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Negotiating limits to accessing medicines for rare cancers:An ethical perspectivePresented byDr Wendy LipworthCentre for Values, Ethics and the Law in Medicine, Sydney Medical School

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Overview

1. What is a rare cancer?2. Who wants access to medicines for rare cancers?3. Who provides access to medicines for rare

cancers?4. Why can decisions about access be difficult?5. Implications for rare cancer advocacy

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What is a rare cancer?

– Various definitions of rare cancers– 2-15 per 100,000 people– Australia 6 per 100,000

– Can be rare because– Arises in unusual site– Is a biologically unusual variant of a common cancer– Needs special treatment

– In a typical year over 44,000 Australians will be diagnosed with a rare or less common cancer and nearly 24,000 will die

– Little change in incidence to mortality rations over past 20 years

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Overview

1. What is a rare cancer?2. Who wants access to medicines for rare

cancers?3. Who provides access to medicines for rare

cancers?4. Why can decisions about access be difficult?5. Implications for rare cancer advocacy

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Who wants access to medicines for rare cancers?

– Review of Life Saving Drugs Program (2014)– Cancer Drugs Alliance submission:

• LSDP “absolutely essential” for patients with rare cancers

• Should be revised to accommodate cancer

– Senate Inquiry on the availability of new, innovative and specialised cancer drugs in Australia (2015)– Rare Cancers Australia submission:

• “some cancer patients have suffered from systematic neglect”

• Need for an “interim access fund” prior to PBS listing• Need to consider licensed usages of medicines

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Overview

1. What is a rare cancer?2. Who wants access to medicines for rare cancers?3. Who provides access to medicines for rare

cancers?4. Why can decisions about access be difficult?5. Implications for rare cancer advocacy

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Who provides access to medicines for rare cancers?– The Federal Government via the PBS– “Special” government programs

– Life saving drugs program– Herceptin program

– Hospital therapeutics committees– Pharmaceutical companies (compassionate

access)– Researchers by enrolling people in clinical trials– Individuals who can afford to pay themselves

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Overview

1. What is a rare cancer?2. Who wants access to medicines for rare cancers?3. Who provides access to medicines for rare

cancers?4. Why can decisions about access be

difficult?5. Implications for rare cancer advocacy

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Why can decisions about access be difficult?1. There is a need to consider a number of

different values2. These values can be difficult to fulfill when it

comes to rare cancers3. These values also compete and conflict with

each other

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Doing good/avoiding harm

– The desire to provide benefit to patients, without harming them

– The desire to fulfill the related the “rule of rescue”– the moral and psychological imperative to help those in

desperate need, irrespective of cost or uncertainty

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Why it is difficult to consistently do good/avoid harm– Treatments for rare cancers are not always as

safe and/or effective as we hope they will be – Like any other treatment

JAMA Internal Medicine October 2015• 2/3 of new cancer drugs in the past five

years approved by FDA on surrogate outcomes e.g. tumour shrinkage, progression free survival

• They have not been shown to extend or improve life

• They may be toxic• Includes treatments for rare cancers

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Equity

– The desire to promote equity—ensuring that patients are not disadvantaged simply because they have rare cancers or rare subsets of cancers

– Currently receive a disproportionately small % of PBS funds (15%)

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Why it is difficult to ensure equity of access– Government payers, hospitals and

pharmaceutical companies can’t fund everything for everyone– Some people will always be disadvantaged relative to

others– Groups of cancer patients are being divided up

more and more according to molecular profiles• E.g. 1% of NSCLC have ROS1 mutation and respond

to crizotinib• E.g. 10 % of stomach cancer have HER-2 mutation

and respond to trastuzumab (Herceptin)– This is good for some people who can receive “targeted

therapies”– But also means there are more people to be “left

behind” relative to others

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Why it is difficult to ensure equity of access– Modes of access other than through the Federal

Government will always be uneven, based on:– Who has access to/is eligible for clinical trials– Who has access to/is eligible industry compassionate

access programs– Who has doctors advocating for supply through hospitals– Who has money in the absence of the above

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Efficiency, affordability, fairness of price– The desire to allocate resources efficiently

– producing the ‘greatest good for the greatest number’ – in an affordable and cost-effective manner

– And paying a ‘fair’ price for medicines, based on their clinical value

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Why it is difficult to ensure efficiency and affordability– Many treatments for rare cancers are very

expensive– Development is expensive (e.g. time to recruit to trials)– Markets are small

– The number of rare cancers is collectively large (and increasing as cancers are divided up according to particular molecular profiles)

– The costs add up when all rare cancers are considered together

– Even medicines that are “cost-effective” may not be affordable

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Why it is difficult to negotiate a “fair” price– Main justification for high prices is need to recoup

investment in innovation and promote further innovation

BUT– No agreement as to how much it costs to develop

a new drug– Tufts estimate >$2 billion

• incl. considerations of risk and need to attract investors

– But critics say the figure is overblown by• Ignoring public investment in basic science• Including marketing costs• Overstating risk and what it takes to attract

investment– Some believe that prices are whatever the “market will

bear”– Small markets have less bargaining power

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Scientific evidence

– The desire to make decisions based on sound scientific evidence of – Effectiveness– Safety– Cost effectiveness

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Why it is difficult to make “evidence-based” decisions– Can’t always do large RCTs and meta-analyses

– Numbers of patients are (by definition) small difficult to recruit to trials and achieve adequate power

– Patients are often desperately ill and need to “crossover” upon progression difficult to maintain a control group

– For targeted therapies have to factor in • “Companion diagnostics”• Appropriate comparator

– Difficult to demonstrate effectiveness and cost-effectiveness

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How values compete and conflict

In order to achieve… There is often a need to compromise on…

Clinical benefit Safety

Equity of access EfficiencyAffordabilityPower to negotiate a “fair” price

Quick access Quality of evidence used to make decisions

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Lessons for advocacy

– Not enough to just advocate for “more access” – Need to recognise the complexity of decisions

and advocate for “procedural justice”:– Stakeholder engagement

• So that we can identify all the relevant values– Transparency

• So that we can see that all values are being considered and balanced in a “reasonable” way

– Accountability• So that decisions can be overseen e.g. re conflicts of

interest • So that decisions can be appealed