ISSUE: JCA will roll out in 2025, starting with all new oncology products. This will expand to include products with orphan designation in 2028. Assessment of rare diseases will face additional challenges compared with non-orphan products in terms of clinical trial design, low patient numbers, heterogenous populations, and differing standards of care across the 27 European Union member states. It is important that companies start preparing now to ensure studies are designed appropriately and to understand the PICOs (population, intervention, comparator, and outcomes) that will apply. It may be appropriate to use pragmatic approaches to reduce the research questions to be appraised and innovative methodology to consider the available evidence, both from clinical trials and real-world data. Understanding the potential options to efficiently consider all relevant populations and to ensure that a voice is given to patients across these populations within rare diseases will be vital. All stakeholders will need to collaborate to balance challenges due to the constraints of the time and data available with the needs of patients.
OVERVIEW: This panel discussion will convene experts in rare diseases, patient advocacy, and statistics to share their insights and experiences regarding JCA requirements. Caroline Ling will moderate the session, introducing the topic and the panellists. Abigail Stevenson will explore the considerations and potential challenges associated with evidence generation across all 27 European Union member states for rare diseases. Emma Hawe will consider efficient approaches to evidence generation from the perspective of literature reviews and indirect comparisons where multiple PICOs exist. The patient advocate will articulate the significance of giving a voice to specific populations in rare diseases, emphasizing the urgency of access to new treatments and importance of patient-relevant outcomes. The panel presentations will span 45 minutes, followed by a 15-minute question-and-answer session, allowing the audience to engage with the panellists.
