Consolidated Health Economic Evaluation Reporting Standards for Interventions That Use Artificial Intelligence (CHEERS-AI).

OBJECTIVES: Economic evaluations (EEs) are commonly used by decision makers to understand the value of health interventions. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS 2022) provide reporting guidelines for EEs. Healthcare systems will increasingly see new interventions that use artificial intelligence (AI) to perform their function. We developed Consolidated Health Economic Evaluation Reporting Standards for Interventions that use AI (CHEERS-AI) to ensure EEs of AI-based health interventions are reported in a transparent and reproducible manner. METHODS: Potential CHEERS-AI reporting items were informed by 2 published systematic literature reviews of EEs and a contemporary update. A Delphi study was conducted using 3 survey rounds to elicit multidisciplinary expert views on 26 potential items, through a 9-point Likert rating scale and qualitative comments. An online consensus meeting was held to finalize outstanding reporting items. A digital health patient group reviewed the final checklist from a patient perspective. RESULTS: A total of 58 participants responded to survey round 1, 42, and 31 of whom responded to rounds 2 and 3, respectively. Nine participants joined the consensus meeting. Ultimately, 38 reporting items were included in CHEERS-AI. They comprised the 28 original CHEERS 2022 items, plus 10 new AI-specific reporting items. Additionally, 8 of the original CHEERS 2022 items were elaborated on to ensure AI-specific nuance is reported. CONCLUSIONS: CHEERS-AI should be used when reporting an EE of an intervention that uses AI to perform its function. CHEERS-AI will help decision makers and reviewers to understand important AI-specific details of an intervention, and any implications for the EE methods used and cost-effectiveness conclusions.

A systematic review of studies that estimated the burden of chronic non-communicable rare diseases using disability-adjusted life years.

BACKGROUND: Initiatives aiming to assess the impact of rare diseases on population health might be hampered due to the complexity of disability-adjusted life years (DALYs) estimation. This study aimed to give insight into the epidemiological data sources and methodological approaches used in studies that estimated DALYs for chronic non-communicable rare diseases (CNCRD), and compare its results. METHODS: A literature strategy was developed for peer-review search in Embase and Medline, and also performed on grey literature databases and population health and/or rare disease-focused websites. We included studies that determined the burden of CNCRD listed on the Orphanet's and/or the Genetic and Rare Diseases information center (GARD) websites. We excluded communicable and occupational diseases, rare cancers, and cost-effectiveness/benefit studies. Two researchers independently screened the identified records and extracted data from the final included studies. We used the Guidelines for Accurate and Transparent Health Estimates Reporting (GATHER) statement to assess the quality of reporting of the included studies. The data synthesis depicted the studies' characteristics, their distribution by geographic coverage and the group of disease(s) they focused on, the methods and data input sources used and estimated DALY per case. RESULTS: In total, 533 titles were screened, and 18 studies were included. These studies covered 19 different CNCRDs, of which most fell in the disease category "Diseases of the nervous system". Diverse methodological approaches and data input sources were observed among burden of CNCRD studies. A wide range of DALY per case was observed across the different studies and diseases included. CONCLUSIONS: A low number of burden of CNCRD studies was observed and most estimates resulted from multi-country studies, underlining the importance of international cooperation to further CNCRD research. This study revealed a lack of epidemiological data and harmonization of methods which hampers comparisons across burden of CNCRD studies.

Valuing the "Burden" and Impact of Rare Diseases: A Scoping Review.

Introduction: Rare diseases (RDs) are a severe, chronic, degenerative and often life-threatening group of conditions affecting more than 30 million people in Europe. Their impact is often underreported and ranges from psychological and physical symptoms seriously compromising quality of life. There is then a need to consolidate knowledge on the economic, social, and quality of life impacts of rare diseases. Methods: This scoping review is the result of 9 qualitative interviews with experts and a literature search on Cost-of-Illness (COI) studies and quality of life (QoL) studies following the PRISMA methodology. Grey literature was also included to complement findings. Results. 63 COI studies were retrieved, covering 42 diseases and a vast majority of them using a prevalence-based approach (94%). All studies included medical costs, while 60% included non-medical costs, 68% productivity losses and 43% informal care costs. 56 studies on QoL were retrieved, mostly from Europe, with 30 different measurement tools. Grey literature included surveys from the pharmaceutical industry and patient organisations. Discussion: The majority of studies evaluating the impact of RDs on the individual and society use the COI approach, mostly from a societal perspective. Studies often vary in scope, making them difficult to consolidate or compare results. While medical costs and productivity losses are consistently included, QoL aspects are rarely considered in COI and are usually measured through generic tools. Conclusion: A comprehensive study on impact of rare disease across countries in Europe is lacking. Existing studies are heterogeneous in their scope and methodology and often lack a holistic picture of the impact of rare. Consensus on standards and methodology across countries and diseases is then needed. Studies that consider a holistic approach are often conducted by pharmaceutical companies and patient organisations exploring a specific disease area but are not necessarily visible in the literature and could benefit from the sharing of standards and best practices.