Stress on science

Hamburg is intent on positioning FDA as a science-based organization whose regulatory decisions are based on medical and technical judgments rather than politics. The agency hopes to deal effectively with internal disputes over product-safety and approval actions and to avoid public controversies such as that which delayed approval of an over-the-counter version of the Plan B emergency contraception pill.

At the August meeting of FDA's Science Advisory Board, Hamburg highlighted the importance of strengthening regulatory science as an academic field, noting that it has atrophied compared with biomedical research, which has surged following investment by the National Institutes of Health and other research organizations. She emphasized the need to "revitalize and re-energize" support for the Critical Path Initiative and acknowledged concerns about the slim pipeline for new drugs and medical products.

One sign of the times is Hamburg's decision to replace the head of the Center for Devices and Radiological Health (CDRH). Veteran FDA official Dan Schultz resigned in August as CDRH director in the wake of several high-profile device-safety problems. CDRH staff had complained that management had ignored its concerns regarding the safety and efficacy testing of new products seeking market approval.

Under the Hamburg regime, FDA Chief Scientist Jesse Goodman will ensure scientific integrity in agency deliberations and encourage research and innovation as the head of a new Office of Science and Innovation (OSI) for resolving scientific disputes within the agency. Goodman will coordinate responses to charges of inappropriate action, manage appeals, and oversee advisory committees.

Another of Goodman's assignments will be to coordinate FDA's involvement in counterterrorism and emerging threats, which involves managing 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 and authorize emergency use of needed therapies.

As head of OSI, the chief scientist also will make the case for FDA support of high-quality scientific projects. That effort will involve expanding the Critical Path Initiative and working with FDA's Center for Toxicological Research in Arkansas. Goodman anticipates playing a role in encouraging personalized medicine and in promoting comparative-effectiveness research. FDA hopes to tap into biomedical data resources at the Department of Health and Human Services that can identify patient populations likely to benefit from certain treatments. Such information should help design clinical studies that can produce useful information about how medical products should best be used to yield benefits and avoid harm.

Access and information

In addition to these organizational changes, FDA is rolling out new programs and policies important to manufacturers. One set of new rules aims to expand patients' access to investigational medicines by finalizing proposals made in 2006 and long sought by patient advocates. The policy makes it easier for seriously ill individuals to obtain unapproved drugs under the treatment investigational new drug policy and to have early access to an approved drug for an unapproved indication. Manufacturers also gained clarification about when they may charge patients for an experimental drug outside a clinical trial.

An important FDA initiative is to promote the electronic submission of postapproval adverse event (AE) reports for drugs, biologics, and medical devices, as outlined in a new proposed rule that aims to eliminate paper submissions of safety reports. Many large pharmaceutical manufacturers already use FDA's Electronic Submission Gateway to file about 80% of individual case safety reports under a pilot program. The new policy aims to prompt small biotechnology firms and device makers to follow suit by offering a new web portal for agencywide safety reporting. FDA is establishing the portal as part of its MedWatch Plus initiative.

The proposed rule makes no changes in the scope and timing of manufacturers' AE filings and continues to use the standards for data elements and format established by the International Conference on Harmonization (ICH) and adopted in Europe and Japan. For these reasons, FDA anticipates that firms already submitting AE reports electronically will have few additional costs to comply with the new requirements. FDA, however, will save $2.4 million per year by eliminating the manual conversion of paper reports to electronic form. More important will be speedier access to important safety information. FDA says that reviewers can obtain electronic safety submissions in two days, compared with two weeks to get paper reports into the agency's Adverse Event Reporting System.