RTW

Group Calls for FDA Consistency, RTW Sends Letter to Government Leaders

April 1, 2026

"The Rare Disease Advocacy Biotechnology and Investor Coalition sending a letter today to HHS Secretary Robert F. Kennedy Jr., the Centers for Medicare and Medicaid Services Administrator Dr. Mehmet Oz, and FDA Commissioner Dr. Marty Makary. The RTW Institute has been calling for flexibility and consistency at the FDA as the regulator reviews applications for new rare disease treatments."

Rare disease advocacy group urges Trump administration to restore FDA clarity

April 1, 2026

April 1 (Reuters) - A rare disease advocacy coalition on Wednesday urged the Trump administration to restore regulatory clarity as it considers new leadership at the U.S. Food and Drug Administration's Center for Biologics Evaluation and Research.

9 Months In, FDA's New Priority Voucher Program Still Clouded With Uncertainty

March 23, 2026
“In theory, the benefits look great,” Kinnari Patel, CEO of think tank RTW Institute, told BioSpace of the scheme, which was introduced in June 2025. “The idea of any drug that has a huge unmet medical need or need in the American population to be reviewed in one or two months, is great, full stop.”

FDA’s One Trial Policy Not a Revolution but a Potentially Risky Evolution

February 26, 2026
The fact that the policy was announced via a medical journal itself raises questions, according to Kinnari Patel, CEO of think tank RTW Institute.

“What I would like to see is all of these criteria to become a statutory requirement and/or draft guidance documents,” Patel, who previously served as head of R&D and chief operating officer at Rocket Pharmaceuticals, told BioSpace. “Because I think only when that happens, every single reviewer at the FDA is trained on it and can consistently apply it.”

The White House has asked for the resignation of a top aide to FDA chief

November 21, 2025

“The recent wave of FDA reversals, late-cycle objections, and shifting evidentiary standards across multiple rare disease programs has led to a clear increase in unexpected [complete response letters] and refusals despite expedited designations and positive prior meetings,” the Rare Disease Advocacy, Biotechnology, and Investor Coalition wrote.

Top FDA drug regulator raises alarms about expediting approvals

November 21, 2025
"The drug industry relies on a predictable FDA to understand what it needs to do to win approval of new treatments and has recently raised concerns publicly, including in a letter directed at Trump, Makary and Health Secretary Robert F. Kennedy Jr. “Mr. President, recent FDA actions risk undermining your legacy of medical innovation,” biotechnology companies, rare disease patient advocates and investors wrote, warning that China could displace America’s global leadership in the biotechnology field..."

Health care leaders' letter to the FDA: Concerns about approvals for rare disease therapeutics

November 21, 2025
The vision of RARE Hope is to drive collaborative research efforts to find a cure for Annabel and for the other AHC patients around the world, to add new research on critical brain function that could help patients with other related neurological disorders, and to provide a model of patient advocacy for defeating other rare genetic diseases.