title: Orphan drugs policies: a suitable case for treatment.
abstract: BACKGROUND
  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.
authors.affiliation: Centre for Health Economics, University of York, Heslington, York, YO10 5DD, UK, mike.drummond@york.ac.uk.
authors.forename: Michael
authors.initials: M
authors.lastname: Drummond
authors.forename: Adrian
authors.initials: A
authors.lastname: Towse
mesh_terms: D003362:Cost-Benefit Analysis; D016527:Drug Costs; D006291:Health Policy; D006801:Humans; D007349:Insurance, Health, Reimbursement; D009965:Orphan Drug Production; D011050:Policy Making; D035583:Rare Diseases / Q000188:drug therapy*; D013673:Technology Assessment, Biomedical