Federal Advisory Committees in the United States:
Estimating the Outpatient Burden of Venous Thromboembolism in the United States: An Analysis of Google Trends Data from 2004 to 2015
Abstract Background: Analysis of internet search traffic has provided a new proxy measure into the trends and patterns of patients' diseases and their information-seeking behaviors. In recent years, Google Trends has become a data resource of interest given its status as the largest internet search provider in the world with publicly-viewable, passively-collected, and expansive data on any searchable term or combination of terms. For instance, search terms related to influenza (e.g. fever) predicted influenza spread faster than standard surveillance as shown by Ginsberg et al in Nature 2009. The true outpatient burden and incidence of venous thromboembolism (VTE) has long been debated. Extant VTE data are limited to cases that present to medical attention, thus missing any cases that do not come to an emergency department or clinic. We hypothesized that Google Trends analysis offers potential insight into the general populations' blood clot burden and awareness. This study aimed to explore general trends of VTE-related terms and seasonal variation in searches. Methods: Google Trends was utilized to obtain relative search engine traffic values (defined as search volume indices, SVIs) for terms related to DVT in the United States from summer 2004 - winter 2015. Terms related to LEG SWELLING, CALF PAIN, VARICOSE VEINS, and LEG CLOT were used and combined with Boolean operators to combine similar terms. A separate search occurred for BLOOD CLOT and related terms to investigate awareness of VTEs. Analysis was performed in R (V3.1.1) in accordance with previously published Google Trends investigations. Results: The average relative volume of searches was highest for VARICOSE VEINS and lowest for LEG SWELLING by approximately 3.2 fold. A seasonal pattern was seen with summer months (May-Aug) having higher SVIs than winter months (Nov-Feb) for all terms in the 11 year study period except for BLOOD CLOT. Using a Wilcoxon signed rank test, mean SVI difference comparing summer to winter for LEG SWELLING showed W = 66 (p = 0.004), for CALF PAIN W = 66 (p = 0.003), for VARICOSE VEINS W = 67 (p = 0.004), and for LEG CLOT W = 65 (p = 0.005). For BLOOD CLOT, a gradual increase in SVIs was seen and characterized by a Mann-Kendall test as having a significant positive trend, S = 898, p = 0.024. Conclusions: Search terms related to VTE in the United States show a strong seasonal pattern with greater search activity in summer months compared to winters months. These data suggest a higher incidence and burden of VTE in the summer. This challenges previous notions of a weakly higher incidence of VTE in winter months, calculated as a relative risk of 1.143 by Dentali et al in 2011. The gradual increase in relative search traffic for BLOOD CLOT terms reflects a likely rising awareness and/or true rise in the incidence of VTEs in the United States from 2004-2015. Further studies should investigate whether internet search traffic correlates directly with total yearly DVT incidence rates. Keywords: Population, venous thromboembolism, incidence Figure 1. Figure 1. Figure 2. Figure 2. Disclosures Khorana: Leo Pharma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Daiichi Sankyo: Consultancy, Honoraria; sanofi: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Boehringer-Ingelheim: Consultancy, Honoraria.
Abstract Background Protein C (PC) is a naturally occurring, vitamin-K dependent anticoagulant produced by the liver. Deficiency of PC, which can be congenital or acquired, results in a hypercoagulable state. Patients with severe PC deficiency may manifest severe, often life-threatening, disseminated intravascular coagulation (DIC), purpura fulminans (PF), and/or thromboembolism. Highly purified protein C concentrate prepared from human donor plasma (Ceprotin; Baxter Healthcare Corporation, Westlake Village, CA) is an approved therapy for prevention and treatment of venous thrombosis and PF in patients with severe congenital PC deficiency (SCPCD) in the United States. It is indicated in patients with SCPCD for PF and coumarin-induced skin necrosis, and short-term prophylaxis in the European Union and other countries. Use of protein C concentrate (human) has been reported in patients with acquired PC deficiency. The Ceprotin Treatment Registry is a prospective, international, multi-center, open-label, non-interventional, observational study designed to examine the long-term safety and effectiveness of protein C concentrate (human) in the clinical setting. This is the first large, real-world assessment of the treatment of a rare disease with protein C concentrate (human). Here we report data from the first interim analysis in June 2013 covering a 3-year enrollment period. Methods Patients of any age who received, or were initiating/receiving protein C concentrate are included. The study duration is 5 years (3-year enrollment period plus 2 years of follow-up). All study visits/assessments are in accordance with standard of care, with protein C concentrate dose, dose frequency, duration and route of administration determined by the investigator. The study objectives are to determine the most common medical diagnoses associated with protein C concentrate treatment, protein C concentrate treatment regimens, and safety information based on all serious adverse events (SAEs) and rate of treatment-related AE. Other objectives include: an examination of the relationship between protein C concentrate treatment and outcomes in all registry participants, as well as various subgroups. Descriptive statistical analyses are used. Results At the time of data extraction, 34 patients were enrolled from 26 centers; 10 centers in the United States and 16 centers in Europe. Half of the patients were males. The primary diagnosis of PC deficiency was congenital in 25 patients (73.5%) and acquired in 9 patients (26.5%). The median age at diagnosis was 0.03 years (range 0–19.9). Mean plasma PC activity level was 9% (range 1–40.0). The most common (in ≥3 patients) thrombotic disease-associated conditions were PF (50.0%); blindness (44.1%); thromboembolic disease (41.2%), which included deep vein thrombosis, arterial thrombosis and DIC; stroke (32.4%), and renal dysfunction (8.8%). Of the 23 patients being treated with protein C concentrate, 15 were administered protein C concentrate intravenously, and 8 patients received protein C concentrate subcutaneously. The body weights of patients receiving subcutaneous treatment ranged from 10.0 kg to 57.9 kg. A total of 15 patients received an anticoagulant treatment in addition to protein C concentrate. Eight patients reported 23 SAEs, all of which were considered not related to treatment. Eighteen (52.9%) patients reported 72 AEs; only 1 of them, an episode of upper respiratory infection, was considered possibly treatment-related. In 18 patients in whom PC activity recovery was determined after protein C concentrate treatment, there were no patients documented with poor recovery. Protein C concentrate was used during 18 surgeries/invasive procedures and considered effective in all interventions for which data were available. Conclusions Data from the first interim analysis of the Ceprotin Treatment Registry demonstrate that patients with both congenital and acquired severe PC deficiency who are treated with protein C concentrate (human) have a low incidence of treatment related SAEs and AEs, and treatment with protein C concentrate appears to be effective when used during surgery/invasive procedures. Further patient follow up will shed light onto clinical treatment outcomes. Disclosures: Manco-Johnson: CSL Behring: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Novo Nordisk: Membership on an entity’s Board of Directors or advisory committees; Biogen Idec: Membership on an entity’s Board of Directors or advisory committees; Baxter BioScience: Membership on an entity’s Board of Directors or advisory committees; Bayer HealthCare: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Eisai: Research Funding. Knoebl:Novo Nordisk: Consultancy, Honoraria; Baxter: Consultancy, Honoraria. Shapiro:Cangene: Research Funding; Biogen Idec: Research Funding; Baxter BioScience: Research Funding; Bayer HealthCare: Research Funding. Finnerty:Baxter: Employment, Equity Ownership. Yel:Baxter: Employment, Equity Ownership. Gelmont:Baxter: Employment, Equity Ownership.
Conflicts of interest permeate the governance of the federal advisory committees that issue recommendations to consumer protection agencies, such as the Food and Drug Administration (FDA) and the United States Department of Agriculture (USDA), and therefore, American consumers need a federal solution to protect their health from biased recommendations. In order to promote a business-friendly food pyramid, agribusinesses and food industrialists lobby for dietary guidelines that boost their sales. The resulting guidelines cause great damage to public health, spur environmental pollution, and result in a loss of democratic freedoms. As a result, the FDA and USDA's bifurcated task of protecting both food producers and consumers, creates a conflict of interest within the agencies that often favor the food industry over consumer protection.This paper describes the problems embedded within the FDA and USDA's conflict of interest and the resulting revolving door of the heavily invested lobbyists, and finally, suggests statutory amendments to solve this problem. The proposed amendments will dispense with ineffective disclosure requirements and eliminate the possibility of waiving conflicts of interest for advisory committee members. By rebalancing the composition of the advisory committees and the scientific basis for the dietary recommendations, the proposed amendments will close the loopholes that large food industrialists currently abuse. As a result, consumer protection agencies, such as the FDA and USDA, are empowered to police the federal advisory committees issuing the dietary recommendations and prevent government officials from breaching their fiduciary duties to American consumers.
Abstract Introduction: Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm characterized by clonal platelet production and an increased risk for thrombotic and hemorrhagic events. Limited real-world data exist regarding the clinical characteristics and treatment patterns of ET in the United States; most prior data have been generated outside the United States. The Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST) is an ongoing observational study being conducted to describe contemporary demographics, burden of disease, clinical management, and patient-reported outcomes in patients with specific risk categories of myelofibrosis (MF) or ET in the United States. This analysis describes the clinical characteristics of patients with ET currently enrolled in MOST. Methods: MOST is a multicenter, noninterventional, prospective, observational study in patients with a clinical diagnosis of specific risk categories of MF or ET (NCT02953704). Eligible patients with ET included high-risk patients (≥ 60 years of age and/or a history of thromboembolic events [TEs]), or low-risk patients currently receiving ET-directed therapy. Key exclusion criteria included participation in blinded investigational drug studies, life expectancy ≤ 6 months, or diagnosis of other malignancy. Data regarding disease and clinical characteristics are collected at usual-care visits over a planned 36-month observation period. Patient demographics and clinical characteristics at enrollment were described with descriptive statistics. Results: At data cutoff (May 18, 2018), 793 eligible patients were enrolled from 85 sites since November 29, 2016. The median age at enrollment was 70 (range, 24-95) years, 66.5% were female, and 89.8% were Caucasian. The median time from ET diagnosis to enrollment was 4.2 (range, 0.0-42.1) years with a proportion of patients diagnosed within 1 year (19.5%), 1 to < 5 years (35.0%), 5 to < 10 years (21.7%), or ≥ 10 years (23.8%) of enrollment. Approximately 40% of patients were retired and 42.7% were working full- or part-time at enrollment. A total of 212 patients (26.7%) had a history of TE at the time of enrollment. The type of TE was available for 148 patients, the most common was arterial events (53.4%); 33.1% had venous, and 13.5% of patients had both arterial and venous events. Six hundred and eighty-eight patients (86.8%) were classified as high-risk. Assessments at the time of ET diagnosis, among evaluable patients, included bone marrow biopsy (51.4%; 393/765) and mutational testing (77.2%; 590/764). Three hundred and forty-nine patients had mutation test results reported at the time of diagnosis; of patients with JAK2 V617F test results reported at the time of diagnosis (n = 313), 78.6% were positive for JAK2 V617F (Table 1). Laboratory values and peripheral blood counts were reported for patients with available data (Table 2). The majority of patients (87.9%) had received at least 1 ET-directed therapy prior to enrollment, which in some cases was the same medication the patient was receiving at the time of enrollment. At the time of enrollment, 740 patients (93.3%) were receiving at least 1 current ET-directed therapy, including HU (71.6%; 530/740), anagrelide (13.1%; 97/740), ruxolitinib (4.7%; 35/740), interferon (3.0%; 22/740), and busulfan (0.3%; 2/740). Of 793 patients, the most frequently occurring relevant comorbid conditions were hypertension (52.7%), history of smoking (44.7%), and hyperlipidemia (24.1%). Among 761 patients with ET-related symptoms assessed at diagnosis, the most common symptoms documented by healthcare providers included constitutional (22.9%), vasomotor (16.0%), and spleen-associated symptoms (3.9%), and pruritus (2.6%). Conclusions: Prior real-world data in ET has predominately been generated outside of the United States or has been reported from single institutional experiences. The MOST study will provide a more complete picture of the patient characteristics and outcomes of patients receiving ET-directed therapy in the United States. Ultimately, these data will be important for determining ET treatment gaps and areas of unmet need. Disclosures Yacoub: Cara Therapeutics: Equity Ownership; Inycte: Honoraria, Speakers Bureau; Novartis: Honoraria, Speakers Bureau; Ardelyx, INC.: Equity Ownership; Seattle Genetics: Honoraria, Speakers Bureau; Dynavax: Equity Ownership. Verstovsek:Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Italfarmaco: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees; Incyte: Consultancy. Shao:ASH: Membership on an entity's Board of Directors or advisory committees; ASCO: Membership on an entity's Board of Directors or advisory committees. Agrawal:Incyte: Speakers Bureau. Sivaraman:Incyte: Employment. Colucci:Incyte: Employment, Equity Ownership. Yao:Incyte: Employment. Mascarenhas:Celgene: Membership on an entity's Board of Directors or advisory committees; Promedior: Research Funding; CTI Biopharma: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding; Merck: Research Funding; Incyte: Membership on an entity's Board of Directors or advisory committees, Research Funding; Roche: Research Funding; Janssen: Research Funding.
National advisory committees have considered the obligations owed to research participants in the event of research-related injuries. These committees have repeatedly concluded that injured research participants are entitled to compensation for their injuries, that the tort system provides inadequate remedies, and that the United States should adopt no-fault compensation. But because the advisory committees have made no concrete proposals and have taken no steps toward implementing no-fault compensation, the United States continues to rely on the tort system to compensate injured research participants.This Article argues that recent legal developments and a transformation in the global research landscape make maintaining the status quo morally indefensible and practically unsustainable. Recent legal developments exacerbate the longstanding difficulties associated with the tort system as a method of compensation; nearly every injured research participant will have difficulty recovering damages, and certain classes of injured research participants—those in federal research and those abroad—are prevented from recovering altogether, resulting in substantial unfairness. In the past ten years, many of the countries substantially involved in research have mandated systematic compensation. By not mandating compensation, the United States has become a moral outlier and risks having its noncompliant research embargoed by foreign ethics committees, thereby delaying important biomedical advances.This Article examines alternative compensation mechanisms and offers a concrete no-fault compensation proposal built on systems already in place. The proposed system can be implemented in the United States and countries around the world to help harmonize various national compensation systems and to more equitably and effectively make those injured by research whole.
Abstract Introduction Black (B) patients with Mycosis fungoides and Sézary Syndrome (MF/SS) have inferior survival and distinct clinical presentations, including many high risk features compared to non-black (NB) patients with MF/SS in large registry studies from the United States. We sought to characterize clinical differences in presentation, treatment, and outcomes to identify drivers of disparities among B patients with MF/SS. Methods We performed a retrospective review of 417 patients with MF/SS diagnosed between 1990 and 2020 at 6 academic institutions in the United States that serve populations with large numbers of Blacks and minorities. Patients with pathologic review and a confirmed histopathologic diagnosis of MF or SS with a stage >1B as their highest TNMB stage were eligible for inclusion. The primary objective was to assess differences in clinical characteristics, treatment patterns, and survival between B and NB patients. Clinical variables included demographics, insurance, zip code, tumor characteristics, and treatment. Descriptive analysis was performed for each variable. Comparison between B and NB patients utilized ANOVA for numerical covariates and chi-square test or Fisher's exact test for categorical covariates. Kaplan-Meier curves for OS were generated, and subgroups were analyzed separated. Results Among 417 patients, 204 (48.9%) were Blacks, 196 (47.0%) were White, and (4.1%) were other races (8 Asian, 5 unknown, 4 Hispanic). The diagnosis was MF in 366 (87.8%), SS in 47 (11.3%), and other 4 patients. Stage at diagnosis was available for 384 patients. 37 (9.6%) were stage 1A, 165 (43%) were stage 1B, 34 (8.9%) were stage 2A, 58 (15.1%) were stage 2B, 44 (11.5%) were stage 3, and 46 (12%) were stage 4 at diagnosis. The median survival of the whole cohort (n=417) was B patients were younger (median 50.1 years in B vs. 60.5 in NB, p<0.001), more often female (49.5% in B; 35.7% in NB, p=0.004), had higher rates of reported lymphadenopathy (78.5% vs. 70%, p=0.039), longer delays from symptom onset to diagnosis (2.0 years in Black vs. 1.2 years in NB, p=0.014), and higher rates of bacteremia at any time in their disease course (19.7% in Black, vs. 6.6% in NB, P< 0.001) compared to NB patients. There was no difference in insurance status (private, Medicare/Medicaid, or uninsured) or comorbidities by race. There was also no differences in overall TNMB stage at diagnosis (p=0.14) compartment stage, or highest stage (p=0.076). Progression to a higher T, N, M, or B stage occurred at similar frequencies in B and NB patients (n=83 (43.2%) in Black and n=73 (38.8%) in NB, p=0.38). Time to first therapy, time to systemic therapy, or total lines of therapy also did not differ by racial group. A greater fraction of B compared to NB patients received systemic therapy but this did not reach statistical significance (72.6% in Black vs. 62% in NB, p=0.060). There was no difference in large cell transformation at any point, or white blood cell count (WBC), lactate dehydrogenase (LDH), or T-cell receptor (TCR) clonality in skin/blood at diagnosis. On univariate analysis, race was not associated with survival; the median survival was 10.5 years (95% CI 8-13.2) in B patients and 10.9 years (95% CI 7.2-14.1) in NB patients. Covariates associated with inferior survival included older age (p<0.001), number of comorbidities (p< 0.001), bacteremia (p< 0.001), higher overall TNMB stage, higher individual T, N, M and B stage, progression to a higher N or M stage, positive TCR in the blood, WBC value, and LDH value. Insurance status and year of diagnosis (prior to 2010, vs. following 2010) were not associated with survival. On multivariate cox proportional hazard model, age at diagnosis, comorbidities, extra-cutaneous disease, bacteremia, TNMB stage at diagnosis, and progression to a higher nodal stage remained statistically significant for survival. Conclusions We present the first multicenter analysis of racial disparities in MF/SS, with nearly 50% B patients. B patients had distinct presentations, delay in diagnosis, and higher rates of bacteremia compared to NB patients. In contrast to US-registry studies, there was no difference in survival between B and NB patients. This finding could be explained by access to academic centers and/or higher rates of insurance coverage among B patients in our cohort. Additional analyses will be updated at the time of the presentation. Figure 1 Figure 1. Disclosures Allen: ADC Therapeutics: Consultancy; Kyowa Kirin: Consultancy; Secure Bio: Consultancy. Mehta: Seattle Genetics; Incyte; TG Therapeutics: Consultancy; Seattle Genetics; Incyte; TG Therapeutics: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Affirmed; Kite/Gilead; Roche-Genetech; Celgene/BMS; Oncotartis; Innate Pharmaceuticals; Seattle Genetics; Incyte; Takeda; Fortyseven Inc/Gilead; TG Therapeutics; Merck; Juno Pharmaceuticals/Bristol Myers Squibb: Research Funding. Huen: Rhizen: Research Funding; Elorac: Research Funding; Kyowa Kirin: Research Funding; Tillium: Research Funding; Innate: Research Funding; Galderma: Research Funding; Miragen: Research Funding. Porcu: Viracta: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Innate Pharma: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; BeiGene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Incyte: Research Funding; Daiichi: Honoraria, Research Funding; Kiowa: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Spectrum: Consultancy; DrenBio: Consultancy.
BACKGROUND: The American Board of Internal Medicine (ABIM) offers separate board examinations for adult hematology and medical oncology, yet the vast majority of fellowship training programs in the United States are structured as combined hematology/oncology programs. Single-board hematology tracks or programs may help increase recruitment and retention in non-malignant hematology; however, the barriers and attitudes toward hematology-only fellowship programs are unknown. We administered a survey to ACGME-accredited hematology/oncology program directors to explore their perceptions toward training in non-malignant hematology and their attitudes toward single-board hematology training. METHODS: In collaboration with the ASH Medical Educators Institute, we developed an anonymous online survey with 30 multiple-choice and open-ended questions to characterize attitudes toward specialization in non-malignant hematology and to assess program director interest and perceived barriers toward single-board hematology training. The survey was electronically administered to program directors of active hematology/oncology fellowship programs in the United States (n=139) in March/April 2019 using Qualtrics software. RESULTS: Of the 139 program directors who received the survey, 90 (65% response rate) completed the survey. The majority of program directors characterized their institutions as academic (87%), with only 9 (10%) describing their programs as community-based. Seventy-eight (87%) program directors believed that there is a shortage of exclusive non-malignant hematologists in the United States, and 59% felt that training more fellows to practice exclusive non-malignant hematology could help address the shortage (Table 1). Jobs for for exclusive non-malignant hematologists were perceived to exist only in academic settings by 47 (52%) respondents. In terms of fellowship training, program directors reported that an average of 5% of fellows per program pursued a career largely or exclusively focused on non-malignant hematology. In addition, 39 (43%) program directors felt that fellows were dissuaded from pursuing a career exclusively in non-malignant hematology. Regarding single-board hematology training, 73% of program directors believe that hematology-only training is both necessary and sufficient for fellows specializing in non-malignant hematology. The most commonly perceived barriers to single-board hematology fellowship programs were: 1) concerns for job availability for single-board hematology trainees, 2) concerns about limiting the training option to hematology only, and 3) lack of interested applicants to the program (Table 2). If barriers were addressed, 37% of programs directors reported that they would be interested in implementing a single-board hematology track at their institution. CONCLUSIONS: Combined hematology/oncology fellowship training is nearly exclusive to the United States. Our survey demonstrates that the percentage of fellows specializing in non-malignant hematology is significantly low (5%), a number that remains unchanged compared to a prior ASH program director survey in 2003. Our results also suggest that programs directors are interested in training fellows in non-malignant hematology and that single-board hematology training is generally acceptable among program directors. It will be important to address perceived barriers to hematology-only programs in order to promote implementation. Disclosures Naik: Elsevier: Other: Content Editor. Nagalla:Alnylam: Membership on an entity's Board of Directors or advisory committees.
Advisory committees are part and parcel of policy-making in every government agency. Their input is used at many levels, ranging from high level strategic initiatives and planning to very specific input into narrowly defined decisions. This chapter describes an operational overview of the different approaches used by science agencies in the United States and documents the heterogeneous way in which they are used. It suggests that some of the principles that motivated the establishment of advisory committees are not always fully served in practice. It also finds that that the financial structure and incentives of some entities, might usefully be reviewed. The chapter concludes by observing that the structure of committees might be improved by developing a more scientific approach to their constitution.
Abstract Background: Daratumumab (dara) is a human CD38-directed monoclonal antibody indicated for the treatment of patients (pts) with multiple myeloma (MM) who have received ≥3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMID) or who are double-refractory to a PI and an IMID. Accelerated approval was granted in the United States (US) in November 2015, based largely on the results of MMY2002, a pivotal phase 2 study in this pt population. Methods: The objectives of this multicenter, open-label early access treatment protocol (EAP) were to provide early access to dara treatment and collect safety and patient-reported outcome (PRO) data in this pt population. Eligibility criteria were similar to those of pivotal study MMY2002 and included age ≥18 years, documented MM, progression by IMWG criteria following the most recent therapy, ≥3 prior lines of therapy including a PI and an IMID or disease double-refractory to a PI and an IMID, ECOG performance status score 0-2, no known chronic obstructive pulmonary disease or persistent asthma, no ongoing MM therapy, and no prior exposure to anti-CD38 antibody therapy. Pts received dara 16 mg/kg IV weekly for 8 weeks, then every 2 weeks for 16 weeks, and then every 4 weeks until disease progression, unacceptable toxicity, or 60 days after US approval. Pre- and post-infusion medications were administered as in study MMY2002. Serious adverse events (SAEs), grade 3-4 AEs, infusion related reactions (IRRs), and PRO data were collected. Results: In total, 400 pts were screened and 348 pts were enrolled and dosed at 39 US sites from July to November 2015. Median age was 65 (range 27-94) years; 72% were white, and 17% were African American. Three-fourths of pts were symptomatic with an ECOG score of 1 (58%) or 2 (16%). Pts received a median of 8 (range 1-17) doses, and median treatment exposure was 1.9 (range 0.03-6.0) months. Median durations of infusion were 7.4, 4.4, and 3.5 hours for the first, second, and all subsequent infusions, respectively. Half of pts (52%) transitioned to commercial drug after marketing authorization, whereas 37% discontinued due to progressive disease. Treatment emergent grade >3 AEs were reported in 51% of pts. The most common grade ≥3 AEs were thrombocytopenia (15%) and anemia (14%). SAEs occurred in 35% of pts, including 12% of pts with SAEs which were reported by investigators as drug-related. The most common SAEs were infections, which occurred in 11% of pts. Nine percent of pts discontinued therapy due to AEs, including 3% for drug-related AEs. Thirteen (4%) pts had an AE with a fatal outcome, including 2 (0.6%) pts with drug-related AEs (pyrexia, thrombocytopenia/ subdural hematoma). IRRs occurred in 56% of pts, including 8% with grade >3 IRRs. IRRs occurred in 56%, 2%, and 2% of first, second, and all subsequent infusions, respectively; the most common IRRs were respiratory or thoracic symptoms (cough, dyspnea, throat irritation, nasal congestion), which occurred in 31% of pts. The median change from baseline in all the domains of the EQ-5D-5L and EORTC QLQ-C30 scales after 1 and 2 cycles as well as at pts' last assessment was 0, with the exception of EQ-5D-5L VAS, which showed a median increase of 1 and 2 units after 1 and 2 cycles, respectively. Conclusions: EAP results in US pts confirmed the safety profile of dara in MM pts with >3 prior therapies including a PI and IMID or who were refractory to both PI and IMID. SAEs occurred in one-third of pts, but only 12% of pts experienced a drug-related SAE. More than half of pts experienced IRRs, which primarily occurred during the first infusion and were grade 1-2 in severity. Pts maintained their health-related quality of life during a median duration of 2 months of therapy. Disclosures Chari: Array Biopharma: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Janssen: Consultancy, Research Funding; Celgene: Consultancy, Research Funding; Amgen Inc.: Honoraria, Research Funding; Takeda: Consultancy, Research Funding; Pharmacyclics: Research Funding. Mark:Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Takeda: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Research Funding, Speakers Bureau; Bristol Myers Squibb: Speakers Bureau. Stockerl-Goldstein:Janssen: Speakers Bureau. Usmani:Novartis: Speakers Bureau; Pharmacyclics: Research Funding; Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Millenium: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Array: Research Funding; Skyline: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Britsol-Myers Squibb: Consultancy, Research Funding; Onyx: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Amgen: Consultancy, Research Funding, Speakers Bureau; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding; BioPharma: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Londhe:Janssen Scientific Affairs, LLC: Employment. Etheredge:Janssen Research & Development, LLC: Employment. Parros:Janssen Research & Development, LLC: Employment. Fleming:Janssen Global Services, LLC: Employment. Liu:Janssen Research & Development, LLC: Employment. Freeman:Janssen Scientific Affairs, LLC: Employment. Ukropec:Janssen Scientific Affairs, LLC: Employment. Lin:Janssen Scientific Affairs, LLC: Employment. Lonial:BMS: Consultancy; Novartis: Consultancy; Celgene: Consultancy; Janssen: Consultancy; Onyx: Consultancy; Merck: Consultancy; Onyx: Consultancy; Janssen: Consultancy; BMS: Consultancy; Celgene: Consultancy; Millenium: Consultancy; Novartis: Consultancy.
A Review of: Moulaison Sandy, H., & Dykas, F. (2016). High-quality metadata and repository staffing: Perceptions of United States–based OpenDOAR participants. Cataloging & Classification Quarterly, 54(2), 101-116. http://dx.doi.org/10.1080/01639374.2015.1116480 Objective – To investigate the quality of institutional repository metadata, metadata practices, and identify barriers to quality. Design – Survey questionnaire. Setting – The OpenDOAR online registry of worldwide repositories. Subjects – A random sample of 50 from 358 administrators of institutional repositories in the United States of America listed in the OpenDOAR registry. Methods – The authors surveyed a random sample of administrators of American institutional repositories included in the OpenDOAR registry. The survey was distributed electronically. Recipients were asked to forward the email if they felt someone else was better suited to respond. There were questions about the demographics of the repository, the metadata creation environment, metadata quality, standards and practices, and obstacles to quality. Results were analyzed in Excel, and qualitative responses were coded by two researchers together. Main results – There was a 42% (n=21) response rate to the section on metadata quality, a 40% (n=20) response rate to the metadata creation section, and 40% (n=20) to the section on obstacles to quality. The majority of respondents rated their metadata quality as average (65%, n=13) or above average (30%, n=5). No one rated the quality as high or poor, while 10% (n=2) rated the quality as below average. The survey found that the majority of descriptive metadata was created by professional (84%, n=16) or paraprofessional (53%, n=10) library staff. Professional staff were commonly involved in creating administrative metadata, reviewing the metadata, and selecting standards and documentation. Department heads and advisory committees were also involved in standards and documentation selection. The majority of repositories used locally established standards (61%, n=11). When asked about obstacles to metadata quality, the majority identified time and staff hours (85%, n=17) as a barrier, as well as repository software (60%, n=12). When the responses to questions about obstacles to quality were tabulated with the responses to quality rating, time limitations and staff hours came out as the top or joint-top answer, regardless of the quality rating. Finally, the authors present a sample of responses to the question on how metadata could be improved and these offer some solutions to staffing issues, the application of standards, and the repository system in use. Conclusion – The authors conclude that staffing, standards, and systems are all concerns in providing quality metadata. However, they suggest that standards and software issues could be overcome if adequate numbers of qualified staff are in place.