Opening the Post-Approval Summit at Harvard on May 14 and 15, keynote speaker Janet Woodcock, M.D., director of the Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), outlined her agency’s expanded authorities in the area of post-approval research oversight brought about by the passage of the FDA Amendments Act (FDAAA) into law at the end of March.

“FDAAA stipulates many new tasks and authorities for CDER,” said Woodcock.

Another common theme discussed by presenters who spoke later in the day at the conference was that, with the passage of FDAAA into law, the bifurcation of pre-approval and post-approval drug oversight would be eliminated. Woodcock described FDAAA as a “profound change” that would lead to “equal focus and accountability [being] placed on overseeing the safety of marketed drugs as on the evaluation of new drugs.”

Among its new authorities, CDER will be able to require a Risk Evaluation and Mitigation Strategy (REMS), which is a strategy to manage a known or potential serious risk associated with a drug or biological product. A REMS will be required if FDA finds that a REMS is necessary to ensure that the benefits of the drug or biological product outweigh the risks of the product, and FDA notifies the sponsor.

A REMS can include a Medication Guide, Patient Package Insert, a communication plan, elements to assure safe use, and an implementation system, and must include a timetable for assessment of the REMS. Some drug and biological products that previously were approved/licensed with risk minimization action plans (RiskMAPs) will now be deemed to have REMS...

At the end of Woodcock’s talk and others’, audience members from pharma and CROs asked what the difference between a REMS and a RiskMAP was and whether the REMS would replace the RiskMAP. These questioners were invariably referred to the agency’s lawyers. REMS relationship to RiskMAPs was not made clear at the conference.

A common theme throughout the presentations was the need for new technology to help the agency offer better pharmacovigilance.

CDER’s implementation strategy is called Safety First/Safe Use. It is the initiative under which the agency’s goals will be accomplished.

Woodcock discussed plans for the agency’s pharmacovigilance and active surveillance, acknowledging that it had to build new ways to do post-marketing surveillance instead of the Medwatch program, which involves voluntary reporting of spontaneous adverse events from marketed drugs by physicians. The goals for the agency’s new system are to include 25 million patients by July 1, 2010 and 100 million patients by July 1, 2012.

“It’s no problem to set up the system, analyzing data is the problem,” said Woodcock. But she did also express budgetary concerns.

Under the new law, FDA must identify trends and patterns and provide reports to Congress. It must ensure timely, routine and systematic analysis and reporting.

“The situation is more urgent than the timeframes recommended by Congress…Four years after enactment, we hope we can give a dynamite report,” said Woodcock.

But she emphasized the need for better analytic tools and that posting information on the web was not as simple as it sounded. Woodcock was pleased with the fact that Congress allowed CDER to form a Risk Communication Advisory Committee, considering the fact that there are limitations on the number of advisory committees the agency is allowed.

Woodcock cited the “new realities in the 21st century,” alluding to the fact that two decades ago there was a lack of effective treatments for most life-threatening illnesses and today there are more treatments but that a lot of the harm done out in the healthcare system with drugs is through inappropriate and unsafe use. “Congress didn’t pay attention because we didn’t have the tools,” she said.

The Medwatch program has clearly been inadequate, but perhaps the best the agency could offer at the time, given its lack of technological tools, and its budget and staff.

Woodcock then described a new initiative that is being introduced at the agency, the “Sentinel Concept,” whose purpose is “to ensure drug safety throughout the drug lifecycle by giving equal focus to oversight of drug development and post-marketing safety.” The FDA has made no public announcement to the general public yet about this new concept but will be soon.

The Sentinel Concept will use transactional data such as health claims and electronic health records. The agency plans to partner with data owners to figure out how they can share the information and form a distributed network. “We’ve had tremendous enthusiasm and encouragement from the data owners. There are a tremendous number of pilot projects,” she said.

These pilot projects are being sponsored by private parties that are doing research about how best to set up the system as well as evaluating the feasibility and robustness of analyses.

But there are many issues to be ironed out relating to governance, communication, privacy, access to data and healthcare provider burden that CDER will be addressing over the next six to nine months. Woodcock also expressed the need for the “right mix of staff with appropriate skills” and the need to “work in partnership with the healthcare system.”

But Woodcock stressed that the timing was right. “We couldn’t have done this before because we didn’t have the tools.” She expressed the desire to work in partnership with the healthcare system and remove the tension that has existed between them.

“What we’re dealing with here is uncertainty, uncertainty, uncertainty … We know whatever decision we make, we will be criticized,” concluded Woodcock.