Abstract:
:Pharmacoeconomic data may be obtained within the context of randomised clinical trials (RCTs) and from effectiveness studies in the 'real world'. The differences between the 2 types of study design have implications for the types of data that can be obtained and the interpretation of the resulting findings. Because RCTs are designed to assess the safety and efficacy of pharmaceuticals, and because the study design of RCTs emphasises internal validity over generalisability, the pharmacoeconomic data collected from them are limited. The data may not be applicable to the more heterogeneous patients encountered in actual clinical practice, and cost estimates may be inaccurate because of protocol requirements. Effectiveness studies, in which treatments are studied under real-world conditions, remedy some of these limitations. Generalisability to actual users is generally enhanced in effectiveness designs, but data may be biased in other ways. This brief review compares the 2 study designs as they relate to pharmacoeconomic evaluations in terms of the research questions they address, design differences and their implications for study bias, data collection and data analysis and the generalisability of their results.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Revicki DA,Frank Ldoi
10.2165/00019053-199915050-00001subject
Has Abstractpub_date
1999-05-01 00:00:00pages
423-34issue
5eissn
1170-7690issn
1179-2027journal_volume
15pub_type
杂志文章,评审abstract:BACKGROUND:Bipolar disorder has a significant impact upon a patient's quality of life, imposing a considerable economic burden on the individual, family members and society as a whole. Several medications are indicated for the acute treatment of mania and depression associated with bipolar disorder as well as for maint...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11594930-000000000-00000
更新日期:2012-06-01 00:00:00
abstract:OBJECTIVE:To estimate indirect costs in Spanish ambulatory patients with HIV/AIDS and to identify changes in employment status and their current QOL. METHOD:Information was obtained through 32 interviews/enquiries carried out with ambulatory patients receiving medical attention at Gregorio Marañón and Puerta de Hierro...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200321150-00005
更新日期:2003-01-01 00:00:00
abstract::As part of its single technology process, the National Institute for Health and Care Excellence (NICE) invited the manufacturers of aripiprazole (Otsuka Pharmaceutical Co. and Bristol Myers Squibb) to submit evidence of the clinical and cost effectiveness of aripiprazole for the treatment and prevention of acute manic...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-013-0091-0
更新日期:2013-11-01 00:00:00
abstract::Currently, the most important issue in US pharmaceutical policy is probably cost containment. Research into pharmaceutical issues should therefore concentrate on this area. Analysis of changing trends in pharmaceutical expenditures, particularly during the early 1990s, would be a useful starting point. Research into t...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199600102-00019
更新日期:1996-01-01 00:00:00
abstract::In the current climate of cost containment and quality control, nosocomial infection is a worrisome adverse event in hospital care. Hospitalised patients require care for increasingly severe illnesses, and are therefore more susceptible to infection, especially by opportunistic micro-organisms. It is thus necessary to...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199507020-00005
更新日期:1995-02-01 00:00:00
abstract:BACKGROUND:Interventions in health care often not only have an effect on patients, but also on their informal caregivers. Caregiving can have a profound impact on the health and wellbeing of carers. Ignoring these spillovers in economic evaluations risks labelling interventions mistakenly as cost-effective, at the expe...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0724-4
更新日期:2019-04-01 00:00:00
abstract::Non-melanoma skin cancer (NMSC) and actinic keratosis are becoming an increasingly important healthcare problem. There are approximately 1 million cases of NMSC in the US each year, primarily basal cell carcinomas, and the incidence is increasing. Although NMSC is significant in terms of both health risk and the resou...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422020-00002
更新日期:2004-01-01 00:00:00
abstract::The recent licensing of interferon-beta for use in patients with multiple sclerosis has caused concern, in view of the equivocal evidence of efficacy, pressure of public expectation towards its use and the high expected cost if widespread use were to be sanctioned. Whether such alarm is justified remains a moot point....
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199711030-00002
更新日期:1997-03-01 00:00:00
abstract:OBJECTIVE:To evaluate the cost effectiveness of dabrafenib versus dacarbazine and vemurafenib as first-line treatments in patients with BRAF V600 mutation-positive unresectable or metastatic melanoma from a Canadian healthcare system perspective. METHODS:A partitioned-survival analysis model with three mutually exclus...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-014-0241-z
更新日期:2015-04-01 00:00:00
abstract::Since the inception of the National Health Service in 1948, successive British governments have taken various measures to restrain the growth of the medicines bill. A total of 10 different measures have been introduced with very limited success. The most effective measures have been those directed at increasing the le...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199610030-00003
更新日期:1996-09-01 00:00:00
abstract::Lung cancer remains the leading cause of cancer-related death and economic burden worldwide. Despite the heavy toll of lung cancer, multiple new advances have improved patient outcomes, largely through precision medicine and targeted therapy. The associated rising economic burden however may impact the uptake of novel...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-017-0563-8
更新日期:2017-12-01 00:00:00
abstract::As part of the Single Technology Appraisal (STA) process, the National Institute for Health and Care Excellence (NICE) invited the manufacturer (AstraZeneca) of durvalumab (IMFINZITM) to submit evidence for the clinical and cost effectiveness of durvalumab for the treatment of patients with locally advanced, unresecta...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-019-00870-w
更新日期:2020-04-01 00:00:00
abstract::Pneumocystis carinii pneumonia (PCP) is the most common severe opportunistic infection, and one of the most costly, among people with AIDS. Over 50% of patients experience toxic effects of the major anti-PCP medications- cotrimoxazole (trimethoprim-sulfamethoxazole) and pentamidine. Recently, the US Food and Drug Admi...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章
doi:10.2165/00019053-199609060-00007
更新日期:1996-06-01 00:00:00
abstract:BACKGROUND:Several vaccine and antibody candidates are currently in development for the prevention of lower respiratory tract infections caused by the respiratory syncytial virus (RSV). METHODS:We searched MEDLINE, Embase, and SCOPUS and included model-based evaluations of RSV vaccinations. Two reviewers performed the...
journal_title:PharmacoEconomics
pub_type:
doi:10.1007/s40273-020-00991-7
更新日期:2021-01-19 00:00:00
abstract::In an effort to control escalating health expenditures, especially in hospitals, many countries are planning or experimenting with prospective budgeting systems. Belgium is no exception and has recently introduced, with some success, limited fixed charges per hospital admission and/or per hospitalisation day for labor...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199609030-00006
更新日期:1996-03-01 00:00:00
abstract:BACKGROUND:Cost-effectiveness analyses worldwide assume that the price of any single drug increases with inflation. New guidance from the Pharmaceutical Management Agency in New Zealand suggests that, when it is known that a generic drug will be available in the near future, a best estimate of the lower price of the ge...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200826070-00006
更新日期:2008-01-01 00:00:00
abstract::Until a few years ago, griseofulvin and ketoconazole were the only 2 oral agents available for the treatment of dermatophyte onychomycosis of the toenails. With the availability of the newer antifungal agents, such as itraconazole, terbinafine and fluconazole, the armamentarium of drugs available to treat onychomycosi...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199813020-00007
更新日期:1998-02-01 00:00:00
abstract:OBJECTIVE:The aim of the present study is to describe a refinement of a previously presented method, based on the concept of point sensitivity, to deal with uncertainty in economic studies. DESIGN:The original method was refined by the incorporation of probability distributions which allow a more accurate assessment o...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199916010-00004
更新日期:1999-07-01 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::This article explores the need to perform pharmacoeconomic evaluations of herbal medicines and assesses the extent to which this approach has been applied so far to these products. There seems to be no compelling need for pharmacoeconomic analyses of herbal over-the-counter medicines, but such analyses are certainly w...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200018010-00001
更新日期:2000-07-01 00:00:00
abstract::The lack of a uniform costing methodology is often considered a weakness of economic evaluations that hinders the interpretation and comparison of studies. Standardisation is therefore an important topic within the methodology of economic evaluations and in national guidelines that formulate the formal requirements fo...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200220070-00002
更新日期:2002-01-01 00:00:00
abstract:BACKGROUND:Invasive fungal infections in neutropenic patients treated for haematological malignancies are associated with a high mortality rate and, therefore, require early treatment. As the diagnosis of invasive fungal infections is difficult, effective antifungal prophylaxis is desirable. So far, fluconazole has bee...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200826010-00007
更新日期:2008-01-01 00:00:00
abstract::Disease management is a systematic approach to a health condition or a healthcare intervention that organises preventative, interventional and care approaches throughout the continuum of care and which measures outcomes in terms of populations, not individuals. Disease management's advantage over the current component...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199814010-00002
更新日期:1998-07-01 00:00:00
abstract::Proper economic evaluation involves comparative analysis of alternative courses of action in terms of both costs and outcome. This is difficult for general anaesthesia as there are few alternatives and measurement of outcome is difficult to define. All that is generally available is a cost description of different a...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199303020-00005
更新日期:1993-02-01 00:00:00
abstract:OBJECTIVE:Patients with cancer-associated neutropenia are at high risk of developing severe infections which can be fatal if treatment is not promptly administered. For this reason, fever is treated as soon as possible with broad spectrum antibacterial therapy. The objective of this study was to conduct a cost analysis...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章
doi:10.2165/00019053-199915010-00006
更新日期:1999-01-01 00:00:00
abstract::Constipation is one of the most common digestive complaints. It is a symptom, not a disease. The subjectivity that this involves means that assessments of clinical epidemiology, socioeconomic costs and pharmacotherapy are difficult, since there is no definition of 'normal' bowel habit. Although constipation can affect...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199507010-00003
更新日期:1995-01-01 00:00:00
abstract:OBJECTIVE:To estimate the economic value of pharmacological treatment of type 2 diabetes mellitus in overweight and obese patients using orlistat in addition to standard diabetes therapy (i.e., a sulphonlyurea, metformin or insulin) and weight management strategies as compared with standard diabetes therapy and weight ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200321070-00005
更新日期:2003-01-01 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::This study evaluated the cost utility of adding interferon-alpha 2b to conventional treatment in patients with multiple myeloma. It also provides a methodology for transforming complex quality-of-life profiles into a single index value on the conventional 0 to 1 quality-adjusted life-year scale (QALY). From 1990 to 19...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-199712010-00009
更新日期:1997-07-01 00:00:00
abstract:BACKGROUND:Swedish formulary committees are expected to influence prescribing practice by establishing and issuing drug lists and clinical practice guidelines, particularly now that financial responsibility for prescription drugs has been transferred from the national to the county council level. OBJECTIVE:The purpose...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200017020-00006
更新日期:2000-02-01 00:00:00