1,721,131 research outputs found
Endpoint surrogati e politiche sanitarie: l’importanza di metodi di validazione adeguati
No full textPolitiche sanitarie e decisioni di accesso al mercato o rimborso di tecnologie sanitarie dovrebbero idealmente basarsi su studi clinici robusti che valutano outcome rilevanti per i pazienti, come la sopravvivenza o la qualità della vita correlata alla salute. La spinta crescente a ridurre i tempi di accesso a trattamenti innovativi per i pazienti ha promosso negli ultimi anni diverse tendenze in questo senso, inclusa la diffusione di endpoint surrogati su cui basare decisioni regolatorie o di rimborsabilità.1 La definizione comunemente accettata di endpoint surrogato è “biomarcatore o esito intermedio usato per sostituire e predire un esito rilevante per il paziente”. Questi ultimi sono identificati come esiti che riflettono direttamente sopravvivenza, sintomi o funzionalità.
Value attribution for combination treatments: two potential solutions for an insoluble problem
No full textValue Attribution for Combination Treatments: Two Potential Solutions for an Insoluble Proble
Urinary Tract Infections – An Economic Issue
No full textIt is important that the change of approach to prophylactic treatment is supported by economic data and also that quantitative economic data are essential to back healthcare budget allocation applications to regional policy makers. In the absence of European economic data on UTIs, data from the US suggest a US1 billion) and the rest attributed to direct costs (healthcare services). The distress experienced by patients with recurrent UTIs has been estimated to be similar to that caused by more recognisable conditions that are also way better funded and supported, such as Alzheimer’s and dementia
Valorizzare gli esiti riferiti dal paziente : opportunità e sfide
No full textDopo avere sostenuto insieme a FADOI e Fondazione IRCCS Policlinico San Matteo di Pavia nel
2020 la redazione del Libro Bianco sul conflitto di interessi, e nel 2021 quello sulla ricerca biomedica e il rapporto pubblico-privato, nel 2022 la Fondazione Roche ha deciso di approfondire la
tematica dei dati personali e del loro utilizzo nel sistema sanitario.
Si tratta di un tema di grande interesse, che si pone in sostanziale continuità con gli argomenti
trattati nelle precedenti edizioni. Infatti, i dati rappresentano evidentemente una risorsa di valore
inestimabile per la ricerca e per la migliore cura dei pazienti, ma è altrettanto palese che è necessario che vi sia un contesto, normativo e fattuale, all’interno del quale sia realmente possibile valorizzarli in tutta la loro potenzialità, salvaguardando, al contempo, il rispetto dei diritti della persona.
In questa prospettiva è dunque indispensabile perseguire e realizzare un corretto equilibrio tra
interessi e diritti diversi, individuando la loro possibile conciliazione non in termini astratti, ma sulla
base delle esperienze concrete. Seguendo questa traiettoria argomentativa, la pubblicazione si
struttura quindi su aree tematiche differenti che, grazie all’intervento di autorevoli esperti, mirano
a esplorare i molteplici profili della questione, da quelli giuridici, nazionali ed europei, a quelli etici,
sino a soffermarsi su alcuni modelli operativi che hanno dato prova di efficacia nell’utilizzo dei dati
a favore dei cittadini e dei ricercatori. Pertanto, nei vari interventi si è cercato di evitare le prevedibili lamentazioni sulle rigidità burocratiche e interpretative, che pure in talune circostanze ostacolano l’efficienza operativa, per indirizzarsi invece ad analizzare soprattutto le potenzialità del dato
nel sistema sanitario, a vantaggio di tutti gli interessi coinvolti, che vanno dal rafforzamento del
Servizio Sanitario Italiano (SSN), alla migliore tutela dei pazienti, sino al beneficio per l’impresa
Validation of surrogate end points for overall survival in advanced colorectal cancer: a harmonized approach is needed
No full textA letter invited from the Editors as a response to a comment on a previous article published by us in the same journal
An economic perspective on urinary tract infection: the "costs of resignation"
No full textBackground: Urinary tract infection (UTI) is a leading cause of morbidity in the female population, with high levels of prevalence and recurrence within 6 months.
Objectives: Our objective was to estimate annual costs and health resource utilization by women with UTIs, and the impact on quality of life (QoL) due to lower urinary tract symptoms (LUTS), vaginal pain and dyspareunia. The secondary objective was to estimate the cost per episode of cystitis in a subgroup of patients with recurrent UTIs.
Design and participants: We conducted a retrospective analysis of medical records of a cohort of 309 female patients with a history of UTI. Annual costs and costs per episode were estimated as a composite of costs per diagnosis, costs per pharmacologic therapy and costs per physician visit, according to the Italian NHS perspective.
Results: The mean number of episodes of cystitis per patient was 4.5 and the median time from the onset of symptoms was 7 years (interquartile range (IQR): 3-11). Recurrent UTIs were diagnosed in 122 (39%) patients. Overall, 243 (78%) patients had an impaired sexual life and 51 (17%) had interrupted sexual lives as a result of UTIs. Patients’ median rates of discomfort due to LUTS or dyspareunia on a VAS scale of 0-10 were 8 (IQR 7-10) and 8 (IQR 8-10), respectively. Annual patient direct costs due to UTI were €229 (median €107, IQR €53─241), estimated cost per episode in patients with UTI was €236, whilst in women with recurrent UTI the cost per episode was €142 (p˂0.000).
Conclusion: Annual costs and impact on quality of life caused by UTIs are not negligible. Persistence of the disease and the difference in costs per episode in recurrent UTI vs. UTI patients, which we have called “costs of resignation”, suggest that appropriate early evaluation and effective treatment measures for the disease are still difficult in clinical practice
Valutare l’impatto dell’HTA come strumento di governo dell’innovazione tecnologica: modelli teorici e studi empirici
No full textIn this article we present the results of a literature review aimed at investigating critical issues and major challenges while evaluating HTA’s impact. Our search in Embase identified ten studies, classified as theoretical or empirical, that we analyzed in detail synthetizing the most important elements. HTA’s
impact may be influenced by a variety of factors that should be considered when seeking appropriate methods for impact assessment. HTA’s impact evaluation may represent the trigger for future HTA diffusion and dissemination and for the implementation of its outputs and recommendations
The role of health technology assessment bodies in shaping drug development
No full textThe use of health technology assessment (HTA) to inform policy-making is established in most developed countries. Compared to licensing agencies, HTA agencies have different interests and, therefore, different evidence requirements. Criteria for coverage or reimbursement decisions on pharmaceutical compounds vary; however, it is common to include, as part of the HTA, a comparative effectiveness evaluation. This type of clinical data might go beyond that required for market authorization, thus creating an additional evidence gap between the regulatory and the reimbursement submission. The relevance of submissions to HTA agencies is consistently increasing in a pharmaceutical company's perspective, as market prospects are strongly influenced by third-party payers' coverage. In this study, we aim to describe current HTA activities with a potential impact throughout the drug development process of pharmaceuticals, with a comparative emphasis on the systems in place in Italy and in the UK. Based on an extensive literature and website review, we identified three major classes of HTA activities, beyond mainstream HTA, with the potential to influence the drug development program: 1) horizon scanning and early HTA; 2) bipartite and tripartite early dialogue between manufacturers, regulators, and HTA assessors; and 3) managed market entry agreements. From early stages of clinical research up to postauthorization studies, there is a trend toward increased collaboration between parties, anticipation of market access evidence collection, and postmarketing risk-sharing. Heterogeneity of HTA practices increases the complexity of the market access environment. Overall, there are signals that market access departments are gaining importance in the pharmaceutical companies, but there is still a lack of evidence and reporting on how the increasing relevance of HTA has reshaped the way clinical development is designed and managed
Hospital-based HTA: quale valore per la valutazione delle tecnologie nelle Aziende Sanitarie italiane?
No full textThe early development of Italian Health Technology Assessment (HTA) is largely based on experimental approaches to the hospital-based technology assessment, which are argued to be the most important peculiarity of the country and still the real driver of the HTA movement. HTA in hospitals can serve as a tool for aiding administrative procedures or increasing dialogue and transparency as well as supporting the bottom-up awareness generation around HTA principles. The objectives of this study are to present the results of a survey of HTA activities at the hospital level in Italy and to assess the relationship between the implementation of HTA principles and variables, namely consumption and expenditure for orthopaedic prostheses, which have managerial implications for healthcare providers. Our results are based on 38 hospitals located in 13 Italian Regions of which 53% have a commission working on MDs assessment. Nineteen hospitals declare a link exists between procurement and HTA activities. Our findings suggest that HTA could potentially impact on health technologies’ uptake and expenditure when realized in coordination with other hospital units, thus becoming a useful tool for budget control and planning to local healthcare managers
The importance of using the appropriate model for systematic reviews and meta-analyses-reply
No full textNonfatal Myocardial Infarction—Poor Surrogate for Mortalit
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