Clinical trials are the systematic way that modern medicine determines whether new treatments are safe and effective. But while the process is critically important to improved health care, the number of new drugs submitted to the U.S. Food and Drug Administration in recent years is only about half what it was in the 1990s.
To experts at a conference cosponsored by AAAS, this is symptomatic of a system ill suited for 21st-century medicine. Today’s clinical trials, in an effort to measure the often subtle effect of new therapies, tend to be huge and unwieldy. Now the search is under way for more streamlined, cost-effective, and sensitive ways to assemble patients and collect data.
“Our clinical trials system is falling behind the needs of society, and we have to figure out a way to do it more efficiently and better,” said Duke University’s Robert M. Califf, a leading U.S. expert on the process.
He delivered this blunt assessment in a keynote address to more than 200 participants at “Clinical Trials: Present Challenges and Future Opportunities,” a conference held 6 to 7 June on the campus of the National Institutes of Health (NIH). The National Library of Medicine and the Friends of the National Library of Medicine joined AAAS as cosponsors.
When the National Library of Medicine created www.clinicaltrials.gov in 2000, the Web site was seen as revolutionary. It now includes most new U.S. clinical trials and many overseas, detailing where and how a study will be conducted, the type of patients recruited, and trial results.
The Web site makes this information public in a structured format for the very first time, said its director, Deborah A. Zarin, “thereby ensuring objective and comprehensive reporting and mitigating opportunities for positive ‘spin’ of study results.”
PatientsLikeMe, a privately owned Web site, aims to empower patients in the clinical trial process by making it easier for them to share information, said Paul Wicks, the site’s research and development director.
He described how a group of patients with amyotrophic lateral sclerosis greeted initial reports that low doses of the drug lithium carbonate slowed progression of their disease. One patient located a paper abstract in Italian, used Google software to translate it into English, and shared it online.
“Before it was even published [in English], there were more patients in our system taking lithium prescribed for them by their doctors than were in the [Italian] clinical trial,” Wicks said. While the treatment proved ineffective, the response showed how patients might actively engage in managing their own care.
Participating in a clinical trial means multiple visits to researchers, and many patients decide that it is not worth the hassle. But the pharmaceutical company Pfizer used the conference to introduce its test of the first virtual clinical trial, using a drug approved for bladder control.
Web-based and mobile phone apps are being used to manage recruitment, enrollment, data entry, and monitoring. Blood draws can be scheduled online, and the drug is shipped directly to the patient. Physician assistance is available 24/7 by phone.
Some conference participants expressed curiosity but also unease with a virtual approach that could eliminate face-to-face contact with study patients.
Another strategy for reducing the size and complexity of future trials could be to examine how a new drug or device works at the cellular or molecular level, Califf said, and evaluate these responses in patients. This approach could help researchers better anti-cipate risks and benefits earlier in a trial.
“It is critical that we consider how these developments will affect the more traditional ways that clinical research has been done,” said Mark S. Frankel, director of AAAS’s
Scientific Responsibility, Human Rights and Law Program, “and what that will mean for translating findings into treatments.”