OP65 Pharmacoeconomic Evaluation Of Orphan Drugs: Impact Of Extra Criteria?

2019 ◽  
Vol 35 (S1) ◽  
pp. 16-16
Author(s):  
Orla Maguire ◽  
Laura McCullagh ◽  
Cara Usher ◽  
Michael Barry
Keyword(s):  
Cost Effectiveness ◽  
Orphan Drug ◽  
Societal Perspective ◽  
Evaluation Process ◽  
Orphan Drugs ◽  
Pharmacoeconomic Evaluation ◽  
Qaly Gain ◽  
Minor Effect ◽  
Orphan Medicinal Products ◽  
A Minor

IntroductionThere is ongoing debate as to whether conventional pharmacoeconomic evaluation (PE) methods are appropriate for orphan medicinal products (OMPs). The National Centre for Pharmacoeconomics (NCPE) in Ireland has a well-defined process for conducting pharmacoeconomic evaluations of pharmaceuticals, which is the same for OMPs and non-OMPs. The objective of this study was to identify whether supplementary criteria considered in the pharmacoeconomic evaluation of OMPs would affect final reimbursement recommendations.MethodsA literature search was conducted to identify criteria. Orphan drug pharmacoeconomic evaluations completed by the NCPE between January 2015 and December 2017 were identified and supplementary criteria, where feasible, were applied.ResultsFourteen pharmacoeconomic evaluations were included in the study. Three criteria that could feasibly be applied to the NCPE evaluation process were identified, all three of which essentially broadened the economic perspective of the pharmacoeconomic evaluation. Higher cost-effectiveness threshold: Despite being arbitrarily raised from EUR 45,000/QALY to EUR 100,000/QALY, only one orphan drug demonstrated cost-effectiveness at this higher threshold. Weighted QALY gain: here, a weighted gain of between one and three is applied to drugs demonstrating QALY gains between 10 and 30, respectively. No OMPs included in the study showed a QALY gain of more than 10. Thirteen demonstrated QALY gains less than 10 and one could not be evaluated. Societal perspective: six submissions incorporated societal perspective as a scenario analysis. Despite incremental cost-effectiveness ratios (ICERs) being reduced between 4 percent and 58 percent, only two OMPs demonstrated cost-effectiveness at the higher threshold (EUR 100,000/QALY).ConclusionsApplication of supplementary criteria to the pharmacoeconomic evaluation of OMPs had a minor effect on three products assessed. However, for the majority, the final cost-effectiveness outcomes remained the same. The study highlights that other criteria are being considered in the decision to reimburse.

scholarly journals European and Italian regulation on orphan medicinal products

2013 ◽  
Vol 14 (2) ◽  
pp. 89-98
Author(s):  
Roberta Joppi
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Potential Benefit ◽  
Orphan Drugs ◽  
Pharmaceutical Companies ◽  
Drug Status ◽  
Medicinal Products ◽  
Eu Legislation ◽  
Orphan Medicinal Products ◽  
The Eu

The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

scholarly journals COST-EFFECTIVENESS OF ORPHAN DRUGS FOR LEUKEMIA TREATMENT: A SYSTEMATIC REVIEW

2021 ◽  
Vol 506 (1-2) ◽  
Author(s):  
Nguyen Do Hong Nhung ◽  
Tran Thi Ngoc Van ◽  
Hoang Thy Nhac Vu
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Orphan Drug ◽  
Financial Burden ◽  
Cost Effective ◽  
Orphan Drugs ◽  
Measurement Unit ◽  
Comparison Results ◽  
Leukemia Treatment ◽  
The Cost

Introduction: Leukemia is a rare disease related to hematologic cancer stemming from the bone marrow. The Vietnam Ministry of Health (MOH) promulgated the Orphan Drugs List, in which there were 37 orphan drugs indicated for leukemia. This study aimed to systematically review all studies on the cost-effectiveness analysis (CEA) of these orphan drugs in leukemia treatment. Materials and methods: This study conducted a systematic review on all studies published till August 2021 on the National Center for Biotechnology Information (NCBI), SpringerLink, and Biomed Central. 23 studies were selected in this systematic review, which were studies that had available full-texts, were written in English, aimed to analyze the cost-effectiveness of leukemia drugs listed on the Vietnam MOH’s Orphan Drugs List. The results were presented by describing CEA findings by five different leukemia types, with a focus on the incremental cost-effectiveness ratio (ICER) of each orphan drug and the comparison to the willingness-to-pay (WTP) threshold. All the cost currency values were converted to USD in 2021 to make comparison. Results: Of 37 drugs on the Vietnam MOH’s orphan drug list, 24 drugs were cost-effectiveness analyzed with available full-texts. Of 23 selected studies in the review, there were 10 studies regarding lymphocytic leukemia (43.5%) and 13 studies regarding myeloid leukemia (56.5%). 60.9% studied on relapsed/refractory patients, 39.1% conducted cost-analyses with a social perspective, and 47.7% used overall survival combined with progression-free survival (OS-PFS) as a clinical endpoint. Quality-adjusted life year (QALY) was used as an effectiveness measurement unit in 65.2% of the total selected studies. 15 studies made a conclusion that their studied orphan drugs were cost-effective. 100% of the studies regarding acute lymphoblastic leukemia showed the orphan drugs of interest were cost-effective (ICER < WTP threshold); while about 50% of the studies regarding other leukemia types showed the orphan drugs of interest were not cost-effective (ICER > WTP threshold) and thus required suitable financial aid. Conclusion: This study provided information on the cost-effectiveness of 24 out of 37 orphan drugs for leukemia treatment listed on the Vietnam MOH's Orphan Drugs List. These orphan drugs could be considered as a financial burden for leukemia patients and other potential payers such as the Vietnam Social Security due to their considerably high cost.

Improvement of monitoring of tertiary filtration with particle counting

2006 ◽  
Vol 6 (1) ◽  
pp. 1-9
Author(s):  
V. Miska ◽  
J.H.J.M. van der Graaf ◽  
J. de Koning
Keyword(s):  
Particle Surface ◽  
Minor Effect ◽  
Particle Size Distributions ◽  
Particle Volume ◽  
Mass Distributions ◽  
Particle Counting ◽  
Total Particle ◽  
Particle Counts ◽  
A Minor ◽  
Sample Dilution

Nowadays filtration processes are still monitored with conventional analyses like turbidity measurements and, in case of flocculation–filtration, with phosphorus analyses. Turbidity measurements have the disadvantage that breakthrough of small flocs cannot be displayed, because of the blindness regarding changes in the mass distributions. Additional particle volume distributions calculated from particle size distributions (PSDs) would provide a better assessment of filtration performance. Lab-scale experiments have been executed on a flocculation–filtration column fed with effluent from WWTP Beverwijk in The Netherlands. Besides particle counting at various sampling points, the effect of sample dilution on the accuracy of PSD measurements has been reflected. It was found that the dilution has a minor effect on PSD of low turbidity samples such as process filtrate. The correlation between total particle counts, total particle volume (TPV) and total particle surface is not high but is at least better for diluted measurements of particles in the range 2–10 μm. Furthermore, possible relations between floc-bound phosphorus and TPV removal had been investigated. A good correlation coefficient is found for TPV removal versus floc-bound phosphorus removal for the experiments with polyaluminiumchloride and the experiments with single denitrifying and blank filtration.

PCV21 Cost-Effectiveness of Atorvastatin Versus Simvastatin in Prevention of Cardiovascular Events from a Thailand Societal Perspective

2021 ◽  
Vol 24 ◽  
pp. S70
Author(s):  
A. Sukonthasarn ◽  
N. Thongtang ◽  
V. Ektare ◽  
F. Du ◽  
G. Brizuela ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Societal Perspective ◽  
Cardiovascular Events

Are Discount Rates Too High? Population Health and Intergenerational Equity

2021 ◽  
Vol 41 (2) ◽  
pp. 245-249
Author(s):  
R. Scott Braithwaite ◽  
Mark S. Roberts
Keyword(s):  
Health Policy ◽  
Cost Effectiveness ◽  
Decision Theory ◽  
Health Outcomes ◽  
Societal Perspective ◽  
Environmental Damage ◽  
Healthy Population ◽  
Discount Rates ◽  
Policy Decisions ◽  
Effectiveness Analysis

Increasing attention is being paid to policy decisions in which shorter-term benefits may be eclipsed by longer-term harms, such as environmental damage. Health policy decisions have largely been spared this scrutiny, even though they too may contribute to longer-term harms. Any healthy population or society must sustain itself through reproduction, and therefore, transgenerational outcomes should be of intrinsic importance from a societal perspective. Yet, the discount rates typically employed in cost-effectiveness analyses have the effect of minimizing the importance of transgenerational health outcomes. We argue that, because cost-effectiveness analysis is based on foundational axioms of decision theory, it should value transgenerational outcomes consistently with those axioms, which require discount rates substantially lower than 3%. We discuss why such lower rates may not violate the Cretin-Keeler paradox.

scholarly journals Linkage mapping identifies a non-synonymous mutation in FLOWERING LOCUS T (FT-B1) increasing spikelet number per spike

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Jonathan Brassac ◽  
Quddoos H. Muqaddasi ◽  
Jörg Plieske ◽  
Martin W. Ganal ◽  
Marion S. Röder
Keyword(s):  
Flowering Time ◽  
Aspartic Acid ◽  
Synonymous Substitution ◽  
Minor Effect ◽  
Phenotypic Variance ◽  
Spikelet Number ◽  
Wheat Varieties ◽  
Trait Locus ◽  
A Minor ◽  
Spikelet Number Per Spike

AbstractTotal spikelet number per spike (TSN) is a major component of spike architecture in wheat (Triticumaestivum L.). A major and consistent quantitative trait locus (QTL) was discovered for TSN in a doubled haploid spring wheat population grown in the field over 4 years. The QTL on chromosome 7B explained up to 20.5% of phenotypic variance. In its physical interval (7B: 6.37–21.67 Mb), the gene FLOWERINGLOCUST (FT-B1) emerged as candidate for the observed effect. In one of the parental lines, FT-B1 carried a non-synonymous substitution on position 19 of the coding sequence. This mutation modifying an aspartic acid (D) into a histidine (H) occurred in a highly conserved position. The mutation was observed with a frequency of ca. 68% in a set of 135 hexaploid wheat varieties and landraces, while it was not found in other plant species. FT-B1 only showed a minor effect on heading and flowering time (FT) which were dominated by a major QTL on chromosome 5A caused by segregation of the vernalization gene VRN-A1. Individuals carrying the FT-B1 allele with amino acid histidine had, on average, a higher number of spikelets (15.1) than individuals with the aspartic acid allele (14.3) independent of their VRN-A1 allele. We show that the effect of TSN is not mainly related to flowering time; however, the duration of pre-anthesis phases may play a major role.

scholarly journals Cost-effectiveness analysis of ranibizumab for retinal vein occlusion patients in China from the societal perspective

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Weiyi Ni ◽  
Jia Liu ◽  
Yawen Jiang ◽  
Jing Wu
Keyword(s):  
Cost Effectiveness ◽  
Retinal Vein Occlusion ◽  
Societal Perspective ◽  
Laser Photocoagulation ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Gdp Per Capita ◽  
Base Case ◽  
Vein Occlusion ◽  
Per Capita

Abstract Background Clinical trials in China have demonstrated that ranibizumab can improve the clinical outcomes of branch retinal vein occlusion (BRVO) and central retinal vein occlusion (CRVO). However, no economic evaluation of ranibizumab has been conducted among Chinese patient population. Methods To provide insights into the economic profile of ranibizumab among Chinese RVO population, a Markov state-transition model was used to predict the outcomes of ranibizumab comparing to laser photocoagulation and observational-only care from the societal perspective. This model simulated changes in patient visuality, quality-adjusted of life years (QALY), medical costs, and direct non-medical costs of individuals with visual impairment due to BRVO or CRVO in lifetime. The base-case analysis used an annual discount rate of 5% for costs and benefits following the China Guidelines for Pharmacoeconomic Evaluations. Deterministic and probabilistic sensitivity analyses were performed to test the robustness of the model. Results The base-case incremental cost-effectiveness ratio (ICER) comparing ranibizumab to laser photocoagulation was ¥65,008/QALY among BRVO patients and was ¥65,815/QALY among CRVO patients, respectively. Comparing to the 2019 gross domestic product (GDP) per capita of ¥71,000, both two ICERs were far below the cost-effective threshold at three times of GDP per capita (¥213,000). The deterministic and probabilistic sensitivity analyses demonstrated the base-case results were robust in most of the simulation scenarios. Conclusion The current Markov model demonstrated that ranibizumab may be cost-effective compared with laser photocoagulation to treat BRVO and cost-effective compared to observation-only care to treat CRVO in China from the societal perspective.

scholarly journals IL-6 Is Not Absolutely Essential for the Development of a TH17 Immune Response after an Aerosol Infection with Mycobacterium tuberculosis H37rv

Cells ◽  
2020 ◽  
Vol 10 (1) ◽  
pp. 9
Author(s):  
Kristina Ritter ◽  
Jan Christian Sodenkamp ◽  
Alexandra Hölscher ◽  
Jochen Behrends ◽  
Christoph Hölscher
Keyword(s):  
Immune Response ◽  
Inflammatory Diseases ◽  
Mycobacterial Infection ◽  
Minor Effect ◽  
Mycobacterium Tuberculosis H37rv ◽  
Th 17 ◽  
Anti Inflammatory ◽  
A Minor ◽  
Aerosol Infection ◽  
Deficient Mice

Anti-inflammatory treatment of chronic inflammatory diseases often increases susceptibility to infectious diseases such as tuberculosis (TB). Since numerous chronic inflammatory and autoimmune diseases are mediated by interleukin (IL)-6-induced T helper (TH) 17 cells, a TH17-directed anti-inflammatory therapy may be preferable to an IL-12-dependent TH1 inhibition in order to avoid reactivation of latent infections. To assess, however, the risk of inhibition of IL-6-dependent TH17-mediated inflammation, we examined the TH17 immune response and the course of experimental TB in IL-6- and T-cell-specific gp130-deficient mice. Our study revealed that the absence of IL-6 or gp130 on T cells has only a minor effect on the development of antigen-specific TH1 and TH17 cells. Importantly, these gene-deficient mice were as capable as wild type mice to control mycobacterial infection. Together, in contrast to its key function for TH17 development in other inflammatory diseases, IL-6 plays an inferior role for the generation of TH17 immune responses during experimental TB.

scholarly journals Sweet taste of heavy water

2021 ◽  
Vol 4 (1) ◽  
Author(s):  
Natalie Ben Abu ◽  
Philip E. Mason ◽  
Hadar Klein ◽  
Nitzan Dubovski ◽  
Yaron Ben Shoshan-Galeczki ◽  
...  
Keyword(s):  
Heavy Water ◽  
Calcium Release ◽  
Homology Modelling ◽  
Taste Receptor ◽  
Chemical Properties ◽  
Sweet Taste ◽  
Minor Effect ◽  
Dynamics Simulations ◽  
A Minor ◽  
Physical And Chemical

AbstractHydrogen to deuterium isotopic substitution has only a minor effect on physical and chemical properties of water and, as such, is not supposed to influence its neutral taste. Here we conclusively demonstrate that humans are, nevertheless, able to distinguish D2O from H2O by taste. Indeed, highly purified heavy water has a distinctly sweeter taste than same-purity normal water and can add to perceived sweetness of sweeteners. In contrast, mice do not prefer D2O over H2O, indicating that they are not likely to perceive heavy water as sweet. HEK 293T cells transfected with the TAS1R2/TAS1R3 heterodimer and chimeric G-proteins are activated by D2O but not by H2O. Lactisole, which is a known sweetness inhibitor acting via the TAS1R3 monomer of the TAS1R2/TAS1R3, suppresses the sweetness of D2O in human sensory tests, as well as the calcium release elicited by D2O in sweet taste receptor-expressing cells. The present multifaceted experimental study, complemented by homology modelling and molecular dynamics simulations, resolves a long-standing controversy about the taste of heavy water, shows that its sweet taste is mediated by the human TAS1R2/TAS1R3 taste receptor, and opens way to future studies of the detailed mechanism of action.

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