Real World Data (RWD) Access and Availability in Patient Safety

The RWD Access and Availability in Patient Safety initiative seeks to identify key challenges in accessing and applying RWD for patient safety, revealing top barriers and opportunities across a variety of use cases.

 

This initiative will deliver a survey and a corresponding paper that validates these barriers and highlights existing solutions, offering industry stakeholders options for actionable next steps.

Key resources

Rationale

Post-marketing safety assessments have traditionally relied on limited data sources that often lack contextual, longitudinal insights critical to patient safety. Over time, patient safety has expanded to incorporate real-world data (RWD) driven by the need for more timely, comprehensive, and representative safety data across diverse populations to monitor and evaluate post-market drug safety.1

Industry stakeholders have worked to identify bottlenecks in signal assessment and explore ways to incorporate all available evidence, particularly by leveraging RWD and developing alternatives to full study protocols. Various frameworks and guidelines support the use of real-world data (RWD) in pharmacovigilance (PV), such as the ICH M14 guideline, which outlines principles for non-interventional post-authorization safety studies (PASS) and aims to harmonize global expectations for RWD use in PV.2,3

Although conceptual models and checklists help guide database selection and quality evaluation, operational gaps persist – particularly in accessing RWD to assess and study feasibility.4,5,6,7 Challenges such as missing data, variable data quality, representativeness, and measurement bias are well-documented and remain critical to the overall utility of RWD in PV.8

There is an opportunity to leverage RWD to support benefit-risk assessments, help reduce post-marketing study demands, and better align with regulatory expectations without the need for additional studies. TransCelerate seeks to assess how RWD is currently used in pharmacovigilance across pharmaceutical, biotech, and CRO organizations, aiming to identify barriers to RWD access and availability by surveying key stakeholders in the RWD ecosystem and delivering subsequent findings.

1.U.S. Food and Drug Administration. Real-world evidence. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence

2.TransCelerate BioPharma Inc. Rapid signal assessment using real-world data solutions. https://www.transceleratebiopharmainc.com/assets/rapid-signal-assessment-using-real-world-data-solutions/

3.International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. General Principles on Planning, Designing, Analysing, and Reporting of Non-interventional Studies That Utilise Real World Data for Safety Assessment of Medicines. Amsterdam:
https://database.ich.org/sites/default/files/ICH_M14_Step4_Final_Guideline_2025_0905.pdf

4.Patel D, Guleria S, Titievsky L, Flaherty S, Everage N, Korjagina M, Porkess S, Kou TD, Layton D. Contemporary Practice and Considerations for Real-World Data Source Identification and Feasibility Assessment. Pharmacoepidemiol Drug Saf. 2024 Sep;33(9):e5862.

5.Wang SV, Pinheiro S, Hua W, Arlett P, Uyama Y, Berlin JA, Bartels DB, Kahler KH, Bessette LG, Schneeweiss S. STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies. BMJ. 2021 Jan 12;372:m4856.

6.Hall GC, Sauer B, Bourke A, Brown JS, Reynolds MW, LoCasale R. Guidelines for good database selection and use in pharmacoepidemiology research. Pharmacoepidemiol Drug Saf. 2012 Jan;21(1):1-10.

7.Miksad RA, Abernethy AP. Harnessing the Power of Real-World Evidence (RWE): A Checklist to Ensure Regulatory-Grade Data Quality. Clin Pharmacol Ther. 2018 Feb;103(2):202-205.

8.Rudrapatna VA, Butte AJ. Opportunities and challenges in using real-world data for health care. J Clint Invest. 2020 Feb 3;130(2):565-574

Solution

Surveying Access and Availability of Real-World Data in Patient Safety: An Industry Perspective: This article will showcase findings from a recent industry survey demonstrating the shared challenges of leveraging RWD and highlighting opportunities to expand application across patient safety.

Benefits

  • Greater understanding of diverse use cases to leverage RWD to support benefit-risk assessments
  • Motivation for ecosystem stakeholders (drug sponsors, CROs, data providers, etc.) to consider new ways to reduce post-marketing study demands and increase RWD safety use case uptake for more comprehensive evidentiary evaluations
  • Enhanced awareness of opportunities to better align with regulatory expectations without the necessity of conducting additional studies