Six Essential Tips For Developing Orphan Products

By Ed Miseta, Chief Editor, Clinical Leader

Developing orphan products is not an easy endeavor, but one that pharma may be getting more involved with as a result of the patent cliff and erosion of the blockbuster model. Knowing how to select the right development partner can greatly accelerate the approval of orphan products, reduce the cost, and result in a better sponsor/CRO relationship. David Shoemaker, SVP of R&D for contract research firm Rho, notes Pharma firms may not be looking for a blockbuster, but would still like to find drugs that can serve a large population.

Shoemaker notes orphan product patients are rarer, recruiting them may have to involve patient advocacy groups, and the science is more esoteric. He also believes financial incentives offered by the FDA have not sufficiently enticed most pharma companies, and for that reason Big Pharma has not been anxious to get involved in the orphan space. “Big Pharma is certainly in a difficult situation, and is looking for ways to grow drug pipelines,” he says. “This has led to pharma giving more money to academic institutions in addition to putting more scientists in university labs. The largest pharma firms will continue to evolve into sales and marketing companies, and will increasingly rely on technologies developed by government, academia, and small companies.”