Evidence generation with external control arms in rare diseases: a multistakeholder perspective

Evidence generated for rare diseases from studies involving external control arms (ECAs) is increasingly being used to support decision-making by regulatory and health technology assessment (HTA) agencies. Yet the standards and guidance for their use varies, making the generation of harmonized fit-for-purpose evidence challenging. At the recent ISPOR Europe 2023 meeting, a multistakeholder panel convened to discuss ‘Use of External Control Arms in Rare Disease: Are We Moving Towards an International Gold Standard and How Can We Facilitate Progress?’ Here, the panelists, Carla Vossen (Senior Director Epidemiology, RWE, Syneos Health), Pierre Net (Associate Project Manager, HEOR, Syneos Health), Amr Makady...