Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies

Abstract

BackgroundReimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in health technology assessment (HTA) of melanoma drugs by European HTA agencies, as well as differences in RWD use between agencies and across time.MethodsHTA reports published between 1 January 2011 and 31 December 2016 were retrieved from websites of agencies representing five jurisdictions: England [National Institute for Health and Care Excellence (NICE)], Scotland [Scottish Medicines Consortium (SMC)], France [Haute Autorité de santé (HAS)], Germany [Institute for Quality and Efficacy in Healthcare (IQWiG)] and The Netherlands [Zorginstituut Nederland (ZIN)]. A standardized data extraction form was used to extract information on RWD inclusion for both REAs and CEAs.ResultsOverall, 52 reports were retrieved, all of which contained REAs; CEAs were present in 25 of the reports. RWD was included in 28 of the 52 REAs (54%), mainly to estimate melanoma prevalence, and in 22 of the 25 (88%) CEAs, mainly to extrapolate long-term effectiveness and/or identify drug-related costs. Differences emerged between agencies regarding RWD use in REAs; the ZIN and IQWiG cited RWD for evidence on prevalence, whereas the NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed.ConclusionIn general, RWD inclusion was higher in CEAs than REAs, and was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies’ use of RWD; however, no visible trends for RWD use over time were observed.Electronic supplementary materialThe online version of this article (10.1007/s40273-017-0596-z) contains supplementary material, which is available to authorized users.

Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making

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Integrating Health System Data With Systematic Reviews: A Framework for When and How Unpublished Health System Data Can Be Used With Systematic Reviews

“…In other examples, unpublished data were used to provide contextual information (other than effectiveness or harms/safety information); for example, to detail uptake of services, patient characteristics, epidemiology, natural history, or cost or data for cost-effectiveness analyses. [19][20][21][22][23] In some instances, the rationale for using unpublished data was not evident and/or appeared opportunistic (i.e., researchers had access to their own unpublished data and included it because it was available), and was not applied to healthcare decision making. Overall the examples we identified applied clinical practice data or specific registry data from a single health system (e.g., Mayo Clinic) or regional or nationally representative data (e.g., clinical registries, hospital database).…”

The Role of Digital Strategies in Financing Health Care for Universal Health Coverage in Low- and Middle-Income Countries

“…Another driver will be the progressive use of so-called real-world data—data obtained not from randomized trials but from more heterogeneous groups of patients already under treatment or observation. 37 In the future, strategic purchasers will make greater use of granular data on population and health services. The main challenge will probably remain the same as described earlier: solutions will have to combine data of very different natures from very different sources.…”