FDA leadership promotes scientific integrity, quality standards

Key Points

FDA officials are taking steps to erase the charges of incompetency and ineffectiveness that have plagued the agency for several years. FDA is announcing new rules and policies almost daily and strengthening enforcement of research, manufacturing, and marketing requirements to encourage adherence to regulatory standards.

FDA Commissioner Margaret Hamburg began by reorganizing her immediate office to emphasize the importance of scientific integrity and to encourage more transparency in regulatory decisions. An expanded office of external affairs aims to improve communications with the media, healthcare professionals, and interest groups. Several cross-cutting activities-bioresearch monitoring, combination products, orphan drugs, and pediatrics-will be managed by an Office of Special Medical Programs.

Budget development will shift to the policy office to improve the agency's ability to explain to Congress how FDA allocates its resources and why it may need additional funding. Hamburg is benefiting from a record $3 billion budget for the coming year (including some $600 million in user fees) but will be hard-pressed to maintain the recent growth in resources.

At the August meeting of FDA's Science Advisory Board, Hamburg highlighted the need to strengthen regulatory science as a field, noting that it has atrophied compared to the surge in biomedical research investment made by the National Institutes of Health. She emphasized the importance of "revitalizing and reenergizing" support for FDA's Critical Path Initiative and acknowledged concerns about the slim pipeline for new drugs and medical products. Efforts to make the FDA review process as efficient as possible can benefit R&D, she commented, whereas the current lack of transparency about FDA programs can be "very stifling to innovation."

Under the Hamburg regime, FDA chief scientist Jesse Goodman has the task of ensuring scientific integrity in agency deliberations, along with encouraging research and innovation. Goodman will head a new office for resolving scientific disputes among FDA staffers, coordinate responses to charges of inappropriate action, manage appeals, and oversee advisory committees. Another assignment will be to coordinate FDA's involvement in counterterrorism and emerging threats, which involves management of the agency's response to the influenza pandemic and to other diseases that threaten public health. Goodman's office will facilitate access to effective medical countermeasures, authorize emergency use of needed therapies, and oversee FDA counterterrorism and pandemic planning.

As head of the Office of Science and Innovation, the chief scientist also will make the case for FDA support of high-quality scientific projects. That will involve expansion of FDA's Critical Path Initiative, encouragement of personalized medicine, and promotion of comparative effectiveness research. FDA hopes to tap into data resources that can identify patient populations likely to benefit from certain treatments. Such information, says Goodman, should help design clinical studies that can produce useful information on how medical products should best be used to yield benefits and avoid harm.

FDA is also rolling out new programs and policies important to drug R&D. One set of new rules would expand patient access to investigational medicines by finalizing proposals made in 2006 that have been long sought by patient advocates. The intent of the policy is to make it easier for seriously ill individuals to obtain unapproved drugs under the treatment IND (investigational new drug) policy or to have early access to an approved drug for an unapproved indication.

One proposed FDA rule is designed to expand electronic submission of postmarket adverse event (AE) reports for drugs, biologics, and medical devices. Most (80%) of the drug AEs from manufacturers already are submitted to the agency online or by digital media. FDA seeks to make it easier for small companies, healthcare professionals, and individuals to report safety problems through a new Web portal for agency-wide safety reporting, which FDA is establishing as part of its MedWatch Plus initiative. FDA anticipates that for manufacturers, the cost of compliance with the new e-reporting policy will be relatively low and that the agency will save $2.4 million/y by eliminating the manual conversion of paper reports to electronic form. More important, though, will be speedier access to important safety information; FDA says that reviewers are able to obtain electronic safety submissions in 2 days, compared to 2 weeks to get paper reports into the agency's Adverse Event Reporting System.

Ms Wechsler is a Washington-based reporter specializing in federal and state healthcare issues.