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FDA and clinical trials industry experts focus new attention on rare disease studies

The Association of Clinical Research Professionals (ACRP)

With the recent launch of the U.S. Food and Drug Administration’s (FDA’s) Rare Disease Endpoint Advancement (RDEA) Pilot Program, the clinical research enterprise gains new support for efforts aimed at efficacy endpoint (or clinical outcome) development and the timely approval of drugs and biological products that treat rare diseases, including rare diseases in children.

This FDA action makes it all the more timely that several articles appearing in the forthcoming October 2023 issue of ACRP’s Clinical Researcher journal focus on various aspects of rare disease studies that many research professionals have seldom or never had to think about.

In his article on “Ensuring Timely, Secure Delivery of Therapies for Rare Diseases,” Dea Belazi, PharmD, MPH, President and CEO of AscellaHealth, notes that clinical trial teams are increasingly focused on the development of specialty drugs and cell and gene therapies to provide an estimated 300 million people worldwide with potentially lifesaving, curative treatment for the 7,000 known rare diseases.

“Today, there are [approximately] 200 clinical trials under way in Phase III targeting patients with rare, complex diseases who often face death, disability, or poor quality of life from lack of effective treatments,” Belazi writes. “As specialty drugs continue to be the main category in the FDA’s pipeline for new approvals as of mid-2023, and it has been projected that 13 new cell or gene therapies could be approved in the U.S., Europe, or both by the end of 2023, optimizing the supply chain must a priority throughout the pre-commercialization phase of drug development.”

Belazi goes on to examine how, among the myriad challenges to researchers are the unique requirements of these new, innovative therapies and the burdens tied to establishing protocols for flexible, agile supply chain logistics in support of their research and development. The need for risk management is paramount, he notes, given the multiple stakeholders involved in the supply chain. Read more …