Bill would enable two-year provisional approval of drugs to treat life-threatening illnesses, such as ALS, based on substantial evidence of safety and early evidence of efficacy. At a Senate committee hearing, rare disease advocates said it would remedy the FDA’s inconsistent application of regulatory flexibility, but a bioethicist cautions it would lower the bar for approval.
A provisional drug approval pathway is needed for serious, life-threatening conditions that lack good biomarkers and cannot use the US Food and Drug Administration’s accelerated approval pathway, rare disease advocates said in urging Congress to pass the Promising Pathway Act.
Patients, advocacy group representatives and industry representatives who spoke at a 26 October Senate Aging Committee hearing, and at a...
Exploring How Everyday Industrial Compounds Enter Our Systems, Their Health Implications, and the Ongoing Quest for Safer Alternatives .
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.
Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.
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.
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.
