Legislation

The FDA's interim leadership takes shape, but the sense of transition chaos still lingers.

Pink Sheet reporter and editors discuss the uncertainty created by the Trump Administration freezing most communications at the Health and Human Services Department, new travel and telework bans, as well as the delay in announcing an acting FDA leader and the impact of new executive orders on the FDA and CMS.

The cell and gene therapy field has reached an inflection point in the US as the second Trump Administration begins and advocates argue the sector could fit well with the “MAHA” agenda.

The strategy includes a committee made up of pharmaceutical industry representatives and ministers to discuss the impact of regulatory proposals.

The White House is expected to name Sara Brenner, chief medical officer for in vitro diagnostics at CDRH and a veteran of the first Trump administration, as acting commissioner, but the lack of a formal announcement is creating uncertainty. New executive orders on telework and the senior executive service also could impact agency staff.

Pink Sheet editors consider ex-FDA officials’ advice for the Trump Administration on implementing FDA reforms, comments CDER Director Patrizia Cavazzoni made before her departure from the agency about wanting to release review documents for applications that received complete response letters, and diverging trends between CDER and CBER novel application approvals.

Robert Califf’s final appearance on Capitol Hill as US FDA commissioner, a 5 December Senate hearing focused on the agency’s food and nutrition functions, was an appropriate coda to his tenure and preview of things to come under the Trump Administration.

The European Commission's 100-day countdown to deliver a Critical Medicines Act, aimed at improving the EU's resilience to supply chain disruptions and price volatility, began last December. Meanwhile, negotiations over the proposed package that will reform the over 20-year-old EU pharmaceutical legislation are now in the hands of the Council of the EU. The Pink Sheet examines what to expect for these key pieces of legislation this year.

The UK’s “world-first” tailored regulatory framework for point of care manufacturing is set to enter into force this summer.

Former FDA commissioners Mark McClellan and Scott Gottlieb, former acting commissioner Janet Woodcock and current commissioner Robert Califf offered advice on successfully implementing reforms and preventing a mass exodus of FDA employees as inklings emerge that the Trump team is already engaged on this front.

EU joint clinicals assessments introduced under the HTA Regulation have now gone live for cancer medicines and advanced therapies.

Stakeholders have until 15 January to respond to a European Commission proposal that aims to harmonize pharmacovigilance activities by marketing authorization holders, national competent authorities and the European Medicines Agency.

Pharmaceutical industry says the transition from paper to electronic product information, as part of EU pharmaceutical reforms, should not be held back by member states' readiness.

6 January 2025 will be remembered in Washington, DC, as the date Congress certified Donald Trump’s second presidential election victory and a snowstorm shut down most of the city, but for FDA watchers it will be the day the agency released more than two dozen new draft and final guidances.

The agency’s actions may signal its optimism about near-term reauthorization despite the PRV program and other bills aimed at tackling rare and childhood diseases not making the December 2024 government spending bill.

Despite GOP criticism of the Part D stabilization program and its budgetary impact, the Trump Administration may decide to keep it in part to avoid blame for a premium increase.

The FDA will stay open, but its rare disease priority review voucher program will wind down after not being renewed on 20 December. PBM reform and other industry priorities also were nixed at the last minute in an effort to avoid a government shutdown.

President-elect Trump is backing a government funding bill that cuts pharma priorities, but it remains unclear whether the plan will garner enough votes to prevent a government shutdown.

A Catalyst fix, a pediatric rare disease voucher reauthorization that should prevent future workload imbalances for FDA, and a boost for out-of-state coverage in Medicaid are among Congress’s year end commitments to pediatric and rare disease patients that now seem in doubt.

As application of the Health Technology Assessment Regulation nears, the European Commission has launched two new websites to help developers kick start the joint clinical assessment process and request a joint scientific consultation.