Executive Director, Rare Disease, Center of Excellence
Jonathan Schwartz, MD
Chief Gene Therapy Officer
Jay A. Barth, MD
Chief Medical Officer
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Pharma’s interest in rare diseases has been re-invigorated in recent years, leading to a flurry of major deals and approvals for new drugs. But to drive success in rare diseases, leaders have to think differently while navigating a number of unique challenges around clinical trial design, regulations and more.
Join our journalists at PharmaVoice, along with key industry leaders across biotech and pharma, for an immersive and thought-provoking event. During this 60 minute virtual event we’ll share what it takes to make it in the rare disease space including sound business strategies, working with patients and why the time is right to be a player in this burgeoning field.
Register now to secure your free spot at the Biotech Roundtable.
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Navigating clinical trial design and regulations in rare diseases
Tackling the complexities and costs of clinical trial design in the rare disease space remains a chief goal for the industry. And while there are lingering hurdles around patient enrollment and the need to improve DEI, opportunities are also emerging through advanced technologies.
Meanwhile, rare disease approvals have gained significant regulatory momentum, and in 2022, half of the new meds OK’d by the FDA were for orphan drugs. But significant work in the rare disease space still needs to be done to bring more medications to market.
In this session, our panel will discuss:
Patient recruitment challenges and opportunities
Incorporating DEI into trial design
The role of DCTs and other advanced clinical technologies
Designing clinical trials to meet regulatory goals
How recent FDA changes are impacting drug development efforts