Ensuring Timely, Secure Delivery of Therapies for Rare Diseases

Clinical Researcher—October 2023 (Volume 37, Issue 5)

GOOD MANAGEMENT PRACTICE

Dea Belazi, PharmD, MPH

Clinical trial research teams are increasingly focused on the development of specialty drugs and cell and gene therapies (CGTs) to provide an estimated 300 million people worldwide with potentially lifesaving, curative treatment for the 7,000 known rare diseases. Today, there are more than 2,000 clinical trials under way{1} with 200 in Phase III targeting patients with rare, complex diseases who often face death, disability, or poor quality of life from lack of effective treatments. As specialty drugs continue to be the main category in the U.S. Food and Drug Administration’s (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,{1} optimizing the supply chain must a priority throughout the pre-commercialization phase of drug development.

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