International Journal of Technology Assessment in Health Care最新文献

{"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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"title":"PP147 What Does Real World Evidence (RWE) Offer Health Technology Assessment (HTA) Procedures In Australia?","authors":"Gabrielle Challis, Michelle Hill, Liesl Strachan, Kate King, Shajedur Rahman Shawon, Oluwadamisola Sotade, Louisa Jorm","doi":"10.1017/s0266462323002519","DOIUrl":"https://doi.org/10.1017/s0266462323002519","url":null,"abstract":"<span>Introduction</span><p>Medical device health technology assessment (HTA) in Australia is largely coordinated by the Medical Services Advisory Committee (MSAC). Its remit to improve the public’s health by deciding where to allocate public healthcare funding, can be enhanced by considering real world evidence (RWE). Existing data sources have limitations that can be addressed through RWE, including coverage of Australian patient populations who may not meet trial eligibility criteria, and long-term follow-up through data linkage and datasets. We partnered with a university to explore what information could be gained from an analysis of linked administrative patient data, with a view to addressing current evidence gaps and/or limitations. The findings can be used as a source of local data to define patient populations, estimate actual costs of care, and enable more comprehensive economic modeling to inform medical device HTA.</p><span>Methods</span><p>The University-developed New South Wales Cardiovascular Cohort dataset, comprising person-level longitudinal NSW administrative data for all patients admitted to hospital with a cardiovascular diagnosis from 2001 onwards, linked to national Medicare Benefits Schedule and Pharmaceutical Benefits Scheme claims data, was interrogated.</p><span>Results</span><p>Working with RWE is resource intensive in terms of time and costs. The potential of these data was revealed as the research progressed. It was possible to continually refine the data analyzed and reported,as well as expand the data requested. Varied expertise is required to accurately analyze the administrative datasets, particularly clinical classification skills and expertise in methods for causal inference using observational data. Findings from this study will enable the refinement of information for MSAC submissions, including identifying the most relevant patient population and reporting comprehensive costs, beyond an admitted hospital setting. The data will enhance engagement with clinicians and refine messaging, for example regarding patient risk factors.</p><span>Conclusions</span><p>RWE enhances Australian HTA applications. Local data, extended periods of time and insights not apparent from a focus on admitted hospital episodes can be revealed. Data can be refined during the process for specificity and applicability.</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":"138631827","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}

{"title":"PP64 Cost-Effectiveness Of Fractional Flow Reserve As Diagnostic Tool In Coronary Artery Disease Versus Angiogram Alone In Indian context","authors":"Monika Pusha, Arif Fahim, Kirti Kataria","doi":"10.1017/s0266462323002015","DOIUrl":"https://doi.org/10.1017/s0266462323002015","url":null,"abstract":"<span>Introduction</span><p>Fractional Flow Reserve (FFR) is a diagnostic tool that aids decision-making in the treatment of coronary artery disease (CAD). FFR provides an objective measurement and is used as an adjunct to an angiogram. The clinical and cost-benefit of using FFR have been well established across published literature. This research was aimed at evaluating the economic impact of using FFR as an adjunct to angiogram versus an angiogram alone, in the Indian healthcare context.</p><span>Methods</span><p>A study from a tertiary care public hospital in India estimated the impact of using FFR as an adjunct to angiogram in management of CAD. This study was used to create a mathematical simulation model to estimate cost-effectiveness and economic impact of using FFR over seven years’ time horizon, from the Indian health systems perspective. A targeted literature review was performed to collect the clinical inputs for the model, and the national public health insurance program data was referenced to obtain the cost inputs.</p><span>Results</span><p>A hypothetical cohort of 100,000 patients in the model reported 30 percent reduction in unnecessary stenting. Moreover, 14,025 deaths were averted with the adoption of FFR. In addition, there was a cost-saving of INR46,986 (574USD) per death averted and INR5,169 (63USD) per patient treated over a seven-year time horizon. The analysis demonstrated that FFR inclusion in the current clinical practice saves INR2,651 (32USD) per patient in overall upfront cost and INR2,518 (31USD) per patient in overall follow-up cost over a seven year follow-up period owing to improved diagnosis and prognosis.</p><span>Conclusions</span><p>In conclusion, FFR prevents unnecessary stenting, reduces overall mortality, and proves to be a cost-saving intervention in the long-term when used as a decision-making criterion in CAD patients in the Indian context.</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":"138631954","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}

{"title":"PP155 Should Breast Cancer Patients Avoid Venipuncture In The Ipsilateral Arm? A Rapid Review Of The Evidence","authors":"Keng Ho Pwee","doi":"10.1017/s0266462323002568","DOIUrl":"https://doi.org/10.1017/s0266462323002568","url":null,"abstract":"<span>Introduction</span><p>This rapid review clarified the evidence supporting avoidance of venipuncture on the ipsilateral arm in breast cancer patients who have had sentinel lymph node biopsy (SLNB) or axillary lymph node clearance (ALNC), as a preventive measure against lymphoedema.</p><span>Methods</span><p>A systematic search was carried out for systematic reviews with the following elements:</p><ul><li><p><span>•</span> Population – breast cancer patients who had SLNB or ALNC</p></li><li><p><span>•</span> Intervention – avoidance of venipuncture in the ipsilateral arm</p></li><li><p><span>•</span> Comparator –use of either arm for venipuncture</p></li><li><p><span>•</span> Outcomes – risk of lymphoedema in the ipsilateral arm</p></li></ul><p></p><p>Databases searched included PubMed (MEDLINE), Epistemonikos and the Cochrane Database of Systematic Reviews. Included reviews were critically appraised with the AMSTAR2 instrument and the primary studies were extracted and tabulated in a narrative synthesis.</p><span>Results</span><p>Six reviews were included; none of the reviews self-identified as systematic reviews in their titles/abstracts. Four reviews did report methods, including systematic search strategies and describing studies in adequate detail. However, all reviews did not meet most criteria on the AMSTAR2 checklist. The reviews concluded that the evidence base for avoiding venipuncture was inconsistent. An evidence table was consequently drawn up of the primary studies included in the reviews as a narrative synthesis of the primary evidence base.</p><p>The primary evidence base comprised 12 observational studies – six prospective cohort or descriptive studies and 6 retrospective studies. These studies were inconsistent and inconclusive; studies that found an association or reported cases following ipsilateral venipuncture were subject to recall bias or other potential confounders. Guidelines or patient information recommending avoidance of ipsilateral venipuncture do so based on expert opinion rather than consistent findings from empirical studies.</p><span>Conclusions</span><p>All reviews concluded that the evidence base for avoiding venipuncture was inconsistent. Review authors consistently recognized there was no strong basis for the prevalent recommendations to avoid ipsilateral venipuncture to prevent lymphoedema. Such recommendations lead to unnecessary measures that may be detrimental to patients. Stakeholders should reconsider advice to patients in the light of existing evidence and weigh up the uncertain benefits against potential harm to patients.</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":"138631963","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}

{"title":"PP125 Why Understanding The Burden Of The Population Is Fundamental","authors":"Franziska J. Schöb, Julie Hviid Hahn-Pedersen","doi":"10.1017/s0266462323002386","DOIUrl":"https://doi.org/10.1017/s0266462323002386","url":null,"abstract":"<span>Introduction</span><p>Despite intense efforts in development of new treatments over the last two decades, symptomatic treatments remain the only option for the vast majority patients diagnosed with dementia due to Alzheimer’s disease (AD). There remains a significant unmet need for disease modifying therapies (DMTs) to slow or stop AD progression. DMTs in development are targeting early stages of AD (pre-clinical, mild cognitive impairment and mild dementia stages), thereby creating an entirely new treatment paradigm for patients, clinicians, and payers. A key challenge will be in identifying the appropriate patient for treatment in a very heterogenous population. We have performed a literature review to better understand and define the AD population, with a view to enabling more targeted treatment in future.</p><span>Methods</span><p>Embase, MEDLINE and the Cochrane Library were searched to identify publications between 2010-2021 on observational studies reporting evidence on prevalence and subgroup identification, including clinical feasibility of identification. The search was restricted to English language.</p><span>Results</span><p>We identified 45 studies, mostly from Europe, USA and Asia. Populations were primarily grouped based on generic demographic factors (e.g., age, sex, gender), AD staging, comorbidities or biomarkers. Prevalence data was available for six subpopulations: pre-dementia stage, mild dementia, age, Apolipoprotein E (APOE) genotype, comorbid obesity and hypertension. Across these, data on prevalence were heterogenous depending on study design and country of origin, and ranging between 66 million to 102 million for people with mild AD dementia, or as another example, ranging between 46 million to 92 million for APOE genotype carriers worldwide.</p><span>Conclusions</span><p>The heterogeneity and the uncertainty in prevalence of the AD population represent big challenges to clinicians and payers. Future discussions on target patient identification for new treatments should be aligned and integrated with current clinical practice e.g. leveraging validated biomarkers as diagnostic tools. Additional research on an integrated approach to identify patients who would benefit the most from DMTs will be needed.</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":"138631672","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}