A Dolon Institute campaign
Innovation in rare diseases has transformed the lives of many patients, yet significant unmet need remains. Among the 300 million people living with a rare disease in Europe today, a substantial number are without an approved treatment option, experience shortcomings with existing treatments, or struggle to access these.
To mark Rare Disease Day 2024, Dolon published a series exploring what it will take to see continued rare disease innovation in Europe.
This year, we turn our attention to EU Joint Clinical Assessment (JCA), which is now upon us and has the potential to transform the evaluation of medicines in Europe, including orphans. Building on our recent publication “Joint Clinical Assessment: The finger on the scale for orphan sustainability in Europe?”, we explore the potential impact of JCA across three key dimensions important to sustaining investment and launch of new rare disease treatments in Europe.