Revised draft guidance includes recommendations on preparing for FDA inspections, considerations for real-world data and digital health technologies, and the need for service level agreements between sponsors and IT providers.

The UK pharma industry body has set out bold proposals for a new drug pricing agreement from 2024, but not everyone is convinced. Generics companies are pressing for more of a say in the negotiations, while patient groups want to know more about how the deal would help improve access to medicines.

US FDA Commissioner Califf praises both Project Pragmatica and the ‘very powerful’ partnership between OCE Director Rick Pazdur and National Cancer Institute Director Monica Bertagnolli.

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

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

FDA seeks advisory committee input on whether there is ‘convincing evidence’ to support regular approval for treatment of SOD1-ALS patients even though lone Phase III trial failed its primary endpoint; companies filed for accelerated approval on the basis that a reduction in plasma neurofilament light chain is reasonably likely to predict clinical benefit.

Health technology assessment institute NICE has reversed its provisional rejection of Oxlumo for treating the rare disease, primary hyperoxaluria type 1.

The US Medicare agency intends to treat all dosage forms of the same active ingredient as a single drug for purposes of its new price negotiation authority. The approach will have implications for product branding and line extension strategies.

Calculating a ‘fair price’ for new versions of already negotiated moiety is also one of several that CMS is requesting input on in its recently released initial guidance on the negotiation program.

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.

The European Medicines Agency has responded to an analysis that argued that generating evidence for decision making in health care using observational real-world data, and thus potentially replacing randomized controlled trials, “is the wrong remedy for the challenges in drug development.”

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.