North America

Former Food and Drug Law Institute CEO Amy Comstock Rick will take on patient engagement for the US FDA Rare Disease Hub as director of strategic coalitions.

US FDA’s Rare Disease Innovation Hub should shepherd the practical transformation of the diverse wealth of patient-generated data held by advocacy organizations into information for regulatory use, Reagan-Udall public meeting hears.

Republican presidential candidate Donald Trump says he will let Robert F. Kennedy Jr. “go wild on medicines” if he wins the White House. That could spell challenges for the US FDA in 2025.

Another record-breaking year for novel approvals looks out of reach, but the six novel agents with November goal dates show the continued strength of rare disease drug development.

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.