The new leadership at the US Food and Drug Administration is wasting little time in taking steps to make the agency more efficient
and effective. The FDA is announcing new rules and policies almost daily and strengthening enforcement of manufacturing and
marketing requirements to show the industry it means business.
FDA Commissioner Margaret Hamburg began by reorganizing her immediate office to emphasize the importance of food safety and
scientific integrity and to encourage speedy compliance with rules. An expanded office of external affairs will improve communications
with the media, health professionals, and special interest groups. Several crosscutting activities—bioresearch monitoring,
combination products, orphan drugs, and pediatrics—will be managed by a new Office of Special Medical Programs.
Budget development will shift to the policy office to better explain to Congress how the FDA spends 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 further expand resources.
A prominent addition is the Center for Tobacco Products, authorized by Congress in June and launched by the FDA in record
time. The Center will be headed by Lawrence Deyton, an MD with experience in academia, public health, and Congress, and successful
in revitalizing smoking cessation programs as a top health official at the Department of Veterans Affairs. The tobacco legislation
charges the FDA with enforcing marketing restrictions on tobacco products, regulating product ingredients, and taking action
to reduce youth smoking. Leeway for the FDA to promote smoking cessation could stimulate development of new nicotine replacement
STRESS ON SCIENCE
Hamburg is intent on positioning the FDA as a science-based regulatory agency where politics don't override medical judgment
in making regulatory decisions. The agency hopes to deal effectively with internal disputes over product safety and approval
actions and to avoid public controversies, such as the one that delayed approval of an over-the-counter version of the Plan
B emergency contraception pill.
At the August meeting of the 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 by the National Institutes of
Health. She emphasized the importance of "revitalizing and re-energizing" support for the Critical Path Initiative and acknowledged
concerns about the slim pipeline for new drugs and medical products. An increased focus on regulatory science and efforts
to make the FDA review process as efficient as possible can benefit innovation, she commented, while the current lack of transparency
about the FDA review process can be "very stifling to innovation." Here Hamburg echoed opinions expressed by biotech companies
and venture capitalists at a recent meeting organized by the FDA and the White House's Office of Science and Technology Policy
to address innovation and transparency.
Under the Hamburg regime, FDA Chief Scientist Jesse Goodman will spur innovation as head the Office of Science and Innovation.
He also heads up a new office of scientific integrity that will resolve scientific disputes within the agency, coordinate
responses to outside charges of inappropriate actions, resolve appeals, and oversee advisory committees. And Goodman will
oversee counterterrorism and emerging threats, which involves coordinating the FDA's response to the current influenza pandemic,
facilitating access to effective medical countermeasures, and authorizing emergency use of needed therapies.
In addition to these organizational changes, the FDA has proposed new rules designed to expand patient access to investigational
medicines, finalizing proposals made in 2006 and long sought by patient advocates. The policy makes it easier for seriously
ill individuals to obtain unapproved drugs and clarifies for manufacturers when they may be able to charge patients for an
experimental drug outside a clinical trial.
The FDA also has issued a proposed rule requiring electronic submission of postapproval adverse event (AE) reports for drugs,
biologics, and medical devices. Most (80%) of drug AEs already are submitted to the agency online or in digital media, and
the new policy aims to prompt small biotech firms and device makers to follow suit. The FDA is establishing a new web portal
that it says will be easier for small companies and individuals to use for AE reporting. There are no changes in the scope
and timing of AE filings, which should make it fairly easy for manufacturers to comply. However, the FDA estimates it will
save $2.4 million a year and, more importantly, will gain access to safety information much more quickly.