User Fee Agreements

FasterCures, a center of the Milken Institute, is a non-profit, non-partisan action tank driven by a singular goal: to save lives by speeding up and improving the medical research system. Since its inception in 2003, FasterCures has been working to integrate patients as partners in biomedical research and development.

Therefore, FasterCures was an active participant in the stakeholder meetings conducted as part of the Prescription Drug User Fee Act (PDUFA) VI and Medical Device User Fee Amendments (MDUFA) IV renegotiation process between the U.S. Food and Drug Administration (FDA) and industry representatives.

Listed below are some of the key provisions in the PDUFA VI and MDUFA IV commitment letters that we believe are critical to helping FDA advance its efforts to deepen and expand engagement with the patient community.

PDUFA VI

Section I.J.1: ‘Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making’
The Patient-Focused Drug Development initiative that FDA launched under PDUFA V has helped give more prominence to the patient voice in the drug development process. However, more work is needed to advance the science of patient input, and we believe the steps outlined in this section of the commitment letter are critical to moving this science forward and integrating the patient voice more effectively.

Section III: ‘Improving FDA Hiring and Retention of Review Staff’
To carry out the ambitious goals articulated in the PDUFA VI commitment letter, FDA must be able to hire and retain staff with appropriate skills and expertise. FasterCures has long advocated for the adoption of policies that will strengthen the agency’s workforce, and we believe the enhancements identified in this section will help achieve more sustainable and long-lasting improvements to FDA’s workforce.

Section I.J.2: ‘Enhancing Benefit-Risk Assessment in Regulatory Decision-Making’
Patient perspectives are critical to making informed benefit-risk assessments, and the proposed enhancements and guidance documents will further the effective integration of those perspectives.

Section I.I.6: Enhancing Use of Real World Evidence for Use in Regulatory Decision-Making
Real-world evidence is an area with huge potential to enhance the patient experience, and we are pleased to see a commitment to better understand the opportunities and challenges of this evidence in PDUFA VI. We commend FDA for committing to hold at least one public workshop bringing together patients and other key stakeholders for input on how to best move forward.

MDUFA IV

Section IV.F: ‘Patient Engagement & the Science of Patient Input’
We support FDA in its commitment in the MDUFA IV goals letter to “advance patient input and involvement in the regulatory process.” We believe that the proposed activities outlined in this section will help all stakeholders be better positioned to effectively integrate patient perspectives, leading to more efficient and effective development of medical devices that address the needs of patients.

Section III.B: ‘Scientific and Regulatory Review Capacity’
To ensure that patient input continues to be meaningfully integrated into the regulatory process, it is critical that FDA has qualified staff to execute the underlying work. While we believe there is more that can be done outside of the MDUFA process to strengthen the agency’s workforce, we are grateful to see this issue addressed so explicitly in the performance goals letter.

Section IV.H: ‘Real World Evidence’
FDA continues to explore how real-world evidence can be more effectively used in regulatory decision-making for medical devices, and we support the use of user fee revenue to support the National Evaluation System for health Technology (NEST). This national system for evaluating medical devices is intended to generate evidence across the total product lifecycle of medical devices, leading to improved quality of real-world evidence that health-care providers and patients can use to make better informed treatment decisions.

History of User Fee Agreements

User fee agreements are carefully negotiated between FDA and industry, outlining expectations and commitments on both sides to facilitate efficient review of certain drugs and medical devices.

Each user fee agreement must be renegotiated and reauthorized every five years. PDUFA, the first user fee agreement, has been successfully renegotiated and reauthorized each cycle since it was signed into law in 1992.

PDUFA I (1992)
  • Original legislation permitting the FDA to charge a fee to pharmaceutical companies for the review of their products
  • Fees supplemented Congressional appropriations with the goal of accelerating the review process and allowing new cures to reach the market more quickly
PDUFA II (1997)
  • Enacted stricter performance expectations for FDA to make timely and efficient reviews
  • Introduced the investigational phase of review as under the scope of the law
PDUFA III (2002)
  • Began FDA post-market review
  • Funding used for new hires of skilled employees
PDUFA IV (2007)
  • Increased fees
  • Improved post-market review
PDUFA V (2012)
  • Institutionalized mechanism for feedback from the patient community through tools such as benefit/risk analysis, patient-reported outcomes and the Patient-Focused Drug Development program

New user fee agreements covering different medical products have been added since 1992, with each agreement following the same five-year renegotiation cycle. The existing agreements will sunset on Sept. 30, 2017.

Enacting Legislation Acronym Years Enacted Current legislative mandate ends
Prescription Drug User Fee Act PDUFA 1992, 1995, 2002, 2007, 2012 Sept. 30, 2017
Medical Device User Fee Amendments MDUFA 2002, 2007, 2012 Sept. 30, 2017
Generic Drug User Fee Amendments GDUFA 2012 Sept. 30, 2017
Biosimilar User Fee Act BSUFA 2012 Sept. 30, 2017

The reauthorization of these agreements is vital to support a robust, fully functional FDA that is well-positioned to support the advancement of biomedical innovation.

FasterCures’ Public Comments on User Fee Agreements

FasterCures has been an active participant in the stakeholder meetings conducted as part of user fee agreements renegotiation process.