Orphan drugs policies: a suitable case for treatment

Michael Drummond, Adrian Towse

Research output: Contribution to journalArticlepeer-review


Context: Current orphan drug policies are unsatisfactory when viewed from almost all perspectives. Patients find that, although therapies are available for many rare conditions, access to care is sometimes restricted. Pharmaceutical manufacturers have responded to the incentives for research embodied in orphan drug legislation, only to find that funds are not made available to pay for therapies once developed. Those funding health care find that most orphan drugs do not justify funding based on standard value for money criteria, yet that they face political problems if they fail to provide funding for therapy.

Methods: A literature review was conducted in order to determine the precise nature of the problems and to suggest potential solutions.

Results: Current orphan drug policies are not fit for the purpose and initiatives need to be taken in the areas of (1) clarifying society's views about the priority to be given to orphan drugs, (2) revising the arrangements for pricing and reimbursement of orphan drugs, (3) defining the priorities for research into rare diseases and (4) developing 'joined up' policies to deal with these issues.

Conclusions: Without changes in the current policies, pharmaceutical companies will eventually cease responding to the incentives to develop orphan drugs, because they will increasingly be uncertain whether the drugs, if developed, will be reimbursed.
Original languageEnglish
Pages (from-to)335-340
Number of pages6
JournalEuropean Journal of Health Economics
Issue number4
Early online date17 Jan 2014
Publication statusPublished - May 2014


  • Rare diseases
  • Cost-effectiveness analysis
  • Drug reimbursement
  • Health technology assessment

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