bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP97 Recommendations For Generating South African Health-Related Quality Of Life Data For Cost-Utility Analyses
Authors: Marsh Sophia Emmarenzia , Truter Ilse 
Published: December 2022

IntroductionHealth-related quality of life (HRQoL) data are necessary for conducting cost-utility analyses (CUAs) as part of health technology assessments (HTAs), but the lack of robust HRQoL data may delay or even prevent patient access to medicines when National Health Insurance is introduced in South Africa. This study aimed to make recommendations for evidence generation activities to support national HTA in the public health sector, with a focus on creating South African HRQoL data suitable for CUAs.MethodsA systematic review of HRQoL research in South Africa formed the basis for three analyses. The first analysis quantified and evaluated the suitability of HRQoL studies for CUAs. The second analysis determined the performance indicators of the research output and identified collaborative networks through bibliometric analyses. The third analysis critiqued the translation methodology of the HRQoL instruments retrieved in the systematic review.ResultsBased on the published literature, existing HRQoL data are unlikely to support CUAs because they were derived from observational or cross-sectional studies that lacked the methodological details necessary to determine their scientific merit according to HTA requirements. Overall, there was a lack of research continuity in this field, with numerous isolated research networks. Despite the strong contribution of South African based researchers and organizations in this area, their performance was below that of international counterparts. Since only a few HRQoL instruments suitable for CUAs would be valid in the South African context, HRQoL research output in South Africa could be optimized by using more rigorous study designs and by the expansion of researcher networks to include those working in HTA and related fields. The three-level EQ-5D is the tool best suited for use in South Africa, so its utilization should be encouraged and supported by establishing a South African value set.ConclusionsFuture data generation activities should incorporate the recommendations from this study because existing South African HRQoL data are likely to be inadequate for conducting CUAs in national HTA.
PP107 Scale For Measuring Fatigue In Patients With Parkinson’s Disease: Scientific Technical Report
Authors: Figueiredo Caroline Vieira , De Assis Patroclo Maria Aparecida , Nicaretta Denise Hack 
Published: December 2022

IntroductionFatigue in Parkinson’s Disease (PD) remains poorly understood. The comprovation derives from and is proven by psychometric scales. The objective is to prepare a Technical Scientific Report on the performance of scales in PD.MethodsBased on the Methodological Guideline for the Preparation of Technical Scientific Report, Brazil 2014, we conducted a search on MEDLINE / PubMed, Virtual Health Library (BVS) and Cochrane, and then a review and critical evaluation of the studies and the quality of the evidence.ResultsNine studies were analyzed: three systematic reviews, one case-control and five cross-sectional studies. The following were evaluated: Fatigue Severity Scale (FSS), Fatigue Assessment of Chronic Illness Therapy (FACIT-F) and Parkinson’s Fatigue Scale (PFS-16). In Brazil, FSS and PFS-16 were validated. The studies have methodological weaknesses and moderate, low and very low evidence. However, FSS, FACIT-F and PFS-16 show potential for assessing fatigue in PD but further studies are needed.ConclusionsConsensus on fatigue are recommended for Brazilian studies comparing FSS and FACIT-F with PFS-16 and longitudinal monitoring of patients.
PP114 The Influence Of Implicit Factors On The Health Technology Assessment Deliberative Process: A Survey In Five European Countries
Authors: Monleon Clara , Toumi Mondher , Späth Hans-Martin , Dussart Claude , Crespo Carlos 
Published: December 2022

IntroductionImplicit factors can be defined as any criteria that play a role in the health technology assessment (HTA) deliberative process but are not part of the HTA framework. To date, very few studies have explored the influence of implicit factors on this process. This survey of HTA experts in five European countries aimed to analyze the influence of implicit factors on the HTA deliberative process.MethodsSemi-structured interviews with 20 HTA experts from five European countries (France, Germany, Italy, Spain, and the United Kingdom) were conducted from February to May 2021. The main topics of the interviews were: the HTA deliberative process; the degree of influence on the HTA deliberative process of a set of factors previously identified in a systematic literature review performed by the authors; and recommendations for improving the deliberative process.ResultsAll but two of the experts concurred that implicit factors played a role in the deliberative process. German experts considered that the factors explored had a low influence on the process. Burden of disease and unmet need scored highest, followed by the professional experience of the people involved in the HTA deliberative process. To improve the deliberative process, experts suggested expanding the external stakeholder perspective (i.e., including patients, the pharmaceutical industry, and the public), increasing transparency when revealing implicit factors, and implementing a methodology to mitigate the influence of implicit factors.ConclusionsOur survey indicates a need to increase external involvement in the process and to develop a methodology for unmasking the implicit factors in the deliberative process. This may be achieved by either updating the current frameworks to include these implicit factors or by developing new methods to address them. Further research may explore approaches to acknowledge the implicit factors in the HTA deliberative process in a systematic manner.
PP115 Analysis Of Previous Joint Clinical Assessment And Potential Transferability To Four European Countries: Case Study And Conceptual Approach
Authors: Kern Jeanne , Pascha Victoria Malek 
Published: December 2022

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) has led together, with member states, several joint actions, including Joint Clinical Assessments (JCA), under the form of project-based voluntary cooperation, which outputs and transferability of those projects in other European countries remains somehow limited. In June 2021, the European Council has reached an agreement on the European Health Technology Assessment (HTA) regulation, which is entering into force gradually. Initially limited to oncology products, then extended to orphan/advanced therapy, and after a five to eight-year additional period, it will apply to all centrally approved products. The JCA will consist of a focused scientific analysis on relative effectiveness assessment, including the health condition, technology description, clinical effectiveness, and safety. These analyses will also include information relating to the degree of certainty. We consider that the evidence appraisal might have limitations, and transferability would not be generalizable. We aim to determine the potential drivers and barriers for HTA transferability in EU4, employing the analysis of a case example where JCA was conducted.MethodsEmploying an oncology JCA, we will compare an HTA analysis conducted in EU4 countries (Germany, France, Italy, and Spain). Overview and background information on countries involved in the JCA, and EU4 HTA system will be provided, followed by HTA outcome and main evidence requirements, reimbursement outcome, and pricing agreements.ResultsStudy results supporting HTA outcomes may focus on the population assessed, the comparator considered, and uncertainty management. A conceptual adaptation about the scope of the EU JCA regulation will be discussed, to understand its potential advantages to individual HTAs in Europe and remaining gaps to effectively inform HTA or decision-making process.ConclusionsThe analysis of pricing and reimbursement outcomes can further help understand potential barriers and drivers for JCA transferability and potential areas of evidence generation requirements.
PP117 Larger Impact Of An Appropriate Care Program By Involving Healthcare Professionals: A Case Report On Cervical Pre-malignancy
Authors: Böcker Koen , de Bruijn Thom , Vink Maarten 
Published: December 2022

IntroductionAppropriate care programs differ in the way and the extent to which they involve or collaborate with stakeholders. Here we describe the collaboration of two national appropriate care programs with gynecologists to improve the curative care of women with cervical intraepithelial neoplasia (CIN), a cervical pre-malignancy.MethodsThe present case report describes the collaboration based on project documentation.ResultsThe Appropriate Care program from the Dutch National Health Care Institute performs a systematic cyclic health technology assessment to examine the degree to which care in the insured package is provided to patients. The full cycle consists of four phases: screening, in-depth analysis, implementation, and evaluation. The results of the in-depth analysis are discussed with the stakeholders. This is followed by written agreements on multiple actions to improve healthcare from the patient perspective. For CIN these actions encompass improvements in a top-down fashion; for example, by updating guidelines to eliminate unwanted practice variation and creating tools for shared decision-making. These actions were supplemented by the development of audit and feedback information on a national and local level. The development was supported by a second national appropriate care program, Healthcare Evaluation and Appropriate Use. The results of the first production run of the audit and feedback information were disseminated by the Dutch Society of Obstetrics and Gynaecology and more than 50 healthcare institutions. This information was used to prioritize modular guideline updates and helped pinpoint the main areas of improvement of individual healthcare institutions. A future production run of audit and feedback information will facilitate the Plan-Do-Check-Act cycle on a local and national level.ConclusionsIn the case we present, the collaboration between appropriate care programs and healthcare professionals led to a synergy between top-down (updating and disseminating guidelines and tools for shared decision-making) and bottom-up (learning from audit and feedback information) activities to improve curative care for women with CIN.
PP128 Development And Piloting Of An Online Training Course On Health Technology Assessment For Patients
Authors: Vicente-Edo María José , Triñanes Yolanda , Toledo-Chávarri Ana , Bono-Vega María , Gavín-Benavent Patricia , Prieto-Remón Lucía , Suñe Begoña , Segovia Margarita , Novella-Arribas Blanca , Pilar Loeches-Belinchón , Cantero-Muñoz Paula , Perestelo-Pérez Lilisbeth , Jayo Nerea Arias , Reviriego-Rodrigo Eva 
Published: December 2022

IntroductionPatients have knowledge, perspectives and experiences that are unique and can make an essential contribution to Health Technology Assessment (HTA). However, in order for their participation to be effective, they need to be able to understand how HTA reports are generated and the decision-making processes that they inform. The aim is to describe the development and virtualization of training materials for patients, as well as to their implementation in a pilot study.MethodsA working group from Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS) agencies was created to develop educational materials in collaboration with patients. The content was based on international initiatives and feedback from the working group. The project was initiated in November 2020. The team consisted of HTA researchers, technicians with experience in training and virtualization and patients. The final version was obtained after an iterative process and refinement of the content and design.ResultsThe materials were published in complete and summary versions, and they were translated into Catalan, Basque and Galician. The online course was designed in an e-learning platform (Moodle) with the aim of being implemented by each of the agencies. The materials include relevant and summarized information on HTA processes, current framework at national and European level, and the role of patients in HTA. Health research and the importance of qualitative and quantitative methods are also addressed. The course also includes a module of practical aspects of patient and citizen participation for achieving an effective contribution to HTA. The course is being piloted with patients in different regions in Spain. The objective of the pilot is to evaluate the usefulness and satisfaction with the course, and it has been designed with the purpose of incorporating the pertinent modifications in the course.ConclusionsThe online training course is intended to facilitate the acquisition of knowledge related to the processes and tools of HTA for patients, as well as to inform them in what phases and in what way they can participate. The pilot will provide relevant information on its use in practice. It is expected that the course will favor capacity building and patient involvement.
PP140 Economic Evaluation Of Several Vaccination Strategies Against Rotavirus In Spain
Authors: Imaz-Iglesia Inaki , Carmona Montserrat , García-Carpintero Esther E. , Martínez-Portillo Alejandro , Alcalde-Cabero Enrique , García-Pérez Lidia , Linertová Renata 
Published: December 2022

IntroductionThe Spanish Ministry of Health asked us about the efficiency of extending the current rotavirus vaccination strategy to all newborns. The current strategy is to vaccinate only to high-risk newborns (premature and those qualified as high-risk by a pediatrician). The objective of this research was to compare three strategies: no-vaccination, vaccination of high-risk newborns and universal vaccination, considering the two vaccines available in Spain: RotaTeq® and Rotarix®.MethodsA cost-utility analysis, based on a de novo Markov model, was carried out both from a societal and a healthcare system perspective. The model follows a cohort of newborns during their life-course. The cycle length is annual and a half-cycle correction was applied. A discount rate of 3 percent was applied in the base case both to costs and utilities. Most of the incidence, probabilities and costs data were Spanish. The Quality Adjusted Life Year (QALY) data were taken from international literature. We assumed a willingness to pay threshold of EUR 25,000 per QALY gained. We performed deterministic one-way sensitivity analysis.ResultsCompared to no-vaccination, the high-risk vaccination strategy is cost-effective assuming the above- mentioned threshold only with Rotarix® from a societal perspective (RotaTeq® EUR 32,008 per QALY; Rotarix® EUR 23,368 per QALY). Universal vaccination is not cost-effective either compared to no-vaccination or compared to the high-risk vaccination strategy and with both perspectives. Vaccine prices and efficacy data are highly sensitive variables. We find that universal vaccination would be cost-effective with a discount of 44.6 and 36.9 percent of the current price of RotaTeq® and Rotarix®, respectively.ConclusionsUniversal vaccination would not be a cost-effective strategy for Spain with either of the two vaccines at current prices. Vaccination of high-risk newborns would be cost-effective at current prices and from a societal perspective only with Rotarix®. Substantial vaccines price reductions could make the universal vaccination a cost-effective option in Spain.
PP142 Time-Driven Activity-Based Costing (TDABC) Of Brazilian Public Healthcare System (SUS): Preliminary Results For Osteogenesis Imperfecta (OI)
Authors: Azevedo Camila , Souza Juliana , Gargano Ludmila , Salvador Bianca , Santana-da-Silva Luiz Carlos , Ogata Gabriel , Nita Marcelo , Félix Têmis 
Published: December 2022

IntroductionImproving the value of healthcare delivered requires accurate cost information, which can be challenging for rare diseases. We report direct costs of patients with OI using the TDABC methodology.MethodsThis research is part of a nationwide observational study to assess the “Value of Healthcare Journey for Patients With Rare Diseases” in SUS. Patient journey and economic data was collected for the actual clinical practice in each medical center enrolled in the project. We set the starting point of disease and map a patient’s path in the healthcare journey, including treatment, exams, procedures and appointments needed. Unit cost, time and amount of resources to perform each activity, such as human resources, materials and medicines, was assessed, disregarding indirect costs. Here we present preliminary results for OI. We considered medical centers from different regions of Brazil. The results are presented in terms of percentage and/or mean and its standard deviation (SD).ResultsThree medical centers completed the data collection. The average [SD] cost of a one year journey of a patient diagnosed with OI is BRL 16,308.07 [11,005.21] (USD 2,886.91 [1,948.36]) per center. Activities with greater cost are medicines, with an average cost of BRL 11,919.47 [12,629.45] (USD 2,109.76 [2,235.52]), followed by materials and human resources, with an average cost of BRL 2,881.91 [3,311.57] (USD 509.92 [585.84]) and BRL 1,506.70 [1,300.46] (USD 266.54 [230.24]), respectively. When assessing the moment of a patient’s journey, the percentage of appointments, diagnosis, treatments and follow-up were 11.2, 25.8, 32.5 and 30.5, respectively. Only 3.3 percent of consumed resources were external to the center (out-of-pocket or private insurance).ConclusionsThe TDABC can efficiently draw the processes and costs associated with it. Medicines are the main driver of annual costs for OI patients in the SUS. This study was funded by the National Council for Scientific and Technological Development – CNPq and the Ministry of Health of Brazil – MoH.
PP146 The Use Of Indirect Comparisons For Reimbursement Decision Making In The Netherlands And England
Authors: Kalf Rachel , Dawoud Dalia , Bregman Caroline , Leckenby Emily , de Vries Marijke J.S. , Delnoij Diana M.J. , Bouvy Marcel L. , Goettsch Wim G. 
Published: December 2022

IntroductionReimbursement decision making is based on a relative effectiveness assessment (REA), which may be combined with a cost-effectiveness assessment, by national Health Technology Assessment (HTA) agencies. These assessments are based on clinical data where new interventions are compared to the current standard of care, which may differ between countries. Since most pivotal trials only include a limited number of interventions, indirect treatment comparisons (ITCs) can be used to compare multiple interventions. The aim of this study was to evaluate the use of ITCs in HTA decision making in the Netherlands and England.MethodsAll pharmaceutical assessments published between 2015 and 2019 by the National Health Care Institute (ZIN) and the National Institute for Health and Care Excellence (NICE) were reviewed to determine whether an ITC had been used. For detailed analysis we included all assessments of ZIN using an ITC, and a random sample of assessments of NICE using an ITC (10 assessments per publication year).ResultsBetween 2015 and 2019 a total of 106 and 265 assessments were conducted by ZIN and NICE, respectively. Of these assessments 48 from ZIN and 150 from NICE included an ITC. The detailed analysis showed that pharmaceutical assessments including indirect comparative evidence led to the REA conclusion of similar therapeutic evidence in 57 percent of 48 assessments by ZIN and in 52 percent of 50 assessments by NICE. Reimbursement recommendations including indirect comparative evidence most often resulted in positive recommendations by ZIN (57% assessments), and in restricted recommendations by NICE (50% assessments). Different methods were employed to incorporate indirect comparative evidence, such as naïve ITCs and network meta-analysis.ConclusionsOur results showed a significant variability in the use of ITCs between NICE and ZIN, which may contribute to differences in their recommendations. Further analysis will provide deeper insight in these differences and may provide suggestions for a clearer international guidance on the use of ITCs for HTA.
PP150 The Role Of Expert Consensus In UK Guidance: Patient Selection For Hydrogel Spacer Use During Prostate Cancer Radiotherapy
Authors: Saunders Rhodri , Payne Heather Ann , Jain Suneil , Peedell Clive , Edwards Albert , Thomas Andrew , Das Prantik , Hedblom Amanda Hansson , Woodward Emily , Bahl Amit 
Published: December 2022

IntroductionIn UK males, prostate cancer is the most common cancer, with over 47,500 diagnosed annually. Radiotherapy is a highly effective curative treatment but can be limited by dose to surrounding normal-tissues such as the rectum. Radiation to the rectum can be reduced by increasing the distance between prostate and rectum with a hydrogel spacer. Despite National Institute of Health and Care Excellence guidance, spacers are not widely funded in the UK. Limited funding has necessitated patient prioritization, without any existing consensus on method.Studies have shown generally homogenous results in reduction of rectal toxicity across assessed subgroups, but the requirement to prioritize remains. One way of addressing the appropriate use of beneficial health technologies is the inclusion of end-user experts in decision-making. The study aim was to identify consensus among radiation oncologists on patient prioritization for rectal hydrogel spacers.MethodsWe conducted a Delphi study where six leading clinical oncologists and one urologist from across the UK experienced in using rectal hydrogel spacers participated in two rounds of online questionnaires and two virtual advisory board meetings.ResultsThe experts estimated that 83 percent of patients who could potentially benefit from a spacer were denied access. Overall, ten points of consensus were reached. Key ones concerning patient-access were:•Spacer use in eligible patients significantly reduces radiation dose to the rectum and toxicity-related adverse events.•Increased benefit is expected in patients on anticoagulation, with diabetes and with inflammatory bowel disease.•Increased benefit can be expected with ultra-hypofractionated radiotherapy, but radiotherapy modality is not a key consideration for patient selection.•Patients should have the opportunity to actively participate in the discussion regarding the use of a spacer.ConclusionsCurrently, not all patients who would benefit can access funding for hydrogel spacers. Consensus in this study indicates that appropriate health policy and funding mechanisms are warranted for patients, to provide equitable access to technologies improving quality of life.