Hi Derek,
I apply causal inference methods and the target trial emulation framework in many of the projects I work on. But not in the regulatory environment. However, I know of two papers from a team at Harvard working with the FDA that you might find interesting:
- Franklin, J.M., Glynn, R.J., Martin, D. and Schneeweiss, S., 2019. Evaluating the use of nonrandomized real‐world data analyses for regulatory decision making. Clinical Pharmacology & Therapeutics, 105(4), pp.867-877.
- Franklin, J.M., Pawar, A., Martin, D., Glynn, R.J., Levenson, M., Temple, R. and Schneeweiss, S., 2020. Nonrandomized real‐world evidence to support regulatory decision making: process for a randomized trial replication project. Clinical Pharmacology & Therapeutics, 107(4), pp.817-826.
I also have experience with generalizability and transportability studies (see here). I know that NICE (UK) is updating its methodology recommendation in this field to include a better description of how to apply generalizability and transportability methods in the evidence submitted to NICE for decision-making. The recommendation also includes other methods.
I'm happy to chat more if you would like.
Maria
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Maria E. Montez Rath, PhD
Director of the Biostatistics Core, Division of Nephrology
Stanford University
Chair Membership Outreach & Diversity Committee
ASA Statistical Consulting Section
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Original Message:
Sent: 05-23-2024 10:46
From: Derek Blankenship
Subject: Causal Inference
Hello All,
As a biostatistician, I find causal inference theory and the movement toward allowing well designed observational studies to have causal interpretations fascinating (for example see: Dahabreh IJ, Bibbins-Domingo K. Causal Inference About the Effects of Interventions From Observational Studies in Medical Journals. JAMA. 2024 May 9. doi: 10.1001/jama.2024.7741.). I am sure many of you, like me, have used analysis methods common to causal inference such as propensity scores. Does anyone in this group directly apply causal inference methodology and framework such as target trial emulation in your work? If so, do you have experience applying these methods for drugs or devices in a regulated environment? If so and willing, I would love to hear your experience and discuss further!
Best,
Derek
Derek Blankenship, PhD, MBA
Sr. Director, Global Biostatistics Lead
Clinical Advanced Analytics
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Fresenius Medical Care
Global Medical Office
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Cell: 214-361-2892
derek.blankenship@freseniusmedicalcare.com
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Derek Blankenship
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