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  • 1
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    Online Resource
    Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study
    Frontiers Media SA ; 2022
    In:  Frontiers in Pharmacology Vol. 13 ( 2022-5-16)
    Details
    In: Frontiers in Pharmacology, Frontiers Media SA, Vol. 13 ( 2022-5-16)
    Abstract: Background: Orphan drugs are used for the diagnosis, prevention and treatment of rare diseases that, in the European Union, are defined as disorders affecting no more than 5 persons in 10,000. So far, a total of around 800 orphan medicinal products have been approved by the European Medicines Agency, however the utilization profile of orphan drugs has yet to be explored. This study aimed at assessing the utilization profile of orphan drugs authorized for marketing by the Italian Medicines Agency using population-based data. Methods: A total of 21 orphan drugs used in outpatient settings, approved in the European Union before or during the 2008–2018 period and involving 15 rare diseases, were included in the study. The monitored population included patients with one of the conditions surveilled by the population-based Tuscany Registry of Rare Diseases and diagnosed between 2000–2018. A multi-database approach was applied, by linking data from the registry with information collected in drug prescriptions databases. The prevalence and intensity of use were estimated for the selected orphan drugs and other non-orphan medications, used to treat the same rare disease and for which a change in the prevalence of use was hypothesized after authorization of the orphan drug. Results: For some diseases (acquired aplastic anemia, tuberous sclerosis complex, most metabolic diseases) a low prevalence of orphan drugs use was observed (range between 1.1–12.5%). Conversely, orphan drugs were frequently used in hemophilia B, Wilson disease and idiopathic pulmonary fibrosis (maximum of 78.3, 47.6 and 41.8%, respectively). For hemophilia B and Leber’s hereditary optic neuropathy, there are currently no other medications used in clinical practice in addition to orphan drugs. Six orphan drugs were used for the treatment of pulmonary arterial hypertension, appearing the elective therapy for this disease, albeit with different utilization profiles (range of prevalence 1.7–55.6%). Conclusion: To the best of our knowledge, this is the first study investigating the utilization profile of orphan drugs prescribed in a defined geographical area, and providing relevant information to monitor over time potential changes in the prevalence of these medications as well as in the health care decision making.
    Type of Medium: Online Resource
    ISSN: 1663-9812
    URL: Article
    DOI: 10.3389/fphar.2022.869842
    Language: Unknown
    Publisher: Frontiers Media SA
    Publication Date: 2022
    detail.hit.zdb_id: 2587355-6
    SSG: 15,3
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  • 2
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    Sustained Response to Interferon–Ribavirin Combination Therapy Predicted by a Model of Hepatitis C Virus Dynamics Using Both Hcv Rna and Alanine Aminotransferase
    SAGE Publications ; 2003
    In:  Antiviral Therapy Vol. 8, No. 6 ( 2003-08), p. 519-530
    Details
    In: Antiviral Therapy, SAGE Publications, Vol. 8, No. 6 ( 2003-08), p. 519-530
    Abstract: Standard models simulate the dynamics of hepatitis C virus (HCV) infection using HCV RNA kinetics and show a correlation between the clearance of infected hepatocytes and response to interferon. However, they are unable to predict whether the response will be maintained. The aim of our work was to identify criteria by which sustained responses may be predicted and defined. Methods In our model the clearance rate of infected cells is a function of their number and the baseline rate is computed by the alanine aminotransferase (ALT) kinetics during the first month of therapy. We simulated the variations of viral and infected cell loads in 31 consecutive patients treated with IFN-α2b 3–5 MU every other day, with or without ribavirin, for 6 or 12 months depending on HCV genotype. Results At baseline the computed (in 28 of 31 cases) percentage of infected cells was lower in seven sustained responders (mean: 11.7%, range: 1–24.6%) than in 14 transient responders (mean: 28.2%, range: 7.4–75.5%) and in seven non-responders (mean: 41%, range: 8.8–86%) ( P=0.036). At the end of therapy the computed infected cell number was 〈 100 cells/ml of extracellular fluid in all sustained responders (mean: 18.4, range: 1.7–48), in three transient responders (mean: 3500, range: 1.52–17500) and in none non-responders (mean: 28500, range: 1200–96000) ( P=0.003). Overall of 10 patients with less than 100 infected cells/ml at the end of therapy seven (six had combination therapy) showed sustained response. All three relapsers received interferon monotherapy. Conclusion The analysis of infected cells dynamics using the new model might be useful to tailor treatment duration in patients with combination therapy.
    Type of Medium: Online Resource
    ISSN: 1359-6535 , 2040-2058
    URL: Article
    DOI: 10.1177/135965350300800602
    Language: English
    Publisher: SAGE Publications
    Publication Date: 2003
    detail.hit.zdb_id: 2118396-X
    SSG: 15,3
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  • 3
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    Economic evaluation of the chronic hepatitis B treatment strategies in Italy
    Seed SRL ; 2013
    In:  Farmeconomia. Health economics and therapeutic pathways Vol. 14, No. 3 ( 2013-09-10), p. 111-118
    Details
    In: Farmeconomia. Health economics and therapeutic pathways, Seed SRL, Vol. 14, No. 3 ( 2013-09-10), p. 111-118
    Abstract: BACKGROUND AND OBJECTIVE: Pharmacological approaches available in chronic hepatitis B (CHB) are based on 48-weeks finite course of peg-interferon (PEG-IFN) or continuous administration of nucleoside analogues. Recent studies gave way to early identification of non-responders to peg-interferon with a stopping rule based on virologic/serologic markers at week 12. A pharmacoeconomic simulation model was developed to support the economic evaluation of HBeAg-negative CHB treatment strategies, which either involve first line peg-interferon with stopping rule and switch to current most effective analogue entecavir or tenofovir, or nucleosides analogues in first line treatment. This model showed a good cost-effectiveness profile of the first line treatment with peg-interferon. Aim of the present study was the estimation of the impact of a wide adoption of a first line with PEG-IFN and the stopping rule on total overall cost related to CHB over the lifetime of patients in Italy.METHODS: The Markov model was developed to perform a lifetime simulation of the disease progression considering the following health-related states: active CHB, virologic response, HBsAg clearance, compensated cirrhosis with active CHB, compensated cirrhosis with virologic response, decompensated cirrhosis, hepatocellular carcinoma, liver transplant, post-liver transplant and death. The outcomes provided by the model were average survival, quality-adjusted life years (QALY) and costs, calculated from the Italian National Health Service (SSN) perspective. A “current” mix of treatments, where 80% patients received ETV or TDF in first-line, was compared with an “hypothetical” mix defined by a 100% adoption of a PEG-IFN first-line with the stopping rule, to estimate the impact on total lifetime CHB-related costs for a cohort of 100 HBeAg-negative CHB patients in Italy.RESULTS: Results obtained from the evaluation of overall total treatment cost over patients’ lifetime appeared to confirm the cost-effectiveness of peg-interferon previously assessed. Lifetime overall total cost resulted significantly lower with the “hypothetical” treatment mix as compared to the “current” mix.. In fact, estimated lifetime overall total costs for the former and latter scenario were equal to €7,239,050 and €9,060,150 respectively, thus generating a saving of approximately 20% associated to CHB treatment with first-line peg-interferon.CONCLUSION: Based on the results obtained from the economic evaluation of the CHB treatment strategies, first-line peg-interferon associated with the stopping rule and switch to nucleoside analogues consistently represents a convenient strategy on both the clinical and economic perspective.
    Type of Medium: Online Resource
    ISSN: 2240-256X
    URL: Article
    DOI: 10.7175/fe.v14i3.665
    Language: Unknown
    Publisher: Seed SRL
    Publication Date: 2013
    detail.hit.zdb_id: 2715873-1
    SSG: 15,3
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  • 4
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    Online Resource
    Individualized Treatment of Hbeag-Negative Chronic Hepatitis B Using Pegylated Interferon-α2A as First-Line and Week-12 HBV Dna/Hbsag Stopping Rule: A Cost-Effectiveness Analysis
    SAGE Publications ; 2013
    In:  Antiviral Therapy Vol. 18, No. 4 ( 2013-05), p. 1-11
    Details
    In: Antiviral Therapy, SAGE Publications, Vol. 18, No. 4 ( 2013-05), p. 1-11
    Abstract: Hepatitis B e antigen (HBeAg)-negative chronic hepatitis B (CHB) is the most frequent and difficult-to-treat viral hepatitis worldwide. HBV DNA and hepatitis B surface antigen (HBsAg) serum levels, which help the early identification of non-responders to pegylated interferon (PEG-IFN), prompt more flexible individualized therapeutic strategies exploiting the benefits of both PEG-IFN and nucleoside/nucleotide analogues (NAs). We assessed the cost-effectiveness of week-12 HBV DNA/ HBsAg stopping rule for early interruption and switch to currently most effective NA treatments (entecavir [ETV] or tenofovir disoproxil fumarate [TDF] ). Methods A decision-analytic Markov model was developed in the following health-related states: CHB, compensated cirrhosis (CC) and decompensated cirrhosis, hepatocellular carcinoma, liver transplant, post-liver transplant, death, virological response, relapse and HBsAg clearance. Simulated strategies included: ETV/TDF in CHB; ETV/TDF delayed until CC; first-line PEG-IFN followed by switch to ETV/TDF for either patients meeting the week-12 stopping rule or week-48 null-responders/relapsers; and first-line PEG-IFN followed by switch to ETV/TDF delayed until CC. ETV and TDF were considered alternatively for a total of eight strategies. A lifetime simulation horizon was applied. Results Early treatment strategies using NAs with or without first-line PEG-IFN provided the highest results (approximately 22 life-years and 15 quality-adjusted life years [QALYs]). Delayed treatments until cirrhosis development resulted in poorer outcomes. The average per-patient lifetime costs ranged from €33,500 (TDF in CC) to €68,900 (TDF in CHB). Costs using ETV were 20–50% higher. First-line PEG-IFN strategies ranged from dominant (that is, more effective and less costly) to highly cost-effective, although differences in QALYs were always very narrow. Conclusions The cost-effectiveness of antiviral therapy of HBeAg-negative CHB could be improved significantly using first-line PEG-IFN followed by a switch to NAs in either patients meeting the week-12 HBV DNA/HBsAg stopping rule or week-48 non-responders/relapsers.
    Type of Medium: Online Resource
    ISSN: 1359-6535 , 2040-2058
    URL: Article
    DOI: 10.3851/IMP2555
    Language: English
    Publisher: SAGE Publications
    Publication Date: 2013
    detail.hit.zdb_id: 2118396-X
    SSG: 15,3
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  • 5
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    Liver Stiffness, a Non-Invasive Marker of Liver Disease: A Core Study Group Report
    SAGE Publications ; 2010
    In:  Antiviral Therapy Vol. 15, No. 3_suppl ( 2010-04), p. 69-78
    Details
    In: Antiviral Therapy, SAGE Publications, Vol. 15, No. 3_suppl ( 2010-04), p. 69-78
    Abstract: The ability to evaluate liver stiffness non-invasively in clinical practice by measuring transient elastography using FibroScan® has resulted in considerable interest and enthusiasm. A core study group, organized by the Italian Association for the Study of the Liver, has assessed the usefulness of FibroScan® in the diagnosis and management of liver disease in clinical practice. The group concluded that FibroScan® is a valuable, non-invasive technique and have developed a consensus report form for registering transient elastography results. In this article, we report the findings of the study group.
    Type of Medium: Online Resource
    ISSN: 1359-6535 , 2040-2058
    URL: Article
    DOI: 10.3851/IMP1626
    Language: English
    Publisher: SAGE Publications
    Publication Date: 2010
    detail.hit.zdb_id: 2118396-X
    SSG: 15,3
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  • 6
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    Online Resource
    Do the circulating Pre‐S/S quasispecies influence hepatitis B virus surface antigen levels in the HBeAg negative phase of HBV infection?
    Wiley ; 2020
    In:  Alimentary Pharmacology & Therapeutics Vol. 51, No. 12 ( 2020-06), p. 1406-1416
    Details
    In: Alimentary Pharmacology & Therapeutics, Wiley, Vol. 51, No. 12 ( 2020-06), p. 1406-1416
    Abstract: Virus, host factors and their interplay influence Hepatitis B surface Antigen serum levels during Hepatitis B Virus (HBV) infection course and treatment. Aim To study the Pre‐S/S circulating quasispecies in a cohort of untreated, HBeAg negative, genotype‐D, HBsAg carriers. Methods We studied 260 carriers: 71 with HBeAg negative infection (ENI; HBV‐DNA ≤2000 IU/mL); 42 Grey Zone (GZ; HBV‐DNA ≤20 000 IU/mL); 82 chronic hepatitis (CH) and 65 cirrhosis (CI) (HBV‐DNA  〉  20 000 IU/mL). Population sequencing was applied to identify Pre‐S/S gene mutations responsible for any amino acid substitution or potential biological/antigenic implications (M‐muts) on HBsAg. Results HBsAg serum levels were lower in ENI + GZ than in CH + CI (2.61 [−1.10/4.06] vs 3.62 [2.41/4.92] log 10 IU/mL, P   〈  0.001) and in CI than CH (3.48 [2.41/4.38] vs 3.66 [2.57/4.92] log 10 IU/mL, P   〈  0.001). M‐muts were found in 73 (28.1%) cases: 5 (7.0%) ENI, 3 (7.1%) GZ, 26 (31.7%) CH, 39 (60.0%) CI ( P   〈  0.001) and mostly in Pre‐S2 (17.6%) than Pre‐S1 (5.8%) and Small‐S (10.8%; P   〈  0.001). Overall HBsAg serum levels were higher in carriers with M‐muts (3.56 [0.95/4.38] vs 3.17 [−1.10/4.92] log 10 IU/mL, P   〈  0.001), but comparable in carriers with or without M‐mut when considering separately ENI + GZ (2.84 [0.95/3.89] vs 2.61 [−1.10/4.06] log 10 IU/mL, P  = 0.330] and CH + CI (3.57 [2.67/4.38] vs 3.63 [2.41/4.92] log 10 IU/mL, P  = 0.37). Infection phase ( β : 0.422, P   〈  0.001), age ( β : −0.260, P   〈  0.001), ALT ( β : −0.103, P  = 0.045), liver stiffness ( β : −0.118, P  = 0.039) and HBV‐DNA ( β : 0.384, P   〈  0.001), but not M‐mut were independently associated with HBsAg serum levels. Conclusions In HBeAg negative, genotype‐D, carriers Pre‐S/S heterogeneity increases with severity of liver disease, but does not influence HBsAg serum levels, that in low viraemic carriers are associated with an effective control of HBV.
    Type of Medium: Online Resource
    ISSN: 0269-2813 , 1365-2036
    URL: Issue
    URL: Article
    DOI: 10.1111/apt.v51.12
    DOI: 10.1111/apt.15753
    Language: English
    Publisher: Wiley
    Publication Date: 2020
    detail.hit.zdb_id: 2003094-0
    SSG: 15,3
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  • 7
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    Budget impact of an individualized treatment for HBeAg-negative chronic hepatitis B in Italy with peg-interferon alpha-2a combined to 12 weeks’ stopping-rule
    Springer Science and Business Media LLC ; 2013
    In:  PharmacoEconomics Italian Research Articles Vol. 15, No. 3 ( 2013-12), p. 123-130
    Details
    In: PharmacoEconomics Italian Research Articles, Springer Science and Business Media LLC, Vol. 15, No. 3 ( 2013-12), p. 123-130
    Type of Medium: Online Resource
    ISSN: 1590-9158 , 2035-6137
    URL: Article
    DOI: 10.1007/s40276-013-0015-1
    Language: Italian
    Publisher: Springer Science and Business Media LLC
    Publication Date: 2013
    SSG: 15,3
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