Impact of delayed access to cancer medicines on cancer patients
Throughout the inquiry the committee heard that timely, affordable
access to treatments and quality care are inherently linked for cancer
patients. A strong message in the submissions was that most cancer patients,
particularly those with advanced disease, cannot afford to wait for effective treatment
options to become available in a subsidised form in Australia.
The committee heard that the quality of cancer care is negatively
impacted when the appropriate course of treatment for a patient's cancer
profile is either not available in Australia or is not subsidised via the Pharmaceutical
Benefits Scheme (PBS).
The committee received evidence that suggested underuse of potentially
successful treatments has played a part in the low survival rates of patients
with lung, colorectal and ovarian cancers in Denmark and the United Kingdom.
This chapter examines the impact difficulties in accessing cancer
medicines can have on the quality of care available to cancer patients. It
considers the avenues available to individual cancer patients to access cancer
medicines that have not been listed on the PBS before considering the
particular challenges faced by rural and regional cancer patients, patients
with rare and less common cancers and children, adolescents and young adults.
The impact of delayed access on cancer patients and their families
The committee received many submissions from individual cancer patients
and their families urging the committee to support the expedited listing of
specific cancer medicines. Personal accounts from cancer patients and their
families highlighted the distress experienced by patients and their families
when apparently superior treatment options are not available in Australia via
the PBS. The committee heard that delays in the approval of cancer treatments,
together with delays in the commencement of treatment due to the need to seek
special approval or organise finance is a source of significant stress for
The committee heard repeatedly that cancer patients with advanced disease
cannot afford to wait and that the knowledge that a particular drug may enable
them to gain more valuable time or improve their quality of life, but at a
price outside their ability to pay, is a source of great anguish.
Ms Lorraine Hoskin's account of the plight of a young family friend is
indicative of a great many submissions to the inquiry:
A young family friend has recently been diagnosed with a rare
form of cancer. After the chemotherapy options have now been exhausted without
a positive result his only option to gain more valuable time is a very
expensive cancer drug. The decision whether to start this treatment is a major
stress to him because of the financial cost which will affect the future of his
young wife and family.
On top of the extreme suffering he has endured fighting this
wretched disease, the added torment of knowing there is a drug which could help
him, but at a huge cost is very unfair.
Submitters described the significance to cancer patients and their
families of maintaining a normal life. Many described the physical, emotional
and mental toll associated with cancer treatments that disrupt everyday
activities and separate cancer patients from family life. Cancer patients spoke
of the importance of being able to return to the workforce and contribute to
society, and emphasised the distress felt when this is not possible. Ms Lee
McKerracher told the committee:
The cost of treatment may be expensive, but how can you put a
price on giving a family member an extra 6 months, 12 months, 2 years with
their family. Some may even be able to return to the workforce or their studies
to contribute to society with whatever time they have left – we should give
them the chance to do so.
Evidence to the committee emphasised the potential of many new cancer
medicines to improve the quality of life of many cancer patients by reducing
the need for costly and invasive treatments such as surgery or hospitalisation.
For example, Medicines Australia (MA) noted that consumer comments received by
sponsors indicate that a simple and short outpatient administration of a single
new targeted cancer drug has greater benefit compared to a treatment regimen
involving multiple chemotherapies, long infusion times and the requirement to
travel to the hospital several times per week or month.
For Ms Karen Cowley, a breast cancer survivor, access to the drug
Kadcyla has stabilised her disease and allowed her to feel a degree of control
in her life:
Living with cancer affects a person physically, emotionally,
and mentally. It can interfere with everyday activities. For many years I felt
I was on a tightrope having many different regimes of drugs to manage my
disease with many side effects. Kadcyla has been a revelation, since May 2014,
I feel that I have some control in my life as my disease is stable and I have
less side effects. Administration of the drug is also less invasive, with only
one infusion taking less than an hour whereas other medications took several
Such benefits extend beyond the patient to those supporting them who are
spared months of worry and stress and caregiver time.
The committee received many submissions from cancer patients and their
families highlighting the significant physical, financial and psychological
impacts of being unable to access new and innovative cancer drugs. Many
patients are faced with the harsh dilemma of either paying a significant amount
of money to access the latest, most effective treatments for their cancer or
being unable to access the treatment at all.
Ms Louise Marshall of the Australian Melanoma Consumer Alliance told the
committee that many cancer patients are faced with the choice of selling their
home, accessing their super or relying on the fundraising efforts of friends
Bowel Cancer Australia told the committee:
The alternative for many is forgoing treatment and therefore
the possibility of precious extra time with their loved ones.
A number of submitters gave accounts of their frustration at finding the
medicine needed to treat their cancer was not listed on the PBS for their
cancer, or for their stage of the disease. Ms Bridget Whelan, who has ovarian
cancer, told the committee that:
I finished chemotherapy in May 2014 and spent some time
recovering and getting my health back. Chemo takes quite a toll on your body
and your mind.
Since then I have been on what is called a "maintenance
drug" – it's a drug to slow tumour growth and prolong your remission. I
pay for this drug. When I started it, it wasn't on the PBS at all. It now is,
but not for me. Only for women diagnosed for the first time. So I entered into
a co-pay arrangement with the pharmaceutical company. It’s expensive. It’s more
than another mortgage. I am so lucky I am in a position to do this. I imagine
only a handful of women in Australia can and that disturbs me greatly. This is
a drug which has shown good results in worldwide trials in prolonging the
remission of women just like me. The alternative, the standard Australian
Government funded approach, is that after my last chemo, I would do nothing and
just wait for it to come back.
Mr Scott Beyer, who has non-Hodgkin lymphoma, told the committee of the
disappointment of knowing the preferred treatment for his cancer is not listed
on the PBS:
When we first met with the head oncologist at the Alfred
hospital to discuss my options he said I would be better off to stay on
Bentuximab for an extended period of time or until the drug possibly failed to
keep the cancer under control. We alluded to the fact that it was going to be
such financial burden on our family that this wasn't going to be possible. He
was of the understanding that this drug was on the PBS, but what he didn't
realise was that it didn't cover my type of cancer. The fact that he believed I
would be better suited staying on Brentuximab for an extended period of time in
lieu of going through an arduous and sometimes fatal procedure shows how
important the access to these drugs is, but the fact of the matter is it is
financially out of my reach.
In this day and age this is just unacceptable and truly
With the support of family and friends, Mr Beyer has been able to raise
funds to support his treatment, but expressed concern that 'this generosity
can't last forever'.
Submitters expressed concern that Australian patients appear to be
missing out on new cancer treatments and are relying on older alternative
treatments, with harsh side effects, compared with cancer patients in many
Mr Janis Kinne told the committee of the constant stress of living with
advanced prostate cancer and not being able to access best practice treatments
available in other countries:
It is very tense and very stressful for anyone afflicted and
their families. Hormone resistant cancer can appear at any time for no evident
reason. When it does the prognosis is not good and life expectancy starts down
the slippery slope. Men with prostate cancer in United States are able to be
treated successfully with Zytiga or Xtandi before chemotherapy. It is
frustrating that Zytiga and Xtandi are not available in Australia on the
Pharmaceutical Benefits Scheme before chemotherapy. Instead of getting best
practice treatment, I have to undergo chemotherapy with its harsh side effects
before I can get access to Zytiga and Xtandi on the Pharmaceutical Benefits
Impact on medical advice
Delays in access to subsidised medicines also pose challenges for
medical practitioners. The committee heard that cancer physicians often
encounter the moral and ethical dilemma of raising the potential of treatment
with particular cancer medicine with a patient who may not be able to access
that medicine in a subsidised form in Australia.
Breast Cancer Network Australia (BCNA) told the committee that past
research indicates that women have not always been told about expensive new
treatments if the medical oncologist thought the patient could not afford it,
for fear of distressing the patient and her family.
Rare Cancers Australia (RCA) raised a similar concern:
... where a drug is not funded for the particular indication
that afflicts the patient, the clinician is faced with less optimal choices,
Prescribe a "second
choice" medication that is funded through the PBS.
Seek compassionate or charitable
access to the first choice medicine through a compassionate program or clinical
trial. This option is not always available.
Present the facts to the patient
and let them decide if they can fund their own treatment.
Deliberately misdiagnose the
patient so that the patient can access the treatment through the PBS for a
funded indication. In these circumstances we are confronting clinicians with
the choice of fraud or inadequate care.
Mr Anthony Steele, Head of Blood Cancer Support, Leukaemia Foundation of
Australia (LFA) said:
Patients are not being told about all available therapies. We
think there should be some sort of onus on health professionals to provide
information on all available therapies. We get stories from patients who are
told that to access a therapy they need to pay for it. They have been prepared
to sell their house and been very devastated by it. In consultation with the
Leukaemia Foundation we have put them through other means of getting access to
the therapies, sometimes free of charge or highly discounted. It was at maybe a
competing hospital or a different centre, across the river or in a different
state. They are not advised of all available therapies. We think patients
should be advised of all available therapies.
Submitters argued that a cancer patient should have the choice of trying
new and innovative treatments, particularly if these have the potential to
offer an improved quality of life. Ms Jolanda Visser told the committee that
she would like to have the option of accessing drugs that may improve her
quality of life or slow the progression of her disease:
I respect the system and also understand that the government
does not want to make drugs available without [them] being thoroughly tested. However,
I also think that we patients should be given a choice. It should be my choice
to try other drugs, as if I am forced to continue to take the medication I am
currently taking, I will also have to face some realities of a diminished
quality of life, progression in my disease which could introduce new risks and
even a shorter life span.
Alternative pathways for access to cancer drugs
Cancer patients have a limited number of options available to them to
gain special or off- label access to cancer medicines that are not available
through the PBS. Cancer patients may receive off-label access via:
compassionate access programs, where a pharmaceutical company may
provide patients with access to new medicines, often free of charge;
hospital formularies, where the hospital agrees to pay for the
treatment for that individual patient;
clinical trials; or
by meeting the associated costs out of private funds.
For cancer medicines that are not listed on the Australian Register of
Therapeutic Goods, the Therapeutic Goods Administration (TGA) manages the
Special Access Scheme (SAS). The SAS provides for the import and/or supply of
an unapproved therapeutic good for a single patient, on a case-by case basis.
The term off-label refers to the use of a medicine in ways other than
specified in the TGA approved product information. Off-label use includes when
a medicine is prescribed or administered:
for another indication;
at a different dose;
via an alternate route of administration; or
for a patient or an age or gender outside the registered use.
The committee notes that off-label prescribing is an integral part of
patient care for many cancer patients. The Council of Australian Therapeutic
Advisory Groups has stated:
In some circumstances, off-label use of a medicine may
represent the best available option for a patient or the standard of care. The
off-label use of medicines allows patients to access innovative and potentially
useful new medicines or older medicines for new indications, dose or routes
based on recent evidence. In patient groups, such as paediatrics, oncology,
psychiatry and palliative care, off-label use of medicines is prevalent and may
provide the only treatment option.
Off-label prescribing is common in paediatric oncology and is not
limited to new medicines. The Australian and New Zealand Children's
Haematology/Oncology Group (ANZCHOG) submitted:
A recent study at the Sydney Children’s Hospital showed that
68% of standard chemotherapy agents were prescribed "off label". During
the same time period over 80% of "new" anti-cancer therapies were
prescribed "off label" (personal communication). The extent of
"off label" prescribing reflects the fact that the levels of evidence
required by regulatory authorities are not available for rare diseases such as
The committee notes that both patients and medical practitioners face a
level of risk in the use of off-label medicines as there are clinical, safety,
ethical, legal and financial issues associated with such use which require
Off-label medicines may have less supporting evidence and have undergone less
scrutiny for efficacy, safety and cost-effectiveness than medicines registered
by the TGA. Prescribers are therefore expected to exercise professional
judgement in prescribing off-label use of medicines.
At the same time, the committee notes evidence that some cancer patients
have a higher propensity for the risks associated with such use. Mr Barrie
Littlefield, Head of Engagement, Cure Brain Cancer Foundation (CBCF) told the
It is fair to say that it is something that is very often
overlooked by the medical community and others. In the vast majority of cases,
people living with brain cancer have a very short time to live. Therefore,
their risk propensity is extremely high in many cases—far higher than their
doctors sometimes realise. They are quite prepared to take extreme risks when
they need to if they think they have hope of a cure. So I think there needs to
be a resetting around the ethical boundaries that are currently being set
around access as well. I think it is well worth considering that.
Compassionate or early access
Compassionate and early access programs are initiated by the sponsors of
a medicine and approved by the drugs or therapeutics committees of
Most compassionate access programs provide access for a limited time or to a
pre-specified financial commitment. MA told the committee that in more than two
thirds of cases this access is used to cover the gap between TGA registration
and PBS reimbursement.
MA referred the committee to research undertaken by Deloitte Access Economics
(DAE), which found that nearly 5000 patients were provided with compassionate
access in Australia during 2011-12 from a sample of nine pharmaceutical
companies. In most cases the access was provided free of charge.
The DAE report states that approximately $10 million of cancer medicines are
provided to patients prior to PBS listing, or even experimentally prior to TGA
approval, through specialist cancer centres.
However, MA noted that the sustainability of such programs is a
significant issue for the pharmaceutical industry, particularly when there are delays
in achieving PBS listing:
...companies are frequently criticised by clinicians, the
Government and PBAC when unanticipated, lengthy delays in listing decisions
mean that ongoing access cannot be commercially sustained indefinitely.
Mr Timothy James, Chief Executive Officer, MA, told the committee:
...we support our members in their efforts to provide access to
new medicines to patients, but we point out that these are not themselves
sustainable access models and should not be performing the role of the PBS.
Key concerns raised in relation to compassionate access programs are that
cancer patients are reliant on their treating clinician to lobby for them to
gain compassionate access to treatment and that such access is not assured.
Ovarian Cancer Australia (OCA) noted that access to compassionate access
program is often subject to strict eligibility criteria, and that even if a
patient is successful in gaining entry, many patients still face significant
While compassionate access schemes do sometimes exist in the
interim period between TGA approval and PBS listing, these are at the
discretion of the drug sponsor and they do not necessarily cover all of the
drug costs. We have seen many instances of patients facing considerable
financial burden to meet the costs of non-PBS listed medicines.
The committee heard a number of personal accounts indicating the
magnitude of the financial burden faced by cancer patients. Mrs Karen Cowley
described for the committee the personal impact of making a significant
contribution to receive the drug Kadcyla
through a pharmaceutical company's compassionate program:
I paid $15,000. I am now on a disability pension and my
husband has just turned 60 and working 6 days a week to make ends meet. It's
too much for him. Going forward we are unable to fund another drug. Even though
I am a skilled professional, I am trying to get part-time work to make ends
meet but being in my late 50s with cancer, difficult to find work. The irony is
that Kadcyla has made me well enough to feel I can work again. I live in hope,
as it goes against my grain to be a pensioner which has been my last option as
our savings are depleted due to my loss of income and 8 years of out of pocket
In some cases, cancer patients must reach a threshold of treatments
before they become eligible for a compassionate program. Mr Chris Brugger, who
is currently taking the drug brentuximab vedotin to treat Hodgkin's lymphoma,
told the committee that his treatment costs his family $16 000 per dose every
three weeks. In the event that Mr Brugger requires more than nine doses, the
pharmaceutical company will supply the drug free of charge:
The drug company has a compassionate program where, once you
get to nine doses, they will supply it after that. There is research on this
drug, in America, that shows it used to be capped at 16 doses but now you can
stay on it indefinitely, as maintenance. My oncology nurses have spoken to the
drug reps and they have said the compassionate program will be fine for me. If
I need to get over nine doses, they said I am a perfect candidate for it,
because I am young and otherwise healthy. I am a perfect candidate for it. If I
were in my 60s or something it probably would not be as good a prospect.
Mrs Lesley Royle's account of her access to the drug Agrylin on
compassionate grounds prior to it being added to the PBS highlights the
uncertainty surrounding a patient's ability to secure private health insurance
coverage to defray out-of-pocket costs:
My Private Health Cover agreed to pay the difference between
the private script price (which was $75.00 at the time) and the actually cost
of the drug. I was prescribed 3 months' supply and my health fund pulled their
funding, leaving me and my husband to pay nearly $2000.00 in pharmaceutical
The committee heard that there has not been a systematic effort to gauge
the ability and willingness of health funds to fund non-listed PBS items. The DAE
report noted that health fund payments appear predominately to be ex gratia
and legislative requirements surrounding the coverage of non-PBS listed
medicines are unclear.
Public hospital formulary
The committee also notes concerns raised about the availability of
cancer medicines through public hospital formularies. Access to subsidised
medicines for admitted public patients in public hospitals is dependent on the
formulary of individual hospitals and in Queensland, the state-based formulary.
The decision to list pharmaceuticals on the formulary of Australian hospitals
is a consideration for the drug committees of individual hospitals or states
The committee heard that as there is no single streamlined process
across institutions and jurisdictions to assess proposed formulary listing of a
medicine, the timeframe of each listing process is variable.
Requests to prescribe drugs outside a hospital's list of approved medications,
such as new anti-cancer therapies, usually involves an application to the
hospital executive or jurisdictional advisory body. 
CanTeen told the committee that varying policies between hospitals and states
can lead to inequities in access:
While clinicians can request that their hospital pay for such
drugs via individual patient usage applications, most public hospitals also cannot
afford such drugs. Moreover, while one hospital may approve an individual
patient usage application for a specific agent, another hospital may not,
thereby creating further inequity of access.
Link HealthCare submitted that funding of cancer medicines through the
hospital system is limited for both in-patient and out-patient medicines and
poses an additional burden on hospital budgets.
Link Health Care illustrated this with a case study of the drug Defibrotide,
used in the treatment of a serious complication resulting from haematopoietic
stem-cell transplantation, hepatic veno-occlusive disease:
Defibrotide is currently only available to patients under the
[Special Access Scheme] and is prescribed throughout the transplantation
centres across Australia. Funding is provided through the hospital budgets and
a three week course of treatment can cost around $40,000 to $100,000 per
patient based on the recommended daily dose of 25mg per kg.
The South Australia Medicines Evaluation Panel, at their
meeting on 14 November 2012, recommended rejecting funding of defibrotide
"due to the number of Individual Patient Use (IPU) requests for this
medicine exceeding the threshold for review as directed under SA Health
ANZCHOG also told the committee that it is not realistic to expect new
and innovative cancer medicines to be funded from hospital operational budgets
and that some hospitals have had needed to fund the supply of such drugs from
Another important potential avenue for early access to new cancer
medicines that have not yet received TGA approval or PBS listing is through
participation in clinical trials. Clinical trials may be sponsored by
pharmaceutical companies or may be initiated by researchers and health professionals.
However, as noted in chapter 3, the committee heard that there are
challenges associated with clinical trials and for a number of reasons
participation in clinical trials is not an option for many cancer patients:
not all new cancer drugs are tested through clinical trials
conducted in Australia;
patients must be referred to hospitals and clinicians
participating in the trial, meaning participation may not be possible due to
the patient's location; and
strict eligibility criteria may mean some patients are ineligible
OCA told the committee:
Most ovarian cancer trials are large international studies
and getting a spot on one of these trials is itself fraught with difficulty.
Australia is often given only a handful of spots at a few hospitals. In
addition, the eligibility criteria are often so restrictive as to rule them out
as an option for most women, if geography has not done that already.
Mr Andrew Warden, who has been diagnosed with Waldenstrom's Macroglobulinemia
(WM), a type of non-Hodgkins Lymphoma, told the committee that he had been
unsuccessful in gaining access to a trial as he did not meet the eligibility
The consensus of leading world experts identifies WM
treatments including IMBRUVICA, Idelalisib, Ofatumumab, Velcade and RIBOMUSTIN.
I do not have access to these treatments. There are Australian clinical trials
(with limited patient intakes) for all these treatments except Ofatumumab which
is only for Chronic Lymphocytic Leukaemia (CLL). My Haematologist late last
year unsuccessfully sought my participation in the IMBRUVICA clinical trial. I
did not then meet the specified criteria as my relapse had not then reached the
stipulated level. The trial is now closing before my condition is within the
defined criteria, so my chance has passed.
A number of submitters called for greater access to clinical trials for
Australian cancer patients.
Mr Anthony Steele told the committee that there is a need to facilitate
increased access to clinical trials by simplifying the process for establishing
trials, establishing a national ethics approval system and increasing access to
Cancer Voices South Australia told the committee that clinical trials
are often undertaken only in limited locations and that access often depends on
patients and families searching out the information and asking clinicians to
refer them to those locations.
The Australian Leukaemia and Lymphoma Group (ALLG) told the committee
that one of the reasons more Australian's are not able to access new drugs via
the clinical trial framework is because the framework is 'slow, unresponsive to
emerging trends, and focused to aid the clinical trial activity generated from
the pharmaceutical industry whereby commercial outcomes of the drug in use is
at the forefront of the development investment'. ALLG argued for better support
for the Australian clinical trial environment, noting that:
Cooperative clinical trial groups, like the ALLG, provide the
fertile ground for patients to have access to drugs that are designed for use
with the patient and their health outcomes as the focus – not the commerciality
of the drug.
ALLG further stated:
If this continues in this way, Australians will only ever
continue to get access to new drugs at the discretion of a company that has
valued and determined access by a commercial gain.
Professor Brendon Coventry, Research Director with the Australian
Melanoma Research Foundation, also noted the need for greater support in
Australia for clinical trials of innovative approaches to cancer treatment.
Professor Coventry, and Mr Martin Ashdown, a Research Fellow in the Faculty of
Medicine at the University of Melbourne, told the committee of their research
into the operation of the immune system and the importance of accurate timing
of cancer treatments. Professor Coventry told the committee:
Martin and I have subsequently identified how the immune
system seems to be working, by switching on and switching off repetitively, and
that when treatment occurs is vitally important. The delivery of relatively
inexpensive agents at the correct time for the patient can have a dramatic
effect on the effectiveness of many therapies for cancers of many different
Mr Ashdown further commented:
Continuing on, this relatively simple approach potentially
offers near-immediate and less expensive opportunity to use cheaper off-patent
existing drugs and some of the newer drugs more effectively. Timing of therapy
seems to govern efficacy and this principle is finding its way into clinical
literature year by year. Regarding the use of accurate timing of therapy,
according to two highly experienced and prominent New York oncologists who have
been reviewing our work, this finding has the potential to dramatically change
the treatment landscape of cancer immunotherapy.
The committee notes that this work has been self-funded and reliant on
philanthropic support. Professor Coventry told the committee that the current
grants system 'does not really serve original research and innovative research'.
The committee heard that a lack of information about clinical trials may
also contribute to inequities in access to clinical trials. For example, the
committee heard that patients may experience difficulty accessing information
about clinical trials for medicines that may be suitable for them if the trials
are not offered at the hospital or oncology unit where they are receiving
treatment. BCNA told the committee:
We know that some women receiving treatment in rural and
regional areas, older women and women from lower socio-economic and culturally
and linguistically diverse backgrounds are poorly represented in clinical
The committee notes the establishment of the Australian Clinical Trials
website is intended to assist patients to be aware of trials available in
Australia and access information about them. The website is also intended to
assist trials to recruit participants.
Private funding, fundraising and
For those cancer patients who have not been able to access treatment
through trials or compassionate access, the alternatives are limited.
The committee heard evidence of patients travelling overseas to receive
treatment at significant expense and personal cost. Submitters noted that the
expenses associated with seeking treatment overseas are 'beyond the
capabilities of most Australian families' and noted the associated emotional
and social burden.
While the Australian Government funds a Medical Treatment Overseas
Program for proven therapies not available in Australia, the committee heard
that treatments on a clinical trial are specifically excluded 'presumably
because by definition, if a therapy is still undergoing a clinical trial, its
efficacy is not yet proven'.
The committee heard that many cancer patients rely on the generosity and
fundraising efforts of family, friends, colleagues and the community to finance
the cost of their cancer treatment.
While submitters expressed their gratitude for this support, they noted the
personal impact fundraising has on cancer patients and their families.
Mrs Naomi Brugger told the committee:
We are fundraising at the moment. We have managed to raise
close to $60,000 since January. That has been hard going. I have run the
campaign mostly by myself, so that means I have been away from the boys as
well. So they are not only missing out on dad; they are also missing out on
mum, who is trying to keep dad alive.
RCA told the committee that it has established a charitable Cancer
Medicines Fund to address what it sees as profound inequities in the current
In its submission, RCA provided the committee with two examples of patient
experience to illustrate how these inequities can arise:
The first of these examples, Anita, has been diagnosed with
non-small cell lung cancer and has been diagnosed as having an ALK+ genetic
mutation as a contributing factor. Her oncologist prescribed a drug called
Crizotinib and Anita has responded well for a number of months. Crizotinib has
been recommended for listing by the PBAC for Anita's cancer but as the
contractual process unfolds, it may yet take some months for it to be listed.
In the meantime our Cancer Medicines Fund continues to fund her treatment at
$7,400 per month.
Our second patient is Lillian who has also been diagnosed
with non-small cell lung cancer but in Lillian's case her much rarer mutation
is in the ROS1 gene. Her highly respected oncologist has also prescribed
Crizotinib as there is substantial evidence of benefit. Lillian is also
responding well but because her cancer or indication is so rare there is
currently no application to PBS for re-imbursement.
Hence we face a situation where both Lillian and Anita need
to self fund today at a cost of over $7,400 per month yet simply because of the
random genetic mutations they have, Lillian will never receive funded medicines
through the PBS whilst Anita hopefully will.
Same cancer, same treatment but no fairness.
The fund, which has been established under the campaign banner, is
supported by fundraising, corporate support, public donation and events and
campaigns under the 'Sick or Treat' banner.
RCA told the committee:
That we needed to establish this site says everything we need
to say about the current state of cancer medicines in Australia.
Impact on rural patients
The committee heard that cancer patients living in rural and remote
areas frequently suffer greater challenges in accessing cancer treatment. MA
told the committee:
There is commonly a lack of easily accessible diagnostic and
treatment services in rural areas where treatment services are often
rudimentary comparted to large urban centres. They will also likely lack access
to current research and clinical trials, which are commonly conducted in larger
urban centres. These factors contribute to later diagnosis; diagnoses at more
advance stages of disease; and higher mortality rates.
This is of particular concern for patients with rare or less common
cancers, as rural centres are not equipped to treat less common cancers. The
Unicorn Foundation, which assists and supports patients with neuroendocrine
cancer, submitted that 40 per cent of neuroendocrine cancer patients live in
Medical Oncology Group of Australia (MOGA) and LFA expressed concern
that cancer patients receiving treatment outside major treatment centres, such
as those from regional and rural areas, may also experience difficulty
accessing compassionate and early access programs and clinical trials.
LFA told the committee :
In our survey, of the
patients who accessed a new drug, 17% had to relocate for treatment at their
own cost. However, none of the State or Territory Patient Assisted Travel
Schemes (PATS) provides a subsidy to cover rural, regional or remote patients
to travel to a metropolitan hospital to take part in a clinical trial. Even if
the clinical trial covers the cost of the drug, rural, regional and remote
patients must bear ongoing accommodation and travel expenses as well as the
cost of other medications. It means accessing new drugs through clinical trials
is an inequitable, user-pays system that penalises non-metro patients and their
LFA underscored this evidence with the following four patient case
studies, which demonstrate the additional challenges faced by rural and
regional cancer patients seeking to take part in clinical trials:
Patient case study 6:
I made the decision to move from Canberra to Melbourne, where
I had no family, to avoid having to commute regularly to receive the drug
(which had to be given intravenously as part of the clinical trial program).
This was a very hard thing to do, but looking back it doesn't seem there really
was any other option for me at the time.
Patient case study 7:
My wife and I had to live in Melbourne for 14 weeks while
undergoing the treatment, which wasn’t available in our regional centre, 300 km
from Melbourne. Our first month cost $5550 for accommodation. Thereafter we
were lucky enough to gain access to Leukaemia Foundation accommodation, which
meant a saving in the order of $10,000.
Patient case study 8:
My treatment regime involved two consecutive days each four
weeks. It was too far (and way too exhausting) to travel to and from home two
days in a row so I stayed in a motel located near the hospital. It was pretty
lonely going back to a motel after an exhausting day of treatment, monitoring
any symptoms and then going back in a taxi the next day to do it all again.
Patient case study 9:
I'm taking part in an international clinical trial. This
requires driving for treatment every two weeks to Gosford from Tamworth. While
in Gosford I live in in rented accommodation. Fortunately, our children are old
enough to be self-sufficient and my wife's still working, so we’re able to pay
for the costs from her personal savings. So far, the treatment's been
The committee notes that for families of young cancer patients the only
option may be to relocate the whole family to be nearer to treatment and
To undergo this intense treatment schedule, Zoe's family had
to leave their farm – 18,000 acres of mixed cropping and livestock – and
relocate to Perth to be closer to the Princess Margaret Hospital for Children.
Zoe's older sister...and little brother...(who was born just a few months after
Zoe's diagnosis), together with her mum...and dad...fought with Zoe all the way.
Roche Products submitted that the potential benefits for regional and
rural patients of new forms of cancer therapy should be accorded greater value
in the assessment process. For example, Roche Products stated that 'oral and
sub-cutaneous forms of intravenous cancer therapies may be more easily used
outside of major metropolitan hospitals and may support patients completing
their full course of therapy'.
Mrs Jill Delahoy's experience taking the drug Ibrutinib as part of her
treatment for Chronic Lymphocytic Leukaemia also highlights the impact some new
cancer medicines can have on both the quality of care and quality of life for
This drug comes in capsule form and is taken by the patient
at home. This means no hospital admission required, either for day chemotherapy
or by admission. My haematologist is of the view that in a short time this will
become the normal treatment for CLL patients. Imagine what this means for rural
residents, no going to hospital which may be many hours away at a time when the
patient is feeling dreadful. This should reduce costs and pressure on the
Impact on patients with rare cancers
The committee heard that, as research priorities, commercial imperatives
and advocacy tend to focus on oncology medicines and treatments for more common
cancers, the impacts of a lack of access to subsidised cancer medicines has a
disproportionate impact on the quality of care for patients with rare and less
common cancers, for whom there are
often few treatment options.
The evidence base for rare cancers, which have small patient
populations, is more likely to have some level of uncertainty.
Clinical trials for rare cancers are often conducted through collaborative
trial groups with less industry support and the data collected may be less
suited to registration and reimbursement requirements.
Dr Christopher Fraser, Chair of ANZCHOG, told the committee that most
drugs for rare cancers, which includes most childhood cancers, are not covered
by the PBS and are not likely to be:
The problem is, to get to that PBS approval, the data is
almost never going to be available to prove clinical and cost-effectiveness in
extremely small patient groups, and the economic incentive to try and collect
the data is not going to be available. The drug company is going to say: 'Let's
apply for a listing for breast cancer.' They are not going to be worried about
a rare liver tumour in children. At the moment in our system we have to beg our
hospital executives to pay for these drugs, and they may or may not approve it.
There are inequities amongst different state thresholds. For rare diseases,
like just about every childhood cancer, we feel there needs to be an improved
mechanism that assesses the eligibility for federal funding or a federal
subsidy for these drugs that does not rely upon extremely stringent clinical
and cost-effectiveness data and that is not subject to the vagaries of
individual jurisdiction or approval.
In its submission, the Department of Health noted the difficulties
associated with funding for treatments for rare cancers:
Making judgements about the level of support for rare cancer
patients is especially difficult, noting that it involves spending significant
amounts of taxpayer dollars on a very small, but very sick, sub-group. The
trend in increasingly expensive, personalised medicines will continue to place
pressure on both the family and national budgets.
As Ms Lee McKerracher submitted, an individual's access to medicines
should not be dictated by the type of cancer they have or their financial
For those diagnosed with a rare cancer, they are in a
different position. They are not lucky like I was and have ready access to a
range of therapies that are reimbursed. So in addition to the emotional stress
they are under, these people need to try and fund treatments to get well. These
treatments can be extremely expensive and many can't afford them. Access to
medicines should not be dictated by the type of cancer someone has, nor the
income they earn. These people do not put their hands up to get cancer, they
are a victim of circumstance and should not be discriminated against just
because their disease is rare.
A number of submitters argued that alternative approval and funding
pathways are required to address the particular challenges faced by patients
with rare cancers. For example, LFA proposed that consideration be given to
automatic conditional acceptance of treatments for rare cancers that have been
approved in the United States and Europe. Mr Steele told the committee:
We know there are some life-saving drugs that may be
available to rare cancers but are not brought into the country because it is
not profitable. We would like to have some other methods to access those drugs
for the non-profitable therapies to the rare cancer groups.
The account of Mr John Canning summarises the sentiments of many
submitters. Mr Canning has inoperable metastatic stage 4 lung cancer. Despite
his prognosis, Mr Canning describes himself as mentally and physically fit. Mr
Canning has a rare genetic mutation known as anaplastic lymphoma kinase and is
being treated with the drug crizotinib:
What does this mean for me? I actually do not feel like a
patient. I can take this drug orally, in the form of a tablet twice a day
wherever I am, whether I am travelling or out at dinner with family and
friends. If I was on the current PBS listed alternative pemetrexed, I would
have to book in for an infusion at a public hospital or cancer centre every two
to three weeks. The side effects of the targeted therapy that I am on have been
proven and shown to be much milder and much more manageable. I have some
control over my life as a cancer sufferer. I have quality of life. For me and
for patients like me, that is what it is all about: quality of life.
There is no plan B. At this point in time, I do not have
other options. I am still working, albeit fewer hours. I continue to carry out
the charitable and pro bono work that I have done during my professional
career. It means an enormous amount to me emotionally, psychologically and
physically, but it makes an enormous difference to my wife and my family, who
are my primary carers.
There is a challenge with all this because, although
crizotinib was approved at PBAC's November meeting, it is still not listed on
the PBS. It is not reimbursed. It is an expensive drug. I am one of very few
people in this community who can afford to purchase this drug. For every one of
me, there are 30-plus people who cannot. There are currently around 340 people
with my cancer in Australia today. There are only around 40 who are likely to
be diagnosed each year. What we are after is a fair access scheme for people
with rare cancers. It is not an expensive access scheme but it is one which
means a truly remarkably different experience than the brutal, debilitating and
exhausting process of normal cancer chemotherapy treatments.
Evidence to the committee indicated a move toward more personalised use
of existing medicines in the treatment of rare cancers. For example, CBCF told
the committee that it is tackling the paucity of medicines to treat brain
A significant, increasingly
global, research collaboration, to accelerate new treatment options for
A concerted move towards a
'personalised medicine' approach, whereby tumour genetics are established early
on and high-throughput screening of existing medicines (many of which are
currently PBS listed for other indications) occurs. If any of the screened
drugs show activity against an individuals' tumour, then this information is
conveyed to the treating oncologist (and hopefully patient) to be used for
(most likely off-label treatment.
As noted in chapter 2, commercial realities often mean that
pharmaceutical companies do not seek registration of cancer medicines for rare indications
with small patient populations. Rare Cancer Australia (RCA) told the committee:
The potential outcome is that today we have medicines registered
with the TGA for one indication meaning they are deemed safe and for that
indication also efficacious. However, their utilisation for other indications
is prevented simply by the lack of application for registration.
RCA expressed the strong view that where a medicine is already
registered in Australia for one indication, clinicians/and or patient groups
ought to be able to apply to the TGA to extend the registration to additional
indications where reasonable evidence of efficacy exists.
Impact on children, adolescents and young adults
The committee notes that the concerns raised above are amplified in the
case of young cancer patients. The committee also received evidence that
children, adolescents and young adults (AYAs) diagnosed with cancer face unique
challenges in accessing new, innovative and specialist cancer drugs.
The committee heard that childhood cancers are different from adult
cancers. While the most common form of adult cancer is carcinoma, this form of
cancer constitutes less than three per cent of paediatric cancers. ANZCHOG told
Consequently, the molecular targets seen in childhood cancers
are often different to those in adult cancers. However, currently the approach
taken to applying targeted therapy to paediatric oncology is to see which drugs
being developed for adult cancers might have some activity in children. The
paediatric oncology community would argue that children deserve their own drugs
developed specifically for their diseases. The explosion of new agents, a
relative lack of pre-clinical research and the small numbers of paediatric
patients makes prioritisation of agents for clinical trials extremely
CanTeen submitted that AYAs face exceptionally difficult cancer journeys,
noting that the number of young people aged between 15-24 years is higher than in
the younger age groups. AYAs present with a greater range of cancer types and,
due to delays in diagnosis, the prognosis for AYAs is not as good:
The number of young people aged 15-24 years diagnosed with
cancer is 1.5 times the number of children aged 0-14 years that are diagnosed.
Young people have significantly poorer survival rates than children and older
adults in some of the cancers common in this age group. Many of the cancers
that affect young people are rare. Young people also present with a larger
array of cancer types compared to older adults: 90% of the cancer burden is
accounted for by 20 different cancer types. Furthermore, young people also tend
to present with cancer at a more advanced stage due to longer delays before
diagnosis and suffer higher rates of inferior psychological outcomes compared
to other age groups. This in turn, is associated with poorer prognosis and a
heightened risk to survival. Consequently, for some cancers, young people show
a much poorer response with the same treatments given to older adults or
Similarly, AYAs encounter disproportionate difficulty in accessing new
and innovative cancer medicines. There is a lack of clinical trials for the
cancer subtypes commonly seen in AYAs because pharmaceutical companies do not
usually devote the same research effort to rare diseases. CanTeen noted that,
unlike the United States and Europe, where legislation has been passed
encouraging pharmaceutical companies to develop drugs with paediatric
indications, Australia has not provided a legislative incentive for
pharmaceutical companies to seek the PBS listing of indications relating to
cancers in children and adolescents.
One young submitter told the committee:
There are no drugs for kids like Eva to try and save their
life. The big drug companies need help from the government to find better ways
to help kids get new drugs. The government needs to help out scientists find
drugs for little kids so they don't just have to use drugs for old people. Kids
are different and the government needs to treat them differently to grown up
The Kids Cancer Project submitted:
Australia now needs to develop a policy for incentives and/or
require industry to participate in the supply of drugs for childhood cancer and
eliminate inequities. The fear is that Australia will not keep pace
internationally with improvements in survival rates and children and families
will continue to seek international options.
The rarity of the cancers that affect children and AYAs means it is
extremely challenging to conduct trials necessary to satisfy the requirements
to achieve subsidised funding through the PBS.
It is not economically attractive for pharmaceutical companies to invest in
drug development for childhood cancers:
The success of frontline therapy and the fact that new
therapies target smaller sub-groups of already rare diseases means that there
are fewer patients eligible to test these therapies and makes the conduct of
trials to prove clinical effectiveness extremely challenging.
While noting the dramatic improvement in survival rates for children
with cancer 'from less than 30% in the 1960s to 80% currently', ANZCHOG states
that the most important factor in this improvement, has not been access to new
cancer medicines, but global collaboration in incorporating clinical research
as an essential part of the care of children.
The committee heard that all of Australia's major paediatric hospitals have
access to international trials and it is estimated that more than 50 per cent
of Australian children with cancer will participate in a US or European
originated children's cancer clinical trial.
However, meeting the costs associated with treatment of childhood
cancers poses significant challenges for hospital operation budgets. ANZCHOG
told the committee 'most Australian paediatric oncologists would be very
reluctant to ask a family to pay for a high cost therapy themselves because of
the ethical challenges that this presents'.
Reliance on childhood cancer clinical trials based and funded in the
United States and Europe is also not without its complications. Unlike adult
trials, which are often initiated by the pharmaceutical industry, childhood
cancer clinical trials are initiated by organisations such as the United States
based Children's Oncology Group and the European based Consortium for
Innovative Therapies for Children with Cancer funded by the United States and
European governments. The committee heard that Australia's access to some
childhood cancer clinical trials funded by the Children's Oncology Group is
being limited due to budgetary constraints.
The committee also heard that there can be difficulties associated with
children under the age of 18 gaining access to trials. CBCF noted that children
are often not allowed to enter trials because the medicines must be tested on
CanTeen argued that there is no valid biological justification for
age-eligibility criteria and told the committee that the practice has been
criticised internationally by AYA cancer advocacy groups.
The Kids Cancer Project told the committee that decisions to register and
subsidise drugs for use in adults but not children can also lead to inequities.
For example, the drug clofarabine, used in the treatment of acute lymphoblastic
leukaemia, is subsidised for use in adults but not in children. The annual cost
of treatment for a child can be as much as $100 000 compared to $37 for an
Finally, as noted earlier, many childhood cancers are rare and patient
populations are small. This lack of a commercial incentive can hamper the
development of trials to test the effectiveness of drugs used in the treatment
of adult cancers in treating the types of cancers seen in AYAs. For example, CanTeen
said Ruxolitinib, a drug developed for older adults with myeloproliferative
disease has been reported to be effective in some AYAs with Philadelphia-like
acute lymphoblastic leukaemia. Despite the apparent effectiveness of this drug
in treating this disease, CanTeen stated it is unlikely that the PBAC will
approve the indication without clinical trials and there is currently no
incentive for pharmaceutical companies to undertake trials in such a
comparatively small market.
The committee notes that for cancer patients and their families,
maintaining a normal life and enhancing the quality of that life is of utmost
importance. The uncertainty and significant financial cost associated with
off-label use of cancer drugs results in significant physical, emotional and
financial stress associated with the uncertainty of securing that access. As Ms
Robyn Lindley noted:
You can't work, your husband needs to work less to help so
his income is lower. Then you also have the expenses of pharmacy products to
counteract side effects. So where do we get this money from? You're so worried
about surviving and beating this dreaded disease and wondering if you are going
to be here for your children to then add another huge stress of MONEY.
The committee understands that often the patients with the highest
medical need are also the patients with the least capacity to fund their own
Many patients are already experiencing financial difficulty and do not have the
capacity to meet significant out of pocket costs. Access to new treatments
through clinical trials and compassionate programs is by no means certain, and
in the case of the latter, can involve a significant contribution on the part
of the cancer patient. The burden is even greater for those cancer patients
with rare or less common cancers, particularly children and young adults, or
who live in rural and regional Australia, who have fewer treatment options to
Special access schemes, compassionate access programs and clinical
trials are an important source of assistance, but help only a small minority of
cancer patients and are neither sustainable nor equitable alternatives to
subsidised access through the PBS. Over reliance on such schemes also risk
creating a two tiered health system in which some cancer patients can afford
treatment and others cannot.
The committee is particularly concerned about the impact of delays in
access to innovative cancer medicines to treat patients diagnosed with rare and
less common cancers, including children and young people and those with brain
cancers. Proportionally, research in these areas of cancer research is less
well supported than other areas of cancer research, while at the same time
expenditure on treatment for these forms of cancers is much greater. For
example, the committee notes that treatment of brain cancer costs more per
person than any other form of cancer, yet receives only a small fraction of Australian
Government cancer research funding.
In this context, the committee recognises the importance of supporting
innovative research that may lead to more effective use of existing cancer
treatments with consequential savings in the longer term. The committee notes
that work described by Professor Coventry and Mr Ashdown in relation to a
locally produced vaccine to treat advanced metastatic melanoma highlights the
potential for the delivery of relatively inexpensive agents at the correct time
for an individual patient to have a dramatic impact on the effectiveness of
many therapies for many different types of cancers.
The committee considers the long-term implications of research of this
type merit further investigation. Similarly, the committee considers that there
is merit in considering the extent to which legislative and other incentives
implemented overseas might have application in an Australian context to
encourage greater focus on the development of cancer treatments for rare and
less common cancers.
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