Will Precision Medicine Displace the Randomized Trial?

Maurie Markman, MD
Published: Sunday, May 20, 2018
Maurie Markman, MD
Maurie Markman, MD
There is a most difficult struggle within the cancer research establishment—the often intense debate regarding the future direction of clinical investigation. The tension comes from the conflict between academic traditionalists and the newer set of targeted medicine enthusiasts. The traditionalists continue to firmly believe that the phase III randomized trial examining clinical outcomes within a defined population of patients with cancer should remain the only true gold standard for the evaluation of antineoplastic therapeutic efficacy. The opposing contingent’s views on optimal research design have substantially shifted to a focus on the need for clinical decisions to be far more personalized based on knowledge of the unique biology of the cancer within the individual patient (aka, precision medicine).

It should be acknowledged that many who support the need for randomized studies fully recognize that in certain settings, biologically precise data are realistically not possible to obtain (at least not within a period of time acceptable to society), but this reluctant acceptance comes with the loudly proclaimed caveat that any other form of evidence is of far less quality and value. Two recently published peer-reviewed cancer research efforts help to define the divergent views.

Molecularly Targeted Strategy

The first paper presents data from a basket trial, MyPathway, designed to explore the potential clinical utility associated with the off-label use of several commercially available molecularly targeted antineoplastic agents.1 (Note: My employer, Cancer Treatment Centers of America, participated in this trial, and I am a member of the sponsoring company’s [Genentech] gynecologic cancer speaker’s bureau.) The study involved 251 patients with 35 different tumor types; 230 patients were evaluable for response or discontinued treatment before they could be evaluated. This trial focused on an objective evaluation of off-label drug use. One-quarter of the population achieved an objective response, including 14 (38%) of 37 patients with colon cancer whose malignancies overexpressed the HER2 receptor and 6 (43%) of 14 patients with non–small cell lung cancer and a BRAF V600 mutation who were treated with a specific rationally considered molecularly targeted antineoplastic strategy.1

Investigators were able to report early results from these targeted cohorts. They believe the results may help confirm the validity of administering targeted therapy based upon molecular aberrations in multiple tumor types and encourage additional studies.

Traditional Approach

To represent the opposite perspective, one would be hardpressed to find a better example of the search for statistical certainty regarding a particular question in clinical oncology than the results of the international IDEA study designed to determine the utility of 3 versus 6 months of adjuvant oxaliplatin-based (folinic acid, fluorouracil, oxaliplatin [FOLFOX]; or capecitabine, oxaliplatin [CAPOX]) chemotherapy for stage III colon cancer.2
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Online CME Activities
TitleExpiration DateCME Credits
Community Practice Connections™: 22nd Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas and MyelomaMay 30, 20192.0
Online Medical Crossfire®: 5th Annual Miami Lung Cancer ConferenceMay 30, 20196.5
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