While historically on the margins of R&D spend, orphan drug development increasingly looks like a strategy whose time has arrived for delivering value in drug development. The benefits of shifting focus to orphan drugs are numerous, but as competition moves into these niche spaces, the need to demonstrate the economic and clinical value of drugs will only intensify.

Developing a successful “rolling blockbuster” strategy based on the orphan drug model will require biotech and pharmaceutical companies to adopt a new approach to both product development and commercialization. In their article, Orphan Drugs — Niche Strategy, Mainstream Potential, published this month in BioPharm International, NAI President Rita E. Numerof, Ph.D. and Managing Partner, Michael N. Abrams, M.A. explore the implications of the orphan drug approach for pharma companies considering adopting this strategy.