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University of Basel

Is it worth researching rare diseases, Stefan Felder?

Text: Stefan Felder

Does it make sense to promote research into diseases that only affect a very small number of people? A health economist and a medical ethicist offer contrasting views.

A rare disease is defined as one that affects fewer than five in 10,000 people. This definition applies to approximately 7,000 different diseases, some of them with severe clinical, social and economic consequences for those who suffer from them. In Switzerland, around half a million people suffer from a rare disease – as many as have been diagnosed with diabetes (40,000 cases for type 1, and 460,000 for type 2). Each rare disease affects 70 people on average. This presents research in the field of rare diseases with a major challenge: Whereas a given investment in diabetes research benefits half a million patients, research into a rare disease benefits only 70. From this perspective, research into rare diseases is not actually worthwhile.

Yet, this “cold” economic rationale is not politically popular. In the fall of 2014, the Swiss Federal Council adopted the “National Strategy for Rare Diseases” as part of the “Health 2020” policy framework, and instructed the Federal Department of Home Affairs to formulate a plan of action. In doing so, Switzerland joined many other countries committed to promoting research into rare diseases.

Is it reasonable to weigh up the fate of 70 patients sufferring from a rare disease against that of half a million diabetics? According to the principles of utilitarianism, this is in fact imperative. Under this philosophy, all patients should be treated equally, while taking into consideration the sometimes considerable differences in severity among the various diseases. This is because the money spent on a disease affecting 70 people entails opportunity costs: Half a million patients with diabetes are deprived of the potential benefits of research into their disease.

Utilitarianism has a long tradition in countries such as the UK, where it is applied relatively consistently in healthcare. A recently published representative survey reveals support for this policy. According to the survey, the British public does not consider rarity to be a sufficient argument for certain diseases to be given preferential treatment.

The same phenomenon was observed in Norway when the population was consulted on the prioritization of “orphan drugs”, as medication for rare diseases is known. When asked to consider the trade-off s, respondents did not attach any special importance to rare diseases.

A similar position was reflected in the ruling of the Swiss Federal Supreme Court in the highly publicized “Myozyme case” of 2010. This case involved a patient with Pompe disease – a lung disorder whose treatment with Myozyme at that time cost half a million Swiss francs a year. In its verdict, the court noted that 2.8 percent of the country’s inhabitants suffered from a comparable impairment of lung function. If half a million francs were spent annually on treating each of these patients, Switzerland’s entire health budget of 90 billion francs would be exhausted. Accordingly, the court ruled against the reimbursement of Myozyme on grounds of fairness. And this is the surprising thing about utilitarianism: It brings considerations of efficiency and fairness into harmony.

Stefan Felder is Professor of Health Economics at the University of Basel. He is President of the Swiss Society of Health Economics and Executive Secretary of the European Association of Health Economics. His interests include regulation of health insurance and healthcare markets. He has also published on medical decision-making and prioritization of healthcare services.

More articles in the current issue of UNI NOVA.

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