Duke-Margolis Center for Health Policy
Agency’s plan for advanced manufacturing seeks more harmonization, while also seeking to codify internal practices with guidance and training.
The agency’s plan for advanced manufacturing seeks more harmonization, while also seeking to codify internal practices with guidance and training.
The current regulations were written when clinical trials involved far fewer entities, and a certain amount of ‘contortion’ is necessary when sponsors are trying to introduce decentralized, community-based and other modern elements, the FDA’s Kevin Bugin says in an interview with the Pink Sheet.
Gestational age and a binary, neonatal composite endpoint are insufficient to assess outcomes that are important to babies and families, clinicians and researchers say, suggesting development of continuous variables or scales with weighted components and a comparison of outcomes among newborns in gestational age ‘buckets.’
US FDA’s oncology advisory committee will discuss two meta-analyses supporting use of MRD as a surrogate endpoint to support accelerated approval in multiple myeloma trials.
Anticipating a ‘tsunami of therapies’ for rare diseases, commissioner says the agency will have to think of creative approaches and employ regulatory flexibility for them. FDA considers copying the oncology center’s Project Facilitate for expanded access to other diseases.
Sponsor concerns about commercial confidential information may be a sticking point in getting patient groups the desired data on when and how the FDA uses patient input in regulatory decision making.
Move over big data, a biostatistician calls for more people to think small for rare diseases and FDA officials indicate openness to the trial approaches laid out.
With the only FDA-approved treatment off the market, the FDA is looking for ideas for development of new preterm birth treatments.
Experts discuss how master protocols can help produce more comparable real-world evidence and draw more investigators into studies. FDA CMO Hilary Marston says payers must be more involved in discussion of the value of real-world data.
Panelists at a recent Duke-Margolis meeting cite growing use of native ads and patient influencers, as well as the limited attention given to presentation of risk information, in calling for a re-examination of how pharmaceutical promotion on digital platforms is regulated.
US FDA will be transitioning from the ‘diversity plans’ outlined in recent guidance to the new, legislatively directed ‘diversity action plans’ by the end of 2023. The oncology group is leading the way in gathering early perspectives on industry approaches.
Whether subjects can use their own personal devices, how to manage mid-study technology updates, and the extent to which data gathered will be shared with patients in real-time are all issues that sponsors, investigators and regulators need to consider.