Fresenius Kabi’s Omegaven is most recent of 13 approvals, including three with breakthrough therapy designations, where FDA has relied on real world evidence to make a regulatory decision about efficacy.
The exigencies of ultra-orphan drug development have provided the early testing ground for the US FDA’s limited use of real-world evidence (RWE) in approval decisions, resulting in 13 approvals identified by the Pink Sheet that relied on external or historical controls to place single-arm efficacy trials in context or derived data from case reports and expanded access protocols.
FDA’s most recent approval using RWE – the July 27, 2018 clearance of Fresenius Kabi AG’s I.V. lipid emulsion...
Exploring How Everyday Industrial Compounds Enter Our Systems, Their Health Implications, and the Ongoing Quest for Safer Alternatives .
With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.
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the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance.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 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.
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.
