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Use of Real-World Data for the Research, Development, and Evaluation of Oncology Precision Medicines
Jan R R Lewis1, Ian Kerridge1, Wendy Lipworth1
1All authors: Sydney Health Ethics, University of Sydney, Sydney, New South Wales, Australia.
Abstract:
Although randomized controlled trials remain the scientific ideal for determining the efficacy and safety of new treatments, they are sometimes insufficient to address the evidentiary requirements of regulators and payers. This is particularly the case when it comes to precision medicines because trials are often small, deliver incomplete insights into outcomes of most interest to policymakers (eg, overall survival), and may fail to address other complex diagnostic and treatment-related questions. Additional methods, both experimental and observational, are increasingly being used to fill critical evidentiary gaps. A number of modified early- and late-phase trial designs have been proposed to better support earlier biomarker validation, patient identification, and selection for regulatory studies, but there is still a need for confirmatory evidence from real-world data sources. These data are usually provided through observational, postapproval, phase IIIB and IV studies, which rely heavily on registries and other electronic data sets-most notably data from electronic health records. It is, therefore, crucial to understand what ethical, practical, and scientific challenges are raised by the use of electronic health records to generate evidence about precision medicines.
Insights
Randomized controlled trials are insufficient for precision medicine evidence. Real-world data from electronic health records (EHRs) are crucial but present ethical, practical, and scientific challenges.
Area of Science:
- Pharmacoeconomics and Health Policy
- Biomedical Informatics
- Clinical Trial Design
Background:
- Randomized controlled trials (RCTs) are the gold standard for treatment efficacy and safety.
- RCTs often provide insufficient evidence for precision medicines due to small sample sizes and limited outcome data.
- Regulators and payers require comprehensive evidence beyond traditional RCTs.
Purpose of the Study:
- To explore the necessity of supplementary data sources beyond RCTs for precision medicine.
- To identify the role of real-world data (RWD) in addressing evidentiary gaps.
- To examine the challenges associated with using electronic health records (EHRs) for generating evidence on precision medicines.
Main Methods:
- Review of current literature on clinical trial designs and RWD utilization.
- Analysis of the limitations of traditional RCTs for precision medicine.
- Identification of modified trial designs and observational study approaches.
- Focus on the use of registries and EHR data in post-approval studies (Phase IIIB/IV).
Main Results:
- Modified trial designs aim to improve biomarker validation and patient selection.
- Real-world data, particularly from EHRs, are increasingly vital for regulatory and payer evidence.
- Significant ethical, practical, and scientific challenges exist in using EHRs for evidence generation.
Conclusions:
- Traditional RCTs are often inadequate for the complex needs of precision medicine.
- Real-world data sources, especially EHRs, are essential to fill critical evidence gaps.
- Addressing the challenges of EHR data utilization is crucial for advancing precision medicine research and policy.
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