Assessing Treatment Benefit in Immuno-Oncology

The management of cancer patients in clinical practice is undergoing a revolution due to the introduction of effective immunotherapy. Several different strategies currently rely on harnessing the immune system to fight tumors, leading to unprecedented therapeutic success. However, immunotherapy has also created the need to revisit the assessment of treatment benefit, possibly because its mechanism of action leads to effects on short- and long-term outcomes in a manner that differs from those of other modalities. For example, clinical trials of immune checkpoint inhibitors have disclosed novel issues related to the assessment of objective responses and have reinstated overall survival as a reliable primary endpoint, often to the detriment of progression-free survival (PFS). Moreover, pseudoprogression has become an issue in some tumor types, and the duration of responses has emerged as a relevant endpoint. In parallel, the limitations of current statistical methods of survival analysis that are based on the proportional-hazards assumption have become more apparent, and novel methods have been proposed.

In this webinar, featured speakers will start by reviewing key issues in the recent literature related to endpoints in immuno-oncology, with a brief overview of the four different sets of criteria that have been proposed for response assessment in immunotherapy. Examples from the recent literature will be used to illustrate the limitations of current statistical methods in the assessment of progression-free survival and overall survival, and review the novel approaches that have been proposed both for the design and analysis of clinical trials, including the assessment of the net benefit through the use of generalized pairwise comparison.

Speakers

Marc Buyse, ScD, Chief Scientific Officer, International Drug Development Institute (IDDI)

Marc Buyse 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 (http://publicationslist.org/marc.buyse).

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Everardo Saad, MD, Medical Director, International Drug Development Institute (IDDI)

Everardo Saad has over 15 years of experience in Medical Oncology and clinical trial designs. 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 therapies for cancer patients.

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