FDA's Califf Seeks New “Ecosystem” for Clinical Trials

Current clinical trials are regarded as “too slow, too expensive, not reliable, and not designed to answer the important questions,” according to FDA’s new deputy commissioner for medical products &tobacco, Robert Califf. A veteran of multiple initiatives to modernize the biomedical clinical research enterprise, Califf emphasized the importance of improving the quality and efficiency of clinical trials as key to improving public health and to encouraging biomedical innovation.

Califf has long advocated for building a “learning health care system,” where  information from individual health records can be accessed to inform treatment decisions and support medical product development. Now we’re “on the verge of a tipping point” in clinical trial reform, Califf predicated at a Washington seminar on “Re-Engineering Clinical Trials” organized by the Tufts Center for the Study of Drug Development and ICON. Multiple efforts so far have realized “incremental improvements” in research operations, many negotiated as part of drug user fee agreements, Califf observed; he sees real change on the horizon due to important advances in data systems, integration of health care delivery operations, plus greater public attention to the flaws in the clinical research enterprise.

FDA staff is implementing initiatives designed to achieve more efficiencies in clinical research, such as electronic informed consent, adoption of mobile technologies to measure clinical response, use of e-health records in designing research protocols, and adoption of common data standards and terminologies in research studies to support applications filed with FDA. Further development of research networks at the National Institutes of Health (NIH) and the Patient-Centered Outcomes Research Institute (PCORI), along with expansion of FDA’s Sentinel System, will provide added infrastructure for conducting more efficient clinical research.

Califf spoke at a meeting to announce publication of “Re-Engineering Clinical Trials,” a book presenting multiple articles on the issue, edited by ICON executives Peter Schuler and Brendan Buckley (Elsevier’s Academic Press, 2015), with an introduction and contributions from Kenneth Getz and Kenneth Kaitin of Tufts. Getz, Schuler, Buckley and other experts at the seminar presented strategies for optimizing drug development by improving clinical site operations, simplifying protocol design, and improving systems for recruiting trial participants.

The book is timely, as House Energy and Commerce Committee chairman Fred Upton (R-Mich) emphasized the importance of modernizing clinical trials to achieve personalized medicine. Upton sees provisions in the 21^st Century Cures legislation furthering this goal, including measures that make it easier to conduct pediatric studies and achieve more flexibility by utilizing biomarkers and adaptive trial designs. Clinical trials “are slow and expensive,” said Upton, urging efforts to make them “faster, safer and more personalized” by agreeing on a final Committee bill this month.  

Califf similarly described a new “ecosystem” for clinical research as key to supporting the Precision Medicine Initiative and efforts by FDA to “put patients first.” The traditional clinical trial enterprise, he advised, “ignores these forces at its peril.”