The Dilemma Between Efficacy as Defined by Regulatory Bodies and Effectiveness in Clinical Practice

Reduced mortality and morbidity burden • Improved quality of life • Prolonged overall survival • Remission-free interval.Frequently used surrogate parameters such as progression-free survival are inaccurate, and a correlation with overall survival can usually not be demonstrated.However, such parameters are found in most presented licensing studies (4, 5). Licensing studies are not studies of benefitHowever, such trials do little to enable physicians to provide patients with suitable explanations of the benefit of a new drug immediately after it has been authorized, or to make a joint decision with patients on therapeutic intervention.Patient-oriented benefit cannot be deduced on the basis of such licensing studies, particularly when preapproval clinical trials have not provided a single comparison of patient-oriented outcomes such as data on quality of life with drugs that are already available.Although the absolute efficacy of the drug has been investigated as required by the German Medicines Act, this does not allow determination of its relative efficacy in comparison to other drugs or interventions, or even of how the benefit of the drug compares with that of standard treatment.Among the reasons for this are the following