Real-World Evidence Generation: Overcoming Concerns

Real-World Evidence (RWE) is a term that’s been around for many years, but as our team was reminded at ISPOR, many folks are interested to see where it’s headed next.

The landscape of evidence development, long rooted in the Randomized Clinical Trial (RCT), is at the beginning of what will likely be a major shift in how and by whom medical evidence is developed for the next generations.

Concerns over the limitations in generalizability of results from RCTs are not new. However, the increasing availability of electronic healthcare data and rapidly improving methods on how to repurpose these data for evidence development have led researchers, payors, regulators, healthcare providers, life sciences companies, and contract research organizations to begin to shift their strategy and policies to accommodate these new designs.

This shifting landscape has not been lost on lawmakers. The 21st Century Cures Act that passed the House in 2015 contained references to RWE. This year, the bipartisan Senate Health, Education, Labor, and Pensions (HELP) Committee, primarily focused on supporting and advancing the pace of medical research, innovation, development, and delivery of treatments and cures in America, is working through its own version of 21st Century Cures.

Though many groups, including the Bipartisan Policy Center (see Advancing Medical Innovation for a Healthier America) are strongly encouraging its inclusion, it is unclear whether the Senate will include RWE in its version of the Bill. In review and discussion of numerous potential policies regarding the use of RWE in the context of pre, post, and even new approval models, senators have expressed most uncertainty about the latter. More specifically, there exist some fundamental concerns over a product life-cycle approach that must be addressed.

The following are concerns of some within the Senate:

  • Lowering the “gold standard”
  • Increasing risk to patients
  • In general, the inability to properly ascertain risk/benefit

In the blogs to follow, I will speak to each of these concerns and offer differing perspectives and potential solutions for and benefits of advancing the generation and use of RWE.

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