Abstract:
BACKGROUND:While there is growing demand for information about comparative effectiveness (CE), there is substantial debate about whether and when observational studies have sufficient quality to support decision making. OBJECTIVE:To develop and test an item checklist that can be used to qualify those observational CE studies sufficiently rigorous in design and execution to contribute meaningfully to the evidence base for decision support. METHODS:An 11-item checklist about data and methods (the GRACE checklist) was developed through literature review and consultation with experts from professional societies, payer groups, the private sector, and academia. Since no single gold standard exists for validation, checklist item responses were compared with 3 different types of external quality ratings (N=88 articles). The articles compared treatment effectiveness and/or safety of drugs, medical devices, and medical procedures. We validated checklist item responses 3 ways against external quality ratings, using published articles of observational CE or safety studies: (a) Systematic Review-quality assessment from a published systematic review; (b) Single Expert Review-quality assessment made according to the solicited "expert opinion" of a senior researcher; and (c) Concordant Expert Review-quality assessments from 2 experts for which there was concordance. Volunteers (N=113) from 5 continents completed 280 article assessments using the checklist. Positive and negative predictive values (PPV, NPV, respectively) of individual items were estimated to compare testers' assessments with those of experts. RESULTS:Taken as a whole, the scale had better NPV than PPV, for both data and methods. The most consistent predictor of quality relates to the validity of the primary outcomes measurement for the study purpose. Other consistent markers of quality relate to using concurrent comparators, minimizing the effects of bias by prudent choice of covariates, and using sensitivity analysis to test robustness of results. Concordance of expert opinion on the quality of the rated articles was 52%; most checklist items performed better. CONCLUSIONS:The 11-item GRACE checklist provides guidance to help determine which observational studies of CE have used strong scientific methods and good data that are fit for purpose and merit consideration for decision making. The checklist contains a parsimonious set of elements that can be objectively assessed in published studies, and user testing shows that it can be successfully applied to studies of drugs, medical devices, and clinical and surgical interventions. Although no scoring is provided, study reports that rate relatively well across checklist items merit in-depth examination to understand applicability, effect size, and likelihood of residual bias. The current testing and validation efforts did not achieve clear discrimination between studies fit for purpose and those not, but we have identified a critical, though remediable, limitation in our approach. Not specifying a specific granular decision for evaluation, or not identifying a single study objective in reports that included more than one, left reviewers with too broad an assessment challenge. We believe that future efforts will be more successful if reviewers are asked to focus on a specific objective or question. Despite the challenges encountered in this testing, an agreed upon set of assessment elements, checklists, or score cards is critical for the maturation of this field. Substantial resources will be expended on studies of real-world effectiveness, and if the rigor of these observational assessments cannot be assessed, then the impact of the studies will be suboptimal. Similarly, agreement on key elements of quality will ensure that budgets are appropriately directed toward those elements. Given the importance of this task and the lessons learned from these extensive efforts at validation and user testing, we are optimistic about the potential for improved assessments that can be used for diverse situations by people with a wide range of experience and training. Future testing would benefit by directing reviewers to address a single, granular research question, which would avoid problems that arose by using the checklist to evaluate multiple objectives, by using other types of validation test sets, and by employing further multivariate analysis to see if any combination or sequence of item responses has particularly high predictive validity.
journal_name
J Manag Care Spec Pharmjournal_title
Journal of managed care & specialty pharmacyauthors
Dreyer NA,Velentgas P,Westrich K,Dubois Rdoi
10.18553/jmcp.2014.20.3.301subject
Has Abstractpub_date
2014-03-01 00:00:00pages
301-8issue
3eissn
2376-0540issn
2376-1032pii
2014(20)3: 301-308journal_volume
20pub_type
杂志文章abstract::The AMCP Abstracts program provides a forum through which authors can share their insights and outcomes of advanced managed care practice through publication in AMCP's Journal of Managed Care & Specialty Pharmacy (JMCP). Most of the reviewed and unreviewed abstracts are presented as posters so that interested AMCP mee...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.4.S1
更新日期:2016-04-01 00:00:00
abstract::We write to comment on a recently published study by Delea et al. in the January 2015 issue of JMCP that evaluated the cost-effectiveness (CE) of sunitinib (SU) versus pazopanib (PAZ) as first-line treatment for metastatic renal cell carcinoma (mRCC) from a U.S. third-party payer perspective.1 This analysis was based ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 评论,信件
doi:10.18553/jmcp.2015.21.9.834
更新日期:2015-09-01 00:00:00
abstract:BACKGROUND:Adherence to biologic disease-modifying antirheumatic drugs (bDMARDs) among patients with rheumatoid arthritis (RA) is often suboptimal in routine clinical practice. Low or nonadherence can reduce the effectiveness of bDMARD therapies. OBJECTIVE:To evaluate filling of newly prescribed initial bDMARDs for th...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.3.209
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:Treatment patterns for metastatic colorectal cancer (mCRC) patients have changed considerably over the last decade with the introduction of new chemotherapies and targeted biologics. These treatments are often administered in various sequences with limited evidence regarding their cost-effectiveness. OBJECT...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.23.1.64
更新日期:2017-01-01 00:00:00
abstract:BACKGROUND:Geographic variation in the use of prescription drugs, particularly those deemed harmful by the FDA, may lead to variation in patient exposure to adverse drug events. One such drug is the glucose-lowering drug rosiglitazone, for which the FDA issued a safety alert on May 21, 2007, following the publication o...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2015.21.12.1214
更新日期:2015-12-01 00:00:00
abstract:BACKGROUND:Opioid pain relievers can be highly effective in providing relief for patients suffering from pain. At the same time, prescription opioid abuse, dependence, overdose, and poisoning (hereinafter "abuse") have become a national public health concern. Opioid abuse is also costly: previous estimates of the annua...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.16265
更新日期:2017-04-01 00:00:00
abstract:BACKGROUND:Diabetes mellitus is associated with substantial morbidity and mortality. With the rise in prevalence of diabetes, there has been an increased need for clinical pharmacy services focused on diabetes management in ambulatory clinics. However, more data IS needed to determine the overall impact that clinical p...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2014.20.9.914
更新日期:2014-09-01 00:00:00
abstract:BACKGROUND:The implementation of Medicare Part D provided insurance coverage for outpatient medications, but when persons reach the "gap," they have very limited or no medication insurance coverage until they reach a second threshold for catastrophic coverage. In addition, some patients have a low-income subsidy (LIS),...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2014.20.8.862
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Three pharmacist-specific Current Procedural Terminology (CPT) codes exist to facilitate medication therapy management (MTM) reimbursement (codes 99605, 99606, and 99607). However, no studies have used CPT codes in administrative claims databases to identify subjects who have received MTM services. OBJECTIV...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.26.10.1297
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Encouraging generic drug use has reduced health care costs for payers and consumers, but the availability of therapeutically interchangeable medications or generic medications of choice is not equal across disease states. The extent to which systems of care are able to substitute with generics is not well un...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2014.20.11.1093
更新日期:2014-11-01 00:00:00
abstract:BACKGROUND:New 3-drug regimens have been developed and approved to treat multiple myeloma (MM). The absence of direct comparative data and the high cost of treatment support the need to assess the relative clinical and economic outcomes across all approved regimens. OBJECTIVE:To evaluate the cost-effectiveness of trea...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.1.29
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:In recent years, comparative effectiveness tools and methods have evolved to assist health care decision makers in identifying optimal therapies. In-person training programs on comparative effectiveness research may be helpful in understanding and applying this information. OBJECTIVE:To provide a follow-up ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.6.618
更新日期:2016-06-01 00:00:00
abstract:BACKGROUND:Individuals with Down syndrome (DS) experience various comorbidities in excess of the prevalence seen among the non-DS population. However, the extent of the excess burden of comorbidities specifically within commercially and publicly insured DS populations aged < 21 years is not currently known. OBJECTIVES...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.23.7.761
更新日期:2017-07-01 00:00:00
abstract:BACKGROUND:Prescription opioid abuse is associated with substantial economic burden, with estimates of incremental annual per-patient health care costs of diagnosed opioid abuse exceeding $10,000 in prior literature. A subset of patients diagnosed with opioid abuse has disproportionately high health care costs, but lit...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2015.21.10.902
更新日期:2015-10-01 00:00:00
abstract::Improving medication adherence has been identified as a crucial step towards improving health outcomes for patients with chronic disease and has provided the motivation for many changes in our health care system. Despite the volume of research done on this topic, however, we still lack important basic information abou...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章,评审
doi:10.18553/jmcp.2014.20.8.775
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Acromegaly is a chronic disorder characterized by excess growth hormone secretion and elevated insulin-like growth factor-1 levels most often caused by a pituitary adenoma. Clinical presentation of the disease includes coarsening of the facial features, soft-tissue swelling of the hands and feet, and overgro...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2015.21.12.1106
更新日期:2015-12-01 00:00:00
abstract:BACKGROUND:Nearly half of statin users discontinue therapy within the first year of treatment. Nonadherence to statin therapy may lead to an increased risk of atherosclerotic cardiovascular disease and, thus, higher costs due to hospitalizations. Value-based care models, such as accountable care organizations (ACO), ar...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2019.25.5.588
更新日期:2019-05-01 00:00:00
abstract:BACKGROUND:Rosacea is a chronic skin disorder that presents with abnormal vascular and inflammatory conditions. Clinical manifestations include flushing, facial erythema, inflammatory papules and pustules, telangiectasias, edema, and watery or irritated eyes. OBJECTIVE:To discuss the evolving pathophysiology of rosace...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章,评审
doi:10.18553/jmcp.2014.20.6.623
更新日期:2014-06-01 00:00:00
abstract:BACKGROUND:The impact of formulary management strategies on utilization and expenditures in overactive bladder (OAB) treatment has not been extensively investigated. In 2013, step therapy (ST) policies for 2 branded OAB treatments, mirabegron and fesoterodine, were removed from Humana Medicare Advantage Prescription Dr...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.23.1.27
更新日期:2017-01-01 00:00:00
abstract:BACKGROUND:Multiple sclerosis (MS) is a neurological degenerative chronic condition without cure. However, long-term disease-modifying therapies (DMTs) help reduce the severity of MS symptoms. Adherence to DMTs is key to their success. Several studies have analyzed what makes patients adherent to their DMTs. As new DMT...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.12.1394
更新日期:2016-12-01 00:00:00
abstract:BACKGROUND:In the United States, many children with cerebral palsy (CP) obtain health care coverage through managed Medicaid, but little is known about the current demographics or management of this high-need, complex population. OBJECTIVE:To develop U.S. population-level information about the prevalence of CP, manage...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2019.25.7.817
更新日期:2019-07-01 00:00:00
abstract:BACKGROUND:Psoriasis is a chronic, hyper-proliferative dermatological condition associated with joint symptoms known as psoriatic arthritis (PsA). In a 2013 review, the total economic burden of PsA was estimated at $51.7-$63.2 billion. The economic burden of moderate to severe psoriasis patients has reduced significant...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.7.654
更新日期:2018-07-01 00:00:00
abstract:BACKGROUND:Value-based insurance design (VBID) waives or reduces prescription copayments in order to decrease member cost barriers to refilling medications. Medication therapy management (MTM) is a member clinical intervention designed to reinforce members' knowledge of their medications, which addresses barriers to me...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.11.1303
更新日期:2016-11-01 00:00:00
abstract:BACKGROUND:Employers have increased efforts to engage employees in health and wellness programs. Providing employees with incentives to participate in these programs has been shown to improve overall enrollment and engagement. One program that has had challenges with enrollment and engagement is medication therapy mana...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.23.11.1140
更新日期:2017-11-01 00:00:00
abstract:DISCLOSURES:The author provides consulting services to the pharmaceutical industry, including manufacturers, health plans, employers, and specialty pharmacies, and is a member of the editorial advisory board of Specialty Pharmacy News. The author reports no financial or other conflicts of interest related to the subjec...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.16091
更新日期:2016-08-01 00:00:00
abstract:UNLABELLED:Medication adherence is a problem that has received widespread attention in the medical literature and health policy circles. With the increased emphasis on recognizing and rewarding quality in the U.S. health care system, medication adherence measures are increasingly being adopted to assess quality of medi...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.5.598
更新日期:2016-05-01 00:00:00
abstract:DISCLOSURES:No funding was involved in the writing of this letter. Outside of the submitted work, Hiligsmann has received research grants through institution from Amgen, Radius Health, UCB, and Teva/Theramex. Reginster has received research grants and/or consulting fees from Servier, Novartis, Negma, Lilly, Wyeth, Amge...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 评论,信件
doi:10.18553/jmcp.2020.26.3.334
更新日期:2020-03-01 00:00:00
abstract:BACKGROUND:Poor medication adherence is a predictor of poor health outcomes, especially in populations with chronic diseases. Although several self-reported measures of medication adherence exist, the scope of each is limited. OBJECTIVE:To identify barriers to medication adherence in order to facilitate effective del...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2015.21.5.391
更新日期:2015-05-01 00:00:00
abstract:BACKGROUND:Immunoglobulin (Ig) is a costly blood product prescribed as immune replacement or modulation therapy to treat a wide spectrum of medical conditions. While the FDA has approved Ig for a limited number of indications, there are multiple off-label uses that have demonstrated proven clinical benefit or are curre...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2014.20.4.357
更新日期:2014-04-01 00:00:00
abstract:BACKGROUND:The management of sickle cell disease (SCD), an inherited, chronic, and multifaceted condition, is associated with considerable health care resource utilization (HRU) and costs, especially for Medicaid. Anemia affects most patients with SCD and correlates with end-organ damage (EOD), such as stroke, chronic ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.20009
更新日期:2020-09-01 00:00:00