Lomustine with or without reirradiation for first progression of glioblastoma, LEGATO, EORTC-2227-BTG: study protocol for a randomized phase III study.

BACKGROUND: Chemotherapy with lomustine is widely considered as standard treatment option for progressive glioblastoma. The value of adding radiotherapy to second-line chemotherapy is not known. METHODS: EORTC-2227-BTG (LEGATO, NCT05904119) is an investigator-initiated, pragmatic (PRECIS-2 score: 34 out of 45), randomized, multicenter phase III trial in patients with first progression of glioblastoma. A total of 411 patients will be randomized in a 1:1 ratio to lomustine (110 mg/m2 every 6 weeks) or lomustine (110 mg/m2 every 6weeks) plus radiotherapy (35 Gy in 10 fractions). Main eligibility criteria include histologic confirmation of glioblastoma, isocitrate dehydrogenase gene (IDH) wild-type per WHO 2021 classification, first progression at least 6 months after the end of prior radiotherapy, radiologically measurable disease according to RANO criteria with a maximum tumor diameter of 5 cm, and WHO performance status of 0-2. The primary efficacy endpoint is overall survival (OS) and secondary endpoints include progression-free survival, response rate, neurocognitive function, health-related quality of life, and health economic parameters. LEGATO is funded by the European Union's Horizon Europe Research program, was activated in March 2024 and will enroll patients in 43 sites in 11 countries across Europe with study completion projected in 2028. DISCUSSION: EORTC-2227-BTG (LEGATO) is a publicly funded pragmatic phase III trial designed to clarify the efficacy of adding reirradiation to chemotherapy with lomustine for the treatment of patients with first progression of glioblastoma. TRIAL REGISTRATION: ClinicalTrials.gov NCT05904119. Registered before start of inclusion, 23 May 2023.

Roadmap to improve the organized cancer screening programs - The case of colorectal cancer screening in Montenegro.

BACKGROUND: Implementation of organized cancer screening programs comes with many challenges and barriers, which may inhibit the achievement of the screening activities' desired benefits. In this paper we outline a plan for improving the colorectal cancer (CRC) screening system in Montenegro. METHODS: We formulated a roadmap, which was generally defined as a country-specific strategic plan to improve cancer screening programs. The roadmap development was an iterative, step-by-step process. First, we described the current screening program, then identified and described key barriers, and finally proposed actions to overcome them. Multiple sources of information (e.g., documents, expert opinions) were collected and processed by local and international stakeholders. RESULTS: The CRC screening program was implemented between 2013-2019 by gradually increasing the invitation of the target population. Key barriers of the implementation were defined: 1) Lack of colonoscopy capacity in the northern part of the country; 2) Inadequate information technology systems; 3) Inadequate public promotion of screening. The defined actions were related to overcoming lack of available resources (e.g., financial, human and technological), to improve the policy environment and the knowledge, and to facilitate information sharing. CONCLUSION: The collaboration between local stakeholders of CRC screening and researchers experienced in planning and evaluating screening programs resulted in the first comprehensive description of CRC screening in Montenegro, detailed understanding of key barriers that emerged during implementation and a carefully designed list of actions. The implementation of these actions and the evaluation of whether barriers were solved will be captured in the upcoming period by maintaining this collaboration.

Systematic Literature Review of Economic Evaluations of Treatment Alternatives in Chronic Lymphocytic Leukemia.

BACKGROUND: Economic evaluations are widely used to predict the economic impact of new treatment alternatives. Comprehensive economic reviews in the field of chronic lymphocytic leukemia (CLL) are warranted to supplement the existing analyses focused on specific therapeutic areas. METHODS: A systematic literature review was conducted based on literature searches in Medline and EMBASE to summarize the published health economics models related to all types of CLL therapies. Narrative synthesis of relevant studies was performed focusing on compared treatments, patient populations, modelling approaches and key findings. RESULTS: We included 29 studies, the majority of which were published between 2016 and 2018, when data from large clinical trials in CLL became available. Treatment regimens were compared in 25 cases, while the remaining four studies considered treatment strategies with more complex patient pathways. Based on the review results, Markov modelling with a simple structure of three health states (progression-free, progressed, death) can be considered as the traditional basis to simulate cost effectiveness. However, more recent studies added further complexity, including additional health states for different therapies (e.g. best supportive care or stem cell transplantation), for progression-free state (e.g. by differentiating between with or without treatment), or for response status (i.e. partial response and complete response). CONCLUSIONS: As personalized medicine is increasingly gaining recognition, we expect that future economic evaluations will also incorporate new solutions, which are necessary to capture a larger number of genetic and molecular markers and more complex patient pathways with individual patient-level allocation of treatment options and thus economic assessments.

Overcoming barriers: Modelling the effect of potential future changes of organized breast cancer screening in Italy.

OBJECTIVES: Organized breast cancer screening may not achieve its full potential due to organizational and cultural barriers. In Italy, two identified barriers were low attendance in Southern Italy and, in Italy as a whole, underscreening and overscreening in parts of the eligible population. The objective of this study was to identify potential changes to overcome these barriers and to quantify their costs and effects. METHODS: To assess the impact of potential measures to improve breast cancer screening in Italy, we performed an evaluation of costs and effects for increasing adherence for Southern Italy and harmonizing screening intervals (biennial screening) for the whole of Italy, using an online tool (EU-TOPIA evaluation tool) based on the MIcrosimulation SCreening ANalysis (MISCAN) model. RESULTS: Increasing adherence in Southern Italy through investing in mobile screening units has an acceptable cost-effectiveness ratio of €9531 per quality-adjusted life year gained. Harmonizing the screening interval by investing in measures to reduce opportunistic screening and simultaneously investing in mobile screening units to reduce underscreening is predicted to gain 1% fewer life-years, while saving 19% of total screening costs compared to the current situation. CONCLUSIONS: Increasing adherence in Southern Italy and harmonizing the screening interval could result in substantial improvements at acceptable costs, or in the same benefits at lower costs. This example illustrates a systematic approach that can be easily applied to other European countries, as the online tools can be used by stakeholders to quantify effects and costs of a broad range of specific barriers, and ways to overcome them.

[New opportunities offered by digital technology in melanoma malignum screening.] / A digitális technológia kínálta új lehetoségek a melanoma malignum szurésében.

INTRODUCTION: In Eastern and Central Europe, Hungary has the third highest incidence of melanoma after the Czech Republic and Slovakia. In order to improve the efficiency of melanoma diagnostics and screening, several technological developments have been made in recent years, one is the integrated automated total body mapping and digital dermatoscopy. OBJECTIVE: The aim of this targeted literature review is to demonstrate the potential of new digital technologies developed for total body mapping in relation to melanoma screening. METHOD: Targeted literature review was performed to collect information. We searched in publicly available databases for guidelines and recommendations as well as scientific publications on screening and early detection of melanoma. RESULTS: We identified 15 international guidelines and recommendations summarizing screening and early detection of melanoma. We did not find any Hungarian guidelines on melanoma screening. We analyzed 2 clinical, 5 observational studies and 8 systematic reviews on the effectiveness of digital dermatoscopy and automated total body mapping. We found 10 studies that investigated the complementary role of machine learning algorithms in the early diagnosis of melanoma. DISCUSSION: Digital dermatoscopy and total body mapping can help identify skin malignancies or new melanoma at high-risk individuals. Due to the lack of reliable data of melanoma screening programmes on cancer mortality, population-based melanoma screening is not recommended by international guidelines. CONCLUSION: International guidelines highlight the role of automated total body mapping and sequential digital dermatoscopic imaging in screening of individuals at increased risk of melanoma. Orv Hetil. 2022; 163(49): 1943-1951.

Improved survival of non-small cell lung cancer patients after introducing patient navigation: A retrospective cohort study with propensity score weighted historic control.

OnkoNetwork is a patient navigation program established in the Moritz Kaposi General Hospital to improve the timeliness and completeness of cancer investigations and treatment. The H2020 SELFIE consortium selected OnkoNetwork as a promising integrated care initiative in Hungary and conducted a multicriteria decision analysis based on health, patient experience, and cost outcomes. In this paper, a more detailed analysis of clinical impacts is provided in the largest subgroup, non-small cell lung cancer (NSCLC) patients. A retrospective cohort study was conducted, enrolling new cancer suspect patients with subsequently confirmed NSCLC in two annual periods, before and after OnkoNetwork implementation (control and intervention cohorts, respectively). To control for selection bias and confounding, baseline balance was improved via propensity score weighting. Overall survival was analyzed in univariate and multivariate weighted Cox regression models and the effect was further characterized in a counterfactual analysis. Our analysis included 123 intervention and 173 control NSCLC patients from early to advanced stage, with significant between-cohort baseline differences. The propensity score-based weighting resulted in good baseline balance. A large survival benefit was observed in the intervention cohort, and intervention was an independent predictor of longer survival in a multivariate analysis when all baseline characteristics were included (HR = 0.63, p = 0.039). When post-baseline variables were included in the model, belonging to the intervention cohort was not an independent predictor of survival, but the survival benefit was explained by slightly better stage distribution and ECOG status at treatment initiation, together with trends for broader use of PET-CT and higher resectability rate. In conclusion, patient navigation is a valuable tool to improve cancer outcomes by facilitating more timely and complete cancer diagnostics. Contradictory evidence in the literature may be explained by common sources of bias, including the wait-time paradox and adjustment to intermediate outcomes.

The role of low-dose computed tomography in lung cancer screening / A kis sugárdózisú komputertomográfia szerepe a tüdorákszurésben

Introduction: Lung cancer is a serious public health problem in Hungary, but currently there is no nationwide screening program for the early detection of the disease. Several technological developments have been carried out recently to improve the effectiveness of lung cancer screening. Low-dose computed tomography (LDCT) is one of these technologies. Objective: The objective of this study is to provide an overview on guidelines and recommendations related to the application of LDCT as a novel lung cancer screening modality and to summarize the scientific literature and screening practices of other countries. Method: We performed a targeted literature review to collect information about LDCT in lung cancer screening. We searched in publicly available databases for guidelines and recommendations as well as scientific publications on screening and early detection of lung cancer. Results: In our literature search, we identified 16 guidelines and recommendations for lung cancer screening and LDCT. Regarding the efficacy of LDCT lung cancer screening, 10 foreign randomized controlled trials and 2 Hungarian trials were reviewed. Information on screening practices of 10 European countries were identified. Discussion: Evidences suggest that LDCT screening improves the detection of lung cancer, especially at early stages, and reduces cancer-specific mortality. Conclusion: In summary, in the high-risk population, LDCT can be considered an effective screening modality for the early-stage detection of lung cancer and for reducing lung cancer mortality. The ongoing Hungarian and foreign pilot programs may provide futher evidence for the implementation of a nationwide LDCT lung cancer screening program.

Prioritization of implementation barriers related to integrated care models in Central and Eastern European countries.

The importance of integrated care will increase in future health systems due to aging populations and patients with chronic multimorbidity, however, such complex healthcare interventions are often developed and implemented in higher income countries. For Central and Eastern European (CEE) countries it is important to investigate which integrated care models are transferable to their setting and facilitate the implementation of relevant models by identifying barriers to their implementation. This study investigates the relative importance of integrated care models and the most critical barriers for their implementation in CEE countries. Experts from Croatia, Hungary, Poland, Romania and Serbia were invited to complete an online survey within the SELFIE H2020 project. 81 respondents completed the survey. Although experts indicated that some integrated care models were already being implemented in CEE countries, the survey revealed a great need for further improvement in the integration of care, especially the managed care of oncology patients, coordinated palliative care of terminally ill patients, and nursing care of elderly with multimorbidity. Lack of long-term financial sustainability as well as of dedicated financing schemes were seen the most critical implementation barriers, followed by the lack of integration between health and social care providers and insufficient availability of human resources. These insights can guide future policy making on integrated care in CEE countries.

Review of the scientific literature on the use of automated breast ultrasound for screening / Az automatizált emloultrahang-daganatszurés szakirodalmi áttekintése

Several technological developments have been carried out recently to improve the effectiveness of breast cancer screening. Most of them have emerged as a complementary method to mammography. Automated breast ultrasound is one of these technologies. The objective of this study is to provide an overview on guidelines and recommenda-tions related to the application of automated breast ultrasound as a screening modality and to summarize the scien-tific literature. Targeted literature review was performed to collect information. We searched in publicly available databases for guidelines and recommendations as well as scientific publications on screening and early detection. We found substantial amount of information about automated breast ultrasound mainly for patients with dense breast; however, breast cancer screening guidelines have not yet incorporated this technology. 9 clinical studies were in-cluded in the review, most of them were single-arm studies with relatively short follow-up time. Most of them were performed in the USA. Results were presented mainly for short-term outcomes of breast cancer screening: sensitiv-ity, specificity, tumor detection rate and recall rate. The opportunity for retrospective evaluation of the images and the reproducibility are considered the most important advantages. Evidence suggest that the cancer detection rate can be improved compared to mammography alone in women with dense breast. The main disadvantages of this technology are the high recall and false positive rates. Further scientific evidence is required to reduce uncertainty related to the use of automated breast ultrasound for breast cancer screening.

Heterogeneous impact of the COVID-19 pandemic on lung, colorectal and breast cancer incidence in Hungary: results from time series and panel data models.

OBJECTIVE: During the COVID-19 pandemic, health system resources were reallocated to provide care for patients with COVID-19, limiting access for others. Patients themselves also constrained their visits to healthcare providers. In this study, we analysed the heterogeneous effects of the pandemic on the new diagnoses of lung, colorectal and breast cancer in Hungary. DESIGN: Time series and panel models of quarterly administrative data, disaggregated by gender, age group and district of residence. PARTICIPANTS: Data for the whole population of Hungary between the first quarter of 2017 and the second quarter of 2021. MAIN OUTCOME MEASURES: Number of patients newly diagnosed with lung, colorectal and breast cancer, defined as those who were hospitalised with the appropriate primary International Classification of Diseases Tenth Revision diagnosis code but had not had hospital encounters with such a code within the previous 5 years. RESULTS: The incidence of lung, colorectal and breast cancer decreased by 14.4% (95% CI 10.8% to 17.8%), 19.9% (95% CI 12.2% to 26.9%) and 15.5% (95% CI 2.5% to 27.0%), respectively, during the examined period of the pandemic, with different time patterns across cancer types. The incidence decreased more among people at least 65 years old than among the younger (p<0.05 for lung cancer and p<0.1 for colorectal cancer). At the district level, both the previously negative income gap in lung cancer incidence and the previously positive income gap in breast cancer incidence significantly narrowed during the pandemic (p<0.05). CONCLUSIONS: The decline in new cancer diagnoses, caused by a combination of supply-side and demand-side factors, suggests that some cancer cases have remained hidden. It calls for action by policy makers to engage individuals with high risk of cancer more in accessing healthcare services, to diagnose the disease early and to prepare for effective management of patient pathways from diagnosis to survival or end-of-life care.

Effects of lower screening activity during the COVID-19 pandemic on breast cancer patient pathways: Evidence from the age cut-off of organized screening.

We examined the effects of the COVID-19 pandemic on the screening, diagnosis and treatment of breast cancer in Hungary based on administrative data until June 2021, covering three pandemic waves. After correcting for trend and seasonality, the number of mammography examinations decreased by 68% in 2020q2, was around its usual level in 2020q3 and was reduced by 20-35% throughout 2020q4-2021q2. The reduction was caused by a combination of supply-side (temporary suspensions of screening) and demand-side (lower screening participation during the pandemic waves) factors. The number of new breast cancer diagnoses and mastectomy surgeries responded with a lag, and were below their usual level by 15-30% in all quarters between 2020q2 and 2021q2, apart from 2020q4, when there was no significant difference. Using a regression discontinuity framework, we found that the partial mastectomy rate (indicative of early diagnosis) dropped more substantially in 2020q2 in the 61-65 years old age group that was just below the age cut-off of organized screening than in the 66-70 years old age group, and this difference was partially offset in 2021q1. We suggest that policymakers need to motivate the target population (by providing both information and incentives) to catch up on missed screenings.

Pricing strategies, executive committee power and negotiation leverage in New Zealand's containment of public spending on pharmaceuticals.

This paper explores policy mechanisms behind New Zealand's remarkable track record of cost containment in public pharmaceutical spending, contrasting with most other advanced economies. We drew on a review of official policy documents and 28 semi-structured expert interviews. We found that decision making in pricing and reimbursement policy was dominated by a small group of managers at the Pharmaceutical Management Agency (PHARMAC), the country's drug reimbursement and Health Technology Assessment Agency, who negotiated pharmaceutical prices on behalf of the public payer. In formal negotiation over patented pharmaceutical prices these managers applied an array of pricing strategies, most notably, 'bundling' consisting of discounted package deals for multiple pharmaceuticals, and 'play-off tenders', whereby two or more pharmaceutical companies bid for exclusive contracts. The key pricing strategy for generic drugs, in contrast, was 'blind-tenders' taking the form of an annual bidding process for supply contracts. An additional contextual condition on bargaining over pharmaceutical prices was an indirect strategy that involved the cultivation of the PHARMAC's 'negotiation leverage'. We derived two cost containment mechanisms consisting in the relationship between pricing strategy options and various reimbursement actors. Our findings shed light on aspects of the institutional design of drug reimbursement that may promote the effective use of competitive negotiations of pharmaceutical prices, including specific pricing strategies, by specialist public payer institutions. On this basis, we formulate recommendations for countries seeking to develop or reform policy frameworks to better meet the budgetary challenge posed by pharmaceutical expenditure.

Secondary primary malignancies after treatment with chemo-immunotherapy in treatment-naïve patients with CLL: a systematic literature review.

OBJECTIVES: In chronic lymphocytic leukemia (CLL), therapy-related cytotoxicity and the resulting immunodeficiency are thought to contribute to the development of secondary primary malignancies (SPM). Here, we analyzed clinical trial data on the occurrence of SPM following chemo-immunotherapy (CIT) regimens in treatment-naïve CLL patients. METHODS: A systematic literature search was conducted covering multiple databases between 2003 and 2019. Data from relevant clinical trials on the proportion of patients with SPMs were extracted. Then, the number of SPM patients/person-years was calculated by taking into account the trials' follow-up time. Finally, a random-effects meta-analysis to pool the rates from individual studies was performed. RESULTS: We identified 22 studies reporting SPM data available for analysis. Random-effects meta-analysis estimated that the number of SPM patients/1000 person-years was 24 (95%CI: 19-29). Results from trials with cancer-specific data indicated 19 (95%CI: 14-26) solid and 9 (95%CI: 6-12) hematological SPM patients/1000 person-years. These estimations did not change significantly when sub-groups were analyzed by CIT regimens. CONCLUSION: Although pooling data with the intention to analyze adverse event rates is challenging, our study concluded that for CIT regimens, SPM should be considered an important adverse outcome. Different regimens showed similar trends; however, other clinical and demographic factors also have profound impact.

Burden of Chronic Heart Failure in Romania.

Chronic heart failure (CHF) affects millions of people across the world, with increasing trends in prevalence, putting ever increasing pressure on the healthcare system. The aim of this study was to assess the financial burden of CHF hospital care on the public healthcare sector in Romania by estimating the number of inpatient episodes and the associated costs. Additionally, societal costs associated with missed work and premature death of CHF patients were also estimated. The national claims database was analyzed to estimate the number of CHF patients. Cost data was extracted from a pool of nine public hospitals in Romania. In 2019, 375,037 CHF patient episodes were identified on specific wards at the national level. The average cost calculated for the selected nine hospitals was EUR 996. The calculated weighted national average cost per patient episode was EUR 1002, resulting in a total cost of EUR 376 million at the national level. The cost of workdays missed summed up to EUR 122 million, while the annual costs associated with the premature death of CHF patients was EUR 230 million. In conclusion, the prevalence of CHF in Romania is high, accounting for a large proportion of hospitalizations, which translates into large costs for the national payer.

Cost-effectiveness of colorectal cancer screening in Slovakia.

BACKGROUND: Colorectal cancer (CRC) is an ideal disease for screening due to known and detectable precursor lesions and slow progression from benign adenoma to invasive cancer. The introduction of organized population-based screening programs reduces the burden of colorectal cancer and increases the quality of the screening process with a more favorable harm to benefit ratio compared to opportunistic screening. METHODS: The study used the microsimulation screening analysis-colon simulation model for the estimation of the effect of various factors on cancer incidence and mortality. The model simulated the Slovakian population from 2018 to 2050. Study includes the analysis of two screening strategies the fecal immunochemical test (FIT) every 2 years and annual FIT. Cost-effectiveness parameters were evaluated comparing each simulated screening scenario with no screening. RESULTS: Compared to no screening, the biennial FIT would detect 29 600 CRC cases and annual FIT 37 800 CRC cases. Mortality due to CRC showed benefits for both strategies with 17,38% reduction in biennial FIT and 24,67% reduction in annual FIT approach. Both screening programs were more costly as well as more effective compared to no screening. The ICER for biennial FIT strategy was 1776 EUR per 1 QALY and for the annual FIT 3991 EUR per 1 QALY. CONCLUSIONS: In summary, this is the first cost-effectiveness analysis focusing on multiple national CRC screening strategies in Slovakia. Both strategies demonstrated cost-effectiveness compared to no screening. However, for optimal population-based programmatic screening strategy, the policymakers should also consider human resources availability, acceptability of screening test among the population or additional resources including the screening funding.

A 'patient-industry complex'? Investigating the financial dependency of UK patient organisations on drug company funding.

We examined the minimum extent of dependency of UK patient organisations on pharmaceutical industry funding using drug company disclosure reports and patient organisation financial accounts from 2012 to 2016. We used linear regression to explain the overall share of industry funding ('general dependency') and top donor funding ('company-specific dependency') in organisations' income. Predictors included patient organisations' goal; having members and volunteers; geographical scope of activity; headquarter location; expenditure/income ratio; and disease area. The prevalent low levels of general dependency (IQR, 0.1%-6.0%) and company-specific dependency (IQR, 0.1%-4.3%) made a widespread capture of patient organisations unlikely, though only if one excludes the possibility of significant payment under-reporting. However, organisations with considerably higher dependency than others might be more prone to co-optation by industry. Of the 398 organisations, 18 (4.5%) and 8 (2.0%) had general and company-specific financial dependency over 50%, respectively. However, the shares of outliers exceeding the third quartile plus 1.5 times IQR were 51 (12.8%) and 56 (14.1%) for each dependency type. Certain characteristics including activity profile (advocacy) or indicating limited access to resources (remote location) made organisations vulnerable to developing financial dependency. Future research should examine both financial and non-financial links between the two sides and their impact on patient organisations' activity.

Development of a breast cancer screening protocol to use automated breast ultrasound in a local setting.

Introduction: The sensitivity of mammography screening is lower in women with dense breast. Increasing the efficacy of breast cancer screening have received special attention recently. The automated breast ultrasound (ABUS) shows promising results to complement mammography. Our aim was to expand the existing breast cancer screening protocol with ABUS within a Hungarian pilot project. Methods: First, we developed a protocol for the screening process focusing on integrating ABUS to the current practice. Consensus among clinical experts was achieved considering information from the literature and the actual opportunities of the hospital. Then we developed a protocol for evaluation that ensures systematic data collection and monitoring of screening with mammography and ABUS. We identified indicators based on international standards and adapted them to local setting. We considered their feasibility from the data source and timeframe perspective. The protocol was developed in a partnership of researchers, clinicians and hospital managers. Results: The process of screening activity was described in a detailed flowchart. Human and technological resource requirements and communication activities were defined. We listed 23 monitoring indicators to evaluate the screening program and checked the feasibility to calculate these indicators based on local data collection and other sources. Partnership between researchers experienced in planning and evaluating screening programs, interested clinicians, and hospital managers resulted in a locally implementable, evidence-based screening protocol. Discussion: The experience and knowledge gained on the implementation of the ABUS technology could generate real-world data to support the decision on using the technology at national level.

Access to high-priced medicines in lower-income countries in the WHO European Region

Lower-income countries (LICs) in the WHO European Region generally have poorer health status and more limited resources than higher-income countries. This creates pressing issues when making decisions on financing high-priced medicines. The market access strategies for innovative medicines in LICs usually follow the low-volume, high-price business model, which does not guarantee access for all potentially eligible patients. If the political will to make changes exists, however, several policy tools could be used that may improve patient access to these medicines. This technical report describes specific constraints of LICs related to public financing of high-priced medicines, and summarizes policy considerations to improve access to high-priced medicines in LICs in three areas, including price-control mechanisms, tools to increase the negotiation power of health-care payers and tools to facilitate appropriate prescribing.

Implementation of evidence-based health policy in the field of integrated care: A Ritka Betegségek Nemzeti Eroforrás Központjának tervezési folyamata / Bizonyítékokon alapuló egészségpolitikai döntéshozatal az integrált ellátásban: A Ritka Betegségek Nemzeti Eroforrás Központjának tervezési folyamata

Összefoglaló. Bevezetés: A ritka betegséggel élok ellátásában fontos elorelépések történtek az elmúlt években. Egy következo lépés lehetne hazánkban a Ritka Betegségek Nemzeti Eroforrás (Uni-Versum) Központjának (a továbbiakban: Központ) létrehozása, amely az egészségügyi, szociális és köznevelési szolgáltatásokat összehangolva és kiegészítve személyközpontú ellátást nyújtana a betegek és támogatóik részére. Célkituzés: Célunk az volt, hogy egy nemzetközi tudományos módszertan alapján javaslatokat tegyünk arra, hogy milyen eszközökkel lehet feloldani a Központ megvalósításának lehetséges korlátozó tényezoit. Módszer: A Központ megvalósíthatóságának értékelésére interdiszciplináris szakmai egyeztetést szerveztünk különbözo érintett érdekcsoportok részvételével, a SELFIE H2020 kutatási projekt által kidolgozott módszertan alapján. Az elozetesen rangsorolt legfontosabb korlátozó tényezokre lehetséges megoldási javaslatokat tettünk. Eredmények: A lehetséges korlátozó tényezoket a résztvevok relevánsnak tartották a Központ létrehozásával kapcsolatban, és ezekre összesen 17 olyan konkrét javaslat született, amelyben a résztvevok között egyetértés alakult ki. A javaslatok kiterjedtek az ellátás tartalmára, az alkalmazott technológiák támogató szerepére, a humáneroforrás-korlátok megoldására, a hatékony vezetés és szervezés kialakítására, az összetett finanszírozási struktúra kialakítására és a kutatási lehetoségek megteremtésére is. Megbeszélés: A Központ megvalósítása esetén a ritka betegséggel élok ellátása az egészségügyi, szociális és köznevelési tevékenységeket integráló megközelítés felé mozdulna el. A kutatás során megfogalmazott javaslatok hozzájárulhatnak a Központ létrehozásához, amennyiben megvan az ehhez szükséges szakpolitikai támogatás is. Ezen túlmutatóan, a leírt munkamódszer más integrált ellátási modellek bevezethetoségének elemzéséhez is mintaként szolgálhat. Következtetés: Összefoglalva megállapíthatjuk, hogy a Központ létrehozásához számos, elozetesen is látható korlátozó tényezot kell feloldani. Az érdekcsoportok közös javaslatai alapján kialakítható egy olyan muködési forma, amely az ellátórendszerek kiegészítésével és összehangolásával jelentos társadalmi értéktöbbletet eredményezhet. Orv Hetil. 2021; 162(45): 1818-1825. INTRODUCTION: In Hungary, significant achievements have been made in the care of patients with rare diseases in recent years. A next step could be the establishment of the National Resource Centre for Rare Diseases (hereinafter: Centre) to facilitate patient-centered complex care by the integration and supplementation of existing health, social and educational services. OBJECTIVE: This research aimed to develop recommendations based on international scientific methodology to overcome potential implementation barriers of the aforementioned Centre. METHOD: To evaluate the feasibility of the implementation, we organized an interdisciplinary workshop with representatives of different stakeholder groups, adopting the methodology developed in the SELFIE H2020 research project. During the workshop, we discussed the previously ranked, most significant implementation barriers and made recommendations for potential solutions. RESULTS: The potential implementation barriers were considered relevant by the participants and, reflecting on these barriers, altogether 17 recommendations were developed by consensus. These recommendations were related to the content of service delivery, use of supportive technologies, overcoming workforce issues, establishing effective leadership, implementing a complex financing structure and creating research opportunities. DISCUSSION: Implementation of the Centre would shift the care of rare diseases towards personalized and integrated health, social and educational services. Our recommendations will contribute to the establishment of the Centre, subject to positive policy decision. Furthermore, our methodological approach could support the feasibility assessment of future integrated care solutions and programs. CONCLUSION: Several predictable barriers must be overcome to establish the Centre. Recommendations developed by representatives of relevant stakeholders could support successful implementation and societal value generation. Orv Hetil. 2021; 162(45): 1818-1825.

Early palliative care associated with lower costs for adults with advanced cancer: evidence from Hungary.

OBJECTIVE: Palliative Care Consult Service (PCCS) programme was established in Hungary to provide palliative care to hospitalised patients with complex needs and to coordinate integrated care across providers. The aim of this study was to measure the association of PCCS with healthcare costs from payer's perspective. METHODS: Study population consisted of patients with metastatic cancer, who were admitted to the Clinical Centre of the University of Pécs between 2014 and 2016. Patients who did not die within 180 days from enrolment were excluded. Patients receiving services from PCCS team (intervention patients) were compared to patients receiving usual care (controls). The two populations were matched using propensity scores. Data were obtained from electronic medical records linked to claims data. RESULTS: For patients who were involved in PCCS at least 60 days before their death, the costs of care outside the acute hospital were higher. However, this was offset by savings in hospital costs so that the total healthcare cost was significantly reduced (p = 0.034). The proportion of patients who died in the hospital was lower in the PCCS group compared to the usual care group (66% vs. 85%, p = 0.022). CONCLUSION: Timely initiation of palliative care for hospitalised patients is associated with cost savings for the healthcare system.