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“21st Century Cures” Require Improved Trials Process

By Amanda Conschafter, Blog Editor

Innovation, data sharing and public-private partnerships could help modernize clinical trials, agreed witnesses at Wednesday’s “21st Century Cures” hearing by the House Energy and Commerce Committee Health Subcommittee. Citing an “expensive and antiquated” clinical trials model, Chairman Joseph Pitts (R-PA) and his colleagues queried representatives from the pharmaceutical industry, health care settings and academia about how to address clinical trials’ growing cost and complexity.

Witnesses repeatedly noted the challenge of patient participation. Paula Brown Stafford of Quintiles explained that finding participants can consume a disproportionate amount of the time required for clinical trials. Stafford estimated that identifying and enrolling trial participants takes “anywhere from 10 months to four years.” Roy Herbst, Ph.D. of the Yale Cancer Center echoed this sentiment; he recalled a trial in which researchers had to screen 100 patients to find only the 10 participants needed – inefficiency he characterized as “totally unacceptable.”

Several witnesses suggested using electronic health records to identify and engage patients in clinical trials. As Stafford explained, trial staff would “de-identify” patient data; pinpointing physicians who treat applicable patients instead of tracking the patients themselves. Asked how HIPAA laws affect access to electronic health records, Sundeep Khosla, M.D. of the Mayo Clinic acknowledged that regulations prohibit patient data from leaving the patient’s medical center – though that data could aid clinical trials. Jay Siegel, M.D. of Janssen Research and Development explained, “It’s important to protect privacy, but it can be done in ways that still advance research.”

Witnesses also offered suggestions for streamlining the trials process. Robert Meyer, M.D. of the University of Virginia School of Medicine recommended master protocols, which coordinate aspects of multiple clinical trials to reduce the logistical and cost burdens. He also suggested incorporating data collected via medical technology. Pulmonary patients, he offered as an example, can measure airflow easily from home. Using the data they collect there would reduce the need to assess them in clinic – potentially encouraging broader participation and improving patient retention. It could also cut costs.

Modernized clinical trial models also sparked discussion. Dr. Siegel suggested broader use of biomarkers, while several witnesses debated the use of surrogate endpoints to allow researchers to assess trial results sooner. Witnesses also promoted collaboration among public and private entities, as well as data sharing in pre-competitive trial stages. Several witnesses applauded the use of clinical trial networks such as Lung-MAP, a collaborative, multi-drug trial that uses multiple biomarkers to assign participants to one of several interrelated trials.

Few witnesses requested congressional action beyond continued financing. But the group expressed unanimous concern that clinical trials’ complexities not inhibit patients’ timely access to new treatments. As Ms. Stafford reminded the members of Congress, “Patients are waiting.”

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