Recent and upcoming US FDA advisory committee meetings and a summary of topics covered. 

The EMA hopes that the use of a single portal by drug companies and EU/EEA inspectorates to coordinate on good manufacturing practice inspections will drive greater efficiency.

The European Medicines Agency also expects to reach some conclusions on the use of MAb-based COVID-19 treatments by the end of October.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the European Union, and updates on EU marketing authorization changes recommended by the CHMP.

Age limit wasn’t part of advisory committee’s vote on third shot of the COVID vaccine, but aligns with concerns about myocarditis in younger recipients.

US FDA’s John Concato cites rigorous collection of data and study design in getting Prograf lung transplant indication. Use of real-world evidence in Opdivo trials had mixed results, BMS exec notes.

Advisory Committee on Immunization Practices fails to pass an interim recommendation endorsing third dose of vaccine in health care workers and others at risk of occupational or institutional exposure, with panelists raising concern that the target population was overly broad.

Members of Congress, companies and government agencies want the Patent Office to change policies that they say impede generic drug competition. The latest effort is focused on PTO’s discretionary denial of inter partes review petitions, which is the subject of a bipartisan congressional letter and cert petition to the US Supreme Court.

Only about one-quarter of the 40 gene therapies projected to launch by the end of 2025 would have potential US patient populations of more than 50,000. But a handful of treatments could target millions of individuals.

With Intarcia's request, the rarely used mechanism could now produce two hearings this year, but outcomes of the few other times the process has been undertaken should not generate a lot of confidence for sponsors that this a path that they want to go down.

Cancer drug developers should come to the agency early with a package of clinical trials to support accelerated approval and confirm clinical benefit rather than with plans to sequentially conduct such studies, Oncology Center of Excellence’s Richard Pazdur says; he also suggests closer scrutiny ahead of whether postmarketing trials are being conducted with ‘due diligence.’

US FDA has pushed sponsors away from the traditional dose-finding designs for cancer drugs in recent years. But academics continue to make a case for keeping the 3+3 design with some improvements.

Drug and medical device company payments to doctors and hospitals for non-research related activities dropped for the first time in 2020 since CMS’s Open Payments database began collecting the information. 

Pink Sheet reporters and editors discuss Bluebird Bio’s decision to pull its gene therapy business from Europe, Eli Lilly potentially pushing regulatory boundaries with its Olympic TV spots, and the CMS decision to scrap the Trump aministration’s most favored nation reimbursement rule.

Novartis gets some praise for being willing to embrace cost-effective drug prices, but health policy analysts are skeptical the industry would truly adopt the idea for its best-selling drugs, particularly if the appropriate guardrails were put in place to ensure broader budget impact was controlled.  Holes were also poked in the way Novartis determined the total cost of drug utilization management on the US health system. 

After decades without an approval pathway for excipients, FDA pilot program paves the way for innovation.

The EMA hopes that the use of a single portal by drug companies and EU/EEA inspectorates to coordinate on good manufacturing practice inspections will drive greater efficiency.

Summary judgment puts 20-year-old effort to define ‘inordinate’ out-of-state drug compounding back on slow track.