For the past 50 years, Randomized Clinical Trials (RCT’s) have been a staple of pre-approval research; often referred to as the “gold standard” for their ability to minimize bias in outcomes. They are designed to maximize internal validity – or our confidence that the product being studied caused the outcomes reported under the circumstances the product was evaluated. Unfortunately, in far too many cases, this internal validity comes at the sacrifice of our ability to generalize the results to a broader population often treated under very different circumstances to those studied. It is for this very reason that so many have begun to place an emphasis on real world research designs meant to supplement the findings from RCT’s when informing policy and now potentially regulatory decisions.
As the regulatory and policy frameworks across the U.S. begin to move toward the incorporation of Real-World Evidence (RWE), we are beginning to see a major shift in how and by whom medical evidence is developed for the next generation of products.
With this shift comes concerns by some within Congress that we may be lowering the gold standard by implementing RWE earlier in the drug development process. However, under certain circumstances, opening up the design options in the pre-approval phases of drug development to include Pragmatic Clinical Trials (PCT’s) has the potential to greatly enrich and complement the findings from traditional RCT’s and, earlier in the drug approval process, broaden the generation of evidence more relevant to treated patients.
Though the creation of the infrastructure to accomplish this requires strong collaboration across multiple stakeholders in the healthcare system, the “raw materials” for establishing a robust, prospective, real-world evidence development environment are in place. What is needed now is for the regulatory path to be clear for those desiring to put it in place. Regulators, payers, and providers alike also must be aligned in their demand for more relevant evidence upon which to base their decisions.
Though pure gold cannot corrode or tarnish, in this case, our current gold standard is not so pure. With the increasing availability of robust real-world data environments and improving analytic methodologies, we have the opportunity to greatly enrich our development process by combining the relative strengths of RCT’s and RWE, bringing us to a new, much purer gold standard.
Please join us on Thursday, June 23, 2016 from 9:00 – 11:00 a.m. for the Bipartisan Policy Center’s event on Real World Evidence for Safe and Effective Cures. This event will feature a panel discussion on the latest advancements in medical innovation legislation, as well as the release of new recommendations on the importance of using real world evidence to support finding safe and effective cures for Americans.