The European Medicines Agency has published for public consultation a draft reflection paper on establishing efficacy based on single arm trials submitted as pivotal evidence in a marketing authorisation.
Randomised controlled trials (RCTs) are the standard for providing confirmatory evidence on the efficacy of a new treatment. However, in a relevant proportion of marketing authorisation applications the pivotal clinical data stems from single-arm trials (SATs). This is observed across different therapeutic areas, including for rare diseases.
The purpose of this reflection paper is to outline the current thinking about SATs that are submitted as pivotal evidence for establishing efficacy in marketing authorisation applications. Defining general conditions under which SATs may be considered acceptable as pivotal evidence for marketing authorisation is outside the scope of this reflection paper. Such considerations strongly depend on the clinical context and other things such as the drug treatment modality. It is the responsibility of the applicant to adequately justify to regulators why a SAT, which deviates from the standard approach of providing pivotal evidence on efficacy through RCTs, can provide clear pivotal evidence of efficacy. Obtaining scientific advice is therefore strongly recommended to discuss whether pivotal evidence from SATs may be considered acceptable for seeking marketing authorisation for a specific development programme.
Comments should be provided using a specific template and sent to RP-SATs@ema.europa.eu by 30 September 2023.
Read the draft here.
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