Pink Sheet - News And Expert Analysis On Pharma Policy And Regulation since 1939

An enhanced role for patients in the European Medicines Agency was a key proposal within the EU pharmaceutical legislation overhaul – but patient groups warn this provision could be scrapped or weakened due to ongoing negotiations.

The buyout excludes reviewers, but not project managers. Experts said product reviews likely would be harmed if review team-specific project managers could take the buyout.

The UK drug regulator has made a number of improvements to its innovative licensing and access pathway, including a more “core” role for National Health Service partners, which is expected to facilitate flexible commercial discussions around drug reimbursement.

The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment. 

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.

In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.

Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.

Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

A new collaboration of health technology appraisal bodies in the US, England and Canada could lead to better alignment on HTA methodology in the future.

The European Commission has released three exercises to help pharma companies prepare for joint clinical assessments under the new EU Health Technology Assessment Regulation. Experts explain how manufacturers should interpret these and what their limitations are.

Company dossiers submitted for EU-level joint clinical assessments under the Health Technology Assessment Regulation will be made public. This could conflict with other strategic business decisions and should be planned for in advance, experts say.

Retiring CDER Deputy Director Douglas Throckmorton spoke to the Pink Sheet ahead of his early 2025 retirement about regulatory flexibility, AI and other topics.