If the US Congress continues to target trials at Chinese military hospitals, the impact could be relatively small, while a broader focus could upend multinational development programs. 

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With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be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

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked.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

The agency’s Antimicrobial Drugs Advisory Committee suggested a more limited indication for Interum’s antibiotic for uncomplicated urinary tract infections than the sponsor proposed, though it wasn’t necessarily clear committee members favored approval.

Teva requested the change to the draft guidance, arguing that otherwise the meetings would be “completely one-sided conversations.”

CBER Director Peter Marks said recent leaps in gene therapy science have not been matched by more affordable manufacturing technology and that the field needs to set a cost-effectiveness target. 

The European Medicines Agency’s safety committee has concluded that the benefits of medicines containing metamizole continue to outweigh the risks but that new measures are needed to minimize the serious outcomes of agranulocytosis.

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

Glenmark Pharmaceuticals agreed to pay $25m in the US to resolve its alleged liability under the False Claims Act linked to price-fixing allegations.

The US FDA is returning to the pre-COVID in-person advisory committee format and the agency suddenly has a flood of meetings lined up for the fall.

If the US Congress continues to target trials at Chinese military hospitals, the impact could be relatively small, while a broader focus could upend multinational development programs. 

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.