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How do payers use patient reported outcomes?

This is the question asked by a study conducted by Brogen et al. (2017).  To see how payers use patient reported outcomes (PRO) in oncology, the authors conducted a literatures search, and searched the websites of the following health technology assessment bodies:

  • Pharmaceutical Benefits Advisory Committee (PBAC) in Australia,
  • Canadian Agency for Drugs and Technologies in Health (CADTH),
  • Haute Autorité de Santé (HAS) in France
  • Institute for Quality and Efficiency in Health Care (IQWiG) in Germany,
  • Scottish Medicines Consortium (SMC),
  • National Health Service Scotland (NHS Scotland),
  • National Institute for Health and Care Excellence (NICE) in the UK,
  • Drug Effectiveness Review Project in the United States,
  • Agency for Healthcare Research and Quality (AHRQ) in the United States.

The authors also interviewed key stakeholders through an online survey and a one-on-one survey.

The literature review found that:

Of the 324 HTAs reviewed, 91 (28%) reported PRO data. The use of PRO data increased over time, from 11.1% in 2005 to 42.5% in 2011…Use of PRO data varied by HTA body, from 9.8% in HAS HTAs to 66.7% in HTAs by IQWiG and NHS Scotland.

The authors claim that much of the growing interest in PROs was driven by FDA guidance on PROs published in 2009.  In fact, more clinical trials are explicitly incorporating PROs as trial endpoints.

How do payers consider PROs in market access decisions?

Payer participants interviewed generally indicated that they consider PRO evidence in their decisions but that such evidence is usually considered complementary to clinical and safety endpoints. PRO evidence appears to have greater influence outside the United States: When asked whether PRO data affect decision making when one therapy is being considered over another, 47% of U.S. payers and 78% of ex-U.S. payers in the web-based survey indicated that PRO data have an impact.

Overall the authors found that some PROs are more likely to be used than others.  Specifically:

Characteristics of PRO data that maximize influence on payer decision making were reported to be (a) quality, well-controlled, and transparent PRO evidence; (b) psychometric validation of the PRO measure in targeted populations; and (c) publication in peer-reviewed journals.


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