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

PharmacoEconomics, Dec 2017

Background Reimbursement 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. Methods HTA 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. Results Overall, 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. Conclusion In 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.

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

PharmacoEconomics March 2018, Volume 36, Issue 3, pp 359–368 | Cite as Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies AuthorsAuthors and affiliations Amr MakadyArd van VeelenPáll JonssonOwen MoseleyAnne D’AndonAnthonius de BoerHans HillegeOlaf KlungelWim Goettsch Open Access Original Research Article First Online: 06 December 2017 9 Shares 2.8k Downloads 1 Citations Abstract Background Reimbursement 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. Methods HTA 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. Results Overall, 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. Conclusion In 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 material The online version of this article ( https://doi.org/10.1007/s40273-017-0596-z) contains supplementary material, which is available to authorized users. Key Points for Decision Makers Real-world data (RWD) may provide useful evidence on relative effectiveness (REAs) and cost effectiveness assessments (CEA) for reimbursement decisions. This study showed that RWD is more often included in CEAs than REAs. In REAs and CEAs, RWD is often used to describe the effectiveness/safety of a new drug in clinical practice and to predict the long-term effectiveness of the new drug, respectively. Differences emerged between agencies in how they use RWD for reimbursement decisions. 1 Introduction Melanoma is the most serious and fatal form of skin cancer [1], and its incidence has been increasing, largely caused by increased exposure to ultraviolet radiation [1, 2, 3]. Primary tumours are most often removed by surgical excision; however, after tumour metastasis, surgical excision is often no longer feasible and pharmacotherapy becomes the remaining option [1, 4]. According to the literature, prior to 2011 dacarbazine was the standard chemotherapeutic of choice for the treatment of metastatic (or non-operable) melanoma (henceforth melanoma) [5, 6]. Since 2011, multiple drugs for the treatment of melanoma have entered the market, representing four novel mechanisms of action, thereby substantially increasing treatment options [1, 7]. Regulatory approval of new therapeutics in Europe is centralized, with decisions being issued by the European Commission [8]; however, each European jurisdiction decides nationally on drug reimbursement and pricing, conventionally based on assessments and appraisals of available evidence conducted by national health technology assessment (HTA) agencies. These involve relative effectiveness assessments (REAs), sometimes in combination with cost-effectiveness assessments (CEAs), based on evidence submitted by the marketing authorisation holders of drugs. For the purposes of this article, we define REAs as assessments that examine the extent to which an intervention does more good than harm, when compared with one or more alternative interventions for achieving the desired results and when provided under the routine setting of healthcare practice [9, 10]. Meanwhile, CEAs examine the relationship between relative effects and the respective costs of implementing the intervention versus its comparators [11]. Evidence on drug effectiveness informing HTA submissions is conventionally (...truncated)


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Amr Makady, Ard van Veelen, Páll Jonsson, Owen Moseley, Anne D’Andon, Anthonius de Boer, Hans Hillege, Olaf Klungel, Wim Goettsch. Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies, PharmacoEconomics, 2017, pp. 359-368, Volume 36, Issue 3, DOI: 10.1007/s40273-017-0596-z