Viewpoints in JAMA

The Safety Risks of Innovation - The FDA's Expedited Drug Development Pathway


Thomas J. Moore, A.B., of the Institute for Safe Medication Practices, Alexandria, Va., and Curt D. Furberg, M.D., Ph.D., of the Wake Forest School of Medicine, Winston-Salem, N.C., examine the FDAs program for speeding approval of new drugs, known officially as the "Expedited Drug Development Pathway." The authors discuss the complex safety issues raised by the expedited approval process, and cite three examples involving drugs for treatment of cancer, multiple sclerosis, and stroke prevention, with these new drugs including some agents with limited clinical trial data and substantial risks.

“It takes years of development, costly clinical trials, and extensive analyses to establish the clinical conditions under which new drugs will do more good than harm. Risks persist, even with standard approvals. Meanwhile, the U.S. Senate, in approving a regular 5-year update to the Food, Drug, and Cosmetic Act, proposed a further expansion of expedited review, with a new category for ‘breakthrough drugs.’ For physicians, the FDA's emphasis on rapid drug approval underlines the importance of the 6 ‘principles of conservative prescribing’ that include the warning, ‘Exercise caution and skepticism regarding new drugs.’”

(JAMA. 2012;308[9]:869-870.


Clinical Trial Data as a Public Good


Marc A. Rodwin, J.D., Ph.D., of Harvard University, Cambridge, Mass., and John D. Abramson, M.D., M.S., of Harvard Medical School, Boston, discuss the need to improve public access to clinical trial data, and suggest mandatory disclosure of the standardized Clinical Study Report (CSR) for all clinical trials involving FDA-approved drugs.

The CSR summarizes the trial, clinical end points, methods, key data, and data analysis and includes the most pertinent information about a clinical trial in an easily analyzed format. “Drug manufacturers already produce these reports to meet international and national regulatory requirements. Making CSRs publicly available would not be expensive, yet disclosure would promote research integrity, medical knowledge, and public health. Furthermore, CSRs are more likely to be reliable than other summaries. Drug manufacturers submit CSRs to public authorities when they seek marketing approval and cannot alter or delete data without potentially jeopardizing their relationships with regulatory agencies and risking criminal prosecution,” the authors write.

They add that “to expand public access to clinical trial data, Congress could require the FDA to disclose all CSRs that drug manufacturers submit when seeking marketing approval and also could require drug firms to disclose CSRs for all clinical trials they conduct for drugs that they market in the United States. Those actions would go a long way toward making the knowledge derived from clinical trials truly a public good.”

(JAMA. 2012;308[9]:871-872.