The study of results is a broad umbrella concept that does not have a permanent definition. The results study examines the final results of medical care, in other words, the effect of the process of providing medical services on the health and well-being of patients. In other words, clinical outcome studies aim to monitor, understand, and optimize the impact of medical treatment on a specific patient or a specific group. Such studies describe scientific research that is related to the effectiveness of health measures and medical services, that is, the results obtained through such services.
Attention is often focused on the person suffering from the disease — in other words, on the clinical
конечные точки (overall outcomes) that are most relevant for that patient or group of patients. Such endpoints may be
качество жизни or the degree of pain. However, outcome studies can also focus on the effectiveness of health care delivery, with measurement parameters being
эффективность затрат, health status and severity of the disease (the impact of health problems on a person).
The difference between evidence-based medicine and outcome research is the focus on different issues. While the main objective of evidence-based medicine is to provide optimal medical care to the patient in accordance with clinical evidence and experience, research on the results is primarily aimed at determining the endpoints. In a clinical outcome study, these points usually correspond to clinically relevant endpoints.
In outcome studies, relevant endpoints often become symptoms or parameters measuring functional abilities and care needs — something that the patient receiving treatment considers important. For example, a patient suffering from an infection who has been injected with penicillin may pay more attention to the fact that he does not have a fever and his general condition has improved than to the effect of penicillin on the actual level of infection. In this case, the symptoms and how he feels are considered as a direct assessment of his state of health — and these are
конечные точки,which are the focus of attention when conducting a study of the results. The patient is also likely to be interested in the possible side effects associated with penicillin, as well as the cost of treatment. In the case of other diseases, such as cancer, an important clinical outcome relevant to the patient will be
риск fatal outcome.
If the study is lengthy in time, when studying the research results, the following methods can be used: «
суррогатные конечные точки».
Суррогатная конечная точка involves the use of a biomarker to measure an outcome, acting as a surrogate for a clinical endpoint measuring
эффективность. Рассмотрим
клиническое исследование, in which the effect of penicillin is tested by reducing the amount of one type of protein (C-reactive protein), which is always present in the blood. The amount of this protein in the blood of a healthy person is very small, but with an acute infection it increases rapidly. Thus, measuring the level of C-reactive protein in the blood is an indirect way to determine the presence of infection in the body, therefore, in this case, the protein serves as a "biomarker" of infection..
Биомаркер — this is a measurable indicator of the state of the disease. This parameter is also correlated with the risk of developing or progressing the disease or with how the prescribed treatment will affect the disease. A blood sample is taken daily from the patient for analysis to measure the amount of the biomarker in the blood.
It should be emphasized that in order to use a surrogate endpoint for the purpose of control and supervision, the token must be confirmed or verified in advance. It is necessary to demonstrate that changes with the biomarker are correlated (consistent) with the clinical outcome in the case of a specific disease and the effect of treatment.