Why do so Many Phase III Fail?

Randomized controlled trials (RCTs), particularly in phase 3, are the preferred sources of evidence for drug approval and reimbursement decisions. Moreover, phase 3 trials are at the top of the evidence-based medicine pyramid, thus representing the gold-standard for judgements about the efficacy and safety of new treatments and their worth in clinical practice. Nevertheless, regulatory and clinical decisions sometimes need to be made on evidence of lower level, such as that provided by phase 2 trials, particularly in oncology. As a matter of fact, nearly one-third of anticancer drug approvals by the US Food and Drug Administration (FDA) between 1992 and 2017 were accelerated approvals, one of the four types of expedited programs offered by that Agency.1,2  

Authors:

Marc Buyse, ScD, and Everardo Saad, MD

Conversation between a Sponsor and a Statistician about Phase II/III trials in oncology

Phase I trials in oncology: Pros and cons of current designs

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AUTHORS 

Marc Buyse, ScD, Chief Scientific Officer and Founder, IDDI

Marc holds a ScD in biostatistics from the Harvard School of Public Health (Boston, MA). He is the founder of the International Drug Development Institute (IDDI) and of CluePoints, two biostatistical service organizations based in the US and Europe. He is interested in clinical trial design, meta-analysis, validation of biomarkers and surrogate endpoints, statistical methods in oncology, statistical detection of errors and fraud, statistical monitoring of clinical trials, and medical data sharing.

 

Everardo Saad, MD, Medical Director, IDDI

Dr. Everardo Saad has nearly 20 years of experience in Medical Oncology and clinical-trial design. He graduated in Medicine and trained in Internal Medicine in Sao Paulo, and did his fellowship in Medical Oncology at the University of Texas M.D. Anderson Cancer Center, in Houston. After practicing for several years, he shifted his professional career towards education and research in Medical Oncology, and has a special interest in clinical trial methodology, the assessment of endpoints, and the development of novel therapi

Why do so Many Phase III Fail?

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