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. 2024 Jun 24:11:1408553.
doi: 10.3389/fmed.2024.1408553. eCollection 2024.

Balancing ethical norms and duties for the introduction of new medicines through conditional marketing authorization: a research agenda

Mariia V Maksimova  1 , Ghislaine J M W van Thiel  1 , Yke Tromp  1 , Rosan Lechner  2 , Johannes J M van Delden  1 , Lourens T Bloem  3
Affiliations

Balancing ethical norms and duties for the introduction of new medicines through conditional marketing authorization: a research agenda

Mariia V Maksimova et al. Front Med (Lausanne). .

Abstract

The European Medicines Agency's conditional marketing authorization (CMA) aims to expedite patient access to medicines for unmet medical needs by shifting a part of the drug development process post-authorization. We highlight ethical issues surrounding CMA, comprising (i) the complexity of defining unmet medical need; (ii) poor understanding of CMA and its impact on informed consent; (iii) hope versus unrealistic optimism; (iv) implications of prolonged post-authorization studies and potential patient harm; (v) rights and duties of patients surrounding participation in post-authorization studies; (vi) access to previously authorized CMA medicines; and (vii) the "benefit slippage" phenomenon, defined as the gradual shift of strict criteria to less strict criteria. We propose a comprehensive research agenda to address these ethical issues, and stress the need for multi-stakeholder engagement to ensure patient-centered use of CMA.

Keywords: European Medicines Agency; clinical ethics; conditional marketing authorization; expedited regulatory pathways; informed consent; research ethics; uncertainty; unmet medical need.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

FIGURE 1
FIGURE 1
Number of medicines granted conditional marketing authorization in 2006-2023 in the European Union. The gray curve excludes the seven coronavirus disease 2019 (COVID-19) vaccines and treatments that were granted CMA (N = 2 in 2020; N = 4 in 2021; and N = 1 in 2022). Adapted from Bloem et al. (3) and updated with data from the European Commission’s Union Register of medicinal products (4). CMA, conditional marketing authorization.
FIGURE 2
FIGURE 2
Relationships between characteristics of conditional marketing authorization (CMA) and ethical issues. CMA requirements result in three ethically relevant characteristics of CMA: addressing an unmet medical need, uncertainty about the benefit-risk balance, and mandatory CMA studies. These characteristics and their intersections provoke ethical issues, which are listed in bold. Below each ethical issue, the implicated ethical principles and values are shown. CMA, conditional marketing authorization.
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References

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