Question

"Orphan drugs are pharmaceutical and biological products that target diseases affecting under 200,000 patients in the US. What is not an incentive given to manufacturers to encourage the development of orphan drugs?"

-Clinical research subsidies are provided
-No other version of the drug will be approved during a twelve year market exclusivity
-Tax incentives are given to the manufacturer
-Smaller patient numbers are required for clinical trials
-PDUFA fees are waived

Answer 1

Incentives for orphan drug development include clinical research subsidies, tax incentives, market exclusivity, and smaller trial sizes. PDUFA fees being waived is not a typical incentive.

Step-by-step explanation:

The incentives given to manufacturers to encourage the development of orphan drugs, which are pharmaceutical and biological products targeting diseases affecting fewer than 200,000 patients in the US, include clinical research subsidies, tax incentives, market exclusivity for twelve years, and the requirement for smaller patient numbers in clinical trials. However, PDUFA fees being waived is not typically one of these incentives. The Prescription Drug User Fee Act (PDUFA) fees are required to be paid by companies to the FDA when they submit an application for a drug to be approved, and these fees are not generally waived for orphan drugs.

The conversation around the development of new drugs often touches on whether pharmaceutical companies have an ethical obligation to prioritize the public interest and focus on high-utility drugs rather than just profitable ones. Government subsidies are one way to entice pharmaceutical companies to develop drugs that may not have a large market but are crucial for patients with rare conditions.