OBJECTIVES: Adaptive trials allow modifications to the trial design following initiation of the trial, and are of particular interest for research in oncology, increasing trial flexibility, efficiency, and the speed at which novel therapies can be evaluated. This study aimed to identify the likelihood for oncology therapies, investigated via adaptive trial designs, to obtain regulatory approval and positive HTA recommendations.

METHODS: Adaptive trials, investigating oncology treatments, were identified from searches in PubMed, ClinicalTrials.gov and from grey literature. Two types of data were gathered for each treatment. First, the status of regulatory approval by the EMA and the MHRA was verified for the treatments investigated in each trial, and second, whether the adaptive trial was used to support the regulatory approval decision. The available HTA reports in the UK, France, Germany, Italy, and Spain were investigated as well as the utilization of adaptive trials in HTA submissions.

RESULTS: Preliminary data identified 30 adaptive trials, representing 22 Phase I and/or Phase II trials and eight Phase III trials, investigating more than 100 oncology treatments. Until April 2022, regulatory approval was achieved for more than 20 treatments in the indications investigated, of which 15 are supported by adaptive trials. Adaptive trials were not used as the pivotal trial for any of the treatments to have achieved regulatory approval. There were only two treatments in which adaptive trials were used to support HTA submissions.

CONCLUSIONS: The results of this study indicate that adaptive trials have been used in the earlier stages of development of oncology therapies, and to identify the most promising therapies to investigate into Phase III development.