Health Disparities in Rheumatology in the United States.

Objective: Underserved populations are often at risk of experiencing systematic healthcare disparities. Existing disparities in care access, quality of care received, and treatment outcomes among patients with rheumatic disease are not well understood.

Methods: We conducted a targeted literature review to understand disparities in health outcomes, treatment patterns, and healthcare management faced by rheumatology patients in the United States, with a focus on rheumatoid arthritis (RA), psoriatic arthritis (PsA), and ankylosing spondylitis (AS).

Intra-articular Hyaluronic Acid for Osteoarthritis of the Knee in the United States: A Systematic Review of Economic Evaluations.

Background: The economic impact of intra-articular hyaluronic acid (IAHA) for the treatment of knee pain associated with osteoarthritis (OA) has been evaluated in the United States, but not systematically summarized.

Objective: We reviewed the literature to determine the economic impact of IAHA for pain associated with knee OA in the United States.

Methods: A literature review was performed in PubMed (including MEDLINE and MEDLINE In-Process), Embase, the Cochrane Database of Systematic Reviews, and National Health Service Economic Evaluation Database and was limited to English language human studies published from January 2000 to October 2020.

Natural History and Disease Burden of Neurofibromatosis Type 1 with Plexiform Neurofibromas: A Systematic Literature Review.

Neurofibromatosis type 1 (NF1) is an incurable genetic condition that frequently includes the development of plexiform neurofibromas (PNs) in patients. A systematic literature review was conducted to identify data on the natural history, disease burden, and treatment patterns among patients diagnosed with NF1 and PN, as well as to identify evidence gaps in these areas. MEDLINE and MEDLINE In-Process, Embase, and Cochrane Library Searches were searched using predefined terms.

Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs.

The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as health-related quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients.

Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology.

The complement system is part of the innate immune response system, which comprises more than 50 distinct plasma and serum proteins that interact to opsonize pathogens (i.e., mark pathogens for destruction) and induce inflammatory responses to fight infection.

Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease.

Patients with paroxysmal nocturnal hemoglobinuria (PNH) often experience a lengthy path to diagnosis. Fewer than 40% of patients with PNH receive a diagnosis within 12 months of symptom onset, and 24% of all PNH diagnoses can take 5 years or longer. Diagnostic delay is a source of distress and can affect emotional well-being for patients with PNH.

Systematic review of sequencing of ALK inhibitors in -positive non-small-cell lung cancer.

The objective of this study was to understand outcomes of patients treated with ALK inhibitors, especially when ALK inhibitors are followed by other ALK inhibitors. A systematic literature review was conducted in PubMed, Embase, and Cochrane through July 17, 2017. Conference abstracts (three meetings in past 2 years) also were searched.

Direct costs associated with adverse events of systemic therapies for advanced melanoma: Systematic literature review.

Background: Treatments for advanced melanoma are associated with different adverse events (AEs), which may be costly to manage. This study aimed to evaluate direct costs associated with managing treatment-related AEs for advanced melanoma through a systematic literature review.

Methods: Systematic searches were conducted of the PubMed, Embase, Cochrane, BIOSIS, and EconLit medical literature databases to identify studies providing estimates of direct costs and health care resource utilization for the management of AEs of melanoma treatments, published between January 1, 2007, and February 23, 2017.

The epidemiology of medically attended respiratory syncytial virus in older adults in the United States: A systematic review.

Objective: This review was undertaken to assess the historical evidence of the disease incidence and burden of laboratory-confirmed respiratory syncytial virus (RSV) in medically attended older adults.

Design: A qualitative systematic literature review was performed; no statistical synthesis of the data was planned, in anticipation of expected heterogeneity across studies in this population.

Methods: A literature search of PubMed, Embase, and the Cochrane Library was conducted for studies of medically attended RSV in older adults (≥ 50 years) published in the last 15 years.

The Patient Experience with Soft Tissue Sarcoma: A Systematic Review of the Literature.

Background: Soft tissue sarcomas (STS) are a heterogenous group of rare tumors that involve the connective tissue in the body (e.g. muscle, tendons).

Impact of Measuring Patient-Reported Outcomes in Dermatology Drug Development.

Although some symptoms of dermatologic diseases, such as pruritus and pain, can be subjectively assessed only by patients, the most commonly used endpoints in dermatology drug research traditionally have been clinician-reported outcomes. Research has found that patient-reported outcomes (PROs) were included in only one-quarter of 125 trials conducted between 1994 and 2001. Our objective was to characterize the impact of PROs in dermatology drug development from the patient, prescriber, regulator, payer, and manufacturer perspectives using a case study approach.

Utility estimates for patients with Type 2 diabetes mellitus after experiencing a myocardial infarction or stroke: a systematic review.

A systematic review identified studies eliciting utility decrements from myocardial infarction (MI) and stroke in patients with Type 2 diabetes mellitus (T2DM) and examined their use in economic models of new diabetes treatments. In 16 utility studies in patients with T2DM, utility decrements in the first year ranged from 0.017 to 0.

Factors affecting the health-related quality of life of patients with cervical dystonia and impact of treatment with abobotulinumtoxinA (Dysport): results from a randomised, double-blind, placebo-controlled study.

Objective: To describe the health-related quality of life (HRQOL) burden of cervical dystonia (CD) and report on the HRQOL and patient perception of treatment benefits of abobotulinumtoxinA (Dysport).

Design: The safety and efficacy of a single injection of abobotulinumtoxinA for CD treatment were evaluated in a previously reported international, multicenter, double-blind, randomised trial. HRQOL measures were assessed in the trial and have not been previously reported.

Symptoms and toxicity of rituximab maintenance relative to observation following immunochemotherapy in patients with follicular lymphoma.

Objectives: The randomized phase 3 PRIMA trial established that 2 years of rituximab maintenance therapy after attaining disease response to immunochemotherapy as first-line treatment of follicular lymphoma, reduced the risk of disease progression, compared with observation, without adversely affecting patient-reported quality of life (QoL). We now report additional analyses of symptom burden and toxicity.

Methods: Symptom burden was measured by the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 items.

Patient-reported outcome labeling claims and measurement approach for metastatic castration-resistant prostate cancer treatments in the United States and European Union.

Background: Metastatic castration-resistant prostate cancer (mCRPC) and its treatment significantly affect health-related quality of life (HRQOL). Our objectives were to evaluate and compare patient-reported outcome (PRO) claims granted by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for 5 recently approved mCRPC treatments and to examine key characteristics, development, and measurement properties of the PRO measures supporting these claims against current regulatory standards.

Methods: Five products approved for treatment of mCRPC by the FDA and the EMA (2010-2013) were examined: enzalutamide, abiraterone, sipuleucel-T, cabazitaxel, and radium Ra 223 dichloride.

Incremental costs associated with myocardial infarction and stroke in patients with type 2 diabetes mellitus: an overview for economic modeling.

Objective: To identify cost estimates related to myocardial infarction (MI) or stroke in patients with type 2 diabetes mellitus (T2DM) for use in economic models.

Methods: A systematic literature review was conducted. Electronic databases and conference abstracts were screened against inclusion criteria, which included studies performed in patients who had T2DM before experiencing an MI or stroke.

Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy.

Patients with myelofibrosis (MF) have significant debilitating symptoms, physical disabilities, and poor health-related quality of life (HRQoL). Here, we report post-hoc analyses of the impact of ruxolitinib, a potent and selective JAK1 and JAK2 inhibitor, on disease-related symptoms and HRQoL in MF patients from the large phase 3 COMFORT-II study (N = 219). During the follow-up period of 48 weeks, HRQoL and MF-associated symptoms improved from baseline for ruxolitinib-treated patients but remained the same or worsened for best available therapy (BAT)-treated patients.

Evidence-based medicine, appropriate-use criteria, and sports medicine: how best to develop meaningful treatment guidelines.

We propose using appropriate-use criteria (AUC) as the methodology of choice for formulating and disseminating evidence-based medicine guidelines in sports medicine and arthroscopy. AUC provide a structured process for integrating findings from the scientific literature with clinical judgment to produce explicit criteria for determining the appropriateness of specific treatments. The use of AUC will enable surgeons to treat patients in a more consistent manner based on expert clinical consensus and evidence-based medicine.

Do different clinical evidence bases lead to discordant health-technology assessment decisions? An in-depth case series across three jurisdictions.

Background: Health-technology assessment (HTA) plays an important role in informing drug-reimbursement decision-making in many countries. HTA processes for the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia, the Common Drug Review (CDR) in Canada, and the National Institute for Health and Clinical Excellence (NICE) in England and Wales are among the most established in the world. In this study, we performed nine in-depth case studies to assess whether different clinical evidence bases may have influenced listing recommendations made by PBAC, CDR, and NICE.

High burden and unmet patient needs in chronic kidney disease.

Chronic kidney disease (CKD) is a complex debilitating condition affecting more than 70 million people worldwide. With the increased prevalence in risk factors such as diabetes, hypertension, and cardiovascular disease in an aging population, CKD prevalence is also expected to increase. Increased awareness and understanding of the overall CKD burden by health care teams (patients, clinicians, and payers) is warranted so that overall care and treatment management may improve.

Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010.

Objectives: Previous analyses of patient-reported outcome (PRO) label claims concentrated only on successful label claims. The goal of this research was to explore the reasons why PRO label claims were denied and to compile regulatory feedback regarding the use of PROs in clinical trials.

Methods: By using the Food and Drug Administration's Drug Approval Report Web page, all new molecular entities and biologic license applications approved between January 2006 and December 2010 were identified.

A review of patient-reported outcome labels in the United States: 2006 to 2010.

Objective: In 2004, Willke and colleagues reviewed the efficacy endpoints reported in the labels of new drugs approved in the United States from 1997 through 2002 to evaluate the use of patient-reported outcome (PRO) endpoints. Of the labels reviewed, 30% included PROs. Our study aimed to build on this work by describing the current state of PRO label claims granted for new molecular entities (and biologic license applications since February 2006 after the release of the US Food and Drug Administration (FDA) draft PRO guidance.

Differences among formulary submission guidelines: implications for health technology assessment.

Objectives: This article provides a detailed understanding of the differences in selected formulary submission guidelines supplied by various health technology assessment (HTA) agencies and indicates how these differences can impact the evidence base used to populate the HTA.

Methods: Detailed summaries of the recommended methods for evidence generation, organized by topic areas relevant for clinical and economic data, for twelve countries in Europe, North America, and Australia where HTA processes are well developed were prepared. Using these summaries, we provide examples of the likely impact these differences in recommended methods could have on the evidence base used to evaluate new health technologies.

Preliminary development of a responder index for chronic low back pain.

Objective: One of the greatest obstacles to identifying the most effective therapy for chronic low back pain (CLBP) is the lack of standardized outcome measures for assessing treatment effect in clinical trials. The aim of the OMERACT Special Interest Group was to discuss the development and validation of a preliminary responder index in CLBP.

Methods: Patient data from 5 clinical trials of celecoxib and valdecoxib use in CLBP were used to assess the reliability and validity of multiple items in the outcome domains of pain, functioning, and overall impression of health.

Are chemotherapy patients' HRQoL importance weights consistent with linear scoring rules? A stated-choice approach.

Objective: To compare a linear scoring rule with the subjective importance of different domain and symptom levels of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) among patients undergoing chemotherapy.

Methods: Using a stated-choice or choice-format conjoint analysis survey instrument, we elicited patient preferences for varying levels of physical, role, social, emotional, and cognitive function along with chemotherapy-related side effects and financial difficulties. A total of 375 patients completed the questionnaire: 159 breast cancer, 117 colorectal cancer, 99 non-small-cell lung cancer; and 21 with unknown tumor type.