Advisory committee to discuss proposed changes to isotretinoin REMS to minimize burden and treatment delays while preventing risk of fetal exposure. Agency recommends against at-home pregnancy tests.

Pink Sheet reporter and editors discuss Moderna CEO’s Stéphane Bancel’s defense of his company and COVID-19 vaccine pricing at the Senate Health, Education, Labor and Pensions Committee, the NIH denying a petition to use government march-in rights for Xtandi, and Tofersen receiving a positive US FDA advisory committee recommendation for use in ALS.

Draft guidance gives recommendations for conducting one randomized controlled trial to generate the evidence for accelerated approval and confirm clinical benefit.

A report from the EMA summarizing the mid-term achievements of its Regulatory Science Strategy to 2025 covers health technology assessments and a range of other topics.

Plans for the overhaul of the EU’s medicines framework are missing key analysis on the effect of reductions in data and market protection on the pharmaceutical industry, despite an abundance of studies on how such incentives influence company decisions on whether to develop new products, say lawyers from Sidley.

EU regulators have issued a new Q&A document on the key aspects that sponsors of complex trials should consider when making new submissions or moving ongoing studies to the Clinical Trials Information System.

Moderna’s time in the hot seat of the US drug pricing debate went about as well as it could for the company. The firm astutely set a tone of an All-American success story – but the hearing also showcased other ‘only in America’ themes.

A report from the EMA summarizing the mid-term achievements of its Regulatory Science Strategy to 2025 covers health technology assessments and a range of other topics.

The Biden administration decision not to pursue ‘march in’ proceedings for Pfizer/Astellas’ Xtandi sounds like good news. The reality is that the prostate cancer therapy is likely to face a more certain federal pricing intervention in the first year of the new Medicare ‘negotiation’ process.

Draft guidance gives recommendations for conducting one randomized controlled trial to generate the evidence for accelerated approval and confirm clinical benefit.

Any program changes would not occur until after a thorough assessment, the agency said.

The European Medicines Agency says that when it comes to products developed under its priority medicines scheme, dialog is crucial to ensure that the data generated for marketing applications are sufficiently robust and mature and facilitate a timely review.

Novo Nordisk has been given a two-year suspension from membership of the Association of the British Pharmaceutical Industry for breaching the association’s code of practice.

CMS chose the prices of therapeutic alternatives and these competitors’ comparative benefits as the ‘foundation and starting point’ for setting an initial offer for drugs subject to negotiation, ahead of other factors Congress laid out it could consider that are more singularly focused on the specific drug at issue.

Alzheimer’s product is following a different playbook than Aduhlem, reflecting better data – and hard-earned lessons. Eisai estimates that 80-90% of veterans who are eligible for Leqembi treatment based on the FDA label would also fit the VA’s criteria.

Commissioner Califf underscores need to keep inspections systems ‘tuned up’ to prevent manufacturers from ‘losing their edge’ on quality.

The International Pharmaceutical Regulators Programme explains how manufacturers of cell and gene therapy products can assess and mitigate the potential risks associated with product raw materials.

Agency’s proposals for pandemic preparedness reauthorization would build on CARES Act provisions to gain greater visibility into global pharmaceutical supply chains, while also seeking deeper understanding of manufacturing quality management activities. Also included in FY 2024 budget request: long-sought drug destruction and drug recall authorities.