Based on designations, FDA already could have enough products in the pipeline to trigger the mandatory program stop and assessment.
While FDA has issued only one pediatric rare disease priority review voucher, the program already has generated enough industry interest that the agency soon may be forced to stop it temporarily.
The new PRV program, created in the 2012 FDA Safety and Innovation Act, allows sponsors receiving approval of a product treating a rare pediatric disease to gain a voucher
Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.
The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.
US trade and tariff leverage might successfully push European and other countries to pay more for medicines, but if not, President Trump’s executive order includes potential US drug withdrawal and other options to entice industry to lower US prices.
Decision in AstraZeneca’s lawsuit against the Health and Human Services Department is a precedent-setting victory for the government and a blow to manufacturer efforts to block the price negotiation program.
Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.
Pink Sheet editors discuss the appointment of Vinay Prasad as the new director of the Center for Biologics Evaluation and Research and revelations that FDA layoffs now are hindering development of guidance documents.
Agency staff once again have access to the Lexis-Nexis legal and news database, but are still waiting for access to journals, and laid off librarians have not been reinstated.
