CDER Planning Guidances On Single-Trial Approvals, Real-World Evidence
US FDA drugs center’s ambitious guidance agenda lists 105 new or revised guidances, most of which carried over from the prior year; new to the list are three guidances each on real-world data and individualized antisense oligonucleotide drug products, and a document focused on demonstrating efficacy with a single adequate and well-controlled trial.
You may also be interested in...
Sponsor preferred a tropical disease voucher because it could be redeemed for a supplemental application, but the FDA awarded a voucher under the medical countermeasures program, which prohibits redemption for supplements; agency says it takes a case-by-case approach to deciding whether tropical disease and rare pediatric disease vouchers can be redeemed for supplements.
Draft guidance will help sponsor-investigators working on individualized gene therapies. The focus on basic administrative and procedural aspects of interaction with the FDA leaves many policy and commercialization questions about how these personalized investigational products should be studied unanswered.
The first biosimilar with interchangeability status compared to its reference will be entitled to one year of exclusivity, a largely overlooked advantage in the early days of the US biosimilar market.