Ethical Challenges in Biomarker‐Driven Drug Development

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The Belmont Report, one of the foundational documents for the field of research ethics, outlines three ethical principles: respect for persons, beneficence, and justice.1 Much of the literature on the ethical implications of biomarker‐centered research has focused on these principles. For example, there has been vigorous debate about whether informed consent should be required for future research use of patient biospecimens.2 Informed consent is the primary way in which the research enterprise expresses the principle of respect of persons. However, given that biomarker discovery typically depends on access to large quantities of specimens, some worry that biomarker research could effectively grind to a halt if investigators were required to get patient consent every time they wanted to study a sample of biobanked specimens. To address this ethical concern, while preserving patient privacy and confidentiality, some research institutions and regulators have proposed guidelines for coding or anonymizing specimens.3
The principle of beneficence requires that all experiments with human subjects have a favorable risk/benefit balance. This principle is fundamental to the precision medicine movement, which aims to redefine disease in terms of particular biomarker signatures. By studying how to tailor treatments to biomarker‐defined patient populations, precision medicine can improve the overall risk/benefit profile of research—study participants will be more likely to benefit, the results of research will be more applicable to the relevant clinical populations, and the biomarker‐negative patients can be spared the burdens of ineffective treatments. Yet some commentators have argued that the research investment required to realize these benefits from precision medicine could do far more good—i.e., better express beneficence—if it were put toward research and translation of public health interventions.4
The principle of justice requires that the burdens and benefits of research should be distributed equally across society, and that those who participate in research—and thereby contribute to the advancement of knowledge—should have access to the fruits of this labor. Some commentators have argued that as biomarkers are used to “salami slice” patient populations into smaller and smaller groups, the costs of therapies targeted to these groups is likely to rise considerably.5 If this is correct, and the predictable result of biomarker research is higher drug costs, which will restrict future patients' access to the benefits of the research, then this research activity itself may be unjust.
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