Pave the Way to Joint Clinical Assessment for Your Orphan Drug in Europe
Anne Heyes, MBA
VP, Head of Value Strategy
RTI Health Solutions
Navigating the New Era of Orphan Product Registration in Europe
In January 2028, a new era begins for orphan product manufacturers in Europe with the introduction of Joint Clinical Assessments (JCAs), and I am hearing a lot of concern from clients who are hoping to register their orphan products in Europe in 2028 and beyond.
This pivotal change mandates that manufacturers demonstrate the comparative effectiveness and safety of their new products against all relevant comparators. While this process is already in place for oncology products and Advanced Therapy Medicinal Products, the same rigorous standards will soon apply to rare diseases.
The unique hurdles of clinical development in the rare disease space—such as low patient numbers, limited data, and analytical uncertainties—necessitate a strategic and innovative approach to meet JCA requirements and other challenges. Showing patient-relevant and payer-relevant benefits needs meticulous planning and robust methodologies. Single-arm studies, ethical considerations, and varying management strategies post launch across EU member states further complicate the landscape. In addition, developers need to consider implementation science and astute ways of addressing budget impact challenges.
RTI Health Solutions Provides Expert Support for Joint Clinical Assessments
At RTI Health Solutions, we understand these complexities and are here to help. Our team of experts offers integrated solutions tailored to meet the stringent requirements of Joint Clinical Assessment for rare diseases. From strategic planning to tactical delivery, we provide comprehensive support to ensure your product's success in the European market.
Partner with RTI Health Solutions and navigate the Joint Clinical Assessment process with confidence. Together, we can turn challenges into opportunities and pave the way for groundbreaking treatments in the rare disease space.
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