MedicalResearch: What is the background for this study? What are the main findings?
Dr. Onakpoya: Several orphan drugs have been approved for use in Europe. However, the drugs are costly, and evidence for their clinical effectiveness are often sparse at the time of their approval.
We found inconsistencies in the quality of the evidence for approved orphan drugs. We could not identify a clear mechanism through which their prices drugs are determined. In addition, the costs of the branded drugs are much higher than their generic or unlicensed versions.
MedicalResearch: What should clinicians and patients take away from your report?
Dr. Onakpoya: Because of inconsistencies in the evidence regarding the benefit-to-harm balance of orphan medicines, coupled with their high prices, clinicians and patients should assess whether the orphan drugs provide real value for money before making a decision about their use for a medical condition.
MedicalResearch: What recommendations do you have for future research as a result of this study?
Dr. Onakpoya: We need more clinical trials to assess the benefits and harms of orphan drugs, especially for those which currently have low levels of evidence. Systematic reviews of some orphan medicines are outdated, and these need to be updated since further clinical trial results have become available. Furthermore, research aimed at providing a standard, transparent and robust mechanism for determining the prices of orphan drugs is imperative.
Igho Onakpoya MD MSc, & Clarendon Scholar (2015). Evidence of Value of Orphan Drugs Inconsistent