Biomarker-guided drug development provides value for patients, payers and drug developers: lessons learned from 25 years in the biomarker industry

Abstract

Introduction

There is an urgent, persistent, need for better biomarkers in clinical drug development. More informative biomarkers can increase the likelihood of drug advancement or approval, and implementing biomarkers increases the success rate in drug development. Biomarkers may guide decisions and allow resources to be directed to the projects most likely to succeed. However, biomarkers that are validated to high standards are needed, reflecting biological and pathological processes accurately. Such biomarkers are needed to develop treatments faster, and to improve and guide clinical trial design by selecting and de-selecting patients.

Methods

In this review based on the authors’ previous published experience and interaction with pharmaceutical- and biomarker stakeholders, we highlight the use and value of biomarkers in clinical development according to the BEST guidelines. We highlight the value of 3 types of biomarkers that may provide optimal value to stakeholders: diagnostic, prognostic and pharmacodynamic biomarkers.

Results

A more appropriate clinical trial design, increasing the ratio between benefits and side effects, may come from a more tailored biomarker-approach identifying suitable molecular endotypes of patients to treat.

Discussion

Biomarkers may guide drug developers in selecting the optimal projects to progress, when designing clinical studies and development paths. Biomarkers may aid in the diagnosis and prognostic assessment of patients and assist in matching the molecular endotype to the selected treatment, which improves the success rate of clinical development progression. The aim of this paper is to provide a comprehensive ideation framework for how to utilize biomarkers in clinical development, with a focus on utility for patients, payers and drug developers.

Keywords:

• Biomarkers
• clinical development
• drug development

Disclosure statement

All authors are employees at Nordic Bioscience A/S. ALRP, SR, MBA, CSA, RV and MK are shareholders in Nordic Bioscience A/S.

Data availability statement

The authors confirm that the data supporting the findings of this study are available within the article.

Notes

1 https://pubmed.ncbi.nlm.nih.gov/27010052/.

2 https://www.fda.gov/drugs/biomarker-qualification-program/letter-support-los.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.