The Business Group on Health survey also highlights employers’ complicated attitudes on prescription drug rebates in their health benefit programs.

With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.

Under current law, drugs or biologics must receive rare pediatric disease designation before 1 October to be eligible for a priority review voucher. The sunset provisions caused a spike in designation requests and a push by pediatric and rare disease advocates for an extension.

New therapies in the wings include two candidates for Niemman-Pick disease type C, a first-in-class antipsychotic, a targeted oncology therapy under Real-Time Oncology Review, and a new drug for gastroparesis.

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked.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.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

Pink Sheet reporter and editors discuss the impact of a US and EU drug approvals analysis that found the US FDA still clears many novel products first, including most new cell and gene therapies and cancer treatments.

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

Pink Sheet reporter and editors discuss the potential fallout if the US FDA’s rare pediatric disease priority review voucher program is allowed to sunset, as well as an upcoming agency advisory committee meeting on potentially banning compounding of the active ingredient in the now-withdrawn pre-term birth prevention drug Makena.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add seven new products, including Winrevair, MSD's treatment for pulmonary arterial hypertension.

The Democratic Republic of Congo, the country worst hit by the mpox crisis, has now received the first batch of vaccines for the disease. Meanwhile, the World Health Organization is expected to complete its review for emergency use listing of mpox vaccines soon.

Medicines regulators in the EU have “much to gain” from using AI models in their processes, but this technology must be used in a “safe and responsible” way, says the European Medicines Agency.

The US Medicare agency is encouraging use of observational trials to fulfill Coverage With Evidence Development requirements, but details will await an upcoming guidance on “fit for purpose” studies.

The Business Group on Health survey also highlights employers’ complicated attitudes on prescription drug rebates in their health benefit programs.

A second pivotal study may be enough to push Iterum’s antibiotic through its second FDA review, but data gaps raised concerns about inappropriate prescribing contributing to antimicrobial resistance and could limit marketing differentiation.

Despite its legal stay of execution, lack of confirmatory data means the European Commission’s decision for market removal is likely to stand.

MSD welcomes the decision but says the drug should have gone through NICE’s Highly Specialized Technologies route that allows more flexibility regarding uncertainty around rare disease drugs.

South Korea has yet to come up with detailed plans for a major national biopharma cluster, but is considering a virtual approach to connect existing industry hubs nationwide. At a recent policy forum, experts discussed related issues including governance and insufficient investment, as well as the need to attract foreign firms and capital to compete with rival clusters in Asia.

The going rate to buy a voucher for a faster US FDA application review is about $100m, but the France-headquartered firm received considerably more from an unnamed big pharma.