With interinstitutional negotiations on the EU Pharmaceutical Legislation now underway, the European Society of Cardiology (ESC) acknowledges the efforts of the Member States and views the Council of the EU's position as a long-awaited step forward. At the same time, we remain seriously concerned about several critical shortcomings that must be addressed to ensure a truly impactful reform.
A core concern for the ESC is the limited and optional nature of stakeholder consultation, particularly regarding healthcare professional organisations. This marks a step back with respect to the European Parliament's position, which had rightly prioritised stronger engagement throughout the regulatory lifecycle.
"Healthcare professionals bring critical clinical insights to the regulatory process, helping ensure that policies reflect real-world care and work effectively in practice. Clinical expertise must not be an afterthought, but the foundation of effective regulation" said Professor Piotr Szymanski, Chair of the ESC Regulatory Affairs Committee.
The ESC also calls for the preservation of voting rights for healthcare professionals and patient representatives in the Committee for Human Medicinal Products – rights which are removed under Article 148a of the Council's position. "Healthcare professionals and patients – the ultimate beneficiaries of the reform - must have a say", continued Professor Szymanski.
Other key recommendations
1. Unmet Medical Needs (Article 83, Directive)
- The ESC calls for a comprehensive and inclusive definition of Unmet Medical Needs (UMN) that reflects the real-world burden of disease and patient experience, including factors such as quality of life and treatment-related adverse effects.
- As the EMA develops guidelines to clarify UMN criteria, healthcare professionals must be actively involved. Their clinical expertise is essential to ensure the criteria are grounded in everyday practice and aligned with patient needs. The Council's position does not ensure stakeholder consultation, in contrast to the Parliament's more inclusive approach. This should be rectified in the final text.
2. PRIME Scheme (Article 60, Regulation)
The ESC calls for clearer criteria for "exceptional therapeutic advancement," developed with input from healthcare professionals and patients. The current focus on rarity or novelty is too narrow and risks excluding high-impact treatments. Notably, only 2 of 384 PRIME applications granted between 2016 and 2021 related to cardiovascular disease, despite being the EU's leading cause of death1 . The scheme's scope should be expanded to better support innovation in chronic, high-burden conditions, improving patient outcomes and health system resilience.
3. Medicine Shortages (Chapter X, Regulation)
The ESC welcomes the expanded scope of the Union List of Critical Medicines, including products which might become critical in case of supply issues, but calls for:
- Systematic consultation with healthcare professionals in developing the list, taking into consideration clinical practice guidelines.
- Preservation of the Parliament's provisions on stakeholder input in shortage prevention plans, information sharing, and guidance development.
- Stronger provisions for joint procurement, expanding beyond the Joint Procurement Agreement and the recently proposed Critical Medicines Act.
4. Repurposing of Medicines (Article 48, Regulation)
The ESC supports extending repurposing beyond unmet needs and urges clarification regarding product information updates. Since repurposing can expand access to treatments and deliver substantial benefits to patients, its scope should be as inclusive as possible.
5. Transparency (Article 57, Directive)
The ESC supports enhanced disclosure by manufacturers of received public R&D funding, including indirect support and information on licensed products, as outlined in the Parliament's text.
6. Added Therapeutic Value (Article 153, Regulation)
ESC advocates for mandatory consultation with healthcare professionals and patient organisations when developing EMA guidelines on therapeutic value. We also call for the inclusion of evidence generated through publicly funded registries and other real-world data analyses conducted by healthcare professionals as an integral part of the process, leveraging the European Health Data Space as a key enabler.
7. Redispensing (Article 207a, Regulation)
While supportive in principle, the ESC stresses the need for robust safety safeguards, traceability, exclusion of high-risk products, and informed patient consent.
A Call to Action
The ESC urges EU policymakers to ensure that the final legislative text results in a future-proof pharmaceutical framework, one that is grounded in clinical care, prioritises patient needs, and co-created with those on the front lines of healthcare.
A detailed overview of ESC's recommendations is available in the full statement here .
