Reauthorization of FDA Legislation Puts Focus on Enhancements that Benefit Patients

The Parkinson’s community has reason to celebrate today.  Years of advocacy have culminated in the passage of significant Food and Drug Administration (FDA) legislation.  The Food and Drug Administration Safety and Innovation Act, which reauthorizes so-called user fees that partially fund the FDA, contains an unprecedented number of patient-centric provisions.

Included in the final legislation, which we expect to be signed by the president shortly, are portions of the PAN-endorsed TREAT, FAST, and EXPERRT Acts, geared to expediting drug approval and delivery to market, enhancing opportunities for patient engagement and involvement, and maximizing the FDA’s access to scientific and medical experts.  For a more in-depth discussion of patient-centric provisions and their anticipated impact on the Parkinson’s community, please click here.

Background
Every five years, Congress must reauthorize legislation – the Medical Device User Fee Act (MDUFA) and the Prescription Drug User Fee Act (PDUFA) – enabling the FDA to collect fees from the pharmaceutical and medical device industries that partially fund the agency’s efforts to approve new drugs and therapies.  The legislation incorporates, by reference, agreements negotiated between the FDA and medical device and pharmaceutical industries.  During the reauthorization process, Congress often simultaneously considers and approves related legislation which has significance to the Parkinson’s community.

Impact of the Parkinson’s Community’s Voice
Recognizing the urgent need for breakthrough therapies for Parkinson’s disease, PAN staff has met regularly with FDA officials to provide high-level input and guidance on the importance of increasing patient engagement and input, enhancing the FDA’s review process, and increasing the agency’s access to scientific and medical experts.  Last fall, PAN delivered remarks on these topics at a public meeting before FDA officials and hundreds of patient and consumer advocates.

PAN staff also worked closely with the Biotechnology Industry Organization (BIO) on legislative recommendations for broad, sweeping systemic changes at the FDA.  Congressional allies sought PAN’s input on policy proposals throughout 2011, which ultimately resulted in the introduction, separate from the user fee legislation, of FDA-related bills.  Parkinson’s advocates around the country urged support for these bills.

Summary of Patient-Centric Provisions
What follows is a summary of provisions that we anticipate will impact and benefit the Parkinson’s community. For a more in-depth summary of these provisions, please click here.

Expediting Drug and Device Approval and Availability
The legislation includes portions of the PAN-endorsed Faster Access to Specialized Treatments (FAST) and Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Acts, which encourage the use of modern scientific tools, such as a broadened range of surrogate or clinical endpoints in drug development and review.

Enhancing the FDA’s Access to External Experts
Also included are provisions of the PAN-endorsed Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act, and revisions to the agency’s restrictive conflict-of-interest policy, to facilitate collaborations between FDA reviewers and external scientific and medical experts.

Establishing a Benefit-Risk Framework that Reflects Patient Input
The FDA must use a consistent approach to incorporating benefit-risk into decision making and communicating benefits and risks of new drugs to patients.  The agency will establish a five-year plan to implement and evaluate a structured benefit-risk assessment.

Increased Solicitation and Consideration of Patient Input
Over the next five years, the FDA will convene 20 disease-specific meetings between FDA officials, patient advocates, and other stakeholders and determine how input received will impact decision making.  The FDA will also increase use of patient representative consultants and better incorporate patient views into regulatory discussions.

Enhancing the Clinical Trial Process to Include and Incorporate Patient Input
The FDA must publish safety and effectiveness data provided by sponsors in applications, broken down by subgroups including sex, age, and ethnicity, and will promote best practices for the review and qualification of patient-reported outcome assessment tools.

Promoting Innovation through Enhanced Communication between FDA and Industry during Drug Development
The FDA will hire staff to liaise with sponsors and establish staff trainings to improve communication and, by 2015, will issue guidance describing best practices for communication between the agency and sponsors during drug development.

Promoting the Use of Telemedicine and Electronic Health Resources to Treat and Monitor Diseases
The Department of Health and Human Services must publish a report on an “appropriate, risk-based regulatory framework pertaining to health information technology, including mobile medical applications, that promotes innovation, protects patient safety.”  Because this provision is written in such a way that its significance is not entirely clear, PAN will be following up with the FDA for clarification.

PAN applauds the swift passage of this important legislation and the inclusion of an unprecedented number of measures designed to benefit individuals with Parkinson’s and other chronic, progressive diseases.