Following the political agreement reached at the end of last year, both the Council and the European Parliament’s Committee on Public Health (SANT) have endorsed the compromise texts for a revised EU medicines framework – the first major overhaul in more than 20 years - which is now entering the final steps toward formal adoption. The reform package was agreed after years of political discussion as well as continuous engagement with stakeholders, including the ESC.
The ESC welcomes the more inclusive approach to unmet medical need (UMN) and stakeholder engagement in the compromise texts.
"The agreed definition of UMN appears more flexible and reflects a broader understanding of clinical benefit. It is expected to go beyond mortality and morbidity, allowing consideration of additional patient-centred dimensions, such as quality of life and treatment-related side effects", Prof. Piotr Szymanski, Chair of the ESC Regulatory Affairs Committee said.
Now attention turns to the European Medicines Agency (EMA), which will devise Guidelines on UMN. Crucially, this work will be developed with input from stakeholders, including patients and healthcare professional representatives.
The final text also features a more inclusive approach to stakeholder engagement. This is exemplified by the restoration of voting rights for healthcare professionals and patient representatives within the EMA Committee for Medicinal Products for Human Use (CHMP), which had been curbed in the Council position. For the ESC, this decision is a welcome change and reaffirms the central role of clinical expertise in regulatory decision-making. "That means the future EMA guidance on UMN is more likely to support comparative, patient-relevant and access-relevant endpoints, rather than morbidity and mortality endpoints only", Prof. Szymanski added.
"We welcome the new text which ensures that we are not only observers, but can speak and vote", he said. "On some topics voting rights will be key to shaping decisions on which medications reach the market. The voice of patients and healthcare professionals has been preserved."
While the conclusion of negotiations on the EU pharmaceutical reform is a major milestone, there is still much work to do. The ESC’s advocacy efforts are now focusing on the key issue of medicine shortages.
"The revised pharmaceutical regulation strengthens engagement with healthcare professionals and patients when Member States identify critical medicines. However, the criteria for defining critical medicines should reflect the priorities of clinicians and patients", Prof. Kostrubiec said. A medicine may remain critical for patients even if an alternative product exists – especially if those alternatives are not clinically equivalent for a specific patient subgroup.
"The current list of critical medicines primarily includes medicines that are not widely used, but it does not include products which many of our patients rely on, such as antiarrhythmic or heart failure drugs", Prof. Kostrubiec said. "We need to better understand how this list was devised and ensure that the needs of cardiovascular patient populations are taken into account."
The list provides the basis for targeted EU-level actions to improve security of supply, including measures reflected in the Critical Medicines Act currently under negotiation. When it comes to this file, the ESC is calling for stronger European-level coordination, including joint procurement, common approaches to stockpiling, and enhanced information-sharing between countries.