Clinical-Trial System Said Ripe for Reform

The Food and Drug Administration (FDA) is collaborating with Duke University researchers to find ways to improve the efficiency and quality of clinical trials.

The Clinical Trial Transformation Initiative (CTTI), first announced in November 2007, is a public-private partnership hosted and coordinated by Duke University with participation from all areas of clinical research.

The FDA has been criticized in recent years for inefficiency and lack of sufficient oversight. The number of new drug approvals, especially new compounds, has declined in recent years. In 2007, the FDA approved only 19 new drugs, the lowest since 1983, according to a Bloomberg/Arizona Daily Star report on January 10. Meanwhile, the cost of conducting a late-phase clinical trial can amount to tens of millions of dollars.

Drug companies are increasingly conducting global clinical trials outside of the United States so that more patients can be enrolled at more sites simultaneously and more data can be obtained sooner to meet FDA requirements for new-drug review. However, global trials, including some conducted in Third World countries, increase the agency's difficulty in monitoring the quality of these trials.

The CTTI partnership is intended “to develop new standards and identify new methods and technologies that improve safety, boost the quality of information derived from clinical trials, and make the research process more efficient,” according to its Web site.

The CTTI “was set up by the FDA in response to growing frustration over the complexity in conducting clinical trials and the long time frames involved in completing them,” said Judith Kramer, M.D., executive director of the CTTI and an associate professor of medicine at Duke University School of Medicine. She told Psychiatric News that the CTTI has heard from many people directly involved in clinical trials who are frustrated by the inefficiencies in the system.

The cost and time of conducting trials limit the number of clinical questions that can be answered. Industry-sponsored clinical trials tend to focus on data needed for regulatory approvals. Physicians often find themselves in a position of prescribing FDA-approved drugs“ off-label” without adequate clinical-trial data for diseases affecting a small patient population without an approved treatment.

Fundamentally changing the existing framework, however, requires stakeholders from different fields with different perspectives to develop global solutions for these problems, Kramer emphasized. Although the CTTI is still in the planning and organizing stage, Kramer expressed optimism that it can achieve its goals: “We ... announced the executive committee in May that consists of FDA representatives in the drug and medical device divisions; a patient advocate; representatives from the pharmaceutical industry, biotechnology, device companies, and contract research organizations; academic researchers; an at-large representative from the Tufts Center for the Study of Drug Development; and a liaison from the National Institutes of Health.”

Kramer explained that CTTI plans to solicit ideas and participation from a variety of members. “We already have 36 organizations that have committed to join CTTI,” she said. “So far our membership includes a broad cross-section of industry, contract research organizations, professional societies, academic institutions, investigators, trade organizations, even a law firm specializing in regulatory law, and a private investment company.”

Among the current members are the American College of Neuropsychopharmacology and American College of Clinical Pharmacology.

If successful the findings derived from the CTTI will lead to changes in not only industry-sponsored clinical trials but also in pure academic research. “The quality of clinical trials is important to not only new drug approvals but also for medical practice,” said Kramer.

The FDA has committed personnel but provides no funding for the research and other activities in the CTTI. The projects are expected to be funded by membership fees, which are based on a tiered structure depending on the type of the organization or company.

Additional information about the CTTI is posted at<trialstransformation.org>.▪