How the 21st Century Cures Act and FDA Guidelines for Patient-Focused Drug Development Impact Real-World Research

Beginning in 2012 with the Food and Drug Administration Safety and Innovation Act (FDASIA) reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA has recognized two things: that patients are experts in their disease, and that endpoints, symptoms, and the impacts of disease that are important to patients do not always align with the clinical endpoints used to support medical product development. This initiative to capture the ‘patient voice’ was further reinforced as a priority for the FDA within Section 3002 of the 21st Century Cures Act, which aims to standardize the collection methods, types of data, and analysis of the patient experience.

As part of the 21st Century Cures Act, FDA is obligated to develop guidance(s) on the collection of patient experience data, and the use of such data and related information in drug development. This obligation includes defining the methodology for collection and measurement of patient experience data, how such data and related information may be incorporated during product development, and presentation of such data for consideration in regulatory decision making, including product submission for approval.

The FDA has stated their intent to satisfy this obligation through public workshops with all Patient-Focused Drug Development (PFDD) stakeholders (eg., academia, healthcare providers, communities, advocacy groups, and drug/device developers), which will culminate in the release of four guidance documents. The first of these guidance documents describing methodological considerations for data collection, reporting, management, and analysis of patient experience data was released in draft format in June 2018. Additionally, FDA is also scheduled to hold a public meeting in early October on methods to identify factors of importance to patients.

Within the context of real-world research and the impact of the 21st Century Cures Act on current and future design and implementation strategies, this webinar will explore:

• The history of the 21st Century Cures Act, Section 3002 on incorporating patient experience into drug development and FDA fulfilment of these obligations to-date
• Key concepts from the June 2018 draft FDA guidance on Patient-Focused Drug Development (PFDD)
• Preliminary themes from the October public meeting on PFDD methods

Speaker

Juliane Mills, MPH, MS, Real World Solutions - Scientific Affairs, PRA Health Sciences

Juliane Mills has a comprehensive understanding of drug development from the research laboratory to the patient’s bedside gained from over 20 years of experience in various scientific roles in CROs, academic institutions, and pharmaceutical companies. For the last 9 years, Ms. Mills has exclusively supported peri- and post-approval programs and real-world research. She has extensive experience in the operational strategy and scientific design of various study types such as disease and product registries, post-authorization safety studies (PASS), non-interventional studies (NIS), biomarker studies, and chart reviews. In her position prior to joining PRA, Ms. Mills worked extensively in risk management and post-marketing safety requirements, providing consulting services for the development of programs to meet FDA and EMEA mandates for post-approval risk assessment and risk minimization regulatory commitments, as well as presenting at regulatory meetings with the US FDA. She earned a master’s in both public health and science communication from Drexel University.

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