For many new drugs, there are important outcomes research questions relating to epidemiology, patient-reported outcomes, and healthcare economics. The research needs and strategies may be different for potential blockbuster drugs than for traditional products. A potential blockbuster drug that advances efficacy may require reevaluation of the current state of treatment and prevalence of disease. In terms of patient-reported outcomes, a new blockbuster drug may arise in a disease area where patient-reported outcomes are especially important and innovation is needed to evaluate them optimally. In terms of healthcare economics, a potential blockbuster drug requires a truly international outcomes research program to address cost-effectiveness in different countries. The outcomes research used to support several existing and potential blockbuster products is reviewed.
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