Pub Date : 2023-12-14DOI: 10.1017/s0266462323000673
Rosalia Ragusa, Dott Fabio Didonè, Laura Botta, Antonina Torrisi, Maria Alessandra Bellia, Gemma Gatta, BENCHISTA Italy working group
Introduction
Pediatric cancers are rare tumors, heterogeneous in location and biologically very different from adult cancers. Documented survival variation across European countries and Italian regions shows that there is still room for further improvement by reducing inequalities. We aim to understand why there are differences in survival. The BENCHISTA-ITA project (National Benchmarking of Childhood Cancer Survival by Stage at diagnosis), that is the Italian twin project of the International BENCHISTA, collects stage at diagnosis of solid pediatric tumors, according to the Toronto Guidelines. We will compare how far the cancer has spread at diagnosis and test if differences in tumor stage explain any survival differences between Italian regions.
Methods
The project study involved the stage distribution and the survival of 9 pediatric solid tumors diagnosed between 2013 and 2017 in Italy. All patients therefore had at least 3 years of follow-up in 2021 for life-stage definition. The study involves the identification of all new diagnoses of cancer, evaluation of the clinical documentation of cases eligible for research, and international classification and coding. Analyses of stage distribution and survival rates for each tumor type will be described.
Results
Data from 35 population-based cancer registries from 18 out of 20 Italian regions were collected covering about 84 percent of the Italian child population. In particular, data on: imaging/examination performed before any treatment; source used for staging; primary treatment defined as given within one year from diagnosis; relapse/ recurrence/ progression; follow up and status of life. The study tested the applicability of the Toronto Guidelines as a tool to obtain population-level comparable stage information for childhood cancers. There were 1,343 cases collected (242 Neuroblastoma, 124 Wilms Tumour, 145 Medulloblastoma, 148 Osteosarcoma, 135 Ewing sarcoma, 115 Rhabdomyososarcoma, 54 Ependymoma, 47 Retinoblastoma, 333 Astrocytoma). Toronto stage could be assigned in more than 90 percent in the majority of tumors. Tumors in which it was more difficult to assign the stage using the Toronto staging guidelines were ependymoma, astrocytoma, and retinoblastoma. It was easier to retrieve data for patients in the 0-14 years of age range than adolescents (14-18 years). Differences in stage distribution and survival differences between regional grouping were presented.
Conclusions
The Italian BENCHISTA project, improving the connection between pediatric cancer registries, aims to improve care of children with cancer across the nation, reducing possible disparities.
The wide adoption of the Toronto Guidelines will facilitate international comparative incidence studies, strengthen the interpretation of survival data, and contribute to more appropriate solutions to improve childhood cancer outcomes.
{"title":"OP22 Benchmarking Of Population-Based Childhood Cancer Survival By Toronto Stage: Know The Differences To Propose Effective Interventions","authors":"Rosalia Ragusa, Dott Fabio Didonè, Laura Botta, Antonina Torrisi, Maria Alessandra Bellia, Gemma Gatta, BENCHISTA Italy working group","doi":"10.1017/s0266462323000673","DOIUrl":"https://doi.org/10.1017/s0266462323000673","url":null,"abstract":"<span>Introduction</span><p>Pediatric cancers are rare tumors, heterogeneous in location and biologically very different from adult cancers. Documented survival variation across European countries and Italian regions shows that there is still room for further improvement by reducing inequalities. We aim to understand why there are differences in survival. The BENCHISTA-ITA project (National Benchmarking of Childhood Cancer Survival by Stage at diagnosis), that is the Italian twin project of the International BENCHISTA, collects stage at diagnosis of solid pediatric tumors, according to the Toronto Guidelines. We will compare how far the cancer has spread at diagnosis and test if differences in tumor stage explain any survival differences between Italian regions.</p><span>Methods</span><p>The project study involved the stage distribution and the survival of 9 pediatric solid tumors diagnosed between 2013 and 2017 in Italy. All patients therefore had at least 3 years of follow-up in 2021 for life-stage definition. The study involves the identification of all new diagnoses of cancer, evaluation of the clinical documentation of cases eligible for research, and international classification and coding. Analyses of stage distribution and survival rates for each tumor type will be described.</p><span>Results</span><p>Data from 35 population-based cancer registries from 18 out of 20 Italian regions were collected covering about 84 percent of the Italian child population. In particular, data on: imaging/examination performed before any treatment; source used for staging; primary treatment defined as given within one year from diagnosis; relapse/ recurrence/ progression; follow up and status of life. The study tested the applicability of the Toronto Guidelines as a tool to obtain population-level comparable stage information for childhood cancers. There were 1,343 cases collected (242 Neuroblastoma, 124 Wilms Tumour, 145 Medulloblastoma, 148 Osteosarcoma, 135 Ewing sarcoma, 115 Rhabdomyososarcoma, 54 Ependymoma, 47 Retinoblastoma, 333 Astrocytoma). Toronto stage could be assigned in more than 90 percent in the majority of tumors. Tumors in which it was more difficult to assign the stage using the Toronto staging guidelines were ependymoma, astrocytoma, and retinoblastoma. It was easier to retrieve data for patients in the 0-14 years of age range than adolescents (14-18 years). Differences in stage distribution and survival differences between regional grouping were presented.</p><span>Conclusions</span><p>The Italian BENCHISTA project, improving the connection between pediatric cancer registries, aims to improve care of children with cancer across the nation, reducing possible disparities.</p><p>The wide adoption of the Toronto Guidelines will facilitate international comparative incidence studies, strengthen the interpretation of survival data, and contribute to more appropriate solutions to improve childhood cancer outcomes.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138689288","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323001198
Patrick Wincy Reyes, Lara Alyssa Liban, Ma. Cecilia Victoria Arellano, Joshua Santillan, Sheena Jasley Samonte, Anne Julienne Marfori, Anna Melissa Guerrero, Marc James delos Santos, Catherine Manuela Lee-Ramos, Agnette Peralta, Department of Health – Medical Device Unit, Health Technology Assessment Council
Introduction
Pursuant to the Universal Health Care Act of 2019, the Health Technology Assessment Council (HTAC) in the Philippines was mandated to make recommendations for government financing of health technologies, including medical devices. The development of a Philippine Essential Medical Device List (PEMDL) will serve as a guide for the procurement of medical devices and supplies in government health facilities and as the basis for creating a price reference index.
Methods
The HTAC and a team of medical device experts (the Expert Advisory Committee) developed a streamlined pathway and guidelines using a Delphi technique with the Philippine Food and Drug Administration (FDA) and the Department of Health (DOH) to determine the necessary criteria for fast tracking the inclusion of medical devices. The pathway was also revised throughout a pilot processing of commonly procured medical devices (e.g., consumables) that were either already covered by existing national health insurance packages or funded by DOH programs, among others.
Results
The final pathway consisted of validating whether the device was already approved by the Philippine FDA and whether it was already part of standard of care. Similar to the usual HTA nomination requirement, the local FDA approval attested to the safety and quality of the device. Meanwhile, the inclusion of the device in the standard of care guaranteed that it was essential in the healthcare setting. This can be written in the World Health Organization technical documents and databases such as the Medical Devices Information System, health facility listing requirements, or locally adopted clinical practice guidelines. The initial PEMDL for release contained a total of 307 medical devices across 16 categories.
Conclusions
Unlike the mandate for the Philippine National Formulary, government health facilities are not yet required to base their procurement on the PEMDL because the list is still in its infancy. Moving forward, the list will include big ticket items and will be updated through consultations with specialty centers and hospitals.
导言根据 2019 年《全民医疗保健法》,菲律宾卫生技术评估委员会(HTAC)的任务是就政府对医疗技术(包括医疗器械)的资助提出建议。菲律宾基本医疗器械清单(PEMDL)的制定将作为政府卫生机构采购医疗器械和用品的指南,并作为创建价格参考指数的基础。方法菲律宾卫生技术评估委员会和医疗器械专家团队(专家咨询委员会)采用德尔菲技术,与菲律宾食品药品管理局(FDA)和卫生部(DOH)共同制定了简化路径和指南,以确定快速纳入医疗器械的必要标准。结果最终的路径包括验证设备是否已经获得菲律宾 FDA 的批准,以及是否已经成为医疗标准的一部分。与通常的 HTA 提名要求类似,当地 FDA 的批准证明了设备的安全性和质量。与此同时,将该设备纳入医疗标准则保证了它在医疗环境中的重要性。这可以写入世界卫生组织的技术文件和数据库(如医疗器械信息系统)、医疗机构列表要求或当地采用的临床实践指南。结论 与《菲律宾国家处方集》的规定不同,政府卫生机构尚未被要求根据《菲律宾国家处方集》进行采购,因为该清单仍处于起步阶段。今后,该清单将包括大型项目,并将通过与专科中心和医院的协商进行更新。
{"title":"OP107 Streamlining Health Technology Assessment Of Medical Devices Through Development Of The Philippine Essential Medical Device List","authors":"Patrick Wincy Reyes, Lara Alyssa Liban, Ma. Cecilia Victoria Arellano, Joshua Santillan, Sheena Jasley Samonte, Anne Julienne Marfori, Anna Melissa Guerrero, Marc James delos Santos, Catherine Manuela Lee-Ramos, Agnette Peralta, Department of Health – Medical Device Unit, Health Technology Assessment Council","doi":"10.1017/s0266462323001198","DOIUrl":"https://doi.org/10.1017/s0266462323001198","url":null,"abstract":"<span>Introduction</span><p>Pursuant to the Universal Health Care Act of 2019, the Health Technology Assessment Council (HTAC) in the Philippines was mandated to make recommendations for government financing of health technologies, including medical devices. The development of a Philippine Essential Medical Device List (PEMDL) will serve as a guide for the procurement of medical devices and supplies in government health facilities and as the basis for creating a price reference index.</p><span>Methods</span><p>The HTAC and a team of medical device experts (the Expert Advisory Committee) developed a streamlined pathway and guidelines using a Delphi technique with the Philippine Food and Drug Administration (FDA) and the Department of Health (DOH) to determine the necessary criteria for fast tracking the inclusion of medical devices. The pathway was also revised throughout a pilot processing of commonly procured medical devices (e.g., consumables) that were either already covered by existing national health insurance packages or funded by DOH programs, among others.</p><span>Results</span><p>The final pathway consisted of validating whether the device was already approved by the Philippine FDA and whether it was already part of standard of care. Similar to the usual HTA nomination requirement, the local FDA approval attested to the safety and quality of the device. Meanwhile, the inclusion of the device in the standard of care guaranteed that it was essential in the healthcare setting. This can be written in the World Health Organization technical documents and databases such as the Medical Devices Information System, health facility listing requirements, or locally adopted clinical practice guidelines. The initial PEMDL for release contained a total of 307 medical devices across 16 categories.</p><span>Conclusions</span><p>Unlike the mandate for the Philippine National Formulary, government health facilities are not yet required to base their procurement on the PEMDL because the list is still in its infancy. Moving forward, the list will include big ticket items and will be updated through consultations with specialty centers and hospitals.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138689385","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323000612
Liu Liu, Yi Yang, Dunming Xiao, Yingyao Chen
IntroductionTreatment with atezolizumab plus standard chemotherapy can prolong the overall survival of patients with metastatic non-squamous non-small cell lung cancer (NSCLC). However, the economic value of this treatment regimen is unknown. This study aimed to estimate the cost effectiveness of atezolizumab plus chemotherapy in the first-line treatment of metastatic non-squamous NSCLC from a healthcare system perspective in China.MethodsA partitioned survival model consisting of three discrete health states was developed to estimate the cost and effectiveness of atezolizumab plus carboplatin or cisplatin plus pemetrexed (APP) versus carboplatin or cisplatin plus pemetrexed (PP) in the first-line treatment of metastatic non-squamous NSCLC over a 12-year lifetime horizon. Key clinical data were generated from the IMpower132 trial. Local direct medical and non-medical costs were used and health preference data were collected from patients with NSCLC in 13 tertiary hospitals across five provinces in China. Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were measured. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the model.ResultsCompared with the PP regimen, APP therapy yielded a gain of 0.21 QALYs at an increased cost of CNY145,602 (USD22,574), resulting in an ICER of CNY684,894 (USD106,185) per QALY gained. The ICER was significantly higher than three times the gross domestic product per capita for China in 2021 (USD37,663). One-way sensitivity analyses revealed that one of the most influential factors in this model was the cost of atezolizumab. Probabilistic sensitivity analysis showed that there was 14.7% probability that atezolizumab plus chemotherapy was cost effective at a willingness-to-pay value of CNY242,928 (USD37,663) per QALY gained.ConclusionsThe APP regimen could prolong survival and improve health benefits over standard chemotherapy in the first-line treatment of patients with metastatic non-squamous NSCLC, but it is unlikely to be a cost-effective treatment option in China.
{"title":"OP11 Cost-Effectiveness Of Atezolizumab Plus Chemotherapy As A First-Line Treatment For Metastatic Non-Squamous Non-Small Cell Lung Cancer","authors":"Liu Liu, Yi Yang, Dunming Xiao, Yingyao Chen","doi":"10.1017/s0266462323000612","DOIUrl":"https://doi.org/10.1017/s0266462323000612","url":null,"abstract":"IntroductionTreatment with atezolizumab plus standard chemotherapy can prolong the overall survival of patients with metastatic non-squamous non-small cell lung cancer (NSCLC). However, the economic value of this treatment regimen is unknown. This study aimed to estimate the cost effectiveness of atezolizumab plus chemotherapy in the first-line treatment of metastatic non-squamous NSCLC from a healthcare system perspective in China.MethodsA partitioned survival model consisting of three discrete health states was developed to estimate the cost and effectiveness of atezolizumab plus carboplatin or cisplatin plus pemetrexed (APP) versus carboplatin or cisplatin plus pemetrexed (PP) in the first-line treatment of metastatic non-squamous NSCLC over a 12-year lifetime horizon. Key clinical data were generated from the IMpower132 trial. Local direct medical and non-medical costs were used and health preference data were collected from patients with NSCLC in 13 tertiary hospitals across five provinces in China. Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were measured. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the model.ResultsCompared with the PP regimen, APP therapy yielded a gain of 0.21 QALYs at an increased cost of CNY145,602 (USD22,574), resulting in an ICER of CNY684,894 (USD106,185) per QALY gained. The ICER was significantly higher than three times the gross domestic product per capita for China in 2021 (USD37,663). One-way sensitivity analyses revealed that one of the most influential factors in this model was the cost of atezolizumab. Probabilistic sensitivity analysis showed that there was 14.7% probability that atezolizumab plus chemotherapy was cost effective at a willingness-to-pay value of CNY242,928 (USD37,663) per QALY gained.ConclusionsThe APP regimen could prolong survival and improve health benefits over standard chemotherapy in the first-line treatment of patients with metastatic non-squamous NSCLC, but it is unlikely to be a cost-effective treatment option in China.","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138689386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323001265
Lauren Pretorius, Debjani Mueller, Anke Peggy Holtorf
IntroductionIn building health technology assessment (HTA) and related decision processes in Southern Africa, institutions and stakeholders face region-specific challenges such as disease prevalence and population makeup. These can be addressed by collaboratively discussing patient engagement solutions that fit the local culture and systems and serve to ensure equitable and sustainable access to patient-relevant health technologies. Our aim is to initiate a collaboration for driving patient involvement (PI) suitable for the Southern African context and Sub-Saharan patient communities. In addition, we explore current experiences in PI, including the stakeholder expectations, gaps, limitations, and new opportunities.MethodsA one-day hybrid multi-stakeholder PI in HTA workshop was held in Johannesburg, South Africa. Co-created by the participants, the outputs are a call to action and a concept draft for the vital success criteria for PI in the region. The content of the call to action is gathered from pre-workshop surveys, interviews, and outcomes from historic meetings held in conjunction with the Health Technology Assessment International (HTAi) PI workstream as well as facilitated discussion from the actual workshop.ResultsThe workshop was attended by 42 participants from nine countries, representing diverse stakeholder groups. The attendees represented multiple PI stakeholder groups. The workshop survey was completed by 44 respondents, while 12 participants completed the post-event survey. A workshop outcomes document highlighting a high level of alignment and identifying seven key success factors was developed. A workshop proceeding detailing the outcomes is now being drafted.ConclusionsOver 95 percent of respondents to pre-and post-surveys indicated an interest in contributing to a more in-depth description of PI in their country. While the majority of participants were from South Africa, participants from Tanzania, Ethiopia, Zambia, and Zimbabwe emphasized that trans-African-engagement for HTA will provide an additional opportunity for HTA in Africa and patient and community participation in HTA and healthcare decision-making. Hence, a collaborative platform could help all African countries to advance and benefit from improved healthcare decision processes.
导言在南部非洲建立卫生技术评估(HTA)和相关决策流程时,机构和利益相关者面临着疾病流行和人口构成等地区特有的挑战。要解决这些问题,可以通过合作讨论适合当地文化和系统的患者参与解决方案,以确保公平、可持续地获取与患者相关的卫生技术。我们的目标是开展合作,推动适合南部非洲环境和撒哈拉以南地区患者社区的患者参与(PI)。此外,我们还探讨了目前患者参与方面的经验,包括利益相关者的期望、差距、限制和新机遇。方法在南非约翰内斯堡举办了为期一天的混合型多利益相关者患者参与 HTA 研讨会。与会者共同起草了一份行动呼吁书和一份该地区 PI 重要成功标准的概念草案。行动呼吁的内容来自于研讨会前的调查、访谈、与国际卫生技术评估(HTAi)PI 工作流共同举行的历史性会议的成果,以及实际研讨会上的协助讨论。与会者代表了多个 PI 利益相关者群体。44 名受访者完成了研讨会调查,12 名与会者完成了会后调查。研讨会编制了一份成果文件,强调了高度一致,并确定了七个关键成功因素。结论95%以上的会前和会后调查答复者表示有兴趣为更深入地介绍本国的 PI 做出贡献。虽然大多数与会者来自南非,但来自坦桑尼亚、埃塞俄比亚、赞比亚和津巴布韦的与会者强调,跨非洲参与 HTA 将为非洲的 HTA 以及患者和社区参与 HTA 和医疗决策提供更多机会。因此,合作平台可帮助所有非洲国家推进医疗决策进程并从中受益。
{"title":"OP120 Developing A Call To Action For Patient Involvement In Health Technology Assessment (HTA) In Southern Africa","authors":"Lauren Pretorius, Debjani Mueller, Anke Peggy Holtorf","doi":"10.1017/s0266462323001265","DOIUrl":"https://doi.org/10.1017/s0266462323001265","url":null,"abstract":"IntroductionIn building health technology assessment (HTA) and related decision processes in Southern Africa, institutions and stakeholders face region-specific challenges such as disease prevalence and population makeup. These can be addressed by collaboratively discussing patient engagement solutions that fit the local culture and systems and serve to ensure equitable and sustainable access to patient-relevant health technologies. Our aim is to initiate a collaboration for driving patient involvement (PI) suitable for the Southern African context and Sub-Saharan patient communities. In addition, we explore current experiences in PI, including the stakeholder expectations, gaps, limitations, and new opportunities.MethodsA one-day hybrid multi-stakeholder PI in HTA workshop was held in Johannesburg, South Africa. Co-created by the participants, the outputs are a call to action and a concept draft for the vital success criteria for PI in the region. The content of the call to action is gathered from pre-workshop surveys, interviews, and outcomes from historic meetings held in conjunction with the Health Technology Assessment International (HTAi) PI workstream as well as facilitated discussion from the actual workshop.ResultsThe workshop was attended by 42 participants from nine countries, representing diverse stakeholder groups. The attendees represented multiple PI stakeholder groups. The workshop survey was completed by 44 respondents, while 12 participants completed the post-event survey. A workshop outcomes document highlighting a high level of alignment and identifying seven key success factors was developed. A workshop proceeding detailing the outcomes is now being drafted.ConclusionsOver 95 percent of respondents to pre-and post-surveys indicated an interest in contributing to a more in-depth description of PI in their country. While the majority of participants were from South Africa, participants from Tanzania, Ethiopia, Zambia, and Zimbabwe emphasized that trans-African-engagement for HTA will provide an additional opportunity for HTA in Africa and patient and community participation in HTA and healthcare decision-making. Hence, a collaborative platform could help all African countries to advance and benefit from improved healthcare decision processes.","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138689534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Health technology assessment (HTA) bodies support healthcare decision-making by producing different kind of products. The high speed of the healthcare innovations and the scenarios such as the COVID-19 pandemic challenge HTA organizations to adapt their services to better respond to these demands. The Spanish Network of HTA Agencies (RedETS) is redefining the services and the products in its portfolio. The first step has been conducting a review in order to identify the most relevant HTA products.
Methods
A scoping review with two sections was conducted: (i) analysis of results from a bibliographic search performed in the main biomedical databases; and (ii) analysis of results from a manual review of the official websites of seven international HTA agencies: CADTH (Canada), INESSS (Canada), SBU (Sweden), NICE (United Kingdom), IQWIG (Germany), HAS (France), IECS (Argentina) and IETS (Colombia). The EUnetHTA website was also reviewed.
Results
The search identified 1,311 references; 21 studies were considered relevant. The main topic found was about rapid responses services. The standard timeline for these should be less than six months, with even some produced in days. Transparency about methodology and involvement of decision-makers were considered key points to be included. Website analysis revealed similar HTA reports production but variation in the domains and elements considered. The timeframe for conducting a full HTA report can be up to 24 months, with a median of 12 months. Agencies also offer some kinds of rapid response services. Scientific consultation and horizon scanning systems for emerging technologies are other services performed by some agencies.
Conclusions
The review reveals that agencies have different products to address different needs throughout the life cycle of technologies: from scientific advice to full HTA. In addition, HTA agencies have incorporated rapid responses into their services. According to literature, these products could support short-term decision-making.
{"title":"PP133 What Services And Products Should A Health Technology Assessment Agency Provide?","authors":"Maria-Jose Faraldo-Valles, Maria-Carmen Maceira-Rozas, Beatriz Casal-Acción, Patricia Gomez, Yolanda Trinanes","doi":"10.1017/s0266462323002453","DOIUrl":"https://doi.org/10.1017/s0266462323002453","url":null,"abstract":"<span>Introduction</span><p>Health technology assessment (HTA) bodies support healthcare decision-making by producing different kind of products. The high speed of the healthcare innovations and the scenarios such as the COVID-19 pandemic challenge HTA organizations to adapt their services to better respond to these demands. The Spanish Network of HTA Agencies (RedETS) is redefining the services and the products in its portfolio. The first step has been conducting a review in order to identify the most relevant HTA products.</p><span>Methods</span><p>A scoping review with two sections was conducted: (i) analysis of results from a bibliographic search performed in the main biomedical databases; and (ii) analysis of results from a manual review of the official websites of seven international HTA agencies: CADTH (Canada), INESSS (Canada), SBU (Sweden), NICE (United Kingdom), IQWIG (Germany), HAS (France), IECS (Argentina) and IETS (Colombia). The EUnetHTA website was also reviewed.</p><span>Results</span><p>The search identified 1,311 references; 21 studies were considered relevant. The main topic found was about rapid responses services. The standard timeline for these should be less than six months, with even some produced in days. Transparency about methodology and involvement of decision-makers were considered key points to be included. Website analysis revealed similar HTA reports production but variation in the domains and elements considered. The timeframe for conducting a full HTA report can be up to 24 months, with a median of 12 months. Agencies also offer some kinds of rapid response services. Scientific consultation and horizon scanning systems for emerging technologies are other services performed by some agencies.</p><span>Conclusions</span><p>The review reveals that agencies have different products to address different needs throughout the life cycle of technologies: from scientific advice to full HTA. In addition, HTA agencies have incorporated rapid responses into their services. According to literature, these products could support short-term decision-making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631673","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323002192
Himani Jaiswal, Anna D’Ausilio, Matthew Bending
Introduction
Currently, no cure exists for the 1 in 2,500 Australian babies born with potentially fatal cystic fibrosis (CF). The authors conducted a health technology assessment (HTA) case study analysis of all regulatory approved CF treatments in Australia from January 1994 to July 2022. Submissions were also made under the Therapeutics Goods Administration and Pharmaceutical Benefits Advisory Committee (TGA-PBAC) parallel process.
Methods
Public summary and source materials were researched to understand relevant clinical and health economic evidence requirements, and access decisions from Australia’s lead HTA body, PBAC.
Results
The review found that there are more than seven approved products in Australia. Of those, all four novel CF transmembrane conductance regulator (CFTR) modulating medications, which treat the underlying disease, received an orphan drug designation and were eventually listed. However, initial HTA decisions were mixed, with one recommended (25%), one not recommended (25%), and two deferred (50%). Clinical efficacy, cost-effectiveness, clinical need, as well as patient/carer-centric perspectives were most influential in HTA recommendations. Like other rare disease treatments, price, high incremental cost-effectiveness ratios (ICERs), uncertainty around cost-effectiveness and/or efficacy were key barriers to positive decisions. Notably, Australian stakeholders did not recommend CF medicines when their ICERs significantly exceeded a threshold of AUD200,000 (USD134,700) per quality-adjusted life year (QALY) gained. Administratively, Australia addresses risks associated with poor cost-effectiveness and high costs through managed access programs, risk-sharing agreements (RSA) and special pricing arrangements.
Recently approved elexacaftor-tezacaftor-ivacaftor would be inaccessible to many Australian patients without inclusion in the Pharmaceutical Benefits Scheme (PBS); this placement increases access by limiting patients’ payments to AUD42.50 (USD28.62) maximum per prescription. Alternatively, manufacturers of therapies for other chronic or rare life-threatening conditions can participate in Australia’s Highly Specialised Drugs Program and/or Life Saving Drugs Program to facilitate access.
Conclusions
Companies can accelerate and optimize market access by using the TGA-PBAC parallel process. Other Asia-Pacific countries can model components of Australia’s approach to advancing access to innovative, live-saving therapies.
{"title":"PP93 Health Technology Assessments For Rare Diseases In Australia: A Case Study On Cystic Fibrosis","authors":"Himani Jaiswal, Anna D’Ausilio, Matthew Bending","doi":"10.1017/s0266462323002192","DOIUrl":"https://doi.org/10.1017/s0266462323002192","url":null,"abstract":"<span>Introduction</span><p>Currently, no cure exists for the 1 in 2,500 Australian babies born with potentially fatal cystic fibrosis (CF). The authors conducted a health technology assessment (HTA) case study analysis of all regulatory approved CF treatments in Australia from January 1994 to July 2022. Submissions were also made under the Therapeutics Goods Administration and Pharmaceutical Benefits Advisory Committee (TGA-PBAC) parallel process.</p><span>Methods</span><p>Public summary and source materials were researched to understand relevant clinical and health economic evidence requirements, and access decisions from Australia’s lead HTA body, PBAC.</p><span>Results</span><p>The review found that there are more than seven approved products in Australia. Of those, all four novel CF transmembrane conductance regulator (CFTR) modulating medications, which treat the underlying disease, received an orphan drug designation and were eventually listed. However, initial HTA decisions were mixed, with one recommended (25%), one not recommended (25%), and two deferred (50%). Clinical efficacy, cost-effectiveness, clinical need, as well as patient/carer-centric perspectives were most influential in HTA recommendations. Like other rare disease treatments, price, high incremental cost-effectiveness ratios (ICERs), uncertainty around cost-effectiveness and/or efficacy were key barriers to positive decisions. Notably, Australian stakeholders did not recommend CF medicines when their ICERs significantly exceeded a threshold of AUD200,000 (USD134,700) per quality-adjusted life year (QALY) gained. Administratively, Australia addresses risks associated with poor cost-effectiveness and high costs through managed access programs, risk-sharing agreements (RSA) and special pricing arrangements.</p><p>Recently approved elexacaftor-tezacaftor-ivacaftor would be inaccessible to many Australian patients without inclusion in the Pharmaceutical Benefits Scheme (PBS); this placement increases access by limiting patients’ payments to AUD42.50 (USD28.62) maximum per prescription. Alternatively, manufacturers of therapies for other chronic or rare life-threatening conditions can participate in Australia’s Highly Specialised Drugs Program and/or Life Saving Drugs Program to facilitate access.</p><span>Conclusions</span><p>Companies can accelerate and optimize market access by using the TGA-PBAC parallel process. Other Asia-Pacific countries can model components of Australia’s approach to advancing access to innovative, live-saving therapies.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323002362
Miyoung Choi, Jimin Kim, Byung Min Choi, Jeonghee Shin, Chanmi Park, Gisu Ha, Hong Joo Shin, Eui Kyung Choi
Introduction
Critical congenital heart disease (CCHD) refers to a group of heart defects that cause serious, life-threatening symptoms in the neonatal period and requires timely surgical or catheter interventions. We reviewed evidence for incorporating a mandatory neonatal CCHD screening test as a national public health project for all neonates born in Korea by analyzing the validity and cost-effectiveness of neonatal CCHD screening using pulse oximetry in Korea.
Methods
We performed a rapid literature review to establish models for the diagnostic accuracy and economic evaluation of pulse oximetry. Also, we analyzed the prevalence, mortality, and medical expenditure for different types of CCHD using the national health insurance (NHI) data. We analyzed the cost-effectiveness of pulse oximetry by comparing the group of neonates who received a combination of a physical examination and pulse oximetry, and group of neonates who only received a physical examination. For the cost-effectiveness analysis for the CCHD screening test in this study, we used a duration of one year, diagnostic accuracy as the clinical endpoint, and Life Year Gain (LYG) as the effectiveness indicator.
Results
We used recent systematic review he pooled sensitivity can be enhanced from 76.5 percent (pulse oximetry alone) to 92 percent (combined with physical examination). We used a total of 2,334 neonates with CCHD data for the economic model. Our analysis revealed that adding pulse oximetry to the routine neonatal physical examination leads to 2.34 of LYG and a cost difference of USD1,080,602, showing a ICER of KRW610,063,240 (USD461,857)/LYG.
Conclusions
Considering the benefit of LYG and cost of reducing the complications and after effects of newborns with CCHD who survived early diagnosis, it is considered to be worthwhile in Korea for a mandatory screening test.
{"title":"PP122 The Feasibility Assessment For Domestic Introduction Of Newborn Pulse Oximetry Screening For Critical Congenital Heart Disease","authors":"Miyoung Choi, Jimin Kim, Byung Min Choi, Jeonghee Shin, Chanmi Park, Gisu Ha, Hong Joo Shin, Eui Kyung Choi","doi":"10.1017/s0266462323002362","DOIUrl":"https://doi.org/10.1017/s0266462323002362","url":null,"abstract":"<span>Introduction</span><p>Critical congenital heart disease (CCHD) refers to a group of heart defects that cause serious, life-threatening symptoms in the neonatal period and requires timely surgical or catheter interventions. We reviewed evidence for incorporating a mandatory neonatal CCHD screening test as a national public health project for all neonates born in Korea by analyzing the validity and cost-effectiveness of neonatal CCHD screening using pulse oximetry in Korea.</p><span>Methods</span><p>We performed a rapid literature review to establish models for the diagnostic accuracy and economic evaluation of pulse oximetry. Also, we analyzed the prevalence, mortality, and medical expenditure for different types of CCHD using the national health insurance (NHI) data. We analyzed the cost-effectiveness of pulse oximetry by comparing the group of neonates who received a combination of a physical examination and pulse oximetry, and group of neonates who only received a physical examination. For the cost-effectiveness analysis for the CCHD screening test in this study, we used a duration of one year, diagnostic accuracy as the clinical endpoint, and Life Year Gain (LYG) as the effectiveness indicator.</p><span>Results</span><p>We used recent systematic review he pooled sensitivity can be enhanced from 76.5 percent (pulse oximetry alone) to 92 percent (combined with physical examination). We used a total of 2,334 neonates with CCHD data for the economic model. Our analysis revealed that adding pulse oximetry to the routine neonatal physical examination leads to 2.34 of LYG and a cost difference of USD1,080,602, showing a ICER of KRW610,063,240 (USD461,857)/LYG.</p><span>Conclusions</span><p>Considering the benefit of LYG and cost of reducing the complications and after effects of newborns with CCHD who survived early diagnosis, it is considered to be worthwhile in Korea for a mandatory screening test.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631812","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323002398
Ada PM Batcagan-Abueg, Swee Sung Soon, Hong Ju, Kwong Ng
Introduction
Medical technologies are evolving rapidly, with many new and expensive technologies entering the market constantly, challenging the sustainability of the public healthcare system. Early health technology assessment (HTA) to inform subsidy decision for innovative medical technologies, before they diffuse into the public healthcare system, may drive appropriate early adoption or curtail inappropriate use. This abstract describes the Agency for Care Effectiveness (ACE)’s experience in conducting early HTAs and key challenges faced.
Methods
During ACE’s 2021 topic prioritization exercise, ACE took a proactive approach by considering medical technologies identified from horizon scanning (HS) for subsidy evaluation. Two topics were shortlisted from HS. Standard HTA evaluation framework and local clinician consultation were used to define the evaluation scope and clinical pathways. Literature search and appraisal were conducted for safety, effectiveness, and economic evidence. Budget impact estimations and organizational feasibility assessment were additional domains considered for subsidy decision-making by the Ministry of Health Medical Technology Advisory Committee (MTAC).
Results
MTAC did not recommend subsidy for the two technologies due to weak evidence base, largely due to a lack of comparative evidence, small samples, short-term follow-ups, or heterogeneity of population. Additional considerations included potentially high budget impact or organizational feasibility issues such as substantial capital and maintenance cost and infrastructure reconfiguration required. During the evaluation, key challenges of assessing such technologies in their early diffusion within the healthcare system were: (i) differing clinical opinions on whether the technology meets an unmet need; (ii) uncertain place in the clinical management algorithm for the relevant indications; (iii) sparse and weak evidence; (iv) uncertain financial implications to the healthcare system due to a lack of available local costs.
Conclusions
Early HTA on medical technologies identified from HS can be a useful tool to guide subsidy decisions; however, several challenges exist. Careful selection of technologies and timing of evaluation are critical. Seeking stakeholder inputs earlier would ensure shortlisting appropriate technologies with greater clinical need for HTA.
{"title":"PP127 Early Health Technology Assessment (HTA) Of Medical Technologies To Inform Subsidy Decision-making In Singapore","authors":"Ada PM Batcagan-Abueg, Swee Sung Soon, Hong Ju, Kwong Ng","doi":"10.1017/s0266462323002398","DOIUrl":"https://doi.org/10.1017/s0266462323002398","url":null,"abstract":"<span>Introduction</span><p>Medical technologies are evolving rapidly, with many new and expensive technologies entering the market constantly, challenging the sustainability of the public healthcare system. Early health technology assessment (HTA) to inform subsidy decision for innovative medical technologies, before they diffuse into the public healthcare system, may drive appropriate early adoption or curtail inappropriate use. This abstract describes the Agency for Care Effectiveness (ACE)’s experience in conducting early HTAs and key challenges faced.</p><span>Methods</span><p>During ACE’s 2021 topic prioritization exercise, ACE took a proactive approach by considering medical technologies identified from horizon scanning (HS) for subsidy evaluation. Two topics were shortlisted from HS. Standard HTA evaluation framework and local clinician consultation were used to define the evaluation scope and clinical pathways. Literature search and appraisal were conducted for safety, effectiveness, and economic evidence. Budget impact estimations and organizational feasibility assessment were additional domains considered for subsidy decision-making by the Ministry of Health Medical Technology Advisory Committee (MTAC).</p><span>Results</span><p>MTAC did not recommend subsidy for the two technologies due to weak evidence base, largely due to a lack of comparative evidence, small samples, short-term follow-ups, or heterogeneity of population. Additional considerations included potentially high budget impact or organizational feasibility issues such as substantial capital and maintenance cost and infrastructure reconfiguration required. During the evaluation, key challenges of assessing such technologies in their early diffusion within the healthcare system were: (i) differing clinical opinions on whether the technology meets an unmet need; (ii) uncertain place in the clinical management algorithm for the relevant indications; (iii) sparse and weak evidence; (iv) uncertain financial implications to the healthcare system due to a lack of available local costs.</p><span>Conclusions</span><p>Early HTA on medical technologies identified from HS can be a useful tool to guide subsidy decisions; however, several challenges exist. Careful selection of technologies and timing of evaluation are critical. Seeking stakeholder inputs earlier would ensure shortlisting appropriate technologies with greater clinical need for HTA.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631813","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323002490
Silvana Kelles, Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, Lélia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Borin
Introduction
The Corona Virus Disease 2019 (COVID-19) pandemic has impacted the functioning of health systems, imposing the need for adaptations. Elective surgeries also needed to adapt, and research has shown higher mortality in newly infected surgical patients after or during procedures. Thus, was recommended the suspension of elective surgeries during the pandemic. Early studies evaluating the effect of COVID-19 pandemic on bariatric surgery have reported a substantial reduction in procedures performed.
Methods
This retrospective study evaluated the impact of the suspension of bariatric surgeries for a Brazilian Health Maintenance Organization: UNIMED-BH, based on the analysis of data from before and during the pandemic of COVID-19.
Results
There were 2,641 bariatric procedures conducted in 2019 with a 14.1 percent reduction in volume to 2,314 procedures in 2020. In 2021, there were 2,813 bariatric procedures and 1,700 procedures were observed from January to August 2022. Therefore, it appears that in 2022 the demand for bariatric procedures will be similar to the year 2019, which was before the COVID-19 pandemic.
Conclusions
From the analysis of the data, a decrease in bariatric surgical volume was evidenced during the year 2020 when compared to 2019. Post-pandemic, monitoring is necessary to assess whether the system was able to meet the demand for bariatric surgical procedures.
{"title":"PP145 The Impacts Of The Corona Virus Disease 2019 Pandemic On Bariatric Surgeries In The Private Healthcare In Brazil","authors":"Silvana Kelles, Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, Lélia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Borin","doi":"10.1017/s0266462323002490","DOIUrl":"https://doi.org/10.1017/s0266462323002490","url":null,"abstract":"<span>Introduction</span><p>The Corona Virus Disease 2019 (COVID-19) pandemic has impacted the functioning of health systems, imposing the need for adaptations. Elective surgeries also needed to adapt, and research has shown higher mortality in newly infected surgical patients after or during procedures. Thus, was recommended the suspension of elective surgeries during the pandemic. Early studies evaluating the effect of COVID-19 pandemic on bariatric surgery have reported a substantial reduction in procedures performed.</p><span>Methods</span><p>This retrospective study evaluated the impact of the suspension of bariatric surgeries for a Brazilian Health Maintenance Organization: UNIMED-BH, based on the analysis of data from before and during the pandemic of COVID-19.</p><span>Results</span><p>There were 2,641 bariatric procedures conducted in 2019 with a 14.1 percent reduction in volume to 2,314 procedures in 2020. In 2021, there were 2,813 bariatric procedures and 1,700 procedures were observed from January to August 2022. Therefore, it appears that in 2022 the demand for bariatric procedures will be similar to the year 2019, which was before the COVID-19 pandemic.</p><span>Conclusions</span><p>From the analysis of the data, a decrease in bariatric surgical volume was evidenced during the year 2020 when compared to 2019. Post-pandemic, monitoring is necessary to assess whether the system was able to meet the demand for bariatric surgical procedures.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631821","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-14DOI: 10.1017/s0266462323002283
Silvana Kelles, Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, Lélia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Borin
Introduction
Most new drugs have only clinical studies focused on a single population at the time of first registration, hence their indications for use are restricted to this population. For clinical conditions when there are no other treatments available, new drugs have higher costs in Brazil. There is no review of prices when these medications broaden their therapeutic areas, and this can have a significant financial impact. This study’s objective is to assess the financial implications of pembrolizumab’s incremental indication after its initial registration.
Methods
We calculated the annual cost to treat all Brazilian patients with indications for use in the first registration and all incremental indications of pembrolizumb. Populations were estimated by epidemiological data from the pembrolizumab clinical trials called, KEYNOTE studies, and the INCA 2023 cancer estimate for the Brazilian population. Costs were calculated by CMED-ANVISA price value and considering the dosing of 200mg every 3 weeks.
Results
In 2016, pembrolizumab was granted registration in Brazil was restricted to patients with advanced melanoma. In 2022 the indication was expanded to more than 20 new indications, with several studies in progress that potentially will lead to further inclusions. The estimate of patients eligible for indications increase of 1,796 to 99,544 patients with an increased total cost from BRL625,802,837 to BRL34,685,366,192 (USD121,185,677.4 to USD6,716,763,399.04).
Conclusions
The financial burden of pembrolizumab’s expanded uses after it was first approved could significantly rise, endangering the long-term viability of healthcare systems. In Brazil, where medicine costs are not regularly monitored, the annual inflation adjustment is the only factor that causes prices to change. In order to lower medicine prices in response to the addition of new indications, the expansion of therapeutic options for the same condition, or even obsolescence, regulations are required.
{"title":"PP104 Impact Of New Permbrolizumab Indications After Initial Registration By Brazilian Health Regulatory Agency (ANVISA)","authors":"Silvana Kelles, Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, Lélia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Borin","doi":"10.1017/s0266462323002283","DOIUrl":"https://doi.org/10.1017/s0266462323002283","url":null,"abstract":"<span>Introduction</span><p>Most new drugs have only clinical studies focused on a single population at the time of first registration, hence their indications for use are restricted to this population. For clinical conditions when there are no other treatments available, new drugs have higher costs in Brazil. There is no review of prices when these medications broaden their therapeutic areas, and this can have a significant financial impact. This study’s objective is to assess the financial implications of pembrolizumab’s incremental indication after its initial registration.</p><span>Methods</span><p>We calculated the annual cost to treat all Brazilian patients with indications for use in the first registration and all incremental indications of pembrolizumb. Populations were estimated by epidemiological data from the pembrolizumab clinical trials called, KEYNOTE studies, and the INCA 2023 cancer estimate for the Brazilian population. Costs were calculated by CMED-ANVISA price value and considering the dosing of 200mg every 3 weeks.</p><span>Results</span><p>In 2016, pembrolizumab was granted registration in Brazil was restricted to patients with advanced melanoma. In 2022 the indication was expanded to more than 20 new indications, with several studies in progress that potentially will lead to further inclusions. The estimate of patients eligible for indications increase of 1,796 to 99,544 patients with an increased total cost from BRL625,802,837 to BRL34,685,366,192 (USD121,185,677.4 to USD6,716,763,399.04).</p><span>Conclusions</span><p>The financial burden of pembrolizumab’s expanded uses after it was first approved could significantly rise, endangering the long-term viability of healthcare systems. In Brazil, where medicine costs are not regularly monitored, the annual inflation adjustment is the only factor that causes prices to change. In order to lower medicine prices in response to the addition of new indications, the expansion of therapeutic options for the same condition, or even obsolescence, regulations are required.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631826","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}