Position of the Ministry of Health of the Czech Republic on Administrative Proceedings for Reimbursement Applications for Indications of Diseases Meeting the Definition of a Rare Disease, Involving Medicinal Products Not Designated as Orphan Medicines under Regulation (EC) No 141/2000
Act No. 48/1997 Coll., on Public Health Insurance and on Amendments and Supplements to Certain Related Acts, as amended (hereinafter referred to as the "Act"), does not regulate a special procedure for the entry into the reimbursement system for a medicinal product intended for the treatment of a rare disease that has not been granted so-called orphan designation under the relevant Regulation. Such a medicinal product cannot be classified as an orphan medicinal product (LPVO) under the Act, as the second sentence of Section 39da(1) stipulates: “An orphan medicinal product shall mean a medicinal product designated as such under directly applicable EU legislation on orphan medicinal products.” This means that a medicinal product not designated as an orphan under Regulation (EC) No 141/2000 cannot apply for reimbursement under Section 39da, even if it is authorized for a rare disease.
It is worth noting that submitting a reimbursement application under Section 39da is a right, not an obligation, of the marketing authorization holder. Entry into the reimbursement system is also possible via the procedures set out in Sections 39g or 39d of the Act.
Thus, medicinal products that cannot be considered LPVOs under the Act can only enter the reimbursement system via procedures under Sections 39g, 39i, or 39d. In proceedings under Sections 39g or 39i, resulting in permanent reimbursement of a non-orphan medicinal product for a rare disease indication, it is necessary, among other things, to meet the condition of a purposeful therapeutic intervention under the second sentence of Section 15(7) of the Act. However, during cost-effectiveness assessment in such procedures, it is not appropriate to use LPVOs reimbursed under Section 39da as comparators in the base-case scenario, as clarified in the Ministry's statement from 7 April 2025.
Therefore, the more suitable route for non-orphan designated products for rare diseases is through temporary reimbursement under Section 39d, as the features of this procedure are better suited to the nature of such medicinal products than the process for permanent reimbursement.
The first specific feature of the Section 39d procedure is the requirement to demonstrate at least one criterion for granting Highly Innovative Medicinal Product (VILP) status under Section 39d(2), which ensures that the product brings a degree of innovation that significantly impacts the treatment of a serious disease and plays a role in recommended treatment guidelines either in the Czech Republic or abroad. The applicant should specify in the submission, based on the principle of disposition, under which paragraph of Section 39d(2) the product fulfills the VILP criteria.
Fulfilment of at least one criterion for granting VILP status to a non-orphan designated medicinal product for which the first or second temporary reimbursement is requested in therapeutic indications for a condition meeting the definition of a rare disease is then assessed in comparison to therapies with reimbursement established under Section 39g of the Act, i.e., in a manner analogous to the procedure before 1 January 2022, when the amendment introducing the legal framework for the third reimbursement pathway under Section 39da for LPVOs entered into force. Given that the procedure under Section 39da is intended solely for a clearly defined group of LPVOs and applies specific requirements and criteria, where an LPVO reimbursed under Section 39da already exists in the reimbursement system at the time of the application for temporary reimbursement of a non-orphan medicinal product for a rare disease indication, the VILP criteria for such a product should not be assessed in comparison to the LPVO. This preserves the principle of assessing VILP criteria against therapies reimbursed under Section 39g, as is also the case when a temporary reimbursement is requested and a VILP is already reimbursed for the same indication. Otherwise, it would lead to an unjustified cumulative increase in entry requirements for newly entering medicinal products.
The relevant comparator for assessing the VILP criteria under Section 39d(2) and comparative effectiveness should thus be a reimbursed therapeutic intervention with reimbursement under Section 39g or Section 39i, or, if unavailable, another treatment alternative such as best supportive care (BSC). Nonetheless, even a reimbursed LPVO under Section 39da constitutes a therapeutic option, and thus in the clinical evaluation submitted by the applicant, a comparison with the LPVO in the given indication should be made. The entering VILP must meet the requirement of comparable or superior efficacy versus the LPVO in the assessed therapeutic indication—since setting reimbursement for a product with lower efficacy than a reimbursed LPVO cannot be considered in the public interest. Such a product would likely not bring patient benefit over the existing LPVO treatment. Regarding the evaluation of the VILP criterion for the non-orphan designated product, it is appropriate to note that where direct evidence is insufficient, indirect evidence or methodologically appropriate indirect comparisons may be accepted.
The second specific feature of the Section 39d procedure is the approach to assessing the purposefulness of therapeutic intervention, as under the third sentence of Section 39d(3) of the Act, “For temporary reimbursement, it is not required to fulfil the cost-effectiveness condition as one of the conditions for purposeful therapeutic intervention under Section 15(7).” This addresses cases where highly innovative medicines are not cost-effective due, for example, to a lack of sufficient data for a methodologically acceptable cost-effectiveness analysis, yet are reasonably expected to become cost-effective over time and clearly provide innovation.
Although failure to meet the cost-effectiveness condition under Section 15(7), second sentence, is not grounds to reject temporary reimbursement for a product eligible for VILP status, pharmacoeconomic evaluations—cost-effectiveness and budget impact analyses—must still be submitted. As per Section 15(8), fourth sentence, “The budget impact must align with the public interest under Section 17(2),” meaning an unacceptable budget impact may justify denial of temporary reimbursement.
Since the relevant clinical comparator for VILP assessment of a non-orphan medicinal product in a rare disease indication is therapy reimbursed under Section 39g or Section 39i or other alternatives, these should be used in the base-case scenario of the pharmacoeconomic assessment.
However, the alternative cost-effectiveness analysis scenario should include a comparator consisting of the LPVO reimbursed under Section 39da, provided that the analysis is methodologically sound and reviewable. Conversely, the LPVO must be used as the base-case comparator in the budget impact analysis under Section 39d(6), to ensure that estimated budget impacts reflect actual clinical practice and do not overestimate costs by omitting the LPVO as a comparator.
A product not designated as an orphan under Regulation (EC) No 141/2000 but intended for a rare disease indication may enter the reimbursement system under Section 39d if it demonstrates at least one VILP criterion compared to therapies reimbursed under Sections 39g or 39i and shows comparable efficacy to an LPVO reimbursed under Section 39da. The pharmacoeconomic base-case analysis should be performed using comparators reimbursed under Sections 39g or 39i, and the LPVO reimbursed under Section 39da must be included as a comparator in the base-case budget impact analysis and the alternative cost-effectiveness analysis.
This text was fully taken from the website of the Ministry of Health of the Czech Republic.
The conditions for
reimbursement of off-label indications are governed by Section 39b(3) of the
Act on Public Health Insurance. To establish reimbursement, it is necessary to
demonstrate that the use of the assessed medicinal product in the given off-label
indication is sufficiently justified by current scientific knowledge and that
its use represents the only available treatment option.
If it is not the only
treatment option, reimbursement can still be granted provided that the
applicant demonstrates cost-effectiveness compared to existing therapies.
If
the condition of sufficient scientific justification is not met during the
procedure, the Institute does not assess compliance with the remaining
conditions.
Sufficient scientific
knowledge is evaluated based on the evidence related to the specific
indication. Recently, the State Institute for Drug Control (SÚKL) reached
different conclusions on this condition for two indications of the same
product: the treatment of high-grade squamous intraepithelial lesions of the
vulva (HSIL) and extramammary Paget’s disease of the vulva (EMPD). Both
proposed indications, HSIL and EMPD, are off-label.
The Institute determined that,
based on available evidence, the use of the product in the treatment of vulvar
HSIL is sufficiently supported by current scientific knowledge. Additionally,
the product was found to be cost-effective, and therefore the reimbursement
conditions for this off-label indication can be established.
In contrast, for EMPD, the use of the product was deemed
insufficiently justified. The studies provided exhibited several methodological
limitations, such as small patient samples, lack of a control arm, and
observational design. As a result, the Institute proposes not to grant
reimbursement for the EMPD indication.
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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.
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In the course of the administrative
proceedings, the appellant argued that the State Institute for Drug Control
(SÚKL) failed to proceed in accordance with Section 39c(5) of the Act on Public
Health Insurance by not increasing the reimbursement of products containing the
selected active substance to ensure full reimbursement.
This provision stipulates that if, during
the process of determining reimbursement, no fully reimbursed medicinal product
is available in any of the substance groups listed in Annex No. 2—regardless of
therapeutic interchangeability—SÚKL shall adjust the reimbursement so that the
least costly product from the assessed group is fully reimbursed.
The appellant maintained that under the provision in question, full reimbursement must be increased within the group of interchangeable products. According to the appellant, it is not sufficient that a fully reimbursed product exists within the same Annex No. 2 group if it is not therapeutically interchangeable and cannot be used in patients requiring the evaluated active substance under the established reimbursement conditions. Therefore, the appellant argued that the requirement for full reimbursement of at least one product under Section 39c(5) of the Act had not been met.
The Ministry of Health (MoH) responded by stating that the assessed active substances fall under the cytostatic tyrosine kinase inhibitors group—Group No. 116 of Annex No. 2. The medicinal products under review, as well as the fully reimbursed product within the same group, are used and reimbursed for the same disease type—chronic myeloid leukemia (CML). The Ministry noted that the condition of ensuring full reimbursement for at least one product within Annex No. 2 must be assessed in relation to the reference indication (i.e., the broader patient population), not to a specific subpopulation of CML patients. For these reasons, the MoH confirmed that SÚKL had acted correctly, as a reimbursed product was ensured for patients with CML within Group 116 of Annex No. 2 of the Act on Public Health Insurance.
The issue of fully reimbursed medicinal
products under Annex No. 2 has also been addressed by the Constitutional Court
of the Czech Republic. A landmark ruling concerned the full reimbursement of
therapy for prostate and breast cancer.
In decision ref. no. III. ÚS 2332/16, the
Court focused on whether it is constitutionally acceptable for no fully
reimbursed product to be available for patients with prostate cancer, while
such a product exists for breast cancer—even though the relevant products are
categorized in the same Annex No. 2 group under the Act on Public Health
Insurance.
The Constitutional Court held that
interpreting the law in a way that denies full reimbursement of a medicinal
product simply because another fully reimbursed product exists for a different
disease (in this case, breast cancer) undermines the essence of the
constitutionally guaranteed right to health protection under Article 31 of the
Charter of Fundamental Rights and Freedoms. The Court emphasized that the
administrative courts had acted too formalistically and failed to consider the
merits of the claimant’s argument that no fully reimbursed treatment existed
for prostate cancer.
As a result, it followed that if products
contain active substances listed in the same Annex No. 2 group but are intended
to treat categorically different diseases (e.g., one for breast cancer and
another for prostate cancer), then full reimbursement must be ensured not only
within the Annex No. 2 group but also specifically for each individual disease
(i.e., for both breast and prostate cancer).
In a recent appeal review concerning the
expedited reassessment of a reference group, the Ministry interpreted the
Constitutional Court’s ruling to mean that if full reimbursement is ensured for
a product from Annex No. 2 for the treatment of a specific disease type, and
other products from the same group are indicated for entirely different
diseases, then it is not necessary to ensure full reimbursement for those other
products—provided that treatment for the different disease type is already
fully reimbursed by a product outside the same Annex No. 2 group. No court has
yet ruled on this interpretation.
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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.
In an administrative procedure conducted by the State Institute
for Drug Control (SÚKL), objections raised by payers regarding
cost-effectiveness were not upheld. The payers argued that the evaluated
therapy could not be considered cost-effective due to the existence of
cost-reducing agreements concluded between insurers and the marketing
authorization holders of comparably effective medicinal products.
SÚKL, in its first-instance decision, did not take these
cost-limiting agreements into account, reasoning that the agreements were not
decisive for the reimbursement of the comparably effective therapies, which
were cost-effective even without such arrangements.
The correctness of this approach was confirmed by the Ministry of
Health (MoH) during the appeals process, stating that administrative
proceedings must be based on verifiable data. SÚKL cannot rely on claimed cost
levels of comparably effective therapies if such data is not available to it.
Therefore, SÚKL rightly based its assessment on publicly available
reimbursement data listed in the Price and Reimbursement List.
Following this MoH decision, another ministerial ruling was issued
concerning the reimbursement of a product included in the same reimbursement
group as in the aforementioned case.
Again, SÚKL justified its decision not to consider the existence
of agreements in setting the reimbursement for the comparably effective therapy
in a similar manner to the previous case.
The MoH confirmed this approach as well, reasoning that essential
parts of the agreements—specifically the agreed manufacturer’s maximum
price—were confidential. As such, these agreements could not be taken into
consideration during the procedure.
The Ministry of Health’s reasoning may influence SÚKL’s approach
in cases involving confidential agreements affecting the reimbursement of a
comparator/reference product/comparably effective therapy.
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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.
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 full summary report is available on the Ministry of Health’s website.
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:
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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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.
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.
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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.
Our knowledge, your opportunity.
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.
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.
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Our knowledge, your opportunity.
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.