CEE Expert Guide on the reimbursement of orphan drugs in Ukraine

Yes. There are two definitions of this term, which are quite similar.

Under the “Procedure of Expert Evaluation of Materials for Medicinal Products of Limited Use Submitted for State Registration (Re-registration)”, approved by the Order of the Ministry of Health of Ukraine No. 55 dated 26 January 2010, a drug of limited use (orphan drug) is a medicinal product intended for diagnostics, prevention or treatment of a rare disease, i.e. a disease that is life-threatening or disabling, which does not usually affect more than five in 10,000 people at the time the application for state registration is submitted.

Under the recently adopted new Law on Medicines, which entered into force on 28 July 2022 but will be enacted only 30 months after the end of martial law in Ukraine, an orphan drug is a medicinal product for which the marketing authorisation holder can demonstrate that:

• the medicinal product is intended for diagnostics, prevention or treatment of a rare disease, i.e. a disease that is life-threatening or disabling, which does not usually affect more than five in 10,000 people at the time the application for state registration is submitted;
• the medicinal product is intended for diagnostics, prevention or treatment of a life-threatening or seriously immunosuppressive disease, which is seriously debilitating or chronic, and, without state incentives, it is unlikely that such medicinal products will be registered in Ukraine. In this case, a medicinal product intended for the treatment of diseases that occur in Ukraine in more than five in 10,000 people might be recognised as orphan;
• there is no available method of diagnostics, prevention or treatment of the respective disease in Ukraine, or, if such method exists, the medicinal product will be of great benefit to patients with that disease.

Yes. There is no separate procedure for orphan drugs, but the main avenues for the reimbursement of drugs (including orphan drugs) in Ukraine are:

• state healthcare programmes, where medicines listed in the National Essential Medicines List, or centralised nomenclatures approved on an annual basis are procured through centralised public procurement procedures;
• regional/municipal healthcare programmes, where medicines are procured through regional or hospital public procurement;
• an outdated quasi-reimbursement procedure approved by the Resolution of the Cabinet of Ministers of Ukraine No. 1303 dated 17 August 1998, where medicines for certain diseases and certain groups of patients are dispensed for free or with a discount.

The Ministry of Health of Ukraine funds state healthcare programmes. It outsources the centralised procurement to its centralised procurement agency the State Enterprise Medical Procurement of Ukraine (the “MPU”), as well as to several international procurement organisations, including Crown Agents, UNDP and UNICEF. In recent years, all of these agencies have procured orphan drugs paid for by the central budget account.

Regional/municipal governments and public hospitals subordinated to them procure orphan medicines covered by regional/municipal healthcare programmes, which vary region to region.

The outdated quasi-reimbursement procedure defined by the Resolution of the Cabinet of Ministers of Ukraine No. 1303 provides for free or discounted Rx medicinal products (including orphan drugs) dispensed by municipal pharmacies for outpatient treatment for particular groups of people and particular diseases (including, e.g. phenylketonuria and cystic fibrosis). Due to the lack of transparency and efficiency of the procedure, it is gradually being replaced with more sustainable state and regional/municipal programmes.

Finally, outpatient medicinal products can receive reimbursement though the outpatient national reimbursement programme. So far, no orphan drugs have received such reimbursement, as the programme is designed for relatively cheap and mostly generic medicines. Moreover, at this point the programme does not include an option to reimburse the price confidentially, which demotivates most companies and prevents them submitting their innovative medicines for outpatient reimbursement.

While Ukraine has been reimbursing some orphan drugs for more than a decade, the recently implemented health technology assessment mechanism (the “HTA”) has significantly transformed the market access process.

Currently, there are two HTA procedures:

• the company-initiated HTA, where a company prepares and submits a drug dossier to the HTA indicating the proposed reimbursement price;
• the MOH-initiated HTA, where the Ministry instructs the HTA to assess the therapy considering the historic reimbursement price.

For now, to be reimbursed by the local state budget, the drug must successfully undergo either of the two HTA procedures.

As a result of the HTA and subject to the HTA’s recommendation, the MOH takes its decision on the drug and:

• lists it on the National Essential Drugs List (normally not applicable to expensive orphan drugs);
• lists it in the state nomenclature for the centralised healthcare programmes (if the reimbursement price for the drug is not confidential);
• proceeds with confidential negotiations with the company, and, if the negotiations are successful, procures the drug under a confidential risk-/cost-sharing managed entry agreement (“MEA”).

Additionally, based on the HTA outcomes, regional health authorities can list a drug in the regional nomenclatures for the regional healthcare programmes.

All reimbursement decisions are temporary.

The Fundamentals of the Legislation of Ukraine on Health Care declare a commitment by the government to guarantee uninterrupted full reimbursement of orphan drugs. However, in practice all reimbursement decisions are linked to the annually approved central/regional budgets.

Regarding drugs procured based on managed entry agreements, the timeframe of reimbursement is limited by the agreements (up to three years), and the price is subject to annual revision based on the fast-track MOH-initiated HTA procedure.

Yes. As Ukraine is not an EU-member state, there is no formal reference to the EMA orphan drug list in the local legislation/practice of the authorities.

As of now, only one new orphan therapy received national reimbursement in 2022, namely Roche’s SMA therapy redisplay. The product successfully passed the HTA procedure and the company then successfully negotiated and entered into a three-year managed entry agreement to supply the drug.

Eight other orphan drugs (therapies from BioMarin, Sanofi-Genzyme and Takeda) have successfully undergone HTAs and received three-year reimbursement decisions according to the confidential terms of risk-/cost-sharing agreements between the government and the MAHs of the products. None of these therapies is new to the market but had been supplied under the usual centralised public procurement procedures.

A few other orphan therapies (never before reimbursed and already on the Ukrainian market) are currently undergoing HTAs. Thus, we expect reimbursement decisions soon.

There is no unified source of data on the reimbursement of orphan drugs at the regional/local level. Thus, we cannot exclude that some products might have received reimbursement under the regional healthcare programs.

Under the applicable regulation, an HTA procedure normally takes up to 110 business days, not including justified breaks. A fast-track HTA takes up to 40 business days. In practice, with all the stop-clocks, the standard company-initiated procedure takes between six months to a year.

Following the HTA, if successful, the drug is listed on one of the lists. Alternatively, the company can negotiate a managed entry agreement. In practice, the full cycle from HTA application submission to the actual start of reimbursement usually takes between one to two years for a company-initiated HTA, and between 3 and 12 months for an MOH-initiated HTA.

In 2022, parliament and the MOH adopted legislation and regulations on expanded access programmes, where patients can obtain free access to a therapy under the HTA/authorisation process. However, no reimbursement is provided for medicines supplied under expanded access programmes. In addition, expanded access is currently permitted only for not-yet-authorised drugs.

In 2021, the Cabinet of Ministers of Ukraine approved the Concept for the Development of a System to Provide Medical Care to Patients Suffering from Rare (orphan) Diseases for 2021-2026 (the “Concept”).

The Concept provides for the creation and maintenance of a state register of patients with rare diseases, professional development for specialists in early diagnostics, prevention and treatment of rare diseases and other measure which may affect the reimbursement process/practices.

The new Law on Medicines, due to be enacted 30 months after the end of martial law in Ukraine, also provides for a special fast-track procedure for marketing authorisations for orphan drugs, as well as a lower bar for the MAH of an orphan drug.