As the debate rages on the price tags associated with orphan drugs an opinion presented in The Journal of Clinical Pharmacology discusses whether the high price associated with them are feasible. The authors discusses the legislation passed in the United States (US), European Union (EU), Japan and Australia which has led to orphan drug development to be financially viable with more marketing authorizations in the subsequent years. The authors make a special mention of the 21st Century Cures Act and the expedited regulatory programs in the United States, which promises to expedite drug development including those for orphan drugs.
According to the authors the approval rates for orphan drugs in the US and EU are low and are less than 20% among those receiving orphan drug designations. Most of the designations and approvals are for “ultra rare diseases with limited market potential and any improvement in disease stratification of “orphan subset” will enhance treatment outcome and cost effectiveness, and should be encouraged.”
The authors are aware of the public apprehension about the high pricing of orphan drugs and the fact that the high drug prices associated with low cost-effectiveness could limit reimbursement. Even then the author believes the high cost could be worth the potential medical breakthrough. They ask for public support for the development of orphan drugs, and a system which will ensure sufficient return of investment.