Abstract:
BACKGROUND:To reach the French market, a new drug requires a marketing authorization (MA) and price and reimbursement agreements. These hurdles could delay access to new and promising drugs. Since 1992, French law authorizes the use of unlicensed drugs on an exceptional and temporary basis through a compassionate-use programme, known as Temporary Authorization for Use (ATU). This programme was implemented to improve early access to drugs under development or authorized abroad. However, it is suspected to be inflationary, bypassing public bodies in charge of health technology assessment (HTA) and of pricing. OBJECTIVE:The aim of this study is to observe the market access after the formal licensing of drugs that went through this compassionate-use programme. METHODS:We included all ATUs that received an MA between 1 January 2005 and 30 June 2010. We first examined market access delays from these drugs using the standard administrative path. We positioned this result in relation to launch delays observed in France (for all outpatient drugs) and in other major European markets. Second, we assessed the bargaining power of a hospital purchaser after those drugs had obtained an MA by calculating the price growth rate after the approval. RESULTS:During the study period, 77 ATUs were formally licensed. The study concluded that, from the patient's perspective, licensing and public bodies' review time was shortened by a combined total of 36 months. The projected 11-month review time of public bodies may be longer than delays usually observed for outpatient drugs. Nonetheless, the study revealed significant benefits for French patient access based on comparable processing to launch time with those of other European countries with tight price control policies. In return, a 12 % premium, on average, is paid to pharmaceutical companies while drugs are under this status (sub-analysis on 56 drugs). CONCLUSIONS:In many instances, the ATU programme responds to a public health need by accelerating the availability of new drugs even though this study suggests an impact of the programme on the market access of these drugs for which the standard administrative path is longer than usual. In addition, pharmaceutical companies seem to market compassionate-use drugs with a presumed benefit/risk ratio at a price that guarantees a margin for future negotiation.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Degrassat-Théas A,Paubel P,Parent de Curzon O,Le Pen C,Sinègre Mdoi
10.1007/s40273-013-0039-4subject
Has Abstractpub_date
2013-04-01 00:00:00pages
335-43issue
4eissn
1170-7690issn
1179-2027journal_volume
31pub_type
杂志文章abstract::The broad spectrum of antibacterial activity and favourable pharmacokinetic profile of ciprofloxacin permit oral treatment of many serious infections which have traditionally necessitated parenteral antibacterial therapy. This has been demonstrated in comparative studies, in which ciprofloxacin was as effective and w...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199303050-00007
更新日期:1993-05-01 00:00:00
abstract::The use of pharmacoeconomic tools has grown dramatically in the past decade as provision of healthcare throughout the industrialised world has required increased cost consciousness. However, pharmacoeconomic analysis has not yet been fully exploited as a conceptual underpinning for public or private health policy deci...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199916050-00001
更新日期:1999-11-01 00:00:00
abstract::Asian healthcare systems are very diverse, representing cultures, political systems and economies from more than 30 countries with varying histories. Despite the diversity in the region, there has been enormous growth in health economics and outcomes research since the beginning of the 21st century. Whilst Japan has s...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11538360-000000000-00000
更新日期:2010-01-01 00:00:00
abstract::Barrett's oesophagus is a premalignant complication that occurs in approximately 10% of patients with gastro-oesophageal reflux disease (GORD). In patients with Barrett's oesophagus, the risk of adenocarcinoma of the oesophagus approaches 0.5% per patient-years. Therefore, practice guidelines have been developed that ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200119100-00003
更新日期:2001-01-01 00:00:00
abstract::This study examined the costs of treatment of, and quality of life in, patients with antineoplastic therapy-induced neutropenic fever who were treated with antibacterials, with or without granulocyte-macrophage colony-stimulating factor (GM-CSF). Patients with haematological malignancies (n = 47) or solid tumours (n =...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-199712030-00007
更新日期:1997-09-01 00:00:00
abstract:BACKGROUND:The ARTEMIS trial compared first-line antiretroviral therapy (ART) with lopinavir/ritonavir (LPV/r) to darunavir plus ritonavir (DRV + RTV) for HIV-1-infected subjects. In order to fully assess the implications of this study, economic modelling extrapolating over a longer term is required. OBJECTIVE:The aim...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-013-0048-3
更新日期:2013-05-01 00:00:00
abstract::Deterministic sensitivity analyses (DSA) remain important to interpret the effect of uncertainties in individual parameters on results of cost-effectiveness analyses. Classic DSA methodologies may lead to wrong conclusions due to a lack of or misleading information regarding marginal effects, non-linearity, likelihood...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00979-3
更新日期:2021-01-01 00:00:00
abstract::Total hip arthroplasty (THA) is a major orthopaedic procedure with a high risk of postoperative thromboembolism. Increasing demand for this type of surgery, together with its high cost, has led to examination of means by which the cost of THA may be minimised. Current clinical opinion favours the use of suitable pharm...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199610020-00009
更新日期:1996-08-01 00:00:00
abstract:BACKGROUND AND OBJECTIVE:Patient heterogeneity is the part of variability that can be explained by certain patient characteristics (e.g. age, disease stage). Population reimbursement decisions that acknowledge patient heterogeneity could potentially save money and increase population health. To date, however, economic ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-012-0015-4
更新日期:2013-02-01 00:00:00
abstract:BACKGROUND:In Ireland, all new drugs are considered for a formal pharmacoeconomic evaluation (PE) prior to a reimbursement decision. All evaluations are conducted by the National Centre for Pharmacoeconomics (NCPE). The objectives of this study were to describe the evaluation process and to examine the movement of drug...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-016-0437-5
更新日期:2016-12-01 00:00:00
abstract::The current US drug innovation financing framework rests on the notion that a defined period of marketing exclusivity combined with the expectation of reimbursement for clinically valuable, cost-effective therapies, followed by vigorous price competition from generic drugs and biosimilars ensures a sufficient return o...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00926-2
更新日期:2020-09-01 00:00:00
abstract::Economic analysis of healthcare interventions is increasingly reliant on decision models to estimate the long-term costs and benefits of new therapies. Models permit analysts to take short-term clinical data to forecast long-term costs and benefits. Models should follow certain basic principles and can be appraised in...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200220001-00002
更新日期:2002-01-01 00:00:00
abstract::Cost-utility analysis (CUA) is a technique that can potentially be used as a guide to allocating healthcare resources so as to obtain the maximum health benefits possible under a given budget constraint. However, it is not clear that current practice captures societal preferences regarding health benefits. In analyses...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200321020-00001
更新日期:2003-01-01 00:00:00
abstract:BACKGROUND:The symptoms of attention-deficit hyperactivity disorder (ADHD) are associated with impairment in multiple domains of health-related quality of life (HR-QOL). HR-QOL of children with ADHD has been assessed by relatively long multidimensional questionnaires. A review of the literature found no studies using t...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200523080-00004
更新日期:2005-01-01 00:00:00
abstract:OBJECTIVE:Cost is an issue when prescribing two drugs with equivalent efficacy. We compared the direct medical costs of topical brinzolamide 1% (twice a day or three times daily) with topical dorzolamide 2% (twice a day or three times daily) in France, Italy, Portugal and Spain in patients with ocular hypertension or p...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-200321030-00005
更新日期:2003-01-01 00:00:00
abstract:BACKGROUND:Head and neck cancer (HNC) and its treatment can affect communication, nutrition, and physical appearance, and the global impact of this disease on patients' quality of life may be substantial. OBJECTIVE:The aim of this systematic literature review was to describe the impact of HNC and its treatment on the ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-014-0199-x
更新日期:2014-12-01 00:00:00
abstract:OBJECTIVES:This study provides the results of a cost-effectiveness analysis of levetiracetam as an adjunctive treatment for refractory epilepsy from the Canadian Ministry of Health perspective. The main objective is to estimate the expected cost-effectiveness ratio expressed as the incremental cost per seizure-free day...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200523050-00008
更新日期:2005-01-01 00:00:00
abstract:INTRODUCTION:The objective of this study was to evaluate the potential economic implications of using etoricoxib versus non-selective NSAID alternatives in the treatment of patients with osteoarthritis (OA) or rheumatoid arthritis (RA) in the UK. STUDY DESIGN:Decision-analytical modelling was used to calculate the exp...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200422100-00003
更新日期:2004-01-01 00:00:00
abstract::There is a global perspective to the economic consequences of diabetes, which is illustrated in the many publications now being produced on this subject. Because of the higher prevalence of type II diabetes, its impact is greater than that of type I diabetes in both developed and developing countries (but particularly...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199500081-00017
更新日期:1995-01-01 00:00:00
abstract::As a consequence of its prevalence, early onset and chronicity, schizophrenia imposes clinical and economic impediments to healthcare practitioners and society alike. Among the many antipsychotics available to treat the symptoms of this devastating illness, clozapine has emerged and differentiated itself from the othe...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200321010-00001
更新日期:2003-01-01 00:00:00
abstract:BACKGROUND:The process of "mapping" is increasingly being used to predict health utilities, for application within health economic evaluations, using data on other indicators or measures of health. Guidance for the reporting of mapping studies is currently lacking. OBJECTIVE:The overall objective of this research was ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-015-0312-9
更新日期:2015-10-01 00:00:00
abstract::The original article can be found online. ...
journal_title:PharmacoEconomics
pub_type: 已发布勘误
doi:10.1007/s40273-020-00950-2
更新日期:2020-10-01 00:00:00
abstract::Decision-analytic models play a key role in informing healthcare resource allocation decisions. However, there are ongoing concerns with the credibility of models. Modelling methods guidance can encourage good practice within model development, but its value is dependent on its ability to address the areas that modell...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0499-z
更新日期:2017-06-01 00:00:00
abstract:OBJECTIVE:The objective of this study was to estimate the per-patient-per-month (PPPM) costs of medications in the six Medicare Part D protected classes based on findings among Medicare and dual eligible beneficiaries with drug coverage before the enactment of the benefit. DESIGN:Data were from the Thomson Medstat Mar...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200624003-00007
更新日期:2006-01-01 00:00:00
abstract::Randomized controlled trials provide unbiased estimates of treatment effect through reducing the attribution of observed differences in outcome to the play of chance or the treatment of interest. Issues in the design of randomized trials may lead to a dilution or magnification of treatment effect when compared with cl...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200927010-00002
更新日期:2009-01-01 00:00:00
abstract::Discrete choice experiments (DCEs) are regularly used in health economics to elicit preferences for healthcare products and programmes. There is growing recognition that DCEs can provide more than information on preferences and, in particular, they have the potential to contribute more directly to outcome measurement ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200826080-00004
更新日期:2008-01-01 00:00:00
abstract:BACKGROUND:A wide variety of oral antiplatelet trials have been carried out, and a large number of cost-effectiveness estimates based on them have been published. OBJECTIVE:To assess the cost effectiveness of oral antiplatelet treatments in the prevention of cardiovascular events. METHODS:A comprehensive literature s...
journal_title:PharmacoEconomics
pub_type: 杂志文章,meta分析,评审
doi:10.2165/00019053-200725120-00007
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Health technology assessment has been increasingly used in China, having been legally mandated in 2019, to inform reimbursement decisions and price negotiations between the National Healthcare Security Administration and pharmaceutical companies around the price of new pharmaceuticals. The criteria currently...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00954-y
更新日期:2020-12-01 00:00:00
abstract:BACKGROUND:Simulation models utilizing real-world data have potential to optimize treatment sequencing strategies for specific patient subpopulations, including when conducting clinical trials is not feasible. We aimed to develop a simulation model to estimate progression-free survival (PFS) and overall survival for fi...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00951-1
更新日期:2020-11-01 00:00:00
abstract:OBJECTIVE:The study objective was to evaluate and compare quality-of-life (QOL) parameters between patients with type 2 (non-insulin-dependent) diabetes mellitus who changed therapy from an oral hypoglycaemic agent (OHA) to insulin and those who remained on an OHA. DESIGN:The World Health Organization Quality of Life ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199814020-00008
更新日期:1998-08-01 00:00:00