European Parliament Holds Key Hearing on Pharmaceutical Legislation: A Focus on Innovation, Incentives, and Patient-Centred Governance
The European Parliament’s Intergroup on Cancer and Rare Diseases convened a high-level hearing today in Strasbourg to discuss critical developments in the revision of the EU pharmaceutical legislation (often referred to as the ‘Pharma Package’). The session brought together Members of the European Parliament (MEPs), patient advocates, researchers, and industry leaders to debate the future of incentives, unmet medical needs, and governance at the European Medicines Agency (EMA).
This hearing was held at a critical juncture for the legislative file, as it enters the trilogue phase. The European Parliament will play a crucial role in shaping the final outcome and ensuring the legislation delivers meaningful benefits for patients across Europe.
Strong Political Commitment from Parliament
Opening the session, MEP Sokol (HR, EPP) welcomed all the participants and stressed the importance of having this hearing when the Revision of the General Pharma Legislation arrived at trilogues. He expressed hope that the final position will arrive before the end of the year as conceptually, despite current divergencies, there are not irreconcilable differences between the Parliament and the Council’s positions.
MEP Voiculescu (RO, RE) continued the discourse but invited those present to think beyond the Pharma Package, highlighting the need to really work in the next four years to shape health policy in a constructive way. He underlined the importance of taking actions on the shortages of cancer treatments and new incentives targeting the most vulnerable populations, in particular the paediatric population.
MEP Metz (LU, Greens) agreed with MEP Voiculescu: the Pharma Package will not solve all of the existing problems, as there is need to work beyond this file. The Critical Medicines Act and its provisions on joint procurement will help strengthen Member States in negotiations, with beneficial repercussions on access and affordability. She stressed the importance of ensuring that rewards truly go to those bringing added value to patients.
MEP Bosse (DK, RE) echoed her colleagues while calling for a streamlined and purposeful legislative approach, warning against overcomplexity that could hinder effective outcomes. She underlined the need to secure adequate health funding in the MFF and to strengthen cross-border collaboration, both in clinical trials and access to care. MEP Bosse also urged the EU to lead in areas where collective action delivers clear added value for patients, particularly in the field of rare diseases.
MEP Moretti (IT, S&D) concluded by calling for reforms to create a fairer and more efficient EU pharmaceutical system, centred on patients’ needs. She urged stronger conditions for regulatory incentives, including equitable access, affordable pricing, and transparency, especially where public funding is involved.
Section 1: Regulatory Incentives and Access to Innovation
The first panel focused on adapting regulatory incentives to better address unmet medical needs and ensure timely patient access to innovation.
Dr. Virginie Hivert (EURORDIS) and Prof. Gilles Vassal (Gustave Roussy, FR & SIOPE) addressed the importance of refining incentives for orphan medicinal products and promoting a ‘first-in-child’ approach in paediatric medicine development.
"Given that 95% of rare diseases still lack an approved treatment, it is imperative to establish a regulatory framework that proportionally rewards the level of effort and innovation invested. EURORDIS supports a graduated system of incentives for orphan medicinal products, ensuring that developers addressing the most underserved and complex rare diseases receive commensurate recognition and reward. Aligning incentives with unmet needs is essential to fostering equitable progress across the rare disease landscape.” (Virginie Hivert)
"The Paediatric Medicine Regulation and the Orphan Regulation have had only limited impact on advancing the development of paediatric anticancer medicines, leaving significant unmet needs in childhood cancer treatment. As the EU revises its General Pharmaceutical Legislation, we call for the inclusion of a ‘first-in-child’ marketing authorisation incentive. Such a measure is expected to boost commercial interest in developing medicines specifically for paediatric cancers and other paediatric rare and ultra rare diseases.” (Gilles Vassal)
Tina Taube (EFPIA), joining online, presented perspectives on balancing incentives with equitable access, while boosting EU competitiveness.
"In light of Draghi’s warnings on declining competitiveness, maintaining a strong and predictable IP system is crucial—weakening protections risks undermining Europe’s innovation environment. Policy has shown it can drive progress, as with orphan drugs, but future frameworks must stay patient-centric, especially in defining unmet medical needs. While we support better access, equity-based pricing requires flexibility: there’s no one-size-fits-all, after all, it takes two to launch.”
Denis Lacombe CEO of EORTC, emphasised the incentives from drug-centredness to patient-centredness.
“Pharmaceutical legislation can better support patient outcomes by recognising the role of treatment optimisation as part of the development pathway. This approach may help ensure that therapies are used more effectively and appropriately across diverse patient populations.”
Lydie Meheus (Anticancer Fund) highlighted the unmet medical need and the prominence of repurposing.
“Repurposing authorised medicines is a proven, cost-effective strategy to meet unmet medical needs. The EU pharmaceutical legislation can play a transformative role by recognising the vital contribution of non-commercial research actors and supporting them with an appropriate regulatory framework.”
Yannis Natsis (European Social Insurers Platform) emphasised that we live in difficult times for the welfare state, under attack by conspicuous budget cuts everywhere.
“Competitiveness cannot come at the cost of solidarity. Public budgets carry the weight of extended exclusivities, with billions at stake and lives impacted. This isn’t just about medicines—it’s about the societal contract. We need fewer blank cheques to industry, more accountability, and a definition of unmet medical need that serves patients, not profits. Biotech must have a seat at the table—this system cannot be shaped by big pharma alone."
Section 2: Strengthening EMA Governance
The second panel explored the role of the EMA in maintaining excellence in patient and healthcare professional engagement.
Claudia Louati (European Patients' Forum) and Dr. Tomasz Grybek (CEO of Foundation of Borys the Hero, and member of the EMA's Paediatric Committee (PDCO)) underlined the need to safeguard meaningful patient involvement in regulatory processes, particularly in paediatric and rare disease contexts.
“We urge EU institutions to build on EMA’s longstanding experience and maintain meaningful patient involvement across the regulatory lifecycle, including patient participation in CHMP and PRAC with voting rights. As trilogues proceed in the coming weeks, it is essential that EU institutions uphold the commitment to meaningful patient involvement. This also includes consultation of patients in drawing up the Union's list of critical shortages and critical medicines; safeguarding the expertise of patients currently involved in scientific committees despite the EMA’s restructuring; allowing patient representatives to become co-rapporteurs in the evaluation of MAA and including patient representatives in the Coordination Group for Mutual Recognition and Decentralised Procedures for human medicines (CMDh)."(Claudia Louati)
“Giving voting rights to patients is not a revolution, but a necessary evolution. Granting patients a vote reflects a mature system — one where those most affected share responsibility, co-create solutions, and help shape decisions that truly serve all.” (Tomasz Grybek)
Conclusions and Next Steps
The hearing concluded with a collective call to ensure that the revised pharmaceutical legislation delivers on its promise: to serve patients' real needs through smarter incentives, inclusive governance, and a steadfast focus on public health outcomes.
The Intergroup will continue to monitor and contribute to the legislative process, engaging all relevant stakeholders as trilogue negotiations unfold. A report of the Group on the hearing will follow.