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Adapting the randomised controlled trial (RCT) for precision medicine: introducing the nested-precision RCT (npRCT)
Nils Kappelmann1,2, Bertram Müller-Myhsok3,4, Johannes Kopf-Beck5
1Department of Translational Research in Psychiatry, Max Planck Institute of Psychiatry, Kraepelinstraße 2-10, 80804, Munich, Germany. n.kappelmann@gmail.com.
Trials
|January 7, 2021
Summary
A novel nested-precision randomized controlled trial (npRCT) design efficiently combines traditional and precision trials. This approach supports precision medicine algorithm development and testing while increasing statistical power for comparative effectiveness research.
Area of Science:
- Clinical Trials
- Precision Medicine
- Biostatistics
Background:
- Traditional randomized controlled trials (RCTs) face challenges with high costs and large sample sizes.
- Precision medicine requires algorithms to optimize treatment allocation for individual patients.
- Existing trial designs do not fully integrate algorithm development with rigorous testing.
Discussion:
- The nested-precision RCT (npRCT) integrates a traditional RCT with a precision RCT.
- This design enables online development and testing of precision medicine algorithms.
- Joint analysis of data from both trial components enhances statistical power for comparative effectiveness.
Key Insights:
- The npRCT offers a unified platform for algorithm development and validation.
- It allows for efficient comparison of intervention A versus B using combined data.
- Sample size requirements are quantified, demonstrating potential cost savings.
Outlook:
- The npRCT presents a novel strategy for precision medicine trial design.
- Practical considerations like blinding and bias require careful attention.
- Further research should explore optimal application contexts for the npRCT.
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