Conclusions and recommendations
As noted in this report, Australia has the highest incidence of cancer
in the world. While Australia also has some of the best cancer survival
outcomes in the world, the provision of timely and affordable access to new and
innovative cancer medicines provides a significant challenge for the Australian
Government, clinicians and patients. These challenges stem in part from the
fact that cancer medicines are among the most expensive medicines, and from
Australia's relatively small patient populations.
These challenges are also a consequence of an increasingly sophisticated
understanding of cancer as not one, but many hundreds of diseases requiring an
equally sophisticated and individualised method of treatment. The committee
heard that advances in the treatment of cancers are frequently incremental and
increasingly targeted at small patient populations. More targeted medicines and
therapies have the ability to increase the range of treatment options for
cancer patients, resulting in improved quality of life and survival for many
patients. At the same time, cancer is an area of high clinical need meaning
that, even with access to subsidised medicines, many cancer patients face
significant financial hardship. These challenges are exacerbated for those
patients with rare or less common cancers, particularly children and young
people, and those who live in rural and remote communities.
These factors pose a significant challenge for all governments as they
seek to facilitate affordable cancer care while maintaining the sustainability
of the overall health budget. The current trends in cancer research can be
expected to continue.
Throughout this inquiry, the committee has been acutely aware that
cancer patients are not the only patients who experience difficulty in
accessing new and innovative medicines in a timely way. The committee considers
that the concerns identified in this inquiry could easily apply to those
diagnosed with a range of chronic or less common diseases. What sets cancer
patients apart from many other patients is time. The vast majority of cancer
patients do not have time on their side.
The committee considers that if the process for the assessment and
listing of medicines can be enhanced to address the particular concerns that
arise in relation to cancer medicines, it will inevitably serve the needs of
all Australians more effectively.
Evidence to the inquiry has underscored the fact that access to
medicines ultimately depends on the ability of patients to pay for them. The
listing of medicines on the Pharmaceutical Benefits Scheme (PBS) plays a
significant role in ensuring this access is equitable for all Australian
As mentioned earlier, evidence to the inquiry has demonstrated that for
many cancer patients access to new and innovative treatments comes at
significant personal and financial cost. Those who require access to cancer
medicines not currently listed on the PBS must resort to access through compassionate
programs or clinical trials. Evidence to the committee has demonstrated these
avenues of access are neither equitable nor certain and frequently incur
The committee heard that the inability to access cancer medicines,
either because the preferred course of treatment is not registered in Australia
or is not subsidised via the PBS, has significant flow on consequences for
cancer patients and the people who care for them. The committee received
numerous accounts describing the personal experience of cancer patients. These
accounts underscored the grim reality that for cancer patients delays in access
to new and innovative cancer medicines can be measured in loss of quality life
years and lives lost.
The committee notes that a key factor in the timely availability of new cancer
medicines is the timing of applications for registration and reimbursement by
pharmaceutical companies. This is a commercial decision made in the context of
a global industry. The committee understands the commercial imperatives that
may lead a pharmaceutical company to seek regulatory approval in the United
States or Europe in the first instance, in preference to a country with a small
population, like Australia.
However, while the timing of the lodgement of applications is outside
the control of Australian regulatory authorities, the committee notes that
there is scope for the Australian Government to ensure that the regulatory
processes in place for the consideration of applications are efficient and do
not act as a disincentive to companies to seek listing and reimbursement.
Enhancing the operation of the TGA and the PBAC
Evidence to the inquiry has stressed the value that stakeholders place
on the PBS and the Pharmaceutical Benefits Advisory Committee (PBAC) system.
Submitters noted the importance of decisions regarding the registration and
reimbursement being based on a rigorous, evidence based assessment of safety,
efficacy and value.
However, while submitters consistently emphasised that the current PBS
and PBAC process has served Australia well, they also emphasised the need for
the system to be reviewed to ensure that it is capable of dealing with the
challenges posed by the rapid development of cancer treatments in particular.
While some submitters expressed concern that a one-size-fits-all assessment
process is no longer fit for purpose, the committee considers that the concerns
raised in relation to the current process should be able to be addressed
without creating a parallel process. The committee also notes that there is
considerable commitment and goodwill within the pharmaceutical industry and the
stakeholder community to work with government to explore avenues for addressing
More streamlined and flexible
While the committee has noted that the current assessment processes are
appropriately rigorous and based on clearly cyclical timelines, the committee
notes that there is scope to improve the timelines for consideration of
applications. The committee also notes that greater flexibility regarding
evidentiary requirements and provision for entities other than the sponsor of a
medicine to seek registration of new indications for medicines in appropriate
circumstances may address concerns regarding the responsiveness of the current
registration system to changes in the clinical setting.
The committee notes evidence to the inquiry regarding fast track
processes employed by overseas regulators and has noted that key features of
such programs are early and frequent engagement between the regulator and the
sponsor to address any issues associated with assembling data in support of an
application and some form of 'rolling review'.
The committee also notes concerns regarding the evidential requirements
of the current system. The committee considers that greater formal emphasis
should be placed on quality of life considerations. In this context, the
committee welcomes the current review of Parts II and III of the PBAC
Guidelines and notes that the review provides a timely opportunity clarify the
information requirements for applications for PBAC assessment. The review also
offers an opportunity to identify new developments with regard to current
methodology, along with any issues of scientific debate and consideration of
Australian and international best practice.
The committee also supports greater collaboration between the TGA and
the PBAC, along with continued examination of current parallel processing
arrangements, to identify options for streamlining processes and minimising
duplication in order to achieve compressed timeframes where possible.
Improved managed access programs
The committee notes the potential for managed access programs to address
some of the concerns raised in relation to evidential requirements while at the
same time providing more timely access to subsidised medicines. While
Australia's initial managed entry scheme has not been enthusiastically
embraced, the committee welcomes the work of the Access to Medicines Working
Group (AMWG) in developing a new framework for a managed access program. The
committee encourages the AMWG to consult closely with clinicians and consumers
in finalising the framework.
The committee also notes evidence emphasising the need for consideration
of a number of possible avenues to address demand for early access to new
medicines. The committee notes that the provision of sustainable subsidised
access to medicines, particularly expensive cancer medicines, will continue to
pose a significant challenge for the Australian Government. The committee
therefore supports the examination of a range of possible access models.
An increased role for consumers and
The committee considers that consumers and clinicians should play a more
substantial role in the evaluation of new medicines. The committee commends the
PBAC for its efforts to facilitate consumer engagement through the introduction
of consumer and patient hearings.
The committee considers that consideration should be given to avenues
for facilitating more formal discussion with the Australian community. The
committee notes evidence received regarding the operation of formal mechanisms
overseas to capture community expectations around broader moral and ethical
considerations and considers there is merit in considering how similar
mechanisms might operate in the Australian context.
The committee considers that greater transparency throughout the
regulatory system will enhance the engagement of all stakeholders and will
support a clearer understanding of the reasons for delays in listing of
particular cancer medicines. Greater transparency also has the potential to
support greater procedural efficiency and a commitment continuous improvement.
The committee notes the PBAC's commitment to increasing the transparency
of its processes and the level and clarity of information available to consumer
and patient groups. The committee notes the implications of commercial in
confidence considerations for these initiatives, but encourages the PBAC and
industry to work together to address these.
Improved monitoring and data
The committee notes the importance of establishing effective mechanisms
for collecting and analysing clinical data in relation to the use of cancer
Evidence to the committee has underscored the importance of effective
review of medicines after their listing on the PBS as a means of supporting the
listing of medicines through managed entry programs. The committee welcomes the
new guidance for post market reviews produced by the AMWG. The committee
encourages the AMWG to continue to consult widely on the operation of the post
market review program as greater use is made of managed access programs and
more flexible assessment criteria to explore ways in which the program could
support such initiatives.
The committee notes calls for the establishment of a national cancer
registry, and, while it sees merit in this proposal, considers that a review of
existing data collection mechanisms is a necessary precursor to the
establishment of such a registry. The committee considers that a review of data
collection must consider options for linking existing databases, facilitating
wider access to the data collected and avenues for collecting data regarding
the off-label use of cancer drugs.
The case for an interim specialist
cancer drug fund
Evidence to the committee stressed that, while a comprehensive review of
the current PBAC processes was necessary, such a review would take time to complete
and cancer patients do not have time on their side. Submitters advocated the
introduction of an interim cancer drug fund pending completion of a review,
particularly for patients diagnosed with rare cancers.
The committee is cautious around suggestions that advocate for the
establishment of separate regulatory mechanisms specifically to deal with
cancer drugs. The committee is mindful of concerns raised about the operation
of such funds overseas. In particular the committee is concerned at the potential
for such funds to exacerbate some of the issues identified with the current
PBAC system around cost and access to cancer medicines, and the impact of
separate assessment processes on the rigour and integrity of the PBAC system.
The committee notes NHS England's current review of its Cancer Drug Fund
and the unintended consequences arising from the operation of the fund. The
committee notes that NHS England is considering a managed access pathway as an
alternative to a cancer fund.
The committee considers that if such a fund were to be established, it
is preferable that it is established within the current regulatory framework
and operates consistently with existing processes. The committee considers that
the current Life Saving Drugs Programme (LSDP) may offer a basis for the
delivery of an expanded government funded compassionate access program for
patients with rare or less common cancers.
The operation of the LSDP is currently the subject of a post-market
review. While a technical assessment of the LSDP has raised questions regarding
the sustainability of the program in its current form, it has also highlighted
options for enhancing its operation. The committee considers that there is
merit in drawing on this current review to examine the scope for modifying the
administration of the LSDP to provide an interim means of subsidised access to
medicines for the treatment of rare cancers.
The need for a coordinated review of access pathways for cancer medicines
The findings of this inquiry are not new. Similar findings have been
identified in previous reviews initiated by the Parliament and the Australian
Government. However, the evidence to this inquiry has underscored the
importance of acting to address the concerns raised in order to ensure that
Australia has a system that is capable of meeting both the challenges posed by
rapid developments in medical research and the demand for subsidised access to
new and innovative medicine in a way that is timely, equitable and sustainable.
The committee has acknowledged that the current work of the independent
Review of Medicines and Medical Devices Regulation also overlaps with the terms
of reference of this inquiry and has produced findings that are consistent with
the evidence the committee has received. The committee notes that the review
panel has made recommendations to:
expand the pathways by which sponsors can seek marketing approval
for a medicine or medical device, including making provision for utilisation of
assessments conducted by comparable regulators, and for expedited assessments
in defined circumstances;
identify comparable overseas national regulator authorities using
enhance post-market monitoring of medicines and medical devices
and streamline post-market requirements in respect of products in the
Australian Register of Therapeutic Goods; and
improve transparency and predictability of processes and
decisions to build trust and confidence in the NRA's ability to ensure
Australians have timely access to high quality, safe and efficacious products.
The committee urges the Australian Government to give careful
consideration to the implementation of these recommendations.
The committee also acknowledges work undertaken by the pharmaceutical
industry and other key stakeholders. In particular, the committee notes the
outcomes of the work streams initiated by the Cancer Drugs Alliance as a result
of its forum in March 2014. The work of the AMWG in relation to the managed
access program, transparency of PBS processes and post-market reviews, also has
the potential have a positive impact on access to new cancer medicines. The
committee considers that this work within the stakeholder community speaks to
the considerable value placed on the PBAC system and the commitment and good
will expressed by all stakeholders to working closely with government to
improve its operation.
The committee has also noted initiatives that have the potential to
impact on the assessment of medicines for listing on the PBS. While some of
these, such as the review of the PBAC Guidelines and initiatives to enhance
consumer engagement throughout the PBAC process, are positive interim steps
towards enhancing the operation of the current system, the impact of others,
such as the Pharmaceutical Benefits Scheme Access and Sustainability Package,
are not yet known.
The committee recognises the importance of timely, interim changes but
is concerned that an incremental approach to reform in this area risks being
piecemeal and may squander the opportunity to identify synergies and
efficiencies that a more coordinated and comprehensive review could identify.
The committee considers that it is incumbent on the Australian Government to
respond to the challenges facing the operation of the PBAC and the ongoing
sustainability of the PBS in a comprehensive and considered manner.
The committee also wishes to emphasise the importance of consulting
widely in the development and implementation of changes to the current system. In
particular, while the committee welcomes the work of the AMWG, the committee encourages
broader consultation with all relevant stakeholders prior to the implementation
of changes as a result of the AMWG's work program.
Finally, as noted above, while this inquiry has focussed on access to
cancer medicines, the committee considers that its findings have broader
application. A review that seeks to address the concerns raised with regard to
access to new and innovative cancer drugs, will inevitably address the concerns
of all of those patients who rely on the PBS for timely and affordable access
to best practice medical treatment.
The committee recommends that the Australian Government initiate a
comprehensive review of the system for the registration and subsidisation of
medicines. The review should examine:
all available pathways for the registration and listing of new
medicines, or new indications for medicines already registered on the ARTG and
listed on the Pharmaceutical Benefits Scheme, including making provision for
utilisation of assessments conducted by comparable overseas regulators; provision
for clinicians and/or patient groups to apply for an extension of existing
registrations to additional indications, managed access programs and
risk-sharing, and the adoption of more flexible evidential requirements;
options for improving the operation of assessment processes
enhancing engagement with sponsors and other stakeholders to
better tailor their applications to the requirements of the PBAC, including
consideration of pre-application planning meetings;
applying tiered assessment processes as a means of matching
resources to the complexity of applications;
encouraging greater cooperation between the PBAC, the TGA and the
Medical Services Advisory Committee, including examination of options for
enhancing the operation of parallel processing arrangements; and
ensuring greater transparency throughout the assessment process;
options for expanding the post-market review of medicines;
enhancing and formalising mechanisms for consumers and clinicians
to play a more central and substantial role in the evaluation of new medicines
and new indications for already listed medicines, including:
consideration of options for expanding consumer and clinician representation
on the PBAC;
enhancing existing avenues for stakeholder input, including the
use of consumer and patient hearings; and
avenues for incorporating public perspectives on overarching
moral, ethical and opportunity cost considerations into PBAC decision making
processes, including consideration of models employed by comparable overseas
options for ensuring that the necessary administrative and
technical resources are available to support the implementation of an enhanced
The committee recommends that the Australian Government commission a
review of current data collection mechanisms for cancer medicines, including
obstacles to the integration of existing databases and potential
avenues for addressing these;
opportunities to incorporate data from post-market evaluations;
avenues for capturing data relating to the off-label use of
The committee recommends that the Australian Government establish a
Steering Committee to examine the feasibility of establishing a national
register of cancer medicines.
Senator Rachel Siewert
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