Regulatory Affairs Professionals Society (RAPS) – EU patient groups say proposed definition of ‘unmet medical need’ is too restrictive (Catapult’s regulatory round-up – March 2024)

The overhaul of the EU pharmaceutical legislation, which includes the definition of unmet medical need, was adopted by the EC in April 2023. This definition carries significant implications as it shapes pharmaceutical companies’ eligibility for regulatory data protection, conditional marketing authorisations, and enhanced regulatory and scientific support. Most notably, it has implications for the post-authorisation phase and health technology assessment (HTA). According to the European Patients’ Forum (EPF) It is imperative that this definition is aligned with the true needs of patients. As the voice of patient organisations in Europe, the EPF calls for patient involvement in drawing up the definition.

EPF proposes a future framework to characterise “unmet medical need” as part of the revision of EU pharmaceutical legislation. According to the proposed framework it should:

• Apply to medicinal products intended to treat, but also medicinal products intended to prevent or diagnose a disease, or to restore, correct or modify physiological functions as per the definition of medicinal products in the EU.
• Acknowledge both immediate therapeutic needs perceived by patients that are unmet by current treatments and longer-term societal needs, including the impact on caregivers and potential public health concerns.
• Embrace inclusivity, recognising that many chronic diseases, while not life-threatening, substantially burden patients, caregivers, and healthcare systems. It should consider not only mortality and morbidity but also their broader impact on patients’ lives and health-related quality of life.
• Assess patients’ therapeutic needs based on various criteria, encompassing the impact of the condition on life expectancy, the appropriateness of current standards of care, and patient experience data (PED), including their quality of life and any mental health implications.

Please find further information here.

Furthermore, in order to steer pharmaceutical investment into medicines for unmet medical needs the EC proposed the provision of targeted regulatory protection incentives and regulatory support for medicines addressing unmet medical need (UMN) and high unmet medical need (HUMN). Please find further details here.

Published on the 2nd of April 2024 in Catapult’s regulatory round-up for March 2024.