Health Technology Assessment Agencies Research Articles

PP14 The Role Of Health Technology Assessment In The Public Hospitals Of The National Health Service Of Catalonia

IntroductionHospitals play an essential role to facilitate the appropriate introduction of health technologies into the National Health System. We aimed to analyze if the decision-making at the hospital level is based on health technology assessment (HTA).MethodsWe developed a seventeen-question survey to assess the decision-making mechanisms for the introduction of health innovation into the public hospitals of the National Health Service (NHS) of Catalonia. All questions were related to the field of HTA and health innovation. We asked about the process of new health technology acquisition, the existence of HTA units, and the impact those units might have within the center. We invited the three main public healthcare providers (n= 68 centers). We performed a quantitative analysis grouping the results by type of healthcare provider.ResultsThirty-five different health providers (51.5%) remitted forty-two responses. We identified two differential mechanisms for the introduction of health innovation in those centers: 51.4 percent used a directive committee for decision-making, whereas 37.1 percent employed a technical commission. Some centers (8.6%) used both options. Both the directive committees and technical commissions predominantly considered the professional experience or scientific evidence provided by the healthcare professionals of the center (80%) or external HTA reports or clinical practice guidelines (65.7%) to guide their decision-making. Using HTA products developed within the center was less frequent (34.3%). Only 57.1 percent of the healthcare providers had an HTA unit, but 75 percent of them declared that HTA products had a direct and quantifiable impact on their decision-making regarding the introduction of innovation. Healthcare providers without HTA units manifested their willingness to adopt HTA practices into their decision-making processes.ConclusionsHTA at the hospital level is not a common practice when deciding to introduce innovative health technologies in the NHS of Catalonia. However, the main healthcare providers agreed that the introduction of HTA is necessary for improving their decision-making and that the HTA agency, Agency for Health Quality and Assessment of Catalonia (AQuAS) will play a key role (training, promoting, and assessing).

PP07 Vaccine Decision-making In Canada: Processes And Guidelines For Using Economic Evidence

IntroductionCanada’s National Advisory Committee on Immunization (NACI) makes recommendations on the use of human vaccines. Provinces and territories subsequently use the advice to make decisions on public funding and program implementation. Traditionally, NACI reviewed vaccine characteristics and burden of illness.With its recent expanded mandate, NACI now considers cost-effectiveness via economic evaluations, among other decision determinants. As such, new processes and guidelines were needed to formalize the incorporation of economic evidence into federal vaccine decision-making.MethodsTwo task groups were convened respectively to develop NACI’s “Economic Process” and “Guidelines for the Economic Evaluation of Vaccination Programs in Canada”. The groups conducted environmental scans to inform their work, as well as engaged with government partners, decision-makers, academics, national immunization technical advisory groups from other countries, health technology assessment agencies, industry, patient groups, among others.ResultsThe Economic Process outlines when and how NACI incorporates economic evidence for vaccine recommendation. For instance, it describes how policy questions are prioritized given institutional capacity constraints for generating economic evidence. It also describes how policy questions are assessed to determine the appropriate type of economic evidence required (i.e., systematic review, economic evaluation, multi-model comparison of external models).The Economic Guidelines provide recommendations in 15 chapters on how to conduct economic evaluations (i.e., from defining the decision problem to reporting). Unlike other health technologies, vaccines have the potential to affect both vaccinated and unvaccinated individuals. Hence, the Guidelines consider population-level impacts such as externalities (e.g., herd immunity, age-shifting of disease) and spillover effects. They also discuss equity considerations and non-health impacts of vaccines such as to productivity, consumption and education.ConclusionsThe Economic Process and Economic Guidelines promote the generation and use of credible and standardized economic evidence. They advocate for transparency, allowing evidence to be used across jurisdictions beyond Canada. Next steps include documentation of user feedback, incorporation of Indigenous considerations, and formal evaluations.

HTA283 A Scoping Review of Health Technology Assessment Agency (HTA) and Health Economic (HE) Modelling Guidelines for Identification of Health Economic Model Inputs

HTA283 A Scoping Review of Health Technology Assessment Agency (HTA) and Health Economic (HE) Modelling Guidelines for Identification of Health Economic Model Inputs

OP135 Machine Learning And Cancer Registry: Evaluation Of The Effectiveness Of Case Coding

IntroductionMachine learning (ML) algorithms are computational procedures that use pattern recognition and inference by learning from previously categorized documents to predict the category to which a new document belongs. The role of machine learning within cancer registries remains unclear given the lack of in-depth testing and guidance from health technology assessment (HTA) agencies. We evaluated the effectiveness of coding new cases through machine learning at the Integrated Cancer Registry.MethodsThe Integrated Cancer Registry covers the eastern area of Sicily in Italy, which has an annual average incidence of about 10,000 cases of malignant neoplasm. Potential new cancer cases were retrieved from pathology services and processed by pathologists who confirmed the neoplastic nature of supposed cases and specified the morphological type and location of the tumors. The current method involves identification by reading the free-text report when International Classification Diseases for Oncology information was not provided. We used the new Microsoft ML.Net Library, a framework developed in response to the challenge of facilitating machine learning pipeline utilization in large software applications. A total of 1,050,952 free-text pathology reports published from 2003 to 2018 were selected separately from all Sicilian pathology services and uploaded to machine learning software that explored eight binary classification algorithms.ResultsWe evaluated each algorithm’s performance by calculating metrics (the number of true positives, true negatives, false positives, and false negatives) from the classification procedure applied to the test dataset. The metrics used were accuracy, F1 score, and area under the receiver operating characteristic curve. With a test set of around 210,000 text diagnoses, each algorithm reached an F1 score of up to 95 percent.ConclusionsMachine learning algorithms capture relevant information about tumors from free-text pathology reports, optimizing the process and reducing waste. With the help of machine learning systems, cancer registries can provide more timely data for research and evaluation of all types of new cancer technologies (drugs, devices, radiology and radiotherapy equipment, diagnostic devices, robotic surgery, and vaccines).

RWD23 Comparison of the Use of Real-World Evidence (RWE) by Health Technology Assessment (HTA) Agencies in Reviews of Drugs for Rare Diseases (DRDS)

RWD23 Comparison of the Use of Real-World Evidence (RWE) by Health Technology Assessment (HTA) Agencies in Reviews of Drugs for Rare Diseases (DRDS)

PP13 Results Of An International Survey About Barriers In Patient Involvement In Health Technology Assessment Of Digital Health Technologies

IntroductionIn recent years, several efforts have been initiated by health technology assessment (HTA) agencies to increasingly incorporate patient involvement (PI) into their assessment processes. The rationale behind PI in HTA is that patients can give their perspective and experiences about health, illness and the use of health technologies, complementing clinical and healthcare system standpoints.A recent systematic review summarized current evidence on the barriers and facilitators of PI in HTA. Barriers and facilitators were classified according to the context in which they appear, as organizational, decision-making, political and community-related.Digital health technologies offer a singular opportunity to address some challenges faced by healthcare systems. However, we hypothesized that their intrinsic characteristics may have some implications for PI in digital HTA. We conducted a survey to gain knowledge about barriers in PI in digital HTA.MethodsThe survey was elaborated using Microsoft Forms and consisted of 31 questions divided into four blocks (organizational, decision-making, political and community-related). It was sent to three Spanish, one Catalan and one International patient umbrella organizations. The results were analysed using Microsoft Excel.ResultsA total of four responses were received from three Spanish and one Catalan patient umbrella organizations. Results showed high, partial, and low concordance among organizations in 65 percent, 19 percent and 16 percent of the answers received, respectively. According to the results, most of the barriers for PI seem to be the same for HTA of digital health technologies as for other types of health technologies. However, two or three of the four umbrella organizations consider that HTA of digital health technologies might pose greater challenges in terms of achieving significant patient participation, sufficient patient training and knowledge of HTA processes, proper preparation and tools of the HTA experts and logistics during the HTA process.ConclusionsBarriers in PI are mostly the same for digital or other types of health technologies. Few differences are related to patient training, tools, and logistics during HTA process.

OP114 Health Technology Assessment Of Digital Technologies: Results From Two Past Examples And Thoughts For The Future

IntroductionDigital health care is a rapidly growing and increasingly diverse area of medicine and social care. Health technology assessment (HTA) agencies around the world are engaging with digital health technologies and developing methods for appropriately assessing their value. Given our experience in HTA for pharmaceuticals and how methodologies must differ when assessing medical devices and diagnostics, we must be proactive in rapidly developing useful methods for assessing a variety of digital health technologies.MethodsWe will first present the results of two completed HTAs from the UK and contrast their methods and results, while looking at the UK approach to assessing digital health care in general. Next, we will present preliminary results from an ongoing consensus conference study of HTA’s for integrated digital technologies. This study will use a modified Delphi methodology to develop statements for discussion at a consensus conference. The primary research question is “how digital technologies can help build resilient healthcare systems and how HTAs can assess their value”.ResultsAn HTA conducted in the UK of an artificial intelligence-enabled ambulatory electrocardiogram device resulted in a recommendation for further real-world evidence generation. Another HTA of a digital therapeutic app for insomnia, which was also conducted in the UK, resulted in a positive recommendation but the process was lengthy and needed to be flexible. The preliminary results of an upcoming consensus conference study will also be presented. The consensus panel will consist of a range of stakeholders from digital experts, HTA practitioners, clinicians, health economists, industry representatives, and policy makers.ConclusionsDigital technology is now a broad field with a wide range of very different technologies included in its definition. Questions remain as to whether digital technologies should be assessed using a traditional pharma-based framework for HTA, a MedTech model, or something else entirely. Given our past experience of developing flexible methods for HTA, we have an opportunity to develop useful recommendations and best practices from the ground up.

OP43 Translating Cell And Gene Therapy HTA Into Practice – Building The Plane As We Fly It

IntroductionAssessing, funding, and implementing cell and gene therapies are usually based on limited evidence. This requires health technology assessment (HTA) agencies to develop new methodologies; payers to accept risk-based funding; industry to consider the right evidence and price; hospitals to consider if they have the necessary requirements; patients to consider their risk appetite for gene therapies; and health departments to consider all the above. Ensuring timely patient access to these therapies is challenging in Australia’s federated health system. To ensure stakeholders are aligned, all must come to the table and share their respective insights, experiences, and expertise to support planning, decision-making, funding, and commissioning of these therapies. It is time to develop a new consultative and decision-making paradigm to expedite HTAs, funding, commissioning, and timely patient access for cell and gene therapies.MethodsAustralian federal, state, and territory government representatives agreed to develop a framework that clarifies processes around information sharing, HTA, funding, commissioning, and monitoring of highly specialized therapies and services (including cell and gene therapies). A draft national framework was developed that addresses assessment, funding, and implementation of high cost, highly specialized therapies and services (e.g., bone marrow transplants). However, it is unclear whether non-government stakeholders have been consulted.ResultsThe framework for assessing, funding, and implementing high cost, highly specialized therapies and services across Australia’s public hospital system is pending endorsement by each of the jurisdictional governments. High-level in nature, the framework’s primary audiences are industry and public hospitals. While not all processes are in place, the framework is forward-looking. A detailed implementation plan is warranted to better inform the roles and requirements of each stakeholder.ConclusionsThe framework allows stakeholders to better understand government processes regarding assessment, funding, and implementation of high-cost therapies and services, thereby fostering a collaborative environment that supports timely patient access. Articulating process details in a follow-up implementation plan is essential to gain the trust of, and input from, industry, clinicians, and patient representatives.

OP109 The Conceptualization And Value Of A Disease Management Approach To HTA In Canada: Findings From A Qualitative Study

IntroductionOur objective was to conceptualize and assess the potential value of a disease management approach to health technology assessment (HTA) in Canada.MethodsWe conducted 18 semi-structured interviews between April 2022 and October 2022 to elicit informant views on potential opportunities for re-conceptualizing the decision-problem in HTA as a disease management problem versus a technology management problem. Participants were purposefully sampled from national and provincial HTA agencies and related organizations in Canada to achieve representation across the disease pathway including prevention, screening, and treatment, and the decision-making pathway including HTA organizations, expert committee members, and decision-makers. Data were analyzed using thematic organizations (based on the interview guide) and manual line by line coding of the data. Ethics approval was received from the Health Sciences Research Ethics Board at the University of Toronto.ResultsThree key features of a disease management approach to HTA (i.e., disease-based, multi-interventional, and dynamic) emerged from informants that differed from traditional HTA processes in Canada. The concept was generally not perceived to be a new idea – some informants indicating that it was implicit in the HTA analysis framework. There was general support for an explicit disease management approach to HTA if the impact of the approach could be demonstrated, if the assessment could be completed within an appropriate time frame, and if the assessment could include the equity, ethical and implementation domains of HTA. Informants indicated that the reconceptualization of HTA could lead to effective and efficient decision-making throughout a technology’s lifecycle, help breakdown system silos, and offer a platform for greater consideration of non-drug alternatives and upstream interventions. The impact of this approach was anticipated to contribute to a proactive health system that could improve population health, enhance the patient experience, and ensure appropriate stewardship of health care resources.ConclusionsA disease management approach to HTA has international relevance as an approach that could promote integrated, proactive, sustainable, and resilient health systems.

PP11 Patient Involvement In Drug Evaluations To Inform Funding Decisions: A Singapore Case Study

IntroductionPatient involvement has become increasingly integral to health technology assessment (HTA) processes globally due to greater recognition of the important contribution patients make to address uncertainties in the scientific evidence base and interpret results for real-world implementation. To align with best practices and encourage meaningful patient input in HTAs in Singapore, patient involvement processes were established by the Agency for Care Effectiveness (ACE) in 2021. This presentation discusses how patient groups were identified in Singapore and describes the new patient involvement processes.MethodsIn the absence of a centralized database, a stakeholder mapping exercise was undertaken in 2021 to identify all relevant patient and volunteer groups in Singapore. A comprehensive search of the Singapore Charity Portal, hospital websites, standard search engines and social media platforms was conducted.Identified groups were screened in line with specific inclusion criteria and contacted via email and cold calling to find out more about their remits. Plain English resources, targeted training materials and a process guide to encourage patient involvement in ACE’s work were co-developed with local patient organizations by drawing upon best practices from overseas HTA agencies contextualized to local patients’ needs. Supporting resources and processes were revised in 2022 based on ACE’s experience receiving inputs from patients and caregivers to inform drug HTAs.ResultsOne hundred and six patient groups covering 20 conditions were identified including registered organizations and informal support groups. In the first half of 2022, ACE received responses from 82 patients from ten patient organizations to inform seven drug HTAs for cancer, diabetes, HIV, and other conditions in line with the new patient involvement processes. Patient organizations viewed the opportunity to submit testimonials of their lived experience with different conditions as a meaningful and important activity for their members.ConclusionsPatient involvement processes have improved the legitimacy and acceptance of ACE’s work and will be continuously revised to ensure that they remain relevant and meet patients’ expectations and needs.

PP138 Awareness Of Health Technology Assessment And Its Predictors Among Medical Students In Public Universities In Klang Valley, Malaysia

IntroductionLack of awareness has been recognized as one of the most important barriers in the implementation of health technology assessment (HTA) across numerous countries. Medical students have been identified as a vital target group to instill an awareness, basic knowledge, and positive attitude towards HTA from the beginning of their medical career. Presently, there was no study found conducted on HTA awareness in medical students in Malaysia. This study aims to determine the level of awareness of HTA, and its predictors among clinical year medical students in public universities in Klang Valley, Malaysia.MethodsA cross-sectional study using the stratified random sampling method was conducted among clinical year medical students in four public universities in Klang Valley, Malaysia. The data on level of awareness of HTA and its associated factors were collected using a self-administered online questionnaire. Data were analyzed using IBM SPSS version 27 to determine the level of awareness of HTA, its associated factors and predictors.ResultsThe total number of study participants was 323 and the response rate was 90 percent. The analysis showed that 69 percent of respondents had low level of awareness of HTA while 31 percent had high level of awareness of HTA. Factors associated with the level of awareness of HTA was attitude towards HTA (p < 0.001). The predictors of level of awareness of HTA were attitude towards HTA (Adjusted Odds Ratio (AOR) = 7.417, 95% confidence interval [CI]: 3.491, 15.758), peer interaction on HTA (AOR = 0.320, 95% CI: 0.115, 0.888), and previous training on HTA (AOR = 4.849, 95% CI: 1.096, 21.444).ConclusionsThe majority of clinical year medical students in public university have low awareness of HTA which can be predicted by the level of attitude, peer interaction and training of HTA. Therefore, it is important to increase awareness among these future medical practitioners to encourage evidence-based decision-making. Early exposure and introduction of HTA in the current university curriculum in collaboration with a local HTA agency can be recommended to increase awareness as well as to improve students’ attitude towards HTA.

OP09 Developing A Patient And Consumer Training Tool Explaining Health Technology Assessment And Patient/Consumer Participation: The French Experience

IntroductionThe French National Authority for Health’s (HAS) 2019-2024 strategic workplan called for “making public involvement a priority.” This project was designed within the roadmap validated in 2021; “Strengthening public involvement in health technology assessment (HTA) at the HAS”, including an action of building a means of training patient organizations for their contribution. The project’s overall objective was to develop with patients and consumers a first training tool, representing an online knowledge base targeting French patients and consumers to explain the HAS HTA process and assist their participation in these assessments.MethodsThree stages were designed to meet this objective. Firstly, an international benchmark was performed of available online HTA training tools for patients and consumers, notably those used by HTA bodies and European and international patient and consumer groups (PCGs). Secondly, an internal HAS search for e-learning tools, was conducted to identify whether they could meet the training needs of French patients and consumers. Finally, the training tool was developed via a working group composed of patients and HAS scientific officers.ResultsThe benchmark identified 82 online training tools selected according to the specified inclusion criteria. Sixteen international HTA agencies, nine European and international PCGs and 13 other bodies provided online HTA training tools for patients and consumers during the research period, but no journal articles identified such tools. Eleven formats and 12 key themes, divided into two main categories were identified: important content related to HTA, and important content related to public involvement in HTA. The HAS search for e-learning tools resulted in internal e-learning tools offered for clinical experts not meeting the needs and preferences of patients and consumers. Finally, HAS based its training tool development on these preferences and needs to create a PowerPoint in two blocks of modules covering the two main categories above (six modules in total).ConclusionsFrench patients and consumers preferences and needs for a HTA training tool were inspired by the international benchmark, dividing key themes into two main categories: important content related to HTA, and important content related to public involvement in HTA. This resulted in HAS development of two blocks of modules in PowerPoint format covering these two categories.

HTA351 A Targeted Review Exploring How English and German Health Technology Assessment Agencies Differ in Their Appraisal of Digital Health Apps

HTA351 A Targeted Review Exploring How English and German Health Technology Assessment Agencies Differ in Their Appraisal of Digital Health Apps

PP133 What Services And Products Should A Health Technology Assessment Agency Provide?

IntroductionHealth 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.MethodsA 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.ResultsThe 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.ConclusionsThe 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.

HTA28 The Use of Expert Opinions, Patient, and Carer Perspectives and Real-World Evidence in Orphan Disease Submissions Differs for Health Technology Assessment Agencies Across the World

HTA28 The Use of Expert Opinions, Patient, and Carer Perspectives and Real-World Evidence in Orphan Disease Submissions Differs for Health Technology Assessment Agencies Across the World

PP38 Designing A Training And Capacity Building Pathway In Patient Involvement

IntroductionThe Patient Involvement (PI) Interest Group of the Spanish Network of Health Technology Assessment Agencies (RedETS) was set up in 2017 by a group of health technology assessment (HTA) researchers interested in PI. Since its inception, training and capacity-building to support PI and patient-based evidence in HTA processes has been one of its main aims. The objective of this work was to identify the needs and priorities related to training and capacity building activities to be developed within the framework of the PI Interest Group.MethodsThe PI Interest Group met on November 14, 2022, for its Annual Meeting. The group discussed the needs, priorities and possibilities on training, and carried out a prioritization exercise. For this purpose, a self-reported and anonymous questionnaire was used, which included 16 training activities. Every item was scored with a Likert-type scale ranging from 0 to 10.ResultsThe questionnaire was answered by twenty participants. The most highly rated training activities (mean less than or equal to 8) were: qualitative evidence synthesis (8.75); PI case studies (basic (8.65) and advanced (8.56) level); quality assessment tools for qualitative evidence (8.37); and qualitative research (8.11). Other proposals scoring above 7 points were: ethical aspects related to PI, evaluation of patient participation and impact, identification and recruitment procedures, and discrete choice experiments. The group agreed to organize bi-monthly webinars and three structured training activities for the whole RedETS network on: Qualitative Evidence Synthesis, Qualitative Research and PI Case Studies.ConclusionsThe prioritization of training activities according to PI Interest Group members allowed planning a tailored capacity-building program adapted to the needs of RedETS.

Recent changes to adult national immunization programs for pneumococcal vaccination in Europe and how they impact coverage: A systematic review of published and grey literature

ABSTRACT Despite widespread use of pneumococcal vaccines throughout Europe, the burden of pneumococcal disease (PD) in adults is considerable. To mitigate this burden, National Immunization Technical Advisory Groups (NITAGs) and Health Technology Assessment (HTA) agencies assess the value of different vaccine schedules for protecting against PD. The aim of this review was to assess the evidence and rationales used by NITAGs/HTA agencies, when considering recent changes to National Immunization Programs (NIPs) for adults, and how identified changes affected vaccine coverage rates (VCRs). A systematic review was conducted of published literature from PubMed® and Embase®, and gray literature from HTA/NITAG websites from the last 5 y, covering 31 European countries. Evidence related to NIP recommendations, epidemiology (invasive PD, pneumonia), health economic assessments and VCRs were collected and synthesized. Eighty-four records providing data for 26 countries were identified. Of these, eight described explicit changes to NIPs for adults in seven countries. Despite data gaps, some trends were observed; first, there appears to be a convergence of NIP recommendations in many countries toward sequential vaccination, with a pneumococcal conjugate vaccine (PCV), followed by pneumococcal polysaccharide vaccine 23. Second, reducing economic or healthcare burden were common rationales for implementing changes. Third, most health economic analyses assessing higher-valency PCVs for adults found its inclusion in NIPs cost-effective. Finally, higher coverage rates were seen in most cases where countries had expanded their NIPs to cover at-risk populations. The findings can encourage agencies to improve surveillance systems and work to reach the NIP’s target populations more effectively.

Epidemiology of Sjögren's: A Systematic Literature Review.

Primary Sjögren's is a multi-system autoimmune disease affecting patients' physical, mental, and emotional wellbeing. The epidemiology of Sjögren's is not well understood, and up-to-date epidemiological evidence is needed to improve knowledge and awareness of Sjögren's among patients and healthcare professionals, and to ascertain the global burden of disease. The objective of this research was to conduct a de novo systematic literature review (SLR) to identify and synthesise evidence on global epidemiology of primary Sjögren's. This SLR was conducted in May 2021 by searching MEDLINE and Embase databases, relevant conference proceedings, websites of registries, and health technology assessment agencies and databases. Publications were systematically screened for English language articles reporting on the incidence, prevalence, age at symptom onset, and age at diagnosis for people with primary Sjögren's. Of 3510 records identified, 68 publications were included, representing 62 unique studies. Studies reported on age at symptom onset (16/62; 25.8%) and age at diagnosis (43/62; 69.4%) more frequently than incidence (7/62; 11.3%) and prevalence (9/62; 14.5%). Primary Sjögren's was found to have the highest incidence and prevalence in females and in older age groups (incidence: ≥65years; prevalence: ≥75years). Average age at onset and diagnosis of primary Sjögren's ranged between 34-57years and 40-67 years, respectively. This SLR identified a paucity of incidence and prevalence data for primary Sjögren's, highlighting a need for further epidemiological studies. The global Sjögren's community must work together to follow the defined classification criteria of primary Sjögren's and reporting guidelines for incidence and prevalence data to allow for meaningful epidemiological comparisons across studies, settings, and countries.

Criteria adopted in different models of public healthcare systems for the evaluation of reimbursement recommendations of orphan drugs: a scoping review.

Introdução: Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. The difficulty of conducting robust clinical trials limits the quality of evidence and elevates the cost of development, later translated into the drug’s prices. Thus, it is necessary for the Health Technology Assessment (HTA) agencies to have differentiated criteria for the evaluation of reimbursement recommendations when dealing with such drugs. Objetivos: The objective of this research is to identify and summarize the specific criteria used when evaluating reimbursement recommendations for orphan drugs that are adopted by HTA agencies in countries with different models of public healthcare systems. Material e Método: A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus and Embase up to March 2022. We included any publication type (opinion articles, commentaries, editorials, original articles and reviews) that addressed the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs. Resultados: This scoping review summarizes the identified criteria for 18 countries and ranks them within three models of healthcare systems (NHS, NHI and SHI). We identified that NHS countries, such as the UK, Sweden, and Italy, lean towards innovation, the collection of real-world data, and the impact on organizational aspects of the system. Meanwhile, SHI countries, such as Germany, France and the Netherlands, often employ budget ceilings and expedited evaluation processes. All models shared concern over unmet need and disease nature. The 16 included studies range from 2015 to 2022 and the majority consists of reviews of HTA reports and original articles. Discussão e Conclusões: This review provides a good basis for the understanding of each model’s classification and general tendencies when creating differentiated criteria to accommodate and compensate for the lack of evidence and investment around rare diseases.

Perspectives of women living with type 1 diabetes regarding preconception and antenatal care: A qualitative evidence synthesis.

Pregnant women with type 1 diabetes may have an increased risk of complications for both the baby and themselves. Educational programmes, preconception planning, strict glycemic control and comprehensive medical care are some of the antenatal interventions that have been proposed to improve the outcomes of pregnant women with type 1 diabetes. While some evidence-based recommendations about antenatal care are included in clinical practice guidelines (CPGs), the views, and experiences of women with type 1 diabetes about these interventions are not well known. To understand and synthesize the perceptions of women with type 1 diabetes about the interventions before pregnancy. A qualitative evidence synthesis (QES) was carried out with a framework analysis guided by the Cochrane Qualitative and Implementation Methods Group approach. Three online databases (Medline, Embase and Web of Science) were searched. We included qualitative articles that were published from 2011 to 2021 and which were available in English or Spanish. Ten references met the inclusion criteria of the study and were included. Three main themes were identified: (a) acceptability of antenatal care, (b) feasibility and implementation consideration and (c) equity and accessibility difficulties. Continuity of care, coordination between health professionals and services, and a more holistic approach are the key aspects women say need to be considered for more acceptable, feasible and equitable preconception and antenatal care. This QES was carried out as part of the CPGs on diabetes mellitus type 1, carried out as part of the Spanish Network of Health Technology Assessment Agencies. In this CPG, the representatives of the patient associations are Francisco Javier Darias Yanes, from the Association for Diabetes of Tenerife, who has participated in all the phases of the CPG; Aureliano Ruiz Salmón and Julián Antonio González Hernández (representatives of the Spanish Diabetes Federation (FEDE) who have participated as collaborator and external reviewer, respectively.