A doctor writes: Cost of high-tech drugs a bitter pill to swallow

Melanoma patients told State to cover Pembro, while CF drug Orkambi ‘too expensive’

How do you put a price on a life?

In a week when melanoma cancer patients were told the State would reimburse the costs of a new immunotherapy agent, Pembrolizumab (Pembro), the hopes of people with cystic fibrosis (CF) have been dashed as the National Centre for Pharmacoeconomics (NCPE) decided the novel treatment Orkambi was too expensive, relative to its potential benefit, to justify reimbursement.

If you are a patient or a relative of someone with CF, you will feel hard done by today.

Especially if you heard Jillian McNulty tell RTÉ how Orkambi has “transformed” her life and reduced her hospital admissions in the three years she has been on treatment.

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“Orkambi means people with cystic fibrosis using it can live a normal life, or as near a normal life, as possible,” she said, describing it as a life-saving medication.

The drug works on CF patients who have a particular gene mutation, and who make up about half of the 1,200 people with the disease in Ireland.

Cost-effectiveness

The cost of Orkambi is €158,000 annually per patient. The NCPE says the price would have be lower than €30,000 annually to be cost-effective.

Yet, Pembrolizumab, which costs almost €70,000 annually per patient, will be made available to about 130 patients.

Health technology assessment (HTA), the process under which the NCPE evaluates new treatments, works by summarising information on the medical, social, economic and ethical issues related to the new drug.

HTA aims to identify what will deliver the best health gain for the population within the constraints of a fixed healthcare budget.

The process uses a measure called quality-adjusted life years (QALY) for assessing the value of new drug treatments; it looks at the cost of using a drug for a year and weighs it against how much a patient’s life can be extended and improved.

If a treatment costs more than €30,000 per QALY (£30,000 in the UK) – a theoretical unit representing one year in perfect health – it is generally not recommended as cost-effective.

To approve a treatment above this level, advisory bodies such as the NCPE need to make an increasingly strong case for supporting the intervention as an effective use of health system resources.

Variations in views

The QALY system is not without its critics. Researchers from the European Consortium in Healthcare Outcomes have said the system fails to reflect variations in views on illness and disability. Based on the views of some 1,300 respondents, they concluded that the way people rate medical outcomes varies widely and could not be summed up in a neat medical formula.

The worry is that not enough attention is being paid to the subjective views of patients. As part of the commitment in the programme for government to “appropriate patient-care pathways”, it may prove fruitful to explore people’s views of the HTA/QALY system.

However, it is difficult to see how any system, no matter how sophisticated, could decide on the competing needs of, for example, melanoma cancer patients and CF patients. Because, in a system of finite resources, that is the ultimate quandary.

For new, more expensive drugs to be approved for any illness, a cut in services must be made elsewhere in the system. And that, surely, merits a substantial national debate.