LAUSR

A large-scale clinical trial in patients with chronic heart failure and a reduced ejection fraction demonstrates that a new treatment reduces the risk for cardiovascular death. The results are clinically meaningful and statistically persuasive. The drug is approved for use throughout the world. How should we select patients to receive the drug in clinical practice? Many would say that the drug should be prescribed to patients who were similar to and treated similarly to those enrolled in the pivotal trial that demonstrated its benefits. Yet, doing so precisely would represent a practical impossibility. The trial was carried out in specialized centres by research teams, who were expert in the care of patients with heart failure. The protocol specified dozens of entry criteria; many were applied only to make the trial efficient rather than to focus specifically on patients who might benefit. Patients were provided with free study medication and were followed closely by attentive study coordinators. After commercialization, should we require that all these conditions be duplicated for patients to receive treatment? We do not demand that practitioners act as clinical trialists when they manage patients. We do not mandate that treated patients be identical to those studied or be cared for under highly supervised conditions. If we did so, most patients who would benefit from the new life-prolonging drug would never receive it; the findings of the clinical trial would never be translated for the public good.