In the context of administrative
proceedings on the determination of reimbursement and reimbursement conditions
for medicinal products, it is, under certain circumstances, possible to take
into account the so-called subjective parameter. This factor allows for the
reflection of specific features of a medicinal product that cannot be fully
captured through standard objective criteria.
However, subjective
parameters are not always relevant to the proceedings, as demonstrated by the
following examples from practice. In some cases, they may even be perceived as a
limitation of the submitted documentation.
Subjective Parameter in Reimbursement Indication Restriction
The Ministry of
Health upheld SÚKL’s approach of including the MSWS-12 questionnaire—a subjective
parameter—in the reimbursement indication restriction for a particular
medicinal product.
The Ministry
justified the correctness of this procedure by referencing the wording of the Summary
of Product Characteristics (SmPC), which allows the prescribing physician,
already at the initiation of treatment, to evaluate efficacy using the MSWS-12
questionnaire instead of the objective T25FW test.
For this reason,
the Ministry dismissed the appellant’s objection, which questioned the
demonstration of efficacy through a subjective questionnaire. The Ministry
stated that efficacy proven by such a tool is sufficiently supported by the
conditions established during the marketing authorization process.
Moreover, the
Ministry emphasized that the inclusion of subjective parameters in cost-effectiveness
analyses is not considered a flaw or deficiency of such analyses in general.
Subjective Parameter in Clinical Evaluation
In certain cases, the use of a subjectively assessed primary efficacy endpoint in the evaluation of therapeutic appropriateness for reimbursement purposes has been accepted by SÚKL:
The primary efficacy endpoint assessed in clinical trials was based on purely subjective patient assessment. Considering that the MG-ADL score is more sensitive to changes in condition than the QMG score, and given the observed correlation between changes in MG-ADL and QMG scores, this benefit parameter is deemed acceptable. The treatment’s efficacy compared to placebo can be considered statistically significant, even though the difference in the primary endpoint was not clinically significant.
In contrast, in other proceedings, the subjective nature of the primary study endpoint has been labeled an unacceptable limitation for the purpose of reimbursement determination:
SÚKL proposes not
to grant reimbursement for the medicinal product in the indication of
symptomatic treatment of myotonia. In view of the study’s limitations and other
concerns (e.g., potential overestimation of efficacy, the dose used, and the
subjective nature of the primary endpoint)...
The study’s primary
endpoint had a subjective character (VAS scale based on patient evaluation).
The secondary endpoint (chair test) was objectively measurable. Even so,
variability exists between different types of NDM, with some types affecting
upper limbs and facial muscles more, and others affecting the legs. EMA also
stated that direct measurement of muscle strength would have been more
appropriate.
These examples illustrate that the subjective nature of a study’s primary endpoint may in some cases be a limitation for reimbursement purposes. However, if the parameter is embedded in the indication restriction in accordance with the product’s SmPC, it should be accepted by SÚKL for the purpose of reimbursement determination.
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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 text was translated using ChatGPT 4o.