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Julia Thornton Snider Jesse Sussell Mahlet Gizaw Tebeka Alicia Gonzalez Joshua T. Cohen Peter Neumann 《Value in health》2019,22(3):332-339
Background
Payers frequently rely on budget impact model (BIM) results to help determine drug coverage policy and its effect on their bottom line. It is unclear whether BIMs typically overestimate or underestimate real-world budget impact.Objective
We examined how different modeling assumptions influenced the results of 6 BIMs from the Institute for Clinical and Economic Review (ICER).Study Design
Retrospective analysis of pharmaceutical sales data.Methods
From ICER reports issued before 2016, we collected estimates of 3 BIM outputs: aggregate therapy cost (ie, cost to treat the patient population with a particular therapy), therapy uptake, and price. We compared these against real-world estimates that we generated using drug sales data. We considered 2 classes of BIM estimates: those forecasting future uptake of new agents, which assumed “unmanaged uptake,” and those describing the contemporaneous market state (ie, estimates of current, managed uptake and budget impact for compounds already on the market).Results
Differences between ICER's estimates and our own were largest for forecasted studies. Here, ICER's uptake estimates exceeded real-world estimates by factors ranging from 7.4 (sacubitril/valsartan) to 54 (hepatitis C treatments). The “unmanaged uptake” assumption (removed from ICER's approach in 2017) yields large deviations between BIM estimates and real-world consumption. Nevertheless, in some cases, ICER's BIMs that relied on current market estimates also deviated substantially from real-world sales data.Conclusions
This study highlights challenges with forecasting budget impact. In particular, assumptions about uptake and data source selection can greatly influence the accuracy of results. 相似文献5.
Carlos Ferrer Albiach Antonio Conde Moreno Marta Rodríguez Cordón Virginia Morillo Macías Ana Bouché Babiloni Inmaculada Beato Tortajada Ángel Sánchez Iglesias Alicia Francés Muñoz 《Clinical & translational oncology》2010,12(1):22-26
Hypoxia is related to poor prognosis because it is associated to chemo-and radioresistance. During recent years the evolution
of imaging methods like PET/CT and MRI has meant the appearance of new perspectives with direct implications in radiation
therapy. We discuss previous experiences in staging, planning and in the follow-up process with these techniques for measuring
tumour hypoxia. 相似文献
6.
Alberto Castilla M.D. Joan Camps-Bansell M.D. Maria-Pilar Civeira M.D. Jesús Prieto M.D. 《The American journal of gastroenterology》1993,88(2):233-239
Forty patients with chronic hepatitis C (CHC) were included in an open randomized controlled trial of lymphoblastoid α-interferon (L-IFN) versus no treatment. Twenty patients entered each group, and features of therapy and control cases were similar. L-IFN was given in low doses (1.5-4.5 megaunits) for 1 yr. In 16 of 20 patients treated with L-IFN (80%), but in only one of 20 nontreated cases (0.5%; p < 0.001), aminotransferase activities became normal. In four patients there was a reactivation of the disease during treatment after 4, 5, 6, and 8 months with normal aminotransferase levels. A posttherapy reactivation of hepatitis was observed in four additional cases after 1, 1, 1, and 3 months of follow-up. The other eight patients (40%) continued with normal aminotransferase levels for 1.52 ± 0.74 (range, 1-2.1) yr after IFN doses were discontinued. In all treated patients except two nonresponders, but in only one of 20 nontreated cases ( p < 0.001), Knodell's histological activity index decreased. Procollagen type III aminoterminal peptide levels did not change significantly in nontreated and nonresponder patients, diminished slightly in patients with a transient response, and normalized in cases with a long-standing response, suggesting that this serum test may be a reliable marker for monitoring response to IFIN therapy in patients with CHC. Finally, L-IFN treatment induced significant increments in CD4/CD8 index, phytohemagglutinin-induced blastogenesis, and natural killer activity. This study shows that L-IFN diminish inflammatory and fibrogenic activity in most patients with CHC. In 40% of patients treated in this trial, a long-standing remission of the disease was observed. 相似文献
7.
Eduardo E. Castilla María Luísa Martínez-Frías 《American journal of medical genetics. Part A》1995,58(2):106-112
Congenital “healed” cleft lip (CHCL) is an unusual anomaly including a paramedian “scar” of the upper lip, which appears as if a typical cleft lip has been corrected in utero. The CHCL is frequently associated with an ipsilateral notch in the vermilion, and “collapsed” nostril. Twenty-five CHCL cases are presented, eighteen of which were an isolated malformation found among the 3,950,715 births examined in two similar birth defect registries: ECEMC in Spain and ECLAMC in Latin America. Like open cleft lip, of which it seems to be a variant, CHCL is most frequently seen among males (14/18 isolated cases), it preferentially affects the left side (10/18 cases), and it segregated together with cleft lip in one family. The five CHCL cases with other congenital anomalies included: two cases with hydrocephalus, two VACTERL associations, and one atypical oblique facial cleft infant with single umbilical artery. CHCL may result from a defective fusion of the frontonasal and maxillary processes (before week 7 of embryonic life), or from a spontaneously repaired open cleft lip, later on. In either way, these cases heal with a visible scar, and the pre-occurrence of CHCL in two families suggests a familial predisposition to this phenomenon. © 1995 Wiley-Liss, Inc. 相似文献
8.
Alicia E. Ronco M. Cecilia Sobrero Gustavo D. Bulus Rossini Pablo R. Alzuet Bernard J. Dutka 《Environmental toxicology》1995,10(1):35-39
Toxicity testing of sediment samples from the Rio Santiago and its main tributaries was performed using a battery of tests that included the ECHA dipstick biocide monitor, the MetPad test, the lettuce seed germination and the root elongation test, the nematode test, spot plate test, and the SOS Chromotest. Assessment was carried out on organic extracts, pore water, and direct sediment. Results of bioassays showed toxicity sources from tributaries and canals flowing into the river and areas being degraded by insults from industrial spills. © by John Wiley & Sons, Inc. 相似文献
9.
Abstract Pericardial tamponade remains a diagnostic challenge
to the clinician especially when the patient is well compensated
hemodynamically. We report an unusual case
who sought medical help 1 month after having been
stabbed in his chest. An investigation revealed a perforation
of the myocardium and a pericardial tamponade.
The patient survived thanks to a large organized clot
that plugged the perforation. The patient was exposed
to increased risk due to delayed onset, recognition, and
therapy of the tamponade. Most reports on this subject
deal with acute pericardial tamponade. Only few cases
of delayed pericardial tamponade have been reported.
A review of the relevant literature and the therapeutic
approaches are discussed. 相似文献
10.
Alicia Gómez-Barrio David Montero-Pereira Juan J. Nogal-Ruiz José A. Escario Susana Muelas-Serrano Vladimir V. Kouznetsov Leonor Y. Vargas Méndez Juan M. Urbina Gonzáles Carmen Ochoa 《Acta parasitologica / Witold Stefański Institute of Parasitology, Warszawa, Poland》2006,51(1):73-78
A study of some antiparasitic properties of several homoallylamines and related tetrahydroquinolines and quinolines, previously
described, was carried out using in vitro activity assays against the epimastigote form of Trypanosoma cruzi and against Trichomonas vaginalis. Unspecific cytotoxicity against murine macrophages was also studied. Although the antichagasic and trichomonacidal activities
are not comparable to those of the standard drugs, nifurtimox and metronidazole, some of the compounds exhibit an interesting
specific antiparasitic activity. 相似文献