real world Evidence

Goals & Purpose

The group seeks to identify methodologies and approaches that allow the integration of real-world evidence into the evaluation process, thus promoting more informed and contextually relevant decisions within the scope of Health Technology Assessment (HTA). It also aims to explore methodologies and tools for implementation in HTA, and to create a collaborative work and training space between RedETSA nodes.

Members of the group

  • Sebastian Garcia Marti – Institute of Clinical and Health Effectiveness-IECS (Argentina)
  • Guadalupe Montero – Argentine Public Network for Health Technology Assessment - RedARETS (Argentina)
  • Graciela Fernandez – National Resources Fund-FNR (Uruguay)
  • Carlos Rezende – Agência Nacional de Saúde Suplementar-ANS (Brazil)
  • Maricela Curisinche – National Institute of Health-INS-CETS (Peru)
  • Mariana Beatriz Pineda Lopez – National Center for Technological Excellence in Health-CENETEC (Mexico)

Results - Job made by the group

A survey was conducted among redetsa members to identify working groups and conduct a minimum survey on the use of RWE in the context of HTA in the different nodes of the network. A document is currently being prepared that seeks to gather and compile relevant resources on the subject in question that are useful for developers of health technology assessment, such as methodological frameworks or tools that facilitate their use and implementation.

I am a member of RedETSA and I want to join the group!

Links of interest

The following methodologies developed in the Americas region were identified:

NICE FRAMEWORK

This document aims to help identify when real-world data can be used to reduce uncertainties and improve recommendations. The document describes best practices for planning, conducting, and communicating real-world evidence studies to improve the quality and transparency of evidence. It does not set minimum acceptable standards for evidence quality.

CADTH

This guidance has the following main objectives: ensuring that regulators and health technology assessment agencies have sufficient information to assess the suitability of a study for decision-making; providing basic reporting standards for RWE studies that align with global standards to prioritise transparency in reporting; and considering the practical challenges related to the use of RWE.

FDA

This framework aims to assess the potential use of RWE to help support approval of a new indication for an already approved drug or to help satisfy requirements for post-approval studies of drugs. The document includes considerations on the use of trials or studies with RWE for effectiveness decisions, assessment of the appropriateness of RWD, potential for study designs using RWD to support effectiveness.

EMA

This document briefly describes how RWE can be useful in the context of regulatory decision-making, the types of studies that can be conducted, the type of question that can be addressed, and how the EMA can help identify the best resources to address a research question. The process for requesting RWE studies is also explained.

AUSTRALIA

Rapid review conducted by the TGA in 2021, including around 50 targeted interviews with stakeholders on their understanding and use of RWE and PRO by the TGA. The review also examined regulatory documents describing how RWE and PRO are used by some comparable overseas medicines and medical device regulators. The document clarifies the definitions of RWE, the potential source of data and points that need to be updated or clarified in the TGA guidance.

ESMO

The ESMO-GROW guideline is a reporting tool developed specifically for oncology RWE studies. It includes a range of materials for users involved in writing, appraising or publishing oncology RWE studies, including: an interactive checklist and scoring tool, a checklist for assessing reporting quality, and a flowchart to inform case selection for analysis in an RWE study.

ANVISA

This guide provides Anvisa's standards to meet the technical requirements of current regulations regarding demonstrating the safety and efficacy of a drug through studies with real-world data. The document includes procedures and items such as recommendations for real-world studies, challenges in generating real-world evidence, types of study designs, real-world data with regulatory standards, and essential elements for the development of real-world studies.