Balancing costs, efficacy, and side effects.

Drug approval processes in most developed countries generally do not provide the comparative information necessary for clinicians, patients or policymakers to make informed choices between similar agents, balancing efficacy and toxicity. These questions have been made exponentially more complex with the addition of cost as a third dimension to consider in making therapeutic choices. Application of cost-containment pressure in the absence of adequate data provides a poor foundation for optimal decision making. A systematic, disciplined process is needed for the comparison of similar drugs along each of the three dimensions of efficacy, safety, and cost. Such an inquiry will require use of several methodologies, from conventional randomised controlled trials, to pharmacoepidemiological studies and cost-effectiveness analyses. Such information need not form the basis for drug registration, but instead would be made available to practitioners, patients, and payers to inform their therapeutic choices. The cost of such an ambitious research endeavour would be more than offset by the opportunity for enormously greater efficiencies in the rational use of cost-effective drug therapy.