Abstract:
INTRODUCTION:The traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-specific outcome measures. Incorporating qualitative research into rare disease clinical trials may help sponsors, regulators, payers, and prescribers to better understand the real-world and patient-specific impact of a potential therapy. This paper provides a methodologic overview of the use of Patient and Caregiver Perception of Change (PPC and CPC) Assessments utilizing patient and caregiver video interviews to complement the data captured by traditional endpoints in rare disease clinical trials. METHODS:Incorporating qualitative patient and caregiver video interviews into clinical trials allows for the rigorous capture of patient experiences and caregiver observations. Interview guides informed by input from key stakeholders provide the opportunity to solicit structured feedback on experiences before, during, and after the clinical trial. Patients and caregivers can complete their video interviews in a study mobile application, and interview transcripts are analyzed by independent coders. Themes are summarized by the treatment group and individual patient, which adds context to the clinical outcome measures of how patients feel and function, as well as elucidates the degree of change that is meaningful to patients and caregivers. The qualitative results can be compared to the data captured in clinical trials to assess data concordance. CONCLUSION:Capturing patient experience data with sufficient rigor allows it to contribute to the body of evidence utilized in regulatory, payer, and prescriber decision-making. Adding PPC and CPC Assessments to rare disease clinical trials offers an innovative and powerful way to tap into the unique insights of patients and their families to develop a fuller picture of the patient experience in the clinical trial. FUNDING:Stealth BioTherapeutics Inc.
journal_name
Adv Therjournal_title
Advances in therapyauthors
Contesse MG,Valentine JE,Wall TE,Leffler MGdoi
10.1007/s12325-019-00920-xsubject
Has Abstractpub_date
2019-05-01 00:00:00pages
997-1010issue
5eissn
0741-238Xissn
1865-8652pii
10.1007/s12325-019-00920-xjournal_volume
36pub_type
杂志文章,评审abstract:INTRODUCTION:Sevelamer hydrochloride (SH) and lanthanum carbonate (LC) are calcium-free phosphate binders used for the management of hyperphosphatemia in patients with end-stage renal disease (ESRD). The objective of this analysis was to evaluate the real-world dose-relativity between SH and LC monotherapy in US patien...
journal_title:Advances in therapy
pub_type: 杂志文章,多中心研究
doi:10.1007/s12325-013-0077-5
更新日期:2013-12-01 00:00:00
abstract::Coronary heart disease is the major cause of morbidity and mortality in industrialized countries, and its prevalence is predicted to grow as the population ages. Current drugs for chronic stable angina (such as beta-blockers, calcium-channel blockers, long- and short-acting nitrates, and potassium-channel activators) ...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-009-0005-x
更新日期:2009-02-01 00:00:00
abstract:PURPOSE:To evaluate the impact on cost, time, resource use, and clinic workflow of converting the route of drug administration from a neurokinin-1 receptor antagonist (NK-1 RA) 30-min intravenous (IV) infusion to aprepitant IV, and more specifically to IV push, within a multicenter community oncology practice. METHODS...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01377-z
更新日期:2020-07-01 00:00:00
abstract::In an open-label 12-week study, the safety and efficacy of bimatoprost 0.03% was evaluated in 55 patients with open-angle glaucoma or ocular hypertension inadequately controlled by topical beta-blocker monotherapy. Patients discontinued their topical beta-blocker therapy at the baseline visit and began bimatoprost mon...
journal_title:Advances in therapy
pub_type: 临床试验,杂志文章
doi:10.1007/BF02850101
更新日期:2004-11-01 00:00:00
abstract::Traveling through several time zones results in a constellation of symptoms known as jet lag. These include reduced alertness, daytime fatigue, loss of appetite, reduced cognitive skills, and disruption of the sleep/wake cycle. In susceptible air travel passengers, jet lag may exacerbate affective illness and result i...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-010-0065-y
更新日期:2010-11-01 00:00:00
abstract::In a multicenter, open-label evaluation, 1098 patients with ocular itching and a history of perennial or seasonal allergic conjunctivitis instilled one drop of nedocromil sodium 2% twice daily in each eye. Ocular symptoms, signs, and global improvement were assessed at baseline and 1 month; satisfaction scores, qualit...
journal_title:Advances in therapy
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1007/BF02850056
更新日期:2002-03-01 00:00:00
abstract:INTRODUCTION:In FRESCO (Fruquintinib Efficacy and Safety in 3+ Line Colorectal Cancer Patients) trial, fruquintinib demonstrated a statistically significant and clinically meaningful overall survival benefit in Chinese patients with metastatic colorectal cancer (mCRC). However, its safety profile, including adverse eve...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01477-w
更新日期:2020-11-01 00:00:00
abstract:INTRODUCTION:This study aims to evaluate the early predictive value for postoperative sepsis and 30-day mortality in liver transplant patients using sequential organ failure assessment (SOFA). METHODS:A total of 96 liver transplant patients were enrolled into this study from February 2015 to June 2018. The general inf...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-019-0889-z
更新日期:2019-03-01 00:00:00
abstract:INTRODUCTION:To assess the risk factors associated with regorafenib-related adverse events (AEs) in metastatic colorectal cancer (mCRC) and gastrointestinal stromal tumors (GIST). We also evaluated different measures of combatting AEs and their success rate to aid physicians in early identification and management of re...
journal_title:Advances in therapy
pub_type: 杂志文章,meta分析
doi:10.1007/s12325-019-01013-5
更新日期:2019-08-01 00:00:00
abstract:INTRODUCTION:Idiopathic pulmonary fibrosis (IPF) is the classic progressive fibrosing interstitial lung disease (ILD), but some patients with ILDs other than IPF also develop a progressive fibrosing phenotype (PF-ILD). Information on use and cost of healthcare resources in patients with PF-ILD is limited. METHODS:We u...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01380-4
更新日期:2020-07-01 00:00:00
abstract:PURPOSE:A recent successful clinical trial demonstrated that a less invasive cell-injection procedure is a viable medical modality for treating corneal endothelial dystrophy. This medical advance still relies on human corneal endothelial cell (HCEC) sources derived from rare cornea donations. The progenitor of the corn...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-020-01237-w
更新日期:2020-03-01 00:00:00
abstract:INTRODUCTION:This retrospective study used data from a primary care database to compare two insulin products in routine clinical practice for the treatment of type 2 diabetes in the UK. PATIENTS AND METHODS:Records were analyzed for patients with type 2 diabetes who had been initiated on biphasic insulin aspart 30 (BI...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-010-0079-5
更新日期:2010-11-01 00:00:00
abstract:INTRODUCTION:Partial nephrectomy (PN) has evolved into the surgical standard of care for localized renal lesions. Hemostatic agents (HA) support the surgeon in achieving local hemostasis during PN. We previously reported initial results with the HA Hemopatch® in PN. We now report our experiences with Hemopatch® in a la...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01584-8
更新日期:2020-12-04 00:00:00
abstract::X-linked hypophosphataemia (XLH) is the most prevalent form of hereditary rickets characterized by an alteration of phosphate metabolism which frequently leads to the appearance of fractures, bone deformities and growth delay. Although the mechanism of growth impairment in patients with XLH still needs to be clarified...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-019-01178-z
更新日期:2020-05-01 00:00:00
abstract::This study was performed to investigate the relationships between markers of inflammation in serum (interleukin-6 [IL-6], interleukin-10 [IL-10], and granulocyte elastase [GE]), severity of injury, and clinical outcomes, and to evaluate the predictive value of these markers for major complications and mortality. This ...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/BF02877699
更新日期:2007-09-01 00:00:00
abstract:BACKGROUND:The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) outbreak is a serious health concern. Repurposing of existing drugs indicated for other conditions seems to be the first choice for immediate therapeutic management. The quality of early evidence favoring the different treatment options needs t...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01460-5
更新日期:2020-10-01 00:00:00
abstract:OBJECTIVES:Because clinical guidelines do not offer clear recommendations for treatment options after discontinuing a tumor necrosis factor (TNF) blocker, this study evaluated treatment patterns within 360 days after discontinuation of TNF-blocker treatment. METHODS:The IMS LifeLink Health Plan Claims database was use...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-014-0110-3
更新日期:2014-04-01 00:00:00
abstract::Atrial fibrillation (AF) and venous thromboembolism (VTE) are highly prevalent and relevant healthcare issues. Direct oral anticoagulants (DOACs) are now the first-choice for anticoagulant treatment of these conditions displaying a better efficacy/safety profile than vitamin-K antagonists, mainly due to significantly ...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-020-01307-z
更新日期:2020-05-01 00:00:00
abstract:OBJECTIVE:Examine outcomes and costs of patients with persistent asthma who initiated treatment with beclomethasone dipropionate hydrofluoroalkane (BDP-HFA) or fluticasone propionate (FP). METHODS:MedStat's Commercial Claims and Encounters database (July 1, 2002-June 30, 2007) was utilized. Patients (n=13,968) were in...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-009-0056-z
更新日期:2009-08-01 00:00:00
abstract:INTRODUCTION:The 21-gene breast cancer assay (Oncotype DX(®); Genomic Health, Inc.) is a validated diagnostic test that predicts the likelihood of adjuvant chemotherapy benefit and 10-year risk of distant recurrence in patients with hormone-receptor-positive, human epidermal growth receptor 2-negative, early-stage brea...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-015-0190-8
更新日期:2015-03-01 00:00:00
abstract:INTRODUCTION:To evaluate the retinal function and the relative neural conduction along the visual pathway after treatment with citicoline in liposomal formulation (CLF) eye drops in patients with open angle glaucoma (OAG). METHODS:Twelve OAG patients (mean age ± standard deviation 52.58 ± 11.39 years, intraocular pres...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-019-0897-z
更新日期:2019-04-01 00:00:00
abstract:INTRODUCTION:Stimulant medications are the most effective drugs in the treatment of attention-deficit hyperactivity disorder (ADHD) in children and in adults. However, some patients do not respond to this treatment and other patients suffer from adverse effects. Very often there are also comorbid disorders that warrant...
journal_title:Advances in therapy
pub_type: 杂志文章,meta分析,评审
doi:10.1007/s12325-009-0008-7
更新日期:2009-02-01 00:00:00
abstract::This study was undertaken to evaluate the ocular hypotensive efficacy of brimonidine Purite 0.15% (Alphagan P 0.15%; Allergan, Inc., Irvine, Calif) given as adjunctive therapy with latanoprost 0.005% (Xalatan; Pfizer Inc., New York, NY( to patients with open-angle glaucoma or ocular hypertension. In this multicenter, ...
journal_title:Advances in therapy
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1007/BF02849898
更新日期:2007-03-01 00:00:00
abstract:INTRODUCTION:To evaluate long-term outcomes following stand-alone implantation of two second-generation trabecular micro-bypass stents (iStent inject®, Glaukos Corp., San Clemente, CA, USA) in eyes with predominantly primary open-angle glaucoma (POAG) and considerable preoperative disease burden. METHODS:Eyes with POA...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-019-00984-9
更新日期:2019-07-01 00:00:00
abstract::Unfortunately, the fourteenth author's name was incorrectly published in the Original Article. The correct author name is Cen Hong. ...
journal_title:Advances in therapy
pub_type: 已发布勘误
doi:10.1007/s12325-019-01009-1
更新日期:2019-08-01 00:00:00
abstract:INTRODUCTION:Vitamin E is one of the most promising agents for nonalcoholic steatohepatitis (NASH) treatment, and its drug responsiveness may be closely associated with haptoglobin (Hp) genotype. However, its efficacy and safety remain unknown in China. This clinical trial of vitamin E versus placebo for the treatment ...
journal_title:Advances in therapy
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1007/s12325-018-0670-8
更新日期:2018-02-01 00:00:00
abstract:INTRODUCTION:Type 2 diabetes mellitus (T2DM) is a complex disease in which multiple organs and hormones contribute to the pathogenesis of disease. The intestinal hormone, glucagon-like peptide-1 (GLP-1), secreted in response to nutrient ingestion, increases insulin secretion from pancreatic β-cells and reduces glucagon...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-015-0262-9
更新日期:2015-11-01 00:00:00
abstract::In the past 15 years, improvements in the treatment of HIV infection have dramatically reduced morbidity and mortality. Nucleoside reverse transcriptase inhibitors are the backbone of combination antiretroviral therapy for the treatment of HIV. One of the recommended and commonly used therapies in this class is the on...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-010-0006-9
更新日期:2010-01-01 00:00:00
abstract:INTRODUCTION:The European Medicines Agency (EMA) requires vaccine manufacturers to conduct enhanced safety surveillance (ESS) of seasonal influenza vaccines including a near real-time evaluation of collected data. The objective was to identify whether the use of passive surveillance or active surveillance provides diff...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-018-0747-4
更新日期:2018-08-01 00:00:00
abstract::The present study, which was designed as a prospective, double-masked, randomized, controlled, single-site study, was conducted to compare the effects of 3 approved ophthalmic nonsteroidal anti-inflammatory drugs-nepafenac ophthalmic suspension 0.1% (Nevanac; Alcon Laboratories, Inc., Fort Worth, Tex), ketorolac trome...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/BF02877774
更新日期:2007-11-01 00:00:00