North America

At the advisory committee review of Stealth’s Barth syndrome treatment elamipretide, Office of New Drugs Director Peter Stein clarified the circumstances where clinical data in a related indication could serve as confirmatory evidence for a single adequate and well-controlled study.

A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.

A growing number of future Part B candidates for Medicare price negotiation may avoid price controls in the program with injectable versions.

FDA law experts do not buy the outsourcers’ argument that the agency must go through notice-and-comment rulemaking to remove a drug from the shortage list.

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.

The rolling reimbursement reviews that Canada’s Drug Agency introduced as a pilot scheme to speed up patient access to COVID-19 products can now be used for any drug application. The move to open up the mechanism follows prompts by industry.

England’s NICE has “misclassified” HER2-low metastatic breast cancer as a medium severity disease, say AstraZeneca and Daiichi Sankyo, the co-developers of Enhertu. NICE said it would consider a rapid re-appraisal if the companies offered a “fair price” for the drug.

Three off-patent associations from both sides of the Atlantic want to strengthen their collaborative synergies and secure the supply of generics and biosimilars.