89 research outputs found

    A Guide to Selecting Flexible Survival Models to Inform Economic Evaluations of Cancer Immunotherapies

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    Objectives: Parametric models are routinely used to estimate the benefit of cancer drugs beyond trial follow-up. The advent of immune checkpoint inhibitors has challenged this paradigm, and emerging evidence suggests that more flexible survival models, which can better capture the shapes of complex hazard functions, might be needed for these interventions. Nevertheless, there is a need for an algorithm to help analysts decide whether flexible models are required and, if so, which should be chosen for testing. This position article has been produced to bridge this gap. Methods: A virtual advisory board comprising 7 international experts with in-depth knowledge of survival analysis and health technology assessment was held in summer 2021. The experts discussed 24 questions across 6 topics: the current survival model selection procedure, data maturity, heterogeneity of treatment effect, cure and mortality, external evidence, and additions to existing guidelines. Their responses culminated in an algorithm to inform selection of flexible survival models. Results: The algorithm consists of 8 steps and 4 questions. Key elements include the systematic identification of relevant external data, using clinical expert input at multiple points in the selection process, considering the future and the observed hazard functions, assessing the potential for long-term survivorship, and presenting results from all plausible models. Conclusions: This algorithm provides a systematic, evidence-based approach to justify the selection of survival extrapolation models for cancer immunotherapies. If followed, it should reduce the risk of selecting inappropriate models, partially addressing a key area of uncertainty in the economic evaluation of these agents

    Projecting Pharmaceutical Expenditure in EU5 to 2021: Adjusting for the Impact of Discounts and Rebates

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    Within (European) healthcare systems, the main goal for pharmaceutical expenditure is cost containment. This is due to a general belief among healthcare policy makers that pharmaceutical expenditure—driven by high prices—will be unsustainable unless further reforms are enacted.The aim of the research published in this paper is to provide more realistic expectations of pharmaceutical expenditure for all key stakeholder groups by estimating pharmaceutical expenditure at ‘net’ prices. We also aim to estimate any gaps developing between list and net pharmaceutical expenditure for the EU5 countries (i.e. France, Germany, Italy, Spain, and the UK). We adjusted an established forecast of pharmaceutical expenditure for the EU5 countries, from 2017 to 2021, by reflecting discounts and rebates not previously considered, i.e. we moved from ‘list’ to ‘net’ prices, as far as data were available.We found an increasing divergence between expenditure measured at list and net prices. When the forecasts for the five countries were aggregated, the EU5 (unweighted) average historical growth (2010–2016) rate fell from 3.4% compound annual growth rate at list to 2.5% at net. For the forecast, the net growth rate was estimated at 1.5 versus 2.9% at list.Our results suggest that future growth in pharmaceutical expenditure in Europe is likely to be (1) lower than previously understood from forecasts based on list prices and (2) below predicted healthcare expenditure growth in Europe and in line with long-term economic growth rates. For policy makers concerned about the sustainability of pharmaceutical expenditure, this study may provide some comfort, in that the perceived problem is not as large as expected

    Le système des affections de longue durée (nécessité de santé publique ou mauvaise allocation des ressources ?)

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    Les affections de longue durée (ALD) représentent 15,5% de la population des assurés sociaux et 63% des dépenses de l assurance maladie. Ce système particulier de prise en charge à 100% des soins a été créé en même temps que la sécurité sociale en 1945. Il est composé d une liste, les ALD30, complété par les ALD n31 et 32, qui regroupe plus de 400 pathologies. L assurance maladie assume un déficit colossal depuis de nombreuses années et les ALD sont responsables de 90% de l augmentation annuelle des dépenses de santé. Ce système qui selon les projections ne devrait cesser de grossir dans les années à venir peut-il être pérenne dans sa forme actuelle ? Est-il possible de l améliorer pour le conserver ou alors faut-il commencer à envisager une refonte complète du système de prise en charge des assurés sociaux ?CHATENAY M.-PARIS 11-BU Pharma. (920192101) / SudocSudocFranceF

    Optimisation de la prise en charge globale des patients atteints d'un cancer differencié de la thyroïde

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    LE KREMLIN-B.- PARIS 11-BU Méd (940432101) / SudocSudocFranceF

    Reactivity at low temperature of carbon suboxide (C3O2) with amorphous surface of HCl hydrates monitored by FTIR spectroscopy

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    International audienceAn experimental study, monitored by FTIR spectroscopy, of the spontaneous reaction of C3O2 with HCI on the acid hydrate surface has been performed at low temperature. The first appearing reaction product at 85 K was assigned to chloroformylketene while the second, appearing at 145 K, to malonyl dichloride by comparison with previously reported data in matrix isolation spectroscopy. C3O2 was deposited at 80 K over an amorphous surface of HCl hydrates mainly formed from ionic species (H3O+(H2O)(n-1)Cl-; n = 1-3, 4, 6), prepared by codeposition of a H2O:HCl gaseous mixture at 80 K. The reactions were promoted by warming the substrate from 80 to 200 K. Experiments showed that monohydrate H3O+Cl- was more reactive than higher hydrates by easier C3O2 protonation. Reactions are described by an ionic mechanism catalyzed by ice water and occurring on the surface. Spectral evidence of HCl amorphous hydrates, chloroformylketene, and malonyl dichloride are reported
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