Non-profit biotech founder says permanent pediatric rare disease priority review voucher program will boost incentive, but FDA is concerned about resource drain vouchers cause.
Rare disease advocates want to add more certainty to the pediatric rare disease priority review voucher program, although that may put them at odds with the US FDA.
The pediatric rare disease priority review voucher program has generated hundreds of millions for sponsors, but the program will sunset in October 2020, potentially limiting investment in the space.
The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.
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.
Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.
The budget authority total, which is significantly higher than the amount floated in an April OMB draft document, suggests there may have been successful lobbying to increase FDA funding.
In this first in a series of articles looking at the potential impact of the MFN drug pricing policy on European pharmaceutical markets, EUCOPE’s Alexander Natz tells the Pink Sheet why the US policy underscores the importance of confidential net pricing.
The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.
