Last year the European Medicines Agency (EMA) authorised the first medicinal product for the treatment of certain types of Alzheimer (Leqembi®) based on a favourable benefit–risk assessment. However, recently the National Health Care Institute in the Netherlands (ZorgInstituut Nederland (ZIN)) concluded it does not meet reimbursement criteria for the basic health insurance package. The recent discussion around Leqembi® highlights a recurring challenge in how new medicines translate into real‑world access.
This is not an isolated case, and it’s not just about one drug. Across therapeutic areas, we increasingly see regulatory and health technology assessment (HTA) bodies reach different conclusions, reflecting their distinct roles:
- Regulators like EMA assess whether the benefits outweigh the risks in a controlled clinical setting focusing on safety and efficacy for defined populations.
- HTA bodies (e.g. ZIN) evaluate real‑world effectiveness, added therapeutic value and value for money within a healthcare system considering impact on daily functioning, survival, quality of life and sustainability.
There are also other examples illustrating this gap. For instance, the PARP inhibitors for cancerwere recently (partially) removed from the basic health insurance package after re‑evaluation that showed insufficient survival benefit for some patient subgroups. This decision was driven by updated evidence and concerns the cost‑effectiveness of widespread use.
Dutch initiatives such as the Drug Access Protocol (DAP) aim to offer structured access while collecting real‑world evidence. These programmes exist precisely because EMA approval alone doesn’t guarantee reimbursement, particularly where evidence remains uncertain or unmet value criteria are difficult to satisfy. Across Europe similar patterns have appeared.
Why this all matters:
- Clarifying expectations: patients, clinicians and innovators need to understand that approval is only one step on the path to access.
- Guiding evidence planning: early and aligned evidence strategy with both regulators and HTA agencies is increasingly important to ensure therapies demonstrate meaningful real‑world impact, not just statistical significance.
- Supporting policy evolution: as treatments become more complex and expensive, transparent frameworks that bridge regulatory science and value assessment will be essential for equitable and sustainable patient access.
Leqembi® is not just an example but the underlying issue cuts much wider and encourages cross‑stakeholder dialogue on how we define, measure and reward value in healthcare.
For organisations navigating these complexities, more information on strategic regulatory support can be found at our website www.starodub.nl or contact us at [email protected].
