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Non-comparative trials for marketing authorisations: EMA consultation

 Advancing Healthcare Policy   Prescrire has contributed to a consultation on the use of non-comparative clinical trials to obtain marketing authorisation.
Full article available for download (free)

In September 2023, Prescrire submitted its response to a public consultation organised by the European Medicines Agency (EMA) on the use of non-comparative clinical trials as the main ("pivotal") evidence of efficacy in marketing authorisation applications (1,2).

Prescrire considers that the reflection paper rightly highlighted the methodological weaknesses of non-comparative trials in evaluating the potential efficacy of a drug. It is because of these weaknesses that, with a few rare and substantiated exceptions, marketing authorisations should not be based on such trials. Prescrire felt it was regrettable that the EMA's preparatory document:

  • Does not clearly spell out what these trials can do (generate hypotheses) and what they cannot do (demonstrate a causal relationship between the treatment and the outcomes observed);
  •  And does not define, from the outset, the handful of exceptional situations in which the use of a non-comparative trial might be considered an acceptable basis for marketing authorisation.

Drawing on concrete examples, Prescrire expressed its concern about the fact that, despite the known weaknesses of non-comparative trials, the EMA is increasingly accepting them as the sole basis for marketing authorisations (2).

1- EMA "Reflection paper on establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorisation" 17 April 2023: 15 pages.
2- Prescrire Editorial Staff "Final Prescrire response to EMA consultation guideline single arm trials" 20 September 2023: 9 pages.

©Prescrire 1 July 2024

Source: "Non-comparative trials for marketing authorisations: EMA consultation" Prescrire Int 2024; 33 (261): 195. Free.

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See also:

"Proving a treatment's
efficacy. Double-blind
randomised comparative trials,
the foundation of a rigorous
scientific approach"
Prescrire Int 2023;