Evidence-based medicine arose from a clear need and represents a major advance in the science of clinical decision-making. Despite broad acceptance of evidence-based medicine, however, a fundamental issue remains unresolved: evidence is derived from groups of people, yet medical decisions are made by and for individuals. A new National Academy of Medicine (NAM) Special Publication, Caring for the Individual Patient: Understanding Heterogeneous Treatment Effects, examines how a better understanding of heterogeneous treatment effects (HTE) can lead to safer, more efficacious, personalized care for patients.

By understanding the reasons for treatment effect heterogeneity and developing ways to predict how different patients will respond to a treatment, medical researchers and physicians should be able to personalize medicine to a far greater degree than is possible today. Such an ability would open the door to treatments that are more effective with fewer side effects and would also allow patients to make more informed decisions about the types of medical treatments they choose to receive.

This special publication is based on a workshop that considered patient and stakeholder perspectives on the importance of understanding the methods for predicting treatment effects, and best practices for implementing clinical programs that take HTE into account. For evidence to be more applicable to individual patients, we need to combine methods for strong causal inference (first and foremost, randomization) with methods for prediction that permit inferences about which particular patients are likely to benefit and which are not. Better population-based outcomes will only be realized when we understand more completely how to treat patients as the unique individuals that they are.

The National Academy of Medicine, established in 1970 as the Institute of Medicine, is an independent organization of eminent professionals from diverse fields including health and medicine; the natural, social, and behavioral sciences; and beyond. It serves alongside the National Academy of Sciences and the National Academy of Engineering as an adviser to the nation and the international community. Through its domestic and global initiatives, the NAM works to address critical issues in health, medicine, and related policy and inspire positive action across sectors. The NAM collaborates closely with its peer academies and other divisions within the National Academies of Sciences, Engineering, and Medicine.  The views presented in this special publication – “Caring for the Individual Patient: Understanding Heterogeneous Treatment Effects” – are those of the authors and do not represent formal consensus positions of the NAM; the National Academies of Sciences, Engineering, and Medicine; or the authors’ organizations.


Download the special publication and associated resources: www.nam.edu/IndividualPatient

Join Our Community

Sign up for NAM email updates