In this blog we share a case study in which our statistical consulting team helped a client redesign an oncology pragmatic trial to address regulatory agency questions.
Project Background Our client has a healthcare clinic which aims to provide low-cost, effective therapies to complement existing Standard of Care treatment for a range of cancer indications. Through literature search, they had established that certain approved medicines may demonstrate a positive effect on cancer treatment when taken in combination with conventional therapy approaches. They wished to further assess the effectiveness of a regimen of selected treatments for patients with cancer.
Sponsor Challenge For ethical and practical reasons it is not appropriate to conduct a randomized trial in this setting. The client planned a pragmatic trial to evaluate the effectiveness of the interventions in the real world setting and produce results to allow general claims to be made. However, at the planning stage for the trial, the local regulatory agency rejected our client’s proposed pragmatic design as they considered that a number of aspects were not sufficiently well controlled. The agency was concerned that bias could be introduced, and so the client approached Cytel for support to redesign the trial.
Cytel Solution Cytel statistical consultants used the PICO process as a starting point to review and redesign the pragmatic trial. PICO is a technique used in evidence-based practice to frame and answer a clinical question around the following 4 pillars: P – Patient, problem or population I – Intervention C – Comparison, Control or Comparator O – Outcome
The statistical consultants worked to redesign the trial addressing these issues: • (P) Who precisely are the patients being treated, what cancers do they have, and where do they originate from geographically? • (I) It was not sufficiently clear in the original protocol what cancer indications the drugs were being used for specifically, or what combination of therapies was being used. In the redesigned pragmatic trial, Cytel consultants worked with the client to ensure these aspects were clarified. • (C) In collaboration with the client, Cytel statistical consultants conducted extensive work to establish what the controls would be- whether historical or concurrent within the trial itself. • (O) While in a randomized trial survival is measured from randomization to death, in a pragmatic trial setting, a different definition of survival needs to be created. Cytel statistical consultants worked to create this definition, as well as specifying when the endpoints would be measured for the historical and concurrent controls.
Outcome The new trial design robustly addressed the concerns of the local regulatory agency.
Cytel works extensively with clients in the oncology space creating innovative trial design and implementation solutions across the development life cycle. We will be attending ASCO June 2nd- 6th 2017 in Chicago- visit our experts at booth 20124 or click the button to pre-arrange a meeting.
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