Dr. Jonathan Watanabe Appointed to National Academies of Sciences, Engineering, and Medicine Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union
Dr. Jonathan Watanabe, Founding Associate Dean of Pharmacy Assessment and Quality of the UC Irvine School of Pharmacy & Pharmaceutical Sciences, has been appointed to the Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union. The committee was established, at the request of Congress, by the National Academies of Sciences, Engineering, and Medicine (NASEM). With the formation of this committee, its members and other stakeholders aim to change the way that rare disease treatment is viewed, developed, and administered.
“While rare diseases affect roughly 30 million people in the US, only about 5% have an FDA-approved treatment,” stated Dr. Watanabe. “Based on information gathering and internal deliberations, the committee will develop a report with its findings, conclusions, and recommendations for actions that Congress, federal agencies, the pharmaceutical industry, and nongovernmental organizations can take to support collaborative efforts for bolstering care for rare disease populations.”
Within the committee, Dr. Watanabe is among a group of fellow authorities on pharmacy, drug development, and policy. He was selected for the committee due to his expertise on rare diseases and precision medicine.
In the past, Dr. Watanabe has served on the NASEM Committee on Making Medicines Affordable and their workshop series on Examining the Impact of Real-World Evidence on Medical Product Development, as well as published original research on the increase in high-spend medications covered by Medicare Part D that serve small populations in the US.
His role on the committee is to offer his perspective and advice on real-world data use, clinical research, Orphan Drug Act policy, precision medicine, population health, and policy and regulatory efforts.
“I hope to foster a more sustainable approach to rare disease research and treatment. I’d like to see improved awareness of regulatory approaches for rare disease and orphan drug treatments,” Dr. Watanabe shared. “There is much to be gained and accelerated if there was more effective sharing of knowledge and best practices from agencies around the world, as well as coordination of data approaches.”
He continued, “It’s an honor to be a part of an international effort to enhance development and access to transformative therapies. It’s a fascinating and growing challenge now that, paradoxically, rare diseases are becoming effectively more common with advances in genomics.”
The committee will periodically meet in public sessions via Zoom, which are free to attend. More information about the committee and upcoming events can be found here.