Policies for Use of Real-World Data in Health Technology Assessment (HTA): A Comparative Study of Six HTA Agencies

Makady, Amr; Ham, Renske M.T. ten; Boer, Anthonius de; Hillege, Hans L.; Goettsch, Wim

doi:10.1016/j.jval.2016.12.003

Cited by 158 publications

(149 citation statements)

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“…3 With increasing attention paid to the applicability of evidence to specific target populations, however, clinicians, payers, HTA organizations, regulators, and clinical guideline developers are likely to turn to RWE to sharpen decision making that heretofore had been guided principally by RCTs (frequently using a placebo control) in narrowly defined populations.…”

mentioning

confidence: 99%

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

Berger¹,

Sox

Willke³

et al. 2017

Value in Health

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Purpose: Real-world evidence (RWE) includes data from retrospective or prospective observational studies and observational registries and provides insights beyond those addressed by randomized controlled trials. RWE studies aim to improve health care decision making. Methods: The International Society for Pharmacoeconomics and Outcomes Research (ISPOR)and the International Society for Pharmacoepidemiology (ISPE) created a task force to make recommendations regarding good procedural practices that would enhance decision makers' confidence in evidence derived from RWD studies. Peer review by ISPOR/ISPE members and task force participants provided a consensus-building iterative process for the topics and framing of recommendations.Results: The ISPOR/ISPE Task Force recommendations cover seven topics such as study registration, replicability, and stakeholder involvement in RWE studies. These recommendations, in concert with earlier recommendations about study methodology, provide a trustworthy foundation for the expanded use of RWE in health care decision making. Conclusion:The focus of these recommendations is good procedural practices for studies that test a specific hypothesis in a specific population. We recognize that some of the recommendations in this report may not be widely adopted without appropriate incentives from decision makers, journal editors, and other key stakeholders. KEYWORDScomparative effectiveness, decision making, guidelines, pharmacoepidemiology, real-world data, treatment effectivenessThis is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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confidence: 99%

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

Berger¹,

Sox

Willke³

et al. 2017

Value in Health

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312

239

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“…1,2 The importance and influence of such "real world" evidence is demonstrated by commitment of governments around the world to develop infrastructure and technology to increase the capacity for use of these data in comparative effectiveness and safety research as well as health technology assessments. [3][4][5][6][7][8][9][10][11][12] Research conducted using healthcare databases currently suffers from a lack of transparency in reporting of study details. [13][14][15][16] This has led to high profile controversies over apparent discrepancies in results and reduced confidence in evidence generated from healthcare databases.…”

Section: Introductionmentioning

confidence: 99%

Reporting to Improve Reproducibility and Facilitate Validity Assessment for Healthcare Database Studies V1.0

et al. 2017

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Purpose: Defining a study population and creating an analytic dataset from longitudinal healthcare databases involves many decisions. Our objective was to catalogue scientific decisions underpinning study execution that should be reported to facilitate replication and enable assessment of validity of studies conducted in large healthcare databases. This article is a joint publication by Pharmacoepidemiology and Drug Safety and Value in Health. Methods:This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

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confidence: 99%

“…However, there are no common policies for which type of RWD is needed to generate evidence in the HTA process [24] and how RWE is used [25].…”

Section: Public-private Partnerships and Creating A Critical Mass Of Rwmentioning

confidence: 99%

“…HTAs are adapting a similar approach to recommend on condition that companies can provide RWE to address effectiveness in real world patients in actual healthcare setting [23]. However, there are no common policies for which type of RWD is needed to generate evidence in the HTA process [24] and how RWE is used [25].There is an ongoing discussion on adaptive licensing in Europe, USA and Japan and its implementation will have a huge implication on volume of RWE studies [26].Way forward with RWE RWE is here to stay as it enables VBM's approach. The demand for high-quality RWD sources and RWE will continue to grow.…”

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confidence: 99%

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Deriving more value from RWE to ensure timely access of medicines by patients

Wamala-Andersson

Kyhlstedt²

2017

J. Comp. Eff. Res.

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Keywords: comparative effectiveness research • evidence-based medicine • health technology assessmentReal world evidence (RWE) is defined as a form of evidence generated to answer a question or test a hypothesis using real world data (RWD) and using appropriate design and statistical analyses [1]. Others define RWE as the intersection between the randomized controlled trials (RCTs) and effectiveness of treatment in usual clinical practice [2]. To those with public health expertise, RWE is not totally a new phenomenon. Population-based studies using RWD are often conducted to evaluate the effectiveness and value of health programs and policies. Thus, RWE is an old wine in a new bottle. RWD characterized as patient registries have been a long tradition in the Nordic countries due to publicly financed healthcare and a unique person identifier, which enables linkage of data from different registries [3]. However, the impact of RWD/ RWE on health technology assessment (HTA) decision-making process regarding new treatments is not well assessed and the explanatory factors are not well known. Why RWE now?RWE has recently gained attention from patients, providers, payers, regulators and pharma industry. People are living longer in almost every part of the world with increasing demand for a healthy living. Technological advancement enables quick flow of data and information, thus increasing level of knowledge on health and on what works and what does not [4]. Patients have high expectations on health systems to offer safe and effective treatments.Parallel to this, global public health trends show increasing burden of noncommunicable diseases due to unhealthy lifestyle [5], implying a large proportion of people living a long unhealthy life about 30-40 years.Healthcare costs are rising at a much faster rate than gross domestic product (GDP), creating budget deficiencies [5]. This reinforces payers to ensure that they put money in treatments that are cost effective, add value to patients and improve population health.Thus, regulatory agencies begin to realize the benefit of RWE for regulatory decisionmaking for medicines and medical devices.Thus, in order for the industry to sell their products, they need RWE studies to address effectiveness, efficiency, value and safety of their products. Some companies have developed strategies to generate RWE, others are using ad hoc solutions, while the rest are waiting for the RWE wave to pass. Developing a new treatment is costly, yet a large proportion will never make it to the market. RWE is a potential cost-saver in various phases of the drug development lifecycle [6].RCTs are regarded as the gold standard for providing the scientific evidence of clinical Editorial Andersson & Kyhlstedt efficacy of treatments. However, evidence from the RCTs does not address real life conditions due to strict selection criteria with a limited study population, questionable external validity and generalizability [7]. RWE studies, on the other hand, demonstrate effectiveness in real world clinical pra...

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Policies for Use of Real-World Data in Health Technology Assessment (HTA): A Comparative Study of Six HTA Agencies

Abstract: To facilitate the use of RWD for HTA across Europe, more alignment of policies seems necessary. Recent articles and project proposals of the European network of HTA may provide a starting point to achieve this.

Cited by 158 publications

References 23 publications

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

Reporting to Improve Reproducibility and Facilitate Validity Assessment for Healthcare Database Studies V1.0

Deriving more value from RWE to ensure timely access of medicines by patients

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