Health Affairs: New trial model unlocks power of personalized medicine

Oncology clinicians may soon have hundreds of biomarker tests available to them to assist in developing personalized cancer treatments, but some healthcare professionals believe that novel funding approaches for biomarker tests’ studies—a systematic process to move biomarker tests through regulatory agencies and a body of evidence supporting biomarker tests’ benefits—will all be necessary before healthcare system stakeholders accept their use.

In the December issue of Health Affairs, Scott D. Ramsey, MD, PhD, of the Seattle-based Fred Hutchinson Cancer Research Center and his colleagues proposed a framework for the integrated approach to designing and implementing comparative clinical trials of personalized medicine technologies, including the use of biomarker tests.

Biomarker tests, also known as genomic tests, are used to measure the unique variables of individuals’ genetic material, proteins and other biological molecules. Those measurements can be used to develop individual assessments of the risk of future disease, the aggressiveness of a disease and possible outcomes of different treatments.

Ramsey and his colleagues believe that biomarker tests, which are often introduced into clinical practice without sufficient evidence of their benefits, have “tremendous potential for improved outcomes and lower treatment costs,” but propose comparative effectiveness research to “strengthen what is now a haphazard process for developing and marketing cancer biomarker tests.”

The researchers wrote, “Comparative effectiveness research will be more important at the intersection of personalized medicine and cancer than anywhere else because of the high risks and costs of poor-quality test information about this group of diseases.”

Ramsey and his colleagues suggested that future clinical trials of biomarker tests:

• Identify personalized medicine technologies that multiple stakeholder groups, specifically consumers and clinicians, consider high-priority;
• Be designed to meet the needs of multiple stakeholders;
• Use clinical trial groups with standing infrastructures in order to conduct studies on a large scale; and
• Employ funding mechanisms that distribute costs among private and public insurers.

“For such an approach to be viable,” Ramsey and colleagues wrote, “clinical trials groups, insurers and other interested stakeholders will need to develop standardized methods for assessing clinical validity and clinical utility, particularly in the context of rapidly evolving technology; establish a network of clinical organizations capable of rapidly enrolling patients in these studies; and develop collaborative mechanisms so that payers, clinicians, patients and researchers can work together to design, fund and implement studies.”