Our trend tracker explores the latest news and developments in life science and how they might affect you. In this fast-paced industry, changing market conditions, emerging technologies, and other factors can impact your business. See what current pharma trends might mean for you.
More progress needed in treating rare diseases
Much progress has been made in approving treatments for rare diseases, but more work is needed, asserts Kerry Jo Lee, MD, associate director of rare diseases in the US FDA Office of New Drugs (OND).
Lee said the number of orphan drugs approved by the agency is now on par with other novel approvals. From 2015 to 2022, about half of new drugs and biologics were approved for rare disease indications. “We are very, very cognizant of the fact that 30 million Americans have a rare disease,” said Lee, “and the vast majority of these patients do not have approved treatments.”
Lee addressed the FDA’s work to develop rare disease treatments and updated the agency’s rare disease endpoint advancement (RDEA) pilot program. The FDA will start accepting applications for RDEA on July 1. The agency will accept a single proposal for the pilot for Q4 FY 2023. For FY 2024-2027, the FDA will accept up to one RDEA proposal per quarter for a maximum of three proposals per year.
Further resources
- Learn how a team increased collaboration for a rare disease program with a virtual resource center
- Using technology to design more successful rare disease clinical trials
More news on AI and precision health
Dartmouth launched its Center for Precision Health and Artificial Intelligence (CPHAI) this week, advancing interdisciplinary research into how artificial intelligence (AI) and biomedical data improves precision medicine and health outcomes.
By using technologies like AI and machine learning, CPHAI researchers hope to drive innovations in digital biomarkers, clinical decision-making, predictive analytics, and precision health. Projects at the center will focus on treatment strategy optimization, biomedical data analysis to inform public health policy, and AI-based diagnostic tools.
Further resources
- Learn how AI impacts clinical trials
- Our expert discusses how AI can help pharma teams
Moving closer to customized care
Researchers have proposed the creation of a personalized disease burden assessment and planning tool based on ChatGPT and the Global Burden of Disease study. Using this tool, clinicians could develop customized care plans that consider patients’ preferences and lifestyles.
Such a tool leverages big data from the Global Burden of Disease study. The study provides comprehensive insights into disability and mortality across time, countries, sex, and age. The research contains significant data on injuries, risk factors, and diseases that lead to health loss.
While promising, using tools based on ChatGPT in the healthcare industry requires vigilance. Healthcare professionals must be aware of the potential pitfalls, like accuracy, privacy, and the ability to capture individual nuances.
Further resources
- The distinction between precision medicine vs personalized medicine
- How technology is supporting patient centricity in pharma
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