Consequences for patients
Listing new therapies on the PBS is
a key investment in the health of Australians and not a mere cost. These are
therapies that have been shown to meet a clinical need and improve health
outcomes, for example, increase survival or greater quality of life, often
saving costs elsewhere in the healthcare system. Another benefit is that these
medicines increase the offering of therapies to meet individual patient needs,
which otherwise would not occur if there was a 'one size fits all approach' to
managing illnesses in Australia based on few PBS listings...people in need of
new medicines recommended for listing by the PBAC on the basis they are
effective and a worthwhile investment of taxpayer money should not have access
denied due to (indefinite) PBS listing deferral. Such delay may potentially
create a two tiered system whereby only those able to pay fully for new non-PBS
listed medicines can access them, while many Australians with serious illnesses
are denied access. Is this what Australians want?
The PBS is a fundamental investment in Australians' health,
and is not a cost containment tool to help manage the Federal Budget back to
The effects of the Government's decision to defer the listing of medications
on the Schedule of Pharmaceutical Benefits (the Schedule) on patients,
particularly in relation to the affordability and accessibility of medications,
and the associated repercussions for individual and public health were made
abundantly evident throughout the committee's inquiry.
Evidence received by the committee indicates that the Government's
deferral decision is likely to result in an increased financial impost on
patients who may already be struggling to pay for existing medicines, and as a
result, unsubsidised medications may be inaccessible to a proportion of
patients, resulting in inequitable access to the most appropriate treatment. Further,
inability to access appropriate treatment entails risks not only to the health
of individuals, but also to public health, and will result in further strain on
the health system.
The Consumers Health Forum of Australia (CHF) informed the committee
that it has seen an unprecedented level of concern from consumers about the
changes to the Pharmaceutical Benefits Scheme (PBS) listing process. Following
the deferral announcement, the CHF undertook a survey of the views of
consumers, and 95.1 per cent of respondents indicated that they are concerned
about the Government's decision.
Ms Liliana Bulfone of Deakin University commented that the long-term
effect on patients who are unable to access deferred medicines may not be
known, but stated 'to say that they are not disadvantaged I think is wrong'.
Submitters noted that the PBS plays a central part in ensuring the
affordability of medicines. Without medicines being listed on the PBS, many
appropriate medications would be out of the financial reach of patients.
The Council of Social Service (COSS) Network noted that many low-income and
disadvantaged Australians already struggle financially to access medicines
which are subsidised under the PBS.
National Seniors Australia provided similar comments and noted that there are
some older Australians, including concession card holders, who defer buying
subsidised medicines because of the co-contribution they have to make.
It was noted that medicines are only one of the financial burdens
carried by patients. Submitters commented that patients also face out-of-pocket
expenses for tests, surgery and other medical procedures, loss of income, and
travel expenses, especially for patients from rural and regional areas.
The Chronic Illness Alliance noted that those with chronic illness
already spend a large proportion of their household income on medicines which
are PBS listed, and often go without other essential items in order to afford
their medicines, as health expenditure is rarely discretionary.
This was demonstrated by a survey undertaken by the Chronic Illness Alliance in
2003 of people in regional Victoria with chronic illness. The survey found that
households with chronic illness in rural and regional Victoria will pay for
their health needs regardless of income, and spend more on PBS and
over-the-counter medications than any other health-related item. However, as a
result, these families experience considerable poverty and financial distress,
and will go 'without other essentials such as food, heating, family holidays
and recreation and clothing in order to pay for essential medicines'.
It was also noted that those with mental illness also find it difficult
to find and retain employment and are often on disability support or are
homeless and therefore under financial distress.
Multiple Sclerosis (MS) Australia also commented on the vulnerability of
those with chronic conditions to shifts in government policy:
People with chronic illnesses that are reliant on the health
and welfare system are far more vulnerable to shifts in public policy than
other Australians and have fewer options in being able to adapt to new
arrangements – particularly if they have negative consequences (such as reduced
access to supports, higher levels of compliance or higher costs).
Their lives are already compromised in terms of employment,
community participation and overall quality of life, and decisions such as the
deferrals are a visible reminder of their vulnerability.
The Government's decision to defer the listing of medicines was
characterised as 'a form of cost-shifting' to consumers. Concern was voiced
that consumers, some already burdened with high health-related costs, may now
have no option but to purchase medicines which are not listed on the PBS in
order to obtain the treatment most suitable for them or to go without the
medicines altogether. GlaxoSmithKline Australia (GSK) stated:
Continuing deferral of PBS listing for medicines transfers an
increased proportion of the costs of important medicines from the Government to
the patient. This will force some patients to make difficult choices about
their medical care according to their capacity to pay.
This was illustrated by evidence provided by Mr Robert Pask, who
informed the committee that as a person with multiple sclerosis, he spends over
$250–$300 per month on medications. Mr Pask also suffers from narcolepsy but is
not in a position to afford the approved anti-fatigue medication for this
condition which would cost an extra $300 per month:
I am not in a position to afford it and I am lucky enough
that I can get through most days without falling asleep. When you add that on
you can be looking at $600 a month.
It was strongly argued that any decision which results in cost shifting
to patients is unacceptable, as Ms Carol Bennett of CHF explained:
The issue is that it certainly does create huge barriers to
access to treatment for the most disadvantaged people—the people who have
chronic conditions who cannot work full-time because they are ill and simply do
not have the income to purchase these medicines when they are not subsidised.
We hear stories all the time about people having to make really difficult
choices about whether they buy a medicine or whether they put food on the
table, pay their electricity bill or whatever—and this is for when drugs are
subsidised. When drugs are not subsidised, it puts them out of the reach of
Further, if approved medicines are not subsidised under the PBS,
submitters argued that patients, as taxpayers, are in effect paying twice for
Patients denied PBS access to such cost effective medicines
because they have been deferred, who choose to pay the full cost themselves,
will in effect be paying twice for their medicine.
The committee received direct evidence of the impact of the cost of
medicines not listed on the PBS on consumers. Ms Elizabeth Graham submitted:
The deferral of the listing of these medications means the
burden of cost remains with patients who need them. In my own case, the monthly
cost of treatment by medication is considerable; the need to continue with an
efficacious medication which is unlisted adds to my burden. While I can afford
it, I will continue with its use, but fear the time will come when my financial
situation will make it necessary to change to a PBS listed medication. Should
this scenario become reality, I will be forced to use other medications to
counteract the side effects of the PBS listed medication; this is a situation
of false economy as additional medications will be those listed on the PBS. I
am certain I am not alone in facing this situation.
The COSS Network argued that as a result of the decision to defer
listings, inequitable access to medicines for low-income and disadvantaged
Australians will be exacerbated.
This point was illustrated by one of the respondents to the CHF survey who commented
'I am one of the people who need some of these new drugs, and being on a
pension I cannot afford them unless they are on the PBS'.
Submitters and witnesses also argued that the deferral decision may
result in a two-tiered system in which the most appropriate medicine for some
patients will be beyond their financial reach, and will only be accessible to
those with the financial capacity to purchase unsubsidised medicines:
My concern is that, with that sort of a decision, we are
heading more down the path of a two-tiered health system, where if you are
wealthy you can afford the more convenient treatments. I know there are
arguments about compliance, injections and once a month or twice a month—there
are arguments about those sorts of things—but if you are wealthy you can afford
to get them. If you are not, you have to rely on what is on the PBS. My concern
is that, by going down this path, you are increasingly heading to a stage where
all the older and less effective or less convenient and cheaper stuff is on the
government scheme, but there is a range of treatments that are newer, better
and can be more convenient for patients, and it will be the wealthy who will be
able to access those, not the less wealthy.
Although many consumers do not choose to purchase unlisted medicines,
for those that do, the financial impact is large. Mr Brian Stafford commented
that paying for the most appropriate medicine to treat a condition when that
medicine is not listed on the Schedule can have significant financial ramifications
for patients and their families:
At the outset of the illness we owned 3 houses. By the time
of the patient's death all the properties had been sold in order to pay our
bills over the period of the illness. Not only is it the cost of expensive
medications it is the years of lost earnings from work for the person who has
to give up paid employment in order to become a full time carer. I now live in
a rented house without sufficient capital left to buy any home for myself.
However, my family member who was ill for some 10 years received the best
medical care and medications because they were needed and they worked.
Pfizer Australia further submitted that even if a patient is financially
able to purchase unlisted medicines, these medicines may not always be
available if they are not listed:
Even if the patient is able to self fund there is no
guarantee a medicine will be available privately as the manufacturer may not be
able to support the medicine without PBS subsidisation. Furthermore, if a medicine
is not listed on the PBS, this may affect availability in hospitals.
Diabetes Australia also pointed to other costs to consumers of the
deferral of medicines: if the best medication for a condition is not available,
the consumer may face escalating healthcare costs as effective treatment is
Mr Jose Vieira of AstraZeneca Australia also addressed the costs of the
deferral to consumers and argued that sometimes the alternative medicines can
also offer patients cost-savings in terms of their treatment.
This point was illustrated with reference to Symbicort®, provided by AstraZeneca Australia for the
treatment of chronic obstructive pulmonary disease (COPD), and the alternative
currently available under the PBS:
Each pack of Seretide® contains sufficient doses to provide
for one month's worth of therapy for COPD. In contrast, each pack of Symbicort®
contains sufficient doses to provide for two month's worth of therapy for COPD.
Thus, over the period of a year, a patient receiving Symbicort® for COPD will
pay for 6 prescriptions (with each pack of Symbicort® lasting 2 months). By
comparison, a patients receiving Seretide® for COPD will pay for 12
prescriptions (with each pack of Seretide® lasting one month). As can be seen
from Table 1 below, the net result is that patients pay twice as much for
treatment with Seretide® for COPD as compared to Symbicort®.
It should be noted that an increase in patient co-payments
means that patients contribute more to the cost of their medicines.
Subsequently, because a COPD patient pays less for Symbicort® (compared to
Seretide®), Government pays more. This aspect was the key motivation for the
decision to defer the PBS listing of Symbicort® for COPD, which suggests a
preference to shift costs from the Government to patients.
The Northern Territory Government Department of Health submitted that it
has a mechanism in place to provide affordable access to essential medicines
which have not yet been listed under the PBS:
The Department currently has a policy to facilitate access to
essential medicines where the cost of medicines would otherwise serve as a
barrier. Under this arrangement, medicines that are deemed to be essential by
medical specialists but are not covered by the Australian Government subsidy
arrangements will have the costs met by the Department.
Where a medicine is initiated while it is still under
consideration for PBS listing, the Department meets the full cost until such
time as it is listed...This effectively provides a safety net for limiting the
consequences to patients of such deferrals.
Access to medicines
Many submitters were of the view that the Government's deferral decision
jeopardises access to alternative medicines for Australian health consumers,
and also increases the delay patients experience in accessing affordable
treatments which have been already assessed as cost-effective by the Pharmaceutical
Benefits Advisory Committee (PBAC).
Access to alternative medicines
Submitters argued strongly that patients should have access to the
latest and most effective treatments and the medicines which best suit their
individual needs. A medicine which is an alternative to existing medication,
which does not have unacceptable side-effects, should be available on the PBS.
Dr Simon Fisher from AstraZeneca Australia provided the committee with an
example to illustrate why having access to alternative medications is important
I wanted to talk to you about one elderly gentleman by the
name of George who has chronic obstructive airways disease. I treated him with
the currently listed PBS medicine. He unfortunately had an adverse experience
on that medicine and he came back to see me because he had stopped taking the
medicine and become much more short of breath to the point where his activities
of daily living—washing, cleaning, walking down the street—were now impossible.
So I looked for an alternative medicine, in a hypothetical today situation, on
the PBS and I realised there is no alternative medicine for him, despite one
medicine being TGA registered for the treatment of chronic obstructive
pulmonary disease and in fact recommended by the PBAC. That is the medicine
that AstraZeneca sponsors, Symbicort. I would be unable to use Symbicort for
George because it is not listed on the PBS as a consequence of this policy.
Therefore, I would have to put George in an ambulance to take him to hospital
where he would be admitted, investigated, treated and stabilised for his
chronic obstructive pulmonary disease. That is the doctor-patient interface
consequence of the deferral policy. It is a direct negative effect on patients.
Submitters argued that deferral of listings also serve to restrict
'clinical options to prescribed medications' which in turn reduces health outcomes.
Ms Anna Wise, CHF, explained, in relation to Invega Sustenna®, that:
...with psychiatric medications it can be so tremendously
difficult to find one which actually works for a person. They can have to try
so many that to have an option which is clinically effective and cost effective
not made available to treating clinicians could make a real difference to so
many mental health patients.
This argument was supported by GSK Australia, which submitted:
The first medicine in any therapeutic class to successfully
enter the market and achieve listing on the PBS is not necessarily the best and
it is essential that clinicians have access to a range of treatment choices
where these exist and are proven to meet the cost effectiveness criteria for
listing on the PBS...the deferral of PBS listing for medicines is a barrier to
healthcare and reduces the capacity of treating clinicians to make the best
treatment choice for their patients. Instead of making a decision based on the
clinical requirements of the patient, a doctor must also make a prescribing
decision that takes into consideration the affordability of a medicine for that
One of the respondents to the CHF survey who was concerned about the Government's
deferral decision commented, 'It seems the Government is limiting access to
medicines that a lot of people will need and may not be able to afford, and
changing the goal posts to do it'.
Inequitable access to medicines
A further matter raised by witnesses was the potential for increased
inequity in access to medicines. It was argued that small patient groups, for
example, those with brain tumours, risk becoming more disenfranchised. If the
listing process becomes politicised and more open to the influence of groups
with more political presence and influence, medicines for small patient groups
may not receive equal consideration:
...the loudest groups, the most resourced groups or companies
that are the most able to get the ear of government may well end up getting
their drug listed on the PBS versus a small, niche-market drug for a group of
consumers who may not have the same public profile or benefits to government
that may be delivered from the listing of that drug. It creates a real concern.
It was also argued that inequity can arise within patient groups because
of delays in listing. Ms Liliana Bulfone of Deakin University used Invega
Sustenna® as an
example of how the Government's deferral decision 'introduces inequities' in
...if you are a patient who responds to Consta, which is the
drug that was the comparator in this particular case, you have access to a
subsidised drug, but if you are a patient who does not happen to respond to
that drug or who has adverse events, you do not have the same equity of access
to an equivalent drug, a drug that is as cost effective and as effective.
While it is generally agreed that not all medicines can be listed and
there will always be patients who won't have access to various medications
under the PBS, Dr Brendan Shaw of Medicines Australia stated that in the
past listings have been based on clinical effectiveness and health economics.
The concern with the current situation is that patients will have their access
to better medicines delayed for fiscal reasons as opposed to health economic
Dr Fisher of AstraZeneca Australia further explained that pharmaceutical
companies are trying to facilitate patient access to medicines, but that the
deferral decision impedes patient access to treatment:
We as an industry are dedicated to maintaining that access
and in no shape or form would we or I ever wish to consider withholding that
access or slowing it down. But that is what this policy does.
Delay in access to medicines
A key concern raised by submitters is that due to the deferral of the
listing of these medicines, patients are experiencing a delay in accessing
valuable and cost-effective treatments, which have been assessed and approved
by the PBAC.
Cancer Voices Australia (CVA) cited the example of Erbitux, a medicine
which can extend the lives of those with late stage bowel cancer. Erbitux was
deferred by Federal Cabinet in July 2010. In June 2011 the Minister for Health
and Ageing announced that it would be listed from 1 September 2011,
however, in the interim, this decision has left many patients unable to access
Erbitux for over 15 months.
A number of submitters also raised concerns with the delay in affordable
access to essential medicines caused by deferrals, which in some cases remains
indefinite, as there has not yet been any indication as to when, or if, those
medicines which remain deferred will be reconsidered.
CHF explained that these concerns do not only relate to the medicines
which have already been deferred, but to the uncertainty about which medicines
may be deferred into the future:
Consumers now face even greater uncertainty about when they
will have access to the latest, most effective medications, as even after a
positive PBAC recommendation there is a risk that Cabinet will again decide to
defer listing of some drugs.
Mr Matthew Pitt of the Brain Tumour Alliance of Australia (BTAA) noted
that while no specific brain tumour medicines have as yet been deferred, the
possibility that they could be, and that access to new treatments might be
restricted, is very concerning:
...given that we are so starved for advances in treatment,
the prospect that one day an effective treatment could be deferred absolutely
scares us to the core. It is very worrying.
Ms Helen Tyrrell noted that constituents of Hepatitis Australia are now
concerned about whether they will be able to access new treatments which are
due to be considered by the PBAC in the near future:
There are two new hepatitis C treatment medicines that are
due to be considered by PBAC in the immediate future. In clinical trials, the
addition of either one of these drugs has resulted in significantly improved
cure rates compared to the current standard treatment.
The February 2011 decision to defer PBS listings has created
nervousness amongst our constituents. Those who were delaying treatment until
the new hepatitis C therapies became available are now wondering if they should
start treatment with therapies that have much lower cure rates or keep waiting
and hope that the new therapies are approved before their liver disease
progresses any further, which in itself would make a cure harder to achieve.
The implications of delayed access to the most effective and appropriate
treatment for a condition can be significant, as explained by GSK:
Delays in access to the best, most cost effective medicines
will mean longer periods of debilitating illness for patients, time off work,
increased use of other health services and, in some cases, could be life
threatening. If they continue, the impacts will flow through to the wider
economy through decreased workforce participation, increasing welfare payments,
increasing health and hospital costs.
Mr Denis Strangman noted that both New Zealand and the United Kingdom (UK)
had responded actively to address the issue of timely access to treatment for
particular groups of cancers. In the UK a special fund, the Cancer Drugs Fund,
has been created to provide access to promising new therapies. In relation to
New Zealand he quoted from the Pharma Times of 7 July:
PHARMAC [the Pharmaceutical Management Agency of New Zealand]
is also creating a pathway to assess treatments more quickly for patients whose
condition would significantly deteriorate or who would miss the opportunity for
significant improvement during the usual time taken to assess a Pharmaceutical
Submitters also argued that delays are likely to arise from Cabinet
consideration, as Federal Cabinet will now consider the listing of every new
medicine, and not just those with a financial impact of over $10 million
Ms Bennett explained to the committee that when the $10 million threshold was
in place the listing approval process for high-cost items tended to be more
lengthy due to the requirement for Cabinet consideration, and on this basis
calls had been made to lift the threshold to ensure patients faster access to
People with chronic illnesses should not have to suffer
continued delays in access to medicines because of the Government’s very short
term budgetary goals.
Impact on health outcomes
Many submitters expressed concern that there will be adverse health outcomes
for patients who are unable to meet the cost of medicines not listed on the
PBS. As a result of high costs, patients may instead elect to use an
alternative medicine which is listed on the PBS, but which may not be as
effective, or may have undesirable side-effects.
The Australian Pain Management Association (APMA) submitted:
...if patients cannot afford the non-listed medications, they
may cease medications, take the medications on a basis or frequency less than
medically recommended, or utilize inferior (but cheaper) medications. Each of
these consequences will have implications for the individual, especially over
time. Conditions, and or symptoms, can and will worsen. Consequential medical
costs, health outcomes and quality of life/functionality impacts will result.
The COSS Network noted that inability to access the most appropriate
treatment will impact both individual and public health:
The Government's decision to defer the listing of
pharmaceuticals recommended by the Pharmaceutical Benefits Advisory Committee
(PBAC) will mean that the most vulnerable sick consumers will be unable to
afford some critical medicines and vaccines. This will have negative impacts on
their individual health, and also the broader health and well-being of our
CVA also voiced its concern that patients will not be receiving the best
treatment for their condition due to the deferral decision:
We are deeply concerned that medications that can provide
patients with proven substantial health benefits are not being listed on the
PBS and that, as a result, some Australians will not be receiving the best
possible proven treatment for their medical condition.
These concerns were echoed by Dr Fisher from AstraZeneca Australia, who
explained that patients will continue to be treated, but the issue is that
around 30 per cent of patients will have an adverse event from the
medication they are prescribed, and if medicines which provide an alternative
treatment continue to be deferred, these patients will be at risk of
Mr Stafford described the possible impact on a patient's quality of life
if the appropriate medicine is not subsidised under the PBS:
I found that the proven best treatments in our case were
through pharmaceutical medications that were not subsidised on the PBS. I did
not pay for the necessary medication for over 5 years because I was rich. I
bought the medications because they worked. They worked in that they gave the
patient a quality of life that she would otherwise have been denied.
The alternative was to accept the GP's recommendations of
only PBS subsidised drugs, none of which would have helped the patient, and
have the patient committed to a locked institution until her death.
Submitters also pointed to other benefits which may accrue from the use
of alternative medicine regimes, for example, certain medicines may make
'compliance' with the treatment regimes required for various conditions easier,
and therefore directly improve health outcomes.
A case in point is the deferred antipsychotic medication Invega Sustenna®. Mr Bruce Goodwin,
Janssen-Cilag Australia, referred to the compliance benefits associated with
There is a well-established body of evidence supporting the
use of antipsychotic medications to reduce relapse. In particular, long-lasting
injectables are shown to improve adherence to medication. In fact, around 80
per cent of patients remain adherent on long-lasting injectables whereas only
50 per cent remain adherent on oral medications as an alternative. Of course,
if you do not take your medication you are more likely to get sick again.
NAPWA also raised concerns that health outcomes would be compromised if
new and improved medicines and therapies are delayed, or are withdrawn from or
not made available in the Australian system.
Ms Tyrrell of Hepatitis Australia noted that any deferral in the treatment of
Hepatitis C can have significant implications, including 'increasing liver
fibrosis, cirrhosis, liver cancer, the need for a transplant, liver failure and
In addition, submitters also argued that delay in access to medicines can
impact on the longevity of patients. One respondent to the CHF survey provided
an example to demonstrate that a delay in access to medicine can lead to loss
Delay in listing Azacitidine last year (recommended by PBAC
September 2009, listed February 2011) meant some of Leukaemia Foundation's
patients died waiting for this new drug while awaiting Cabinet approval for PBS
Benefits of effective treatment of medical conditions
Chronic Illness Australia and other submitters explained that access to
affordable medicines is important for those suffering from chronic illness, not
only in terms of staying alive but also in affording them quality of life,
thereby enabling them to participate more actively in the community and the
iNova Pharmaceuticals (Australia) commented:
Listing new therapies on the PBS is a key investment in the
health of Australians and not a mere cost. These are therapies that have been
shown to meet a clinical need and improve health outcomes, for example,
increase survival or greater quality of life, often saving costs elsewhere in the
Further, NAPWA noted that there is a public health benefit in the
effective treatment of medical conditions such as HIV, because as the 'health
of a person with HIV is improved the amount of virus they carry is reduced to
very low levels, thus making onward transmission of the virus very difficult'.
The committee also heard that effectively treating a medical condition
can prevent co-morbidity, or suffering from other conditions caused by or
linked to the original condition, which not only impact on the quality of life
of the patient, but also create further burden on the health system in terms of
resources and cost.
Mr Pask of MS Australia explained to the committee what it means to him
to have access to the medicines he requires:
I have multiple sclerosis and I have type 2 diabetes—even
though I do not admit to it—arthritis and a few other things, so I am dependent
on so many medications. In theory, I am able to work three days a week. But I do
work a lot longer than that...But I am only able to do that because the
medications are there.
...I do not believe we look enough at the value of
medications, as far as keeping people in work and getting people back into
work. Obviously, the cost benefit is what we get out of it. Because of my
medications, I have been given an opportunity to keep going and keep working
and doing stuff that I really love doing. But if I were not working I would not
be able to afford a lot of that stuff...
Consequences of not having access to the deferred medicines
As discussed in chapter 3, the committee heard that not all alternative
medications are equal, and for some patients, alternative treatments are not a
viable option. The committee sought to quantify the impact on patients of the Government's
decision to defer particular medicines, and found that potentially the deferral
of these products could affect:
100,000 people in Australia living with schizophrenia;
an estimated 50,000 to 100,000 Australian patients living with
potentially up to 40,000 patients who would be eligible for the
almost one in five Australians over 40 years of age who are
affected by chronic obstructive pulmonary disease;
about 24,000 patients over five years with venous
potentially 10,000 patients receiving in vitro fertilisation (IVF)
and gamete intrafallopian transfer (GIFT) interventions.
The following discussion focuses on three of the medicines deferred:
Targin® and Botox® (for the treatment of
Case Study 1: Invega Sustenna® (paliperidone palmitate)
is a treatment for schizophrenia which is administered monthly through an
injection to the arm. While it acts in a similar way pharmacologically to
another antipsychotic medication, risperidone, it differs to all other
antipsychotics in the method in which it is administered.
Ms Bennett noted that the CHF has received a particularly strong
response from consumers about the deferral of Invega Sustenna®:
...largely because the concern there is that there are real
issues around compliance of mental health consumers. When you have to attend a
clinic and have two injections a month, versus attending a clinic and having one
injection a month, that may seem fairly insignificant to the average person.
But for somebody who is trying to manage their lives and has to involve a
carer, has to get to a clinic and has to maintain a treatment regime and could
well, if they do not take their medication, end up hospitalised and
psychotic—and all the implications that go with that—that is a pretty
significant quality of life impact. So, for those consumers, that stood out to
me as being one of the drugs that it is pretty hard to argue that there is an
alternative available for, when the alternative can mean the difference between
somebody being hospitalised or not.
Mr David Learmonth of the Department of Health and Ageing (DoHA) noted
that the PBAC recommended Invega Sustenna®
on the basis that its efficacy and toxicity is similar to the existing
treatment, Risperdal Consta®,
but concluded that the claim of clinical superiority was not justified on the
No benefit in reduction of relapses was demonstrated, nor was
there any evidence to support the claim that this alternative drug reduces the
number of days patients spend in hospital.
However, a number of submitters argued quite strongly that Invega
Sustenna® is an
important alternative medicine. Ms Bulfone of Deakin University used Invega
Sustenna® as an
example to illustrate that alternative medications are not equivalent on a
Invega Sustenna for schizophrenia is a really good example of
where the drug on a population level results in the same average benefit to
patients in a clinical trial, but for an individual patient, particularly as
this drug has different pharmacological properties to its comparator, the
profile of side-effects that goes with it is also different. A patient may not
be able to tolerate Consta, which is currently available and was the comparator
in that case, but they will be able to tolerate Invega Sustenna, or they may
respond to one but not the other. Yes, there are alternatives, but they may not
A respondent to the CHF survey also explained the value of an
alternative treatment for patients who have not responded well to existing
The Government has promised to increase funding for mental
health, yet in this recent decision it has refused to allow a new drug to be
listed for the treatment of schizophrenia. As with any drug that treats
conditions of the brain, one drug does not suit everyone, and by refusing to
allow the availability of a new drug, this Government is depriving those with
schizophrenia who have not responded well to existing drugs of an opportunity
to achieve good mental health. This action flies in the face of this
Government's stated position on mental health.
SANE Australia commented that, for people living with schizophrenia, the
difference in administration of Invega Sustenna®
has significant benefits, and should not be trivialised:
A monthly injection makes adherence much more likely and
reduces by half or more the number of visits to a doctor. For people whose
symptoms include reduced cognitive ability and motivation, it increases the
likelihood that they will attend the appointment; it also halves the attendant
trouble and cost of a journey to the doctor's surgery or clinic. This in turn
benefits the carer as well as freeing up the clinician's time and
administration costs. A monthly injection to the arm is also more easily given,
more dignified and less traumatic than a fortnightly injection to the buttock
(especially where the person is being treated involuntarily).
Case Study 2: Targin® (oxycodone/naloxone)
used for the treatment of pain and has been approved by the Therapeutic Goods
Administration (TGA) for managing moderate to severe pain which is unresponsive
to non-narcotic pain relief medication.
Respondents to the CHF survey commented that Targin® has benefits over other available
'alternatives' in that it provides a substitute for the more addictive
Oxycontin, and 'as a combination of oxycodone and naloxone cleverly avoids the
opioid bowel dysfunction seen with other opiod analgesics and reduces
significantly the potential for abuse'.
Submitters explained that Targin®
has the potential to reduce the abuse and diversion of strong prescription
opioids, and is 'the first opioid analgesic that incorporates abuse deterrence
technology to help address this major community issue'.
Opioid diversion is something we cannot just push aside; it
is becoming a much, much bigger problem throughout the world. This preparation
would be extremely unattractive to opioid abusers because it is not designed
for intravenous use. If it was injected intravenously, because of the blocking
drug contained in the preparation it would cause an immediate and very severe
withdrawal. It is the start of our abilities to provide pain management with a
specifically designed preparation that will not have any appeal whatsoever to
The committee was told that it is difficult to quantify the potential
for reduced drug diversion and abuse, and insufficient evidence on this
characteristic was provided to the PBAC for a definite conclusion.
While the technology incorporated into this medicine will not stop abuse
entirely, from a practitioners point of view:
...at least it would overcome the problem, when we are
prescribing the current range of opioid drugs for a patient with medical
indications, of us having to be concerned that this drug might, by various
means, be diverted to other users.
Mr Learmonth of DoHA noted that in its PBAC submission Mundipharma did
not claim that Targin®
was superior to the currently available oxycodone in combination with over the
However, in its submission, Mundipharma explained that opioid-induced
constipation has distinct causative factors, and laxatives do not address the
cause of the condition, rendering them a 'less than adequate treatment'.
Mr Rob Baveystock, Managing Director, Mundipharma, added that while
there are opioid analgesics available to treat chronic pain, these treatments
Whilst these medicines are undoubtedly effective in relieving
severe disabling pain, they are also potentially associated with serious side
effects, including opioid-induced bowel dysfunction, often including severe
opioid-induced constipation, and potential for addiction. What is apparently
not understood by government is that this is not the type of constipation that
otherwise healthy individuals might suffer from time to time. It is far worse;
it is almost inevitable; it results in increased cost to the community and
government; and, importantly, it cannot be treated with simple laxatives.
Severe constipation can lead to impaction and hospitalisation and can aggravate
cancer pain, resulting in a pattern of increasing opioid dosages in an attempt
to relieve pain...there is no strong opioid analgesic available in Australia
which treats chronic disabling pain while simultaneously addressing the cause
of opioid-induced constipation and helping to prevent it.
Case Study 3: Botox® (botulinum toxin type A)
been TGA registered for the treatment of severe primary hyperhidrosis of the
axillae (underarms). This condition manifests through severe, excessive
sweating, and as a result, patients suffer constant wetness and staining of
clothing, and dehydration and maceration of the skin, which can result in
secondary skin infections. Patients can also experience difficulty in grasping
objects and writing. Effective treatment can improve the social functioning and
mental health of those affected by hyperhidrosis who can become withdrawn and
depressed as a result of the condition and its symptoms.
As recognised in treatment guidelines, Botox® provides a unique second-line treatment for
those affected by hyperhidrosis whose condition is not effectively managed
through prescription topical aluminium chloride antiperspirants. It also
provides a less invasive treatment option prior to the consideration of
surgery, which is only undertaken in a minority of patients. In making a
positive recommendation for the listing of Botox®
for the treatment of severe hyperhidrosis, the PBAC noted that there were no
other second-line treatments for severe hyperhidrosis of the axillae on the
PBS, that the condition has significant impact on the quality of life of
patients, and that there was a clinical need for botulinum toxin.
Submitters explained that treatment of severe hyperhidrosis through the
use of lotions or surgery is not necessarily successful, and the side-effects
of these options are often less than desirable. Ms Elizabeth Trapani explained
to the committee that she has pursued a number of local treatment options for her
daughter, Ms Chey-Anne Ellsum, who suffers from severe hyperhidrosis:
...we used every lotion, potion, spray and roll-on we could get
our paws on. We had no luck.
Given the invasive nature of the surgical option, the comparatively low
success rate, the fact that it is not a permanent solution, and the potential side-effects
of the surgery which can include a collapsed lung, palsy of the face and
increased sweating, Ms Trapani explained 'When we found out the dangers of this
surgery, it was not an option I wanted to pursue for my daughter'.
Mr Mark Glover of Allergan Australia noted that in explaining why the
listing of Botox® under
the PBS was deferred, the minister has acknowledged that there is no alternate
treatment available under the PBS, and further, has stated that hyperhidrosis
is a mild condition for many people. However, Allergan Australia took issue
with this reasoning, as the TGA approved indication and PBAC recommended
listing for Botox®
are for severe disease.
treatment is expensive – in Ms Ellsum's case, the initial treatment cost just
under $1900, a cost which for many people is prohibitive. Ms Trapani
explained that they were fortunate that Ms Ellsum's grandparents were able to
assist with the payments.
Ms Ellsum provided the committee with an insight into how living with
hyperhidrosis had affected her life, and the difference it has made to be able
to access the Botox®
treatment. She also noted that if the Botox®
treatment for hyperhydrosis was listed under the PBS, it would be more
accessible not only for her, but also for others suffering from the same
I have had it since I was three, but it got really bad in
puberty. That was when I was in high school, so I was like the magnet for
bullying; everyone went at me, because they did not understand, and no-one
understands. It caused my depression. There were days when I did not want to
get out of bed because it is so controlling. I blamed myself lots of the times
when I had it; I thought it was just me who had it. It draws people back; it
stops people from doing things. It deprived me of most of my youth; I did not
do the things I wanted to do because it was so controlling and conflicting with
my life. I could not do my deb this year because I was afraid of what people
would think, and that really made me sad.
With the Botox, it has been amazing. It is the biggest
improvement that I have ever heard about, and it has worked. I am planning to
do my deb next year, and now I am doing all the things that I have always
wanted to do. I am here now. This is not something I would have done back then.
I am here because there are people out there who cannot get this. I would be
stuck if it were not for my grandparents, because they are the only people who
are getting me through this. I would not have this if it were not for them.
Evidence received by the committee highlights that many Australian
health consumers are under significant financial burden, particularly those
with chronic illness and those on low-incomes. The PBS process should ensure
that the most effective medications are available at an affordable price for
all Australians. However, the Government has now introduced a further
consideration to the listing of medications: an unclear, undefined fiscal
hurdle with no specific timeline.
This a short-term consideration which will have adverse affects on
patients. For some consumers, the deferred medicines represent a more appropriate
treatment regime, or in some cases, the only effective treatment. In the
committee's view, it is unacceptable that the Government is denying access to
The committee is concerned that the Government's decision to defer the
listing of certain medications will exacerbate inequitable access to medicines
for low-income and disadvantaged Australians. The committee is of the view that
this will lead to a two-tiered system in which only those with adequate
financial capacity will be able to access newer, more effective treatments,
which will remain out of financial reach for lower-income patients.
The committee considers that timely access to affordable appropriate
alternative medications is a centrally important feature of the PBS. The
committee notes the consequences in terms of quality of life and adverse events
of not having access to an appropriate and effective medication as illustrated
by the case studies discussed in this chapter. This not only has repercussions
for the health of individuals, but also for broader public wellbeing and
demands on the public health system. In light of this the committee is of the
view that health outcomes of Australian patients should not be compromised by
restricted access to a small or pre-determined number of medicines in a given
class due to the Government's budgetary considerations.
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