MM-2: Contextualizing clinical trials by developing robust historical control arm cohort

Challenge

Currently, there are a relatively large number of approved agents and compounds under development to treat patients with Multiple Myeloma (MM). Given this situation, it is not feasible for clinical trials to capture every possible regimen in a comparator arm. Hence, an alternative solution is pursued here using HARMONY Big Data platform to develop multiple historical control arm cohorts to help contextualize clinical trial research.

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Project Leadership

• Marco DiBonaventura, Pfizer

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Impact

• To create ‘historical control’ arm cohorts to help contextualize clinical trial research in MM

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Project Summary

Patients with MM who have at least one clinical endpoint of interest (e.g., overall survival, progression free survival, or overall response rate) will be included in this study. The availability of patients in the HARMONY database will drive the sample size. Data for analysis will include Phase II, III, IV trials or real-world studies to strengthen the overall sample size. These patients will be categorized into three subgroups;

• Double class relapse – prior treatment with at least one immunomodulatory drug (IMiD) and one proteasome inhibitor (PI).
• Triple class refractory – refractory to at least one IMiD, one PI, and one anti-CD38 antibody.
• Newly diagnosed patients with MM.

 

Initial focus will be on the triple class refractory subgroup with a target sample size of 150-200 patients, then the project will be extended to other subgroups. Since most of the triple class refractory clinical trials are single arm, it creates further challenges and strengthens the need for historical control arm cohorts. Each historical control arm cohort will then be descriptively summarized with respect to demographics and clinical characteristics.                                     

As the HARMONY Big Data Platform is the largest MM register in Europe, the impact of development of such reference cohorts for clinical trial efficacy assessment will be highly valuable for researchers, and ultimately for patients, during the drug development process. The aim of this study is to develop historical control arm cohorts that will help put into context the results of numerous clinical trials in the MM space.

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Timeframe

Project completion is anticipated by the end of 2022.

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