Keywords: Real World Evidence, RWE, Real World Data, post-market, value assessment, value-based contracting
Speaker Submission from Thomas Hubbard, Vice President of Policy Research, NEHI-Network for Excellence in Health Innovation (Cambridge MA and Washington DC) --- "Real World Evidence for Informed Decision-Making: Trends Shaping the Development and Use of RWE in the Pre-Approval and Post-Approval Periods" Real World Evidence seems likely to play an increasing role in health care decision-making in both the pre-FDA approval period and the post-launch, post-market period. RWE can play an important role in complementing data from conventional drug approval trials, while giving payers an earlier, sharper perspective on the best use of new products, a perspective payers increasingly demand as they evaluate and contract purchase of new products. Insights from RWE may be crucial to utilization of new products that meet the tests of health care quality and cost control demanded from new provider payment models. Meanwhile providers and patient advocates look to RWE for insight on directing the right patients to the right care at the right time.
Yet use of RWE remains dependent on the development of shared norms and expectations for appropriate curation of Real World Data, and appropriate "fit for purpose" use of Real World Evidence.
My remarks will present an overview of the multiple trends that are now shaping policy on development and use of RWE in both the public and private sectors. Key trends include: the FDA's increasing interest in novel clinical trial design and the mandates of the 21st Century Cures Act; growing payer utilization of health technology assessment and so-called value frameworks to assess clinical trial data and cost data on use of new products; the role of major health data initiatives such as PCORnet, FDA Sentinel, and other programs; and the impact of "value-based" approaches to both provider payment and medical product reimbursement on the future design and conduct of clinical research. My remarks are based on a multi-year project at our organization that has brought RWE thought leaders from multiple sectors to consider new policy to promote good use of RWE. My objective will be to provide a policy context for the more technical presentations that will be made as part of this parallel session of the conference.