A New Requirement for Publication: Access to Effective Drugs for Ethical Reasons, The Example of Heart Failure.

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The past decade has witnessed the publication of many trials in the field of heart failure, several of which have demonstrated that new drugs could potentially reduce major cardiac events and sometimes even decrease total mortality. Most medical practitioners have experimented with withdrawal of the drug under investigation, having no knowledge of what the patient had received, consistent with a blinded design. In some such cases, the patient's clinical status may be rapidly compromised after withdrawal, suggesting (i) that the patient had been receiving the active drug and (ii) that the drug under investigation might exert beneficial effects. The patients should receive early benefit from the drug, but they are left most often without this beneficial treatment not only until the trial results are complete but also until after the guidelines are updated and after the reimbursement (months to years after the results). Here, we propose that the study sponsor should plan from study conception that all participating patients (regardless of whether they are in the placebo or active drug group) will have access to the drug upon trial completion—once safety is verified and until the results are known. Nowadays, it is not conceivable to submit the results of a large trial for publication without approval from a suitable ethical committee. Similarly, the access to the effective drug should be considered as a requirement for clinical research, not only for ethics committee approval, and for the registration of the trial in an international database until publication.