For healthcare professionals only


Is it time to revisit orphan drug policies?

BMJ 2010; 341 doi: (Published 22 September 2010) Cite this as: BMJ 2010;341:c4777
  1. Christopher McCabe, professor of health economics1,
  2. Tania Stafinski, associate director health technology and policy unit2,
  3. Devidas Menon, professor of health policy and management2
  1. 1Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, Leeds LS2 9LJ, UK
  2. 2Department of Public Health Sciences, School of Public Health, University of Alberta, Edmonton, AB Canada T6G 2V2
  1. c.mccabe{at}

    Yes, for equity’s sake

    The number of new treatments for rare disorders—so called orphan drugs—has increased over the past decade. This is a testament to the success of the Orphan Drug Act in the United States and the Orphan Drugs Regulation in Europe.1 2 The large number of treatments in late stage development indicates that this success is likely to be sustained.3 However, this poses a substantial challenge for healthcare systems because the prices charged for these drugs make it impossible for them to meet conventional measures of good value.4 Increasingly, access to orphan drugs is likely to be restricted, causing political problems for governments and reducing the return to manufacturers from their research investment.

    To date, many healthcare payers have exempted orphan drugs from formal value assessment, arguing that society values equal opportunity for people with rare and common conditions enough to justify the high costs. Until now this has been assumed, rather than being based on robust evidence.5

    In the linked survey (doi:10.1136/bmj.c4715), Desser and colleagues asked a representative sample of the Norwegian general population whether society should pay more to treat rare diseases than it does …

    View Full Text

    Log in

    Log in through your institution


    * For online subscription