BACKGROUND: Performance-based risk-sharing agreements (PBRSA) represent an innovative tool for managing uncertainty and balanced distribution of the financial risk of high-cost drugs. By linking reimbursement to real-world treatment performance, these agreements help mitigate budgetary impacts. This study poses an illustrative patient-level PBRSA reimbursement model for non-small cell lung cancer (NSCLC) immunotherapy based on collected real-world data (RWD). METHODS: A retrospective analysis of 266 patients with NSCLC treated with immunotherapy was performed. Progression-free survival (PFS) served as the primary outcome measure of therapeutic effectiveness. An illustrative patient-level PBRSA model was developed to quantify the manufacturer’s financial participation based on deviations from established PFS thresholds reported in randomised controlled trials (RCT). The manufacturer’s financial responsibility increased proportionally to greater deviations in patient outcomes from the RCT benchmark. Cost calculations were limited exclusively to the acquisition price of immunotherapies, excluding administration, toxicity management, and other indirect costs. The potential PBRSA scenario was compared with the current reimbursement situation. RESULTS: Using this reimbursement method, cost savings per checkpoint inhibitor for healthcare payers could represent between 27.3% and 66.2% of the total cost, depending on the individual PFS reached. For the RWD cohort of NSCLC patients unsuccessfully treated with pembrolizumab monotherapy was 57.5% (a reduction in cost to payers from $27 996 to $11 893 per patient); pembrolizumab in combination 51.7% ($33 595 to $16 237); nivolumab 37.1% ($5 608 to $3 531); atezolizumab 27.3% ($11 799 to $8 583); and durvalumab 66.2% ($44 005 to $14 882). CONCLUSIONS: This study proposes an illustrative patient-level PBRSA reimbursement model leveraging real-world clinical data to enhance risk-sharing for high-cost therapies. Unlike conventional cost-effectiveness analyses, this method directly links clinical performance and manufacturer financial responsibility. Future research should integrate comprehensive cost considerations and validate model performance in broader clinical settings. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13561-025-00646-3.

• Publikační typ
• časopisecké články MeSH

BACKGROUND: Advanced Therapy Medicinal Products (ATMPs) represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Although authorised centrally in the European Union, their market launch differs across Member States (MS). This study aimed to describe the ATMP market availability in MS and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies. METHODS: ATMP availability was defined as the product launch in each MS. Data was collected through open governmental sources, databases, and communication with national competent authorities. Spearman's correlation coefficients were calculated to examine the relationship between ATMP availability and their characteristics (time since granting marketing authorisation, target patient population size, and cost). RESULTS: We collected the availability data on 18 ATMPs from 23 EU MS. Market uptake varied significantly, with Germany (89%), France and Italy (61%) leading. Estonia and Latvia confirmed that no ATMP has been launched on their markets yet. Six ATMPs were available in more than one-third of the analysed MS. No significant correlation was observed between ATMP availability and analysed product characteristics except for time dependency for CAR T-cell therapies. CONCLUSION: Beyond pricing and reimbursement processes, the ATMP commercialisation in particular MS is influenced by the marketing authorisation holder's decision and capacity. ATMPs face product-specific challenges in achieving EU-wide availability, including complex manufacturing, distribution, and administration processes. To increase the accessibility of innovative ATMP-based treatments, implementing the cross-border access framework or individual ATMP production under the hospital exemption is essential, especially in underserved MS.

• MeSH
• dávkové mechanismy * MeSH
• Evropská unie MeSH
• vzácné nemoci terapie   MeSH
• Publikační typ
• časopisecké články MeSH

Managed Entry Agreements (MEAs) play a pivotal role in addressing the challenges arising from escalating prices of innovative medical technologies, especially in areas like oncology, immunology, and rare diseases. Among MEAs, Performance-Based MEAs (PB MEAs) and Outcome-Based MEAs (OB MEAs) stand out as innovative strategies. This study examines the adoption of PB MEAs in the Czech Republic post a 2022 legislative change. Interviews with key stakeholders, including the Ministry of Health, pharmaceutical companies, insurers, and patient groups, were conducted to explore perceptions and challenges. Stakeholders expressed concerns about legislation completeness, data quality, transparency, and methodology. Interestingly, pharmaceutical companies were less concerned about transparency and methodology, likely due to their multinational experience. Despite legislative progress, challenges persist, especially in data infrastructure, risk-sharing perceptions, and stakeholder readiness. Addressing these issues requires collaboration between pharmaceutical companies and payers. Patient involvement, though mandated, remains limited, potentially due to a lack of awareness. This study emphasizes the need for a comprehensive transformation beyond legislation for a successful PB MEA implementation. Trust, technical infrastructure, and data availability are crucial, necessitating a holistic approach. It contributes to the global discourse on PB MEAs, stressing the adjustment of financial frameworks, embracing value-based healthcare principles, and ensuring high-quality health data metrics. A more holistic, value-based MEA approach could reshape pharmaceutical reimbursement in the future.

• Publikační typ
• časopisecké články MeSH

OBJECTIVES: We have developed a scientifically well-grounded, methodological, and reporting checklist for economic evaluation (EE) of medicines in the Slovak health technology assessment process, which serves as a supplement to the Slovak pharmacoeconomic guidelines. METHODS: The checklist was developed using an iterative process in which items were generated and gradually added to the baseline checklist based on shortcomings identified in an analysis of Slovak EEs, using relevant published checklists, and Slovak, as well as international, methodological guidance that was identified in the systematic literature review. The selection of checklist recommendations, their clarity, and relevance to the Slovak setting were validated in the online survey. RESULTS: From the sample of 151 price and reimbursement submissions published between January 2018 and July 2021, almost half of them (n = 73) received at least 1 request from the Ministry of Healthcare to justify or modify the methodology used in the EE; and in 18 proceedings, a negative opinion was issued because of shortcomings identified in the EE. The 25-items preliminary checklist, resulting from an iterative working process, has been validated in an online survey conducted among members of ISPOR Chapter Slovakia. After incorporating relevant comments, the final proposal for the Slovak checklist consists of 55 recommendations. CONCLUSIONS: The research represented the first attempt to create a Slovak EE checklist, which serves as a part of ISPOR Slovakia pharmacoeconomic guidelines. Implementation of the checklist allows checking whether EE meets legislative and methodological requirements and thus helps in improving the appropriateness and standardization of EEs in Slovakia.

• MeSH
• ekonomika farmaceutická * MeSH
• kontrolní seznam * MeSH
• lidé MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• systematický přehled MeSH
• Geografické názvy
• Slovenská republika MeSH

BACKGROUND: The Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) was established in 2013 with the intention of developing a coordinated mechanism between volunteering EU stakeholders and developers of Orphan Medicinal Products (OMPs) to support the exchange of information aimed at enabling informed decisions on pricing and reimbursement at Member State level and to evaluate the value of an OMP based on a Transparent Value Framework. The objective of the collaborative approach was to support more equitable access to authorised therapies for people living with rare diseases, rational prices for payers and more predictable market conditions for OMP developers. Over the past 10 years, the MoCA has conducted a series of pilot projects, examining a variety of different products and technologies at different stages of development; and with contributions from a variety of patient representatives, participation from EU payers from a range of Member States and, recently, with EUnetHTA members and the European Medicines Agency participating in the meetings as observers. RESULTS: 10 years on from the establishment of the MoCA, the European landscape has significantly evolved, not only in the field of drug development with increasingly transformative therapies based on novel technologies, but also in terms of larger numbers of approved treatments, increased budget impact and the resulting associated uncertainties; as well as in terms of stakeholder collaboration and interactions. The value of early dialogue with OMP developers, including the EU payer community via their national decision-making authorities, is a key element within this early interaction and contributes to identifying, managing and reducing uncertainties allowing a prospectively planned approach earlier in development and, consequently, to support more timely, sustainable and equitable access to new OMPs, particularly where there is a high unmet medical need. CONCLUSIONS: The voluntary, informal nature of the MoCA interactions creates a flexible framework for non-binding dialogue. A forum for such interactions is needed to achieve the aims of the MoCA and both to support healthcare systems in planning as well as to underpin timely, equitable and sustainable access to new therapies for patients with rare diseases within the EU.

• MeSH
• lidé MeSH
• rozpočty * MeSH
• vyvíjení léků MeSH
• vzácné nemoci * MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• práce podpořená grantem MeSH
• Geografické názvy
• Evropa MeSH

BACKGROUND: Out of 185 orphan medicinal products (OMPs) registered in 2015-2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. METHODOLOGY: The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients' organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. CONCLUSION: We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of "value-based" decision making.

• MeSH
• hodnocení biomedicínských technologií MeSH
• lidé MeSH
• rozhodování MeSH
• úhrada zdravotního pojištění MeSH
• výroba orphan drugs * MeSH
• vzácné nemoci * farmakoterapie   MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Česká republika MeSH

OBJECTIVES: The comparative efficacy between riociguat and selexipag in patients with pulmonary arterial hypertension (PAH) has never been described in literature. Our aim was to prepare indirect treatment comparison (ITC) to evaluate the cost-effectiveness of riociguat in Czechia. METHODS: A systematic literature review identified two relevant trials with comparable endpoints to inform a Bucher ITC of relative and absolute effects. Given the comparable efficacy of riociguat and selexipag, a cost-minimization analysis (CMA) was conducted. RESULTS: A Bucher ITC provided evidence for the comparable relative efficacy of riociguat defined as the odds of unimproved functional class III 0.761 (95% CI 0.372 to 1.558; p = 0.455) compared to selexipag and a comparable absolute efficacy defined as a difference in the 6-minute walking distance of 10.560 meters (95% CI -10.692 to 31.812; p = 0.330). The CMA identified riociguat as the cost-saving therapy. CONCLUSIONS: Switching to riociguat represents the cost-saving therapy for PAH patients who were inadequately compensated with the PDE5i+ERA therapy. Consequently, riociguat has been introduced to the list of reimbursed medicines in Czechia from October 2021. Based on two global trials, we prepared the first indirect treatment comparison followed with CMA of these therapies that may improve future decision-making for PAH indications.

• MeSH
• antihypertenziva MeSH
• lidé MeSH
• náklady a analýza nákladů MeSH
• plicní arteriální hypertenze * MeSH
• plicní hypertenze * farmakoterapie   MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• systematický přehled MeSH

Při zahájení intenzivní léčby před uzavřením zánětu za hematoencefalickou bariérou je potenciál ovlivnění roztroušené sklerózy největší. Časné nasazení vysoce účinné terapie se tak jeví u většiny pacientů nejlepší terapeutickou strategií, a díky změně úhradových kritérií je možno ji volit již i v České republice. U minoritní skupiny pacientů s mírnou formou nemoci však není důvod podstupovat bezpečnostní riziko a vhodnou volbou je strategie eskalační. Volit lze na základě prognostických markerů, ty ale nefungují stoprocentně a cenou za nedostatečně intenzivní léčbu je nevratné poškození nervových struktur. Klíčová je proto monitorace a přehodnocování strategie s ohledem na její efektivitu i nežádoucí účinky.

When intensive treatment is initiated before the inflammation behind the blood-brain barrier is closed, the potential to affect multiple sclerosis is the greatest. Thus, early initiation of high efficacy therapy appears to be the best therapeutic strategy for most patients, and thanks to the change in reimbursement criteria, this strategy can be chosen in the Czech Republic too. However, in a minority of patients with mild forms of the disease, there is no reason to take a safety risk and the appropriate choice is an escalation strategy. The choice can be made based on prognostic markers, but these are not perfect and the price for insufficiently intensive treatment is irreversible damage to nerve structures. Therefore, monitoring and re-evaluation of the strategy regarding its effectiveness and adverse effects is the key.

• Klíčová slova
• okrelizumab,
• MeSH
• humanizované monoklonální protilátky ekonomika   farmakologie   terapeutické užití   MeSH
• lidé MeSH
• náklady na léky MeSH
• roztroušená skleróza * farmakoterapie   MeSH
• zdravotní pojištění MeSH
• Check Tag
• lidé MeSH

Úvod: Psoriáza (lupénka) patří mezi chronická onemocnění s řadou významných komorbidit a často s velmi výrazným dopadem na kvalitu života pacientů. V poslední době vstoupilo na trh poměrně velké množství přípravků biologické léčby s výrazným účinkem, oproti systémové konvenční léčbě jsou ale také výrazně dražší. V kontextu publikovaného dlouhého období nedostatečné léčby pacientů s psoriázou v ČR, systému zdravotní péče a limitovaných nákladů centrových léčiv je nutno již na úrovni poskytovatele této zdravotní péče vynaložit co nejvyšší úsilí efektivního vynaložení finančních prostředků a zprostředkování vysoce účinné léčby co nejvyššímu počtu pacientů. Metoda: Při modelově nastavené výši jednotkových cen balení přípravků dle maximálních úhrad v ČR v roce 2021, fiktivně nastavené výši bonusů jen u dvou vybraných přípravků (jedná se o modelový příklad neodrážející reálný stav obchodní politiky ve FN Olomouc) a podmínek úhrad přípravků biologické léčby psoriázy v ČR v roce 2021 byla provedena analýza nákladové efektivity (CEA) ve vybraném parametru účinnosti PASI 100. Analýza byla provedena a zpracována interaktivním způsobem v softwaru Excel do grafické podoby výstupů zejména v podobě forest plot grafů (v parametrech CER a ICER) a ve dvourozměrném grafu (náklady vs. účinnost) s vyznačením linií efektivnosti. Výsledky: Rozdíly v nákladové efektivitě jednotlivých přípravků dle bodových hodnot parametrů CER a ICER jsou až několikanásobné. Při zohlednění 95% intervalů spolehlivosti jsou některé rozdíly mezi přípravky i statisticky významné. Při nákladově nejefektivnější posloupnosti léčby na postupně účinnější přípravek u jednoho pacienta během indukční doby léčby můžeme na základě takto ušetřených nákladů nově zahájit indukční léčbu méně účinným biologikem u dvou pacientů navíc než při použití v pořadí druhé nákladově efektivní posloupnosti eskalace léčby. Závěr: Analýza nákladové efektivity (CEA) biologických léčiv v léčbě psoriázy na úrovni poskytovatele zdravotních služeb má velký potenciál při úsilí maximálně zefektivnit léčbu při limitovaných finančních prostředcích či při úsilí zprostředkovat při stejných limitacích účinnou léčbu dalším pacientům navíc.

Introduction: Psoriasis is a chronic disease with a number of significant comorbidities, often having a very substantial impact on the quality of life of patients. Recently, a relatively high number of biological therapy agents have been introduced to the market; however, compared to systemic conventional treatment, they are much more expensive. In the context of a reported long period of inadequate treatment of psoriasis patients in the Czech Republic, system of health care, and limited costs for medicines used in specialized centres, every effort must be made at the very level of the health care provider to spend financial resources effectively and provide highly effective treatment to as many patients as possible. Method: With a model-based amount of unit prices of product packages according to the maximum payments in the Czech Republic in 2021, a fictitiously set amount of bonuses in two selected products only (a model example not reflecting the reality of trade policy in the Olomouc University Hospital), and conditions for reimbursement of biological agents for psoriasis treatment in the Czech Republic in 2021, a cost-effectiveness analysis (CEA) was performed for the selected efficacy parameter of PASI 100. The analysis was performed and processed in an interactive way in Excel software into a graphical form, particularly in the form of forest plot graphs (in CER and ICER parameters) and a two-dimensional graph (cost vs. effectiveness) with effectiveness lines being indicated. Results: There are as much as several-fold differences in the cost-effectiveness of individual products according to the CER and ICER parameter values. When taking into account 95% confidence intervals, some differences between the products are even statistically significant. In the case of the most cost-effective sequence of treatment to a gradually more effective drug in a single patient during the induction phase of treatment, induction treatment with a less effective biological drug can be newly initiated, based on such cost savings, in two more patients than when using the second most cost-effective sequence of treatment escalation. Conclusion: A cost-effectiveness analysis (CEA) of biological drugs for the treatment of psoriasis at the level of a health service provider has great potential in the effort to maximize the effectiveness of treatment with limited financial resources or in the effort to provide, with the same limitations, effective treatment to additional patients.

• MeSH
• biologická terapie ekonomika   MeSH
• biologické přípravky ekonomika   terapeutické užití   MeSH
• hodnocení léčiv MeSH
• lidé MeSH
• management nemoci MeSH
• náklady a analýza nákladů MeSH
• náklady na zdravotní péči * MeSH
• poskytování zdravotní péče ekonomika   MeSH
• psoriáza * ekonomika   farmakoterapie   patologie   MeSH
• Check Tag
• lidé MeSH

BACKGROUND: A core evaluation framework that captures the health care and societal benefits of value added medicines (VAMs, also often called repurposed medicines) was proposed in Report 1, aiming to reduce the heterogeneity in value assessment processes across countries and to create incentives for manufacturers to invest into incremental innovation. However, this can be impactful only if the framework can be adapted to heterogeneous health care financing systems in different jurisdictions, and the cost of evidence generation necessitated by the framework takes into account the anticipated benefits for the health care system and rewards for the developers. AREAS COVERED: The framework could potentially improve the pricing and reimbursement decisions of VAMs by adapting it to different country specific decision-contexts such as deliberative processes, augmented cost-effectiveness frameworks or formal multi-criteria decision analysis (MCDA); alternatively, some of its domains may be added to current general evaluation frameworks of medicines. The proposed evaluation framework may provide a starting point for practices based on which VAMs can be exempted from generic pricing mechanisms or can be integrated into the reimbursement and procurement system, allowing for price differentiation according to their added value. Besides evidence from RCTs, pricing and reimbursement decision processes of VAMs should allow for ex-ante non-RCT evidence for certain domains. Alternatively, relying on ex-post evidence agreements-such as outcome guarantee or coverage with evidence development-can also reduce decision uncertainty. CONCLUSIONS: The core evaluation framework for VAMs could trigger changes in the existing pricing, reimbursement and procurement practices by improving the appraisal of the added value created by incremental innovation.

• Publikační typ
• časopisecké články MeSH
• přehledy MeSH