The U.S. FDA recently issued a statement introducing their new strategic framework to advance the use of real-world evidence (RWE) to support the development of drugs and biologics. The statement highlights how the framework will serve as a roadmap for more fully incorporated real-world data (RWD) and RWE into the regulatory paradigm. The framework, which outlines a number of important RWE-related efforts to explore its potential to support approval of new indications for approved drugs or to support or satisfy post-approval study requirements, will help the FDA and the industry evaluate the potential use of RWE to support regulatory decisions that have visible outcomes for patients. To learn more about this framework and how it will impact sponsors and the broader industry, PAREXEL spoke with Leanne Larson, Corporate Vice President and Worldwide Head, Real-World Evidence Strategy, PAREXEL.
PAREXEL: What does the framework for the FDA’s RWE program include?
Leanne Larson (LL): The 21st Century Cures Act released in 2016 included, among other key points, a mandate for the FDA to issue guidance around its use of RWE in regulatory decision-making, and this Framework represents the foundation, the initial step, in their critical thinking in this area. In this key document, the FDA describes some of the recent initiatives, workshops and decisions that have focused on or incorporated RWE in recent years, as well as the key considerations around RWE that they will be addressing going forward. The Framework offers an outline of their approach to evaluating these considerations, and the steps they will take to define what types of data, analyses and models they will be incorporating into their regulatory decisions in the future.
PAREXEL: Why is this this framework important for the industry?
LL: RWE is the fastest-growing segment of the clinical research industry, and global stakeholders throughout healthcare – including regulatory, payers, clinicians, and even patients – are increasingly looking to this evidence to support their treatment and healthcare decisions. That said, there is still some inconsistency around the most appropriate ways to design, conduct, and even analyze real-world studies. This Framework confirms the essential role that RWE will play in healthcare going forward, and – when FDA concludes the initiatives described within the Framework – will offer stakeholders throughout the industry and healthcare overall a critical foundation in the best-practice approaches to RWD and transforming those data into meaningful and valuable evidence.
PAREXEL: How will this framework impact sponsors?
LL: This Framework offers sponsors today insight into FDA’s current practices with regard to RWE, and confirms the important role that RWE is beginning, and will continue, to play in regulatory decision making going forward. This document confirms the need to incorporate RWE throughout the product development and commercialization lifecycle, and highlights both the definitions and structures of the key research and data models in this area, as well as the questions and challenges to consider in designing and conducting these important programs.
PAREXEL: How will this framework impact patients?
LL: While the Framework isn’t specifically focused on patient-centricity, the FDA’s commitment to including RWE in their decision processes ensures that the patient voice and care paths will be an increasingly central part of regulatory decision making. At its core, RWE reflects the true patient experience – how healthcare is delivered and experienced in actual practice; incorporating this into product-development and approval processes will allow the FDA and other stakeholder to inform their decisions with both the strongest scientific evidence as well as insights into actual practice, and patient healthcare behavior and preferences. This broad and inclusive research and regulatory model will help ensure that patients have access to products that best meet their medical needs while optimizing their healthcare experience.
PAREXEL: How is PAREXEL positioned to help sponsors leverage RWE?
LL: PAREXEL brings to sponsors industry-leading expertise and experience in RWE strategy, science, and operations. With the industry’s first dedicated late-phase team, PAREXEL Access has been designing and leading real-world research programs for over 16 years, and includes experts that have developed and shaped the standards and best practices that guide the industry today in this important field. In addition to our specific expertise in RWE models and practice, PAREXEL also seamlessly integrates our RWE approaches with our Regulatory, Market Access, Reimbursement, and Drug Safety expertise, ensuring that our RWE programs are informed by, and connected to, today’s stakeholder perspectives and requirements, driving product success throughout the product lifecycle.