The State
Institute for Drug Control (SÚKL) has initiated its first ex officio
proceeding to re-evaluate whether a Highly Innovative Medicinal Product
(VILP) continues to meet the required criteria following the entry of a new
comparator into the reimbursement system.
Under the Public
Health Insurance Act, if, during the validity of a decision granting temporary
reimbursement for a VILP, SÚKL subsequently grants reimbursement under Section
39g to another medicinal product with comparable clinical use, the
Institute must promptly initiate ex officio administrative proceedings.
The purpose of these proceedings is to assess whether the VILP still fulfils
the criteria for a highly innovative medicinal product. This obligation does
not apply if the temporary reimbursement would expire in less than 12 months.
Such a
situation has now occurred. The reason for initiating the proceeding was a decision
effective from 1 November 2024, which granted permanent reimbursement
to a new relevant comparator for a similar indication—namely, maintenance
monotherapy in adult patients with BRCA1/2-mutated (FIGO stage III or IV)
advanced high-grade epithelial ovarian, fallopian tube, or primary peritoneal
cancer who are in complete or partial response following first-line
platinum-based chemotherapy.
SÚKL has now
issued a final assessment report regarding the reimbursement of a
medicinal product for patients with advanced ovarian cancer, recommending that the
second temporary reimbursement of the product remain in force. In its
assessment, the Institute compared the efficacy and safety of niraparib
versus olaparib in patients with and without BRCA mutations. It
concluded that comparable efficacy was demonstrated only in patients with
BRCA mutations, and not in BRCA wild-type (BRCAwt) or unknown-status
patients. Due to a lack of clinical evidence, no efficacy comparison
was possible for the BRCAwt/unknown subgroup. For this subpopulation,
the Institute found that the criteria for high innovativeness continue to be
met, and that the appropriate comparator remains best supportive care
(i.e., watch and wait approach).
SÚKL
emphasized that within the scope of this administrative proceeding, it cannot
modify the existing reimbursement conditions by limiting coverage to only part
of the patient population. The decision to revoke or maintain reimbursement
must therefore be based on whether the VILP criteria are met overall.
Given that
the VILP criteria remain fulfilled for a subpopulation of patients
(BRCAwt/unknown), and that the product demonstrated at least a 30%
improvement in the primary endpoint of progression-free survival (PFS)
versus the relevant comparator in the overall population, SÚKL has decided
to maintain the temporary reimbursement.
Timeline:
03/2024 – Medicinal product containing niraparib:
second temporary reimbursement granted in SCAU
11/2024 – Ex officio proceeding initiated
to reassess fulfilment of high innovativeness criteria
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A continuously
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field of pricing and reimbursement is available on the Pharmeca a.s. website.
The State Institute for Drug Control (SÚKL) has rejected a request by
the marketing authorization holder (MAH) of an orphan medicinal product (OMP)
to suspend the proceedings on the setting of the maximum price and
reimbursement.
The Ministry of Health issued a binding opinion for the OMP stating that
the SÚKL is not allowed to grant reimbursement for the given OMP. The MAH
disagreed with the negative binding opinion and submitted a request for its
review. At the same time, the MAH asked the SÚKL to suspend the reimbursement
proceedings for the OMP until the review of the opinion is completed.
The SÚKL argues that it is bound by the binding opinion of the Ministry
and is obliged to issue a decision. According to SÚKL, the submission of a
review request is not a valid reason for suspension because the outcome of the
review is not determinative for this proceeding. The SÚKL holds a binding
opinion from the Ministry, which is key for the decision, and must act in
accordance with it, also considering the legitimate expectations of other
parties to the proceeding. Therefore, the SÚKL will not suspend the
reimbursement proceedings.
Since the new legislation concerning pricing and reimbursement of orphan
drugs came into effect in 2023, the Ministry of Health has issued a total of 31
binding opinions. In 10 of these cases, the Ministry did not agree with the
granting of reimbursement, in 2 cases it proposed changes to the reimbursement
conditions compared to SÚKL’s proposal, and in 1 case it suggested a change in
the reimbursement amount compared to SÚKL’s proposal.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us.
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pharmaceutical and medical device information. We also offer flexible services
that can be tailored to your needs at any time.
Our market position and experience allow us to support you whenever you
need expert guidance.
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 State Institute for Drug Control (SÚKL) has rejected a request by the marketing authorization holder (MAH) of an orphan medicinal product (OMP) to suspend the proceedings on the setting of the...
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.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us.
At Pharmeca, we help you navigate the complex landscape of
pharmaceutical and medical device information. We also offer flexible services
that can be tailored to your needs at any time. Our market position and experience allow us to support you whenever you
need expert guidance.
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.
Full text of the opinion published on the website of the Ministry of Health of the Czech Republic:
Opinion of the Ministry of Health of the Czech Republic on the Selection of a Medicinal Product Intended for the Treatment of a Rare Disease with Reimbursement Granted under Section 39da as a Comparator in Cost-Effectiveness Assessment in Proceedings for the Determination or Modification of Reimbursement in the So-Called Permanent Reimbursement Regime Conducted under Section 39g or Section 39i of Act No. 48/1997 Coll., on Public Health Insurance and on Amendments to Certain Related Acts, as Amended
Cost-effectiveness assessment, as part of evaluating the appropriateness of a therapeutic intervention, is a necessary element of administrative proceedings for the determination or modification of the amount and conditions of reimbursement of a medicinal product conducted under Section 39g or 39i of Act No. 48/1997 Coll., on Public Health Insurance and on Amendments to Certain Related Acts, as amended (hereinafter referred to as "Act No. 48/1997 Coll." or the "Public Health Insurance Act"), in cases enumerated in Section 15(9) of this Act. Fulfilment of the condition of an appropriate therapeutic intervention is one of the basic conditions for granting reimbursement for a medicinal product for a given indication (regardless of whether this indication is defined by a specific formulation of the reimbursement restriction or by the summary of product characteristics), as Section 15(6)(d) of Act No. 48/1997 Coll. stipulates that "The Institute shall not grant reimbursement for medicinal products and foods for special medical purposes that do not meet the conditions of an appropriate therapeutic intervention."
According to the second sentence of Section 15(7) of Act No. 48/1997 Coll., "An appropriate therapeutic intervention shall mean healthcare services provided for the prevention or treatment of disease with the aim of achieving the most effective and safest treatment while maintaining cost-effectiveness." Thus, cost-effectiveness is one of the cumulative conditions of an appropriate therapeutic intervention within the meaning of Section 15(7).
However, the definition of an appropriate therapeutic intervention for a medicinal product intended for the treatment of a rare disease (hereinafter also referred to as "LPVO") with reimbursement granted under Section 39da differs from the definition applicable to a medicinal product in the so-called permanent reimbursement regime, i.e., with reimbursement from public health insurance granted in proceedings conducted under Section 39g or Section 39i of Act No. 48/1997 Coll. According to the third sentence of Section 15(7) of Act No. 48/1997 Coll., "In the case of medicinal products intended for the treatment of a rare disease under Section 39da, an appropriate therapeutic intervention shall mean healthcare services provided for the prevention or treatment of a rare disease with the aim of achieving the most effective and safest treatment, provided that the pharmacotherapeutic impact of this disease has a societal significance and the financial impact on the health insurance system (hereinafter the 'budget impact') is in accordance with the public interest pursuant to Section 17(2)."
This different approach to the requirement of appropriate therapeutic intervention in proceedings for the determination or modification of reimbursement for LPVOs under Section 39da as compared to medicinal products in the so-called permanent reimbursement regime is further expressed in Section 39da(3)(i) of Act No. 48/1997 Coll., which states that "In the proceedings for determining the amount and conditions of reimbursement of a medicinal product intended for the treatment of a rare disease, the following shall be assessed..., (i) cost-effectiveness analysis, however, without taking into account its result in the form of the incremental cost-effectiveness ratio..."
A medicinal product intended for the treatment of a rare disease for which reimbursement has been granted under Section 39da of the Public Health Insurance Act thus does not necessarily meet the condition of an appropriate therapeutic intervention as defined by the requirements of the law for medicinal products under the so-called permanent reimbursement regime. An LPVO reimbursed under Section 39da does not have to meet the condition of maintaining cost-effectiveness and therefore may not qualify as an appropriate therapeutic intervention within the meaning of the second sentence of Section 15(7) of the Act (although it does qualify under the third sentence of Section 15(7)). The anticipated inability of LPVOs to demonstrate their cost-effectiveness as required in proceedings under Section 39g is one of the reasons why LPVOs are granted a different procedure for determining reimbursement under Section 39da.
For the above reasons, when proving the appropriate therapeutic intervention of a medicinal product entering the so-called permanent reimbursement system under Section 39g, or in proceedings for modifying reimbursement under Section 39i of the Act, it is not possible to rely on the appropriate therapeutic intervention demonstrated by an LPVO that entered the reimbursement system under Section 39da. These are two different types of appropriate therapeutic intervention, requiring the demonstration of different circumstances and applicable in different types of proceedings. Therefore, it is not valid to equate them or assume that if an LPVO reimbursed under Section 39da meets the condition of appropriate therapeutic intervention pursuant to the third sentence of Section 15(7) of the Act, it also meets the condition pursuant to the second sentence of that same provision.
Using an LPVO reimbursed under Section 39da as a comparator in the base-case scenario of a cost-effectiveness analysis in proceedings for the determination or modification of reimbursement in the so-called permanent reimbursement regime would lead to methodological inconsistencies in the cost-effectiveness assessment submitted by the applicant and subsequently to inequalities in the Institute's assessment compared to other medicinal products in the permanent reimbursement regime.
Based on the above, it is clear that an assessed intervention in proceedings for the determination or modification of reimbursement in the so-called permanent reimbursement regime cannot be considered to meet the condition of appropriate therapeutic intervention if a medicinal product reimbursed under Section 39da is used as a comparator in the base-case scenario of the cost-effectiveness analysis.
The Ministry is therefore convinced that the use of a medicinal product intended for the treatment of a rare disease reimbursed under Section 39da as a comparator in the base-case scenario of the cost-effectiveness assessment in proceedings for the determination or modification of reimbursement in the so-called permanent reimbursement regime cannot in any way prove that the assessed intervention meets the condition of appropriate therapeutic intervention within the meaning of the second sentence of Section 15(7) of Act No. 48/1997 Coll. Failure to demonstrate appropriate therapeutic intervention under the second sentence of Section 15(7) of Act No. 48/1997 Coll. in reimbursement proceedings under the so-called permanent reimbursement regime constitutes a legal reason for denying reimbursement pursuant to Section 15(6)(d) of the same Act.
This is an unofficial translation generated with the assistance of ChatGPT-4o. It is provided for informational purposes only and does not constitute an official or legally binding translation.
The Ministry of Health of the Czech Republic has published its opinion on the use of a medicinal product for rare diseases (LPVO) with reimbursement granted under Section 39da as a comparator in...
As part of a
streamlined reimbursement review for cost-saving purposes (§ 39p(1)), the State Institute for Drug Control unified reimbursement conditions by removing the “A” symbol (indicating use in ambulatory
care) for products that can be self-administered by patients after prior
training.
The
Institute argued that if all products in a given group serve the same purpose
and can be administered by patients, there is no reason for them to be subject
to different reimbursement conditions. Previously, some products required immediate
administration by a physician (marked with the “A” symbol), while others—therapeutically
interchangeable—were not subject to such restriction.
The appeal
body agreed with this reasoning and described the previous arrangement as inefficient.
The
Institute had already reached the same conclusion in 2022, stating that if the
accompanying texts (SPC/PIL) allow for patient self-administration after proper
training instead of administration in ambulatory care, the “A” prescription
restriction (ZULP for ambulatory care) may be removed without requiring a
pharmacoeconomic assessment, as no expansion in the number of treated patients
occurs.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us. At Pharmeca, we help you navigate the complex landscape of
pharmaceutical and medical device information. We also offer flexible services
that can be tailored to your needs at any time. Our market position and experience allow us to support you whenever you
need expert guidance.
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 Ministry of
Health annulled the decision of the State Institute for Drug Control (SÚKL) on
the grounds that the Institute had failed to respect the Ministry’s previous
binding legal opinion, as set out in its earlier decision — which we reported
in our regular 2021 newsletter.
In 2021, the
Association of Health Insurance Companies (Svaz ZP) appealed against the
extension of reimbursement conditions for products containing inosine pranobex
as part of a comprehensive reimbursement review. The Institute had approved the
extension without assessing the cost-effectiveness and budget impact of the
proposed change.
The Ministry stated
that the removal of reimbursement conditions, such as prescription and
indication restrictions, constitutes an expansion of reimbursement that leads
to an increase in the number of treated patients. Following the change, all
medical specialists could prescribe these products for reimbursement from
public health insurance within the approved therapeutic indications listed in
the SPC. However, the Ministry emphasized that the prevalence of the relevant
conditions in the general Czech population was likely much higher than the
number of patients covered under the prior reimbursement restrictions. The
contested decision would thus logically lead to increased use and higher
reimbursement costs.
By extending
reimbursement without a proper assessment of cost-effectiveness and budget
impact, the Institute acted unlawfully.
In the further
course of the administrative proceedings, the Institute was not allowed to
change the reimbursement conditions in a way that would increase the number of
treated patients without the submission and assessment of the relevant
pharmacoeconomic analyses from the parties to the proceedings. However, after
the participants failed to provide the requested analyses, the Institute
performed its own calculations and again extended the reimbursement conditions.
This decision was again challenged — all payers filed an appeal.
Following further
review, the Ministry of Health reiterated that the Institute may not create its
own pharmacoeconomic analyses for the purpose of changing reimbursement
conditions in the absence of such analyses submitted by the parties. The
Institute is only authorized to perform corrective recalculations or similar
adjustments within the scope of analyses provided by the parties.
For these reasons,
the Ministry annulled the Institute’s decision once again.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us. At Pharmeca, we help you navigate the complex landscape of
pharmaceutical and medical device information. We also offer flexible services
that can be tailored to your needs at any time. Our market position and experience allow us to support you whenever you
need expert guidance.
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.
In its decision,
the Ministry of Health confirms the possibility of ensuring patients’
right to free (i.e., fully reimbursed) medicinal products from the groups
listed in Annex No. 2 to Act No. 48/1997 Coll., even through products
that are not available on the Czech market at the time of the SÚKL decision,
as long as it is certain that such products will be available in
sufficient quantitiesat the time the administrative decision becomes
enforceable.
This typically
applies to contractual arrangements between marketing authorization
holders and health insurance companies, which include a binding
commitment to ensure availability of the respective medicinal
products.
On the other hand, the
right to free medicinal products cannot be ensured through products that
are not present on the Czech market or are available only in
negligible quantities.
Furthermore,
according to the Ministry of Health of the Czech Republic, SÚKL is
not obliged to verify whether a product has been reported as temporarily
unavailable on the domestic market, since such a temporary
unavailability concerns only a transitional period, unlike permanent
discontinuation of supply.
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.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us.
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that can be tailored to your needs at any time.
Our market position and experience allow us to support you whenever you
need expert guidance.
According to the Ministry of Health
(MZ), even a single health insurance company can demonstrate that the presented
budget impact exceeds the current financial capacity of its fund, even if
this applies only to that specific insurer.
Rejecting
the statement of one insurance company solely because other insurers remain
silent or their statements are less convincing (or not sufficiently
substantiated) is, in the Ministry’s view, irrational. If the presented budget
impact poses an economic risk to even one health insurance company, this is
clearly a factor indicating the unacceptability of the proposed budget impact.
The
requirement by SÚKL for statements from all health insurance companies
regarding the acceptability of the budget impact has no legal basis, according
to the Ministry of Health.
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.
Are you interested in reading regular commentaries on decisions by
Pharmeca a.s.? Feel free to contact us.
At Pharmeca, we help you navigate the complex landscape of
pharmaceutical and medical device information. We also offer flexible services
that can be tailored to your needs at any time.
Our market position and experience allow us to support you whenever you
need expert guidance.
