What Is Wrong with Orphan Drug Policies? Suggestions for Ways Forward

We argue that orphan drug policies have been useful in incentivizing socially desirable R&D and that in their absence it is unlikely that treatments of any kind would have emerged. Weaknesses in the current policy framework need to be addressed by refining this framework rather than altogether replacing or dismissing it as inefficient. Improvements can be made in data collection, and efforts are already under way at the European Union level with initiatives concerning registries. Similarly, the legislative framework can be refined to define when an orphan treatment is "sufficiently profitable," at what stage should profits be considered excessive, and, consequently, whether any favorable conditions offered to manufacturers should be removed. Concerns about availability and accessibility of orphan drugs, which are valid in many instances, do not imply that the current orphan drug policy framework is deficient but that the means of assessment need to be improved upon for realistic and affordable prices for payers to become the norm. This implies better data quality, the possible extension of the criteria for value assessment to take explicitly into account the peculiarities of rare diseases, and the availability of appropriate benchmarks around rare disease cost and quality of life to conduct meaningful value assessments.