Precision Medicines’ Impact on Orphan Drug Designation

Orphan drug designation provides critical incentives for the ODA, including significant tax credits for qualified clinical testing.

Developing drugs in minor, molecularly defined, pharmacologically relevant subpopulations with the same disease is increasingly viewed as a viable pathway for bringing drugs to market.

Thus, precision medicines where the drug’s mechanism of action is only relevant in a small subpopulation of patients with a more common disease may qualify for orphan drug incentives as an orphan subset. In 2015, new drug approvals for precision medicines targeting rare subpopulations of otherwise common diseases represented 8% of the orphan drug designations granted.

The BBCR team will empower Sponsors with medical insight, ODA application experience, strategic clinical planning, and streamlined trial design.