Access to reimbursement for orphan medicinal products is subject to specific legislative procedures. However, the procedural pathway chosen by the marketing authorisation holder (MAH) fundamentally determines which criteria will be assessed when evaluating clinical benefit and economic effectiveness. This is illustrated by a comparison of two administrative proceedings concerning the same medicinal product in the same indication. Despite this, the regulator reached different conclusions regarding the choice of comparator, which in clinical practice is primarily reimbursed through individual approval by a health insurance fund’s reviewing physician under Section 16 of the Public Health Insurance Act.
Success in the “Orphan”
Pathway, Failure in the “Standard” Pathway
In the earlier proceedings
specifically designed for orphan medicinal products (Section 39da), a
comparator reimbursed under Section 16 was considered relevant.
However, when the same MAH
subsequently sought permanent reimbursement for the same medicinal product
through the standard reimbursement procedure, different rules applied.
According to the State Institute for Drug Control (the Institute), a medicinal
product reimbursed under Section 16 cannot be used as a legally relevant
comparator in this type of proceeding. This approach was subsequently upheld by
the Ministry of Health.
The main reason given was that
a medicinal product reimbursed under Section 16 is not considered “reimbursed”
in the context of usual therapeutic practice, as such reimbursement is limited
to exceptional, individual cases.
By contrast, in the “orphan”
proceedings, the medicinal product reimbursed under Section 16 was accepted as
an appropriate comparator for demonstrating cost-effectiveness and thus became
a necessary element in establishing eligibility for reimbursement.
In its decision, the Ministry emphasised that the acceptance of a comparator reimbursed under Section 16 in the previous “orphan” proceedings does not establish a right to use the same comparator in standard reimbursement proceedings, where the results of the cost-effectiveness analysis are a key factor in the reimbursement decision.
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Articles on decision-making practice are based on publicly available texts from the decisions of the Ministry of Health of the Czech Republic and the State Institute for Drug Control (SÚKL).
A continuously updated overview of decisions issued by SÚKL and the Ministry of Health in the field of pricing and reimbursement is available on the Pharmeca a.s. website.
The English translation was prepared using the latest available ChatGPT model.