Scott Gottlieb, deputy commissioner for medical and scientific affairs at the US Food and Drug Administration, gave an insightful look into the agency’s ongoing efforts to modernise the clinical research process in a speech delivered to the 2006 Clinical Research Educational Conference earlier this month.
At the heart of his comments was the sense that the clinical research process has stayed largely static in recent times – both in the choice of endpoints and the way the trials are designed an executed – and this needs to change.
Opportunities are being missed to take advantage of information technology tools to improve the way clinical trials are conducted and monitored and clinical investigators collaborate with each other and share information, claimed Gottlieb, while trials are not evolving to make use of new scientific tools such as genomics and proteomics.
That said, clinical trials are undergoing changes tending to become larger and more decentralized, and the FDA needs to adapt its monitoring processes to suit.
For example, the agency intends to improve supervision of multi-site clinical trials by developing new guidance on the use of supervisors who are not principal investigators, to ensure they are accountable to the FDA.
“Is it possible, reasonable or appropriate for a single principle investigator to oversee clinical trials involving 30 to 40 or more sites?” he asked the conference.
Other key issues that need to be addressed in the legislation include a need for more real-time rather than post hoc inspections of clinical trial sites, agreeing common standards for electronic data handling, and setting out clearly the proper role of institutional review boards, including what adverse event information should be reported to them, said Gottlieb.
Meanwhile, the discoveries of basic science – genomics and proteomics – need to be harnessed to help identify patients at risk of adverse events, those who stand to respond well to a drug, “and this will require a move away from the highly empiric approach that we use today,” he said.
Meanwhile, this patient-selection approach could help address another critical challenge, the agonisingly slow pace and low levels of recruitment for clinical trials.
“A recent survey by the American Society for Clinical Oncology, for example, reported that fewer than 4% of eligible cancer patients participate in clinical trials,” said Gottlieb, noting that the FDA is in the process of developing a guidance document on enrichment in clinical trial design that will address this issue.