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Orphan Drugs for Rare Diseases

Is it Time to Revisit Their Special Market Access Status?

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Abstract

Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support market access of orphan drugs. We argue that it is time to revisit the special market access status of orphan drugs. Indeed, evidence suggests that there is no societal preference for treating rare diseases. Although society appears to assign a greater value to severity of disease, this criterion is equally relevant to many common diseases. Furthermore, the criterion of equity in access to treatment, which underpins orphan drug legislation, puts more value on health improvement in rare diseases than in common diseases and implies that population health is not maximized. Finally, incentives for the development, pricing and reimbursement of orphan drugs have created market failures, including monopolistic prices and the artificial creation of rare diseases. We argue that, instead of awarding special market access status to orphan drugs, there is scope to optimize research and development (R&D) of orphan drugs and to control prices of orphan drugs by means of, for example, patent auctions, advance purchase commitments, pay-as-you-go schemes and dose-modification studies. Governments should consider carefully the right incentive strategy for R&D of orphan drugs in rare diseases.

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Acknowledgements

No sources of funding were used to assist in the preparation of this manuscript. The authors have no conflicts of interest that are directly relevant to the content of this manuscript. SS and EP generated the idea and structure for the manuscript and wrote the initial draft. DC and MD made contributions to writing the article and provided feedback.

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Authors and Affiliations

  1. Research Centre for Pharmaceutical Care and Pharmaco-economics, KU Leuven, Onderwijs en Navorsing 2 bus 521, Herestraat 49, 3000, Leuven, Belgium

    Steven Simoens & Eline Picavet

  2. Department of Hepatology, University Hospitals Leuven, Leuven, Belgium

    David Cassiman

  3. Hospital Pharmacy, University Hospitals Leuven, Leuven, Belgium

    Marc Dooms

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  1. Steven Simoens
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  2. David Cassiman
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  3. Marc Dooms
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  4. Eline Picavet
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Correspondence to Steven Simoens.

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Simoens, S., Cassiman, D., Dooms, M. et al. Orphan Drugs for Rare Diseases. Drugs 72, 1437–1443 (2012). https://doi.org/10.2165/11635320-000000000-00000

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