This novel pathway also places significant emphasis on patient engagement and communication, ensuring that people affected by rare diseases are involved throughout development and that informed consent is an ongoing, not one‑off, process. The guidance also explains expectations around adaptive clinical trial approaches, real‑world data, prior knowledge, and predictive modelling – tools that may be essential for generating meaningful evidence in very small or highly heterogeneous populations.
We are seeking feedback from manufacturers, developers, clinicians, researchers, patient organisations, carers, families and other stakeholders on our regulatory approach to rare disease therapies.
To ensure that patients, carers and the wider public receive clear and balanced information about these treatments, we particularly welcome feedback on the sections addressing patient communication, ongoing consent, and support for decision‑making.