Orphan Drugs Policies: A Suitable Case for Treatment

Towse and Drummond take a critical look at deficiencies in policy and suggest revisions.

An editorial just published by OHE’s Adrian Towse and Michael Drummond of the University of York argues that current orphan drug policies are ‘not fit for purpose’ and discusses the issues that need to be clarified as the basis for policy revisions.

An editorial just published by OHE’s Adrian Towse and Michael Drummond of the University of York argues that current orphan drug policies are ‘not fit for purpose’ and discusses the issues that need to be clarified as the basis for policy revisions. The editorial is intended ‘not to provide complete solutions to all the policy problems, but rather to set out a roadmap whereby they can be resolved’. Four groups of issues are identified.

1. Recent surveys appear to suggest that the general public would not necessarily allocate more resources to those with orphan diseases than those with common diseases. The authors suggest that those surveyed may have been influenced by how the questions were framed and that other methods of eliciting preferences should be tested. These include asking directly whether special funding for those suffering from orphan disease should exist and, if so, how much. Clarifying society’s preferences should be the first priority in working towards better public policy.

2. How orphan drugs may be best assessed to determine pricing and reimbursement continues to present challenges. Towse and Drummond identify the primary challenges this has presented, and suggest potential responses. But they also raise an important, and controversial, issue:

If it is known in advance that most of these drugs will not be cost-effective based on standard criteria, why undertake the assessments in the first place, unless there is the possibility that better value for money could be obtained if the drugs were better targeted? It might be better to acknowledge at the outset that high-priced orphan drugs will not be reimbursed, thereby avoiding wasting resources on their assessment.  

3. New approaches to treatment, including advances in targeting treatment to the individual patient, may produce the temptation for companies to make more ordinary diseases appear as ‘orphans’, according to Towse and Drummond. The problem of ensuring that incentives produce the intended consequences may require redefining ‘orphan disease’ or removing orphan status when a product achieves other, non-orphan, indications.

4. ‘Probably the biggest concern about current policies for orphan drugs’, according to the authors, ‘is that the policies for stimulating research and providing reimbursement are at odds with one another, leading to inefficiencies if scarce resources are devoted to the research and development of drugs that are not going to be used’. These policies, they argue, must be ‘joined up’. This might include explicit statements of priority, signaling to drug developers which new products are most likely to receive reimbursement. Setting appropriate priorities, moreover, could be supported by product development partnerships that involve the public and charity sectors.

Towse and Drummond conclude that: ‘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’.

Open access: Drummond, M. and Towse, A., 2014. Orphan drugs policies: a suitable case for treatment. The European Journal of Health Economics. 15(4), pp.335-340. For additional information, please contact Adrian Towse.