SURROGATE ENDPOINT: ALTERNATIVE FOR EARLY ASSESSMENT OF A POTENTIAL TREATMENT EFFECT

Abstract

The efficacy of health technologies, medicines and medical devices should be demonstrated in trails that evaluate final patient-relevant outcomes such as survival or morbidity. We provide a summary of the present use of surrogate end points in health care policy, discussing the case for and against their reviewing and adoption validation methods. Although the use of surrogates can be problematic, they can be validated and selected properly, offers important chances for more efficient clinical trials and faster access to new health technologies that benefit health care systems and patients. In early drug development studies, tumor response is often the true primary endpoint. Usually clinical trials are needed to show that it can be dependent upon to predict, or correlate with, clinical benefit in a context of use. Surrogate endpoints that have undergone this ample testing are called validated surrogate endpoints and these are accepted by the Food and Drug Administration as evidence of benefit. Choosing the right surrogate endpoint and proving that it can predict the intended clinical benefit, however, is not always straightforward. When a disease has been sufficiently studied, surrogate endpoints can measure the underlying cause of a disease (such as low thyroxine levels and hypothyroidism) or an effect that predicts the ultimate outcome (such as measuring diuresis, which is expected to improve symptoms of heart failure).