Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at email@example.com if you need assistance.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
Our new book is a comprehensive look at a vital part of medicines development and regulatory affairs. Grab your copy today!
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
President Donald Trump late Friday signed a bill that reauthorizes the US Food and Drug Administration (FDA) user fee programs for prescription drugs, generic drugs, biosimilars and medical devices through 2022.
Taking effect 1 October, the 86-page law (the text of which can be found here) passed the House via voice vote in July and by a vote of 94-1 in the Senate earlier this month without the addition of any controversial amendments. The law ensures the agency will not have to lay off thousands of employees and it reauthorizes FDA's ability to collect set amounts of money that the biopharmaceutical and medical device industries will pay each of the next five years for consistent reviews of medical products based on timelines agreed to by industry and FDA.
Peter Marks, director of FDA's Center for BiologicsEvaluation and Research, Jeffrey Shuren, director of FDA's Center for Devices and Radiological Health, and Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, discussed some of the major provisions of the new law here.
In general, the fee amounts for each of the programs have increased or been adjusted from the last set of user fee agreements in ways that will speed medical product approvals and allow FDA to hire more employees to conduct reviews.
Outlined below are the major provisions in the law's text and the new performance goals and procedures agreements forged between industry and FDA over the last two years.
The law is divided into nine titles: Fees relating to drugs (Title I), fees relating to devices (Title II), fees relating to generic drugs (Title III), fees relating to biosimilar biological products (Title IV), pediatric drugs and devices (Title V), reauthorizations and improvements related to drugs (Title VI), device inspection and regulatory improvements (Title VII), improving generic drug access (Title VIII) and additional provisions (Title IX).
The first four titles explain the fee amounts and reporting requirements for each of the respective agreements (for more details see the section below on the performance goals), known as the Prescription Drug User Fee Act (PDUFA VI), the Generic Drug User Fee Act (GDUFA II), the Biosimilar User Fee Act (BsUFA II) and the Medical Device User Fee Amendments (MDUFA IV).
Title V focuses on improving access to drugs and devices for pediatric populations, including pediatric cancer treatments (see the Focus article on the RACE for Children Act here for more).
Among other provisions, this section of the law requires FDA, within the next year, to establish, update regularly and post online a list of molecular targets considered to be relevant to the growth and progression of pediatric cancers and a list of molecular targets of new cancer drugs and biologics in development for which pediatric cancer study requirements under this section will be automatically waived.
FDA in the next year will also hold a public meeting on the development, approval or clearance and labeling of pediatric medical devices, and another meeting on overcoming the challenges linked to developing pediatric cancer treatments. And in the next two years, FDA is required to issue final guidance on such cancer drugs for pediatric populations.
Title VI includes slight tweaks and reauthorizations of various laws and programs run by FDA, including the orphan grants program and orphan drugs (with a focus on clinical superiority), pediatric drug labels, expanded access and applications submitted for tropical disease treatments (with changes related to what constitutes such an application).
FDA guidances that must be issued under Title VI include one regarding eligibility criteria for clinical trials and one to streamline the institutional review board review of individual patient expanded access protocols.
Title VII establishes a new risk-based schedule for FDA to inspect medical device facilities (which the Project on Government Oversight has taken issue with), a new risk-based classification system for device accessories, a provision to ease the regulation of over-the-counter hearing aids, provisions related to diagnostic imaging devices used with contrast agents, a new device postmarket pilot project and a new report FDA will issue on the servicing of devices.
Title VIII includes provisions related to speeding the review of certain generic drugs and enhancing transparency on generic competition, though FDA in June already addressed some of these provisions by offering a list of generics with limited or no competition and prioritizing the review of generics for which there are fewer than three applications already approved for the reference listed drug.
And Title IX includes technical corrections to the 21st Century Cures Act, performance reporting requirements, use of funding analyses and FDA facility management analyses.
For a more nuanced look at what each of the user fee agreements will entail, the performance goals letters explain the details:
First created by Congress in 1992, this sixth iteration of the largest of the user fee agreements, supported by industry groups PhRMA and BIO, focuses on pre-market reviews, postmarket safety, regulatory decision tools and other ways FDA is preparing for the future of drug development.
In addition, the agreement seeks to further incorporate the patient voice into drug development, enhance the use of real-world evidence, allow early consultations on new surrogate endpoints, establish a qualification pathway for biomarkers, improve benefit-risk assessments and expand postmarket safety data and evaluations.
PDUFA VI also changes the user fee structure. Previously,
one-third of user fees were collected from
establishment fees, one-third from various application fees and one-third from
product fees. The new structure will collect 20% from application fees
and 80% from fees for approved products. Fees for supplemental
applications and establishments were eliminated.
This latest iteration of the device industry's agreement with FDA will increase fees for device manufacturers in exchange for reduced timelines to decisions for PMAs and 510(k) submissions.
The agreement will also help FDA hire new quality management staff, set new goals for de novo submissions, advance patient input and involvement in the regulatory process, improve the third party device review program with a goal of eliminating routine re-review by FDA, support the National Evaluation System for health Technology (NEST) (with a goal of creating a viable system by 2019), streamline and align FDA review processes for Software as a Medical Device (SaMD) and software inside of medical devices (SiMD), which FDA has already begun with its new digital health innovation plan, establish an Accreditation Scheme for Conformity Assessment (ASCA) program using FDA-recognized consensus standards and report FDA's progress toward meeting these goals.
This first reauthorization of the generic drug agreement creates a new user fee structure and aims to help small businesses, in addition to speeding the review of generic drugs (with 8-month priority reviews) and increasing interactions between the agency and companies working on complex generics.
The new fee structure (see more on the difference between the GDUFA I and II fee structures here) was established because over the first four years of the first GDUFA, FDA received about 1000 new generic drug applications per year, which was about 250 more than what the agency had thought it would receive. To address the increased workload, FDA had to hire additional staff and is projected to spend about $430 million in 2017, the final year of GDUFA I. Under the new GDUFA, industry and FDA agreed that user fees should total $493.6 million annually (adjusted each year for inflation).
The second agreement between biosimilar manufacturers and FDA will allow for additional communications between the agency review teams and biosimilar applicants while also adding days to the review timeframe to accommodate for the new interactions. In addition, the next BsUFA will add new guidance that FDA must issue and allow, in certain circumstances, sponsors to request a written response to questions rather than conduct a face-to-face or teleconference meeting.
PDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2018 THROUGH 2022
MDUFA PERFORMANCE GOALS AND PROCEDURES, FISCAL YEARS 2018 THROUGH 2022
BIOSIMILAR BIOLOGICAL PRODUCT AUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FOR FISCAL YEARS 2018 THROUGH 2022
GDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROGRAM ENHANCEMENTS FISCAL YEARS 2018-2022
Tags: FDARA, FDA user fees, user fee reauthorization, medical product reviews