4. The Patient Voice


The concept of ‘the patient voice’

The Committee’s inquiry attracted strong interest from patients, their families and advocacy organisations.1 They offered many suggestions for improving Australia’s current regulatory and reimbursement system, covering a wide range of issues, but the most dominant theme to emerge from their evidence was the importance of ‘the patient voice’. No exact definition of this concept was offered to the Committee, but when asked whether the current system ‘recruits it’, Ms Deidre Mackechnie, Executive Officer, Australian Patient Advocacy Alliance (APAA), replied:
I think it recruits a patient voice; I don't think it recruits the patient voice. There is certainly an attempt—and that sounds a weaker word than it probably should—by the department to actually consider the perspective of people who are affected by the healthcare system. But often they are—again, for want of a better term—vanilla patients. They often don't include early on in the process, in the design of what they're actually looking at, patients who are specifically affected by that condition. I think that's a real opportunity to actually improve the system, whereby we can include someone who is directly affected, either as a patient or as a carer or parent, so that they are able to more meaningfully contribute to the process.2
The importance of family and carers noted by Ms Mackechnie was emphasised throughout the inquiry. Many submitters were patients themselves, such as Ms Fiona Mobbs and Ms Patricia Pontynen, who wrote to the Committee as sufferers of Type 1 Narcolepsy and Non Small-Cell Lung Carcinoma, a form of lung cancer, respectively.3 However the Committee heard from many parents and carers of patients who are unable to speak for themselves, typically because they are too young or too affected by their illness. These advocates included Dr Elizabeth Patterson, who appeared before the Committee as the mother of an adult son with Prader-Willi Syndrome, and Ms Michelle and Mr Eliot Jones, who wrote on behalf of their eight year old son Joshua, one of the many boys with Duchenne Muscular Dystrophy whose parents submitted to the inquiry.4
Patients were keen to emphasise how different their voice is from that of other key parties to the regulatory and reimbursement system such as government, sponsor companies and clinicians, and how important that difference makes it for their voice to be included properly in the system.
Mr Mike Wilson, the Chief Executive Officer of JDRF Australia, a Type 1 Diabetes group, told the Committee:
The patient voice is of course one that is important, but it is also under recognised in most of our systems and structures in Australia today. It is not the same as a professional voice or a manufacturer voice, but that is its benefit. … A patient's assessment of risk is not the same as that of a regulator. It should be informed by a doctor, but it is also informed by the ultimate need of the individual. I can assure you a patient's assessment of urgency is very different to that of bodies assessing a line-up of drugs and devices awaiting their attention.5
The Committee heard from the Patient Voice Initiative, which describes itself as ‘a multidisciplinary collaboration which advocates for a greater patient voice in health policy.’6 It submitted that:
…researchers and policy-makers overlook critical issues when striving to improve health outcomes because they lack essential contextual knowledge which patients gain from living with a condition or using a treatment. This includes:
Outcomes that are important to patients
Benefits not documented in traditional evidence, including non-health benefits
Risks and adverse events not documented in traditional evidence, including non-health risks
Knowledge of service variation (especially what really happens as opposed to what is meant to happen), often crucially important for people outside of our capital cities
Knowledge of why some patients cannot access existing drugs and services
Knowledge of unmet needs
Knowledge of wider societal consequences.7
Patients insisted that, far from being confined to any one particular stage of the regulatory and reimbursement process, the patient voice must be included throughout the entire system.8

The patient voice and the Therapeutic Goods Administration

Current patient input into Therapeutic Goods Administration decision-making

Adjunct Professor John Skerritt, Deputy Secretary, Health Products Regulation, Department of Health (Adjunct Prof Skerritt), who leads the Therapeutic Goods Administration (TGA), said of the role of the patient voice in TGA decision-making that ‘I think that is an area we need to do more in.’9 He stated that the most important role patient input can play in the TGA’s decisions is through the inclusion of patient-reported outcomes. These enable the TGA to assess the impact a medicine or device has on patients’ quality of life. According to Adjunct Prof Skerritt such outcomes are often more difficult to measure than more traditional clinical trial outcomes, but this difficulty will be minimised in the future as there is a ‘global trend’ towards including such outcomes.10
Adjunct Prof Skerritt explained that patients have a more direct voice in the TGA’s activities through its advisory committees. These committees consider most new drugs and many new devices as part of their registration processes, and include consumer representatives.11 Since a 2017 reorganisation there are seven such committees, one each for biologicals, chemical scheduling, complementary medicines, medical devices, medicines, medicines scheduling and vaccines.12 Since March 2019, the consumer representatives from the Advisory Committees on Medicines and Medical Devices have served as members of the Department of Health’s Health Technology Assessment (HTA) Consumer Consultative Committee alongside the consumer representatives from the Pharmaceutical Benefits Advisory Committee (PBAC), Medical Services Advisory Committee (MSAC) and Prostheses List Advisory Committee (PLAC).13 Adjunct Prof Skerritt noted that the membership of the TGA’s advisory committees is term-limited, and the TGA conducts call-outs for new members, including engagement with consumer groups.14
A final important role that patients already play in the regulation of therapeutic goods is through adverse event reporting, meaning reporting problems with already approved medicines and devices to the TGA.15 The Department of Health (the Department) explained that in January 2018 the TGA introduced the Black Triangle scheme, which provides information about how patients can report adverse events on product labels, for medicines for which ‘use in the general population is yet to be fully characterised.’16
Adjunct Prof Skerritt told the Committee how he and other TGA officials had met with a group of women who were suffering from a rare cancer linked to TGA-approved breast implants. He explained that:
We were working with them on how we could incorporate their voice, and we have a whole program, known as the medical devices action plan, that gives the patient voice a much a much greater input….What we wanted and what we are achieving…was to make sure of our communications for patients around what to do….How do we shape our communications? You don’t want to write in regulator-speak or bureaucrat-speak. Increasingly, we’re sitting across the table and they’re actually shaping the communications.17

Patient views on the Therapeutic Goods Administration

The Therapeutic Good Administration’s engagement with patients and patient evidence

Some patient groups were complimentary about the TGA. Migraine Australia commented that the ‘the TGA process appears to work well and have a high level of transparency and trust’;18 APAA wrote that ‘our regulatory process (TGA and PBAC) is robust and trustworthy,’ a sentiment echoed by Cystic Fibrosis Australia (CFA);19 and Rare Voices Australia (RVA) said that it ‘would like to acknowledge the strengths of the Therapeutic Goods Administration and Australia’s [HTA] approval processes.’20 Nonetheless all three of these groups, along with many other groups and individual patients, had suggested improvements to how the TGA functions.
Patients were clearly of the view that they need more of a voice in the TGA’s activities. CFA stated that there is ‘no consumer consultation at the TGA stage or prior’, stating that ‘consumers must be part of the registration process.’21 The GUARD Collaborative (GUARD), a coalition of genetic, undiagnosed and rare disease organisations, called for ‘dialogue at a very early stage, on a specific disease, in a multi-stakeholder format including patient representatives, rare disease clinicians, regulators, HTA experts and industry…’ to consider a wide variety of issues, and for ‘patient organisations [to] be supported to create Community Advisory Boards composed of trained patient advocates, per disease or group of diseases, in order to enable a structured, high quality, and transparent dialogue with all stakeholders.’22
APAA commented that ‘there is a lack of inclusion of consumers and consumer organisations at all steps in the HTA process,’ and that ‘there are few patient-specific measures included in evaluation.’ It proposed creating ‘a consultative mechanism to co-design process improvements’ to increase engagement, and ‘inclusion of patient measures…in the process’.23 The Consumers Health Forum of Australia (CHF) stated that ‘working collaboratively with consumers and consumer organisations to access and understand real world data around co-design, disease-specific, patient relevant/patient-reported health outcomes (PROMs) and patient-reported experience measures (PREMs), quality of life and patient preference data, must be included as part of the…regulatory…clinical assessments.’24 The CF Patient Pipeline Interest Group asked that the Pharmaceutical Benefits Scheme (PBS) Medicines Status website, discussed below, be expanded to include TGA information.25

Patient comments on other Therapeutic Goods Administration issues

Use of Overseas Regulators

Beyond the issues of engagement with patients and patient evidence, a number of broad themes emerged from patients’ commentary on the TGA. The most popular of these was the need for the TGA to rely more on the work of overseas regulators, or engage in more collaboration and harmonisation with them. Many submitters kept their comments on this issue to the general proposition that this would increase the speed of Australia’s regulatory process and/or result in more products being registered.26
Allergy and Anaphylaxis Australia (A&AA) submitted that the regulators in question should only be those of ‘countries Australia has trusted relationships with’,27 while other submitters proposed: those regulators already designated Comparable Overseas Regulators (CORs) by the TGA;28 the European Union’s European Medicines Agency (EMA) and the United States (US) Food and Drugs Administration (FDA);29 or just the FDA.30 The Australian Federation of AIDS Organisations (AFAO) proposed that the TGA should make use of assessment reports from CORs for diseases ‘with low prevalence among the general population’.31 MND Australia suggested that programs similar to Project Orbis be developed for rare diseases, while Ms Pontynen supported referring to overseas regulators when adjusting a product’s indication after it has been registered.32

Length of review

Another common refrain – reflected in many of the calls for more use of international regulatory work - was the need for TGA process to occur faster. Fabry Australia highlighted the importance of fast registration for patients with chronic progressive conditions, and suggested that current timeframes are not fast enough.33 Migraine Australia submitted that registration ‘could be faster’, and the CF Pipeline Patient Interest Group made a similar point, noting that TGA registration times are slower than the FDA’s and EMA’s.34

Off-Label use

Several patient groups also raised concerns about the reliance currently placed on off-label use of medicines in treatment of certain conditions. The Leukaemia Foundation noted that there are no definitive statistics on off-label usage – which is one of the problems with such usage from a system-wide perspective - but that it appears to be common in treatment of cancers, especially blood cancers.35 The Foundation proposed a ‘Right to Trial’ program to provide ‘a mechanism for the more regular and systematic use and evaluation of off-label medicines’.36 In a similar vein, CHF suggested consideration of ‘right to trial’, a US concept whereby terminally ill patients are allowed access to therapeutic goods that have completed Phase 1 trials but not yet received regulatory approval.37
Rare Ovarian Cancer expressed concern about how common off-label use is in the treatment of rare cancers, since it means these medicines are not being funded by the Government and so are ‘inaccessible to most patients’.38 RVA argued that there are two other problems with ongoing off-label usage: it relies on a prescriber ‘who has an understanding of the rare condition and the benefits of off-label use’ and it often relies on hospital funding which is by individual application, so there is no ongoing certainty for the patient.39

Post-market surveillance

Varying views were expressed concerning the TGA’s post-market surveillance. Migraine Australia commented that ‘there seems to be a very low rate of reporting of side effects and adverse events to the TGA, and perhaps that reporting process could be made simpler and more consumer friendly.’40
AFAO expressed concern that ‘overregulation and the costs incurred with random and unexpected monitoring can act as a disincentive for manufacturers to enter the Australian market.’41 It urged the TGA to ‘strike a balance between conducting essential post market monitoring and assessments of approved devices and creating an environment that encourages innovation’.42

Miscellaneous patient comments on the Therapeutic Goods Administration

In addition to the general concerns with the TGA just discussed, some patients and patient groups had more varied comments. The CF Pipeline Patient Interest Group recommended that the process be changed to allow ’data to be added during the TGA process,’ thereby potentially allowing for indications to be expanded without requiring further applications to the TGA.43 RVA suggested that ‘all rare disease applications should be routinely flagged as complex and may require additional scoping and stakeholder engagement to address potential challenges and uncertainties,’ a comment that ties into the discussion of patient engagement above.44
There were a number of suggestions that the TGA should copy initiatives of the US FDA, including establishing a Priority Review Voucher system, and producing guidance for industry for developing drugs for the rare disease Eosinophilic Esophagitis (EoE).45 The Priority Review Voucher system involves the FDA rewarding a company that has secured approval of a treatment for certain rare diseases with a priority review voucher that can be used to access priority review for a drug that would not normally be eligible for it.46
Ovarian Cancer Australia (OCA) called for the introduction of a ’fast track short-term approval, with subsequent full review’ process, while the AFAO proposed ‘a priority track for the registration of medicines’.47 AFAO recommended changing the TGA’s regulation of advertising ‘to accommodate health promotion campaigns by non-government organisations’, specifically to enable it to promote HIV testing.48
Lymphoma Australia raised the problem of pharmaceutical companies being unwilling to submit a medicine to the TGA for registration if it will not be reimbursed, even though it may be registered in other countries.49 It recommended that this be dealt with through the creation of ‘a pathway for registration…that can also be clinician/research [sic] or patient-initiated’.50 This issue forms part of the broader question of the regulatory and reimbursement system’s reliance on sponsor companies. Lymphoma Australia commented that many clinicians are unaware of the Special Access Scheme - which allows access to unregistered medicines and recommended that they be educated about such matters.51

The patient voice and the Pharmaceutical Benefits Advisory Committee

Current patient input into Pharmaceutical Benefits Advisory Committee decision-making

In its submissions to the inquiry, the Department stated that the mechanisms through which it engages with patients and stakeholders include:
Stakeholder consultation to facilitate access and engagement of specialist clinicians, patient networks, research bodies, registries and international contacts to enable contribution of rare disease expertise
Inputs to submissions through written submissions, consumer hearings, stakeholder meetings, patient/family interview and organisational surveys, for consideration by the committee.52
The Department outlined the work being done by the recently established HTA Consumer Consultative Committee, made up of the consumer representatives from across the regulatory and reimbursement system, including holding workshops and fora for patient organisations, assisting in the development of the Medicines Status website to allow the public to track medicines as they progress through the listing process, and ‘developing a mentoring pilot program for HTA consumer committee representatives.’53
The Department described the work of its HTA Consumer Evidence and Engagement Unit, established in 2019, ‘to support broader consumer participation strategies.’ This Unit has been involved in the mentoring of the consumer committee representatives, and is ‘exploring ways to enhance the transparency of HTA processes further.’ This includes by considering the methods used by the United Kingdom (UK) National Institute of Health and Care Excellence (NICE), and working on a pilot project for sponsors of PBAC submissions to provide patients with a simple summary of their submission.54 Ms Jo Watson, Deputy Chair, PBAC, summarised the Unit’s work as follows:
That Unit has been able to inform the work of the consumer representatives working within committee processes, as well as to start formally developing better ways that we can structure liaison and opportunities for participation with our external patient representatives, their networks and organisations.55
There are currently two patient representatives on the PBAC, including Ms Watson, and other patients have the opportunity to provide input into the assessment of individual submissions. The Department identified four principal processes through which that input is contributed:
Direct input through consumer comments made to the committees
Invitations to present in person at specific hearings
Representation in expert clinical consultations about specific submission items
Representation and input to formal stakeholder meetings and public consultations.56
In its own submission to the inquiry, the PBAC noted the recent changes that have been made to the system, submitting that:
PBAC initiated changes include measures to increase patient engagement, patient hearings, increase transparency of information that informs PBAC decisions and implementing a process for review of PBAC recommendations which have not resulted in a PBS listing of a medicine.57

Patient views on the Pharmaceutical Benefits Advisory Committee

The Pharmaceutical Benefits Advisory Committee’s engagement with patients and patient evidence

Patients were overwhelmingly of the view that the PBAC needs to be more engaged with them and pay more attention to their views. Painaustralia reflected the views of many patients when it submitted that ‘existing mechanisms for consumer input into PBAC processes [are] limited, and inaccessible to grassroots consumers.’ It gave a recent example when it was consulted by the PBAC through its Deputy Chair on belimumab, a treatment for lupus, on a ‘limited timeframe and quick turnaround’, which it said showed ‘the inadequacy of PBAC’s current mechanisms to seek consumer input.’ It recommended the values developed by Health Technology Assessment International’s Interest Group for Patient and Citizen Involvement in HTA as a ‘useful starting point’ for improvement.58
Similar concerns were shared by Migraine Australia, which stated that ‘it is difficult to engage with a PBAC process when there is insufficient information provided from PBAC’ and ‘bringing doctor and patient bodies in for consultations before a submission is made to PBAC, or very early in the PBAC process, should be required.’ It advocated for such bodies to be enabled to ‘initiate stakeholder meetings and appeal decisions of PBAC’.59
The Save Our Sons Duchenne Foundation argued that their ‘community’ needs to be included in HTA and other processes because the disease is so ‘poorly understood’ and lived experience of it is so valuable. It argued that involvement in HTA would help educate the disease community about how the process works and dispel any ‘myths’ about it.60 Duchenne Australia submitted that ‘there needs to be a clear and transparent pathway to provide patient experience data through the HTA process,’ on matters such as ‘lived experience,’ ‘impact on quality and length of life’ and effect on ‘social and civic participation.’ It said that it was ‘essential to embed consumer participation in the HTA processes to flag potential issues early on.’61
MS Australia suggested that the HTA ‘process remains mysterious to most consumers and, if they were to consider making a submission, [would] have to imagine the impact a new drug might have on their life.’ It advocated for patients and clinicians to be provided with ‘appropriate, clear, accessible publicly available information on HTA processes.’62 The Melanoma and Skin Cancer Advocacy Network (MSCAN) stated that ‘consumers bring a crucial lived experience’ and ‘processes for engagement need to be both meaningful, transparent and have a genuine impact/weighting in the decisions.’ It emphasised the need for feedback to be provided to patients ‘to facilitate continuous improvement in the contributions made.’63
Speaking specifically of rare diseases, RVA argued that ‘it is critical that HTA processes formally embed, capture and promote the voice of people living with rare disease and their families and carers’ to ‘provide much needed narrative and context to the data presented.’ It did note that in its view, within Australia’s system, ‘PBAC is the gold standard in terms of…consumer engagement.’64
CHF submitted that patient involvement improves the ‘legitimacy’ of decision-making. It argued that ‘methods are needed to incorporate data and evidence provided by patients’ into HTA’ and ‘HTA systems need mechanisms to incorporate data and evidence provided by patients.’65 The Haemophilia Foundation Australia (HFA) said that HTA should involve ‘evidence that analyses patients experiences and [uses] patients’ words to explain what an outcome means to them to gain a comprehensive understanding of the actual outcome.’66
Another submitter urged that ‘PBAC submissions should include consumer comments.’67 This view was shared by CFA, which submitted that the Government should ‘insist on consumer consultation and sharing of real-life evidence up front in the process,’ and ‘encourage and incentivise patient organisations to be involved in the process.’68 ITP Australia submitted that hearing and considering the patient voice ‘includes, but is not limited to, working with rare disease organisations and consulting effectively on patient criteria.69

Patient reported outcome measures and patient reported experience measures

Many patient organisations called for the inclusion of Patient Reported Outcome Measures (PROMs) and Patient Reported Experience Measures (PREMs) in the HTA process.70 The absence of such measures was noted by CFA and MS Australia, the latter of which emphasised the need for them to be included in clinical trials.71 GUARD emphasised the importance of PROMs, describing them as ‘essential measurements in rare disease development’, and the need for them to be developed ‘at an early stage of product development.’72 The Spinal Muscular Atrophy Association of Australia (SMA Australia) likewise stated that ‘the consumer is not part of the approval process from the beginning with PROMs or PREMs not part of the HTA submission.’73 RVA submitted that companies should be ‘encouraged’ to include such measures and to show that patients were involved in research design.74 Lymphoma Australia recommended that they be included as part of a ‘post market assessment process.’75

Patients calling for more information – submission summaries

CFA apparently spoke for many patients when it stated of ‘lack of transparency with sponsor submissions. Not enough information is available in the public domain.’ It recommended ‘provid[ing] consumers with more information about the submission.’76 The Juvenile Arthritis Foundation Australia (JAFA) similarly submitted that ‘transparency is essential and could be achieved without compromising commercially sensitive information.’77
One particular idea that sparked patient interest was the possibility of sponsors providing simplified summaries of their submissions to patients to enable them to provide better informed input into the assessment process.78 As mentioned above the Department’s HTA Consumer Evidence and Engagement Unit is already testing a pilot of such scheme. Most of the discussions of this idea before the Committee related it to a similar system already in place in Scotland, and is discussed further in the ‘Overseas Models’ section below.

Patient comments on other Pharmaceutical Benefits Advisory Committee issues

Membership and access to expertise

Many patient groups believe that the PBAC needs to engage more closely with clinical experts in the diseases for which it is evaluating treatments, particularly for rare diseases. Mrs Nicole Millis, Chief Executive Officer, RVA, told the Committee:
Rare disease expertise should be sought and accessible on every approval process. All of our approval processes deal with rare disease HTA. We need earlier and ongoing consumer input into HTA—and when I say 'consumer' I mean patient and clinician.79
Similarly Mrs Annette Burke, Chief Executive Officer, CFA, stated that:
So we need the expertise and we need it around precision medicine. There are incredible things that doctors and scientists are doing around organoids, ohmics [sic] and all of those really technical ways of evaluating drugs for the individual, not these big, mass double-blind placebo trials.80
Ms Sharon Caris, Executive Director, HFA, explained that:
…we strongly advocate for the HTA committees to include specialist clinicians and patients at every step to deliver specialised expertise to underpin decision-making; for example, with rare diseases like haemophilia, we could bring together affected patients, treating clinicians, MSAC [the Medical Services Advisory Committee], the NBA [the National Blood Authority] and the sponsor at the beginning of the process to discuss the submission, share expertise and data, and discuss solutions around access before the process begins.81
The National Aboriginal Community-Controlled Health Organisation (NACCHO) noted that it could ‘not identify any member of the HTA consumer committee or PBAC with a primary expertise in Aboriginal and Torres Strait Islander health.’82 It recommended that the Department ‘enhance Aboriginal and Torres Strait Islander people’s representation across Commonwealth HTA committees and agencies.’83 It recommended the establishment of a separate ‘Aboriginal and Torres Strait Islander medicines advisory committee,’ jointly chaired by NACCHO and the Department, to fulfil roles including reviewing current PBS listings for Aboriginal and Torres Strait Islander people, scoping potential new listings, and advising the Department and HTA committees.84

Length of review and resubmissions

Many patient groups expressed concern with how long Australia’s HTA processes currently take.85 MSACN, for example, stated that ‘access to new medicines and treatments is too slow, and lags in reimbursement are directly impacting on too many Australians’ while Duchenne Australia commented that ‘…the approval pathway is lengthy and remains uncertain as to whether it will be successful.86

International cooperation

As in the case of the TGA, patient groups were enthusiastic proponents of increased collaboration with international HTA bodies and harmonisation of HTA processes, with several making general recommendations along those lines.87 CHF focused on what Australia can learn about HTA methods from overseas rather than direct collaboration, and the Alpha-1 Organisation Australia (A1OA) likewise suggested the Government should review international pricing strategies for low volume drugs, such as New Zealand’s bundling approach.88
A&AA called for ‘improved utilisation’ of the COR pathway and ACCESS Consortium, seemingly for HTA purposes, while Lymphoma Australia asked that there be in similar progress in this area for HTA as there has recently been by the TGA with initiatives such as Project Orbis.89 CFA and SMA Australia made arguably the most radical proposal, both suggesting that Australia should jointly negotiate medicine reimbursement with other similar countries such as the UK, Canada and New Zealand.90

Interim access

Many patient groups were strong supporters of some form of ‘interim access’ model, meaning patients would get access to medicines before the final negotiation between sponsor and Government is complete. The CF Pipeline Patient Interest Group encouraged the Government to ‘consider the German model…when long negotiations are likely.’91 The ‘German model’ is discussed in Chapter 6. This was likewise supported by OCA and the APAA, particularly for ‘life-saving drugs,’ and by CFA.92 SMA Australia advocated a similar course, namely ‘immediate access to life-saving drugs following TGA approval.’93 Rare Cancers Australia proposed ‘granting access to treatments once they are assessed as effective and then using real world patient experience to assess pricing after the fact.’94

Real world evidence

Closely linked to the ideas of interim access and patient evidence such as PROMs and PREMs , many patient groups agreed that there needs to be more use made of so-called ‘real world evidence’ (RWE) in HTA.95 CHF supported this proposition and stated that RWE:
…includes electronic medical/health records, registries, patient reported data inclusive of quality-of-life data, qualitative research, use of surrogate outcomes, deciding which outcomes are to be included in an assessment which needs patient and clinician input, costing, monitoring over time, and analysis of uncertainties.96
SMA Australia pointed out that RWE has the advantage over clinical trials that it draws from a broad population, not a narrow one, and does not ‘result in disparities in access for those not enrolled.’97
RVA commented that ‘currently, there is no process in Australia for translating and utilising valuable real world data as it emerges, yet this remains a potentially invaluable strategy to facilitate timely regulatory approval and to enable equitable therapeutic access.’98The CF Pipeline Patient Interest Group recommended a ‘broadening of the range of accepted evidence to include more universal and appropriate use of [RWE]’ and the creation of guidelines to recognise its value;99 it proposed gathering such evidence through data registries, and the ‘German model’, discussed above.100 GUARD noted the importance of ‘continuous generation of RWE post approval to reduce uncertainties.’101

The comparator requirement

Some patient groups called for reform to the PBAC’s comparator requirements. Migraine Australia submitted that a no comparator should be used for new medicines ‘where there is no real comparator drug’ instead of the current procedure of using the nearest alternative, pointing to what it regards as the inappropriate use of onabotulinum toxin A (Botox) as the comparator for a new class of migraine treatments known as Calcitonin Gene Related Peptides (CGRPs).102 The CF Patient Pipeline Interest Group likewise noted that many of the cystic fibrosis treatments in development are ‘highly innovative genetic therapies’, and submitted that consequently ‘the type and use of comparators must be reasonable for the specific mutation, not the entire patient population.’103

Submissions without a sponsor

A number of submitters drew the Committee’s attention to the problem of how submissions can be facilitated when there is no company willing to sponsor them.
CFA and the APAA both submitted that ‘pathways’ should be established where ‘benefit and patient need can be demonstrated.’104 The PFIC Network asked that the rare disease organisations be enabled to work with the Department’s HTA Consumer Evidence and Engagement Unit for ‘medicines with demonstrated benefit for a rare disease,’ a request that was echoed by WMozzies, and by the Metabolic Dietary Diseases Association and Prader-Willi Research Foundation Australia (PWRFA) which both cited Action of the National Strategic Action Plan for Rare Diseases (Action Plan).105
Migraine Australia brought up this issue in the specific context of repurposing. It proposed a ‘quick and affordable…departmental process’ for listed medicines to have their listing altered ‘when requested by third parties such as patient bodies.’106 RVA called for ‘a viable pathway for consumers to make an application’, particularly for repurposed medicines.107
The Australian and New Zealand Headache Society submitted that it has ‘recognised other areas of unmet need in headache over time but has been unable to advocate at any significant level for these changes, since the only avenue is to fund a major submission to PBAC.’ It recommended the creation of ‘an alternative pathway to PBAC consideration of such agents; the capacity for professional bodies such as ours to make such submissions would be an option.’108
Similarly, the Australian and New Zealand Children’s Haematology/Oncology Group submitted that:
We would also support the development of a streamlined system to allow physician-led applications for registration and reimbursement for rare indications in cases where pharmaceutical companies are not inclined to invest in the registration process.109
A doctor who requested name withheld status, called for the establishment of pathways for ‘timely widening of PBS funding of therapies with repurposed use,’ arguing that ‘these pathways should not be dependent on initiation by drug companies. This has the advantage of removing commercial interests.’110
The Macquarie University Centre for the Health Economy (MUCHE) suggested the introduction of a ‘contracted addressment process for listing new orphan and off-patent drugs on the PBS,’ which could be modelled on ‘the MSAC contracted assessment process whereby the Department organises, coordinates and covers the costs associated with developing and preparing the necessary MSAC documents for consideration.’111

Access for Aboriginal and Torres Strait Islander Australians

In addition to its comments on the PBAC’s membership and its advisory committee proposal discussed above, NACCHO suggested the creation of ‘a streamlined pathway to incentivise sponsors to make submissions to PBAC for Aboriginal and Torres Strait Islander populations’ and an ‘update of PBAC guidelines to emphasise the needs and priorities of Aboriginal and Torres Strait Islander populations.’112 It argued for these proposals in part because of the stark gap in expenditure per capita between Aboriginal and Torres Strait Islander Australians and the rest of the population, which was found to be $537 per person for the former compared with $891 per person for the latter in 2020.113

Broader concept of value

Another vital issue for patients was the question of how medicines, particularly for rare diseases, are valued. Narcolepsy Australia recommended that ‘quality of life assessment considerations be permitted in applications.’114 ITP Australia recommended that there be ‘a restructure of the [economic assessment] of treatments to include not just the immediate costs…but the lifelong economics’ especially for rare diseases.115 SMA Australia suggested ‘novel value-based pricing strategies incorporating broad HTA to maximise benefits…could be a way of future access.’116 The Patient Voice Initiative highlighted the importance of including ’benefits not documented in traditional evidence, including non-health benefits,’ as well as ‘non-health risks.’117
Migraine Australia insisted that for ‘new drugs without comparator’ the impact of a potential listing on the health budget should not be considered, but rather the impact on the budget as a whole, thus including factors such as increased tax revenue through patients returning to the workforce. It described this as a ‘holistic cost-benefit analysis.’118 A similar argument was made by HFA, which suggested that the HTA process needs to ‘consider the cost benefits to the whole of government of the whole of life benefits that our community experience,’ not just the impacts on health budgets. Its examples of ‘indirect benefits’ included ‘children being able to attend school regularly’ and relatives being able to spend less time caring for patients and more time working.119
CHF submitted that ‘the current PBAC assessment of medicines…inadequately considers the evaluation of social and economic impacts of a particular intervention….Economic evaluation of an intervention must be conducted within a societal perspective and [with a] broader context in mind.’120 JAFA submitted that ‘…ultimately most decisions are based on cost. While this remains in place, beneficial therapies either are not funded through the PBS or take an unnecessarily long time to be listed.’ It recommended that a formal review of the PBS funding model be undertaken to try to develop a better model.121
GUARD argued that the current approach to ‘setting a price according to…the perceived or estimated value of a medicine does not work, in particular for rare diseases.’ It suggested that more work is needed on correctly valuing medicines according to the outcomes they produce, and raised the possibility of paying lower prices in return for faster reimbursement of medicines.122 The PFIC Network submitted that ‘rare disease therapies [are] unable to meet the criteria for subsidy under current PBAC …pathways as they were designed for the evaluation of common disease therapies.’123
The A1OA argued that subsidisation of new drugs or technologies should be prioritised ‘where a genetic disorder has never had any subsidised treatment.’124 WMozzies called for ‘equity’ to be added to the principles underpinning Australia’s HTA processes.125

The post-Pharmaceutical Benefits Advisory Committee process and price negotiations

Migraine Australia raised the issue of how post-PBAC pricing negotiations are conducted, suggesting that budgetary concerns are too prominent within the PBAC’s decision making and that the Pharmaceutical Benefits Pricing Authority should be re-established to provide independent oversight of the pricing negotiation process.126 SCN2A Australia requested the Government ‘reduce the delay in getting approved medications available to patients’, although it used medicinal cannabis as an example of this, so it is unclear to what extent this refers to price.127
A&AA asked for ‘a more efficient registration and PBS listing process without jeopardising consumer safety,’ giving the example of the atopic eczema treatment dupilumab, which had been recommended by PBAC seven months before the date of submission but had still not been listed.128 CHF stated that ‘improved, streamlined pricing negotiation processes are needed to enable greater transparency of funding arrangements across the health system.’129 Several submitters raised the issue of the lack of any time limit on price negotiations between the Government and sponsors;130 MS Australia, for example, argued that imposing such a limit ‘would provide some certainty regarding access to treatment and managing consumers’ and clinicians’ expectations.131

Listing update and review process

PWRFA touched on another common theme in recommending ‘that there is a process for timely review and updating of PBS listings to ensure equitable and evidenced-based [sic] access to therapies.’132 ITP Australia suggested that such a process ‘utilise evidence from reputable international agencies’, while Lymphoma Australia asked that it include ‘rigorous patient measures’.133 The CF Patient Pipeline Interest Group recommended that the Government ‘allow more flexible models such as “pipeline agreements” to be considered with a particular sponsor, where new medications are provided and the listing can be expanded to include additional patients without additional PBAC meetings’.134 Migraine Australia advocated for ‘automatically listing alternative preparations and pack sizes’.135

The patient voice and the Medical Benefits Advisory Committee

The Department informed the Committee that ‘from 1 July 2021, revised MSAC consultation processes took effect to improve opportunities for stakeholder input, provide procedural fairness and improve transparency.’136

Patient views on Medical Services Advisory Committee

Medical Services Advisory Committee’s engagement with patients and patient evidence

Many patients commented on HTA in general rather than one of the specific HTA committees in particular. Nonetheless, the MSAC is an important body for many patients, and some specifically addressed it in their submissions.
GUARD commented that ‘we welcome the review of MSAC Guidelines and the proposed move to include personal utility as part of the decision-making but are concerned that this will further add time and qualitative measures will not be equal in weight to quantitative measures.’137 The Leukaemia Foundation supported Action 2.2.3.b of the National Strategic Action Plan for Blood Cancers, under which the working group the Action Plan establishes should work with the Government and other stakeholders to:
Continue important reforms to MSAC processes for MBS [Medicare Benefits Schedule] listings, focusing on greater transparency and the rapid adoption of diagnostics…This should include enhancing consumer understanding of and engagement with the MBS listing process, drawing experience from improved consumer engagement in PBS processes.138
RVA likewise submitted that ‘the MSAC certainly lacks transparency around timelines and formal consumer engagement’ and that ‘it is vital that clear timeframes to reach and publish outcomes, similar to the PBAC’s timeframes, are implemented and made public.’ It reported that some patient organisations have ‘a higher level of confidence’ in the PBAC than the MSAC due to these differences in transparency.139
Lymphoma Australia supported the publication of ‘a more comprehensive summary of submissions’ to the MSAC along with the release of the agenda for each meeting.140 HFA called for the involvement of patient organisations from the beginning of the MSAC process, to assist in ‘identifying appropriate evaluation tools, clinical or quality of life outcomes or benchmarks.’ It highlighted the need for clinicians with experience of the particular condition in question to be involved in the MSAC’s assessment, given how PROMs can vary between different conditions. It explained that:
…a culture of stoicism and low expectations of treatment benefits has meant that people with haemophilia often have higher mental, psychological and social scores for health-related quality of life than people with similar chronic health conditions, such as arthritis, while their physical functioning scores are actually very low.141

Patient comments on other Medical Services Advisory Committee issues

Real world evidence and international cooperation

There were numerous calls for more use of RWE and international cooperation in HTA generally. Lymphoma Australia addressed the MSAC specifically, suggesting it needs to learn from the example of the TGA and how it has increased its international cooperation through initiatives like Project Orbis. In particular it wanted to see the MSAC include ‘real-world data that is timely and aligned with approvals from other countries’ in its decision-making.142

Broader concept of value

GUARD welcomed the inclusion of ‘personal utility’ as a consideration in the MSAC’s decision-making, although it expressed concern as to how this was to be done. The PFIC Network submitted that the criteria the MSAC uses for its decisions were designed for therapies for common diseases, making it more difficult for rare diseases therapies to be approved for subsidy. In response to this issue it proposed ‘broadening’ the description and understanding of the principles underpinning Australian HTA processes’ and increasing the availability of rare disease expertise in those processes.143

Miscellaneous patient comments on Medical Services Advisory Committee

Patient organisations raised various other concerns about the MSAC. RVA noted that ‘while the MSAC can use expedited processes, these processes can only be considered for resubmissions.’144 HFA asked that the PBS Medicines Status website be expanded to cover technologies being reviewed by the MSAC.145 Finally, the AFAO recommended that the Government should establish a ‘priority track’ through the TGA and MSAC for therapies ‘needed in the national interest for the protection of the public from health threats.’146

The patient voice and the Prostheses List

The T1DHub was the only patient group to comment on the Prostheses List Advisory Committee (PLAC). It recommended:
Implement mechanisms for the patient voice to be heard in relation to the Prostheses List approval process. Currently, there is no process to ensure the patient voice is heard and when it is, it may not be the right patient at PLAC level. Seeking submissions or statements from health consumers with lived experience could assist greatly in understanding the conditions and lived experience health outcomes for patients.147
It proposed reducing PLAC application times by making more use of international approvals. 148

Other submitters’ views on the patient voice

Many non-patient submitters expressed views on the system’s engagement with patients, whether generally or through a specific part of it. Miss Jessica Pace, a pharmacist completing a PhD on regulatory and funding mechanisms, said that her research shows that clinicians and patients largely believe the system uses ‘fair procedures’, including ‘meaningful opportunities for stakeholder participation,’ although transparency could be improved.149
BioMarin Pharmaceutical Australia advocated for compulsory consumer hearings, together with ‘appropriate processes for local experience from expert clinicians and patients to be considered in the evaluation process.150 LEO Pharma suggested that ‘patient views are currently undervalued as part of the HTA assessment process’, and that ‘improving the current mechanism to allow for better patient contribution will improve decision-making.’151
Merck Healthcare supported a ‘stronger voice’ for patients in the system as a whole.152 Better Access Australia raised a number of questions about how the current system engages with patients, particularly ‘grassroots patients groups and individual consumers. These questions reflected Better Access’ concerns that the system is much more engaged with industry than with patients, and that patients have to reach out to government rather than vice versa; for example patients who provide feedback on a submission are not notified when a decision is reached on that submission.153
The Australian Cardiovascular Alliance recommended the introduction of ‘a consultative process between all HTA committees and researchers, clinicians, patients and patient groups.’154 Merck Sharp & Dohme Australia submitted that ‘a patient-centred approach [to HTA] is required’ because patients bring a’ unique perspective on disease and the value of potential new treatments.’155
The Australian Healthcare and Hospitals Association submitted that ‘it is difficult to balance the type of data and evidence required for current HTAs, which are largely based on clinical outcomes, with patient outcomes or experiences’ and that various ‘data limitations’ exist for patient outcomes. It proposed that the system needs ‘to ensure that patient outcomes and experiences are measured and included in datasets through standardised systems or collections.’156 This recommendation was echoed by Stryker South Pacific, which suggested that funding be adapted ‘to enable providers to focus on outcomes that matter to patients as well as cost efficiencies.’157
Medicines Australia expressed concern that patients ‘with less common conditions’ who do not have access to a patient advocacy group may struggle to contribute to HTA processes. It recommended ‘expanded stakeholder involvement in decision-making, before, during and after HTA consideration.’ It supported strengthening the patient voice through improving patient input processes’ and ‘consistent inclusion of PROMs’.158 It noted that patient involvement in HTA is legislated in Germany, Italy and Taiwan, and suggested that ‘there is an argument’ for legislating it in Australia.159
ViiV Healthcare Australia (Viiv) submitted that ‘stakeholder input into PBAC submissions should be encouraged and valued as meaningful evidence leading to better informed decisions,’ and emphasised that ‘the current process is not consistent across submissions.’ It noted the legislated requirement of patient input in the aforementioned countries, and the practice in the UK and Canada of identifying interested patient groups and inviting them to make submissions. It praised the Canadian practice of publishing ‘the patient document’ online in preference to the PBAC selection of ‘a sample of patient feedback.’160
The Medical Technology Association of Australia claimed that ‘evaluation processes do not sufficiently account for patient input and preference,’ with the option for sponsors to arrange for patient input for applications to the MSAC but uncertainty about how it is used by in assessments, and indeed whether it is used at all. It recommended that ‘the Department should hold an open workshop on the incorporation of patient input and preference into MSAC evaluations with a commitment to implement aligned recommendations.’161 It argued that similar issues apply to the PLAC, and that while it includes patient representation the representatives often do not have specific expertise in the condition to which a particular application relates. It advocated for patient input to the PLAC to be considered in its proposed workshop.162
Commenting on the draft MSAC guidelines that were available at the time it made its submission, Edwards Lifesciences praised the proposals for ‘looking at outcomes that are important to patients (and sometimes family or carers), and the provision of evidence to support the patient relevance of the chosen outcome.’ It supported the proposed inclusion of ‘quantitative patient preference data’ in applications. It recommended that Taiwan be looked to as a model for patient engagement in HTA, that the Department’s HTA Consumer Evidence and Engagement Unit be better resourced and that further clarification be provided about how the MSAC will evaluate patient evidence and what it expects from sponsors in this regard.163
PRISM (Psychedelic Research in Science and Medicine) called for ‘improved mechanisms for consumer and stakeholder involvement and engagement in the assessment process for treatments involving psychedelic compounds.’164 The Australian Antimicrobial Resistance Network recommended the ‘leveraging’ of the knowledge of patients, along with other research stakeholders, in the development of a better response to the problem of antimicrobial resistance.165

Overseas models

The National Institute for Health and Care Excellence

There was considerable interest throughout the inquiry in the approach of the UK’s NICE, which the Macquarie University Centre for the Health Economy submitted ‘is often considered best-practice in terms of HTA.’166 A major focus of that interest was its approach to patient engagement, although other aspects of its operations are discussed in later chapters. Ms Mackechnie of APAA stated that in her view England and Wales (that is, NICE) have the best overall approach to patient engagement, although it is deficient in not providing submission summaries to patients or feedback on their contributions. She described NICE as being ‘very proactive in terms of reaching out to patient organisations and mentoring programs.’167 Ms Simone Leyden, Chief Executive Officer and Co-Founder, NeuroEndocrine Cancer Australia (NECA), likewise praised NICE’s approach to educating patients about HTA.168
Ms Leyden told the Committee:
When a drug or a submission comes up, [NICE] consult the patient organisations that it will affect and they bring them in for a consultation workshop with regard to the submission. They get to see the submission, they get to look at the submission and they get to analyse it before it's even put up for reimbursement….it's something that we should definitely have here.169
The Victorian Comprehensive Cancer Centre indicated its support for such a model.170 Painaustralia likewise singled out NICE for its ‘scoping and consultation workshops,’ as well as patient representation on its committees.171 Viiv Healthcare described its scoping process as ‘one option to improve the current system,’ whereby a scoping document is developed with the input of clinicians and patient groups to determine patient population, place in clinical practice and most appropriate comparator for the therapy.’172
NICE provided evidence to the Committee about how it operates. On the topic of patient engagement and involvement Mr Meindert Boysen, Deputy Chief Executive Officer and Director of the Centre for Health Technology Evaluation, explained to the Committee that:
It starts when we scope a technology evaluation, so we set the question for the work. That's where patients are involved. When we seek submissions not only are we seeking submissions from the company, but we get them from patients, from patient organisations and from clinicians. When our committees meet there will always be patient experts invited to the meeting to give their feedback, usually on what is currently used within the NHS, so not specifically on the new technology. We have lay members on our committees. We have at least two or three lay members that are part of the committee decision-making. They're standing committee members.
Then when the guidance comes out consultation is a public consultation, so the public patients in a broader sense can respond. And there's a chance to challenge the recommendations at the end when we hold the appeal, so that, I guess, across the board patient organisations are involved. I should also say that patient organisations are very much part of our methods and process development work. When we think about new ways of working—and we're currently in the midst of one of those processes—we very much involve patients in the thinking. They're very active as a group. Also, in one of our recent proposals we have asked our manufacturers to provide a specific, patient-focussed summary of their submission, so that the engagement of those patient experts with the evidence that our committee sees is better managed.173


Many submitters highlighted the Scottish system as having a mechanism for providing submission summaries to patients. Ms Mackechnie of APAA told the Committee ‘we believe that a detailed summary template could be co-developed with patients [in Australia], much as they have done in Scotland.’174 She said that so far as she is aware the Scottish system is the only one currently providing such summaries.175
Ms Monica Ferrie, Founder, GUARD, was positive about Scotland’s approach, stating that ‘Scotland does some really terrific things.’ She explained:
So things like the Scottish model of 'We all do things the same' allow groups like RVA and GUARD Collaborative Australia, my organisation, is to understand the process really well for every condition and then be able to assist: 'This is the way that you would go about answering question 1. Let's have a conversation about that, rather than you go away, you do the research, you do all the work yourself and you fill out the form and we'll write a letter to support your submission.'176
In its Guide for Patient Group Partners, Scotland’s HTA body the Scottish Medicines Consortium (SMC) explains to patients that:
Most submitting pharmaceutical companies provide us with a completed Summary Information for Submitting Patient Groups Form, which we can email to you. This provides background information about the medicine and the indication, which can help inform your submission.177
The Summary Information for Submitting Patient Groups Form template is available online for download.178
As mentioned above, Australia has piloted a scheme for providing submission summaries to patients. Mr Neil MacGregor, Managing Director, Australia-New Zealand, Bristol Myers Squibb, told the Committee:
We partnered recently with the Department of Health and PBAC in a pilot to enhance consumer engagement through the PBAC decision-making processes. The scope of the pilot saw BMS in concert with the Department of Health develop plain-language executive summaries specific to two of our recent PBAC submissions. These documents were then provided to the relevant patient groups for their review prior to their own submissions to the PBAC. We believe that this pilot initiative benefited all stakeholders and, importantly, added important patient context for that PBAC consideration.179


Some patient groups praised Canada’s approach to patient engagement, specifically its provision of feedback to patient groups who have commented on a HTA submission. Ms Mackechnie, APAA, for example, gave evidence that ‘providing feedback on the [patient] submission in terms of what worked, what didn't work and how it could be improved for next time is only done by Canada.’180 Ms Leyden, NECA, likewise told the Committee that ‘the way [Canada and Scotland] involve consumers and upskill consumers and train them in what the HTA system is about is what we should be replicating here.’181

Future government engagement with the patient voice

The Department and its staff were keen to emphasise the progress that has been made in engaging with patients in recent years, although they readily accepted that more work is required. Ms Adriana Platona, First Assistant Secretary, Technology Assessment and Access, Department of Health, who has overall responsibility for the Department’s HTA activities, told the Committee that ‘the department has been progressively improving the systematic consumer engagement relating to health technology assessment processes, and that will continue.’ She noted that the Department is creating ‘a new consultation platform’ for HTA online.182
Ms Platona commented on the discussion of overseas systems, particularly NICE and the SMC, and was keen to emphasise that ‘NICE does not do everything’ and does not have all the responsibilities the Department has, such as price negotiation and purchasing.183 On the issue of supporting submissions without a sponsor, she explained:
The reality is that it needs a supplier because, in the end, the agreement to supply the product on the PBS has to be with somebody who has ownership of the product. All the other steps about doing the evidence gathering and preparation of the submissions and waiving fees and charges are all possible with government decision and additional resources. But, to have a product on the PBS, it needs a sponsor.184
The TGA’s Adjunct Prof Skerritt noted that the increased publicity the TGA has received due to the COVID-19 pandemic ‘brings the expectation that we stand up a lot more education and communication about medicines and products that [patients] use.’185 On the issue of patient evidence he commented that ‘what we are moving towards—and this is part of this work we're doing to look at real-world evidence—is to ensure that consumer patient reported outcomes are reflected more extensively.’186
The PBAC noted the ‘need to ensure that further expansion of [its patient engagement] initiatives is adequately resourced.’ It expressed a willingness to trial allowing patient representatives to observe some committee deliberations.187 It submitted that:
A relatively simple matter that requires industry agreement is to inform clinician and patient groups early in the submission process of the specific indications for which reimbursement is being sought. This includes the clinical claim, intended populations, and details on proposed prescribing and clinician access requirements that the sponsor is proposing to PBAC for consideration.188
The Department reported that the pilot on providing PBAC submission summaries to patients ‘is due for evaluation in the final quarter of 2021.’189
The PBAC’s Deputy Chair Ms Watson told the Committee:
We've talked with several patient groups about what some of the potential benefits would be of being able to come in and provide comment earlier on in the cycle—for PBAC, particularly, at the time of submission or at the time of the subcommittee consideration—and have more of a path, if you like, in the cycles along the way. I think that's something that speaks to the need not only for more resourcing internally with the department and our consumer unit but also to have collaboration with the sponsors about that.190
The MSAC did not itself provide any evidence to the Committee, but the Department told the Committee that ‘the Government is committed to continuing to improve MSAC processes, including in respect of stakeholder input, communication and transparency.’ It noted that ‘from 1 July 2021, revised MSAC consultation processes took effect to improve stakeholder input, provide procedural fairness and improve transparency.‘191
As noted in Chapter 2, on 7 September 2021 the Minister announced the signing of five year Strategic Agreements with Medicines Australia and the Generic and Biosimilar Medicines Association. These agreements include ‘the co-design and implementation of an Enhanced Consumer Engagement Process to better capture the patient voice early in the medicines assessment process,’ as well as a comprehensive review of HTA for medicines in general.192

Pharmaceutical Benefits Advisory Committee response to other issues

In response to the concerns discussed above regarding the PBAC’s access to expertise, Professor Andrew Wilson, Chair, PBAC, told the Committee:
In the paper that we've tabled there is an item which is sort of relevant to this, 2.2.6, where I've said:
The PBAC is interested in exploring the mechanisms that might provide greater flexibility in committee membership without increasing what is already a large committee. This might include cross membership with MSAC to facilitate sharing of expertise especially for consideration of co-dependent submissions.
But it may also include situations where we might want to bring in specific experts in relation to it. Having said that, we spend a fair amount of time between sessions meeting with clinical groups and hearing submissions from them. For example, in relation to the new medicines for spinal muscular atrophy, we have probably had close to 10 meetings with experts in that field over the past 12 months. We do also extensively consult outside, where required, in relation to not just rare diseases but also other diseases.193
On the issue of submissions lacking a commercial sponsor the PBAC submitted:
The PBAC notes that while PBAC submissions may be made by other parties (e.g., clinical or patient groups) this is challenging given the PBAC requirements particularly without company sponsor engagement.
The PBAC sees benefit in an alternate mechanism to initiate submissions where there is an unmet clinical need and a potentially useful medicine.
Such an alternative pathway may include alternative sourcing arrangements (e.g., calls for submissions for specific medicines) and would require resourcing a capacity to support the preparation of submissions.194

Committee Comment

The Committee is grateful for the time and effort patients, carers and advocacy organisations put into providing evidence to the inquiry. It appears to the Committee that there is a growing understanding among government, industry and others of the importance of the patient voice. It commends the recent efforts of the Department to pay more attention to the views and experiences of patients in its decision-making, and the ongoing work of the patient representatives on the Department’s various committees to make sure views and experiences are counted.
The Committee is adamant that there is a need for patients to participate in the HTA process at an earlier stage, and to be equipped with more information with which to do so. The Committee appreciates that every HTA system is different, and that submissions for reimbursement contain commercially sensitive information which sponsor companies reasonably want to protect. However, the Committee strongly believes that patients should be involved in the process earlier and should be provided with plain English submission summaries. The Committee encourages the Department to give serious consideration to establishing the patient voice in a similar way to that developed in the UK with NICE. The Committee urges the Department to make these patient voice reforms in conjunction with the review of the HTA system that was recently flagged to begin in July 2022 in the Strategic Agreement 2022-27 between the Government and Medicines Australia.
The Committee considers that it is particularly important that Aboriginal and Torres Strait Islander people are represented on the PBAC and MSAC bodies. While the Committee is greatly concerned with the disparity in access to PBS medicines for Aboriginal and Torres Strait Islander people it does not consider that a separate access pathway is the answer to this problem. Instead the Committee believes that it should be addressed through improvements to patient engagement in the HTA processes. In addition, the review of the HTA system should focus on the assessment of diseases in small patient populations and address equity issues.
The Committee encourages more formal engagement with clinicians during the HTA processes, as the clinicians will bring with them the patient experience using the medicine or treatment. The Committee sees merit in the consideration of cross-membership for certain applications between the PBAC and MSAC and appointing temporary and ad hoc members to either body. Enhancing clinical engagement should be considered by the independent HTA system review in July 2022.
Patient feedback on their contributions to the HTA processes should be developed. This will improve their contributions over time and will assist in developing the patient groups understanding of the HTA system. The Committee considers that the Department should provide a tracking system online for patients to see what progress has been made within the HTA system.
A final difficult issue to emerge from the patient evidence was the problem of how medicines and technologies can be reimbursed when there is no company willing to sponsor them. The Committee notes the Department’s evidence that a company is ultimately required to supply the medicine or therapy, and accepts that if the relevant company is resistant to its product being sold in Australia there is little the Government can do. However, often these will be commercial decisions influenced by market size. Alternative pathways and incentives may overcome barriers relating to what could clearly be a market failure due to the limited size of a potential patient cohort in Australia. The Committee believes the Australia Government should establish a fund to support applications by patients, clinicians and others, in the absence of a sponsor company, but that support should be strictly limited to cases of genuine need, to prevent pharmaceutical and medical technology companies gaming the system to reduce their expenses. The fund should be annually capped with clear eligibility rules. Most instances will be for rare disease medicines and technologies.

  • 1
    The terms ‘patient’ and ‘consumer’ were both used throughout the inquiry, apparently with the same meaning, and indeed some submitters used both interchangeably: Lymphoma Australia, Submission 143, p. [4]; Consumers Health Forum of Australia (CHF), Submission 205, p. 9. The term ‘patient’ is preferred in this report, but references to ‘consumers’ should be read as having the same meaning.
  • 2
    Committee Hansard, Melbourne, 22 April 2021, pages 3-4.
  • 3
    Ms Fiona Mobbs, Submission 38, p. [2]; Ms Patricia Pontynen, Submission 60, p. 3.
  • 4
    Prader-Willi Research Foundation Australia (PWRFA), Committee Hansard, Melbourne, 22 April 2021, pages 10-11; Ms Michelle and Mr Eliot Jones, Submission 132.
  • 5
    Committee Hansard, Sydney, 11 March 2021, p. 23.
  • 6
    Patient Voice Initiative, Submission 71, p. 1.
  • 7
    Patient Voice Initiative, Submission 71, p. 2.
  • 8
    Name withheld, Submission 22, p. [2]. Queensland Genomics Community Advisory Group, Submission 44, p.2; GUARD Collaborative (GUARD), Submission 46, p. 2; MND Australia, Submission 64, pages 7-8; XLH Australia, Submission 81,p. [1]; Rare Voices Australia (RVA), Submission 86, p. 4; Metabolic Dietary Disorders Association (MDDA), Submission 109, p. [7]; PWRFA, Submission 110, p. [4]; Lymphoma Australia, Submission 143, p. [1]; Juvenile Arthritis Foundation Australia (JAFA), Submission 154, p. [3]; FSHD Global Research Foundation, Submission 200, p. 5.
  • 9
    Committee Hansard, Canberra, 18 June 2021, p. 28.
  • 10
    Committee Hansard, Canberra, 18 June 2021, p. 29.
  • 11
    Committee Hansard, Canberra, 18 June 2021, p. 28.
  • 12
    Therapeutic Goods Administration (TGA), TGA Statutory Advisory Committees, Canberra, July 2021, www.tga.gov.au/tga-statutory-advisory-committees, viewed 12 October 2021.
  • 13
    Department of Health, Submission 15, p. 28.
  • 14
    Committee Hansard, Canberra, 18 June 2021, p. 28.
  • 15
    Department of Health, Submission 15, pages 36-37.
  • 16
    Department of Health, Submission 15, p. 34.
  • 17
    Adjunct Prof Skerritt, Committee Hansard, Canberra, 18 June 2021, p. 29.
  • 18
    Migraine Australia, Submission 24, p. 16.
  • 19
    Australian Patient Advocacy Alliance (APAA), Submission 67, p. [1]; Cystic Fibrosis Australia (CFA), Submission 8, p. [5].
  • 20
    RVA, Submission 86, p. 1.
  • 21
    CFA, Submission 8, p. [2].
  • 22
    GUARD, Submission 46, pages 11-12.
  • 23
    APAA, Submission 67, p. [1].
  • 24
    CHF, Submission 205, p. 7.
  • 25
    CF Pipeline Patient Interest Group, Submission 169, p. 3.
  • 26
    Alpha-1 Organisation Australia (A1OA), Submission 29, p. 5; Sanfilippo Children’s Foundation, Submission 36, p. [2]; JDRF Australia, Submission 52, p.[3]; Mrs Melissa Jose, Submission 54, p. [1]; APAA, Submission 67, pages [3]-[4]; SCN2A Australia, Submission 127, p. [2]; Mr and Ms Jones, Submission 132, p. [5]; Ovarian Cancer Australia (OCA), Submission 135, p. [4]; JAFA, Submission154, p. 4; FSHD Global Research Foundation, Submission 200, p. 5; CHF, Submission 205, pages 10-11.
  • 27
    Allergy and Anaphylaxis Australia (A&AA), Submission 128, p. 6.
  • 28
    Eczema Support Australia, Submission 39, p. 2; National Allergy Strategy, Submission 156, p. [4]. For more on the CORs see above Chapter 3.
  • 29
    Migraine Australia, Submission 24, p. 16; A1OA, Submission 29, p. 5; Spinal Muscular Atrophy Association of Australia (SMA Australia), Submission 37, p. [2]; Narcolepsy Australia, Submission 55, p. 4; Duchenne Australia, Submission 77, p. 3; CF Pipeline Patient Interest Group, Submission 169, p. 3.
  • 30
    Save Our Sons Duchenne Foundation (SOSDF), Submission 33, p. 17.
  • 31
    Australian Federation of AIDS Organisations (AFAO), Submission 196, p. 4.
  • 32
    MND Australia, Submission 64, p. 8; Ms Pontynen, Submission 60, p. 2.
  • 33
    Fabry Australia, Submission 4, p. [2].
  • 34
    Migraine Australia, Submission 24, p. 16.
  • 35
    Leukaemia Foundation, Submission 103, p. [6].
  • 36
    Leukaemia Foundation, Submission 103, p. [9].
  • 37
    CHF, Submission 205, p. 11.
  • 38
    Rare Ovarian Cancer, Submission 167, p. [2].
  • 39
    RVA, Submission 86, p. 12.
  • 40
    Migraine Australia, Submission 24, p.16.
  • 41
    AFAO, Submission 196, p. 5.
  • 42
    AFAO, Submission 196, p. 6.
  • 43
    CF Pipeline Patient Interest Group, Submission 169, p. 1.
  • 44
    RVA, Submission 86, p. 9.
  • 45
    Fragile X Association of Australia (FXAA) Submission 159, p. 2; ausEE Inc., Submission 73, p. 4.
  • 46
    FXAA. Submission 159, p. 2.
  • 47
    OCA, Submission 135, p. 4; AFAO, Submission 196, p. 3.
  • 48
    AFAO, Submission 196, pages 3-4.
  • 49
    Lymphoma Australia, Submission 143, p. [2].
  • 50
    Lymphoma Australia, Submission 143, p. [3].
  • 51
    Lymphoma Australia, Submission 143, p. [3].
  • 52
    Department of Health, Submission 15, p. 38.
  • 53
    Department of Health, Submission 15, pages 38-39.
  • 54
    Department of Health, Submission 15.6, p. [25].
  • 55
    Committee Hansard, Canberra, 24 June 2021, p. 4.
  • 56
    Department of Health, Submission 15.6, pages 25-26.
  • 57
    Department of Health, Submission 15.3, p. 1.
  • 58
    Painaustralia, Submission 56, p. 8. See Health Technology Assessment International, Patient and citizen involvement, Edmonton, undated, htai.org/interest-groups/pcig/, viewed 13 October 2021.
  • 59
    Migraine Australia, Submission 24, pages 17-18.
  • 60
    SOSDF, Submission 33, pages 27-28.
  • 61
    Duchenne Australia, Submission 77, pages 2, 6.
  • 62
    MS Australia, Submission 85, p. 10.
  • 63
    Melanoma and Skin Cancer Advocacy Network (MSCAN), Submission 116, p. 4.
  • 64
    RVA, Submission 86, p. 10.
  • 65
    CHF, Submission 205, p. 9.
  • 66
    Haemophilia Foundation Australia (HFA), Submission 119, p. 1.
  • 67
    Name withheld, Submission 22, p. [2].
  • 68
    CFA, Submission 8, p. [4].
  • 69
    ITP Australia, Submission 139, p. 5.
  • 70
    APAA, Submission 67, p. [2]; MSCAN, Submission 116, p. 4; HFA, Submission 119, p. 1; Lymphoma Australia, Submission 143, p. [5].
  • 71
    CFA, Submission 8, p. [2]; MS Australia, Submission 85, p. 11.
  • 72
    GUARD, Submission 46, p. 9.
  • 73
    SMA Australia, Submission 37, p. [2].
  • 74
    RVA, Submission 86, p. 4.
  • 75
    Lymphoma Australia, Submission 143, p. [5].
  • 76
    CFA, Submission 8, p. [4].
  • 77
    JAFA, Submission 154, p. [3].
  • 78
    Lymphoma Australia, Submission 143, p. [5]; Ms Jane Hill, Chief Executive, OCA, Committee Hansard, Melbourne, 23 April 2021, p. 45.
  • 79
    Committee Hansard, Sydney, 11 March 2021, p. 2.
  • 80
    Committee Hansard, Sydney, 11 March 2021, p. 2.
  • 81
    Committee Hansard, Melbourne, 23 April 2021, p. 49.
  • 82
    National Aboriginal Community-Controlled Health Organisation (NACCHO), Submission 190, p. 4.
  • 83
    NACCHO, Submission 190, p. 5.
  • 84
    NACHHO, Submission 190, p. 6.
  • 85
    For example: Name withheld, Submission 91, p. [1]; The Mito Foundation, Submission 125, p. [2]; CHF, Submission 205, p. 8.
  • 86
    MSCAN, Submission 116, p. 2; Duchenne Australia, Submission 77, p. 6.
  • 87
    Sanfilippo Children’s Foundation, Submission 36, p. 1; GUARD, Submission 46, pages 11-12; JDRF Australia, Submission 52, p. 5; OCA, Submission 135, p. 4; Carers and Patients of Braf V600E Colorectal Cancer, Submission 144, pages [3]–[4]; JAFA, Submission 154, p. [4]; FSHD Global Research Foundation, Submission 200, p. 5.
  • 88
    CHF, Submission 205, p. 8; A1OA, Submission 29, p. 5.
  • 89
    A&AA, Submission 128, p. [5]; Lymphoma Australia, Submission 143, p. 5.
  • 90
    CFA, Submission 8, p. [4]; SMA Australia, Submission 37, p. [2].
  • 91
    CF Pipeline Patient Interest Group, Submission 169, p. 2.
  • 92
    OCA, Submission 135, p. 4; APAA, Submission 67, p. [4]; CFA, Submission 8, p. [4].
  • 93
    SMA Australia, Submission 37, p. [2].
  • 94
    Rare Cancers Australia, Submission 166, p. [4].
  • 95
    CFA, Submission 8, pages [2], [4]; Name withheld, Submission 22, p. [2]; Migraine Australia, Submission 24, p. 21; APAA, Submission 67, p. [2]; MS Australia, Submission 85, p. 11; Lymphoma Australia, Submission 143, p. [5].
  • 96
    CHF, Submission 205, p. 8.
  • 97
    SMA Australia, Submission 37, p. [1].
  • 98
    RVA, Submission 86, p. 9
  • 99
    CF Pipeline Patient Interest Group, Submission 169, pages 2-3.
  • 100
    CF Pipeline Patient Interest Group, Submission 169, pages 1, 3.
  • 101
    GUARD, Submission 46, p. 13.
  • 102
    Migraine Australia, Submission 24, pages 18-20.
  • 103
    CF Pipeline Patient Interest Group, Submission 169, p. 3.
  • 104
    CFA, Submission 8, p. [4]; APAA, Submission 67, p. [3].
  • 105
    PFIC Network, Submission 19, p. [3]; WMozzies, Submission 165, p. 4; MDDA, Submission 109, p. [8]; PWRFA, Submission 110, p. [4].
  • 106
    Migraine Australia, Submission 24, p. 22.
  • 107
    RVA, Submission 86, p. 4.
  • 108
    Australian and New Zealand Headache Society, Submission 115, p. [2].
  • 109
    Australian and New Zealand Children’s Haematology/Oncology Group, Submission 120, p. 7.
  • 110
    Name withheld, Submission 48, p. [2].
  • 111
    Macquarie University Centre for the Health Economy (MUCHE), Submission 62, p. 2.
  • 112
    NACCHO, Submission 190, pages 5-6.
  • 113
    NACCHO, Submission 190, p. 3.
  • 114
    Narcolepsy Australia, Submission 55, p. 6.
  • 115
    ITP Australia, Submission 139, p. 5
  • 116
    SMA Australia, Submission 37, p. [1].
  • 117
    Patient Voice Initiative, Submission 71, p. 2.
  • 118
    Migraine Australia, Submission 24, p. 20.
  • 119
    HFA, Submission 119, pages 2, 7.
  • 120
    CHF, Submission 205, p. 8.
  • 121
    JAFA, Submission 154, p. [4].
  • 122
    GUARD, Submission 46, pages 12-13.
  • 123
    PFIC Network, Submission 19, p. [9].
  • 124
    A1OA, Submission 29, p. 2.
  • 125
    WMozzies, Submission 165, p. 3.
  • 126
    Migraine Australia, Submission 24, pages 21-22.
  • 127
    SCN2A Australia, Submission 127, p. [2].
  • 128
    A&AA, Submission 128, p. [5]. Dupilumab was subsequently listed on the PBS on 1 April 2021: Department of Health, Dupilumab, Canberra, May 2021, www.pbs.gov.au/medicinestatus/document/331.html
    , viewed 1 September 2021.
  • 129
    CHF, Submission 205, p. 9.
  • 130
    Name withheld, Submission 22, p. [2]; APAA, Submission 67, p. [3].
  • 131
    MS Australia, Submission 85, p. 11.
  • 132
    PWRFA, Submission 110, p. [3].
  • 133
    ITP Australia, Submission 139, p. 5; Lymphoma Australia, Submission 143, p. [5].
  • 134
    CF Pipeline Patient Interest Group, Submission 169, p. 3.
  • 135
    Migraine Australia, Submission 24, p. 22.
  • 136
    Department of Health, Submission 15.6, p. [25].
  • 137
    GUARD, Submission 46, p.
  • 138
    Leukaemia Foundation, Submission 103, p. [8].
  • 139
    RVA, Submission 86, pages 10-11.
  • 140
    Lymphoma Australia, Submission 143, p.[5].
  • 141
    HFA, Submission 119, p. 9.
  • 142
    Lymphoma Australia, Submission 143, p. [5].
  • 143
    PFIC Network, Submission 19, p. [2].
  • 144
    RVA, Submission 86, pages 10-11.
  • 145
    HFA, Submission 119, p. 10.
  • 146
    AFAO, Submission 196, p. 3.
  • 147
    T1DHub, Submission 192, p. 3.
  • 148
    T1DHub, Submission 192, p. 3.
  • 149
    Miss Jessica Pace, Submission 40, p. 5.
  • 150
    BioMarin Pharmaceutical Australia, Submission 152, pages 3-4.
  • 151
    LEO Pharma, Submission 202, p. 3.
  • 152
    Merck Healthcare, Submission 34, p. 1.
  • 153
    Better Access Australia, Submission 160, pages 11-12.
  • 154
    Australian Cardiovascular Alliance, Submission 76, p.13.
  • 155
    Merck Sharp & Dohme Australia, Submission 63, Appendix A, p. 4.
  • 156
    Australian Healthcare and Hospitals Association, Submission 68, pages 1-2.
  • 157
    Stryker South Pacific, Submission 28, p. 7.
  • 158
    Medicines Australia, Submission 141, p. 8.
  • 159
    Medicines Australia, Submission 141, p. 38.
  • 160
    Viiv Healthcare Australia (Viiv), Submission 80, pages 7-8.
  • 161
    Medical Technology Association of Australia (MTAA), Submission 148, pages 49-50.
  • 162
    MTAA, Submission 148, pages 53-54.
  • 163
    Edwards Lifesciences, Submission 83, pages 31-32.
  • 164
    PRISM (Psychedelic Research in Science and Medicine), Submission 161, p. [5].
  • 165
    Australian Antimicrobial Resistance Network, Submission 53, p. 2.
  • 166
    MUCHE, Submission 62, p. 8.
  • 167
    Committee Hansard, Melbourne, 22 April 2021, pages 2-3.
  • 168
    Committee Hansard, Melbourne, 23 April 2021, p. 43.
  • 169
    Committee Hansard, Melbourne, 23 April 2021, p. 45.
  • 170
    Professor Grant McArthur, Executive Director, Victorian Comprehensive Cancer Centre, Committee Hansard, Melbourne, 23 April 2021, p. 45.
  • 171
    Ms Carol Bennett, Chief Executive Officer, Painaustralia, Committee Hansard, Canberra, 26 March 2021, p. 17.
  • 172
    Viiv, Submission 80, p. 7
  • 173
    Committee Hansard, Canberra, 7 July 2021, p. 3.
  • 174
    Committee Hansard, Melbourne, 22 April 2021, p. 1.
  • 175
    Committee Hansard, Melbourne, 22 April 2021, p. 2.
  • 176
    Committee Hansard, Melbourne, 22 April 2021, p. 23.
  • 177
    Scottish Medicines Consortium, ‘A guide for patient group partners ‘, Glasgow, March 2017 (revised August 2017), p. 8, https://www.scottishmedicines.org.uk/media/5616/guide-for-patient-group-partners-2017.pdf, viewed 13 October 2021.
  • 178
  • 179
    Committee Hansard, Melbourne, 23 April 2021, pages 6-7
  • 180
    Committee Hansard, Melbourne, 22 April 2021, p. 2.
  • 181
    Committee Hansard, Melbourne, 23 April 2021, p. 43.
  • 182
    Committee Hansard, Canberra, 18 June 2021, p. 18.
  • 183
    Committee Hansard, Canberra, 18 June 2021, p. 21.
  • 184
    Committee Hansard, Canberra, 18 June 2021, p. 30.
  • 185
    Committee Hansard, Canberra, 18 June 2021, p. 26.
  • 186
    Committee Hansard, Canberra, 18 June 2021, p. 29.
  • 187
    Department of Health, Submission 15.3, pages 4-5.
  • 188
    Department of Health, Submission 15.3, p. 4.
  • 189
    Department of Health, Submission 15.6, p. [25].
  • 190
    Committee Hansard, Canberra, 24 June 2021, p. 7.
  • 191
    Department of Health, Submission 15.6, p. [13].
  • 192
    The Hon Greg Hunt MP, Minister for Health and Aged Care, ‘Landmark new medicines agreements to bring significant benefits for Australian patients ‘, Media Release, 7 September 2021.
  • 193
    Committee Hansard, Canberra, 24 June 2021, pages 5-6
  • 194
    Department of Health, Submission 15.3, p. 7.

 |  Contents  |