bluuebunny/crossref_metadata_2025 huggingface dataset:
OP94 Online Elicitation Of Personal Utility Functions (OPUF): A New Tool For Eliciting EQ-5D-5L Value Sets On The Societal-, Group-, Subgroup-, And Individual-Level
Authors: Schneider Paul , Brazier John , Devlin Nancy
Published: December 2022

Introduction

The ‘Online elicitation of Personal Utility Functions’ (OPUF) is a new method for valuing health states. It is based on compositional preference elicitation techniques. In contrast to established, decompositional techniques, such as time trade-off or discrete choice experiment (DCE), the OPUF approach does not require hundreds or thousands of respondents, but allows estimating utility functions for small (patient) groups and even on the individual level. The objective of this study was to generate and compare EQ-5D-5L value sets on the societal-, group-, subgroup-, and individual-level.

Methods

A demonstration version of the EQ-5D-5L OPUF Tool is available at: https://eq5d5l.me. It broadly consists of three valuation steps: dimension weighting, level rating, and anchoring. Responses were combined on the individual level to construct personal utility functions. Every respondent also completed three conventional DCEs. Preferences were aggregated across individuals to estimate a societal and various group-level preference functions. We then assessed the heterogeneity of preferences between groups using descriptive statistics and k-means cluster analysis.

Results

A representative sample (n = 1,000) of the United Kingdom (UK) population was recruited through the prolific online platform. On average, it took participants about 7 minutes to complete the survey. Data of 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. The derived utility functions predicted respondents’ choices in DCE with an accuracy of 78 percent. On the societal level, the predicted values for the EQ-5D-5L health states ranged from -0.376 to 1. Health state preference varied greatly between groups. This was largely due to differences in the anchoring, while there was near consensus on the relative importance of the five EQ-5D dimensions between groups. Demographic characteristics explained only a small proportion of the variability.

Conclusions

Using the OPUF approach, we were not only able to estimate a new EQ-5D-5L value set for the UK, but also to examine the underlying individual preferences in an unprecedented level of detail.
OP97 Stakeholders’ Involvement In Brazilian Health Technology Assessment Incorporation In 2021: A Statistical Description Of Demands To The Brazilian National Committee For Health Technology Incorporation
Authors: Komoda Denis Satoshi , Silveira Correa Carlos Roberto , Visacri Marilia Berlofa , Santos Daniela
Published: December 2022

Introduction

The demands for incorporating or excluding health technologies within the Brazilian Public Health System (SUS) can be requested by different stakeholders, including public administration, pharmaceutical companies, specialists’ and patients’ organizations/associations. The Brazilian National Committee for Health Technology Incorporation (CONITEC), part of the Ministry of Health, is the responsible organ to evaluate these demands and emit recommendations. The aim of this study is to show an overview of stakeholders according to the type of technology under request.

Methods

On 26 November 2021, a search was performed at CONITEC website. Health Technology Assessment (HTA) incorporation reports from 1 January to 26 November 2021 were extracted. From these reports, data regarding the demanded technology (e.g., medications, diagnostic tests, etc.), demand (technology incorporation, exclusion, or alteration), pharmacological classification according with the Anatomical Therapeutic Chemical (ATC), and requester’ categorization (e.g., pharmaceutical companies, official administration organs, etc.).

Results

Preliminary results showed a total of 77 health technologies, in 63 HTA incorporation submissions, of which 87 percent (n = 67) were medications, and 9 percent (n = 6) were new medical procedures. Only one medical device, one vaccine and two diagnostic tests were requested. Technology incorporation accounted for 94.8 percent of the demands (73), and 4 demands of exclusion. Seventy-one percent of the requested medications were classified, according to first ATC coding level, within groups L (Antineoplastic and immunomodulating agents), N group (Nervous system), A group (Alimentary tract and metabolism), and R group (Respiratory system), accounting 28.3, 17.9, 8.9 and 8.9 percent, respectively. Regarding stakeholders, the Brazilian Ministry of Health and its associated departments were responsible for 57.1 percent of the demands, while pharmaceutical companies requested 37.6 percent of incorporations. Other requesters included the Federal Justice Department, patients’, and specialists’ organizations, summing up with only four demands.

Conclusions

These results present the numerical weight of stakeholders in the Brazilian HTA incorporation system, with special attention to Brazilian bureaucracy and pharmaceutical companies. Further analysis regarding association between demand and other variables such as budgetary impact, costs, and ICD-10, shall deepen our understanding of different stakeholders’ role in Brazilian HTAi.
PP22 A Lifecycle Approach To The Use Of Real-World Evidence In HTA Submissions And Resubmissions: A Decade’s Experience
Authors: Samaha Dima , Tavares Eliana , van Engen Anke , Szawara Paula
Published: December 2022

Introduction

Health technology assessment (HTA) bodies worldwide recognize the importance of real-world evidence (RWE) in addressing uncertainties around the effectiveness of new drugs at the time of launch and as part of resubmissions. We assessed the use and acceptability of RWE by analyzing HTA recommendations.

Methods

We analyzed 24,841 HTA reports, including original submissions, resubmissions, extensions of original indications, and renewals, published from January 2011 to October 2021 from more than 100 HTA agencies across 37 countries.

Results

Our analysis showed that 3,820 (15%) reports mentioned RWE. Between 2011 and 2021 there was an eight-fold increase in the use of RWE, from 4 percent in 2011 to 34 percent in 2021. RWE was most commonly included in HTAs in oncology (26%) and endocrine and metabolic diseases (13%). The main areas supported were effectiveness (40%), safety (38%), and epidemiology (35%). RWE supplemented evidence on survival and quality of life as well as resource utilization, proxy comparators, and utility. Based on an analysis of the 1,474 reports that mentioned RWE, effectiveness was mainly supported by cohort (22%) and observational studies (13%), safety was mainly derived from pharmacovigilance data (9%), and epidemiology data were collected from registries (23%). The top five HTA bodies mentioning RWE in their reports were from France, Germany, Poland, and the United Kingdom (n=2). RWE was most accepted when it supported safety and epidemiological considerations, and to a lesser extent when it was used for effectiveness aspects or understanding management pathways.

Conclusions

The inclusion and acceptability of RWE in HTA recommendations varies between HTAs according to their data requirements and assessment methods. While it is not always specified how RWE was considered, there is a clear tendency for its increased use and acceptability, albeit not in all areas. Greater use of and transparency around RWE are likely to continue as multiple RWE initiatives emerge globally.
PP24 Organizing Outpatient Parenteral Antibiotic Therapy: Lessons from Denmark
Authors: Loevschall Claus , Thomsen Anne Marie , Risoer Bettina , Groth Lotte , Lou Stina , Palmhoej Camilla
Published: December 2022

Introduction

Outpatient Parenteral Antibiotic Therapy (OPAT) is a complex medical treatment used to treat patients with severe infections. OPAT is provisioned outside hospitals. There is wide variation in the use and organization of OPAT in Denmark. OPAT is increasingly used in Danish regions and municipalities, however, there is limited knowledge on the clinical, economic and organizational consequences of this technology. The purpose of the project was to establish an evidence base for decision-making prior to any further prioritization of OPAT as an alternative to intravenous antibiotic treatment in the hospital (IPAT). The HTA was produced at the request of the Health Directors in the Danish Regions to examine the consequences of using OPAT compared with IPAT.

Methods

The results were based on a systematic literature review and qualitative interviews with leaders (n=5), administrative employees (n=5) and health professionals (n=13) involved in the delivery of OPAT. Furthermore, a micro-costing analysis based on interviews with clinical experts was conducted.

Results

The use of OPAT led to similar or better clinical results when compared with the use of IPAT. Current evidence supports OPAT as a safe model for intravenous antibiotic treatment. The organization of OPAT varied in Denmark as well as internationally. The selection of suitable patients for the different OPAT models was crucial for a successful treatment. Insight into patients’ understanding of the pros and cons of the technology indicated that most patients preferred treatment at home. In a Danish context the microeconomic analysis showed that different OPAT models generally led to a reduction in costs compared with IPAT.

Conclusions

The project contributes to practice and political decision making by identifying challenges and opportunities associated with OPAT. There is no one-size-fits-all solution. The choice of OPAT model must be based on careful clinical considerations. Coordination and communication across municipalities and hospitals is challenging. Reducing organizational complexity is necessary to achieve a more standardized practice.
PP27 Reusing And Adapting Health Technology Assessments (HTAs): An Example From The COVID-19 Time
Authors: Faraldo-Valles Maria-Jose , Regueira-Castro Alba , Triñanes Yolanda
Published: December 2022

Introduction

Health technology assessment (HTA) reports are complex technical documents that address multiple aspects of the incorporation of a technology into the health care system applying complicated methodologies coming from different disciplines. The purpose of HTA is to support decision-makers and these should have an adequate level of training to fully understand these assessments. However, most HTA education programs and courses are intended for HTA doers and there is a lack of practical guidance training aimed at preparing health managers or policy makers in HTA. The objective is to describe an HTA training program developed for decision-makers of the three levels (health care administration, hospital management and clinical practice).

Methods

Rolling Collaborative Review (RCR) 01 of convalescent plasma was identified and selected because it complied with our Population Intervention Comparator Outcome Design Question. The EUnetHTA HTA adaptation Toolkit was used to check the relevance (about research question); reliability (quality of the report) and transferability (application of information to the target setting). Additional considerations regarding the local context were examined. A panel of four professionals and one patient was formed to rate the importance of the outcomes and to carry out the external review

Results

According to the toolkit, information on RCR01 Convalescent Plasma could be adopted for the safety and effectiveness domains. The technical characteristics and current use domains were adapted and extended. It was considered of interest to include the domains of organization and ethics. The organizational aspects were answered through the information retrieved in a search for systematic reviews and guides, and with the collaboration of experts. The ethics domain was answered through a specific literature search on ethical issues related to COVID-19 and transfusions.

Conclusions

The use of the EUnetHTA Toolkit has been helpful in supporting the adaptation process. The adoption of the effectiveness and safety domains from already developed HTA assessments is an efficient way to provide useful information for the decision-making process. However, contextual elements should be included in the adaptation process to ensure a complete framework for the decision.
PP28 Is My Medicine Suitable For An Outcomes Based Agreement? The Feasibility Conundrum
Authors: de la Fuente Graciela Sainz , Halsby Kate , Burton Jessica R.
Published: December 2022

Introduction

Outcomes Based Agreements (OBAs) are financial arrangements that offer the opportunity to align payment to health outcomes in the real-world, and share the financial risk by providing long-term solutions that grant access to medicines, with reimbursement only when performance is achieved. OBAs are most likely to be useful when there is high uncertainty in the clinical data, but they are difficult to design and implement, and other financial options are usually preferred by payers. As a result, OBAs have been more the exception than the norm, and there is not a clear pattern that indicates if an OBA is likely to succeed in practice.

Methods

Through a retrospective OBA exercise with NHS Wales (Project IDEATE: Innovation in Data to Evolve Agreements That Enhance patient health outcomes), we have explored the circumstances under which an OBA might be most appealing to payers, and assessed implementation challenges and solutions, to propose a framework to evaluate the feasibility of a medicine for an OBA.

Results

Along with mitigating some of the clinical uncertainties associated with a lack of mature data at the time of launch, an OBA must also consider other factors: the commercial viability of the agreement, the associated administrative burden, and its cost of implementation. Also, the Health System commitment to a Value-Based Healthcare agenda and, most importantly, its willingness to offer long-term sustainable solutions to optimise treatment, are key to support this approach.

Practical considerations include: how the relevant outcomes are going to be selected and tracked in the real-world, how the whole model is going to fit within the current procurement and finance infrastructures, and how industry works in collaboration with the Health System.

Conclusions

Insights from Project IDEATE will be used to explore how our OBA feasibility framework might be applied in the future.
PP36 Joint Replacement Under Computer Navigation And Robotic Systems
Authors: Akhmedullin Ruslan , Avdeyev Andrey , Benberin Valeriy , Shanazarov Nasrulla , Bariyeva Gulzada , Okesh Makhabbat , Akhmetova Makpal , Aimanova Tansolpan
Published: December 2022

Introduction

Osteoarthritis (OA) is a heterogeneous group of diseases of various etiologies based on the defeat of all components of the joint. OA is one of the main causes of disability in older people. To date, joint replacement is the most clinical and cost-effective method of the terminal stage treatment. The short and long-term success of total joint replacement is closely related to the accuracy of the prosthesis implantation. Published studies show that the accuracy of prosthesis implantation can be intraoperatively controlled by computed navigation and robotic systems better than by traditional methods.

Methods

In order to assess the clinical effectiveness of the technologies, we have conducted a literature search in the MEDLINE database. We included studies that reported a comparison of outcomes between conventional methods, computer navigation and robot-assisted surgery.

Results

The results of this literature review are based on six systematic reviews with meta-analyses (101 studies representing 482,367 cases) and one national joint replacement registry. The outcomes compared included Knee Society Score (KSS)-function, alignment correction, mechanical axis (varus and vagus deviance >3°), prosthesis positioning, soft tissues balancing and functional outcomes. Thus, the cumulative success rate in the computed navigation and robotic systems group was reported to be 86.7 percent, which is crucial considering its lowered rate of revision (3%), correct mechanical axis (≤3%) and functional status.

Conclusions

The literature review demonstrates a high potential of the computed navigation and robotics systems in the intraoperative monitoring of important surgical parameters for achieving the best intervention outcomes. All the clinical endpoints were significantly better by comparison with conventional method.
PP42 Impact Of The COVID-19 Pandemic On Scottish Medicine Consortium Submission Characteristics, Acceptance Rates, And Time To Advice
Authors: Leslie Iain , Berg Guy
Published: December 2022

Introduction

Scottish Medicine Consortium (SMC) meetings were suspended in March 2020 in response to the coronavirus disease 2019 (COVID-19) pandemic. This led to a high number of submissions awaiting appraisal, prompting interim process changes to ensure minimal disadvantages to patient access. We expanded the eligibility criteria for the shorter (abbreviated) submissions process and expedited advice for submissions the New Drugs Committee (NDC) intended to accept. This study aimed to evaluate the impact of the COVID-19 pandemic and these interim process changes on the characteristics of submissions received, acceptance rates, and time to advice publication.

Methods

Data for all submissions received between January 2015 and November 2021 (n=720) were extracted from an organizational database. Characteristics of and acceptance rates for submissions received before and after the start of the pandemic were compared using chi-squared and one-proportion Z-tests, respectively. Additional analyses explored the number of submissions received per month and the time from receipt of submission to NDC and SMC decision.

Results

The numbers of full and abbreviated submissions increased from March 2020 (6% in each case), with a corresponding decrease in the number of medicine-indication pairs (e.g., pembrolizumab for breast cancer) for which companies did not submit (8%; p=0.01). An increase in the SMC acceptance rate was also observed (62 to 72%; p=0.03). Fewer submissions were received in 2020 (n=65), compared with the pre-pandemic average (mean=79.6), whereas the total in 2021 to date was higher than average (n=92). Time series analysis suggested an increasing trend in monthly submissions (from approximately 6 to 9), which is the likely reason for the increase in average time to decision (146 versus 170 days).

Conclusions

Process changes in response to the pandemic have been effective in expediting advice for submissions with sufficiently robust evidence. This demonstrates agility and efficiencies for submitting companies and patient groups, with no perceived impact on process rigor. The average number of submissions has increased since March 2020, and further work is warranted to understand the influence of process improvements on reducing time to advice.
PP57 Collaborating In Times Of COVID: The Launch Of The National Wales COVID-19 Evidence Centre
Authors: Elston Lauren , Winfield Thomas , Washington Jenni , Jarrom David , Myles Susan , Lewis Ruth , Cooper Alison , Gal Micaela , Edwards Adrian
Published: December 2022

Introduction

The COVID-19 pandemic has had a dramatic impact on the health and social care landscape, both in terms of service provision and citizen need. Responsive, evidence-based research is essential to develop and implement appropriate policies and practices that manage both the pandemic itself, and the impact COVID-19 has on other health and social care issues.

To address this, the Wales COVID-19 Evidence Centre (WCEC) was launched in 2021 with the aim of providing the best available, up-to-date, and relevant evidence to inform health and care decision making across Wales.

Methods

Funded by the Welsh Government, the WCEC comprises of a core team and several collaborating partner organizations, including Health Technology Wales, Wales Centre for Evidence-Based Care, Specialist Unit for Review Evidence Centre, SAIL Databank, Public Health Wales, Bangor Institute for Health & Medical Research in conjunction with Health and Care Economics Cymru, and the Public Health Wales Observatory. Over the last year, WCEC has developed its rapid review processes and methodology informed by best international practice and aims to provide around 50 reviews each year. WCEC works alongside various stakeholder groups from health and social care across Wales, and they form an integral part of the review process, from scoping to knowledge mobilization.

Results

To date, the WCEC has produced reviews on a diverse range of COVID-19 topics, including transmission, vaccination uptake (barriers, facilitators and interventions), mental health and wellbeing, as well as face coverings and other preventative interventions. The topics have also covered a wide range of populations, from general public, to healthcare workers, to children. These reviews have been used to inform policy and decision-making, including the Welsh Government’s Chief Medical Officer 21-day COVID-19 reviews.

Conclusions

The WCEC has brought together multiple specialist centers with a diverse range of skills to produce timely reviews of the most up-to-date research to support decision makers across health and social care. These reviews have informed policy and decision-making across Wales.
PP58 SARS-CoV-2: A Rapid Review Of The Transmission Risk From Vaccinated Populations
Authors: Barrate Sasha , Elston Lauren , McDermott Katie , Washington Jenni , Hasler Elise , Jarrom David , Bowles Charlotte , Tom Winfield Jessica Williams , Myles Susan
Published: December 2022

Introduction

Since the vaccine roll out, research has focused on vaccine safety and efficacy, with large clinical trials confirming that vaccines are generally effective against symptomatic COVID-19 infection. However, breakthrough infections can still occur, and the effectiveness of vaccines against transmission from infected vaccinated people to susceptible contacts is unclear.

Health Technology Wales (HTW) collaborated with the Wales COVID-19 Evidence Centre to identify and examine evidence on the transmission risk of SARS-CoV-2 from vaccinated people to unvaccinated or vaccinated people.

Methods

We conducted a systematic literature search for evidence on vaccinated people exposed to SARS-CoV-2 in any setting. Outcome measures included transmission rate, cycle threshold (Ct) values and viral load. We identified a rapid review by the University of Calgary that was the main source of our outcome data. Nine studies published following the rapid review were also identified and included.

Results

In total, 35 studies were included in this review: one randomized controlled trial (RCT), one post-hoc analysis of an RCT, 13 prospective cohort studies, 16 retrospective cohort studies and four case control studies.

All studies reported a reduction in transmission of the B.1.1.7 (Alpha) variant from partial and fully vaccinated individuals. More recent evidence is uncertain on the effects of vaccination on transmission of the B.1.617.2 (Delta) variant. Overall, vaccine effectiveness in reducing transmission appears to increase with full vaccination, compared with partial vaccination. Most of the direct evidence is limited to transmission in household settings therefore, there is a gap in the evidence on risk of transmission in other settings. One UK study found protection against onward transmission waned within 3 months post second vaccination.

Conclusions

Early findings that focused on the alpha variant, showed a reduction in transmission from vaccinated people. There is limited evidence on the effectiveness of vaccination on transmission of the Delta variant, therefore alternative preventative measures to reduce transmission may still be required.
PP62 Recommendations On Methodologies To Obtain Comparator Efficacy In Health Economic Assessments Of Tumor-Agnostic Drugs
Authors: Efe Reva , Vijgen Sylvia
Published: December 2022

Introduction

Guidance on appropriate methods to obtain a comparator arm for the cost-effectiveness analysis of tumor-agnostic drugs is needed. In recent years, multiple tumor-agnostic drugs have been submitted to health technology assessment (HTA) bodies based on data from single-arm basket trials. These target a specific genetic mutation, as opposed to targeting a specific tumor type. Since HTA bodies are interested in the comparative effectiveness of a treatment, manufacturers have used several methods to obtain a synthetic control arm in their submissions. This study provides an overview of the recommendations by HTA bodies on the methodology to obtain comparator efficacy.

Methods

A targeted literature review will be conducted focusing on the methodology used to obtain a comparator arm in the context of tumor-agnostic drugs. The search will cover key HTA organizations; including the National Institute for Health and Care Excellence (NICE), Haute Autorité de Santé (HAS) and the Canadian Agency for Drugs & Technologies in Health (CADTH). Methodologies used in entrectinib and larotrectinib submissions will be extracted. Particular focus will be given on the impact of the applied methodology to the reimbursement decision, as well as key critiques by the HTA bodies. Key search terms will include the following: ‘tumor-agnostic’, ‘histology independent’, ‘HIT’, ‘entrectinib’, ‘larotrectinib’.

Results

An overview of the results will be presented. These will include the applied methodology for obtaining a comparator arm, critiques and recommendations from HTA bodies, and the impact these methodologies had on the overall reimbursement decision. This will enable comparison of HTA decision-making across regions, and key evidence gaps that need to be further explored.

Conclusions

The results of this study could be useful in the future assessment of tumor-agnostic drug submissions, focusing on the methodology used to obtain comparator efficacy.
PP66 Safety, Effectiveness And Cost-effectiveness Of Scalp Cooling Devices For The Prevention Of Chemotherapy-induced Alopecia
Authors: Infante-Ventura Diego , de Armas-Castellano Aythami , Hernández-Yumar Aránzazu , González-Pacheco Himar , Pino-Sedeño Tasmania del , González-Hernández Yadira , García-Pérez Lidia , Ramallo-Fariña Yolanda , Rodríguez-Rodríguez Leticia , Rueda-Domínguez Antonio , Serrano-Aguilar Pedro , del Mar Trujillo-Martín María
Published: December 2022

Introduction

Chemotherapy-induced alopecia (CIA), although reversible, is one of the most common and distressing side effects of cancer therapy, affecting approximately 65 percent of all patients and influencing treatment decisions in some of them. Scalp cooling (SC) is a method aiming to prevent CIA. Our study aims to evaluate the real value of SC devices.

Methods

A systematic review of the available scientific literature on the safety, effectiveness and cost-effectiveness of the use of SC compared with no intervention was performed. Overall effect size was estimated through a meta-analysis. An economic analysis in the Spanish context from the Spanish National Healthcare System (NHS) and social perspectives was performed.

Results

Thirteen randomized controlled trials (n = 832) were included but only nine contributed to the meta-analysis. A large effect in favor of SC reducing hair loss was found (RR=0.57; 95% CI: 0.46-0.69). No differences were observed according to the type of cancer, although there was a small positive effect for breast cancer. A higher effect was found in patients treated with a combination anthracyclines/taxanes treatment compared to those treated only with anthracyclines. The only economic evaluation found in the literature was conducted in The Netherlands and concluded that Paxman system was less costly than usual care from societal perspective and no differences in quality adjusted life years (QALYs) were observed. The de novo economic analysis showed that the strategies including SC devices generated more costs and QALYs (given some assumed utility values) than usual care (not SC), presenting incremental cost-effectiveness ratios below the threshold calculated for Spain (EUR 25,000 /QALY), from both perspectives.

Conclusions

The results suggest that SC are effective for the prevention of CIA. Furthermore, assuming the utility values used in the model, SC devices are cost-effective compared to usual care (not SC).
PP82 First Educational Trainings According To New Health Technology Assessment Guideline For Medicines In Ukraine
Authors: Khmelovska Marharyta , Piniazhko Oresta , Serediuk Valeriia , Masheiko Alona , Romanenko Iryna
Published: December 2022

Introduction

The success of the health technology assessment (HTA) implementation depends on the level of communication efficiency between all stakeholders. Taking this into consideration, the leading HTA experts of HTA Department of State Expert Center of the Ministry of Health of Ukraine launched the new educational initiative which includes training programs for pharmaceutical companies.

Methods

Сomprehensive review of the first developed HTA training programs based on the HTA Guideline (Number 593) “The state health technology assessment for medicines” approved on 29 March 2021 was conducted.

Results

Leading HTA experts of the HTA Department developed a training program, which was conducted for industry representatives according to the provisions of the HTA Guideline. In 2021 over twelve training sessions and two webinars were conducted. These learning events reflect harmonized international recommendations and approaches to HTA training programs, The Professional Society for Health Economics and Outcomes Research, ISPOR short courses in particular, European Network for Health Technology Assessment (EUnetHTA), The National Institute for Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines. Two-hour webinars were aimed at giving theoretical and practical bases for building a Markov model with the help of Excel and TreeAge Software. Training sessions titled “HTA as a tool for assessing the value of health technology” differ by duration (7-hour, 4-hour, 3-hour) and cover core topics adapted to the needs of the audience. The main program components include HTA Roadmap in Ukraine, clinical section, economic section and practical case studies of building a Markov model. All training options included questionnaires at the beginning and i end to assess the quality of each program. Questionnaires are the tool that gives presenters the possibility to trace progress and transform the training material accordingly.

Conclusions

Development of extended capacity building programs in HTA for users and doers is highly prospective for further steps in HTA institutionalization in Ukraine.
PP83 Economic Impact Of Missed Vaccinations On The Italian National Health System
Authors: Di Brino Eugenio , Basile Michele , Rumi Filippo , Cicchetti Americo ,   Instant Report Group
Published: December 2022

Introduction

To provide a complete picture of the economic impact of the coronavirus disease 2019 (COVID-19) emergency for the Italian National Health System (NHS), an estimate was made of the costs to the NHS of vaccination hesitation. The concept of economic impact was investigated with reference to the volume of hospitalizations and days of intensive care required for patients with COVID-19 related to missed vaccinations, considering a vaccine efficacy of less than 100 percent.

Methods

Data from the Istituto Superiore di Sanità were analyzed with respect to the number of people vaccinated in the general population, and the number of people with severe acute respiratory syndrome coronavirus 2 infection who were hospitalized or died in a one-month period stratified by vaccination status. The costs for unvaccinated patients admitted to a general hospital ward (Medical Area) or the intensive care unit were calculated.

Results

Based on the number of preventable hospitalizations among unvaccinated people, the economic impact of missed vaccinations on the NHS in the 30-day period from 13 August 2021 to 12 September 2021 was estimated. Among the unvaccinated hospitalized patients, 5,932 would have avoided hospitalization in the Medical Area and 715 would have avoided admission to the intensive care unit. Thus, each unvaccinated hospitalized patient had an average per capita cost of EUR 17,408. The total costs amounted to EUR 69,894,715, comprising EUR 51,166,079 for hospitalizations in the Medical Area and EUR 18,728,636 for hospitalizations in intensive care.

Conclusions

By evaluating the weekly incidence of hospitalizations per 100,000 people stratified by vaccination status (unvaccinated, partially vaccinated, and fully vaccinated), it is possible to see that we are facing two distinct pandemics running together.
PP89 Stimulating And Assuring Evidence Quality From A Dutch Funders’ Perspective – Introducing The ZonMw Reporting Checklist
Authors: Reijmerink Wendy , Vianen Gerjanne , Bloemers Margreet , Durrani Abida , Stax Annelein
Published: December 2022

Introduction

All studies should report methods and findings in full, following credible and justifiable reporting guidelines. According to the guiding principles of the Ensuring Value in Research (EViR) Funders’ Forum (www.evir.org), this applies irrespective of the nature of the findings or whether the study was completed as planned.

One way for a public funding agency to address evidence quality and transparency is to adaptively implement EQUATOR reporting guidelines (www.equator-network.org) in its funding procedure to ensure research quality ‘from proposal to publication’. The Netherlands Organisation for Health Research and Development, ZonMw, has created the ZonMw Reporting Checklist (ZRC), which was derived from EQUATOR reporting guidelines in order to systematically plan, monitor, and evaluate projects. The next step is experimenting with implementing the ZRC in ZonMw’s grant management system and procedures. Customization is possible based on the ‘comply or explain’ approach (80/20 rule).

Methods

We selected 15 EQUATOR reporting guidelines that covered basic research and health technology assessment through to implementation projects, supplemented with the reporting guideline for implementation studies (StaRI checklist). We conducted comparative content analyses (including rearrangement) to provide a greatest common denominator consisting of both standard and modular reporting elements. We completed the ZRC by adding other current requirements for responsible research practices with respect to diversity and gender, data management, open access, systematic reviews, recruitment and inclusion, registration, and impact.

Results

The ZRC results in structured and validated in-house data on the objectives, design, conduct, and results of ZonMw projects. This is an important source for good research governance, impact assessment, and research on research.

Conclusions

Implementation of the ZRC by a funding agency optimizes the quality, transparency, relevance, and impact of evidence, which legitimately and effectively improves health care for all.
PP96 Joint Clinical Assessments – Implementation And Lessons For The Next Stage Of EU HTA
Authors: Samaha Dima , Newton Max , Tavares Eliana , Kruger Robert , Szawara Paula , van Engen Anke
Published: December 2022

Introduction

The European Network for Health Technology Assessment (EUnetHTA) was a voluntary cross-border initiative on HTA harmonization established by European Commission in 2005. Between 2016-2021, EUnetHTA completed 56 Joint Assessments (28 drugs; 28 other technologies) including 14 reviews of COVID treatments.

Methods

We conducted a review of the 14 EUnetHTA joint assessment reports of drugs in non-COVID 19 indications. We cross-referenced recommendations with national guidance in 30 member countries (including UK) and conducted an analysis of time to national assessment, choice of comparator, direct reference to EUnetHTA assessment, and time to reimbursement decision.

Results

Six products in oncology, 2 in endocrine and metabolic diseases, 2 in infectious and parasitic diseases, and cardiovascular, digestive system, eye disorders and central nervous system (one each) were identified. On average, EUnetHTA published its recommendation 52 days after market authorization for oncology products and 33 days for non- oncology products. EUnetHTA recommendations considered on average 4 comparators (range 1-8) as part of the assessment. All of the 6 oncology products have been assessed by national HTA bodies, however uptake was low with an average of 5 reports referencing the EUnetHTA report. Similarly for the non-oncology products assessed only 3 of 30 HTA bodies cite the EUnetHTA report. Citing HTA bodies were: AETSA (Spain), HAS (France), INFARMED (Portugal), NoMA (Norway), and TLV (Sweden). There was no clear reduction in the time to reimbursement for these products in these markets.

Conclusions

According to EUnetHTA, there has been an increased use and dissemination of joint assessment reports since 2016. Our analysis shows that the level of implementation across countries is heterogeneous despite publication of the EUnetHTA reports shortly after market authorization. The future the EU HTA will depend on the timeliness, rigor and transparency of joint clinical assessment reports and improved uptake of these reports at a national level.
PP100 Improving The Assessment Of Effectiveness For Digital Applications Using The B Statistic: Using WtsWrng As A Case Study
Authors: Abbas Ismail , Barcelo Carla Fernandez , Valdasquina Blanca , Rabasseda Nicolau , Sampietro-Colom Laura
Published: December 2022

Introduction

The performance of diagnostic health technologies is usually assessed by comparing them with standard care using the kappa statistic. These comparisons are made based on comprehensive clinical information (e.g., anamnesis and complementary tests). However, not all digital applications (DAs) execute over complete information, which leads to work under non-uniform distribution of values. Using kappa statistic in this situation has serious methodological limitations. Kappa assumes that the marginal values are uniformly distributed and highly weights the discordant values when calculating concordance, which underestimates the real effectiveness of DAs (i.e., observed concordance). We aimed to present the application of the B statistic to WtsWrng, a symptom triage DA for individuals.

Methods

WtsWrng was used by 382 patients at the emergency department of a hospital. Diagnoses provided by WtsWrng, given 19 symptoms, were compared with those logged in the hospital’s electronic clinical records at discharge. Observed concordance was calculated using contingency tables. The concordance using the kappa and B statistics were compared for the 12 most frequent diagnoses at hospital discharge. Sensitivity and specificity were also calculated.

Results

Real observed concordance fluctuated from 0.4 to 0.98 for the 12 most frequent diagnoses, eight of which had a concordance greater than 0.8. The results ranged from -0.005 to 0.37 when using the kappa statistic and from 0.36 to 0.99 when using the B statistic. The sensitivity and specificity of WtsWrng were greater than 0.8 for three and eight of the 12 diagnoses, respectively.

Conclusions

The results show that the B statistic is closer to the real observed concordance when kappa statistic assumptions are not fulfilled by a DA. Therefore, the B statistic is better suited for assessing the effectiveness of this type of technology. Analysis of WtsWrng using the B statistic showed that its diagnoses were close to those provided by clinicians, which were arrived at using complete clinical information. Moreover, the high specificity of the WtsWrng DA suggests that it is a good tool for determining the appropriate use of healthcare resources.
PP108 Multidimensional Analysis Of Peristeen Plus Medical Device
Authors: Cicchetti Americo , Fiore Alessandra , Rumi Filippo , Di Paolo Maria Giovanna , Fortunato Agostino
Published: December 2022

Introduction

Neurogenic bowel dysfunction occurs in people with central nervous system disease or injury and causes loss of bowel control and severe constipation. These problems involve a lot of anxiety and discomfort and can reduce the quality of life of those who suffer from them, therefore the management of symptoms is very important. Peristeen Plus is a transanal irrigation (TAI) system for bowel dysfunction management, used to empty the rectum and distal sigmoid colon, to prevent uncontrolled bowel movements or to relieve and prevent constipation.

Methods

A literature review was conducted. A total of 14 records were included to evaluate the benefits in terms of efficacy and safety associated with the adoption of the medical device. To assess the economic impact, two different budget impact models have been implemented. The first aimed at evaluating an incremental diffusion in a short-term time horizon (3 years) of the home distribution of the device compared to the direct and indirect distribution methods in the Italian context. The second model aimed at assessing the impact of the diffusion of the device in the clinical practice.

Results

Overall, most studies demonstrate improved endpoints related to the severity of fecal incontinence, constipation and intestinal disorders in patients using the device. The economic assessments conducted estimate that the increase in the Italian care setting is associated with a saving of resources in each year under analysis. The diffusion of home distribution of the device would potentially be able to offer a lower absorption of resources compared to other distribution methods. In addition to this, there is an incremental saving correlated to the degree of diffusion of Peristeen Plus.

Conclusions

TAI is considered a safe and more effective method than conventional treatments for reducing fecal incontinence, constipation and improving quality of life. The results of our study confirm the benefits of TAI as a second-line treatment in case of failure of conventional medical therapy in the management of the neurogenic gut.
PP110 An External Combined Occipital And Trigeminal Neurostimulation Device For Migraine Treatment
Authors: Rodríguez-Ortiz-de-Salazar Begoña , Hijas-Gómez Ana Isabel , del Mar Polo de Santos María , Arias-Pacheco Amparo , Sánchez-Gómez Luis , Luengo-Matos Seteffilla
Published: December 2022

Introduction

Migraine is a multifactorial pathology that affects 12 percent of the world’s population. Relivion® MG (Neurolief Inc., USA) is a non-invasive self-administered device for external combined occipital and trigeminal nerve stimulation that has been recently approved for marketing in Europe and the USA. The objective of this study was to conduct an early assessment of the efficacy and safety of Relivion for the treatment of migraine episodes.

Methods

Relivion was identified by the early awareness and alert system, SINTESIS- nuevas tecnologías, of the Agencia de Evaluación de Tecnologías Sanitarias at the Instituto de Salud Carlos III. An early assessment of the technology was conducted by reviewing relevant clinical studies published to 29 September 2021. The literature was identified by searching PubMed, Embase, the International Clinical Trials Registry Platform, ClinicalTrials.gov, and the Cochrane Library.

Results

Two randomized, sham-controlled double-blind trials were found. They assessed side effects and pain relief, response rate, and pain freedom two hours after treatment. One study included 55 patients and the other (the RIME study) included 131 patients. The rate of pain relief two hours after treatment ranged from 60 to 76 percent in the treatment group and from 31 to 37 percent in the control group (p<0.01). The response rate, defined as at least 50 percent pain reduction two hours after treatment, was significantly higher in the treatment group (67 to 70% versus 32 to 42%). The percentage of patients free of pain two hours after treatment ranged from 42 to 46 percent in the treatment group, compared with 11 to 12 percent in the control group (p<0.0001). No notable adverse events were recorded.

Conclusions

Preliminary results indicated that Relivion effectively and safely relieves or eliminates acute migraine pain. However, more comparative studies are needed. The use of Relivion could improve the control of symptoms and improve quality of life in patients with migraine.
PP112 Reimbursement Decisions for Medical Services in Austria: An Analysis of Influencing Factors for the Hospital Individual Services Catalogue
Authors: Goetz Gregor , Panteli Dimitra , Busse Reinhard , Wild Claudia
Published: December 2022

Introduction

This study aims to (i) describe the (evidence-based) reimbursement process of hospital individual services, (ii) evaluate the accordance between evidence-based recommendations and reimbursement decision of individual services and (iii) elaborate potential aspects that play a role in the decision-making process in Austria.

Methods

The reimbursement process is described based on selected relevant sources such as official documents. Evidence-based recommendations and subsequent reimbursement decisions for the annual maintenance of the hospital individual service catalogue in Austria between 2008 and 2020 were analyzed using a mixed methods approach, encompassing descriptive statistics and a focus group with Austrian decisionmakers.

Results

One hundred and eighteen evidence-based recommendations were analyzed. There were 93 (78.8%) negative and 25 (21.2%) positive evidence-based recommendations. In total, 107 out of 118 evidence-based recommendations (90.1%) did not lead to a deviating reimbursement decision. We identified six aspects that may have played a role in the decision-making process for the annual maintenance of the hospital individual service catalogue, with clinical evidence being the most notable. Further aspects included quality assurance/organizational aspects (i.e., structural quality assurance), costs (if comparable to already existing medical services, not: cost-effectiveness), procedural aspects (e.g., if certain criteria for adoption have not been met formally through the proposals), “other countries” (i.e., taking into account how other countries decided) and situational aspects (such as the COVID-19 pandemic).

Conclusions

There is good accordance between evidence-based recommendations and reimbursement decisions regarding hospital individual services in Austria. Beyond clinical evidence, organizational aspects seem to be considered often with regard to quality assurance but costs do not appear to play a major role. The Austrian system has mechanisms in place that can restrict widespread adoption of novel hospital individual services with uncertain clinical benefits. Future studies could investigate how well these mechanisms work and how they compare to other health systems in Europe.