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

Value Health. 2017 Apr;20(4):520-532. doi: 10.1016/j.jval.2016.12.003. Epub 2017 Jan 27.


Background: Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies' policies for RWD use in relative effectiveness assessments (REA) is lacking.

Objectives: To review policies of six European HTA agencies on RWD use in REA of drugs. A literature review and stakeholder interviews were conducted to collect information on RWD policies for six agencies: the Dental and Pharmaceutical Benefits Agency (Sweden), the National Institute for Health and Care Excellence (United Kingdom), the Institute for Quality and Efficiency in Healthcare (Germany), the High Authority for Health (France), the Italian Medicines Agency (Italy), and the National Healthcare Institute (The Netherlands). The following contexts for RWD use in REA of drugs were reviewed: initial reimbursement discussions, pharmacoeconomic analyses, and conditional reimbursement schemes. We identified 13 policy documents and 9 academic publications, and conducted 6 interviews.

Results: Policies for RWD use in REA of drugs notably differed across contexts. Moreover, policies differed between HTA agencies. Such variations might discourage the use of RWD for HTA.

Conclusions: 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.

Keywords: policy study; real-world data; real-world evidence; relative effectiveness assessment.

Publication types

  • Comparative Study
  • Review

MeSH terms

  • Comparative Effectiveness Research / economics
  • Comparative Effectiveness Research / legislation & jurisprudence*
  • Comparative Effectiveness Research / standards
  • Consensus
  • Cost-Benefit Analysis
  • Europe
  • Evidence-Based Medicine / economics
  • Evidence-Based Medicine / legislation & jurisprudence*
  • Evidence-Based Medicine / standards
  • Government Regulation*
  • Guidelines as Topic
  • Health Care Costs
  • Health Policy / economics
  • Health Policy / legislation & jurisprudence*
  • Humans
  • Insurance, Health, Reimbursement
  • Interviews as Topic
  • Policy Making*
  • Prohibitins
  • Technology Assessment, Biomedical / economics
  • Technology Assessment, Biomedical / legislation & jurisprudence*
  • Technology Assessment, Biomedical / standards