pooled analysis FDA negative phase 3 trials phase 3 trial RCT PRISMA RCT's

Association of Industry and Academic Sponsorship With Negative Phase 3 Oncology Trials and Reported Outcomes on Participant Survival: A Pooled Analysis

by Alfredo Addeo, MD; Glen J. Weiss, MD, MBA; Bishal Gyawali, MD, PhD

May 10^th 2019
8 mins
JAMA Netw Open. 2019;2(5):e193684

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Summary

In this study of trials published in 2016 through 2018, approximately 40% of negative phase 3 RCTs in oncology were conducted without supporting phase 2 trials, and such phase 3 trials were sponsored by both academia and industry. On the basis of our results, proactive steps from regulators and ethical committees should be contemplated to encourage greater consideration before allowing conduct of phase 3 RCTs despite negative results from phase 2 trials in the interest of protecting patients and trial resources.

Phase 3 randomized clinical trials (RCTs) are the final barrier in establishing the efficacy of cancer drugs. One study estimates that only 3.4% of cancer drugs being evaluated in phase 1 trials are approved by the US Food and Drug Administration, with most (35.5%) failing in the phase 3 stage when all indications are considered. For lead indications only, the numbers look better but are still disappointing, with 11.4% probability of success overall and 48.5% probability of success at the phase 3 stage of development. Phase 3 RCTs usually involve more patients and resources than earlier phases and are costly undertakings.

According to a 2014 report, the cost of a cancer drug trial was $22.1 million, $11.2 million, and $4.5 million, for a phase 3, phase 2, and phase 1 trial, respectively. Thus, a drug failing the phase 3 trial (negative phase 3 trial) is an undesirable outcome, even from an economic point of view.....

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