Orphan Drug Designation
A strategic approach creates opportunity for time efficiencies
Celebrating the Orphan Drug Act’s 35th Anniversary, the Orphan Drug Act is an essential piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases and precision medicine impacting disease sub-populations.
On average, a company’s stock price increases by 3.36% after announcing the designation, increasing the company’s value. The results are more pronounced for oncology drugs and small companies.
Our experience and understanding of how the FDA views orphan applications and structuring a development program to deliver the data and rationale to satisfy the FDA can substantially reduce the review period and increase approval.
Demonstrating orphan status may be particularly challenging due to limited prevalence data for ultra-rare and precision medicine sub-population diseases.
Case Study
The OOPD had previously rejected this orphan petition twice due to a lack of incidence and prevalence data on the targeted sub-population.
The OOPD had requested evidence limiting the use of the treatment outside the sub-population included in the orphan petition. BBCR submitted the petition for the first time showing the precise cut of the patient sub-population target of the treatment in development, clarified OOPD’s concerns, and the petition was approved without additional questions.