FDA launches Rare Disease Innovation Hub

Back so soon? We have a lot to share, with FDA’s announcement of a Rare Disease Innovation Hub plus more benefits of COVID-19 vaccines and the latest data on global childhood vaccination rates. (528 words, 2 minutes, 38 seconds)

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FDA launches Rare Disease Innovation Hub to expedite treatments

The Food and Drug Administration (FDA) yesterday announced the creation of a Rare Disease Innovation Hub to “expedite development and approval of safe and effective drugs and biologics” for rare diseases.

What it will do,according to the FDA:

• “Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations.”
• “Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development.”
• “Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.”

Who runs it: Patrizia Cavazzoni, MD, Director, FDA Center for Drug Evaluation and Research, and Peter Marks, MD, Director, FDA Center for Biologics Evaluation and Research—alongside a new Director of Strategic Coalitions for the Hub (Associate Director for Rare Disease Strategy), who “will act as a single point of connection and engagement.”

Why it matters: “By elevating the focus and urgency on rare diseases at the FDA, agency senior leaders are sending a strong message to patients, caregivers, and researchers everywhere of the importance of developing newer and better medicines for every person living with a rare disorder,” said BIO President & CEO John F. Crowley.

BIO’s view: “As a parent of two children with a rare disease, I know how difficult it can be to find information—much less treatments—for a rare condition,” Crowley continued. “This announcement from the FDA, and the personal commitment and leadership of Dr. Cavazzoni and Dr. Marks, is a watershed moment for the rare disease community. BIO welcomes this important news and looks forward to engaging with the FDA and providing input into how the innovation hub can be the success that it needs to be for so many who need hope.”

More reading: We recently spoke to an ALS patient advocate and caregiver about what it will take to improve diagnosis and treatment—read more at Bio.News. 

What Else to Read Today

Study: Vaccines significantly reduce the risk of long COVID. “The percentage of vaccinated people who developed long COVID was much lower than the percentage of unvaccinated people who did,” reports The New York Times. The study, conducted with support from the Department of Veterans Affairs, reinforced the need to keep up with annual vaccines—and since vaccination doesn’t eliminate the risk of long COVID entirely, continue R&D in this area, too.

WHO: Global childhood vaccination ‘stalled’ in 2023.New World Health Organization (WHO) data finds 2.7 million more children were unvaccinated or under-vaccinated in 2023 than in 2019, with 14.5 million “zero-dose children” and only 83% of eligible children receiving their first dose of the measles vaccine. The better news: HPV vaccination rates improved, meaning fewer women and girls will face cervical cancer in the future. Read more at Bio.News.

Beltway Report: What's Ahead in Washington

All eyes are on the Republican National Convention in Milwaukee, where former President Donald Trump will take the stage tonight. Meanwhile, the House and Senate are out and President Biden is recovering from COVID-19 in Delaware.