User Fees: Ensuring a Stronger and Better FDA

By: Margaret Hamburg, M.D.

FDA receives thousands of applications for potentially promising medical products every year. Reviewing these often scientifically-complex submissions is the responsibility of a large team of doctors, chemists, bioengineers, statisticians and other experts who must determine whether a proposed new product is safe and effective for patients, and do so within a certain time period.

Margaret Hamburg, M.D.It takes steady and reliable funding to maintain and support a staff of trained reviewers capable of accomplishing this vital task. We’re gratified that Congress agrees, as demonstrated by today’s passage of the Food and Drug Administration Safety and Innovation Act in the US Senate by a vote of 92-4. Since the House of Representatives passed the bill last week by a voice vote, the bill now heads to the President’s desk, where it is expected to be signed into law. This legislation, when enacted, will authorize the FDA to collect user fees from industry to fund the review of innovator drugs, medical devices, generic drugs and biosimilar biologics.

Such overwhelming support for FDA user fees is a testament to the important role FDA plays in America’s healthcare continuum. FDA’s medical product decisions sit at the intersection of public health, innovation, and commerce and touch the lives of nearly every American every day.

Today’s positive vote also reflects the success of FDA’s commitment to transparency and collaboration in developing user fee proposals that all sides could support. FDA negotiators spent months in discussions with industry and consulted closely with patients, consumers and health care providers before arriving at proposals for each of the four programs.

Driving these discussions is the recognition that user fees have been a big success, reversing what was once a lag in the time needed for drug approvals. Since the prescription drug user fee legislation (PDUFA) was enacted in 1992, time to market for priority drugs has decreased from an average of 2 years to 1.1 years recently. This has provided patients faster access to over 1,500 new drugs and biologics, including treatments for cancer, infectious diseases, neurological and psychiatric disorders and cardiovascular diseases.

Under PDUFA V, fees paid by industry will support continued timely review of new prescription drugs, increase the use of standardized electronic data in product submissions, enhance communications with companies during drug development and implement a structured benefit-risk framework in drug review.  PDUFA V also puts more focus on regulatory science, which seeks to create new tools, standards and approaches for use in assessing the safety, effectiveness, quality and performance of products. Among other things, user fees will advance the development of drugs for rare diseases and encourage the development of biomarkers.

Patient groups in particular are heralding PDUFA V as a turning point because it acknowledges that patients who live with a disease have a direct stake in the outcomes of the drug review process and are in a unique position to contribute to the entire drug development enterprise, including FDA review and decision-making. Thus, PDUFA V will support the advancement of the use of patient-reported outcomes and other tools to assess clinical trial endpoints that can improve quality and reduce risks in drug development. It will also engage patients to obtain their perspective about disease severity and unmet medical needs in a therapeutic area. FDA will use this perspective to inform the context in which the agency weighs drug benefits and risks.

The legislation also reauthorizes the user fee program for medical devices for the third time. Under MDUFA III, user fees will nearly double, rising from 20 percent of the total of FDA’s review activity to 35 percent. The agreement facilitates more timely access to safe and effective devices with the shared goal of reducing average total time to decisions and achieving greater transparency, consistency, predictability and productivity. With the additional funding, the FDA will be able to hire over 200 full-time equivalent workers by fiscal year 2017.

Two other user fee programs in the legislation are new. User fees for generic drugs or GDUFA will address our current generic drug review backlog caused by the increase in generic drug applications, their growing complexity, and the number of generic drug facilities now located overseas where inspections are more challenging. The added money from user fees will reduce this backlog and eventually ensure that FDA is able to inspect overseas facilities as often as it does domestic facilities.

The second new user fee program, BsUFA, would collect fees for products under development shown to be “biosimilar to” or “interchangeable with” an innovator FDA-licensed biological product. The funds would support early meetings with companies.

The legislation also contains dozens of other provisions related to FDA.
Three provisions are worth highlighting: they provide FDA with new tools to better combat drug shortages, ensure the safety and security of the drug supply chain and encourage drug innovation.

It’s been a long, yet productive process since FDA first began meeting with industry and other public stakeholders on the reauthorization of user fees nearly two years ago. What has emerged, I believe, will truly result in a stronger and better FDA for everybody.

 Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration

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