Researchers, policy experts to examine issue of declining enrollment in clinical trials

Leading researchers and policy experts from across the country will come together June 27 and June 28 at Mount Sinai School of Medicine in New York to examine the issue of declining enrollment in clinical trials. The two-day workshop is co-sponsored by Mount Sinai's Institute of Translational Sciences through the Department of Health Evidence and Policy and the Institute of Medicine of the National Academies.

"Remarkable progress has been made understanding disease, but turning scientific findings into better patient care requires clinical studies, and clinical studies require volunteers," said Dennis S. Charney, MD, Dean of Mount Sinai School of Medicine. "Without clinical trials, medical science would have never realized great discoveries such as the value of antibiotics for peptic ulcer disease. But despite the importance of clinical research, there is growing recognition that the U.S. clinical trials enterprise is unable to keep pace with the demand for actionable research results."

Participation in clinical trials is declining nationwide, and over 90 percent of all clinical trials are delayed in large part because of difficulty with patient enrollment. Studies have shown that fewer than five percent of all eligible adult patients are enrolled in therapeutic studies, and this proportion is even lower among the elderly, women, and racial and ethnic minorities. About 20 percent of the principal investigators in clinical trials fail to enroll even one patient, and another 30 percent fail to meet their overall goals for patient enrollment.

The Institute of Medicine found that 40 percent of cancer trials funded by the National Institutes of Health are never completed or published, and of those that made it to completion, many were late. Low enrollment, and disproportionate enrollment of women and minorities, also raises questions about whether trial results can be reliably generalized to the overall population.

"There are many possible causes that have been identified for the decline in clinical trial participation," said Hugh A. Sampson, Dean for Translational Biomedical Research at Mount Sinai School of Medicine. "There is a tremendous lack of public awareness about the importance of clinical trials. Patients also are often hesitant about being randomly assigned to a traditional or control treatment. Misunderstanding or mistrust of the research community also is a growing problem. Cultural, linguistic, and literacy barriers, as well as financial obstacles due to insurance limits, also undermine enrollment."

Clinical trial enrollment issues also affect productivity in the development of new pharmaceuticals. The Tufts Center for the Study of Drug Development has reported that while global research and development spending for new pharmaceuticals grew at a rate of six percent annually from 1997 to 2008, the number of New Drug Application approvals over that time showed a steady downward trend.

"Strong patient desire for investigational therapy has undermined many trials, among them hormone therapy for post-menopausal women, anti-arrhythmic drugs for atrial fibrillation, and high-dose chemotherapy with autologous bone marrow transplantation for advanced breast cancer," said Annetine C. Gelijns, PhD, Professor of Health Policy and Co-Chair of the Department of Health Evidence and Policy, Mount Sinai School of Medicine. "These therapies were all highly touted and widely used—before rigorous clinical trials could demonstrate that they posed more risks than benefits. Off-trial use of these untested therapies absorbed much of the candidate pool and subsequently, the trials took longer than expected to complete."

The two-day workshop at Mount Sinai School of Medicine is intended to:

• Define and discuss the problem of inadequate public engagement in clinical trials.

• Identify the structures and culture of health care systems and delivery organizations that fail to support or engage with the clinical trial enterprise, and suggest potential solutions.

• Discuss how academic medical centers can create successful community partnerships to improve public engagement in clinical trials.

• Highlight and discuss models/methods, both proven and yet to be tried, of public engagement, including the media's role in public engagement.

• Examine novel clinical trial designs that work.

• Move forward a public discussion to create a comprehensive strategy for enhanced public engagement in clinical trials.