Under current law, drugs or biologics must receive rare pediatric disease designation before 1 October to be eligible for a priority review voucher. The sunset provisions have led to a spike in designation requests at the FDA and a push by pediatric and rare disease advocates for an extension, either through a continuing resolution or reauthorizing legislation.
The clock is once again running down on the US Food and Drug Administration’s rare pediatric disease priority review voucher program, and congressional action is needed within the next few weeks to temporarily extend the popular incentive.
The FDA has been dealing with an influx of requests for rare pediatric disease designation before the program’s sunset provisions...
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Brazilian regulator Anvisa is relying on the decisions of its foreign counterparts as a means of ensuring more quality medicines are made available in the country.
The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.
The UK regulator approved 16 medicines containing a new active substance in the first half of this year, while the European Commission issued marketing authorizations for 20 NAS-containing drugs.
The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.
The US FDA Commissioner pushed for a rebalancing of the US’s pharmaceutical supply chains while also stressing that there is a role for US-China commerce that would be risky to compromise.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
