As the EU’s general pharmaceutical legislation enters a decisive phase, EHC have joined EURORDIS–Rare Diseases Europe, the European Patients’ Forum (EPF) and more than 70 additional patient organisations in calling on EU policymakers to safeguard strong and meaningful patient involvement at the European Medicines Agency (EMA) – including the retention of patient voting rights in the Committee for Medicinal Products for Human Use (CHMP).

In a joint statement first published on 10 March 2025, EURORDIS and EPF warned that Council negotiations risked removing patient representatives’ voting rights in the CHMP and reducing the number of voting patient members in the Pharmacovigilance and Risk Assessment Committee (PRAC) – the two EMA committees responsible for authorising and monitoring medicines in Europe.

The European Commission’s original proposal marked a milestone in formalising patient involvement in EU medicines regulation, introducing provisions for four patient representatives with voting rights on the CHMP and two on the PRAC. However, the Council agreement adopted on 4 June significantly weakens these provisions. It falls short of both the Commission’s ambition and the European Parliament’s position adopted in April 2024 in terms of patient involvement.

For over 25 years, patient representatives have contributed their lived experience to EU regulatory processes. Their insights strengthen trust, ensure that medicines respond to real-world needs, and complement clinical and scientific expertise. Reducing patient involvement or removing voting rights would not only undo decades of progress – it would send a damaging signal that patient perspectives are no longer valued in decisions that directly affect their lives.

Following the Coreper I agreement on 4 June, reached under the Polish Presidency of the Council of the EU, trilogue negotiations between the Council, the Parliament and the Commission are set to begin on 17 June. This final phase will shape the future of EU medicines regulation – and with it, the future of patient representation within the EMA.

Hundreds of thousands of Europeans depend on plasma medicines. These medicines can only be made from human plasma. As recently as 2011, enough plasma was collected in the EU to meet the usage of EU patients. Since then, however, despite increases in both public and commercial plasma collections, the increasing demand for plasma has exceeded supply. EU countries do not currently collect enough plasma to meet the needs of EU patients, who have experienced shortages of plasma medicines (plasma-derived medicinal products, or PDMPs).

Europe’s insufficient collection and its subsequent reliance on plasma imports, primarily from the US, endangers patient access and thus undermines the health security of EU Member States and Europe as a whole. Experiences during the COVID-19 pandemic and times of increased geopolitical tensions show the dangerous consequences of relying heavily on imports of plasma to manufacture these essential plasma medicines. Europe must address this strategic vulnerability and no longer leave its citizens exposed to this threat.

Countries with plasmapheresis programmes (public and/or commercial) contribute proportionally more plasma to the manufacturing of PDMPs than countries without such programmes.

The new Regulation on Substances of Human Origin (SoHO) urges Member States to increase their collection capacity and donor base for plasma by investing in non-profit and public plasmapheresis programmes. We strongly believe that by increasing their funding towards public collection programmes, EU Member States will greatly improve the prospects of delivering a secure and resilient supply of plasma medicines for their patients in the coming years.

We, patients, donors, blood establishments, physicians and other healthcare professionals, hospitals, healthcare services and public sector industry representatives therefore endorse this manifesto and urge national and EU authorities to increase their political support and funding to public plasma collection programmes with a view to increase the public collection of plasma for fractionation in Europe by 40%, and at a minimum by 25%, by 2030. This would mean increasing the plasma contribution from Europe from 9 million litres in 2022 to at least 11 million litres in the next 5 years.

Women with bleeding disorders face unique challenges that significantly impact their health and well-being, however, their needs are often overlooked due to a common misconception that bleeding disorders primarily affect men. Conditions such as haemophilia, von Willebrand Disease (VWD), and platelet function disorders can cause severe health complications, including heavy menstrual bleeding, prolonged bleeding after injuries, and difficulties with childbirth. The hormonal fluctuations that occur during menstruation, pregnancy, and menopause can further complicate diagnosis and treatment. Understanding the complexities of bleeding disorders in women is crucial for early diagnosis, effective treatment, and improved quality of life.

Given the barriers that women with bleeding disorders face, the European Union (EU) needs to take proactive steps in addressing these gaps. We urge the EU to prioritise women’s health by focusing on improving access to care, fostering research, and promoting early diagnosis and specialised care.

The EHC is part of the Platform of Plasma Protein Users (PLUS), a coalition of organisations of patients with rare plasma-related disorders whose lives rely on plasma proteins products. Following the annual Stakeholder Consensus Conference on January 23-24 in Estoril (Portugal), the EHC endorsed the PLUS joint statement on the safety of PDMPs.

The EHC acknowledges the recent decisions by several pharmaceutical companies to discontinue or refocus the development or commercialisation of haemophilia therapies since October 2024. The EHC recognises that these decisions come at a time when significant unmet needs persist in the bleeding disorders community. While progress has been made, many people with haemophilia (PWH) or other bleeding disorders still experience treatment burdens, breakthrough bleeding, and reduced quality of life. Access to standard prophylaxis remains a challenge in many European countries, further underscoring the need for continued research, innovation, and equitable treatment availability.

The EHC urges pharmaceutical companies, health authorities, and stakeholders to remain committed to advancing treatment options for haemophilia and other bleeding disorders. Investment in research and development must continue to ensure that the needs of the bleeding disorders community are met and that all patients have access to safe, effective and innovative therapies.

The EHC welcomed the opportunity to contribute to the European Commission’s public consultation on the draft implementing regulation on Joint Scientific Consultations (JSC) for Medicinal Products, for which we issued nine recommendations.

This EU initiative sets out implementing rules detailing how developers can receive early guidance on the evidence and data needed to carry out a joint clinical assessment of their medicinal products.

Read more about this EU Commission’s initiative here.

JCA should not hamper access to transformative ATMPs!

The EHC joins a group of patients, clinicians, academic medical centres, and therapeutic developers and manufacturers in signing a call to action for the successful roll-out of the Joint Clinical Assessment (JCA).

With the adoption of the Implementing Act on the JCA for medicinal products and the HTA Coordination Group’s Methodological and Practical Guidelines on direct and indirect comparison, the implementation of the JCA under the EU HTA Regulation is entering a critical phase. The EHC and other signatories are concerned that European patients with few or no treatment alternatives will experience further delays and limitations in access to potentially life-changing medicines. This would defeat the purpose of the EU HTA and JCA which aspire to increase and accelerate patient access.

Therefore, the joint statement urges the members of the HTA Coordination Group and its relevant subgroups, and JCA assessors to recognise all types of available evidence including single-arm trials and RWE, and to use the JCA report to describe, rather than judge, any resulting uncertainty as to the treatments’ benefits, as called for by the HTA Regulation. A significant portion of outstanding uncertainty can be addressed at the national level during the appraisal phase and through the collection of RWE.

In so doing, the Coordination Group can lead the development of a fit-for-purpose JCA system that efficiently addresses the needs of healthcare systems, without obstructing patient access to transformative therapies.

The EHC joins other leading patient organisations across Europe in releasing a set of ten key recommendations aimed at improving patient involvement in Joint Clinical Assessments (JCAs) under the EU Health Technology Assessment (HTA) Regulation. This collaborative effort highlights the crucial role of patient experiences and insights in shaping effective healthcare assessments and ensuring that the voices of those directly affected by medical innovations are heard and valued. 

Developed with input from a wide range of patient groups and based on extensive consultation and practical experience, the recommendations are designed to foster a more inclusive, transparent, and patient-centered approach to JCAs. The following ten points summarise their input and we highly encourage they are considered to ensure meaningful and practical patient involvement

They’re also a call to action for the HTA Coordination Group to integrate these practices into the guidelines they develop to enhance the quality and relevance of JCAs. By doing so, they can ensure that assessments reflect the real-world experiences of patients, ultimately leading to more patient-centered healthcare solutions.

• Establish a predictable framework for patient involvement in JCAs
• Include input from patients, carers and patient organisations
• Include patient experience data in JCAs
• Streamline patient involvement throughout the process
• Provide plain language summaries of technologies
• Broaden the pool of patients and specify selection criteria
• Provide support to patients
• Make JCA and summary reports available in all EU languages
• Provide feedback to patients
• Adopt a constructive approach to confidentiality and conflict of interest

For the last thirty years, haemophilia A and B patients have been treated using FVIII and FIX replacement concentrates. These therapies are well known to both clinicians and patients. We are now entering a new era in the treatment of haemophilia and rare bleeding disorders. Novel, non-replacement therapies have been approved and are becoming available, which may have a significant impact on the clinical care and quality of life of patients.

The European Medicines Agency (EMA) launched in October 2023 a public consultation on their draft guideline on the clinical requirements for non-replacement therapy in haemophilia A and B. The end of the consultation and deadline for comments was on 30 April 2024.

The European Association for Haemophilia and Allied Disorders (EAHAD), the European Hematology Association (EHA), and the EHC support EMA’s efforts in drafting the guideline which aims to provide both applicants and regulators with much-needed harmonised marketing authorisation requirements for applications of non-replacement therapies for haemophilia A and B.