Exploring the Potential Challenges for Developing Generic Orphan Drugs for Rare Diseases: A Survey of US and European Markets.

Objectives: The US Food and Drug Administration in 1983 and the European Union's European Medicines Agency in 2000 implemented the orphan drug development program for rare diseases. The study aimed to find the potential challenges encountered by generic companies in developing generics for rare diseases.

Methods: We performed a thematic analysis, which consists of qualitative and quantitative research. For data analysis of approved orphan drugs, we used statistical methods, and for the industrial case study, we selected 14 generic companies and conducted semistructured interviews related to 10 critical areas of drug development.

Results: The orphan drug approvals were classified into 4 categories: the number of orphan approvals, pediatric claims, formulation, and therapeutic areas. We analyzed the approvals from 2001 to 2021; the Food and Drug Administration approved 815 drugs and European Medicines Agency approved 258 drugs. The pediatric orphan approvals were analyzed from 2010 to 2021; the average percentage of orphan drugs claim pediatric exclusivity during this period was found to be 31.8%. In formulation, we found the highest percentage of drugs belong to small molecules at 71%. In the therapeutic class, oncology drugs have a majority of approvals at 25%. The industrial case study responses revealed that the major challenge for drug development is the complexity of the disease at 21%, followed by the limited market at 17%.

Conclusions: There is a high need for generic orphan drugs in the developing countries. The generic companies can use the opportunities provided by health authorities for the benefit of both the company and the patient perspective.