CRA is a proud sponsor of the the largest and most established orphan drug & rare disease meeting of its kind across the globe. From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.
Cecile Matthews moderates the session titled “From Clinical Evidence to Equitable Access: Navigating Joint Clinical Assessment in Rare Diseases.”
This session will explore the evolving landscape of Joint Clinical Assessments (JCAs) and their potential impact on equitable access to orphan drugs in Europe. Participants will delve into key lessons learned from ongoing JCAs, examining how evidence frameworks, PICO development, and expert involvement could be shaping clinical evaluations in the future. The discussion will highlight both the new opportunities and risks that JCAs present for orphan medicines. Thought a multi-stakeholder discussion, strategies for overcoming potential challenges will be proposed, including the early identification and simulation of PICOs and approaches for aligning evidence generation with JCA and national HTA requirements. Attendees will gain actionable insights into maximizing access and positive outcomes for patients with rare diseases in the context of rapidly evolving clinical assessment landscape.
Michele Pistollato leads the working group session titled “Every Newborn Counts: The Quest for Equitable Newborn Screening in Europe.”
Description: Over the past decade, newborn screening programs throughout Europe have seen significant advancements, both in the number of countries adopting them and the variety of disorders they screen for. Despite this progress, considerable disparities remain between countries, with the number of screened conditions varying widely. This session will explore the reasons behind these inequalities and discuss the advantages of achieving more equitable access to newborn screening across Europe for both patients and society.
For more information on this event, click here.
