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The Ministry of Health (MoH) has issued a binding opinion agreeing to the reimbursement of a medicinal product for rare diseases (LPVO) after the advisory board unanimously recommended approval.

Although the LPVO under assessment will be reimbursed, it will still be necessary to submit an application for an exceptional individual reimbursement under Section 16 of the Act on Public Health Insurance. This is because the LPVO is used in the given indication in combination with another medicinal product that is not covered by public health insurance. For this second product, an application for exceptional reimbursement will therefore need to be submitted, as stated by the Ministry in its opinion.

The Ministry agrees to the reimbursement terms particularly with regard to the age of the patients for whom the treatment is indicated, the seriousness of the disease, and after careful review of the evidence on the efficacy and safety of the LPVO in question.

Since the entry into force of the new legislation on pricing and reimbursement of rare disease medicines in 2023, the Ministry of Health has issued a total of 35 binding opinions. Of these, in 11 cases reimbursement was not approved, in 2 cases the Ministry proposed a change in the reimbursement conditions compared to the SUKL’s proposal, and in 1 case it proposed a change in the reimbursement amount.

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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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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.

The text was translated using ChatGPT 4o. 

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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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.

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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:

1. 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.
2. 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.
3. 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.
4. 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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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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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. 

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.

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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.

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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) 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 – Medicinal product containing olaparib: permanent reimbursement granted in SCAU
• 11/2024 – Ex officio proceeding initiated to reassess fulfilment of high innovativeness criteria

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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) 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.

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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.

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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 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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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.  

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.

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.

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.