THE NATURAL RESPONSE of an individual or the family of a person with a life-threatening disease is to advocate for funding for the best drug that could save their life.
That is exactly what you do when you’re fighting for your life or the life of a loved one. That drug might be Orkambi, Pembro or Spinraza.
But what is the role of the state in those complex decisions around funding new drugs, where individual rights may conflict with broader principles concerning the distribution of state resources?
Is it possible to achieve an objective process that citizens can agree on and politicians will comply with?
New ‘high-tech’ drugs are extremely expensive, too expensive for countries to pay for all of them at the prices quoted by drug manufacturers. If they did, there would be no money left over for healthcare services.
Moreover, research shows that many new drugs, such as cancer drugs produced during the last decade, do not show sufficient improvement on existing drugs to justify public funding.
All countries have to make incredibly difficult choices in healthcare, to decide what drugs and services to fund and how to do that in a justifiable, ethical, and cost-effective way.
In choosing what to fund, they must consider the ‘opportunity cost’, that is, all of the alternative things that could have been funded with the same money.
How does the state decide which new drugs to fund?
In Ireland, the job of the HSE’s National Centre for Pharmacoeconomics (NCPE), is to review new drugs that have met EU safety standards.
It decides whether or not to recommend the new drug and make it available to Irish citizens by funding it through the public health system.
This work involves an examination of the cost-effectiveness of a drug to see if it provides significant benefit to patients above and beyond similar treatments on the markets and whether that benefit is commensurate with the cost the pharmaceutical manufacturer is charging for the drug.
The measurement of the drug’s value is in QALYs (quality-adjusted life years). This considers the number of additional years a drug might potentially add to a person’s life and the quality of life the patient can expect.
Ireland’s policy is similar to the UK’s.
The NCPE has a cost-effectiveness threshold, a price per QALY over which treatments are less likely to be recommended.
This provides a consistent framework against which the benefit of all new drugs can be measured, while still allowing scope for special consideration for conditions such as rare diseases.
What happened with Orkambi?
The pharmaceutical company Vertex developed the drug Orkambi to treat cystic fibrosis and ran clinical trials of the drug with Irish patients. Ireland has the highest incidence of the disease in the world.
The Orkambi drug then passed EU drug safety standards and, in Ireland, it was referred to the NCPE to assess its potential benefit in the context of the Irish population.
In 2016, the NCPE concluded that Orkambi did not demonstrate sufficient additional health benefits to patients over and above existing drugs at the price it would cost the state.
The decision of NCPE was reported widely in the media. Many patients who had participated in the drug trial and who were already taking the drug were told they would not have access to Orkambi in the future as the state would not cover the cost.
At the time, the final decision regarding the allocation of funding for new drugs rested with a single individual, the Minister for Health.
The NCPE decision prompted a vocal campaign by families of people with CF, and trial participants publicly expressed their fears over the drug being withdrawn from them.
The ensuing media coverage and political scoring by opposition parties put the minister under pressure to ignore the advice of the state’s expert committee, and he eventually relented.
It was a positive result for CF sufferers, but it highlighted serious weaknesses in the drugs approval process. After the Orkambi experience, the Minister announced that responsibility for making decisions on funding new drugs would be transferred back to the HSE.
What happened with Pembro?
During the summer of 2018, Dr David Fennelly, an oncologist, advocated through the media for funding to trial the new cervical cancer drug Pembrolizumab.
Dr Fennelly was treating Vicky Phelan with the drug, which was approved by the FDA in America in June 2018 for some women with advanced cervical cancer but had not yet been licensed to treat cervical cancer by the European Medicines Agency.
The FDA caveated the announcement warning it was an accelerated approval and that further studies were required to confirm the drug’s clinical benefit in patients with advanced cervical cancer.
Ms Phelan, who campaigned successfully to secure access to the drug for women affected by the CervicalCheck affair, continued advocating for the state to extend access to the drug to others suffering from cervical cancer.
In December 2018, over-ruling its own policy of allowing the HSE’s expert committee, NCPE, to decide on the overall benefits of the drug to the population, the government announced its decision to approve Pembro for all women with cervical cancer.
Where do we go from here?
Few would begrudge a person with a life-threatening illness access to life-saving treatment.
For that reason, we need strong policies in place, policies that we as a society agree on so that funding decisions are not made arbitrarily where they can be strongly influenced by media or exploited by the pharmaceutical sector.
These recent events reveal some serious weaknesses in how we allocate critical state resources in one area of healthcare.
The Helsinki Declaration (1964), the gold standard ethical guidance document for all research conducted in human subjects, recommends researchers leading clinical trials to inform a Research Ethics Committee in advance about what will happen to the trial patients’ treatment once the trial is finished.
Given Irish patients participated in the Orkambi drug trial, could it have been foreseen the state would be on the back foot in any negotiation on the price of the drug if the trial was successful and trial participants had not received any guarantee from the manufacturer that they would have access to the drug post-trial?
Although the QALY (quality-adjusted life year) approach to inform decision-making is not without criticism, as yet, we have no better framework for comparing the benefit of drugs for different diseases.
If politicians do not adhere to the guidelines set out, the decision-making process becomes compromised, politized and unfair.
Furthermore, the inconsistency in its decision-making process strengthens the hand of pharmaceuticals in negotiations around drug pricing. One might also ask, what is the point in having a committee and protocols when we don’t stick to them?
The Pembro story raises important ethical questions around doctors advocating for drugs for individual patients or specific groups of patients, especially without discussing the broader questions of opportunity cost, and fair and transparent allocation of resources in healthcare.
What happens when the money runs out?
The Sunday Business Post reported last week that the State has almost fully committed its 2019 budget for new drugs.
Is there a danger that funding will be redirected from other essential services to meet our growing drugs bill?
It is the duty of the state to allocate funding in a way that is fair, efficient and transparent and we need a system that can do this in a consistent and robust way.
From a citizen’s perspective, we need to think about what is the best way to do this and whether we can reach a consensus on the key principles to underpin the distribution of limited funds.
We also need to consider what we are willing to do without if the drugs bill keeps growing. Will we reduce funding for home care packages, primary and community care services, improved maternity care?
Maybe we need to focus our debate on the right to affordable medicines, not just the right to medicines.
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