Dawkowanie i koszt lanreotydu Autogel 120 mg stosowanego w leczeniu akromegalii w Polsce — wyniki 2-letniego badania Lanro-Study

Introduction: To evaluate, over 24 months of prospective follow-up, the dosage and costs of lanreotide AUTOGEL 120 mg (ATG120) administered as part of routine acromegaly care in Poland.Material and methods: A multicentre, non-interventional, observational prospective study on resource utilisation in Polish acromegalic patients treated with ATG120 at 4-week or extended (> 4 weeks) dosing interval. The study population consisted of adult acromegalic patients treated for at least 3 injections of ATG120. The endpoints were: percentage of patients treated with ATG120 at an extended dosing interval (> 4 weeks), mean time between injections, and the cost of ATG120 during a 24-month prospective observation. Costs were calculated in PLN from the public health-care payer and patient perspective for the year 2014.Results: 143 patients were enrolled in, and 132 completed (70% women, 81% macroadenoma, 75% previous surgery) the analysis. During two years, changes in the treatment pattern were reported in 41 patients: 17% of them had increased injection interval and 10% switched to octreotide LAR and then returned to ATG120. Sixty-three patients (48%) received ATG120 at an extended dosing interval. ATG120 was predominantly administered in an out-patient setting (84%) by a health care professional (97%).Conclusions: The results demonstrated that extended dosing interval of ATG120 is used in a substantial proportion of patients in routine clinical practice in Poland. Such findings support the potential for ATG120 in reducing treatment burden in the real-world clinical environment. (Endokrynol Pol 2015; 66 (2): 142–148)Wstęp: Ocena schematu dawkowania i kosztu lanreotydu Autogel 120 mg (ATG120) stosowanego w leczeniu akromegalii w Polsce.Materiał i metody: Wieloośrodkowe, nieinterwencyjne, obserwacyjne badanie prospektywne oceniające zużycie zasobów ochrony zdrowia w populacji polskich pacjentów z akromegalią leczonych ATG120 z zastosowaniem 4-tygodniowego lub wydłużonego (> 4 tygodni) odstępu między dawkami leku. Do badania włączani byli dorośli pacjenci, którzy otrzymali co najmniej 3 dawki ATG120. Horyzont czasowy badania wynosił 24 miesięcy. Koszt leku obliczono z perspektywy płatnika publicznego i pacjenta w 2014 roku.Wyniki: Do badania włączono 143 pacjentów, 132 z nich ukończyło badanie (70% kobiet, u 81% makrogruczolak, 75% było wcześniej leczonych chirurgicznie). W czasie 2 lat zmiany schematu leczenia dokonano u 41 chorych, u 17% z nich wydłużono odstęp między dawkami, u 10% — zmieniono lek na oktreotyd LAR a potem ponownie zastosowano ATG120. U 63 pacjentów (48%) odstęp pomiędzy dawkami ATG120 wynosił > 4 tygodnie. ATG120 był przeważnie podawany ambulatoryjnie (84%) przez fachowy personel medyczny (97%). Wnioski: Badanie wykazało, że w codziennej praktyce klinicznej u znaczącego odsetka pacjentów odstęp czasowy pomiędzy kolejnymi dawkami ATG120 jest dłuższy niż 4 tygodnie. Wynik ten potwierdza tezę, że dzięki stosowaniu ATG120 można obniżyć koszty leczenia akromegalii. (Endokrynol Pol 2015; 66 (2): 142–148

LanroNET — nieinterwencyjne badanie prospektywne oceniające wykorzystanie zasobów medycznych i koszty Lanreotide Autogel 120 mg w populacji polskich pacjentów z objawowymi guzami neuroendokrynnymi

Wstęp: Celem badania LanroNET była ocena wykorzystania zasobów medycznych oraz kosztów objawowego leczenia polskich chorych na nowotwory neuroendokrynne z zastosowaniem lanreotydu autogel 120 mg. Materiał i metody: LanroNET to wieloośrodkowe, nieinterwencyjne, obserwacyjne, prospektywne badanie przeprowadzone w 12 ośrod­kach w Polsce. W badaniu uczestniczyli dorośli chorzy na wydzielające nowotwory neuroendokrynne leczeni lanreotydem autogel 120 mg od przynajmniej 3 miesięcy przed włączeniem do badania. Podczas 24-miesięcznej obserwacji rzeczywistej praktyki klinicznej zbierano dane dotyczące wykorzystania zasobów medycznych oraz przebiegu terapii chorych z wydzielającymi nowotworami neuroendokrynnymi. Wyniki: W badaniu uczestniczyło 54 chorych na wydzielające nowotwory neuroendokrynne. Przeciętny czas stosowania lanreotydu wynosił 1,7 roku (zakres 0,0–2,2 lata). Badanie ukończyło 33 pacjentów, najczęstszą przyczyną przedwczesnego zakończenia leczenia (8/16) była progresja choroby. Całkowity średni koszt wykorzystanych zasobów bez kosztów farmakoterapii oszacowano na 26 307 zł/EUR 6.030,35 na pacjenta/rok. W czasie badania średni odstęp między wstrzyknięciami lanreotydu wynosił 31,7 dni (6,7). Pod koniec obserwacji, po 24 miesiącach od follow-up, 7 pacjentów stosowało 42-dniowe odstępy między dawkami. Średni rzeczywisty koszt lanreotydu autogel 120 mg wyniósł 4216,30 zł/966,49 EUR na pacjenta/28 dni we wspólnej perspektywie płatnika i pacjenta i był niższy o 554,16 zł/127,02 EUR niż koszt stosowania standardowych 28-dniowych odstępów między dawkami. Wnioski: Badanie LanroNET jest pierwszym w Polsce obserwacyjnym dwuletnim badaniem chorych na czynne hormonalnie nowotwo­ry neuroendokrynne żołądkowo-jelitowo-trzustkowe oceniającym koszty codziennej praktyki klinicznej i koszty leczenia lanreotydem autogel.Introduction: The primary objective of the LanroNET study was to evaluate the resource utilisation and cost of symptomatic treatment of patients with neuroendocrine tumours (NET) using lanreotide autogel 120 mg. Material and methods: LanroNET was a multicentre, non-interventional, prospective study conducted at 12 clinical centres in Poland. Eligible patients were adults with symptomatic NET treated with lanreotide autogel 120 mg at least three months before enrolment. During 24-months of observation of real clinical practice, data on medical resource utilisation and the therapy course of patients with symptomatic NET were collected. Results: Fifty-four patients with symptomatic NET were enrolled. The median time of lanreotide exposure was 1.7 years (range: 0.0–2.2). Thirty-three patients completed the study; the most frequent known cause of discontinuation (8/16) was disease progression. The mean cost of consumed resources without the cost of pharmacotherapy was estimated at PLN 26,307/EUR 6030.35 per year. During the study, the mean (SD) interval between injections was 31.7 days (6.7). At the end of observation — after 24 months of follow-up, seven patients were on a 42-day regimen. The average real-world cost of lanreotide autogel 120 mg was PLN 4216.30/EUR 966.49 per patient/28 days from the public payer and patient perspective and was lower by PLN 554.14/EUR 127.02 than the cost for the standard 28-day dosing interval. Conclusions: LanroNET is the first two-year observational study of patients with symptomatic NET evaluating the cost of every-day clinical practice and lanreotide autogel treatment in Poland

Principles of Good Practice for Budget Impact Analysis: Report of the ISPOR Task Force on Good Research Practices—Budget Impact Analysis

AbstractObjectiveThere is growing recognition that a comprehensive economic assessment of a new health-care intervention at the time of launch requires both a cost-effectiveness analysis (CEA) and a budget impact analysis (BIA). National regulatory agencies such as the National Institute for Health and Clinical Excellence in England and Wales and the Pharmaceutical Benefits Advisory Committee in Australia, as well as managed care organizations in the United States, now require that companies submit estimates of both the cost-effectiveness and the likely impact of the new health-care interventions on national, regional, or local health plan budgets. Although standard methods for performing and presenting the results of CEAs are well accepted, the same progress has not been made for BIAs. The objective of this report is to present guidance on methodologies for those undertaking such analyses or for those reviewing the results of such analyses.MethodsThe Task Force was appointed with the advice and consent of the Board of Directors of ISPOR. Members were experienced developers or users of budget impact models, worked in academia, industry, and as advisors to governments, and came from several countries in North America, Oceana, Asia, and Europe. The Task Force met to develop core assumptions and an outline before preparing a draft report. They solicited comments on the outline and two drafts from a core group of external reviewers and more broadly from the membership of ISPOR at two ISPOR meetings and via the ISPOR web site.ResultsThe Task Force recommends that the budget impact of a new health technology should consider the perspective of the specific health-care decision-maker. As such, the BIA should be performed using data that reflect, for a specific health condition, the size and characteristics of the population, the current and new treatment mix, the efficacy and safety of the new and current treatments, and the resourceuse and costs for the treatments and symptoms as would apply to the population of interest. The Task Force recommends that budget impact analyses be generated as a series of scenario analyses in the same manner that sensitivity analyses would be provided for CEAs. In particular, the input values for the calculation and the specific cost outcomes presented (a scenario) should be specific to a particular decision-maker's population and information needs. Sensitivity analysis should also be in the form of alternative scenarios chosen from the perspective of the decision-maker. The primary data sources for estimating the budget impact should be published clinical trial estimates and comparator studies for efficacy and safety of current and new technologies as well as, where possible, the decision-maker's own population for the other parameter estimates. Suggested default data sources also are recommended. These include the use of published data, well-recognized local or national statistical information and in special circumstances, expert opinion. Finally, the Task Force recommends that the analyst use the simplest design that will generate credible and transparent estimates. If a health condition model is needed for the BIA, it should reflect health outcomes and their related costs in the total affected population for each year after the new intervention is introduced into clinical practice. The model should be consistent with that used for the CEA with regard to clinical and economic assumptions.ConclusionThe BIA is important, along with the CEA, as part of a comprehensive economic evaluation of a new health technology. We propose a framework for creating budget impact models, guidance about the acquisition and use of data to make budget projections and a common reporting format that will promote standardization and transparency. Adherence to these proposed good research practice principles would not necessarily supersede jurisdiction-specific budget impact guidelines, but may support and enhance localrecommendations or serve as a starting point for payers wishing to promulgate methodology guidelines

Budget impact analysis: principles of good practice. Report of the ISPOR Working Group on Good Practices for Budget Impact Analysis II, 2012

Background. Budget impact analyses (BIAs) are an essential part of a comprehensive economic assessment of a health care intervention and are increasingly required by reimbursement authorities as part of a listing or reimbursement submission. Objectives: The objective of this report was to present updated guidance on methods for those undertaking such analyses or for those reviewing the results of such analyses. This update was needed, in part, because of developments in BIA methods as well as a growing interest, particularly in emerging markets, in matters related to affordability and population health impacts of health care interventions. Methods. The Task Force was approved by the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council and appointed by its Board of Directors. Members were experienced developers or users of BIAs; worked in academia and industry and as advisors to governments; and came from several countries in North America and South America, Oceania, Asia, and Europe. The Task Force solicited comments on the drafts from a core group of external reviewers and, more broadly, from the membership of the International Society for Pharmacoeconomics and Outcomes Research. Results. The Task Force recommends that the design of a BIA for a new health care intervention should take into account relevant features of the health care system, possible access restrictions, the anticipated uptake of the new intervention, and the use and effects of the current and new interventions. The key elements of a BIA include estimating the size of the eligible population, the current mix of treatments and the expected mix after the introduction of the new intervention, the cost of the treatment mixes, and any changes expected in condition-related costs. Where possible, the BIA calculations should be performed by using a simple cost calculator approach because of its ease of use for budget holders. In instances, however, in which the changes in eligible population size, disease severity mix, or treatment patterns cannot be credibly captured by using the cost calculator approach, a cohort or patient-level condition-specific model may be used to estimate the budget impact of the new intervention, accounting appropriately for those entering and leaving the eligible population over time. In either case, the BIA should use data that reflect values specific to a particular decision maker’s population. Sensitivity analysis should be of alternative scenarios chosen from the perspective of the decision maker. The validation of the model should include at least face validity with decision makers and verification of the calculations. Data sources for the BIA should include published clinical trial estimates and comparator studies for the efficacy and safety of the current and new interventions as well as the decision maker’s own population for the other parameter estimates, where possible. Other data sources include the use of published data, well-recognized local or national statistical information, and, in special circumstances, expert opinion. Reporting of the BIA should provide detailed information about the input parameter values and calculations at a level of detail that would allow another modeler to replicate the analysis. The outcomes of the BIA should be presented in the format of interest to health care decision makers. In a computer program, options should be provided for different categories of costs to be included or excluded from the analysis. Conclusions. We recommend a framework for the BIA, provide guidance on the acquisition and use of data, and offer a common reporting format that will promote standardization and transparency. Adherence to these good research practice principles would not necessarily supersede jurisdiction-specific BIA guidelines but may support and enhance local recommendations or serve as a starting point for payers wishing to promulgate methodology guidelines

Budget-Impact Analyses: A Critical Review of Published Studies

This article reviews budget-impact analyses (BIAs) published to date in peer-reviewed bio-medical journals with reference to current best practice, and discusses where future research needs to be directed. Published BIAs were identified by conducting a computerized search on PubMed using the search term 'budget impact analysis'. The years covered by the search included January 2000 through November 2008. Only studies (i) named by authors as BIAs and (ii) predicting financial consequences of adoption and diffusion of a new health intervention(s) within a specific healthcare setting were included. Relevant studies were evaluated according to the checklist that focuses on issues unique to BIA, highlighting areas of agreement or dissent between published studies and methodological guidelines. A total of 34 studies met the inclusion criteria, the majority published in 2007-8. Of these, 41% were from the US, 54% were prepared for pharmaceuticals and 65% had BIA as their main aim. The published BIAs were heterogeneous in respect of methods for deriving budget-impact estimates, time horizon and population. There is fairly good agreement between published studies and methodological guidelines within the scope of perspective, comparator, cost included and data sources. Specific issues that need to be addressed and/or improved are reporting format, sensitivity analysis and discounting. The results indicate that, recently, BIAs have appeared more frequently in peer-reviewed journals, providing stimulus to development, validation and dissemination of methods. Many published studies fail to reach the desired quality, but this situation should change with good research practice principles that will help codify and clarify important issues and promote standardization and transparency. Future research needs to be directed to quality assurance of published BIAs and investment in data collection for parameters specific to BIAs.

Cost-Effectiveness Analysis of Enoxaparin versus Unfractionated Heparin in Patients with Acute Coronary Syndrome in Poland: Modelling Study from the Hospital Perspective

Aim and perspective: To estimate the cost effectiveness of enoxaparin versus unfractionated heparin (UFH) in patients with acute coronary syndrome (ACS) from a Polish hospital perspective. This was intended to facilitate the decisionmaking process in selecting the most cost-effective treatment for ACS. Method: A decision model was used to quantify costs and effectiveness of alternative treatments. Published results from the Efficacy and Safety of Subcutaneous Enoxaparin in Non-Q-Wave Coronary Events (ESSENCE) study were used to estimate the probability for clinical endpoints (death, myocardial infarction or recurrent angina) at 30 days. Probabilities of patients undergoing revascularisation procedures were obtained from the Global Registry of Acute Coronary Events (GRACE) which included data from 961 patients at six centres in Poland. The analysis assessed only direct medical costs, determined from actual resource consumption on a patient-specific basis (6-month observational study) and estimated using Polish data on unit costs. One- and two-way sensitivity analyses and threshold analysis were performed. Results: At 30 days, 19.8% of patients receiving enoxaparin compared with 23.3% of those receiving UFH reached a composite endpoint consisting of death, myocardial infarction and recurrent angina (p Conclusion: According to our model enoxaparin was more effective at a lower cost than UFH, therefore this treatment was shown to be dominant for patients with ACS in Poland.Acute-coronary-syndromes, Cost-effectiveness, Enoxaparin-sodium, Heparin, Heparins, Low-molecular-weight-heparins, Pharmacoeconomics