Alternative access models for new and innovative cancer drugs
As noted in earlier chapters, while submitters consider that the current
system for providing subsidised access to medicines has served Australia well, they
also consider it is in urgent need of review and modernisation. Submitters
noted that the particular plight of cancer patients highlights the need for a
substantive overhaul of the current system to improve flexibility and
timeliness of decisions, while retaining the rigour of the existing process.
Throughout the inquiry, the committee heard a range of evidence
regarding approaches used by other countries that might be considered as models
for alternate approaches to providing access to cancer drugs. Submitters noted
that some countries have introduced specialist mechanisms to facilitate wider
and more-timely access to cancer drugs. For example, the United Kingdom established
the Cancer Drugs Fund (CDF) in 2010 as a temporary fund to meet the costs of
some cancer drugs either rejected by the National Institute for Health and Care
Excellence (NICE) or not yet evaluated by them.
As noted in Chapter 1, Canada established the pan-Canadian Oncology Drug
Review as a cancer specific national drug review process separate from the
Common Drug Review (CDR) in response to concerns that the CDR process for
consideration of oral oncology products was not meeting the needs of patients
Establishment of a specialist cancer drug fund
Many submitters emphasised the need for Australia to implement a specialist
early access program for cancer drugs. However, a number of submitters
recommended the establishment of such a fund as a temporary measure pending
'modernisation' of the Pharmaceutical Benefits Scheme (PBS) and Pharmaceutical Benefits
Advisory Committee (PBAC) processes.
The Private Cancer Physicians of Australia (PCPA) expressed support for
the 'formation of a separate, novel funding mechanisms for high cost drugs
outside the PBS prior to drugs being considered by the PBAC. PCPA proposed that
once a drug receives PBAC approval, such funding would no longer be required
and suggested that cost recovery mechanisms could be applied in the event that
a drug failed to gain approval.
The Tasmanian Government submitted that a cancer drug fund which
supports access to cancer medications that are not available via the PBS, like
that established by the National Health Service (NHS) in England, could provide
an expedient way of enabling access to cancer medications and could be
preferable to the creation of separate administrative arrangements for specific
[T]he creation of new administrative arrangements for some
newly listed medicines, as has been seen with the introduction of Herceptin and
more recently Eculizumab, should be avoided as it created an additional level
of administrative burden and access ambiguity for clinical staff and patients
Medicines Australia (MA) told the committee that the UK and Canada,
having recognised that the value-for-money measure of cost-effectiveness does
not deliver access to cancer medicines in line with community or political
expectations, had established interim access measures while continuing the
search for a better system. Mr Timothy James told the committee:
I think it is fair to say, without reflecting too much on the
public policy decisions of other governments, that in both the UK and Canada
they saw that a one-size-fits-all approach did not work and that, indeed, that approach
was failing cancer sufferers. Hence, they determined that there should be a
dedicated, specifically focused, resourced part of their system and a
particular capacity to enhance access to cancer medicines. We believe that sort
of focus, the resourcing, the consideration of processes and decisions, the
engagement of stakeholders in a range of respects obviously to have that
greater enhanced focus upon cancer medicines and the particular challenges and
indeed opportunities around cancer medicines, that is obviously something we
would welcome in the local context.
A number of submitters advocated the establishment of an interim access
scheme within or closely aligned with the existing PBAC/PBS mechanisms.
The Tasmanian Government was not supportive of the establishment of a separate
funding stream specifically for cancer medicines outside the PBAC process,
stating that it is preferable for the PBAC process to be streamlined and
tailored where possible.
The Cancer Drugs Alliance (CDA) proposed the establishment of an interim
access scheme while 'the Government commences the process of PBS
Like the Tasmanian Government, the CDA considers that an interim access scheme
should be established within the existing PBAC/PBS mechanisms and should:
be designed to provide access to medicines between Therapeutic
Goods Administration (TGA) registration and PBS reimbursement;
time-limited and operate until revised PBS/PBAC measures have
operate within and in parallel to the existing PBS system, which
would continue to execute its obligations to approve and fund cancer drugs
based on the existing framework;
include clinically driven guidelines for listing and de-listing
include clear guidelines around acceptable pricing taking account
of issues faced by all stakeholders; and
be supported by the establishment of a database of outcomes
following the use of chemotherapy and targeted medicines.
Some submitters saw benefits in establishing an interim fund to provide
expedited access to treatments for rare and less common cancers, including
treatments already listed on the PBS for other indications.
Rare Cancers Australia (RCA) proposed that an interim access scheme should be administered
by the Department of Health (DOH) for the interim approval of medicines not
listed on the PBS. RCA proposed the following process:
applications to be made by a suitably qualified clinician; and
for indications considered to be rare or less common and for
which the company will not be making a submission to the PBAC for that
reviewed by a panel of clinicians and patient advocates who would
provide feedback to the DOH on each drug's safety, efficacy and potential
that the supply of approved medicines could be subject to a range
of other considerations, including a limited time period; a price agreed to
between DOH and the manufacturer, and an agreement with regard to any
outstanding amounts paid to the company in the event the drug is listed by the
RCA also stated that it may be necessary to implement some form of time-limit
penalty to ensure that applications are considered in a timely manner.
However, some submitters cautioned against the establishment of a
dedicated cancer fund, noting that overseas experience suggests that such funds
have not necessarily alleviated issues around cost and access to high cost
For example, Cancer Council of Australia and Clinical Oncology Society of
Australia (CCA/COSA) noted that while the CDF had improved access to new
listings, the fund was over budget and has been criticised for not addressing the
issue of price negotiation with manufacturers.
The Society of Hospital Pharmacists of Australia (SHPA) was also not
supportive of the establishment of alternate funding programs for specific
patient groups such as oncology:
SHPA believes that the current standard assessment and
approval systems are essential to ensuring the safety and cost effectiveness of
medicines funded by public monies or through the PBS. We do not believe that
these evidence-based decision-making principles should be compromised for any
SHPA further stated
...if the Australian government was to expand the range of
medicines funded outside the PBS, the limitations and capacity of systems such
as the Australian Life Saving Drugs Program and the Cancer Drugs Fund in the
United Kingdom must be examined in detail.
In its submission, SHPA noted a range of concerns about the CDF,
the fund's use of less stringent approval processes compared to
the standard NICE process;
a tendency for the price paid by the fund to result in the UK
paying a higher price for cancer medicines than most European countries;
the diversion of funds away from potential treatment
overspending has resulted in only 59 of 84 currently listed
medicines being funded in 2015-16; and
the CDF has undermined the NICE and the principle of
evidence-based decision making.
Roche Products also considered that the CDF has its limitations,
'principally the lack of an acceptable method of prioritising medicines to
list, and the perception of discrimination against other high-burden health
conditions'. Roche Products submitted that it supports systemic reform that
will improve access for all specialised medicines, including cancer. However, Roche
Products noted that, as reform would take time, the government should consider
models for providing interim access to new cancer therapies.
Ms Simone Leydon from the Unicorn Foundation told the committee that,
notwithstanding the difficulties encountered with the CDF, there was merit in
considering such a scheme as an interim measure:
There have been different mistakes probably made with that,
but the essence of it is that it provides an interim model—and we would stress
that these are interim models—while we look at reviving or changing some parts
of the PBS. They would not be there for the long term, which, unfortunately, I
think has happened overseas. And they would be more strictly controlled. So,
again, there would have to be some sort of restrictions put on what drugs could
go in there and how much and that sort of thing.
The Cancer Drugs Fund
A significant number of submitters suggested that an interim specialist
fund could be modelled on the United Kingdom's CDF.
As noted above, the CDF was established in response to criticism over the
timeliness of access to new cancer medicines, to fund access to cancer drug
were not approved by NICE due to insufficient cost effectiveness;
had not yet received final NICE guidance;
were for rare cancer licensed drug indications not selected for
NICE appraisal; or
were planned to be used off label (and therefore could not be
assessed by NICE).
The current operating model for the scheme is due to end on 31 March
2016. Initial funding for the CDF was originally capped at £200 million per
2013-14, the fund was overspent by £30.5 million. The UK government announced
that it would increase the CDF budget to £280 million in 2014-15 (and to £340
million in 2015-16), and that the list of drugs on the CDF would be reviewed,
with a focus on 'value for money'.
NHS England told the committee that while the CDF had substantially
increased access to cancer drugs, particularly for the treatment of rare
cancers or rare cancer indications which had not been assessed by NICE, the CDF
has had unintended consequences:
consequence of the CDF has been to initially reduce the incentive for drug
manufacturers to reduce the prices of their drugs to make their drugs cost
effective via a NICE appraisal. However, the recent incorporation of drug cost
when added to an assessment of clinical impact into the overall CDF
decision-making process has restored this need. The consequence of the setting
up of the CDF has also been to have an ever increasing number of CDF drugs
without final decisions as to whether they should be in baseline commissioning
or not and only definitively making such decisions when the CDF funding envelop
is threatened. An additional observation has been the recent trend for drugs to
be licensed on relatively preliminary data which creates much uncertainty in
NICE's assessment of cost effectiveness. This phenomenon is an additional
factor in explaining the recent higher rate of NICE negative recommendations.
In December 2014, a CDF Working Party was established, comprised of NHS
England, NICE, the Department of Health and representatives of the
pharmaceutical industry and cancer charities, to co-produce a proposal for a
framework for the long-term sustainable evaluation and commissioning of cancer
drugs. The framework is expected to be ready for public consultation during
'the summer of 2015' and for implementation from April 2016.
One of the potential solutions being considered by the Working Party is
a 'managed access' approach to the CDF 'with clear entry and exit criteria and
procedures which would be run jointly between NHS England and NICE.
NHS England told the committee:
recognises NICE's strength in the determination of overall clinical and cost
effectiveness and the ability of NHS England to produce robust clinical
commissioning policies for rare or off label cancer drugs. Both of these routes
of assessment in the new process will incorporate an initial consideration of
clinical promise and thus prioritisation of drugs which deliver the most
important, favourable and meaningful outcomes.
NHS England said the managed access approach is considered to allow the
potential for immediate access to funding on licensing and the collection of
further clinical outcome data if the NICE technology appraisal concludes that
longer term data are required. NHS England describes this as a 'Commissioning
through Evaluation' model.
Managed access models
A number of submitters proposed the adoption of a model for managed access
to medicines as a means of addressing concerns regarding evidence gaps and
timeliness in the PBAC assessment process.
For example, Roche Products expressed support for the more dynamic approach to Health
Technology Assessment (HTA) that a managed access scheme may provide:
Currently, under managed entry, an initial subsidy is
provided at a price justified by the existing data, pending the submission of
more conclusive evidence. Roche consider that the initial price must reflect
the value of the product and be in step with launch prices in other developed
markets. The totality of available evidence needs to be considered, and
subsequent evidence collection must be fit-for-purpose (i.e. address the
The committee notes that Australia has had a framework for a Managed
Entry Scheme (MES) since 2011. Under this framework, the PBAC has had the
ability to recommend PBS coverage for a drug at a price justified by the
existing evidence, pending submission of more conclusive evidence of
The expectation this measure is that the price of the drug could subsequently be
adjusted, either up or down.
The PBAC provided the following explanation of how managed entry works:
In managed entry, a provisional price for the drug is set on
the basis of the sponsor's estimate of effectiveness and toxicity, while data
on outcomes are systematically collected from patients and prescribing doctors.
In this way the clinical risk of lack of benefit and potential for harm are
countered, because those outcomes are detected early because national data are
collected. The financial risks associated with PBS-listing a drug whose
effectiveness is uncertain, but which the PBAC believes is not likely to be
cost-effective at the sponsors' preferred price, are shared between sponsor and
government because the sponsor agrees to repay money if the drug is less
effective in actual use than was predicted.
However, evidence to the committee was that the MES had not been
enthusiastically embraced by industry. The Leukaemia Foundation of Australia
(LFA) noted the limited use made of the MES to date and that the scheme is
currently under review:
The PBAC requires greater flexibility to list new drugs, such
as TGA listed orphan drugs, which have limited clinical data due to small
patient cohorts. The Managed Access Scheme was introduced as a solution to this
in 2011. However, the program has not been a success and is under review. A
less restrictive scheme is essential, allowing conditional listing with further
data to be supplied to demonstrate clinical benefit.
DOH told the committee that changes, such as the MES and 'pay for
performance', are slowly being taken up by industry 'as their value is
DOH submitted that:
...the increasingly expensive price of [cancer medicines]
represents marginal value and that it is difficult to justify continuing
acceptance of high costs for treatments that offer very small benefit. It is
vital that PBS pricing policies continue to put pressure on medicine pricing
and further consideration of 'pay for performance' (ensuring that the price
reflects available evidence of the health benefit) is also warranted.
DOH further stated:
The PBS has adopted innovative pricing models to provide
access to new drugs whilst also supporting the development of a stronger
evidence base. For example, the existing 'managed access' approach is being
reinvigorated to provide options for medicines that are used to treat rare
cancers by allowing a phased evaluation and listing, linked to progressive
payments. Earlier access than would otherwise be obtained could be granted,
where safe to do so, for use in those patients who have no other treatment
options. The health outcomes would be tracked and reviewed, with approval for
broader use only once sufficient evidence of effectiveness becomes available.
MA told the committee that the industry is open to working with 'public
policy makers' on the development of the MES scheme, but noted:
There is a balance to be struck between getting access to
patients who need those medicines most as early as possible and being prepared
to submit to requirements for both clinical and cost effectiveness.
Notwithstanding the limited use made of the MES to date, submitters
noted the potential for such an approach to address concerns regarding the
collection of clinical data and the consideration of broader social and
economic factors, by capturing 'real world' experience of new cancer
treatments. Mr Richard Vines, Executive Chair, RCA told the committee that the
solution requires a bit of flexibililty:
I go back to where you were talking about 'once a submission
is lodged'. Once it is registered and we know there is going to be a
submission, and the TGA says it is safe and the pharmaceutical company is
definitely making a submission, from that point on, if we can run a managed
entry scheme after PBAC consideration, we ought to be able to put something in
place beforehand, not that corrupts the PBS process—it has served us well for a
very long time—but allows it to go through the one to two years, if that is
necessary, and patients still have access. The issue is not the delay; the
issue is the patients who do not get it while the delay takes place.
Mr John Cannings, who appeared in a private capacity, told the
The issues are that currently, under the present guidelines
and PBAC rules, PBAC are not able to take into account some of both social
factors and economic factors in their determination around cost-effectiveness.
Those rules need to be modernised to allow earlier access. Part of that could
be through a managed entry scheme arrangement, where this real-world evidence
is then obtained, accumulated and put back into the system for all cancer
The PBAC noted that there are a number of issues that need to be
considered in formulation recommendations based on a managed entry approach:
the PBAC must have confidence that the clinical data provided at
the initial application shows evidence of likely benefit of treatment to
the sponsor should have additional studies in progress that will
potentially confirm this benefit and allow accurate assessment of the size of
the benefit over existing treatments;
alternatively, the sponsor needs to be prepared to collect data
from Australian patients to establish the benefits, harms and costs of
treatment. Clinicians and patients therefore need to agree to have such data
collected; the committee notes that this raises issues of privacy that are
beyond its remit, but that optimal implementation of managed entry may require
the sponsor needs to propose a price that is potentially
cost-effective, on the basis of the data available at the time of PBAC
the sponsor and DOH need to execute a contract to ensure all of
these issues are agreed, as well as a strategy for delisting the product and/or
recovering excess payments if the hoped-for benefits are not confirmed. This
process, including the fact the drug may be delisted, need to be very clearly
communicated to patients.
The committee notes that some of the PBAC's more recent recommendations
have been on the basis of a MES. For example, in announcing the listing of the
drug Crizotonib, used to treat anaplastic lymphoma kinase – positive non-small
cell lung cancer, on 1 July 2015, the Minister for Health, the Hon Sussan Ley
MP, stated that listing through a MES would speed up access for patients with
the highest need for treatment.
In chapter 4, the committee noted evidence from RCA and Mr Cannings regarding
delays in the listing of Crizotinib.
The PBAC recommended listing of Crizotinib following its November 2014 meeting,
having first considered an application for its listing at its November 2013
meeting. The drug was registered by the TGA on 27 September 2013.
In evidence to the committee, Dr Suzanne Hill, former Chair of the PBAC,
indicated that the PBAC is continuing to make recommendations for listing in
certain circumstances on the basis of managed entry, while the future application
of managed entry is being considered:
In terms of actual
managed entry schemes, the committee has recommended already a couple of
products with listings that are effectively managed entry. One was ipilimumab
for melanoma, nearly 18 months ago, where the agreement was that the price
would be set on the basis of the data that was available to the committee at
the time for the cost-effectiveness evaluation; that survival data would be
collected; and then, when the two years survival data was available, that the
cost-effectiveness would be re-evaluated on the basis of the survival and
toxicity data seen in the real world. Subsequently, the committee has made
another couple of recommendations from November in a similar vein.
So there are two
parts to it. There are the recommendations that are already coming out of the
committee that are effectively managed access or managed entry schemes, and
then there is some more general discussion of managed entry or managed access.
The committee notes that the discussion around the development of
managed access mechanisms appears to be centred in the work of the Access to
Medicines Working Group (AMWG) .
Since June 2014, the Managed Access Programme Sub-Group of AMWG has been
developing a framework for a Managed Access Programme. At its December 2014
meeting the AMWG noted substantive progress on the project and anticipated
broader review of the framework early in 2015. The PBAC reviewed the draft
framework at its March 2015 meeting.
It is not clear if broader consultation with clinicians and consumers is
contemplated in finalising the draft framework.
The committee notes evidence emphasising that managed access is only one
possible avenue for addressing demand for early access to new medicines. Submitters
noted a managed access scheme would work well alongside other mechanisms
currently provided for within the PBS such as risk sharing between the
Australian Government and the sponsor of a medicine.
Submitters also noted that the long-term challenge of funding subsidised
access to cancer medicines requires the consideration of a range of new
approaches. The committee received evidence suggesting that regulatory models
used in other policy domains may serve as useful models for addressing challenges
created by rapid scientific advances in cancer treatment. The committee
received evidence regarding two such models: the licensing of oncology
medicines and social impact bonds.
Risk sharing agreements
The committee notes risk sharing agreements are intended to help
maintain the appropriateness and cost-effectiveness of listed medicines.
CCA/COSA expressed support for use of risk-sharing arrangements as a
means of incorporating the use of surrogate endpoints into the evaluation of
As recommended against ToR a), a scheme based on surrogate
endpoints (also known as performance-based, risk sharing arrangements) could be
implemented in Australia. The scheme could involve new cancer drugs being
submitted for funding based on surrogate endpoints (such as progression-free
survival) with an upfront agreement (not subject to appeal) that funding would
be reduced if the drug, in post-market evaluations, did not realise a major
endpoint such as overall survival or improved quality of life.
Post-marketing surveillance under this type of scheme would
need to be strictly conducted, as the earlier a drug is marketed, the greater
the risk of uncovering unusual or adverse effects.
However, Roche Products submitted that while risk-sharing agreements are
aimed at reducing listing delays following a positive PBAC recommendation, they
are frequently one-sided and may impose requirements that are not based on
clinical best-practice but simply reducing financial costs to government beyond
what is required for cost-effectiveness. However, Roche Products recommended
that industry and government should work together to identify opportunities to
further streamline listing processes.
Merck Sharp and Dohme (MSD) also stated that the reason for the poor
take up of the original MES was the perception that 'all risk in participating
in the scheme would be borne by the companies, with little hope of price
increases even if conclusive evidence was forthcoming'. MSD expressed hope that
the development of a new framework through the AMWG would formalise a more
effective way of giving patients access to new products.
Licensing innovative oncology
RCA questioned whether the current approach of treating medicines as
products and seeking approval on an individual product basis remains an
appropriate model. RCA suggested that treating innovative medicines as
intellectual property and applying a 'service' model, similar to that used for
software, music and film:
The proposition is that, in order to address the failings and
delays of the current system and to avoid the future capacity issues that seem
likely, we look at the model of "Medicines as a service". In other
words instead of pricing and costing each tablet or ampule as a separate
exercise we examine the possibility of licensed usage for a medicine.
RCA states that this type of funding model has the capacity to deliver
benefits in terms of certainty, flexibility and simplicity.
Social impact bonds
The CDA White Paper proposed further consideration of social impact
bonds (SIBs) as means of providing incentives from investments in cost-saving
preventive services. CDA stated that SIBs can ensure that public funding goes
only to interventions that demonstrate their impact through rigorous
outcome-based performance measures.
Under the most common social impact bond model, the
government contracts with a private sector intermediary to obtain social
services. The government pays the intermediary entirely or almost entirely
based upon achievement of performance targets.
The CDA considered that SIBs offer innovative solutions for funding
health initiatives and could have long-term benefits beyond the funding of
access to cancer medicines. Australia's first SIB is the Newpin Social Benefit
Bond, funded by the NSW Government.
The CDA states that in proposing consideration of alternate access models such
as SIBs, it is not attempting to design alternative funding models external to
the PBS. Its intention is to identify models that might 'relieve pressure on
already constrained resources, while ensuring we improve access for Australian
Improved monitoring and data collection
Evidence to the committee underscored the importance of improved data
collection and greater integration of existing data bases to improving the
speed with which cancer drugs are assessed for registration and reimbursement
and the level of information available to assist clinicians. Evidence to the
committee noted that the success of managed access programs for new medicines
is dependent on accurate data capture systems.
Post marketing surveillance
Submitters stressed that it is important to continue to monitor clinical
and cost effectiveness of all medicines after they are listed on the PBS and
recommended the application of greater use of post-market review processes.
The committee heard that currently little is known about patterns of use,
patient outcomes or safety following the grant of marketing approval.
CCA/COSA told the committee that while they consider there is an excess
level of rigidity in the pre-market assessment of medicines, the same rigour is
not applied to assessing the efficacy and cost-effectiveness of medicines once
The absence of rigorous, ongoing post-listing review can lead
to unnecessary expenditure and suboptimal use of listed medicines. Greater
rigour in post-market review is a potential cost offset that could allow for
the listing of new medicines which, while vitally important to a comparatively
small number of patients, do not currently meet cost-effectiveness criteria.
Greater rigour in post-market evaluation would also be a necessary tool for
accepting surrogate endpoints other than disease-free survival as indicators of
efficacy when assessing new PBAC applications – as recommended in response to
the problems of timing and delay.
Agreed future milestones could be monitored through regular
post-market assessment using agreed, pre-determined reporting mechanisms. It
would require a commitment from the sponsor to provide results of ongoing
studies and greater monitoring of safety and efficacy post-market by the TGA.
SHPA also expressed concern that under the current system of post-marketing
surveillance products are reviewed in an ad hoc manner in response to stakeholder
concerns about a particular product. SHPA recommended that all medicines funded
through the PBS should be systematically and routinely assessed against the
criteria that was initially used to approve the product for listing, as well as
any newer, relevant evidence which has been published since the time of
COSA member Ms Suzanne Kirsa told the committee that improved
post-market surveillance to ensure that listed medicines are continuing to
provide value for money, could help to offset some of the costs associated with
the listing of new medicines via more flexible evaluation processes.
A system of post-market reviews was introduced following the 2011-12
budget to assist with improving the sustainability of the PBS. Post-market
reviews provide a means of monitoring medicines in use to inform decision
making at all levels throughout the medicine cycle, from registration to its
use by consumers. The committee notes the post-market review program is
intended to contribute to:
improved patient safety through better understanding of adverse
events and medicine-related harms;
ensuring the ongoing viability of the PBS through targeted
medicines usage and avoiding preventable wastage or inappropriate prescribing;
a better understanding of medicines utilisation, to review
intended clinical benefit and inform medicines evaluation processes;
ongoing cost-effectiveness, including through better management
of clinical and economic uncertainty; and
overall improvements to the quality of use of medicines and education
for patients and prescribers.
The committee notes that new guidance for post-market reviews of
medicines listed on the PBS were announced in March 2015. The new guidance was
produced by the AMWG and has been agreed by the pharmaceutical industry, MA and
Submitters noted that governments in other countries are increasingly
recognising the value of a coordinated national approach to data collection to
justify expenditure on cancer treatments, to provide a framework for earlier
access to new treatments and drive improvements in the delivery of better
outcomes for cancer patients.
Ms Carlene Todd of Roche Products said:
What we can learn from other countries though is around
collection of clinical data in the real world. Italy and the Netherlands do
this well. They have cancer registries in place and they can collect evidence
in clinical practice over time for medicines.
In its White Paper, CDA advocated the establishment of an Australian
national chemotherapy registry (NCR) to enable identification of trends in
clinical practice and patient outcomes. CDA noted that this information could
be used to inform and improve the quality of care across the country:
The NCR's focus will be to improve patient outcomes by
monitoring and improving quality of care. The main purpose of the data
collection would be to:
Monitor current relevant patient
information and linking to medicine use and patient outcomes, including safety
Report risk-adjusted benchmarked
data with the purpose of improving quality of care and delivering optimal
Facilitate decision-making for
access to new cancer medicines on the pharmaceutical benefits scheme (PBS);
Provide a framework that would
support earlier access to cancer therapies such as through managed entry
schemes/managed access programs;
Provide a framework for the
collection of real world data to measure cost effectiveness in Australian
clinical practice; and
Monitor and report on delivery of
equitable cancer care across Australia.
MSD also supported the establishment of a NCR to continually assess the
efficacy of medicines and to help demonstrate the value that medicines bring to
At present, despite significant expenditure on cancer
medicines, no comprehensive picture exists of what happens to patients once
they are placed on therapy, as no national database exists which captures this
information. As well as empowering clinicians with information to improve
cancer care, such data provides a framework to monitor real world cost
effectiveness and to support Managed Access Schemes. This would enable the
health community to continually monitor whether it is getting value for money
out of its investment.
However, the committee also heard that, before investing in a national
clinical cancer registry, there is a need to address to address mechanisms for
the collection of data on a national basis.
We do not have a national database. There are a lot of
disparate registries. What the Medicines Australia submission talks about and
some of the others as well is the need to link some of those registries
together to make sure that they are talking to each other so we can access the
data within those registry silos. That is a big issue at the moment, and
something we should probably look at first before we invest in a national
Greater integration of existing
Submitters noted that greater integration of existing datasets would
provide a means of harnessing the potential of real time monitoring of outcomes,
both from clinical trials and post-PBAC approval, to support managed entry and
improved patient care. For example, COSA suggested the 'integration of post
market surveillance and reporting to track cancer medicines introduced into the
market early, as well as the ongoing effectiveness of approved medicines and
Dr Hill told the committee that more effective collection of clinical
outcome data such as fact of death or adverse effects would be 'an enormous
advance' in supporting the use of managed entry schemes:
Equally, being able to collect clinical outcomes such as fact
of death or adverse effects more effectively than we can at the moment would be
an enormous advance in trying to arrange what we have called managed entry
schemes, where we need to try and monitor drugs that are made available to the
community early to make sure that the benefits and harms are the same as what
we see in the clinical trials.
In its submission to the inquiry, the PBAC emphasised the importance of
linking data sets to support early access.
Professor Zalcberg of CDA also emphasised the merit in linking existing
databases to enable a more comprehensive understanding of cancer drugs:
Clinical trial data is often incomplete at the time. For
example, long-term survival issues are not available; the true toxicity or
safety profile is not understood; we have heard about progression-free survival
and overall survival; there may be interim end-points. So there is a need to
get further data. The way to collect that data is to link existing
databases—like Medicare, like PBAC, like the Australian Cancer Database—in a
way that protects people's privacy but allows information that has already been
collected and information that can be collected into a comprehensive picture
about what these drugs are doing and what they are not doing.
The Australian Cancer Database (ACD) is a data collection of all
primary, malignant cancers diagnosed in Australia since 1982. The ACD is
compiled by the Australian Institute of Health and Welfare (AIHW) from cancer
data provided by state and territory cancer registries through the Australasian
Association of Cancer Registries, which in turn receive information on cancer
diagnoses from a variety of sources including: hospitals, pathology
laboratories, radiotherapy centres and registries of births, deaths and
marriages. Data from the ACD is used to report on national cancer statistics
such as incidence, trends, projections, survival and prevalence. While the
AIHW acts as custodian of state and territory registry data for the purposes of
producing national cancer statistics, the cancer registries retain ownership of
their jurisdiction's data at all times. The AIHW is able to make available a
broad range of cancer statistics subject to a scientific and ethical review
DOH noted that the increasing use of e-health records may also assist in
LFA expressed concern that without access to good information some
patients may be being offered treatments that are no longer considered to be
'best practice'. LFA submitted that Australia needs a centralised process for
all drug access inquiries:
Without a national clinical cancer database, factually-based
information about which therapies are best practice in the Australian community
remains unknown. Therefore, therapy decisions are essentially educated
guesswork and doctor preference, and may be subjected to conflicts of interest.
Professor Brendon Coventry of the Australian Melanoma Research
Foundation also described the difficulty in accessing data:
It is really hard. In the melanoma foundation we have been
trying to get the information about complete response rates, outcomes, stuff
that you should be using to make your decision, your informed choices. We find
it really hard to get. It is buried in papers, it is in tables, it is in
supplementary information and so on. This is extremely difficult for an
experienced clinician and a group of clinicians to do. The patient has almost
no hope of doing it on their own, unless they have the experience. We are
trying to distil that information, compare the studies, compare the trials, put
the information on the table so they walk up to one source and get that
information that they need to have at their fingertips in order to make an
informed choice in the clinic when the clinician starts speaking to them.
Mr Martin Ashdown, Research Fellow in the Faculty of Medicine at the
University of Melbourne suggested that the AIHW might play a role in
facilitating access to clinical data:
It would be very valuable if the Australian Institute of
Health and Welfare could also collect survival and complete response data for
different drug treatments and their associated costs to enable closer
evaluation of clinical effectiveness for patients and clinicians, and especially
to permit better informed choices.
Leukaemia Foundation of Australia (LFA) noted the emergence of private
pay-for-service models, such as Biogrid, which collects data from patients with
a range of cancers and other health conditions that are being treated in a
number of hospitals in Victoria, Tasmania, South Australia, ACT and Queensland.
LFA stated that an example of the value of such clinical databases was 'the
analysis of outcomes for patients with metastatic colorectal cancer' which had 'enabled
differences in practice between centres to be identified, and practice reviewed
and standardised to improve patient outcomes'.
However, LFA argued that such databases need to be established on a
national scale so that they can from part of everyday cancer care delivery and
not be 'available just to the small number of patients who find out about it,
either through their health provider, or online'.
LFA noted that a national clinical database could also facilitate the review of
changes in indication, extending approval to other cancers with the same
genetic mutations and survival benefits, by providing the TGA and the PBAC with
access to extensive data, over and above that provided by the drug sponsor.
Capturing data from off label use
Submitters also noted the importance of capturing information about
outcomes from off label use of cancer drugs. For example, Cancer Voices
Australia recommended the use of post-marketing surveillance of real-life use
of medicines, not just in clinical trials, to assess cancer drugs'
effectiveness and impact on quality of life.
Cancer Voices SA submitted:
We need routinely collected comprehensive, high quality data
to monitor outcomes of all cancer patients. This data should be analysed and
reviewed to ascertain the effectiveness of treatments, particularly high cost
treatments. We need to be able to assess cancer drugs' effectiveness and impact
on quality of life, not just in clinical trials, but through comprehensive post
marketing surveillance. We are missing the information about the outcomes from
all those who access these high cost drugs outside of clinical trial situations
(eg via family and community fundraising) or special access schemes. We need to
ascertain if the expected benefits are achieved when these drugs are used
outside the clinical trial population.
Ms Michelle Stewart of Cure Brain Cancer Foundation told the committee:
People are taking lots of different things and none of that
data is being collected. For the larger group of patients, there is little
benefit. We believe we could capture this off-label use, which is happening
anyway, through a registry. Patients would then disclose what they are taking
and we could track their responses on those treatments.
Clinical trials are set up for a good reason and that is to
gain evidence for using a certain treatment. If you start using off label you
must make sure that you are collecting the data as well.
The committee notes strong support for the introduction of an interim specialist
cancer drug fund, pending review of the current system for listing medicines on
the PBS and the examination of other models for providing expedited access to
medicines. Evidence to the inquiry is that the introduction of specialist
schemes overseas has resulted in both faster approval times and the
availability of a greater range of medicines to cancer patients. However, the
committee notes concerns, both in Australia and overseas, that existing
specialist schemes have not necessarily alleviated issues around cost and
access to cancer medicines and that such funds have the potential to undermine
the rigour of existing evaluation processes.
Evidence to the committee has emphasised the potential for a managed
access program to address concerns regarding the availability of clinical data
to demonstrate the case for listing medicines on the PBS without undermining
the PBAC process. However, the committee notes the importance of effective
monitoring of both clinical and cost effectiveness after PBS listing, both as a
means of improving patient outcomes and as a basis for delisting medicines
Finally, the committee notes the importance of effective collection of
clinical data and the merit in linking existing databases to enable more comprehensive
analysis of the benefits of cancer medicines by the PBAC and clinicians and to
support best practice in patient care.
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