The government approved the conditions for the use of off-label drugs

The Cabinet of Ministers has decided on the requirements for drugs that can be prescribed for the treatment of more than two dozen diseases not according to the instructions. This is especially important, for example, for pediatric oncology and oncohematology, where the share of such drugs in therapy reaches 60–70%. Now the Ministry of Health will be able to include such drugs in clinical recommendations and standards of medical care. The resolution will take effect on September 1, 2024, a year later than originally planned. The All-Russian Union of Patients warns that barriers to treatment must be eliminated as soon as possible.

The government has approved requirements for drugs that can be prescribed regardless of the indications for their use, that is, off-label. According to resolution, signed by Prime Minister Mikhail Mishustin, from September 1, 2024, the use of off-label drugs can be included in clinical recommendations and standards of medical care for children if they meet a number of requirements. In particular, the effectiveness and safety of such use must be confirmed by studies and recommendations adopted by international professional organizations.

The document notes that prescribing off-label drugs is especially important for the treatment of children with severe chronic diseases, as well as children with cancer – in such cases, the share of drugs used off-label can reach 70%.

Back in May 2022, Mikhail Mishustin approved a list of diseases for which the use of drugs outside the instructions is allowed. The government order became a by-law to the law initiated a year earlier by State Duma Vice Speaker Irina Yarovaya and experts from the Dmitry Rogachev National Medical Research Center for Pediatric Hematology, Oncology and Immunology. The law dealt only with cancer and oncohematological patients, but the government list contains 21 diseases and conditions, including neoplasms, diseases of the blood, respiratory system and endocrine system, malformations and chromosomal disorders, COVID-19, diseases of the nervous system, bone marrow donation (for patients under 18 years of age), pregnancy, childbirth and the postpartum period, as well as conditions requiring palliative care.

The government published a draft resolution establishing the conditions for including off-label drugs in treatment standards and clinical guidelines, as well as requirements for them, in November last year. It was assumed that the document would come into force on September 1, 2023. Immediately after the publication of the draft, the All-Russian Patients’ Union (VSP) appealed to the Ministry of Health with a request not to delay the application of the new rules – in order to avoid “serious consequences.” The authors of the appeal indicated that the law “On the Fundamentals of Protecting Citizens’ Health” prohibits the inclusion of off-label drugs in clinical recommendations before the document comes into force “due to the impossibility of establishing the drug’s compliance with the “requirements established by the government”.” As a result, the possibility of using off-label drugs in minors would be delayed until at least 2024 – due to the long time frame for the development and approval of recommendations and standards, the Supreme Court warned. This can lead to irreversible consequences for the health of patients, the authors of the appeal noted.

In addition, the Supreme Court asked to waive the requirement to confirm the effectiveness and safety of off-label drugs by publication in journals “indexed in international databases.” Patients suggested a “more flexible approach”: taking into account scientific publications indexed in Russian information and analytical systems. This was explained by the “current geopolitical situation”, when the publication of the results of research by Russian scientists in foreign publications is “significantly difficult” and there is a risk of stopping the indexing of certain Russian publications “for political reasons.”

As can be seen from the government’s decision, the resolution will come into force a year later than expected by the Supreme Court.

“This means that someone will have serious barriers to drug therapy,” noted co-chairman of the patients’ union Yuri Zhulev. “But we are glad that the document has been adopted and comes into force, albeit a little later than we asked.” Commenting on the need to confirm effectiveness with international publications, Mr. Zhulev assures that the discussion on this matter will continue. According to him, only doctors can decide whether or not to use this or that drug, and they must rely on all available experience, including Russian.

Medical adviser to the Doctis telemedicine service Dmitry Spiridonov says that the practice of using off-label drugs is quite common, especially in pediatrics.

It shortens the path from drug research to the moment it is prescribed to the patient. However, as a rule, now this happens on the basis of a decision of a medical commission. “This resolution is seen as a logical continuation of the initiative of the Ministry of Health to facilitate the work of doctors with such drugs, removing them from the “gray zone,” continues Mr. Spiridonov. “I hope that the trend will continue and in the future there will be more opportunities for the use of drugs in accordance with modern effective international practices.”

Natalia Kostarnova