It’s what you read when you don’t have to that determines what you will be when you can’t help it.
Charles Francis Potter
Novel literature on RWE worth reading. Stay tuned for updates!
- When can real-world data generate real-world evidence? (October 2023) This commentary by Rahman et al. addresses RWD/RWE terminology for drugs and biological products, specifically related to studies of effectiveness submitted to FDA and whether they are classified as RWE by the agency.
- Tactical Considerations for Designing Real-World Studies: Fit-for-Purpose Designs That Bridge Research and Practice (September 2023). This article, by Nancy Dreyer and Christina Mack, offers tactical advice about the value of opportunistic study designs, what to plan for in terms of transparency in data generation and management, and how pharmacy claims are being linked with electronic health records and/or patient-generated health data. The authors also explain that treatment decisions may be made by statistical randomization, not by doctors and patients, but after randomization, naturalistic follow-up can be used.
- Association Between Duration of Immunotherapy and Overall Survival in Advanced Non–Small Cell Lung Cancer (June 2023) In patients with long-term response to immunotherapy for advanced non–small cell lung cancer (NSCLC), should treatment be stopped at 2 years or continued indefinitely? This retrospective RWD cohort study of patients on frontline immunotherapy-based treatment showed that for patients who are progression-free on immunotherapy for NSCLC, it is reasonable to stop therapy at 2 years, rather than continuing indefinitely.
- The next generation of evidence-based medicine. In this Perspective, published in Nature Medicine (January 2023), Subbiah shares his vision of the future of clinical trials and evidence-based medicine, including the role of RWD and RWE.
- Drug repurposing using real-world data. Tan et al. (January 2023) conducted a scoping review to characterize repurposing studies using RWD and discuss their potential challenges and solutions.The use of RWD in this field has emerged due to well-established advantages of drug repurposing in supplementing de novo drug discovery and incentives in incorporating RWE in regulatory approvals.
- Venovenous extracorporeal membrane oxygenation in patients with acute covid-19 associated respiratory failure: comparative effectiveness study. Observational studies are susceptible to confounding, regardless of the study design. However, the emulation of a target trial using observational data will yield the same effect estimate as that of a randomised trial if the emulation is successful. In their paper Urner and colleagues (May 2022) present an emulated pragmatic randomised controlled trial to estimate the effect of treatment with extracorporeal membrane oxygenation compared with conventional mechanical ventilation on hospital mortality within 60 days of admission to the intensive care unit in patients with covid-19 associated respiratory failure. By using the target trial framework, the authors avoided biases common to the design of such observational studies and estimated a per protocol effect using appropriate statistical methods.
- De-identified health datasets promote innovation. In this article (October, 2022), Seastedt et al. address concerns over data anonymization and further explore how increased regulations may inadvertently exclude developing countries over concerns of imperfect data anonymization. The authors argue that limiting data sharing would not only slow the development of future medical innovations and clinical software, but could also potentially expand existing biases that favor high-income countries.
- Real-World Evidence – Where Are We Now? – A NEJM (May 5, 2022) must read that reviews what RWD and RWE mean today from the perspective of the Food and Drug Administration. The article highlights two widespread misconceptions about both terms, which are worth remembering.
- Real-World Data as External Controls: Practical Experience from Notable Marketing Applications of New Therapies. Therapeutic Innovation and Regulatory Science, 2022. Real-world data (RWD) can contextualize findings from single-arm trials when randomized comparative trials are unethical or unfeasible. This article summarizes some lessons learned from such contextualization from 20 notable new drug or biologic licensing applications in oncology and rare diseases.
- Demonstrating Benefit-Risk Profiles of Novel Therapeutic Strategies in Kidney Transplantation: Opportunities and Challenges of Real-World Evidence. Transplant International, 2022. Helantera et al. provide a point of view highlighting that there is an evidence gap in kidney transplantation that calls for innovative clinical trial designs, with RWE providing an opportunity to facilitate longitudinal transplant research with timely translation to clinical practice.
- Real-World Evidence for Regulatory Decision-Making: Guidance From Around the World. Clinical Therapeutics, 2022. In this article, Burns et al. review the available frameworks and existing guidance from across the globe and discuss the observed gaps and opportunities for further development and harmonization.
- Use of Real-World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design. Clinical Pharmacology & Therapeutics 2022, by Dagenais et al. This article reviews how biopharmaceutical companies can leverage RWE to inform internal decisions made throughout the product development process.
- Association of Statin Therapy Initiation With Diabetes Progression: A Retrospective Matched-Cohort Study. JAMA Internal Medicine 2021, by Mansi et al. This large retrospective cohort study found that the diabetes-progression composite outcome was significantly higher among patients with diabetes who used statins than among patients with diabetes who did not use statins. The study examined 12 years of data (2003- 2015)
- Association of E-Cigarettes With Erectile Dysfunction: The Population Assessment of Tobacco and Health Study. American Journal of Preventive Medicine 2021, by El-Shahawy et al. The authors found this association in data from a nationally representative study of 45,971 U.S. adults aged ≥18 years.
- The real-world outcomes of multiple myeloma patients treated with daratumumab, PLOS ONE 2021, by Szabo et al. In this article the authors report that real-world outcomes of all Danish patients with multiple myeloma (MM) treated with daratumumab-based regimens until 1 January 2019 are worse than the results of clinical trials.
- Comparative Effectiveness of BNT162b2 and mRNA-1273 Vaccines in U.S. Veterans. NEJM 2021, by Dickerman et al. In this article, the authors report the results of a RWD analysis to compare how well the two messenger RNA vaccines work. Dickerman and colleagues emulated a target trial using the electronic health records of U.S. veterans.
- Strengthening Causal Inference from Observational Data. NEJM 2021, by Hernán. For researchers using observational data, a useful way to specify a question is to design the target trial that would answer it and then emulate the protocol as closely as possible. In this paper, Hernán explains the reasons behind this argument, illustrating it with an example.
- Weight loss and risk reduction of obesity-related outcomes in 0.5 million people: evidence from a UK primary care database. International Journal of Obesity 2021 March 3, by Haase et al. This study provides objective quantification of the benefit of weight loss for relevant outcomes in a primary care setting.
- Comparative Effectiveness of Initial LAMA versus LABA in COPD: Real-World Cohort Study. COPD 2021, by Suissa et al. This large real-world study in the clinical setting of treatment for COPD supports the most recent recommendations for the initial treatment of COPD. A great example of the clinical utility of RWE studies.
- Value from health data: European opportunity to catalyse progress in digital health. The Lancet 2021, by Vayena, on how innovations in digital health are expected to transform health care, health research, and public health in the years ahead.
- Biases in evaluating the safety and effectiveness of drugs for covid-19: designing real-world evidence studies. Am J Epidemiol 2021, by Renoux et al. In this article, the authors review and illustrate how immortal time bias and selection bias were present in some cohort studies assessing the effectiveness and safety of drugs in patients hospitalized with COVID-19.
- Real-world evidence for assessing pharmaceutical treatments in the context of COVID-19. Clin Pharmacol Ther 2021, by Franklin et al. This article reviews some clinical questions of interest, potential data types, challenges, and merits of RWE in COVID‐19. It provides recommendations for non-randomized RWE designs and analyses based on established RWE principles.
- STaRT-RWE: structured template for planning and reporting on the implementation of real-world evidence studies. BMJ 2021, by Wang et al. This article introduces a structured template for planning and reporting on the implementation of RWE studies.