Abstract:
BACKGROUND:Research describing patient experience and outcomes with extended half-life recombinant factor VIII (EHL rFVIII) outside of clinical trials is limited. Real-world rFVIII consumption studies, when people with hemophilia A (PWHA) switch from standard half-life (SHL) to EHL rFVIII, may help payers and clinicians make more informed treatment choices. OBJECTIVE:To conduct a retrospective, observational, U.S.-based analysis to describe clinical and demographic profiles of PWHA who switched to prophylactic rurioctocog alfa pegol. METHODS:Data were obtained from PWHA treated by 38 prescribers across 21 states using specialty pharmacy database case report forms, electronic medical records, and direct communication with providers, PWHA, or their guardians. Assessments included disease severity, pain severity, number and location of target joints, prior HA therapy, reasons for switching, treatment duration, dosing frequency, adherence, and annualized bleeding rates (ABRs) before and after switching to rurioctocog alfa pegol from SHL or another EHL rFVIII. RESULTS:Data were collected from 56 PWHA. The mean age was 26 years (range = 5-88); median age was 24 years (interquartile range = 14-34); 20% were aged < 12 years; and 89% (50/56) had severe HA. All PWHA had ≥ 12 months of rFVIII treatment before switching to rurioctocog alfa pegol. The population had a mean 1.8 target joints. Baseline subjective pain assessment was mild to moderate for 68% (38/56) of respondents. Before receiving rurioctocog alfa pegol, most PWHA received antihemophilic factor (recombinant) for prophylaxis (73%, 41/56) or breakthrough bleeding (59%, 33/56). Mean dosing frequency for prior prophylaxis was 2.8 per week for SHL rFVIII and 1.8 per week for EHL rFVIII, and 2.2 per week for all PWHA after switching to rurioctocog alfa pegol prophylaxis. The median time on rurioctocog alfa pegol prophylaxis was 12.0 months versus 80.8 months on previous SHL rFVIII and 13.5 months on previous EHL rFVIII. Mean ABRs on prior prophylaxis were 5.9 for SHL rFVIII (n = 35) and 4.7 for EHL rFVIII (n = 3). After switching to rurioctocog alfa pegol, the overall mean ABR reduced by 71% (5.8 to 1.7, P < 0.001) and 20/56 PWHA had no bleeding events. There was also a 20.9% reduction in the mean days per week of factor administration (P < 0.001) after switching to prophylactic rurioctocog alfa pegol. For 47 PWHA who switched from SHL rFVIII, their weekly dose decreased from 109.8 to 100.6 IU per kg with rurioctocog alfa pegol (P = 0.094). The proportion of PWHA with good/complete treatment adherence increased from 68% (38/56) on any prior rFVIII to 80% (45/56) on rurioctocog alfa pegol. The most common reason PWHA switched to rurioctocog alfa pegol was to reduce treatment infusions. CONCLUSIONS:Switching from either an SHL or EHL rFVIII to rurioctocog alfa pegol is associated with fewer bleeding episodes owing to more effective prophylaxis and improved adherence. Those who switched from an SHL rFVIII reported reduced factor consumption with rurioctocog alfa pegol. This long-acting factor is an important additional option for the care of PWHA. DISCLOSURES:This study was funded by Shire Development LLC, a Takeda company, Lexington, MA. Trio Health was involved in study design and acquisition, analysis, and interpretation of data and was funded by Shire Development LLC, a Takeda company. Aledort serves on the data and safety monitoring boards of Baxalta U.S. Inc., a Takeda company, and Octapharma; is chair of the scientific advisory board of Kedrion; and receives consultancy fees and honoraria from Baxalta U.S. Inc., a Takeda company. Milligan is an employee of Trio Health and reports research support from AbbVie, Gilead, Merck, Sanofi, and ViiV, unrelated to this study. Watt is an employee of Shire International GmbH, a Takeda company, and owns stock in the company. Booth was an employee of Baxalta U.S. Inc., a Takeda company, at the time of this study and owns stock in the company. Data from this study were presented at the AMCP Managed Care and Specialty Pharmacy Annual Meeting; April 23-28, 2018; Boston, MA; SETH (2018) Sociedad Espanola de Trombosis y Hemostasia-XXXIV Congreso Nacional; October 11-13, 2018; Grenada, Espana; and Blood 2018 Annual Scientific Meeting; October 21-24, 2018; Brisbane, Australia.
journal_name
J Manag Care Spec Pharmjournal_title
Journal of managed care & specialty pharmacyauthors
Aledort L,Milligan S,Watt M,Booth Jdoi
10.18553/jmcp.2020.26.4.492subject
Has Abstractpub_date
2020-04-01 00:00:00pages
492-503issue
4eissn
2376-0540issn
2376-1032journal_volume
26pub_type
杂志文章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: Measuring patient perspectives of the quality of health care delivery is an essential component of building a patient-centered model of care, which has garnered increasing emphasis under value-based payment models. Although measurements of patient perspectives of physician practices are common, few validat...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2021.27.1.105
更新日期:2021-01-01 00:00:00
abstract:BACKGROUND:The functional impairment associated with migraine can cause physical, emotional, and economic ramifications that can affect occupational, academic, social, and family life. Understanding the relationship between headache-free days (HFDs) and the disease burden of migraine may help with decisions regarding t...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.20103
更新日期:2020-10-01 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: The economic burden of food allergy is large; however, costs specific to individuals with peanut allergy experiencing reactions to peanuts remain to be evaluated. As the prevalence of peanut allergy continues to increase in children, a better understanding of the cost of care is warranted. OBJECTIVE: To as...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2021.20389
更新日期:2021-01-20 00:00:00
abstract:BACKGROUND:Accurate estimates of the effects of cost sharing on adherence to medications prescribed for use together, also called concurrent adherence, are important for researchers, payers, and policymakers who want to reduce barriers to adherence for chronic condition patients prescribed multiple medications concurre...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2015.21.8.678
更新日期:2015-08-01 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:Formulary or preferred drug list (PDL) management is an effective strategy to ensure clinically efficient prescription drug management by managed care organizations (MCOs). Medicaid MCOs participating in Florida's Medicaid program were required to use a state-mandated PDL between May and August 2014. OBJECT...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.2.124
更新日期:2018-02-01 00:00:00
abstract:BACKGROUND:Clinical trials have shown that direct oral anticoagulants (DOACs)-including dabigatran, rivaroxaban, apixaban, and edoxaban-are at least as effective and safe as warfarin for the risk of stroke/systemic embolism (SE) and major bleeding (MB) in patients with atrial fibrillation (AF). However, few studies hav...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.9.911
更新日期:2018-09-01 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
abstract:BACKGROUND:Formulary management within a limited budget is critical, especially for specialty drugs, which are used for serious medical conditions and are very expensive. Despite attempts to summarize the pertinent evidence, it is uncertain whether data needs of formulary decision makers for specialty drugs are satisfi...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.4.368
更新日期:2016-04-01 00:00:00
abstract:BACKGROUND:Previous research finds significant variation in spending and utilization across regions, with little evidence of differences in outcomes. While such findings have been interpreted as evidence that spending can be reduced without compromising patient outcomes, the link between spending variation and outcomes...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.6.504
更新日期:2018-06-01 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: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:BACKGROUND:Several authors have hypothesized that adverse drug events (ADEs) upon switching from reference biologics to biosimilar products are related to the nocebo effect. However, a thorough and current review of the existing literature has not been conducted. OBJECTIVE:To evaluate if patient and/or physician knowl...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.10.952
更新日期:2018-10-01 00:00:00
abstract:BACKGROUND:Adjuvant trastuzumab treatment is administered to early stage breast cancer patients in physician office (POV) or hospital outpatient (HOP) settings. OBJECTIVE:To identify treatment patterns, utilization, and costs by site of care (POV vs. HOP) of patients with adjuvant treatment of breast cancer with trast...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2014.20.5.485
更新日期:2014-05-01 00:00:00
abstract:BACKGROUND:Cancer is a leading cause of death with substantial financial costs. While significant data exist on the economic burden of care, less is known about the indirect costs of treatment and, specifically, the effect on work productivity of patients and their caregivers. To examine the full effect of cancer and t...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章,meta分析
doi:10.18553/jmcp.2017.23.2.136
更新日期:2017-02-01 00:00:00
abstract:BACKGROUND:Tisagenlecleucel was approved for the treatment of pediatric and young adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (ALL) based on the pivotal ELIANA trial. OBJECTIVE:To comprehensively evaluate the total costs associated with tisagenlecleucel treatment, including costs ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.20052
更新日期:2020-08-01 00:00:00
abstract:BACKGROUND:Prescription medication adherence is a known health-related barrier for elderly patients, leading to insufficient disease control and negative health outcomes. The Centers for Medicare & Medicaid Services (CMS) have placed significant emphasis on medication adherence, through the Part D star measures, revolv...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2016.22.3.305
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:Diabetes requires close monitoring to achieve optimal outcomes and avoid adverse effects. Continuous glucose monitoring (CGM) is one approach to measuring glycemia and has become more widespread with recent advances in technology; however, ideal implementation of CGM into clinical practice is unknown. CGM ca...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.26.5.600
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:Adherence to antiretrovirals (ARVs) is critical to achieving durable virologic suppression. OBJECTIVE:To investigate risk factors of poor adherence and the effect of suboptimal adherence on health care resource utilization (HCRU) and costs in Medicaid patients. METHODS:A retrospective longitudinal study wa...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.17507
更新日期:2018-10-01 00:00:00
abstract:BACKGROUND:Overuse and misuse of prescription opioids is associated with increased morbidity and mortality and places a significant cost burden on health systems. OBJECTIVE:To estimate annual statewide spending for prescription opioids in Rhode Island. METHODS:A cross-sectional study of opioids dispensed from retail ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2018.24.3.214
更新日期:2018-03-01 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:DISCLOSURES:No funding supported the writing of this commentary. Brummel has consulted, presented, or was engaged in an advisory board for UCB, Boerhinger Ingelheim, Pfizer, and Lilly. Sorenson has nothing to disclose. ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.26.9.1067a
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:Optimized medication use involves the effective use of medications for better outcomes, improved patient experience, and lower costs. Few studies systematically gather data on the actions accountable care organizations (ACOs) have taken to optimize medication use. OBJECTIVES:To (a) assess how ACOs optimize ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2017.23.10.1054
更新日期:2017-10-01 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:The misuse of prescription drugs is a serious public health problem. Although controlled substance (CS) prescribing, in particular, opioid analgesics, has recently declined, the volume of prescriptions in 2015 was still 3 times higher than in 1999. To curb the high volume of CS prescribing, a national health...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2019.25.3.392
更新日期:2019-03-01 00:00:00
abstract:BACKGROUND:Costs associated with biologic switching and discontinuation can be high in chronic inflammatory diseases. Inappropriate use of medications may have cost implications for both payers and patients. Understanding of biologic utilization and switching rates is lacking among patients with ankylosing spondylitis ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章
doi:10.18553/jmcp.2020.19433
更新日期:2021-01-01 00:00:00
abstract:DISCLOSURES:No funding contributed to the writing of this commentary. Brandsema reports consulting for Alexion, Audentes, AveXis, Biogen, Cytokinetics, PTC Therapeutics, Sarepta, and WaVe and has received research funding as a site investigator from Alexion, AveXis, Biogen, CSL Behring, Cytokinetics, Fibrogen, Pfizer, ...
journal_title:Journal of managed care & specialty pharmacy
pub_type: 杂志文章,评审
doi:10.18553/jmcp.2020.26.4.366
更新日期:2020-04-01 00:00:00