The FDA’s guidance and policies made major changes this year

FDA’s Policy Initiatives

The FDA’s guidance and various policies were at a pace this year. Neverthless the guidance on vaccines and rare disease have attracted most of the deals, with policy shifts focused on enhancing the FDA's efficiency. Alongside the inshoring of the U.S. manufacturing was also considered. Covering rare diseases, focusing on animal testing requirements and vaccines, the biopharma was engaged in contributing the utmost to the healthcare sector.

The biopharma in the headlines of the policy, as Health Secretary, Robert F. Kennedy Jr. and his brilliant team at HHS have proved themselves on the CDC, National Institutes of Health and FDA. Most of the FDA’s 2025 guidance has aimed at leveraging the path of gene and cell therapies to the market, mainly those planned for ultra-rare and rare conditions. Each headline comes with controversy, but what stays unnoticed is the need for effective and reliable therapies for rare diseases.

Changes are the new preparedness for 2026

The FDA watchers and Analysts noticed the sync developed in this space that 2025 will pass on to the next year. The managing director of Equity Research at Mizuho Securities, Graig Suvannavejh, said, “I feel the perspective and that we’re seeing through the FDA’s observation is specifying the rare orphan diseases as an area to engage and invest in for the investors and biopharma.”

“This interest will linger into 2026.” As per the reports, the FDA will start with the requirement of only one pivotal trial, contrary to the standard two. “This will make it easy for rare orphan diseases.”

The next key area of focus for the HHS under the Kennedys’ experience in antivaccine activism has always been in the vaccines area. The radical departure from previous practices, an HHS spokesperson said, “The Health Department declared that it needs the testing of all new vaccines in the placebo-controlled trials before approval.”

This month, the Kennedys re-built the CDC Advisory Committee on Immunisation Practices, initiated another radical departure from the norm. This radical departure is a vote for the late hepatitis B vaccine from birth to two months of age, for a few newborns.

Hatch-Waxman and Biologics chair at Polsinelli Law Firm, Chad Landmon, said, “Kennedy is determined towards a lot of this area covering vaccine staffing, policy and everything based on it. Now, it’s going to be interesting to witness where it leads and whether the same set of individuals will continue in those roles.”