Pasco Reporter

Representatives Gus Bilirakis and Doris Matsui have introduced the Scientific External Process for Educated Review of Therapeutics (EXPERT) Act. This legislation mandates the FDA to conduct externally-led science-focused drug development meetings aimed at rare diseases. Currently, over 90% of rare diseases lack FDA-approved treatments, partly due to challenges faced by FDA staff in addressing specific rare diseases and conflicts of interest among experts.

The EXPERT Act seeks to address these issues by establishing quarterly Externally-Led Scientific-Focused Drug Development (EL-SFDD) meetings. These sessions will facilitate collaboration among medical experts, drug sponsors, scientific organizations, and patient advocates. The focus will be on discussing the hurdles in developing treatments for rare diseases, exploring scientific opportunities, novel clinical trial designs, and aligning on endpoints to meet unmet medical needs. Each meeting will concentrate on a different rare disease topic, with annual reports from the FDA detailing how these discussions influence its review process.

Congressman Bilirakis stated: “We’ve repeatedly seen that tangible progress can be achieved when all stakeholders are brought together and united in a common purpose to share information, brainstorm and develop solutions.” He emphasized the importance of using this approach for tackling challenges in the rare disease community through bipartisan legislation.

Congresswoman Matsui added: “I am grateful for the steps the FDA has taken to advance their engagement with rare diseases, but there is more work to be done to ensure every rare disease patient has the hope of a successful therapy.” She highlighted the need for formalized processes that balance patients' urgent needs with ensuring medication safety and efficacy.

The introduction of this bill has been welcomed by patients, healthcare providers, physicians, and non-profit organizations. Annie Kennedy from EveryLife Foundation for Rare Diseases noted that while progress has been made in developing therapies for some of the estimated 30 million Americans living with rare diseases, significant unmet needs remain. Stacey Frisk from the Rare Disease Company Coalition expressed support for ensuring timely expertise availability during treatment development.

Charlene Son Rigby from Global Genes recognized the necessity of multi-stakeholder input in advancing scientific progress for rare disease treatments. The EXPERT Act aims to establish mechanisms providing clarity and consensus among various stakeholders involved in developing effective therapies.

This legislation is endorsed by several organizations including Everylife Foundation for Rare Diseases, Speak Foundation, Global Genes, and the Rare Disease Company Coalition.