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Real-World Evidence Generation and Evaluation of Therapeutics Proceedings of a Workshop (2017) / Chapter Skim
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Appendix D: Discussion Paper: Real-World Evidence to Guide theApproval and Use of New Treatments
Pages 77-86

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From page 77...
... . The Perspective is intended to help inform and stimulate discussion.
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From page 78...
... Even when traditional efficacy trials evaluate ten fails to address key questions of patients, physi- these effects, the resulting evidence is rarely adequate cians, and health systems regarding the appropriate to guide patients' and physicians' decisions regarding role of new treatments. Those unaddressed concerns alternative treatments -- especially if those treatments include the following: are to be continued for months or years.
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From page 79...
... Traditional producing the data essential for regulatory approval. clinical trials, in which treatment protocols are highly Traditional clinical trials are optimized to efficiently ad controlled, usually offer little information on how new dress key questions in the regulatory process: Is a new treatments affect overall or downstream use of health treatment superior to a placebo or other appropriate services.
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From page 80...
... Enabling Developments The Increasing Use of Standard Patient-Reported The Increasing Use of Electronic Health Records Outcome Measures and Development of Linked Data Resources In addition to traditional clinical data, patient experi Data generated from research and practice have his ences and perspectives are increasingly being incor torically been siloed. However, as the concept of a porated as essential aspects of the medical product learning health system continues to take hold, such evaluation process.
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From page 81...
... a recent demonstration of the potential for using the Example: FDA Sentinel Initiative OHDSI system in large-scale, international observa tional research, the disease treatment pathways for The FDA Sentinel initiative was created with the goal of type 2 diabetes mellitus, hypertension, and depres establishing an integrated, national, electronic system sion were investigated using data aggregated from that monitors the safety of medical products, includ 11 sources providing electronic health records of 250 ing small molecule drugs and biologics. The initiative million patients across four countries (Hripcsak et al., was taken in response to recommendations of the In 2016)
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From page 82...
... While more proval studies, in 2013 FDA approved expanded label- than 60 primary care clinics were involved in the trial, ing for a transcatheter heart valve (Edwards SAPIEN) , patients in the study area are served by one regional allowing its use among a larger segment of patients hospital, and both primary and secondary health pro with aortic stenosis; the decision was based partially viders share one integrated electronic health record on data provided from the TVT Registry (Society of Tho- system.
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From page 83...
... delay in the approval process. Creating appropriate incentives for real-world evidence generation will re- True Integration between Research and Practice quire the following: Ultimately, the evidence necessary to guide policy deci • Guidance regarding appropriate data sources, re- sions regarding new treatments overlaps substantially search methods, and analytic methods to support with the evidence necessary to guide everyday clinical labeling regarding effectiveness, tolerability, value, decisions for individual patients.
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From page 84...
... 2015. Observational Health Data Sciences and real-world effectiveness trial in chronic obstructive Informatics (OHDSI)
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From page 85...
... 2013. FDA approval of Disclaimer device labeling highlights growing use of clinical registry The views expressed in this Perspective are those of data for post-market device surveillance: STS/ACC TVT the authors and not necessarily of the authors' orga Registry™ data cited as part of FDA decision process.
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Key Terms

  • evidence generation
  • traditional clinical
  • electronic health
  • health records
  • medical devices

  • valve therapy
  • efficacy trials
  • clinical data
  • treatment effects
  • transcatheter valve

  • tvt registry
  • phase iii
  • sentinel initiative
  • lung study
  • accessed september

  • published october
  • salford lung
  • data sciences
  • data generated
  • observational health

  • learning health
  • medical product
  • regulatory approval
  • bipartisan policy
  • policy center

  • key questions
  • therapy registry
  • health data
  • health research
  • aortic valve


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