By Alethea Wieland, founder and president, Clinical Research Strategies, LLC
This is the first article in a two-part series addressing real-world evidence (RWE) for life sciences leaders who may be struggling to make sense of the rules.
Part one of this series is intended to give readers a glimpse into what RWE is today, with its potential to utilize staggering volumes of data, digital health technologies,1 and mobile applications. Part two will further describe the challenges and opportunities for life sciences companies, highlight recent examples of RWE, and discuss how this newer way of thinking will transform the way we design and conduct clinical research or evaluate value-based decisions for the better. However, this Cliff Notes-style version is no substitute for reading and becoming better versed in RWE methods and regulatory trends, and more importantly, the work that lies ahead for all of us. There will be, for certain, job security in policy and law, technology, regulatory affairs and strategy, quality assurance, market access, managed care, and data science careers for the long haul.
Over the past few years, it has been difficult to ignore the numerous articles published in medical and science journals and life sciences media channels and the position statements and guidance documents on the FDA website about the new world order in clinical research and value-based payment decision-making: real-world evidence. For those of us in healthcare or clinical research professions, we have seen this coming, due to the rising costs of our healthcare and the countless inefficiencies we encounter in the work we do, including in conformance with process norms, complex regulations, and standards groups in multiple, decoupled federal agencies. Several consolidations of large contract research organizations (CROs) in the past five years were also obvious signs.
If your head has been spinning, trying to establish where we are with the U.S. regulatory framework, development, implementation, and acceptance of RWE programs, you are not alone. You have entered the discussion at a good time, without missing too much in the background. The “reset” button was pressed emphatically a few years ago, including with the passage of the 21st Century Cures Act in late 2016.2
Where Are We Now?
Real-world data (RWD) is data relating to patient health status or the delivery of healthcare routinely collected from a variety of sources (e.g., electronic health records or EHRs, claims and billing data, registries, and digital health apps on mobile devices). Technology has enabled the collection of RWD at an unbelievable rate. RWE, on the other hand, is the clinical evidence regarding usage and potential benefits and risks of a medical product resulting from analysis of RWD that answers specific questions.3, 4, 5
In the wake of the well-documented reproducibility problems in science,6 taxpayer waste and diminished trust in our grants system,7 epic trial failures,8, 9 renewed calls for an end of the use of P values to claim statistical significance,10, 11 and astronomical costs and time to bring a drug to market,12, 13 it seems reasonable that our salvation will come from radical change.
Now imagine for a moment you are watching Dorothy in the Wizard of Oz, when she wakes up after the tornado hits her Kansas prairie family farmhouse depicted in the simplicity of black and white cinema and then we switch to the spectacle of technicolor. Except, you realize that you are taking a tour on the set, standing next to Judy Garland. She is in a rehearsal, still memorizing her lines and going over walkthroughs with a cast of hundreds, a seamstress is adjusting her blue and white gingham dress on a mannequin, the ruby red shoes are ordered but have not yet arrived, the producers are complaining about the budget, the director is nowhere to be found, agents are demanding special treatment for their movie stars, the script writers are handwriting revisions, timelines for editing and distribution are slipping, inspectors are looking over the studio’s scaffolding for hazards, and the construction crew is building the yellow brick road that leads to the Emerald City.
This is essentially where we are with RWE: a state of tactical development with more stakeholders than one could have ever imagined, after the decision for implementation has been legislated and our attention is transfixed by the dramatically new, never going back reality, and the saturation of noise and action. It is cautiously optimistic to think we have advanced beyond crawling to the standing stage, as eloquently supported in the aptly-timed August 2019 Bipartisan Policy Center (BPC) Report, Expanding the Use of Real-World Evidence in Regulatory and Value-Based Payment Decision-Making for Drugs and Biologics, under the leadership of former Senator William Frist, M.D., and three former FDA commissioners, Robert Califf, M.D., Ph.D., Andrew von Eschenbach, M.D., and Mark McClellan, M.D., Ph.D.14
The BPC report makes recommendations on “clearing barriers to the access and use of real-world data to provide an evidence base for regulatory evaluation and value-based payment programs, expanding opportunities to use new data sources and approaches, and advancing new models of collaboration among payers, manufacturers, regulators, clinicians, and –most importantly– patients.”14
A summary of the 12 key policy recommendations includes:
I will let you catch your breath in anticipation of the follow-up article, which will explore challenges, opportunities, recent examples of RWE, and the involvement of multiple stakeholders to propel us forward.
About The Author:
Alethea Wieland is founder and president of Clinical Research Strategies, LLC, an executive-level management consulting firm and boutique, functional service provider for the life sciences industry. Her firm’s solutions include analyzing the intersection of healthcare innovation and policy; providing flexible clinical trial resourcing; developing sustainable corporate affairs programs and policies; training and managing resilient, high-performing clinical operations teams; mitigating risks of clinical trials; and facilitating transparent, accountable sponsor-CRO partnerships. Learn more by connecting with her on LinkedIn and by visiting her website.