Research
Compelling vision, significant objectives & innovative projects
Our Journey
Goals & Outcomes
The GetReal Institute embarked on its journey with a compelling vision, driven by significant objectives and innovative project ideas derived from IMI GetReal. We engage our members and stakeholders to solicit ideas for our areas of activities, while also advancing the continued development and refinement of IMI GetReal outputs, such as the RWE Navigator and GetReal Pragmatic Trial Tool.
With an unwavering dedication to continuous learning, GetReal Institute is poised to shape a future where evidence-based decisions revolutionise the landscape of healthcare.
The Institute ensured the multistakeholder perspective needed to position this study in the RWE community, thereby enabling it to contribute to ongoing efforts on the use of RWE-based external controls.
– Wim Goettsch, Professor HTA of Pharmaceuticals, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, The Netherlands
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External Comparators
The increasing use of single-arm trials and other non-randomised designs for regulatory decision-making has posed challenges in establishing causality of observed effects and controlling for multiple sources of bias. There is limited guidance from regulatory and HTA bodies on expectations around evidence quality and study conduct, particularly in non-randomised studies.
The GetReal Institute commissioned the Division of Pharmacoepidemiology and Clinical Pharmacology at Utrecht University to conduct landscaping of the use of external comparators, including a comparison of regulatory and HTA approaches, and propose recommendations for consideration in any guidance development. This landscaping review will be published as a peer-reviewed paper. The next phase of the project will work collaboratively with other organisations to engage with stakeholder groups through interviews and workshops to develop guidance. The forthcoming guidance will help offer much-needed clarity on the use of external controls, establishing analytical minimal standards for decision-making, identifying data quality and standards, and shedding light on the operational aspects of conducting external control studies.
Through this joint endeavour, we are committed to advancing the field of pharmacoepidemiology and clinical pharmacology, ensuring informed and evidence-based decision-making that benefits patients and healthcare systems alike. By bridging the gaps in current practices, we strive to drive positive impact and shape a more robust and equitable healthcare landscape.
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Criteria for Pivotal Evidence
The lack of clear standards for regulatory assessors to utilise RWE in decision-making presents a significant challenge. While there have been notable advancements in causal modelling, the absence of randomisation and the inability to verify crucial assumptions hinder the exclusion of residual confounding and establishing causality. To address this issue the GetReal Institute commissioned Leiden University Medical Center to develop comprehensive criteria for determining when RWE results are compelling and can be confidently used as pivotal evidence.
The project will initiate essential discussions to establish robust methodological approaches for leveraging RWE as pivotal evidence in decision-making. By providing practical recommendations and guidance, key stakeholders, including patients, industry, academia, regulatory and HTA bodies, will benefit from increased transparency and reliability in the utilisation of RWE.
The project’s discussions encompass crucial aspects, such as the use of thresholds for pivotal evidence, emphasising the need for replication of observations and sensitivity analyses, and highlighting reporting aspects and transparency. Through this collaborative effort, we aim to elevate evidence-based decision-making, empowering regulators and decision-makers with a reliable framework for leveraging RWE as a valuable source of pivotal evidence.
The GetReal Institute is invaluable in providing us with the opportunity to set up and execute an important research project to support the use of RWE in regulatory decision-making.
– Rolf Groenwold, Professor of Clinical Epidemiology, Leiden University Medical Center