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.

Implementation level of best practice policies by Italian Government for healthier food environments: Healthy Food Environment Policy Index (Food-EPI).

BACKGROUND: This work aims to identify policies implemented for healthy food environments in Italy within The Healthy Food Environment Policy Index (Food-EPI) project. METHODS: Food-EPI tool, which includes two components, thirteen domains and fifty good practice indicators, was adapted for the Italian context. Evidence for implementation was gathered and summarized for all fifty indicators from data sources such as governmental websites, non-government organizations publications and websites and via direct contact with Government officials. RESULTS: The highest level of evidence was found within five domains: food composition (2/2 indicators), labelling (3/4), promotion (4/5), provision (4/5) and leadership (5/5). The domains with less identified evidence were food prices (1/4), food in retail (0/4), trade and investment (0/2) and platforms and interaction (1/4). CONCLUSIONS: The evidence summarization and the upcoming stakeholders' meeting to rate the level of implementation for each indicator in Italy have the potential to improve Government commitment to shape healthier food environments.

The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review.

INTRODUCTION: Cost-effectiveness (CEA) and cost-utility analyses (CUA) have become popular types of economic evaluations (EE) used for evidence-based decision-making in healthcare resource allocation. Newborn screening programs (NBS) can have significant clinical benefits for society, and cost-effectiveness analysis may help to select the optimal strategy among different screening programs, including the no-screening option, on different conditions. These economic analyses of NBS, however, are hindered by several methodological challenges. This study explored the methodological quality in recent NBS economic evaluations and analyzed the main challenges and strategies adopted by researchers to deal with them. METHODS: A scoping review was conducted according to PRISMA methodology to identify CEAs and CUAs of NBS. The methodological quality of the retrieved studies was assessed quantitatively using a specific guideline for the quality assessment of NBS economic evaluations, by calculating a general score for each EE. Challenges in the studies were then explored using thematic analysis as a qualitative synthesis approach. RESULTS: Thirty-five studies met the inclusion criteria. The quantitative analysis showed that the methodological quality of NBS economic evaluations was heterogeneous. Lack of clear description of items related to results, discussion, and discounting were the most frequent flaws. Methodological challenges in performing EEs of neonatal screenings include the adoption of a long time horizon, the use of quality-adjusted life years as health outcome measure, and the assessment of costs beyond the screening interventions. CONCLUSIONS: The results of this review can support future economic evaluation research, aiding researchers to develop a methodological guidance to perform EEs aimed at producing solid results to inform decisions for resource allocation in neonatal screening.

Complication Rate of Prepectoral Implant-based Breast Reconstruction Using Human Acellular Dermal Matrices.

Implant-based breast reconstruction (IBR) is currently the most frequently performed reconstructive technique post mastectomy. Even though submuscular IBR continues to be the most commonly used technique, mastectomy technique optimization, the possibility to check skin viability with indocyanine green angiography, the enhanced propensity of patients undergoing prophylactic mastectomies, and the introduction of acellular dermal matrices (ADMs) have paved the way to the rediscovery of the subcutaneous reconstruction technique. The aim of this article is to update the complication rate of immediate and delayed prepectoral IBR using human ADMs (hADMs). METHODS: A literature search, using PubMed, Medline, Cochrane, and Google Scholar database according to Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines, was conducted to evaluate complication rates of prepectoral implant-based reconstructions using hADMs. The following MeSH terms were used: "prepectoral breast reconstruction acellular dermal matrix," "prepectoral breast reconstruction ADM," "human ADM breast reconstruction," and "human acellular dermal matrix breast reconstruction" (period: 2005-2020; the last search took place on April 2, 2020). RESULTS: This meta-analysis includes 1425 patients (2270 breasts) who had undergone immediate or delayed prepectoral IBR using different types of hADMs. The overall complication rate amounted to 19%. The most frequent complication was represented by infection (7.9%), followed by seroma (4.8%), mastectomy flap necrosis (3.4%), and implant loss (2.8%). CONCLUSIONS: The overall complication rate was 19%. The most frequent complications were infection, seroma, and mastectomy flap necrosis, while capsular contracture was rare.

The organizational domain in HTA reports: towards a technology-oriented assessment.

BACKGROUND: Organizational aspects in Health Technology Assessment (HTA) reports play a significant role in managing policies and strategies to implement new health technologies. The organizational domain, however, is often under-represented in HTA reports. This study explored how organizational assessment is carried out in HTA reports and designed a new perspective through an empirical approach after the comparison with EUnetHTA's Core Model 3.0. METHODS: Full HTA reports were extracted from the websites of members of International Network of Agencies for HTA and the aspects relating to the organizational assessment were analysed. HTA reports were classified into 4 typologies of technologies (device, diagnosis/screening, intervention and organizational procedures) and organizational aspects described were explored through a framework of 5 domains and 15 subdomains from Core Model. Specific Multiple Correspondence Analyses were carried out to identify technology-related domains for organizational assessment in reports. RESULTS: The analysis showed that domains and subdomains of Core Model were not covered homogeneously by the organizational assessments in the HTA reports. Through Multiple Correspondence Analyses, four new technology-related dimensions were identified and named according to the Core Model's subdomains in the four new clusters. CONCLUSIONS: This study analyzed the significance of organizational assessments in current literature and the challenges of improving this domain in HTA reports. Through an empirical analysis, we proposed a new methodology to cover the most relevant aspects of organizational appraisal according to new domains to promote support to the assessment of organizational issues in reports and fulfil the needs of future HTA research.

Lean thinking in the hospital setting - Results of a systematic review of literature.

AIM: The aim of this study was to review experiences of use of the Lean methodology in the hospital setting and assess the impact of the interventions in terms of time of processes, productivity, effects on staff and patientd satisfaction. METHODS: PubMed, Scopus and CINAHL databases were searched to identify studies evaluating the lean methodology. Two reviewers screened the citations identified and extracted data according to the PRISMA methodology. RESULTS: In total, 635 citations were identified, of which 27 were included in the present review. Most studies showed a positive outcome related to the implementation of the Lean methodology and tools.

Adjuvanted influenza vaccine for the Italian elderly in the 2018/19 season: an updated health technology assessment.

BACKGROUND: The elderly, defined here as subjects aged ≥ 65 years, are among at-risk subjects for whom annual influenza vaccination is recommended. For the 2018/19 season, three vaccine types are available for the elderly in Italy: trivalent inactivated vaccine (TIV), adjuvanted TIV (aTIV) and quadrivalent inactivated vaccines (QIV). No health technology assessment (HTA) of seasonal influenza vaccination in the elderly has previously been conducted in Italy. METHODS: An HTA was conducted in 2017 to analyze the burden of influenza illness, the characteristics, efficacy, safety and cost-effectiveness of available vaccines and the related organizational and ethical implications. This was then contextualized to the 2018/19 influenza season. Comprehensive literature reviews/analyses were performed and a static mathematical model developed in order to address the above issues. RESULTS: In Italy, influenza is usually less common in the elderly than in other age-classes, but the burden of disease is the highest; >10% of infected elderly subjects develop complications, and about 90% of all influenza-related deaths occur in this age-class. All available vaccines are effective, safe and acceptable from an ethical standpoint. However, aTIV has proved more immunogenic and effective in the elderly. Furthermore, from the third payer's perspective, aTIV is highly cost-effective and cost-saving in comparison with TIV and QIV, respectively. Nevertheless, vaccination coverage needs to be improved. CONCLUSIONS: According to this HTA, aTIV appeared the vaccine of choice in the elderly. HTA should be reapplied whenever new relevant data become available.

Does Clinical Governance influence the quality of medical records?

BACKGROUND: Clinical Governance (CG) is a validated framework for continuous quality improvement in health care settings. Quality medical records may reflect the quality of care delivered and are a viable tool to implement CG skills. AIM: Aim of this study is to investigate the correlation between the level of implementation of CG dimensions and the quality of medical records. MATERIAL AND METHODS: A cross-sectional study was carried out in an Italian Teaching Hospital. CG implementation levels were quantified through a systematic methodology (OPTIGOV©). The overall quality of medical records was measured through a revised version of a National-validated scale. A multiple linear regression model was used to test the likely influence of all the variables constituting the OPTIGOV evaluation on the quality of medical records. 47 hospital wards and 1458 medical records were assessed. RESULTS: A significant and positive association between the quality of medical records and the accountability score (ß = 0.15; p < 0.01) and the clinical audit score (b = 0.11; p = 0.02), was found. Conversely, the risk management score shown a negative and significant correlation (b = -0.17; p < 0.01). This study confirms that CG plays a central role in driving quality improvement and advocates a systematic implementation of such an approach within healthcare organizations.

Using health care audit to improve quality of clinical records: the preliminary experience of an Italian Cancer Institute.

INTRODUCTION: Audit and feedback are recognized as part of a strategy for improving performance and supporting quality and safety in European health care systems. These considerations led the Clinical Management Staff of the "Regina Elena" Italian Cancer Institute to start a project of self-assessment of the quality of clinical records and organizational appropriateness through a retrospective review. MATERIALS AND METHODS: The evaluation about appropriateness and congruity concerned both clinical records of 2013 and of 2015. At the end of the assessment of clinical records of each Care Unit, results were shared with medical staff in scheduled audit meetings. RESULTS: One hundred and thirteen clinical records (19%) did not meet congruity criteria, while 74 (12.6%) resulted as inappropriate. Considering the economic esteem calculated from the difference between Diagnosis Related Groups (DRG) primarily identified as main diagnosis and main surgical intervention or procedure and those modified during the Local Health Unit (LHU) assessment, 2 surgical Care Units produced a high negative difference in terms of economic value with a consequent drop of hospital discharge form (named in Italian "scheda di dimissione ospedaliera", SDO) remuneration, 7 Care Units produced about the same medium difference with almost no change as SDO remuneration, and 2 Care Units had a positive difference with a profit in terms of SDO remuneration. Concerning the quality assessment of clinical records of 2015, the most critical areas were related to medical documents and hospital discharge form compilation. CONCLUSIONS: Our experience showed the effectiveness of clinical audit in assessing the quality of filling in medical records and the appropriateness of hospital admissions and the acceptability of this tool by clinicians.