Health Technology Assessment (HTA) Process assists decision-making in health policies. The COVID-19 pandemic caused a high demand on protocol or guideline updates and incorporation of new drugs or therapies, overwhelming local agencies. A recent study reported that major HTA bodies in England, Scotland, Germany, and Canada reduced their number of drug recommendations in 2020, due to reprioritization of resources and COVID-related challenges. The present study aimed to evaluate the impact of the COVID-19 pandemic at the Brazilian National Committee for Health Technology Incorporation (Conitec) recommendation process.

This descriptive study evaluated all official recommendation reports available on the Government website in 2020 and 2021, extracting the data of disease category, technology type, the aim of the report, Public Involvement, and final result for the recommendation. The results were presented in tabular and graphical form using the machine learning, through the software R studio and excel.

A total of 168 documents were evaluated, including guidelines and recommendation reports, with no reduction in the number of evaluations considering 2019. In 2020, there was a more significant evaluation of guidelines, and in 2021, a report on the non-incorporation of technologies. There were four specific documents about COVID 19, including vaccines and hospital care guidelines. The most incorporated and non-incorporated technologies were medication, targeting rare and highly prevalent diseases in balance. The Brazilian government was the main proposer. These results are part of the study “A Survey about the core methods of the recommendation reports for Brazilian Ministry of Health carried out by Brazilian Health Technology Assessment Centers”, which will characterize and analyze the core methods of the recommendation reports conducted by the Brazilian HTA Centers.

The pandemic had a low impact on demands in the routine of the Conitec. Establish indicators and technological norms applicable to health services, contribute to the identification of possible new practices, methods or criteria.

Hemophilia is a rare, inherited bleeding disorder affecting an estimated 400,000 people worldwide (1). Characterized by spontaneous bleeding and long-term, irreversible joint damage, persons with hemophilia are often limited in normal day-to-day activities, including work/school, and require comprehensive care at specialized treatment centers. With replacement therapies extending survival by decades and vastly improving quality of life (QoL), routine prophylaxis is considered the standard-of-care in developed countries. However, due to the cost of replacement factor, access to treatment remains a challenge, and increased scrutiny over funding has been augmented by growing demands on healthcare budgets (2). Thus, the hemophilia community shares a unified goal of objectively defining patient-centered value in hemophilia care.

Using a three-tiered outcomes hierarchy model initially described by Porter (3), an international, multidisciplinary panel of health economics outcomes researchers and hemophilia experts developed a value framework for decision makers to assess value of various healthcare interventions in hemophilia.

The three tiers for assessing value are: (i) Health status achieved/retained; (ii) Process of recovery; and (iii) Sustainability of health. Tier one measures survival, quality of life (QoL), and hemophilia-specific outcomes of bleeding frequency, musculoskeletal complications, and severe bleeds, as well as function/activity (that is, lifestyle impairment). Tier two measures time to initial treatment or recovery and time missed at education/work, as well as disutility of care (that is, inhibitor development, pathogen transmission/infections, orthopedic intervention, and venous access). Tier three measures avoidance of bleeds, maintenance of productive lives, and long-term health, while capturing long-term consequences of insufficient therapy or age-related complications. Applicability of the framework can be demonstrated in areas of healthcare delivery, treatment regimen, and innovation for new therapies.

This value framework represents an initial collaboration with stakeholders to define and organize an array of patient-centric outcomes of importance in hemophilia into a practical tool that can influence treatment and funding decisions in hemophilia care.

The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International’s Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region.

This article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum.

There is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.

Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations.

The participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.

Social engagement in health encompasses the idea of involving (parts of) society as full partners in the decision-making regarding both development and implementation of health technologies. Evidence shows that patient engagement is linked with fewer adverse events, better patient self-management, fewer diagnostic tests, decreased use of healthcare services and shorter lengths of stay in hospitals. Matching the escalating healthcare requirements to face the ongoing societal and economic challenges regarding access and coverage to (new) health technologies is not an easy task for health providers.

We conducted a systematic review (CRD42017068714) designed to address the institutional implementation of social engagement by the Brazilian Ministry of Health. All systematic reviews were evaluated using the new version of AMSTAR and, once all findings are synthesized, we will use the GRADE-CERQual approach to assess for confidence.

From 399 publications that met the inclusion criteria, 80 described the implementation of social engagement during the development and implementation of (new) health technologies at various levels (local, regional, national, supranational), countries and for different health technologies and social actors. The remaining 319 publications constitute case studies describing barriers and enablers to implementing social engagement in HTA and coverage decision-making processes. By mapping barriers and facilitators, we explored effectiveness and sustainability, further observing how citizen science-based strategies can ultimately reform health service delivery by innovating the social engagement in health technology development and implementation.

This systematic review addresses the know-do gap on social engagement in health technology development and implementation, from a global perspective, as a way of improving the Brazilian Ministry of Health's HTA activities and enabling a Brazilian strategy to reform health service delivery. Enabling social engagement as early as possible, during all the stages of the development cycle, grants a more effective and sustainable health care system.

Cystic Fibrosis (CF) is a genetic disorder caused by mutations in CFTR gene. In Italy, reported prevalence is approximately .70 per 10,000 inhabitants (1). The practice and recommendations for Cystic Fibrosis carrier screening are very heterogeneous in Europe. A proposal of a carrier genetic test in the general population raises many questions. Health Technology Assessment (HTA) could offer a sound methodological basis for this evaluation. The aim of this work was to summarize the available evidence, using the HTA approach, on the genetic tests for Cystic Fibrosis carrier screening.

A systematic literature search was used to find the best available international and national evidence on genetics test for CF carrier screening. In this report, we specifically addressed the health problem of disease, description and technical characteristics of tests – its analytic and clinical validity, and clinical utility. Economic evaluation of different scenarios was synthesized from the literature. Ethical, organizational, and social aspects of CF and genetic screening were also considered.

Several screening strategies have been evaluated in the literature and screening options can be characterized by different timing, model and place of screening (2). The reported cost of a screening test ranged from EUR25 to EUR212 (3). Estimated life time cost of care for CF patients ranged from EUR291,048 to EUR1,105,452. Ethical analysis emphasized that the use of these tests is an advantage in terms of the acquisition of knowledge and of responsible management of choices, but at the same time raises many ethical questions. Social considerations reported among patients and their families an overall positive attitude toward population CF carrier screening.

The advances in the molecular genetics technology have made CF carrier testing reliable and affordable. The multidisciplinary approach of this HTA provided an evidence-based evaluation of genetic tests and offers a firm scientific background for the decision-makers to consider the implementation of a screening for Cystic Fibrosis carriers into the Italian health care system.

In 2020, the Swedish regions cemented a national managed-introduction project for medical devices, to ensure equal, cost-effective, and appropriate use. Health technology assessment (HTA) is an important component of the project. Swedish national health registers have world-class coverage and completeness but may lack information enabling adequate evaluation of medical devices. This study reviews the current situation and ongoing initiatives. Additionally, the potential for medical device health economics and outcomes research (HEOR) using Swedish registers is assessed.

A review of Swedish national health registers was undertaken, focusing on available data, and contextualized for the purpose of HEOR for medical devices. Additionally, the review included an evaluation of the Swedish reimbursement authority's (The Dental and Pharmaceutical Benefits Agency, TLV) ongoing initiatives to improve the potential to follow-up the impact of the technologies they assess and develop value-based pricing schemes.

Five registers were deemed the most relevant national health registers for device research. They include high-quality longitudinal data and are linkable on a per-patient basis. For devices, main limitations include limited data on specialized outpatient care, lags in updating certain registers, lack of laboratory data, and challenges in identifying the specific device used. Reports indicate that certain limitations are being addressed, including pilot-studies investigating the opportunity for automated reporting of data from regional systems, and app-collected patient-reported health data.

Swedish registers provide comprehensive sources for HEOR studies, but limitations related to the assessment of medical device impact remain. As is common with register data reporting grouped diagnoses and interventions, specific devices are not directly identifiable in the national health registers. For some devices, this might be addressable through linkage with other data-sources. Swedish authorities are undertaking several initiatives that will likely improve the potential for HTA and follow-up of medical devices using national health register data.

Written patient evidence is submitted to the National Institute for Health and Care Excellence (NICE) by patient organizations and their nominated patient experts. We reviewed the impact that patient evidence had on the committee's decision making. This local learning can help generate global opportunities for Health Technology Assessment (HTA) bodies and patient groups to further develop their understanding and methodology about how patient evidence can support HTA decision making for ultra-orphan conditions.

There were two phases.

Phase one was an online questionnaire about the impact of patient evidence on the committee's decision making for ultra-orphan HTA evaluations. It was sent to the committee chair, lay committee members and selected other committee members.

Phase 2 developed the initial questionnaire findings using in-depth interviews with the committee chair and the lay members. These gained further understanding of the impact of patient evidence and the themes raised.

Key findings showed patient evidence was helpful to understand the:

• • Burden of disease

• • Patient population

• • Likely uptake of new medicines

• • Impact on carers.

For ultra-orphan conditions, where other forms of evidence are scarcer, patient evidence is fundamental to understanding patient needs, the impact of the disease, patient population and preferences.

Patient evidence was useful for the committee in different ways; it provided the committee with new evidence and it helped the committee understand and interpret the evidence submitted by others. Both are key to committee decision making. It was clear that due to the very small patient population, patient groups knew the patient population, their stage of disease, and their preferences in detail.

The findings will be used to inform an updated patient submission template for ultra-orphan HTAs, and supporting guide. These will be available on the NICE website and offered to the HTAi Interest Group on Patient and Citizen Involvement so they can be shared globally.