Real-world data for efficient health technology assessment

      The new discipline of ‘real-world data science’ has emerged over recent years. Expert practitioners in this embryonic field are finding their feet, not just in terms of role and remit, but also with regards to which questions they can answer and the methods they can bring to bear [
      • Garrison Jr., L.P.
      • Neumann P.J.
      • Erickson P.
      • Marshall D.
      • Mullins C.D.
      Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report.
      ]. But why are pharmaceutical companies employing real-world data scientists and seeking real-world data opportunities on a scale not previously seen? The last two decades saw a similar explosion in organisations with specialist expertise in the delivery of randomised controlled trials (RCTs) and pharmacovigilance to the standards of licencing authorities that include the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). But it is not these regulators that have driven the demand for real-world data (RWD). Instead it is the appearance of the, so-called, ‘fourth hurdle’. It is now necessary to demonstrate cost-effectiveness in addition to the traditional licencing requirements of quality of manufacture, safety and efficacy prior to market access. The clue to why RWD is an important component of any estimation of cost-effectiveness is in the name: the definition of effectiveness, as opposed to efficacy, refers to the measurement of effects in the real-world, rather than under the conditions of experimentation required for the unbiased measurement of efficacy.
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        • Garrison Jr., L.P.
        • Neumann P.J.
        • Erickson P.
        • Marshall D.
        • Mullins C.D.
        Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report.
        Value Health. 2007; 10 ([see comment]): 326-335
      1. Guide to the methods of technology appraisal. NICE, London2013
        • Prasad V.
        • Kim C.
        • Burotto M.
        • Vandross A.
        The strength of association between surrogate end points and survival in oncology.
        JAMA Intern Med. 2015 Aug 1; 175: 1389
        • Freemantle N.
        • Marston L.
        • Walters K.
        • Wood J.
        • Reynolds M.R.
        • Petersen I.
        Making inferences on treatment effects from real world data: propensity scores, confounding by indication, and other perils for the unwary in observational research.
        BMJ. 2013; : 347
        • Caro J.J.
        • Briggs A.H.
        • Siebert U.
        • Kuntz K.M.
        Modeling good research practices–overview: a report of the ISPOR-SMDM odeling Good Research Practices Task Force-1.
        Med Decis Mak. 2012 Jan 1; 32: 667-677
        • van Kampen R.J.W.
        • Ramaekers B.L.T.
        • Lobbezoo D.J.A.
        • de Boer M.
        • Dercksen M.W.
        • van den Berkmortel F.
        • et al.
        Real-world and trial-based cost-effectiveness analysis of bevacizumab in HER2-negative metastatic breast cancer patients: a study of the Southeast Netherlands Breast Cancer Consortium.
        Eur J Cancer. 2017; 79: 238-246
        • Menon D.
        • McCabe C.J.
        • Stafinski T.
        • Edlin R.
        • State B.S.C.
        Principles of design of access with evidence development approaches: a statement from the Banff summit.
        Pharmacoeconomics. 2010; 28: 109-111
        • McCabe C.
        • Edlin R.
        • Hall P.
        • Wallner K.
        Leasing health technologies-an affordable and effective reimbursement strategy for innovative technologies?.

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