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Genotype-based clinical trials in cardiovascular disease

Naveen L Pereira1, Daniel J Sargent2, Michael E Farkouh3

  • 1Division of Cardiovascular Diseases, Department of Internal Medicine, 200 First Street SW, Rochester, MN 55905, USA.

Nature Reviews. Cardiology
|May 6, 2015
PubMed

Insights

Pharmacogenomics aims to personalize medicine by matching drugs to patients, but clinical use is hindered by a lack of genotype-based randomized clinical trials (RCTs). Overcoming ethical and practical hurdles is key for implementing these trials.

Area of Science:

  • Pharmacogenomics and personalized medicine
  • Clinical trial design
  • Genetics and drug response

Background:

  • Randomized clinical trials (RCTs) typically assess average treatment effects in homogeneous populations.
  • Interindividual variability in drug response and adverse effects is well-documented.
  • Pharmacogenomics offers potential for tailored therapies but faces implementation challenges.

Purpose of the Study:

  • To explore the need for and challenges of genotype-based RCTs in pharmacogenomics.
  • To highlight the potential benefits of personalized medicine through pharmacogenetics.
  • To discuss ethical, legal, and practical considerations for genotype-based clinical trials.

Main Methods:

  • Review of existing literature on RCTs, pharmacogenomics, and clinical implementation.
  • Discussion of technological, statistical, and modeling advances in pharmacogenomics.
  • Analysis of ethical, legal, and practical issues related to genotype-based RCTs.

Main Results:

  • RCTs provide average treatment effects, not accounting for individual variability in drug response.
  • Pharmacogenomics has advanced significantly, but clinical application is limited by a lack of benefit-demonstrating RCTs.
  • Genotype-based RCTs could individualize therapy, maximize efficacy, minimize adverse effects, and potentially reduce trial costs.

Conclusions:

  • Genotype-based RCTs are crucial for the clinical implementation of pharmacogenetic principles.
  • Addressing ethical, legal, and practical challenges is essential for conducting these trials.
  • The role of pre-emptive genotyping in electronic health records versus dedicated RCTs requires further investigation.

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