A systematic literature review of health state utility values in head and neck cancer.

BACKGROUND: Health state utility values (HSUVs) are essential parameters in model-based economic evaluations. This study systematically identifies HSUVs in head and neck cancer and provides guidance for selecting them from a growing body of health-related quality of life studies. METHODS: We systematically reviewed the published literature by searching PubMed, EMBASE and The Cochrane Library using a pre-defined combination of keywords. The Tufts Cost-Effectiveness Analysis Registry and the School of Health and Related Research Health Utilities Database (ScHARRHUD) specifically containing health utilities were also queried, in addition to the Health Economics Research Centre database of mapping studies. Studies were considered for inclusion if reporting original HSUVs assessed using established techniques. The characteristics of each study including country, design, sample size, cancer subsite addressed and demographics of responders were summarized narratively using a data extraction form. Quality scoring and critical appraisal of the included studies were performed based on published recommendations. RESULTS: Of a total 1048 records identified by the search, 28 studies qualified for data extraction and 346 unique HSUVs were retrieved from them. HSUVs were estimated using direct methods (e.g. standard gamble; n = 10 studies), multi-attribute utility instruments (MAUIs; n = 13) and mapping techniques (n = 3); two studies adopted both direct and indirect approaches. Within the MAUIs, the EuroQol 5-dimension questionnaire (EQ-5D) was the most frequently used (n = 11), followed by the Health Utility Index Mark 3 (HUI3; n = 2), the 15D (n = 2) and the Short Form-Six Dimension (SF-6D; n = 1). Different methods and types of responders (i.e. patients, healthy subjects, clinical experts) influenced the magnitude of HSUVs for comparable health states. Only one mapping study developed an original algorithm using head and neck cancer data. The identified studies were considered of intermediate quality. DISCUSSION: This review provides a dataset of HSUVs systematically retrieved from published studies in head and neck cancer. There is currently a lack of research for some disease phases including recurrent and metastatic cancer, and treatment-related complications. In selecting HSUVs for cost-effectiveness modeling purposes, preference should be given to EQ-5D utility values; however, mapping to EQ-5D is a potentially valuable technique that should be further developed in this cancer population

The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review

Background Rare diseases negatively impact patients' quality of life, but the estimation of health state utility values (HSUVs) in research studies and cost-utility models for health technology assessment is challenging. Objectives This study compared the methods for estimating the HSUVs included in manufacturers' submissions of orphan drugs to the National Institute for Health and Care Excellence (NICE) with those of published studies addressing the same rare diseases to understand whether manufacturers fully exploited the existing literature in developing their economic models. Methods All NICE Technology Appraisal (TA) and Highly Specialized Technologies (HST) guidance documents of non-cancer European Medicines Agency (EMA) orphan medicinal products were reviewed and compared with any published primary studies, retrieved via PubMed until November 2020, and estimating HSUVs for the same conditions addressed in manufacturers' submissions. Results We identified 22 NICE TA/HST appraisal reports addressing 19 different rare diseases. Sixteen reports presented original HSUVs estimated using EQ-5D or Health Utility Index (n = 12), direct methods (n = 2) or mapping (n = 2), while the other six included values obtained from the literature only. In parallel, we identified 111 published studies: 86.6% used preference-based measures (mainly EQ-5D, 60.7%), 12.5% direct techniques, and 2.7% mapping. The collection of values from non-patient populations (using 'vignettes') was more frequent in manufacturers' submissions than in the literature (22.7% vs. 8.0%). Conclusions The agreement on methodological choices between manufacturers' submissions and published literature was only partial. More efforts should be made by manufacturers to accurately reflect the academic literature and its methodological recommendations in orphan drugs submissions

Mapping health-related quality of life scores from FACT-G, FAACT, and FACIT-F onto preference-based EQ-5D-5L utilities in non-small cell lung cancer cachexia.

BACKGROUND: Health-related quality of life (HRQoL) measurements from disease-specific tools cannot be directly used in economic evaluations. This study aimed to develop and validate mapping algorithms that predicted EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) utilities from Functional Assessment of Anorexia-Cachexia Therapy (FAACT) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and their common component (Functional Assessment of Cancer Therapy-General-FACT-G) in patients with non-small cell lung cancer cachexia. METHODS: Data were collected on five occasions over a 12-week period in two multicenter placebo-controlled trials. EQ-5D-5L utilities were calculated using both English and Dutch value sets. The study sample was divided into development and validation datasets according to patients' geographical residence. Generalized estimating equations were applied to five different sets of independent variables including overall, Trial Outcome Index (TOI), and individual subscales results. The best performing models were selected based on mean absolute error (MAE) and root-mean square error (RMSE). RESULTS: EQ-5D-5L and FAACT/FACIT-F results were available for 96 patients. The developed algorithms showed a good predictive performance, with acceptable MAE/RMSE and small differences between mean observed and predicted EQ-5D-5L utilities. In FACT-G models, Physical Well-Being had the highest explanatory value, while Emotional Well-Being did not significantly affect the EQ-5D-5L score; Anorexia-Cachexia and Fatigue subscales were highly statistically significant in FAACT and FACIT-F models, respectively, as well as the TOI scores. The Eastern Cooperative Oncology Group status was included as covariate in all models. CONCLUSION: The developed algorithms enable the estimation of EQ-5D-5L utilities from three cancer-specific instruments when preference-based HRQoL data are missing

The assessment of patient-reported outcomes for the authorisation of medicines in Europe: a review of European Public Assessment Reports from 2017 to 2022

Objectives: Health regulators have progressively increased their attention and focus on patient-reported outcomes (PROs), driven by the diffusion of a patient-centred approach to the drug development process. This study investigates the consideration of PROs and their measures (PROMs) in the authorisation of medicines in Europe. Methods: All medicines for human use authorised or refused by the European Medicines Agency (EMA) in the period 2017-2022 were identified, and corresponding European Public Assessment Reports (EPARs) were downloaded for review. Medicine and PROs/PROM characteristics were systematically recorded. A multivariate logistic regression was performed to identify variables associated with the use of patient-reported evidence in EPARs. Results: Overall, 497 EPARs of authorised medicines and 19 EPARs of refused medicines were analysed; of these, 240 (48.3%) and 10 (52.6%), respectively, reported any use of PROs/PROMs (p = 0.710). For authorised medicines, the likelihood of using PROs/PROMs was negatively affected by generic (OR = 0.01, p < 0.001) and biosimilar status (OR = 0.46, p = 0.013) and positively affected by orphan status (OR = 1.41, p = 0.177). The use of PROMs (50.6% in 2017 vs 47.9% in 2022) did not show a clear pattern over the 6-year period considered (p = 0.758) and was particularly uncommon in some therapeutic areas (e.g., 15.2% in infectious diseases). A total of 816 dyads of PROs/PROMs were identified. On average each EPAR considered 1.6 (range: 0-14) instruments. Patient-reported outcomes were typically secondary (53.3%) and exploratory endpoints (18.8%); in one-third of cases (32.5%), they assessed generic quality of life. Among the PROMs, 227 (27.8%) targeted general population; EQ-5D (11.0%), SF-36/SF-12 (5.9%) and EORTC QLQ-C30 (5.6%) were the instruments most frequently used. Conclusions: This study suggests PROs/PROMs are considered in less than half of total medicine assessments and even more rarely in some disease areas. The adoption of PROs is key in EMA strategy to 2025 and would be facilitated by consensus development on their measures and optimisation of data collection

Gli ospedali di comunità in Italia: passato, presente e futuro

Il presente capitolo approfondisce il tema degli ospedali di comunità (OdC) attraverso un’analisi approfondita dell’esistente, concentrandosi prima sul contesto internazionale per poi analizzare quello nazionale, con una mappatura aggiornata degli OdC presenti nel nostro Paese e la presentazione di otto casi studio, condotti in sei regioni differenti

Gli Ospedali di Comunità: quali prospettive alla luce del PNRR?

L’obiettivo del presente capitolo è quello di approfondire ulteriormente l’analisi rispetto a quanto emerso nel precedente capitolo sugli OdC pubblicato nel Rapporto OASI 2021 (ibid.), indagando l’evoluzione e le progettualità previste nel breve e medio periodo per queste strutture alla luce delle indicazioni di sviluppo contenute nel PNRR. In particolare, vengono analizzate le priorità strategiche, le vocazioni produttive e i modelli di servizio, il target di utenza e la tipologia di bisogni da soddisfare, la tipologia di personale clinico e amministrativo coinvolto, la dotazione tecnologica e i modelli di integrazione e interconnessione con gli altri servizi e setting di cura e assistenza, sia territoriali che ospedalieri

EQ-5D-5L population norms for Italy

This study aimed to provide normative data obtained in response to the EQ-5D-5L questionnaire in Italy and compare this with data from other countries

Consideration of quality of life in the health technology assessments of rare disease treatments

Objectives Challenges with patient-reported outcome (PRO) evidence and health state utility values (HSUVs) in rare diseases exist due to small, heterogeneous populations, lack of disease knowledge and early onset. To better incorporate quality of life (QoL) into Health Technology Assessment, a clearer understanding of these challenges is needed. Methods NICE appraisals of non-oncology treatments with an EMA orphan designation (n=24), and corresponding appraisals in the Netherlands, France, and Germany were included. Document analysis of appraisal reports investigated how PROs/ HSUVs infuenced decision-making and was representative of QoL impact of condition and treatment. Results PRO evidence was not included in 6/24 NICE appraisals. When included, it either failed to demonstrate change, capture domains important for patients, or was uncertain. In the other countries, little information was reported and evidence largely did not demonstrate change. In NICE appraisals, HSUVs were derived through the collection of EQ-5D data (7/24 cases), mapping (6/24), vignettes (5/24), and published literature or other techniques (6/24). The majority did not use data collected alongside clinical trials. Few measures demonstrated signifcant change due to lack of sensitivity or face validity, short-term data, or implausible health states. In 8/24 NICE appraisals, patient surveys or input during appraisal committee meetings supported the interpretation of uncertainty or provided evidence about QoL. Conclusions This study sheds light on the nature of PRO evidence in rare diseases and associated challenges. Results emphasise the need for improved development and use of PRO/HSUVs. Other forms of evidence and expert input are crucial to support better appraisal of uncertain or missing evidenc

The use of intensive radiological assessments in routine surveillance after treatment for head and neck cancer: An economic evaluation.

BACKGROUND: There is uncertainty around the optimal surveillance of head and neck cancer patients after the primary curative treatment. This study aims at assessing the cost-effectiveness of a post-treatment programme of frequent radiological assessments (maximal approach) compared with a symptom-driven surveillance (minimal approach). MATERIALS AND METHODS: A decision-analytic Markov model is developed to assess the cost utility of two alternative follow-up programmes with a lifetime horizon. The two interventions differ in the number of radiological assessments (i.e. magnetic resonance imaging, computed tomography and positron-emission tomography) performed over a 5-year period. Clinical and utility parameters are derived from published and unpublished literature and expert opinion. The cost analysis is conducted from the perspective of a major Italian region's health care system. Cost-effectiveness results are expressed as incremental cost per life year gained (LYG) and per quality-adjusted life year (QALY) and checked against a cost-effectiveness threshold of €25,000-40,000 per QALY. One-way, two-way and probabilistic sensitivity analyses are carried out. RESULTS: In the base-case analysis, an intensive programme of radiological investigations leads to 0.10 additional QALYs (0.15 LYG) and an increase in costs of €1903 per patient compared with those of a minimal option, resulting in an incremental cost of €19,951/QALY gained (€13,123/LYG). In probabilistic sensitivity analysis, 72% of the results lie below the €40,000 threshold (55% below €25,000). CONCLUSIONS: An intensive post-treatment follow-up with scheduled radiological assessments over time might be cost-effective compared with symptom-driven surveillance in head and neck cancer patients. Further research is needed to check these results in empirical studies or real-world settings

Eliciting Preferences for Clinical Follow-Up in Patients with Head and Neck Cancer Using Best-Worst Scaling.

OBJECTIVES: There are no commonly accepted standards for monitoring patients treated for head and neck cancer. The aim of this study was to assess patients' preferences for different aspects of follow-up. METHODS: A best-worst survey was conducted in a sample of head and neck cancer patients in clinical follow-up at the National Cancer Institute (Milan, Italy). Conditional logit regression with choice as the dependent variable was run to analyse the data. A covariate-adjusted analysis was performed in order to identify socio-demographic and clinical factors related to the selection of best-worst items. The participants were asked to report any difficulties encountered during the survey. RESULTS: A total of 143 patients, predominantly male (74%) and with a mean age of 58 years were enrolled in the survey. The strongest positive preference was expressed for a hospital-based program of physical examinations with frequency decreasing over time. Conversely, the lowest valued item was not performing any positron emission tomography (PET) scan during follow-up. Patients with high educational levels were more likely to value attending a primary care-based program and undergoing intensive radiological investigations. Other patient-specific variables significantly associated with the choice of items were employment and living status, time already spent in follow-up and number of treatments received. CONCLUSIONS: Overall, patients were more likely to choose an intensive follow-up scheme broadly consistent with the program currently administered by the hospital. There is little evidence of preference heterogeneity that might justify customized programs based on demographics. The best-worst scaling task appeared feasible for most participants