The Parkinson’s community has reason to celebrate. 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.
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 the medical device and pharmaceutical industries. During the reauthorization process, Congress often simultaneously considers and approves related legislation with 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 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. 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 of legislation, related to but distinct from the user fee reauthorization, for which Parkinson’s advocates around the country urged support.
Summary of Patient-Centric Provisions
What follows is a summary of provisions that we anticipate will impact and benefit the Parkinson’s community.
Expediting Drug and Device Approval and Availability
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 in drug development and review, are included in the reauthorizing legislation.
Specifically, the legislation amends current law to enhance the FDA’s authority to consider appropriate scientific data, methods, and tools, such as a broadened range of surrogate or clinical endpoints. The hope is that this will result in fewer, smaller, or shorter clinical trials, thus accelerating the approval of drugs for serious or life-threatening diseases.
Additionally, the legislation requires the FDA, within two years from enactment, to issue final guidance regarding implementation of the revised accelerated approval process. The bill also calls for an independent analysis of accelerated approval and its impact on the development of innovative treatments.
These provisions are especially important to our community, as central nervous system drugs, including therapies for Parkinson’s, are slow to move through the drug development pipeline – taking an average of 15-20 years to get to market.
Enhancing the FDA’s Access to External Experts
The legislation includes provisions of the PAN-endorsed Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT ) Act, designed to encourage FDA reviewers to collaborate with external scientific and medical experts in rare diseases and subtypes of diseases. The bill also revises the FDA’s restrictive conflicts-of-interest policy to make it easier for the FDA to access expert advice.
Establishing a Benefit-Risk Framework that Reflects Patient Input
Despite the fact that no drug or therapy has the same effect on everyone and diseases like Parkinson’s affect each individual differently, clinical trial sponsors and FDA staff routinely make generalized decisions around the highly personal and complex issue of risk tolerance. PAN has advocated  the FDA address this by adopting a benefit-risk framework that reflects risk-tolerance differences between disease populations and subtypes.
The legislation requires the FDA to use a consistent approach to incorporating benefit-risk into decision making and communicating benefits and risks of new drugs to patients. Moreover, the FDA commits to create a five-year plan to further develop and implement a structured benefit-risk assessment process. The FDA is required to evaluate this framework and its impact.
Increased Solicitation and Consideration of Patient Input
The FDA also commits to initiate a public process to identify a set of disease areas that could benefit from a more systemic and expansive approach to gathering and evaluating patient input on disease severity and unmet medical needs.
Over the next five years, the FDA will convene a total of 20 disease-specific meetings between FDA officials, the relevant patient advocacy community, and other interested stakeholders. While how and which diseases will be selected for focus is not specified, the agency does commit to develop a proposal for how input shared at the meetings will be incorporated into its decision making.
The agency also commits to increase its utilization of patient representatives as consultants early in the drug review process, and to ensure patient views are considered in regulatory discussions.
Enhancing the Clinical Trial Process to Include and Incorporate Patient Input
Clinical trials are an integral part in the process of bringing much-needed new treatments and cures to patients. From ensuring increased trial participation, to requiring better outcome reporting mechanisms, PAN has weighed in on virtually all aspects of the clinical trial process, most recently in recommendations submitted to the FDA  with the other national Parkinson’s organizations.
The bill requires the FDA to publish, online, information regarding the extent to which sponsors include in their applications safety and effectiveness data, broken down by subgroups including sex, age, and ethnicity. This will provide an added level of transparency in the clinical trial process.
Additionally, the FDA aspires to improve the use of patient-reported outcomes (PROs) by committing to promote best practices for the review and qualification of PRO assessment tools. Effectively measuring PROs is critical to understanding the benefits and risks from an individual patient’s perspective.
Promoting Innovation through Enhanced Communication between FDA and Industry during Drug Development
The agency will hire new staff to liaise with sponsors and establish staff trainings to improve communications with sponsors. By 2015, the agency will issue guidance describing best practices for communication between the FDA and sponsors during drug development. These provisions recognize that timely and interactive communication supports efficient and effective product development.
Promoting the Use of Telemedicine and Electronic Health Resources to Treat and Monitor Diseases
The legislation requires the Department of Health and Human Services to 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.
The bill also directs the FDA to issue guidelines regarding regulation of mobile medical applications. Electronic health resources, such as medical applications for mobile devices, have the potential to deliver crucial health information to patients (particularly those in rural or underserved areas) faster, and may allow for more timely and accurate monitoring of disease symptoms.
PAN applauds passage of this significant legislation and the unprecedented focus on and inclusion of measures to protect, engage, and hasten the delivery of treatments to individuals with Parkinson’s and other diseases. The FDA’s commitment to maintaining transparency and collaboration with the patient community in future reauthorizations is encouraging. PAN is monitoring implementation.