The Advisory Board for the Reimbursement of Medicinal
Products Intended for the Treatment of Rare Diseases at the Ministry of Health
of the Czech Republic has published a summary report evaluating its application
practice from May 2022 to March 2025.
In addition to listing the legal requirements related to
orphan drugs and a comprehensive overview of all proceedings within the
reviewed period, one chapter focuses on the key lessons learned from the
advisory board's experience.
The Advisory Board for the Reimbursement of Medicinal Products Intended for the Treatment of Rare Diseases at the Ministry of Health of the Czech Republic has published a summary report evaluating...
The Ministry
of Health of the Czech Republic (MoH) has issued a decision confirming the
classification of medicinal products containing gliflozins as a group of
essentially therapeutically interchangeable medicinal products for the
treatment of type 2 diabetes mellitus (T2DM). This decision upheld the original
contested ruling of the State Institute for Drug Control (SÚKL).
Gliflozins
are used in the treatment of T2DM, contributing to improved glycaemic control
and offering additional benefits for comorbidities such as heart failure and
chronic kidney disease. The proceeding evaluated medicinal products containing
the following active substances from the group of sodium-glucose co-transporter
2 (SGLT2) inhibitors: dapagliflozin, empagliflozin, and ertugliflozin.
The core
question was whether gliflozin-containing products can be deemed essentially
therapeutically interchangeable within the reference indication "treatment
of type 2 diabetes mellitus." In its
objections to the therapeutic interchangeability of dapagliflozin-containing
products, the appellant presented the following key arguments:
Different
Approved Indications – The company claimed that dapagliflozin has
broader approved indications (e.g., heart failure with reduced and preserved
ejection fraction) that are not approved for all other gliflozins.
Differences
in Efficacy and Safety – The argument was based on variations in
clinical study outcomes and differences in dapagliflozin’s safety profile
compared to other gliflozins.
Varying
Availability of Clinical Data – More robust clinical evidence is available
for dapagliflozin in multiple indications, whereas for other gliflozins data is
either incomplete or lacking.
Irreplaceability
in Specific Indications – In clinical practice, dapagliflozin is
irreplaceable in certain indications due to the lack of alternative treatments
with comparable efficacy and safety evidence.
Despite the
objections, the MoH confirmed that SÚKL correctly assessed gliflozins as
essentially therapeutically interchangeable based on similar efficacy, safety,
and clinical use in the reference indication. The rationale was that while
individual gliflozins may offer different benefits for specific patient
subpopulations, their overall effects in T2DM treatment are comparable.
The MoH also
confirmed that classification into a group of essentially therapeutically
interchangeable medicines is not based on market share or prevalence of use,
but rather on expert considerations such as efficacy, safety, and clinical
application. Therefore, the limited use of one substance in T2DM therapy was
not a valid reason to exclude it from the group.
The MoH
cited a judgment of the Prague Municipal Court dated 22 May 2015, Case No. 5 Ad
11/2010-164, which concluded that terms such as "essentially
therapeutically interchangeable," "similar or close efficacy and
safety," and "similar clinical use" under Section 39c(1) of Act
No. 48/1997 Coll. should not be interpreted narrowly. The law deliberately uses
modifiers such as "similar," "close," and
"essentially" to indicate that products do not need to be completely
identical in all aspects, including safety profiles. This
interpretation was subsequently reaffirmed by the same court in its judgment
dated 23 September 2015, Case No. 7 Ad 19/2011-98. Further support comes from
the Supreme Administrative Court’s ruling (Case No. 2 As 388/2017–66, para.
24), which stated: "There is no reason to equate the terms 'essentially
therapeutically interchangeable with similar or close efficacy and safety and
similar clinical use' for reimbursement purposes with 'interchangeability' of
medicinal products for healthcare provision purposes. The objectives of these
administrative proceedings differ, and the criteria considered cannot be
compared with those evaluated by physicians in selecting appropriate therapy
(...)."
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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) assessed the
objections raised by the Health Insurance Association (“the Association”) and
the General Health Insurance Company (VZP) concerning the allegedly
unacceptable budget impact of the evaluated treatment on the public health
insurance system.
The Association’s calculations were based on a comparison of
per-patient treatment costs – CZK 1,509,429 for the evaluated intervention
versus CZK 151,503 for the reimbursed comparator, representing nearly a tenfold
increase. However, SÚKL concluded that this merely reflects a multiple increase
in unit costs, not the total budget impact. According to SÚKL, such an argument
does not sufficiently reflect the capacity or planning of the public health
insurance system.
Both the Association’s and VZP’s calculations were based on
internal data from the years 2017–2023 (Association) and 2014–2023 (VZP), and
in both cases the projected budget impacts were compared against figures from
the first (older) version of the assessment report. SÚKL considered the projected cost increase presented by the
insurers to be irrelevant, as the conclusions were based on outdated data.
Current costs are lower due to updated external reference pricing and
cost-capping agreements submitted by the applicant in cooperation with health
insurance companies. Therefore, SÚKL did not accept the insurers’ conclusions
as valid for the present decision-making process.
What Should a Statement on Unacceptable Budget Impact Look Like?
In an updated
2023 article, SÚKL based on a decision by the Ministry of Health outlined new
requirements regarding how health insurance funds should submit statements
concerning the unacceptability of budget impact.
SÚKL emphasized, among other things, that it is not acceptable to
apply a universal threshold for all therapies that would automatically indicate
the boundary between acceptable and unacceptable budget impact. Furthermore, it stated that budget impact assessments must not be
limited to pharmaceutical costs alone, but must reflect the overall budget
impact, including other non-pharmaceutical segments of healthcare provision.
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.
At the end of 2024,
the City Court issued a ruling concerning the classification of a
medicinal product as the first similar product or generic. The court reviewed
several claims raised against decisions of the Ministry of Health (MoH) and the
State Institute for Drug Control (SÚKL).
A similar product
is, in simplified terms, a medicinal product with the same active substance(s)
and an identical or similar pharmaceutical form as the reimbursed product
(similarly applicable to biological products).
The court addressed
the question of whether an application could be considered first in line, even
if another product had already been granted reimbursement under legislation
valid until 31 December 2007.
The court held that the definition of a “first similar product” does not
require the application to have been submitted after the effective date of the
amendment to the Public Health Insurance Act.
Furthermore, the
court assessed whether an application for reimbursement modification is
equivalent to an application for reimbursement determination.
In the case of a first similar product, the applicant is required to submit a commitment
to market the product. However, this obligation does not apply to applications
for reimbursement of the second or subsequent similar products.
SÚKL had interpreted the law in such a way that, for determining the order of
applications (i.e. whether the product is the first or not), only the existence
of a previous reimbursement determination application for a similar product was
relevant, while applications for modification of reimbursement were not
considered. The court
disagreed, accepting the plaintiff’s argument that both types of applications
(determination and modification) are materially equivalent and should be taken
into account when assessing the order. Thus, if an application for reimbursement modification for a similar product
was submitted before the application for reimbursement determination, the
latter cannot be considered an application for the first similar product, and
therefore the marketing commitment does not need to be submitted.
The court also
considered whether the medicinal product in question could be regarded as a generic.
The court stated that in order to deny reimbursement due to non-submission of
the marketing commitment for 12 months, it is not sufficient for the product to
be the first similar product—the law also requires that the product must be
registered as either a biological medicinal product or a generic. Since the product under review was not registered as a generic, it cannot be
considered one.
Following the City
Court’s ruling, SÚKL re-evaluated the application for the maximum price and
reimbursement determination of the medicinal product in question and did not
classify it as the first similar product. Instead, it recognized another
product as the first similar one.
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.
A medicinal product
intended for patients with acute myeloid leukaemia (AML) previously held a
temporary reimbursement as a Highly Innovative Medicinal Product (VILP) under
Section 39d, granted for patients with FLT3-mutated AML in first relapse,
reimbursed until disease progression, transplantation, or unacceptable
toxicity.
In a new
application, the same product was submitted for permanent reimbursement as an
orphan drug under Section 39da, this time proposing reimbursement for a
broader population than previously approved. The request aimed to extend
reimbursement to post-transplant AML patients and to remove the first relapse
condition.
The Ministry of
Health (MoH) agreed with SÚKL’s conclusion that the application must be
rejected due to the overlap in indications with the original temporary
reimbursement. According to Section 39da of the Public Health Insurance Act, it is not legally
permissible to apply for reimbursement for an indication already covered by a
previous reimbursement decision. Although the
relevant medical society opposed SÚKL’s decision, citing concerns about
potential negative impacts on patient care and treatment quality, the
Ministry upheld the rejection, stating that rules are consistent and the
perceived medical need for the drug cannot override legal requirements. The appeal was
dismissed by the Ministry of Health, and SÚKL’s original decision was
confirmed.
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 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
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 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.
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 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.
