Pathways & Standards

The European Commission’s new biotech and biomanufacturing hub will make it easier for innovative biotech companies to find legislative documents, funding information and guidance to help them bring new products to the market.

The UK’s drug regulator is developing a “clear and streamlined” regulatory pathway for individualized cancer mRNA immunotherapies.

The US FDA faces many communications challenges with vaccine skepticism rampant. Is doing everything right going to be good enough?

The US FDA wants a smoother transition to newer “low global warming potential” propellants in metered dose inhalers than the prior transition away from CFCs.

The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.

Companies that are admitted to the UK’s revised innovative licensing and access scheme will be mandated to work with the regulator on developing a target development profile for their product.

The inspector general said the council should be more involved in specific approval decisions when there is internal disagreement, but FDA said that would preclude some individuals from adjudicating a subsequent withdrawal proceeding.

Two recent accelerated approval guidances describe the FDA’s policy expectations for trial timeliness, but recognize the need for case-specific determinations. Regulatory actions in 2024 show the FDA is willing to delay application filing or approval over confirmatory trial status.

Pink Sheet editors and special guest Michael McCaughan of Prevision Policy discuss Robert F. Kennedy Jr.’s first act as HHS Secretary, former FDA Commissioner Scott Gottlieb’s suggestion that industry extend the existing user fee agreement rather than negotiate a new one, and President Trump’s policies impacting FDA advisory committee schedules and staffing.

Given the Trump Administration’s intense focus on cutting costs and increasing efficiency, industry and the FDA should focus on what is and is not working in PDUFA VII when negotiating the next agreement, lobbyists said at the BIO CEO and Investor Conference.

Experts worry new restrictions on remote work, plans to eliminate certain employees’ job protections and workforce cuts will make the FDA a less attractive employer and hurt pharma’s efforts to get innovative medicines to patients.

Top areas for potential 2025 approvals were shaped by R&D focused on increasingly tightly targeted therapies, including the eight new candidates to join the still-burgeoning kinase inhibitor class and RNA interference, as well as many varieties of antibodies.