Orphan Drug Rules Hurt Personalized Meds, Follow-ons.

Washington Editor

WASHINGTON u Orphan drug rules intended to encourage the development of treatments for rare diseases are creating an uneven playing field for follow-on biologics and personalized medicine.

Enacted in 1983 when blockbuster drugs seemed to be the ultimate goal for pharma, the Orphan Drug Act offered incentives, including seven years of exclusivity and a tax credit, for developing treatments for diseases or conditions that affected fewer than 200,000 people in the U.S.

Today's regulatory framework makes those benefits out of bounds for branded follow-on biologics and some personalized medicines intended to treat rare diseases. While no one is asking for exclusivity, the other benefits would even the odds and increase the competition in rare disease markets.

For instance, several follow-ons and plasma proteins approved …