RWD and RWE for Regulatory and Health Technology Assessments

This article is based on the session “Real-World Evidence (RWE) for Regulatory and Health Technology Assessment Decision Making: Where Are We?” at the DIA 2024 Global Annual Meeting, in San Diego, June 2024. Many thanks to the presenters, Solange Corriol-Rohou and David Ross, both from AstraZeneca, for their valuable insights. This translation is the author’s rendering of their points and should not be taken as exact quotations.  

In recent years, the use of real-world data (RWD) and real-world evidence (RWE) has evolved significantly in the life science industry. Traditionally utilized for post-licensing for safety monitoring, RWD and RWE are now increasingly employed in pre-production stages and clinical trials. This shift raises numerous questions about the optimal use of such data during product development, including integrating patient experience data and patient-reported outcomes.  

Patient involvement is crucial, yet the primary methods of data collection—registries and digital health tools—pose challenges in terms of data acceptability by regulators. In December 2023, the U.S. Food and Drug Administration (FDA) published, “Realizing the Promise of Real-World Evidence.”¹ To navigate these challenges, continuous communication with regulatory bodies is essential to gain their support for using RWD and RWE in clinical trials. 

We need to continue to implement RWD and RWE into clinical trials to understand disease and natural history, ascertain treatment patterns, optimize patient management, and expose the impacts to public health. RWD and RWE provide valuable insights into the patient perspective which otherwise may go unreported. However, this is not just a U.S.-centric challenge and opportunity.  

International Involvement 

European trade associations have formed specific groups dedicated to focusing on RWD, aiming to identify and establish standards. Within pharmaceutical and biotech companies, it is imperative to engage the right experts across different functions when considering RWD and RWE. This cross-functional collaboration ensures comprehensive evaluation and utilization of the data.  

Various international regulatory agencies have released independent guidance on the use of RWD and RWE in clinical trials, with the International Coalition of Medicines Regulatory Authorities (ICMRA) leading efforts to harmonize global standards. Lessons learned from the COVID-19 pandemic underscored the importance of sharing information and critical knowledge, which facilitated the rapid development and global distribution of vaccines. 

Global private-public partnerships are becoming increasingly common, with regulatory bodies collaborating to create shared learning opportunities. These partnerships encourage the dissemination of findings, provided there is an open dialogue with sponsor companies. 

Members of the International Coalition of Medicines Regulatory Authorities (ICMRA), a global conference of government health bureaucrats, include:

UK MHRA 

The Medicines and Healthcare Products Regulatory Agency is an executive agency of the Department of Health and Social Care in the United Kingdom which is responsible for ensuring that medicines and medical devices work and are acceptably safe. 

Health Canada 

Health Canada is the department of the Government of Canada responsible for national health policy.  

EU 

The primary regulatory bodies in the European Union (EU) are the European Parliament, the Council of the European Union, and the European Commission. 

Swissmedic 

The Swiss Agency for Therapeutic Products is the Swiss surveillance authority for medicines and medical devices. 

PMDA Japan 

Japanese regulatory agency, working together with Ministry of Health, Labour and Welfare.  

NMPA China 

The National Medical Products Administration is a national bureau responsible for drug supervision under the State Council of China and is managed by the State Administration for Market Regulation. 

TFDA Taiwan 

The Republic of China Food and Drug Administration is a Republic of China government agency, which is responsible for the safety and quality of food, drug, medical service and cosmetics. It is part of the Ministry of Health and Welfare. FDA is a regulatory member of ICH association. 

U.S. FDA 

The United States Food and Drug Administration is a federal agency of the Department of Health and Human Services. 

Operational Considerations 

On the operational side, the landscape of RWD and RWE is vast and varied. It includes primary, secondary, and hybrid primary-secondary data sources such as filtered and unfiltered claims data, pharmacy data, pathology studies, electronic medical records (EMRs), and hospital and insurance claims.  

This plethora of information presents several challenges, including: 

• Researchers often face delays in accessing data due to interoperability issues between different systems and inconsistent coding methodologies. 
• National drug codes may vary across data sources, necessitating standardization in analyzing RWD and RWE. 
• Privacy and confidentiality concerns must be addressed. As a result, clinical trials may encounter detrimental impacts tied to their data integrity. 

Additional insights: Understanding the New FDA Guidance for Drug and Biological Product Submissions Containing Real-World Data to Successfully Navigate Regulatory Decisions 

To effectively manage these issues, the integration of metadata, semantic coding, and multiple data sources is essential. This approach ensures the application of FAIR (Findable, Accessible, Interoperable, and Reusable) principles. However, accessibility and reusability of data remain significant challenges. Leveraging artificial intelligence (AI) to process and analyze RWD and RWE can be critical in overcoming these hurdles, enhancing the efficiency and effectiveness of RWD and RWE utilization in regulatory and health technology assessment decision-making. 

Overall, the evolving landscape of RWD and RWE utilization highlights the need for continued innovation and collaboration among regulators, industry stakeholders, and researchers. By addressing the challenges and embracing new technologies, the potential of RWD to improve regulatory outcomes can be fully realized. 

Additional insights: Bringing In-vitro Diagnostics to Market With Real World Evidence  

How Halloran Can Help 

With the surge in technology adoption, clinical trial sponsors invest in diverse systems and services across all development phases, leading to a complex data ecosystem and massive data generation and transfer. Regulatory bodies, globally, are increasingly focused on data integrity and sponsor oversight. Our consultants excel in data integrity across domains, crafting strategies to ensure consistent integrity and inspection readiness. To learn more about how we can help, contact us.  

Reference: 

1. U.S. FDA. Catching Up with Califf. Realizing the Promise of Real-World Evidence. Current as of December 21, 2023. https://www.fda.gov/news-events/fda-voices/realizing-promise-real-world-evidence