New Ukrainian law regulates off-label use of medicines

04/ 01/ 2025

Прослухати онлайн Зупинити   On 21 August 2024, the Ukrainian parliament introduced new legislation that formalises the off-label use of registered medicinal products. This regulation establishes clear procedures for healthcare professionals (HCPs) to prescribe medicines for non-authorised indications, age groups, dosages, or methods of administration, which are commonly known as off-label use. The goal is to ensure patient safety, transparency, and accountability in off-label prescriptions by requiring informed consent, medical advisory board approval, and thorough documentation. The new mechanisms complement the existing legal framework, offering a more structured approach to off-label prescriptions, which was previously applied informally in Ukraine. This new legislation ensures that off-label use follows a transparent and regulated process, aligning with international best practices. Key amendments to the legislation: 1) Patient safety and informed consent: The new regulation requires that HCPs, before prescribing off-label, provide full information to the patient or their legal representative on the risks, expected benefits, alternative treatment options and clinical trials conducted in Ukraine, in which the patient can participate. This will ensure that the patient is fully aware of the implications of the off-label use and can make an informed decision. 2) Eligibility criteria for off-label use: Off-label use is permitted only under the following conditions: The patient suffers from a life-threatening condition or a disease that significantly impairs their quality of life. There are no approved treatment alternatives available in Ukraine that meet the patient’s needs. The expected benefit of using the medicine off-label outweighs the risks of not using it. A medical advisory board of the healthcare institution must confirm the absence of effective on-label alternative treatments and determine whether to recommend off-label use. 3) Documentation and medical justification: The decision to prescribe a medicine off-label must be fully documented in the patients medical records, in accordance with regulations on medical record keeping. The documentation should include: The rationale for prescribing the medicine off-label. References to relevant clinical guidelines (including foreign clinical guidelines, as permitted under the pre-existing legal framework) that substantiate the off-label use. The formal decision of the medical advisory board, which is responsible for recommending off-label use, including the names of the HCPs involved in the board and their consensus on the decision. Comparisons with the EU legislation Ukraine’s new legislation is consistent with EU principles of off-label use, particularly regarding patient safety and informed consent. Moreover, it establishes a more structured process for documenting and justifying off-label prescriptions, which is not uniformly required across all EU member states. Timelines The law will come into effect the day after its official publication, with enforceability starting three months after the effective date. At present, the law awaits formal publication, which is expected within the coming weeks. Full enactment of the law is anticipated by the end of 2024. Possible implications for off-label reimbursement and procurement The new law does not automatically permit the reimbursement or procurement of medicines for off-label use. Historically, off-label procurement has sometimes been practised in Ukraine. For example, oncology treatments without paediatric indications have been procured for paediatric patients where no on-label treatment alternatives were available. With the introduction of this formal legal process for off-label use, centralised procurement agencies of the Ministry of Health, regional governments, or hospitals can procure therapies for off-label use. Individual patient prescriptions, however, will still require adherence to the new process, including medical advisory board approval and informed consent. Implications for life sciences companies The new off-label regulations create important considerations for life sciences companies operating in Ukraine. While the law allows the sharing of information about non-authorised indications with HCPs, companies must be cautious in their approach. The Law on Medicines in Ukraine permits the dissemination of information on medicinal products, including those not authorised or in development, to HCPs through professional publications and events. Companies, however, must ensure that they do not promote off-label use as a standard of care or routine practice. Off-label prescriptions must adhere to the requirements for informed consent and proper documentation since off-label use can only be promoted as an exception. Compliance: Companies must adhere to internal policies on off-label use and promotion, as well as applicable industry codes. Off-label use should be presented only as a special exception to standard care and not as a typical option for treatment. Furthermore, any discussions about off-label use must fully comply with legal standards. Market Opportunities: The clearer regulatory framework around off-label use may open opportunities for engaging with HCPs and payers (e.g. procurement agencies, hospitals) about potential off-label use, provided these discussions are evidence-based and fully compliant with legal standards. Payers may now consider sourcing therapies for off-label use in cases where patients face life-threatening conditions or diseases without approved treatment alternatives available in Ukraine. For more information on Ukraine’s medical and pharmaceutical sectors, contact your CMS client partner or these CMS experts:

On 21 August 2024, the Ukrainian parliament introduced new legislation that formalises the off-label use of registered medicinal products. This regulation establishes clear procedures for healthcare professionals (HCPs) to prescribe medicines for non-authorised indications, age groups, dosages, or methods of administration, which are commonly known as off-label use. The goal is to ensure patient safety, transparency, and accountability in off-label prescriptions by requiring informed consent, medical advisory board approval, and thorough documentation.

The new mechanisms complement the existing legal framework, offering a more structured approach to off-label prescriptions, which was previously applied informally in Ukraine. This new legislation ensures that off-label use follows a transparent and regulated process, aligning with international best practices.

Key amendments to the legislation:

1) Patient safety and informed consent:

The new regulation requires that HCPs, before prescribing off-label, provide full information to the patient or their legal representative on the risks, expected benefits, alternative treatment options and clinical trials conducted in Ukraine, in which the patient can participate. This will ensure that the patient is fully aware of the implications of the off-label use and can make an informed decision.

2) Eligibility criteria for off-label use:

Off-label use is permitted only under the following conditions:

• The patient suffers from a life-threatening condition or a disease that significantly impairs their quality of life.
• There are no approved treatment alternatives available in Ukraine that meet the patient’s needs.
• The expected benefit of using the medicine off-label outweighs the risks of not using it.
• A medical advisory board of the healthcare institution must confirm the absence of effective on-label alternative treatments and determine whether to recommend off-label use.

3) Documentation and medical justification:

The decision to prescribe a medicine off-label must be fully documented in the patient’s medical records, in accordance with regulations on medical record keeping. The documentation should include:

• The rationale for prescribing the medicine off-label.
• References to relevant clinical guidelines (including foreign clinical guidelines, as permitted under the pre-existing legal framework) that substantiate the off-label use.
• The formal decision of the medical advisory board, which is responsible for recommending off-label use, including the names of the HCPs involved in the board and their consensus on the decision.

Comparisons with the EU legislation

Ukraine’s new legislation is consistent with EU principles of off-label use, particularly regarding patient safety and informed consent. Moreover, it establishes a more structured process for documenting and justifying off-label prescriptions, which is not uniformly required across all EU member states.

Timelines

The law will come into effect the day after its official publication, with enforceability starting three months after the effective date. At present, the law awaits formal publication, which is expected within the coming weeks. Full enactment of the law is anticipated by the end of 2024.

Possible implications for off-label reimbursement and procurement

The new law does not automatically permit the reimbursement or procurement of medicines for off-label use. Historically, off-label procurement has sometimes been practised in Ukraine. For example, oncology treatments without paediatric indications have been procured for paediatric patients where no on-label treatment alternatives were available. With the introduction of this formal legal process for off-label use, centralised procurement agencies of the Ministry of Health, regional governments, or hospitals can procure therapies for off-label use. Individual patient prescriptions, however, will still require adherence to the new process, including medical advisory board approval and informed consent.

Implications for life sciences companies

The new off-label regulations create important considerations for life sciences companies operating in Ukraine. While the law allows the sharing of information about non-authorised indications with HCPs, companies must be cautious in their approach. The Law on Medicines in Ukraine permits the dissemination of information on medicinal products, including those not authorised or in development, to HCPs through professional publications and events. Companies, however, must ensure that they do not promote off-label use as a standard of care or routine practice. Off-label prescriptions must adhere to the requirements for informed consent and proper documentation since off-label use can only be promoted as an exception.

• Compliance: Companies must adhere to internal policies on off-label use and promotion, as well as applicable industry codes. Off-label use should be presented only as a special exception to standard care and not as a typical option for treatment. Furthermore, any discussions about off-label use must fully comply with legal standards.
• Market Opportunities: The clearer regulatory framework around off-label use may open opportunities for engaging with HCPs and payers (e.g. procurement agencies, hospitals) about potential off-label use, provided these discussions are evidence-based and fully compliant with legal standards. Payers may now consider sourcing therapies for off-label use in cases where patients face life-threatening conditions or diseases without approved treatment alternatives available in Ukraine.

For more information on Ukraine’s medical and pharmaceutical sectors, contact your CMS client partner or these CMS experts: