Healthcare Policy Implications of Heterogeneity of Treatment Effects

Heterogeneity of treatment effects (HTE) is a phenomenon wherein the same treatment produces different responses in different patients. Following scientific presentations at a conference on HTE in drug therapy, a roundtable panel discussed the policymaking implications of this phenomenon in the current healthcare environment. The presentation of evidence on HTE served as a backdrop for this more pragmatic, solutions-based discussion of how HTE should be addressed in light of the trend in healthcare toward use of evidence-based medicine along with professional society clinical practice guidelines for specific disease states. Overall, the panel concluded that a specific agent should be used when the clinician is equipped with sound data. However, in the absence of such data, care has to be individualized, using the clinician’s best judgment regarding available treatment options. The sharing of data across all levels of the healthcare infrastructure is crucial for policymakers seeking to ensure quality care while considering the phenomenon of HTE and, at the same time, keeping cost-effectiveness a major concern.