The CDDF is pleased to share the key takeaways from our live webinar – “Compassionate Use and EAP for Cancer Drugs – From Fragmentation to a Common Framework in Europe under the New Legislation” held on 26 October 2026. This webinar includes presentations by François Houÿez (Eurordis) and Ramona Reichenbach (Novartis) followed by a Q&A discussion moderated by Ruth Plummer (CDDF) and Nina Heiss (Merck).
📺View the full recording and presentation slides
📑Read the key takeaways:
- Compassionate use (CU) enables physicians to request investigational, locally unauthorised or unavailable medicines for seriously ill patients with unmet medical needs who have exhausted treatment options and cannot participate in a clinical trial—a potential lifeline. CU can bridge the gap between confirmatory trials and marketing authorization, while Early Access Programmes (EAPs) can bridge access inequalities caused by post-authorisation delays, including pricing and reimbursement. Varied use of terminology can be a challenge.
- Challenges for Member States, industry, and clinicians include unclear exit rules, affordability concerns, immature data, including a lack of safety data among patients that differ from the ones enrolled in clinical trials (e.g. kidney or liver impairment, comorbidities), poor data quality and standardization, and complex, burdensome paperwork for executing on CU requests.
- From a patient advocate perspective, recommendations for industry include early planning, communicating more transparently about supply, accepting discussions on CU cannot be confidential, collecting safety and special-population data, establishing responsible exit arrangements, and not restricting the compassionate use programme to clinical trial sites, as well as not letting the cost of EAP be a limitation (relevant for small companies who can charge for the medicine where it is legally and ethically permissible).
- From an industry perspective, under the current legislation, the main challenge is navigating heterogeneous national legislation, compounded by limited transparency, low awareness, and insufficient stakeholder knowledge in this area. There are missed opportunities for systematic real-world data collection to support regulatory or payor decision making. There is a need to provide a framework ensuring quality and standardization of data collected.
- The discussion identified potential opportunities arising from Article 27 of the new European pharmaceutical legislation, which is expected to enter into force in 2028.
- Create greater visibility through the new requirement for the EMA to publicly disclose CU authorised by Member States.
- A more unified legal basis to streamline terminology, scope and operational requirements (e.g. product labeling and release, patient transition / exit plans).
- Reference to greater use of real-world data could strengthen evidence generation and provide an opportunity to define a fit-for-purpose RWD collection framework for CU in Europe.
- Successful implementation will depend on establishing structured and sustained dialogue among all relevant stakeholders – including patients, regulators, healthcare professionals, health technology assessment bodies, and industry- to identify common elements and best practices that can achieve broad acceptance and support effective implementation at the national level.