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Firenze, Italy

Several cases of expensive drugs designed for large patient populations (e.g. sofosbuvir) have raised a complex question in terms of drug pricing. Even assuming value-based pricing, the treatment with these drugs of all eligible patients would have an immense budgetary impact, which is unsustainable also for the richest countries. This raises the need to reduce the prices of these agents in comparison with those suggested by the value-based approach and to devise new pricing methods that can achieve this goal. The present study discusses in detail the following two methods: (i) The approach based on setting nation-wide budget thresholds for individual innovative agents in which a fixed proportion of the historical pharmaceutical expenditure represents the maximum budget attributable to an innovative treatment; (ii) The approach based on nation-wide price–volume agreements in which drug prices are progressively reduced as more patients receive the treatment. The first approach has been developed in the USA by the Institute for Clinical and Economic Review and has been applied to PCSK9 inhibitors (alirocumab and evolocumab). The second approach has been designed for the Italian market and has found a systematic application to manage the price of ranibizumab, sofosbuvir, and PCSK9 inhibitors. While, in the past, price–volume agreements have been applied only on an empirical basis (i.e. in the absence of any quantitative theoretical rule), more recently some explicit mathematical models have been described. The performance of these models is now being evaluated on the basis of the real-world experiences conducted in some European countries, especially Italy. © 2016, Springer International Publishing Switzerland. Source

Facey K.,Evidence Based Health Policy Consultant | Henshall C.,Brunel University | Sampietro-Colom L.,HTA Unit | Thomas S.,University of Southampton
International Journal of Technology Assessment in Health Care

Objectives: Health Technology Assessment (HTA) needs to address the challenges posed by high cost, effective technologies, expedited regulatory approaches, and the opportunities provided by collaborative real-world evaluation of technologies. The Health Technology Assessment International (HTAi) Policy Forum met to consider these issues and the implications for evidence production to inform HTA. This paper shares their discussion to stimulate further debate. Methods: A background paper, presentations, group discussions, and stakeholder role play at the 2015 HTAi Policy Forum meeting informed this paper. Results: HTA has an important role to play in helping improve evidence production and ensuring that the health service is ready to adopt effective technologies. It needs to move from simply informing health system decisions to also working actively to align stakeholder expectations about realistic evidence requirements. Processes to support dialogue over the health technology life cycle need to be developed that are mindful of limited resources, operate across jurisdictions and learn from past processes. Collaborations between health technology developers and health systems in different countries should be encouraged to develop evidence that will inform decision making. New analytical techniques emerging for real-world data should be harnessed to support modeling for HTA. Conclusions: A paradigm shift (to Health Innovation System 2.0) is suggested where HTA adopts a more central, proactive role to support alignment within and amongst stakeholders over the whole life cycle of the technology. This could help ensure that evidence production is better aligned with patient and health system needs and so is more effective and efficient. Copyright © Cambridge University Press 2015 This is an Open Access article, distributed under the terms of the Creative Commons Attribution licence (. Source

Zaccara G.,UO Neurologia | Giovannelli F.,UO Neurologia | Maratea D.,HTA Unit | Fadda V.,HTA Unit | Verrotti A.,University of Chieti Pescara

Purpose Analysis of overall tolerability and neurological adverse effects (AEs) of eslicarbazepine acetate (ESL), lacosamide (LCM) and oxcarbazepine (OXC) from double-blind, placebo-controlled trials. Indirect comparisons of patients withdrawing because of AEs, and the incidence of some vestibulocerebellar AEs between these three antiepileptic dugs (AEDs). Methods We searched MEDLINE for all randomized, double-blind, placebo-controlled trials investigating therapeutic effects of fixed oral doses of ESL, LCM and OXC in patients with drug resistant epilepsy. Withdrawal rate due to AEs, percentages of patients with serious AEs, and the proportion of patients experiencing any neurological AE, nausea and vomiting were assessed for their association with the experimental drug. Analyses were performed between recommended daily doses of each AED according to the approved summary of product characteristics (SPC). Risk differences were used to evaluate the association of any AE [99% confidence intervals (CIs)] or study withdrawals because of AEs (95% CIs) with the experimental drug. Indirect comparisons between withdrawal rate and AEs dizziness, coordination abnormal/ataxia and diplopia were estimated according to network meta-analysis (Net-MA). Results Eight randomized, placebo-controlled, double-blind trials (4 with ESL, 3 with LCM, and 1 with OXC) were included in our analysis. At high doses (OXC 1200 mg, ESL 1200 mg and LCM 400 mg) there was an increased risk of AE-related study withdrawals compared to placebo for all drugs. Several AEs were associated with the experimental drug. Both number and frequency of AEs were dose-related. At high recommended doses, patients treated with OXC withdrew from the experimental treatment significantly more frequently than patients treated with ESL and LCM. Furthermore, the AEs coordination abnormal/ataxia and diplopia were significantly more frequently observed in patients treated with OXC compared to patients treated with LCM and ESL. Conclusions The overall tolerability of AEDs and the incidence of several neurological AEs were clearly dose-dependent. Indirect comparisons between these AEDs, taking into account dose-effect, showed that OXC may be associated with more frequent neurological AEs than LCM and ESL. © 2013 British Epilepsy Association. Source

Messori A.,HTA Unit | Trippoli S.,HTA Unit | Biancari F.,University of Oulu
BMJ Open

Background: Transcatheter aortic valve implants (TAVIs) is indicated as an alternative to surgical valve replacement for patients unfit for surgery. No systematic review has studied survival after 2 years and limited information is available on between-study heterogeneity. Objectives: A systematic review and meta-analysis on intermediate survival after TAVI. Data sources: PubMed, EMBASE, Scopus and references of selected articles. Study eligibility criteria: Clinical studies evaluating TAVI, published between 2010 and 2012, reporting survival at 2 or more years. Participants: About 3500 patients from 14 studies. Study appraisal and synthesis methods: Proportion meta-analysis with 95% CI and heterogeneity assessment (I2 and Cochran's Q). Metaregression analysis was performed as well. Results: Pooled immediate postoperative death rate was 7.8% (95% CI 6.2% to 9.8%, I2=40.8%; Cochran's Q=97.7 with 92.9 df, p<0.0001) and stroke rate was 3.8% (95% CI 2.8% to 5.0%, I2=34.3%; Cochran's Q=96.5 with 92.9 df, p<0.0001). Pooled death rates at 1, 2 and 3 years were 23.2%, 31.0% and 38.6%, respectively. Among studies reporting on concomitant percutaneous coronary intervention, pooled death rates at 30 days, 1 year and 2 years were 6.3%, 17.8% and 25.8%, respectively. Limitations: Although our analysis examined a total of about 3500 patients, only a minority of these were actually followed up after 2 years. Conclusions: Pooled survival rates after TAVI (at 2 years:69.0%; at 3 years:61.4%) can be considered excellent, particularly in the light of the high-risk profile of this patient population. Implications of key findings: The favourable intermediate outcome in patients subjected to TAVI seems to justify its use in patients unfit for surgery. Such pooled results indicate that TAVI is a valid alternative to surgical valve replacement, but lack of data on late durability after TAVI prevents its use in low-risk patients with long expectancy of life. Source

Background and Objectives: We studied the effect of achieving sustained virological response (SVR) on the risk of developing hepatocellular carcinoma (HCC) in patients with hepatitis C receiving anti-hepatitis C virus treatment. Avoiding HCC is considered the main long-term benefit of successful antiviral treatment. Methods: Our literature search extended up to June 2015. We identified all studies that assessed the risk of HCC in patients achieving or not achieving SVR. Meta-analysis was based on a standard random-effect model. The end-point was occurrence of HCC compared between patients with and without SVR; this end-point was expressed as an odds ratio and percent reduction in risk and was also presented separately for patients with and without cirrhosis. All results estimates presented with 95 % confidence intervals (CIs). The presence of any temporal trend in these indexes was investigated by standard meta-regression. Results: Our search identified 25 observational studies (19,822 patients). The odds ratio of HCC for SVR versus no-SVR was 0.19 (95 % CI 0.15–0.24) in the overall series of 25 studies. The difference in this index between patients with any stage of fibrosis/cirrhosis and those with cirrhosis was small. With regard to risk difference, the 25 studies indicated an overall reduction of 10 % (95 % CI 8.00–12.0); this effect was much less pronounced in the group with any stage of fibrosis/cirrhosis (risk difference 6.7 %) than in the selected group with cirrhosis (risk difference 22 %). Meta-regression showed no temporal trend. Conclusion: Our analysis was successful in providing an updated overview on this controversial topic. Some pharmacoeconomic assessments are also presented to interpret the clinical results of our analysis. © 2015, Springer International Publishing Switzerland. Source

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