Next week about 400 million European citizens will be called to elect the 720 members of the European Parliament that will represent them in the next legislative term. Among their responsibilities, the new Members of the European Parliament (MEPs) will be negotiating the reform of the EU pharmaceutical legislation with the Council.
The file is the most extensive reform in the sector in more than 20 years and aims to promote innovation while ensuring timely and equitable access to medicines.
The European Society of Cardiology (ESC) takes this opportunity to congratulate the Parliament on reaching a first reading approval of the file within the current legislative term. While welcoming this step forward and the introduction of some positive additions, we urge the Council and the newly elected MEPs to address some remaining shortcomings.
Professor Piotr Szymański, Chair of ESC Regulatory Affairs Committee, said: "The primary objective of the reform is to guarantee timely and fair access to medications for patients across the EU and to foster the retention of research and innovation activities within Europe. A unified approach at the EU level should offer increased opportunities for incentivising innovation and coordinating efforts to develop and deliver medicinal products addressing unmet medical needs, especially in the areas where innovation remains significantly out of proportion to the disease burden".
The ESC commends the progress made in strengthening the consultation of healthcare professionals in the regulatory process, including for the drafting of scientific guidelines – such as on unmet medical needs - and for the adoption of measures to prevent and manage shortages.
Health professionals' expertise and direct contact with patients are essential to enhance the relevance and effectiveness of regulatory measures. In particular, consultation of health professionals representing medical societies is crucial to gather collective views and foster alignment with clinical practice guidelines. The guidelines play an important role in shaping clinical practice of European physicians, by providing evidence-based recommendations that improve quality of healthcare delivery.
Other improvements introduced by the Parliament that the ESC calls the Council to uphold, concern:
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- Repurposing of authorised medicines, allowing not-for-profit entities to present evidence to regulators for a new therapeutic indication even if not addressing an unmet medical need. Repurposing can effectively broaden access and provide significant benefits to patients and should have a wide scope.
- Prevention and management of shortages, including the requirement for National Competent Authorities to recommend to health professionals alternative medicines to use in the case of shortages.
- Transparency on financial support received for research and development, which was extended to indirect public funding and to licenses or acquisitions from other entities. Enhanced transparency on R&D costs and exchange of expertise on pricing, reimbursement and procurement policies can contribute to the sustainability of health systems.
Nevertheless, the ESC is concerned that while the reform provides targeted measures and incentives for a number of major public health needs, it fails to address the emerging misalignment between burden of disease and innovation in some therapeutic areas, including cardiovascular disease (CVD). Although CVD remains the leading cause of death and morbidity in Europe, research and development is out of proportion to its burden and woefully lagging with respect to other therapeutic areas. Out of 80 new active substances authorized between 2022 and 2023, only one was in CVD[1] and only 4% of the trials started between 2017 and 2022 were for CVD[2]. This misalignment also constitutes an unmet medical need, which should be recognised and addressed to provide European patients with the treatments they need.
Building on the Commission's proposal and on the negotiations held in the Parliament, the ESC recommends the following provisions which would help achieve this objective:
- Identify and address innovation gaps: Regulators and policymakers should proactively identify therapeutic areas where innovation is stagnating despite a remaining high disease burden, representing unmet medical needs. Such information should be used to investigate causes and possible solutions, consulting relevant health professional associations.
An agile and responsive regulatory system should encourage R&D to stay in Europe. In parallel, reinforcing public research is also key to filling existing gaps.
- HERA's & ECDC's mandate: Considering the new role and responsibilities proposed by the Parliament for the Health Emergency Preparedness and Response Authority (HERA) under the European Centre for Disease Prevention and Control (ECDC), such innovation gaps should also be considered, especially when it comes to the definition of a long-term European R&D portfolio and collaboration with third-party research centers, not-for profit entities and academia. As requested by MEPs in the framework of negotiations on Regulation (EU) 2022/2370[3], the ECDC's mandate should be extended to encompass major non-communicable diseases, gathering crucial epidemiological data for research and policymaking.
- Unmet Medical Needs: Unmet needs also exist in chronic, progressive, and highly prevalent conditions, which pose a significant burden to individual and public health.
The definition provided by the directive should take this into consideration and also better reflect patients' and societal needs.
- PRIME: Out of 384 PRIME requests received by EMA between 1 January 2016 and 30 June 2021, only 20 were for CVD and just 2 were granted eligibility to the scheme[4]. Extending the scope of PRIME to encompass chronic conditions associated with high mortality and morbidity would significantly benefit European patients.
