Every five years, Congress must reauthorize legislation – the Prescription Drug User Fee Act (PDUFA) and the Medical Device User Fee Act (MDUFA) – 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. PAN monitors FDA related legislation and was active in an effort to reauthorize the Food and Drug Administration Safety and Innovation Act (FDASIA) (PL 112-44). In July 2012, FDASIA was signed into law. The act reauthorizes the user fees that partially fund the FDA and contains an unprecedented number of patient-centric provisions (summary below).
Prior to the introduction of legislation, 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 many organizations to promote additional legislative recommendations for broad, sweeping systemic changes at the FDA. Congressional allies sought PAN’s input on policy proposals, some of which were included in the final version of FDASIA.
Summary of Patient-Centric Provisions
Below is a summary of provisions that PAN anticipates will impact and benefit the Parkinson’s community:
Establishing a Benefit-Risk Framework that Reflects Patient Input
- Requires the FDA to use a consistent approach to incorporating benefit-risk (the comparison of any inherent health or potential safety risks with the broader benefits of a particular drug or therapy) into decision making and communicating benefits and risks of new drugs to patients.
- Commits FDA 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.
- FDA 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.
- Parkinson’s has been chosen as one of the 20 disease-specific meetings. While our meeting date has not yet been scheduled, PAN is working closely with the FDA to set meeting objectives and outputs.
- Requires 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.
- The FDA has committed to improve the use of patient-reported outcomes (PROs) by committing to promote best practices for the review and qualification of patient-reported outcome (PRO) assessment tools. PROs are questionnaires used in a clinical trial or a clinical setting, where the responses are collected directly from the patient. A PRO questionnaire will most likely assess one of the following issues: symptoms, disability, health status, quality of life, and reports and ratings of health care.
Effectively measuring PROs is critical to understanding the benefits and risks from an individual patient’s perspective.
Expediting Drug and Device Approval and Availability
- Enhances the FDA’s authority to consider appropriate scientific data, methods, and tools, to reduce duration of clinical trials
- Requires the FDA by July 2014 to issue final guidance regarding implementation of the revised accelerated approval process, which allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need.
These provisions are especially important to the Parkinson’s 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. We welcome opportunities to streamline the clinical process, and PAN remains engaged with the FDA on this issue.
Enhancing the FDA’s Access to External Experts
- Includes a provision designed to encourage the FDA to collaborate with external scientific and medical experts in rare diseases and subtypes of diseases when considering a new drug.
Promoting Innovation through Enhanced Communication between FDA and Industry during Drug Development
- Directs agency to hire new staff to liaise with pharmaceutical companies 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.