CEE Expert Guide on the reimbursement of orphan drugs in Czech Republic

Czech law defines an orphan drug as a pharmaceutical which has been determined as an orphan drug under Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products.

Yes, effective as of 1 January 2022, the Czech Republic introduced specific rules for the reimbursement of orphan drugs.

Before 1 January 2022, orphan drugs could have been reimbursed through standard reimbursement procedures or through extraordinary proceedings on a case-by-case basis (i.e. for a specific patient).

The process is set out in Act No. 48/1997 Coll., on Public Health Insurance, as amended (the “Public Health Insurance Act”).

The State Institute for Drug Control (the “Institute”) decides on the amount and conditions of the reimbursement. The application can be filed either by the marketing authorisation holder (the “MAH”) or a sick fund. Participants to the proceedings include the MAH, sick funds, a professional society consisting of professionals specialising in the treatment of the disease that may be affected by the pharmaceutical in question, and a respective patient organisation.

In the reimbursement proceedings, the Institute assesses, among other things:

• therapeutic efficacy and safety;
• substitutability by other treatments covered by health insurance;
• demonstrable contribution to improving the patient’s quality of life;
• recommended practices of professional institutions and relevant professional societies;
• cost-effectiveness analysis, but without considering its result in the form of an incremental cost-benefit ratio;
• projected budgetary impact considering the public interest.

The Institute should issue an assessment report within 110 days from the commencement of the proceedings which is then submitted to the Ministry of Health, together with statements of the participants to the proceedings, for their binding opinion.

The Ministry of Health (a specific advisory board of the Ministry) can either: (i) approve the amount and conditions of reimbursement as set out in the assessment report; (ii) set out the amount and conditions of reimbursement differently than determined in the report; or (iii) reject the reimbursement. Following the binding opinion of the Ministry of Health, the Institute issues a decision.

If an orphan drug receives reimbursement on the basis of an application submitted by an MAH, the MAH must reimburse sick funds for the costs incurred for the reimbursement of the orphan drug in excess of the costs indicated in the budget impact analysis which formed the basis for the Institute's decision.

In principle, the decision on reimbursement is not limited in time. However, the Institute may either on the basis of an application by a sick fund (filed one year from the original decision at the earliest) or ex-officio start a procedure to re-assess the amount and conditions of the reimbursement if certain statutory conditions are met, e.g. the financial impact to the state budget has substantially exceeded original estimates.

A pharmaceutical which does not have orphan designation cannot benefit from the specific procedure described above, but it can receive a reimbursement through a standard reimbursement procedure for “regular” drugs or through a reimbursement procedure for highly innovative pharmaceuticals. An orphan drug could also receive reimbursement through a specific, extraordinary, procedure on a case-by-case basis, e.g. for a specific patient.

In 2022, one orphan product received reimbursement based on the new procedure. In 2023, so far four products have received reimbursement following the new procedure. According to the chairman of the Institute, there are currently an additional 11 pending applications and they expect that they will receive 20 applications this year.

The Public Health Insurance Act provides for certain statutory deadlines for the reimbursement proceedings, but these do not regulate the entire proceedings. The Institute needs to issue its assessment report within 110 days from the commencement of the proceedings. Once the report is issued, the participants have 15 days to provide their statements. After that, the Institute provides the report to the Ministry of Health which should issue its binding opinion within 30 days.

In practice, from the beginning of the effectiveness of new rules, the reimbursement proceedings (from the application to the decision) take around nine months. Currently, the process takes around six months but it always depends on each case, how well the applicants prepare the necessary documentation, and whether they are willing to reach an agreement with all stakeholders.

There are no significant developments planned in the near future at the national level. However, the European Commission is currently working on the Pharmaceutical Strategy for Europe, which may lead to developments at the EU level.