surgoncoКреативная хирургия и онкологияCreative surgery and oncology2076-30932307-0501Башкирский государственный медицинский университет10.24060/2076-3093-2023-13-2-151-158surgonco-812Research ArticleОБЗОР ЛИТЕРАТУРЫREVIEWSЭффективность применения противоопухолевых лекарственных препаратов «вне инструкции» в онкологииEfficacy of Off-Label Use of Anticancer Drugs in Oncologyhttps://orcid.org/0000-0001-6062-5318КарабинаЕ. В.KarabinaE. V.

Елена Владимировна Карабина

отделение противоопухолевой лекарственной терапии

ИДПО

кафедра онкологии с курсами онкологии и патологической анатомии

Тула

Республика Башкортостан

Уфа

Elena V. Karabina

Anticancer Drug Therapy Unit

Department of Oncology with Courses of Oncology and PathologicalAnatomy for Advanced Professional Education

Tula

Ufa

kev-251@yandex.ruhttps://orcid.org/0000-0003-4341-6017СакаеваД. Д.SakaevaD. D.

Дина Дамировна Сакаева, д. м. н., профессор

кафедра фармакологии с курсом клинической фармакологии

Республика Башкортостан

Уфа

Dina D. Sakaeva, Dr. Sci. (Med.), Prof.

Department of Pharmacology with a Course of Clinical Pharmacology

Ufa

https://orcid.org/0000-0002-8867-504XЛипатовО. Н.LipatovO. N.

Олег Николаевич Липатов, д. м. н., профессор

ИДПО

кафедра онкологии с курсами онкологии и патологической анатомии 

Республика Башкортостан

Уфа

Oleg N. Lipatov, Dr. Sci. (Med.), Prof.

Department of Oncology with Courses of Oncology and PathologicalAnatomy for Advanced Professional Education

Ufa

Тульский областной клинический онкологический диспансер; Башкирский государственный медицинский университетРоссияTula Regional Clinical Oncology Dispensary; Bashkir State Medical UniversityRussian FederationБашкирский государственный медицинский университет; Клинический госпиталь «Мать и дитя»РоссияBashkir State Medical University; Mother and Child Clinical HospitalRussian FederationБашкирский государственный медицинский университетРоссияBashkir State Medical UniversityRussian Federation202330062023132151158Copyright © Карабина Е.В., Сакаева Д.Д., Липатов О.Н., 20232023Карабина Е.В., Сакаева Д.Д., Липатов О.Н.Karabina E.V., Sakaeva D.D., Lipatov O.N.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.surgonco.ru/jour/article/view/812

   Применение противоопухолевых лекарственных препаратов «вне инструкции» широко распространено в современной онкологии. Потенциальные преимущества подобных назначений ассоциированы с превышением ожидаемой клинической пользы над рисками возникновения осложнений. Использование противоопухолевых лекарственных препаратов не в соответствии с инструкцией демонстрирует разную эффективность этого подхода в зависимости от типа злокачественного новообразования, от причин назначения указанных агентов и от их принадлежности к той или иной фармакологической группе. В ряде ситуаций клиническая польза от применения лекарственных препаратов «вне инструкции» выглядит более убедительно, чем при наличии официально зарегистрированных показаний. В настоящее время можно с уверенностью констатировать рутинный характер назначения «старых» зарегистрированных противоопухолевых лекарственных препаратов, использующихся в повседневной клинической практике. Однако в инструкциях по медицинскому применению к ним не отражен весь спектр показаний, для которых имеются убедительные доказательства безопасности и эффективности. Наблюдающийся сдвиг парадигмы в сторону воздействия на молекулярные мишени и применения иммунотерапии при различных локализациях злокачественных новообразований способен оказать влияние на увеличение количества использования указанных агентов «вне инструкции». Отсутствие вариантов лечения при редких формах злокачественных новообразований и при исчерпанности возможностей зарегистрированной терапии является ведущей причиной назначения таргетных препаратов не в соответствии с инструкцией на основе выявленных молекулярно-генетических нарушений. Зачастую в подобных случаях концепция прецизионной терапии реализуется путем применения агентов, клиническая эффективность которых подтверждается данными с низким уровнем доказательности или с отсутствием каких-либо доказательств. Изучение эффективности использования противоопухолевых агентов не в соответствии с инструкцией необходимо для систематизации информации и выработки алгоритмов принятия решений о назначении указанных препаратов в рутинной клинической практике.

   The off-label use of anticancer drugs is widespread in modern oncology. The potential advantages of such prescriptions are associated with exceeding the expected clinical benefi ts over the risks of complications. The off-label use of anticancer drugs demonstrates the inconsistent efficacy of this approach depending on the type of malignancy, the reasons for prescribing these agents and their belonging to a particular pharmacological group. In a number of situations, the clinical benefits of off-label drugs are more convincing than in case of authorized indications. Currently, prescribing the “old” registered anticancer drugs, used in everyday clinical practice, is seen routine. However, labeling does not reflect the full range of indications with strong evidence of safety and efficacy. The paradigm shift toward molecularly targeted therapy and immunotherapy in various malignancies may increase the off-label use of the specified agents. Lack of treatment options for rare forms of malignancies and exhaustion of the possibilities for registered therapy are the major reasons for off-label prescribing targeted drugs based on the identifi ed molecular genetic disorders. In such cases, the concept of precision therapy is oft en implemented by using agents, the clinical efficacy of which is confi rmed by data with a low level of evidence or with no evidence. Studying the eff ectiveness of the off-label use of anticancer agents is necessary to systematize information and develop algorithms for making decisions about the prescription of these drugs in routine clinical practice.

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The authors declare that there are no conflicts of interest present.