Developing Clinical Trials Methodology for Drugs Used to Treat Chronic Heart Failure

INTRODUCTION. The issue of chronic heart failure (CHF) is gaining importance not only in clinical medicine, but also for the entire health care system and society. The need to develop new drugs and drug combinations to treat CHF remains highly relevant. Currently, Russia and the EAEU countries lack regulatory documents or guidelines that control the planning, conduct and evaluation of clinical trials for CHF drugs. Therefore, it is essential to develop such a guideline considering national authorization requirements.

AIM. This study aimed to systematize international approaches to clinical trials of CHF drugs and use them as a base for the relevant guidelines effective in the Russian Federation.

DISCUSSION. Clinical trials of CHF drugs are based on such key principles as randomization, control, blinding, clinically significant endpoints, representativeness of the population, and safety prioritization. Randomized controlled trials using the double-blind method should be deemed as a measurement standard for the efficacy and safety of a new drug. To confirm therapeutic efficacy, a careful selection of patients by well-defined diagnostic criteria is warranted, alongside with a sufficient sample size and observation time, similar concomitant baseline therapy, and development of adapted approaches for special populations (elderly and pediatric patients). The endpoints are chosen by their impact on the disease prognosis and patient quality of life, as well as the contribution of the drug. Notably, increasing attention is being paid to current patient-oriented outcomes (such as improved well-being), provided that safety is ensured and there is no negative impact on the survival rate.

CONCLUSIONS. The principles of conducting CHF clinical trials both globally and in the Russian Federation are unified and stem from the fundamentals of evidence-based medicine. Implementing the described scientific principles of clinical research in CHF patients will contribute to improved treatment standards, which, in turn, should have a positive impact on the disease prognosis and outcomes.

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