Global & Regional Health Technology Assessment最新文献

{"title":"Time to market access in Italia: durata del processo di P&R per i farmaci per le malattie rare.","authors":"Andrea Marcellusi, Paola Raimondo, Carlotta Galeone, Pier Luigi Canonico","doi":"10.33393/grhta.2023.2610","DOIUrl":"10.33393/grhta.2023.2610","url":null,"abstract":"Time to market access in Italy: duration of the P&R process for rare disease drugs Objective: This paper aims to investigate the duration of the pricing & reimbursement (P&R) procedures submitted in Italy by pharmaceutical marketing authorization holders (MAH) for drugs indicated for rare diseases. Methods: All the data used in this analysis were publicly available on different sources of the Italian Ministry of Health, the Italian Medicines Agency (AIFA) and other official websites. The information was systematically collected to investigate the timeline (days) needed to complete the P&R process. The process was divided into 6 simplified steps and the median and range of days needed for each phase were estimated based on data reported in official/published documents. The analysis was stratified considering every single step of the assessment phase and included segmentation of drugs into indications for rare diseases, Orphan designation, Innovation assessment and Managed entry agreements (MEAs). Results: Overall, 181 first indication procedures were submitted to AIFA in the period considered and, of these, 167 (92.3%) were completed and 129 procedures were considered for the final analysis and the median duration of the entire process (MAH submission to final Gazette publication) was 434 days (range 176.0-918.0). The duration of procedures for rare diseases (n = 53) was longer than those for non-rare-disease procedures (n = 76) (463.0 days vs 407.5 days respectively). Among rare disease procedures, orphan designation and MEAs represent predictors for time prolongation while innovation is associated with a shorter assessment time. Conclusion: The study describes the time spent in each phase of the assessment and the appraisal process and demonstrates that uncertainty represents the main driver for the increment in the overall time.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"79-88"},"PeriodicalIF":0.5,"publicationDate":"2023-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10628501/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71524075","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Il bisogno insoddisfatto: definizioni, evidenze di letteratura e implicazioni per l’HTA.","authors":"Claudio Jommi, Michela Meregaglia, Carmine Pinto","doi":"10.33393/grhta.2023.2624","DOIUrl":"10.33393/grhta.2023.2624","url":null,"abstract":"Unmet need: definitions, literature evidence and implications for HTA The present paper illustrates the definition of unmet need provided by the peer-reviewed literature and the Health Technology Assessment (HTA) authorities across Europe in the assessment and appraisal process and within the early access schemes for medicines. The analysis relied on a descriptive review of the peer-reviewed literature and HTA documents on the definition of need (disease severity) and the way it is satisfied (existence and validity of alternatives). HTA agencies were found using (i) a narrow definition of need, focused on the clinical impact and the impact on health-related quality of life of the disease and (ii) a broad definition of comparators, including treatments used off-label in the clinical practice. Most of the contributions of the literature advocated for a broader definition of need, including additional dimensions (for example, the socio-economic impact of the disease) and the effects of existing treatments beyond their risk-benefit profile (for example, acceptability to patients). On the one hand, these contributions should be considered by HTA agencies, considering its multi-disciplinary and multi-stakeholder nature. On the other one, the explicit inclusion of the unmet need domains, at present disregarded, should depend on the decisions taken on the ground of the assessment.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"70-78"},"PeriodicalIF":0.5,"publicationDate":"2023-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/96/f6/grhta-10-70.PMC10551671.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41139058","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness analysis of abemaciclib with endocrine therapy (ET) versus ET alone for HR+, HER2-, node-positive, high-risk early breast cancer in Italy.","authors":"Alison Davie,&nbsp;Sory Traoré,&nbsp;Massimo Giovannitti,&nbsp;Giuseppe Pompilio,&nbsp;Mark Lambton,&nbsp;Esra Cakar,&nbsp;Anuja Chatterjee","doi":"10.33393/grhta.2023.2561","DOIUrl":"10.33393/grhta.2023.2561","url":null,"abstract":"<p><strong>Background: </strong>Abemaciclib was recently approved by the European Medicines Agency in combination with adjuvant endocrine therapy (ET) for adult patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), node-positive early breast cancer (EBC) at high risk of recurrence.</p><p><strong>Objective: </strong>To evaluate the cost-effectiveness of abemaciclib plus ET vs. ET alone in patients with HR+, HER2-, node-positive EBC at high risk of disease recurrence, from the Italian healthcare system perspective.</p><p><strong>Methods: </strong>A cohort state transition model was developed with five states: invasive disease-free survival (IDFS), nonmetastatic recurrence, remission, metastatic recurrence, and death. The analysis had a time horizon of 30 years. Individual patient-level data from the monarchE trial (NCT03155997) were used to generate IDFS estimates. Resource use included drug acquisition/administration, best supportive care, terminal care, adverse events, hospitalization, post-progression therapy, and associated resource use in the metastatic disease health state. Health state utilities were derived from monarchE patient-level data and other sources, applying Italian tariffs where feasible.</p><p><strong>Results: </strong>The estimated total discounted costs (€39,249 vs. €16,806; difference: €22,443) and quality-adjusted life years (QALYs) (11.49 vs. 10.50; difference: 0.99) were higher for abemaciclib plus ET compared with ET alone. The incremental cost-effectiveness ratio was €22,651 per QALY gained. The likelihood of abemaciclib plus ET being cost-effective vs. ET alone was 99% at a willingness-to-pay threshold of €30,000 per QALY gained.</p><p><strong>Conclusion: </strong>Abemaciclib plus ET is a cost-effective treatment option vs. ET alone for those with HR+, HER2- node-positive EBC at high risk of recurrence in Italy.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"62-69"},"PeriodicalIF":0.5,"publicationDate":"2023-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/22/ef/grhta-10-62.PMC10551672.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41101679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"COVID-19 impact on the decision process of the Italian Medicine Agency: a quantitative assessment.","authors":"Beatrice Canali, Laura Candelora, Francesca Fiorentino, Tom Halmos, Paola La Malfa, Francesca Massara, Chiara Vassallo, Duccio Urbinati","doi":"10.33393/grhta.2023.2560","DOIUrl":"10.33393/grhta.2023.2560","url":null,"abstract":"<p><strong>Background: </strong>Since the COVID-19 pandemic has placed more attention on drugs' approval process and the importance of rapid decision-making in the healthcare sector, it is crucial to assess how time to market (TTM) of drugs varied.</p><p><strong>Objective: </strong>To estimate the impact of the COVID-19 pandemic on TTM of drugs in Italy.</p><p><strong>Methods: </strong>An IQVIA database was used to retrieve information on drugs that obtained positive opinion from the Committee for Medicinal Products for Human Use between January 2015 and December 2021. The available observations were divided into three groups (Pre COVID, Partially COVID, and Fully COVID) according to the timing of their negotiation process. Differences in average TTM among the three groups were analyzed in three steps: (1) descriptive statistics; (2) univariate analysis; (3) multivariate analysis, using a matching estimator.</p><p><strong>Results: </strong>A total of 363 unique combinations of molecule and indication met the inclusion criteria: 174 in the Pre COVID group, 69 in the Partially COVID group, and 123 in the Fully COVID group. Descriptive statistics and univariate analysis found a statistically significant difference in TTM among the three periods, with average TTM increasing during the pandemic (+136 days, p = 0.00) and then decreasing afterward (-23 days, p = 0.09). In the matching analysis, results for the Partially COVID period were confirmed (+108 days, p = 0.00) while results for the Fully COVID period lost significance but maintained a negative sign.</p><p><strong>Conclusions: </strong>The results suggest that after an adjustment phase in the Partially COVID period, a return to the <i>status quo</i> was reached.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"46-52"},"PeriodicalIF":0.4,"publicationDate":"2023-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0c/76/grhta-10-46.PMC10238912.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9939860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Buone pratiche per lo sviluppo dei modelli di budget impact a livello regionale.","authors":"Andrea Marcellusi,&nbsp;Angela Ragonese,&nbsp;Andrea Marinozzi,&nbsp;Alberto Bortolami,&nbsp;Sara Mucherino,&nbsp;Carolina Moreno,&nbsp;Amalia Antenori,&nbsp;Matteo Ferrario,&nbsp;Claudia Simonelli,&nbsp;Matteo Zanuzzi,&nbsp;Marco Cicoira,&nbsp;Ruggero Lasala,&nbsp;Francesco Russoniello,&nbsp;Francesco Attanasio,&nbsp;Caterina Donati,&nbsp;Chiara Roni,&nbsp;Fabrizio Gemmi,&nbsp;Francesco Saverio Mennini,&nbsp;Pierluigi Russo,&nbsp;Giovanna Scroccaro,&nbsp;Pier Luigi Canonico","doi":"10.33393/grhta.2023.2582","DOIUrl":"https://doi.org/10.33393/grhta.2023.2582","url":null,"abstract":"Good practices for the development of budget impact models at regional level Introduction: The present work aims to discuss the current scenario of procedures and regulations regarding budget impact analysis/models (BIA/BIM) at regional level in Italy and to provide a standardized approach and detailed recommendations for developing these analyses. Method: A systematic review of the literature was conducted in order to collect existing guidelines or specific regional procedures for budget impact analysis in Italy. All the records were analysed in qualitative terms according to a pre-specified analytical framework, based on the ISPOR BIA guidelines. At the end of the analysis, a consensus questionnaire was developed to establish agreed approaches and to provide possible solutions to any critical issues. A list of 39 statements was developed. The survey was distributed to 69 experts who rated their level of agreement with each statement on a 5-point Likert scale. Consensus was predefined as more than 66% of the panel agreeing/disagreeing with any given statement. Results: Sisty-nine experts answered the questionnaire; a total of 30/39 statements achieved consensus. There was agreement on most of the statements. Time horizon to consider and costs were the issues on which no agreement was found. The results allowed the working group to define a list of good practices. Conclusion: While the structure and development of BIM are now well-known and well-applied at national level, there remains a great diversity of management of BIM tools at regional level. Consensus was reached among participating experts, as to the main characteristics, determinants and features of regional BIA/BIM in the perspective of the Italian payer.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"53-61"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e8/b0/grhta-10-53.PMC10318586.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10180422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Costo-efficacia di IMI/CIL/REL rispetto a CMS+IMI per il trattamento di infezioni batteriche Gram-negative non sensibili ai carbapenemi.","authors":"Martina Paoletti,&nbsp;Andrea Marcellusi,&nbsp;Joe Yang,&nbsp;Francesco Saverio Mennini","doi":"10.33393/grhta.2023.2488","DOIUrl":"https://doi.org/10.33393/grhta.2023.2488","url":null,"abstract":"Cost-effectiveness of IMI/CIL/REL for the treatment of carbapenem non-susceptible Gram-negative bacterial infections Objective: The objective of this analysis was to evaluate the cost-effectiveness of imipenem/cilastatin/relebactam compared to colistin-imipenem in the treatment of hospitalized patients with Gram-negative bacterial infections caused by imipenem-resistant pathogens. The perspective was both that of the National Health Service (NHS) and the social one. Methodology: A mixed model was developed to simulate a cohort of patients capable of highlighting the impacts of the disease on the quality of life and the absorption of economic resources of the patients in analysis. Modelled patients were those with hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia (HABP/VABP), complicated intra-abdominal infection (cIAI) or complicated urinal tract infection (cUTI) caused by carbapenem-resistant Gram-negative (GN) pathogens. The model begins with a short-term decision tree describing possible treatment routes and outcomes for patients during the hospitalization period. Patients who are healed in the decision tree enter the long-term Markov model, designed to capture the follow-up costs and health-related quality of life (HRQL) of patients healed over their lifetime. Results: The analysis, conducted on a hypothetical cohort of 1,000 patients, highlights how the use of imipenem/cilastatin/relebactam is advantageous both in terms of diagnosis and treatment in the short term and in terms of cost-effectiveness. In fact, it is dominant compared to colistin-imipenem both in the NHS and in the social perspective since, compared to an average saving of € 2,800.15 and € 3,174.63 respectively, it would generate an increase of 4.76 years of life and of 4.12 QALYs per patient.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"18-28"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0e/55/grhta-10-18.PMC10105322.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9317188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prezzo e rimborso dei farmaci in caso di estensione delle indicazioni: i risultati di una survey sui soci di ISPOR Italy Rome Chapter.","authors":"Eugenio Di Brino,&nbsp;Claudio Jommi","doi":"10.33393/grhta.2023.2562","DOIUrl":"https://doi.org/10.33393/grhta.2023.2562","url":null,"abstract":"Price and reimbursement of medicines when new indications are approved: the results of a survey on ISPOR Italy Rome Chapter members Multi-indication pricing models for medicines and some international impact evidence are available in the literature. Data on the Italian context are more limited. This paper illustrates the results of a study aimed at gathering the opinion on this topic of experts, members of the ISPOR Italy Rome Chapter. The opinion was collected through a structured questionnaire, validated by two potential responders, and administered online in the period October/July 2022. There were 45 responders (20% of the members); 67% of responders work in pharmaceutical companies and 13% in consultancy firms. The remainder belongs to regulators/payers and universities. The survey highlighted a preference for (i) non-automatic models, as automatic approaches are mainly based on price cuts/discount increases in relation to an increase in volumes, (ii) an “indication-based-pricing” model (where prices are differentiated by indication through discounts/risk sharing agreements), since it is more consistent with a value-based approach, even if more complex to manage, (iii) a mix of discounts/agreements according to existing evidence. The opinion collected is consistent with the opinions available in the literature, but not consistent with the Italian trend, where, compared to the past, a blended approach is prevailing. A blended pricing envisages a renegotiation of the single price for all indications, essentially based on a change in the discount. Our hope is that in the future the experts’ opinion will be taken into consideration and that a targeted indication-based-pricing will be adopted again.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"40-45"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/af/b5/grhta-10-40.PMC10158496.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9434963","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analisi di impatto sul budget sanitario italiano di enzalutamide per il trattamento del carcinoma prostatico metastatico ormono-sensibile.","authors":"Francesca Fiorentino,&nbsp;Paolo Di Rienzo","doi":"10.33393/grhta.2023.2507","DOIUrl":"https://doi.org/10.33393/grhta.2023.2507","url":null,"abstract":"ABSTRACT Budget impact analysis of enzalutamide for the treatment of hormone-sensitive metastatic prostate cancer in Italy Backgroud: After demonstrating additional benefit versus standard of care in ARCHES and ENZAMET studies, enzalutamide was reimbursed in Italy in May 2022 by the National Health Service (NHS) for the treatment of hormone-sensitive metastatic prostate cancer (mHSPC). Objective: This study estimates the financial impact associated to the introduction of enzalutamide for patients with mHSPC. Methods: A budget impact model was developed with a dynamic cost calculator approach, in which the impact on subsequent lines of therapy was considered. The analysis considered the NHS perspective and a 3-year time horizon. Included costs were related to drug acquisition and administration, monitoring, patient follow-up and adverse events. Eligible population was estimated based on published literature, real-world data and experts’ opinion while market shares were assessed considering real-world data and assumptions. National tariffs and published literature were considered for unit costs. Results: Eligible population was estimated at 6,200, 6,206 and 6,212 in years 1, 2 and 3 respectively. The introduction of enzalutamide, considering a progressive increase of market shares of 10%, 18% and 25%, is expected to overall increase NHS healthcare expenditure by € 688 thousands and € 2.6 and € 5.6 million in years 1, 2 and 3 respectively, corresponding on average to 1.55% of the overall prostate cancer expenditure. Results are robust across one-way sensitivity analyses, while confidential discount agreements of on-patent drugs might significantly impact the estimates. Conclusion: The introduction of enzalutamide for the treatment of adult patients with mHSPC is expected to increase patients’ health with a moderate impact on costs for the Italian NHS.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"29-39"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d7/c2/grhta-10-29.PMC10105321.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9317186","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-minimization analysis to support the HTA of Radiofrequency Echographic Multi Spectrometry (REMS) in the diagnosis of osteoporosis.","authors":"Ludovica Borsoi,&nbsp;Patrizio Armeni,&nbsp;Maria Luisa Brandi","doi":"10.33393/grhta.2023.2492","DOIUrl":"https://doi.org/10.33393/grhta.2023.2492","url":null,"abstract":"<p><strong>Introduction: </strong>A timely diagnosis of osteoporosis is key to reducing its growing clinical and economic burden. Radiofrequency Echographic Multi Spectrometry (REMS), a new diagnostic technology using an ultrasound approach, has been recognized by scientific associations as a facilitator of patients' care pathway. We aimed at evaluating the costs of REMS vs. the conventional ionizing technology (dual-energy X-ray absorptiometry, DXA) for the diagnosis of osteoporosis from the perspective of the Italian National Health Service (NHS) using a cost-minimization analysis (CMA).</p><p><strong>Methods: </strong>We carried out structured qualitative interviews and a structured expert elicitation exercise to estimate healthcare resource consumption with a purposeful sample of clinical experts. For the elicitation exercise, an Excel tool was developed and, for each parameter, experts were asked to provide the lowest, highest and most likely value. Estimates provided by experts were averaged with equal weights. Unit costs were retrieved using different public sources.</p><p><strong>Results: </strong>Considering the base-case scenario (most likely value), the cost of professionals amounts to €31.9 for REMS and €48.8 for DXA, the cost of instrumental examinations and laboratory tests to €45.1 for REMS and €68.2 for DXA. Overall, in terms of current costs, REMS is associated with a mean saving for the NHS of €40.0 (range: €27.6-71.5) for each patient.</p><p><strong>Conclusions: </strong>REMS is associated with lower direct healthcare costs with respect to DXA. These results may inform policy-makers on the value of the REMS technology in the earlier diagnosis for osteoporosis, and support their decision regarding the reimbursement and diffusion of the technology in the Italian NHS.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"1-11"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/11/a2/grhta-10-1.PMC9923405.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10744081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Il ruolo degli esiti riferiti dal paziente nelle decisioni di rimborso e innovatività dei farmaci in Italia.","authors":"Francesco Malandrini,&nbsp;Cesare Borroni,&nbsp;Michela Meregaglia,&nbsp;Massimiliano Sarra,&nbsp;Oriana Ciani","doi":"10.33393/grhta.2023.2514","DOIUrl":"https://doi.org/10.33393/grhta.2023.2514","url":null,"abstract":"The role of patient-reported outcomes in reimbursement decisions and drug innovation in Italy The attention paid by regulatory authorities to patient-reported outcomes (PROs) and their related measures (PROMs) has progressively increased to allow for a more complete description of the patient’s health status. This work aimed at investigating the use of PROs/PROMs in relation to reimbursement agreement and innovativeness recognition by the Italian Medicines Agency (AIFA). The list of European Public Assessment Reports (EPARs), downloaded from the European Medicine Agency (EMA) website, was analyzed to identify drugs authorized between 2017 and 2021. The associations between the use of PROs/PROMs in the EPARs and the reimbursement and innovativeness status in Italy were explored through Chi2 test and logistic regression. In addition, each innovativeness report was analyzed to identify any further evidence about the consideration of PROs/PROM in the decision-making process. Of the 403 EPARs analyzed, 197 (48.9%) reported the use of PROs/PROMs. A subsample of 229 drugs with a reimbursement class (A, H, or C) officially assigned by AIFA was further investigated. Of them, 49.3% reported the use of PROs/PROMs. The Chi2 test revealed a statistically significant association between the use of PROs/PROMs and innovativeness (p = 0.001) and orphan status (p = 0.012). In the logistic regression, orphan status and availability of PROs/PROMs are significant predictors of the innovativeness status. However, of the 46 innovative drugs, only 9 (19.6%) explicitly reported a consideration of a PRO/PROM in their innovativeness reports. Robust instrument’s validation, reliable data collection and growing potential’s awareness are keys to increase the consideration of PROs at every step of the drug development process.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"12-17"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/37/79/grhta-10-12.PMC9923404.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10744083","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}