28. Tackling uncertainties for rare diseases - The use of RWE to improve access for patients

New innovations require fresh tools and policy approaches to ensure we can adequately and equitably meet the needs of those with rare diseases. This is particularly true given their small patient populations and lack of data which can lead to late or no access. The potential of real world evidence must be exploited to address uncertainties that occur during the evidence generation pathways. The TRUST-4RD multi-stakeholder initiative has created a practical tool to address evidentiary requirements and the use of RWE to enable earlier patient access. This session will: Examine how RWE can be used to address current levels of insufficient data so that regulators, HTA bodies, payers and society can better assess the value of treatments for complex or rare conditions. Discuss what is missing and look into how evidence generation can help meet the unmet needs of patients. Discuss how health data can be used to ensure a patient centric approach to the management and treatment of people with complex or rare conditions. Give context to the debate on access and sustainability of healthcare systems at European and national level. Share best practice from the European Reference Networks and explore the use of data and knowledge for treating patients in EU Member States. Views and insights from academia, the ERNs, national/European policy-makers, patient representatives and industry will be shared during the workshop. The panel will be followed by Q&A with the audience.