The US FDA Pediatric Advisory Committee uses web-posted reviews of “low safety risk” products to keep up with postmarketing monitoring requirements amid rising interest in pediatric development.

Advisory committee concerns cast clouds over Iterum’s oral antibiotic, Intercept’s Ocaliva, and perioperative immuno-oncology regimens, while CSL and Pfizer aim to take their hematology franchises in new directions.

With BIOSECURE's legislative progress on pause until after the election, a Pink Sheet infographic looks back on the Capitol Hill progress to date and looks ahead to the potential impact if it is enacted, using Evaluate Pharma data to highlight the likely holes in pharma’s supply chain.

Inflation accounted for a larger portion of user fee revenue target increases for fiscal year 2025, compared to previous years, according to a Pink Sheet analysis.

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The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked.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

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.

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

NHS England says it is proposing a “realistic but challenging uptake trajectory” that will make “this clinically and cost-effective drug” available to as many patients as possible without overwhelming health care providers.

There is “a lot of flexibility” in the European Medicines Agency’s reflection paper on the use of artificial intelligence during drug development, which is principles-driven rather than setting rigid recommendations, says the agency’s Florian Lasch.

A US FDA webinar for start-up sponsors showcased staff who sound enthusiastic about helping drug developers advance their projects, while also emphasizing the importance of early and continual engagement with the agency.

US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.

Instead of $5bn in 2025, the redesign could cost the federal government two to four times that amount, the Congressional Budget Office told Republican lawmakers. And the Part D premium stabilization demonstration will cost an additional $7bn, the office projected.

Manufacturers that do not answer all questions they are asked during joint clinical assessments under the upcoming EU HTA Regulation could have their applications deemed incomplete, says a co-chair of the JCA subgroup.

Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.

Companies dealing with commercial costings or contracts with the National Health Service for early phase clinical trials or trials of advanced therapy medicinal products should soon be able to conclude their negotiations more quickly.

The final Medicare price negotiation guidance for the next cycle includes improvements from last year’s patient listening sessions.

The FDA is developing several structures and a broad group of experts across disciplines to help craft artificial intelligence policy. But the proliferation of AI-related initiatives raises the question of who, ultimately, will make decisions about when novel applications of AI are acceptable.

The US FDA Commissioner pushed for rebalancing the US’s pharmaceutical supply chains while also stressing that US-China commerce has a role that would be risky to compromise.