Daily Briefing

Around the nation: FDA releases new approval process for personalized treatments


FDA has launched a new "plausible mechanism pathway" to help expedite approvals of personalized treatments for rare diseases, in today's bite-sized hospital and health industry news from the District of Columbia, Maryland, and Washington. 

  • District of Columbia: Ann Mond Johnson, CEO of the American Telemedicine Association (ATA), will retire from her position, effective Dec. 10. Mond Johnson first joined ATA in 2018, and she led the organization through the COVID-19 pandemic, when telehealth use increased dramatically. "It has been a remarkable journey and privilege to guide the work of the ATA through this transformative period. I am proud of our incredible accomplishments, especially our work creating actionable tools to help organizations improve access to needed care, and the talented, dedicated team behind our success," Mond Johnson said. Kyle Zebley, ATA's SVP of public policy and executive director of the organization's policy arm ATA Action, will succeed Mond Johnson as CEO. Before Zebley joined ATA in 2020, he was chief of staff in HHS' office of global affairs and represented the United States in global health diplomacy, including pandemic preparedness, pharmaceutical policy, and digital health standards. "As we look to the future, I'm energized by the opportunity to build on that strong foundation and continue advancing the ATA and ATA Action's mission, driving innovation, strengthening collaboration across the healthcare ecosystem, and ensuring that every American has access to safe, high-quality, technology-enabled care," Zebley said. (Miliard, Healthcare IT News, 11/11; DeSilva, Modern Healthcare, 11/10)
  • Maryland: FDA has launched a new "plausible mechanism pathway" to help expedite approvals of personalized treatments for rare diseases. The new pathway is designed for treatments for ultra-rare conditions that cannot be tested in randomized clinical trials. Treatments that are approved through the pathway are required to act on a known biological mechanism and show clinical improvements in patients. An example is a personalized CRISPR therapy, which was designed for a baby named KJ who had a rare genetic disorder called CPS1 deficiency. Once drugmakers receive approval, they must collect real-world safety and efficacy data for their treatments. Although the pathway initially targets rare conditions, FDA said it could later be extended to diseases with significant unmet needs, though its efficacy in more complex cases is still unclear. (Jeffries, Becker's Hospital Review, 11/13)
  • Washington: Amazon Pharmacy is partnering with healthcare technology platform Experity to allow its patients to order and receive same-day deliveries of prescriptions. This partnership is the first time Amazon is integrating its prescription services into a digital health platform focused on urgent care. Through the partnership, patients at urgent care centers will soon be able to access automatic manufacturer discounts and order medications to be delivered through Amazon. Prime members will be eligible for free two-day delivery, and almost half of Amazon's U.S. customers could access same-day delivery by the end of 2025. According to a study in the Journal of Urgent Care Medicine, patients who received their prescriptions onsite at the place of healthcare service had a prescription abandonment rate of 2%, compared to 23% at community pharmacies. (Jeffries, Becker's Hospital Review, 11/13)

Truly personalized care is possible. Here's how to make it a reality.

Breakthroughs in treatment and diagnostics offer the promise of personalized, tailored care for every patient. However, healthcare leaders face four key challenges in delivering this level of "bespoke care" at scale. Learn how to navigate these obstacles to improve healthcare access for all patients.


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