Impulses for pharmaceutical supply from the patient perspective

Part of the EU pharmaceutical agenda also includes, among other things, increasing patient participation in the European drug approval process. Representatives of patient organizations will be involved in the Management Board of the European Medicines Agency (EMA), the Committee for Medicinal Products for Human Use (CHMP), and the Pharmacovigilance Risk Assessment Committee (PRAC).

Fortunately, Article 143, Paragraph 4a of Commission Proposal ¹ also included provisions for reimbursement of costs for participating patient representatives. However, there is still disagreement regarding the degree of participation. While Articles 148 and 149 still refer to "voting rights," the recent amendment merely states that patient votes should be "taken into account."

Naturally, the European umbrella organizations of patients, the European Patient Forum (EPF) and EURORDIS, have protested against such a reduction. ² Beyond the superficial question of "voting rights" at the EMA, however, it is fundamentally necessary to clarify what methodological concept should be pursued with regard to patient participation in the EMA's committees.

The participation could be a kind of “citizen participation” in order to assure the EMA that a certain residual risk is still acceptable during the approval process on the basis of evidence that may not be entirely reliable.

Patient representatives would then have to consider whether faster access to an innovation or the avoidance of residual risks should be the guiding principle in approval decisions. In Germany, patient participation is viewed less as a source of legitimacy for decisions than as a source of insight in scientific assessment. Clarifying patient preferences, differentiating subpopulations, and understanding the actual healthcare situation are important components of knowledge that are methodologically required in every drug assessment, including approval.

In the area of ​​drug benefit assessment, it is abundantly clear that the patient perspective is not solely intended to qualify an assessment result as "acceptable" or "unacceptable." Rather, it is about enriching the knowledge-gathering process in the HTA procedure with the patient's expertise. Therefore, it is only logical that training on the methodology of HTA processes is a standard offering for patient representatives at the Federal Joint Committee (G-BA) in Germany. A qualitative right to participate in consultations is by no means ineffective in such a setting.

In contrast, patient representatives at the French Haute Autorité de Santé (HAS) naturally have voting rights, but unfortunately, they lack any professional support. With regard to the EMA, it is therefore important to ask: Is there a methodological concept here as to why and for what purpose patient representatives should be involved?

The clarification of this question will also have an impact on the future design of the European benefit assessment procedure for medicinal products.

According to the EU Commission's plans, the EMA's patient participation database will simply be used to identify patient representatives for the EU HTA process. The professional expertise of the individuals consulted does not seem to be particularly important. Nor does it seem to reflect that participation in approval procedures is simply not synonymous with participation in benefit assessment procedures. In any case, training on the methodology of HTA procedures is not planned.

One suspects that patient participation is seen here more as a source of legitimacy than as a source of knowledge. However, there is the possibility of proposing qualified patient representatives from Germany to the Coordination Group's Secretariat for participation in the EU HTA process. Such a right is granted to the National Coordination Bodies under Article 83 of EU Regulation 536/2014. ³

Curiously, this is not the Federal Joint Committee (G-BA), but the Federal Institute for Drugs and Medical Devices (BfArM). Although the BfArM is not normally involved in the benefit assessment of medicinal products, it would be very helpful if it were possible to improve the opportunities for local patient representatives to participate at the European level in this way.

Dr. Martin Danner is a lawyer and the Federal Managing Director of the Federal Working Group for Self-Help of People with Disabilities and Chronic Illnesses and Their Families (BAG SELBSTHILFE). After studying in Heidelberg, he worked for several years as a lawyer specializing in health law before taking over the Health Policy and Self-Help Promotion Department of the BAG SELBSTHILFE. He is the spokesperson for the patient representation at the Federal Joint Committee (G-BA) and is active on the Scientific Advisory Board of the Medical Center for Quality in Medicine and the IQWiG Board of Trustees, among other positions.

¹ European Commission: European Parliament legislative resolution of 10 April 2024 on the proposal for a regulation of the European Parliament and of the Council laying down Union procedures for the authorization and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repeating Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006 (COM(2023)0193 – C9-0144/2023 – 2023/0131(COD)) https://www.europarl.europa.eu/doceo/document/TA-9-2024-0221_EN.html#title2 [accessed April 24, 2025]

² Joint Statement: The added value of a meaningful patient involvement at the EMA level - EPF EURORDIS. https://www.eurordis.org/epf-eurordis-joint-statement-patient-involvement/ [accessed April 24, 2025]

³ Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repeating Directive 2001/20/EC https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32014R0536 [accessed April 24, 2025]