Real-World Treatment Patterns, Healthcare Resource Utilization and Associated Costs Among Patients with Chronic Myeloid Leukemia in Later Lines of Therapy

Introduction: Despite advances in tyrosine kinase inhibitor (TKI) therapy for chronic myeloid leukemia (CML), there remains a sizeable proportion of patients (pts) with CML in chronic phase who are refractory or intolerant to these agents. A good understanding of the most recent real-world patterns of care in these pts is necessary in order to provide context for assessing the potential benefits of new treatments undergoing clinical development. The aim of this study was to evaluate treatment patterns in CML pts cycling through TKI therapies achieving later lines of therapy, and to estimate their healthcare resource utilization (HRU) and costs. Methods: Adult pts with CML in the United States who received at least 3 lines of TKI therapy were identified in the IBM MarketScan Commercial and Medicare Supplement databases (Q1/2001-Q2/2019). Treatment patterns were observed from CML diagnosis. Pt characteristics were measured during the 6 months prior to third line (3L) initiation (baseline period). All-cause HRU (inpatient [IP] admissions and days, days with outpatient [OP] services, and emergency department [ED] visits) and costs (medical and pharmacy) were measured 1) during the course of 3L therapy (from 3L initiation to termination) and 2) between 3L initiation and end of follow-up (stem cell transplant [SCT], or end of data availability/health plan coverage). HRU was reported using monthly incidence rates per 100 pts (IR/100pts); costs (2019 USD) were evaluated per-pt-per-month (PPPM) from a payer's perspective. Results: Of the 48,168 pts identified with CML, a total of 296 CML pts initiated 3L therapy; median age was 58.5 years (30% were aged ≥65 years) and 50% were female. The mean follow-up period from CML diagnosis was 57.8 months and from 3L initiation was 24.5 months. Most pts had their first CML diagnosis in or after 2010 (71%) and achieved 3L in or after 2014 (50%). At baseline, the mean modified Charlson Comorbidity Index score (excluding CML) was 1.6 with 24% of pts with a score ≥3, 64% of pts had a moderate or severe Darkow Disease Complexity Index, and the most prevalent comorbidities were hypertension (45%) and diabetes (25%); 21% of pts (i.e., ≥1 indicator of congestive heart failure, cirrhosis, or end-stage renal disease, or ≥75 years old). The most common sequences of TKIs from first line (1L) to 3L were imatinib → dasatinib → nilotinib (28%), imatinib → nilotinib → dasatinib (16%), imatinib → dasatinib → imatinib (9%), and dasatinib → imatinib → nilotinib (5%). The mean duration of 1L, second line (2L), and 3L therapy was 14.9, 10.4, and 15.6 months, respectively; 62% of pts were still in 3L at the end of follow-up. Only one patient received SCT after 3L. The most common TKIs received at each line were imatinib in 1L (65%), dasatinib in 2L (49%), and nilotinib in 3L (36%). The mean treatment-free period (time between line of therapy termination and next line initiation) between 1L and 2L was 1.3 months, 2.6 months between 2L and 3L, and 1.5 months between 3L and 4L. During 3L therapy, pts had a monthly IR/100pts of 3.4 IP admissions, 21.2 IP days, 248.8 OP days, and 10.2 ED visits (Figure 1A). Pharmacy costs accounted for 69% of the mean total costs of $15,192 PPPM, with medical costs accounting for the remainder (Figure 1B). In 3L therapy and later, pts had a monthly IR/100pts of 3.5 IP admissions, 28.7 IP days, 252.2 OP days, and 10.0 ED visits (Figure 1A). Pharmacy costs accounted for 49% of the mean total costs of $18,767 PPPM, with medical costs mainly driven by IP costs (Figure 1B). Conclusions: This study characterized CML pts receiving later lines of therapy, a clinical population which has not been previously well studied with important unmet treatment needs as they repetitively fail TKI therapy. Although the majority of pts were likely fit for SCT, SCT was rare. In addition, pts quickly switched to the subsequent line of therapy, both facts suggesting that an important proportion of pts were intolerant to previous TKIs. While pharmacy costs accounted for nearly half of the total cost burden during 3L, the proportion of medical costs PPPM took more importance following 3L therapy, with IP costs being the primary cost drivers for this increase. These findings support the need for better treatment options in pts with CML undergoing later lines of therapy. Disclosures Atallah: Novartis Pharmaceutical Corporation: Consultancy; Jazz: Consultancy; Pfizer: Consultancy; Takeda: Consultancy, Research Funding; Abbvie: Consultancy; Genentech: Consultancy. Maegawa:Novartis Pharmaceutical Corporation: Current Employment, Current equity holder in publicly-traded company. Latremouille-Viau:Novartis Pharmaceutical Corporation: Consultancy, Other: Dominique Latremouille-Viau is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.; Sanofi Genzyme: Consultancy, Other: Dominique Latremouille-Viau is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.. Rossi:Novartis Pharmaceutical Corporation: Consultancy, Other: Carmine Rossi is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Guerin:Abbvie: Consultancy, Other; Sanofi Genzyme: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.; Novartis Pharmaceuticals Corporation: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Patwardhan:Novartis Pharmaceutical Corporation: Current Employment, Current equity holder in publicly-traded company.

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Treatment Patterns and Outcomes of Patients with Double-Class Refractory or Triple-Class Refractory Multiple Myeloma: A Retrospective US Electronic Health Record Database Study

Blood ◽

10.1182/blood-2021-152301 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 2705-2705

Author(s):

Feng Wang ◽

Boris Gorsh ◽

Maral DerSarkissian ◽

Prani Paka ◽

Rachel Bhak ◽

...

Keyword(s):

Research Funding ◽

Performance Status ◽

Treatment Patterns ◽

Multiple Drug ◽

Publicly Traded ◽

Analysis Group ◽

Refractory Status ◽

Drug Classes ◽

Immunomodulatory Drug

Abstract Introduction: Patients with relapsed/refractory multiple myeloma (RRMM) resistant to multiple drug classes remain a high unmet need population, despite advances in MM patient care. The objective of this study was to assess real-world treatment patterns and outcomes in patients with RRMM who had failed multiple prior lines of therapy (LOT) and were refractory to multiple drug classes to identify gaps in their treatment pathways. Methods: This longitudinal retrospective cohort study utilized the COTA de-identified real-world database derived from US electronic health records of partnered healthcare providers from Q3 1988 through Q1 2020. Adults with active RRMM previously exposed to a proteasome inhibitor (PI) and an immunomodulatory drug and who received ≥3 prior LOTs were identified. Patients were further categorized as refractory to a PI and an immunomodulatory drug (double-class refractory [DCR]) or additionally to an anti-CD38 monoclonal antibody (i.e. daratumumab; triple-class refractory [TCR]). Determining refractory status was based on International Myeloma Working Group criteria. Index LOT was a new LOT after evidence of DCR or TCR status following ≥3 (DCR) or ≥4 (TCR) prior LOTs. Patient characteristics described included Eastern Cooperative Oncology Group (ECOG) performance status, International Staging System (ISS) stage, cytogenetic risk, age, index date, sex, and follow up time. Treatment pattern assessments included treatments received before/during/after index LOT, reasons for discontinuation, refractory status, and retreatment characteristics. Patient outcomes (overall survival [OS], duration of treatment [DOT], and time to next therapy [TTNT]) were analyzed using Kaplan-Meier survival analysis methods. Results: After excluding patients who were aged <18 years at start of index LOT with no evidence of clinical activity and who participated in a clinical trial during index LOT, 381 (DCR) and 173 (TCR) patients were available for analysis. Median follow-up from index LOT initiation through end of data availability/death was 14 (DCR) and 8 (TCR) months. Demographic characteristics were consistent between DCR and TCR patients. Approximately half were aged ≥65 years (49% DCR; 53% TCR), majority had high-risk cytogenetics (56% DCR; 66% TCR) or prior autologous stem cell transplantation (>62%), and 14-16% had ISS stage III. Patients had a median of 3 (DCR) and 6 (TCR) prior LOTs. Prior to index LOT, bortezomib and lenalidomide were the most commonly received PI and immunomodulatory drug (received by >98% of DCR or TCR patients) and the most common PI and immunomodulatory drugs to which patients were refractory (71% and 84% DCR; 76% and 83% TCR, respectively). At index LOT, PI/immunomodulatory drug-based and daratumumab-based therapies remained the most common therapies. Bortezomib and lenalidomide had the longest time to refractory status and highest retreatment rates among DCR or TCR patients. Approximately 40% of TCR patients were retreated with daratumumab-based therapies after becoming refractory (Table). After index LOT, 70% of DCR and 58% of TCR patients continued to a subsequent LOT. Patients most frequently discontinued index LOT due to disease progression (59% DCR; 60% TCR), toxicity (23% DCR; 25% TCR), or doctor preference (14% DCR; 10% TCR). Median duration of gaps between LOTs generally were shorter than 1 month (Table). Median OS was 22.3 (DCR) and 11.6 (TCR) months. Median DOT was 3.3 (DCR) and 2.8 (TCR) months, and median TTNT was 4.1 (DCR) and 3.2 (TCR) months. In multivariate statistical analyses, inferior baseline ECOG performance scores, and high-risk cytogenetic abnormalities were associated with worse prognosis (higher risk of death, shorter DOT, and shorter TTNT) in DCR and TCR patients. Age was not a significant factor after adjusting for other baseline factors. Conclusions: Treatment options are limited for US patients with DCR and TCR RRMM. DCR and TCR patients were frequently retreated with a PI, an immunomodulatory drug, or daratumumab, despite refractoriness to these agents. Many DCR and TCR MM patients stopped active MM treatment after discontinuing index treatment. Patients with DCR and TCR MM have poor prognosis, especially among high cytogenetic risk and poor performance status patients. This study provides the benchmark for new therapies, like BCMA-targeted agents, to be tested in this population. Funding: GSK (Study 217353). Figure 1 Figure 1. Disclosures Wang: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Gorsh: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. DerSarkissian: Analysis Group, Inc.: Current Employment. Paka: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Bhak: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Boytsov: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Zichlin: GlaxoSmithKline: Research Funding; Analysis Group, Inc.: Consultancy, Current Employment. Sansbury: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Yee: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Ferrante: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Khanal: GlaxoSmithKline: Research Funding; Analysis Group, Inc.: Current Employment. Noman: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Duh: Novartis: Other: I am an employee of Analysis Group, a consulting company that received funding from Novartis for this research study..

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The First Retrospective Commercial Claims-Based Analysis of CAR T Treated Patients with Relapsed or Refractory Large B-Cell Lymphoma (R/R LBCL)

Blood ◽

10.1182/blood-2020-134735 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 31-31

Author(s):

Anna Purdum ◽

Jonathan Johnson ◽

Anthony Bonagura ◽

Liliosa Nyamutswa ◽

Caitlin Elliott ◽

...

Keyword(s):

Health Care ◽

Clinical Characteristics ◽

B Cell Lymphoma ◽

P Value ◽

Publicly Traded ◽

Index Event ◽

Car T ◽

Ed Visits ◽

The Mean

Introduction: Approximately 74,000 Americans are diagnosed with Non-Hodgkin Lymphoma (NHL) each year, of whom 30% are identified as having LBCL. In recent years, two autologous anti-CD19 chimeric antigen receptor T-Cell (CAR T) therapies were approved for the treatment of patients with R/R LBCL with ≥ 2 prior systemic therapies. Objectives: To describe the demographic and clinical characteristics of the CAR T patients and to compare healthcare resource utilization (HCRU) and costs pre- and post-CAR T therapy. Methods: This pre/post-index comparison evaluated in the Optum Research Database included adult (age ≥ 18 years) commercial and Medicare Advantage (MA) enrollees, with medical benefits and evidence of medical/procedure codes indicative of CAR T therapy between 01/01/2017 and 03/31/2019. Patients must have been continuously enrolled in their commercial healthcare plan for at least 3 months prior to and 6 months after the infusion (index) unless death occurred. Patients with evidence of pre-index leukemia were excluded. Baseline demographic and clinical characteristics included age, gender, insurance type, geographic region, and comorbidities. Measures of pre/post HCRU and standardized cost (scaled as per patient per month (PPPM) values to account for variable follow-up durations) included ambulatory visits, emergency department (ED) visits, and inpatient admissions (both Intensive Care Unit (ICU) and non-ICU visits). Information on the CAR T administration index visit and length of stay (LOS) for inpatient admissions were also captured. Data analysis (descriptive statistics) was conducted using Statistical Analysis System (SAS) Version 9.4. Results: 109 patients met all inclusion criteria. The patient mean age was 59.31 (SD = 12.60), 59% were male, 70% were commercially insured, 30% MA, and the mean Quan-Charlson Comorbidity score was 3.60.Thirty patients received CAR T administration through clinical trials. Seventeen percent received CAR T therapy in a Prospective Payment System (PPS) Exempt Cancer Hospital and 83% received treatment in Inpatient PPS hospitals. The CAR T therapy index event was administered inpatient for 82% of patients and twenty patients (18%) received CAR T in the outpatient setting. The median LOS during CAR T administration was 16 days which included 52% ICU admissions. During the pre-index period, all patients experienced an ambulatory visit with an average of 15.09 visits PPPM, whereas 41% had an ED encounter for an average rate of 0.39 visits PPPM, and 44% had an inpatient stay with 0.28 PPPM. The total medical and pharmacy costs were $128K PPPM. With a median follow-up of 8.4 months, patients experienced 46% fewer ambulatory visits (average 8.14 PPPM, p-value < 0.001), 49% fewer ED visits (average 0.20 PPPM, p-value < 0.037), and 18% fewer inpatient visit rates (average 0.23 PPPM, p-value = 0.415) in the post index period (excluding the index event) than pre-index period. The mean total medical and pharmacy costs were also 49% lower at $66K PPPM (p-value < 0.008). Conclusions:Post-CAR T care was associated with lower health care utilization including fewer ambulatory visits, and ED visits and lower overall total health care costs compared to pre-CAR T care. Disclosures Purdum: Kite: Current Employment, Current equity holder in publicly-traded company. Johnson:Optum: Current Employment, Current equity holder in publicly-traded company. Bonagura:Optum: Current Employment, Current equity holder in publicly-traded company. Nyamutswa:Kite: Current Employment, Current equity holder in publicly-traded company. Elliott:Optum: Current Employment. Lal:Optum: Current Employment.

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A Retrospective Database Analysis of Treatment Pathways in US Medicare Patients with Multiple Myeloma and Prior Exposure to Daratumumab, an Immunomodulatory Agent, and a Proteasome Inhibitor

Blood ◽

10.1182/blood-2021-152230 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4019-4019

Author(s):

Natalie Boytsov ◽

Anissa Cyhaniuk ◽

Gary Leung ◽

Feng Wang ◽

Cosmina Hogea ◽

...

Keyword(s):

Index Date ◽

Treatment Patterns ◽

Publicly Traded ◽

Immunomodulatory Agent ◽

Medicare Patients ◽

Treatment Sequencing ◽

The Mean ◽

Retrospective Database Analysis ◽

Retrospective Database

Abstract Introduction: Recentreal-world data for multiple myeloma (MM) treatment patterns and outcomes are limited, particularly for patients with refractory or relapsed MM. As the MM treatment landscape evolves, it is important to understand how new treatments are integrated into patient treatment patterns. The aim of this study was to examine patient demographics, clinical characteristics, treatment patterns, and survival among Medicare patients with MM following exposure to daratumumab (DAR), an immunomodulatory agent, and a proteasome inhibitor (PI). Methods: This was a retrospective database analysis utilizing the Centers for Medicare and Medicaid Services claims data during the study period of January 1, 2016, through December 31, 2018. The start of the study period was chosen to align with DAR market entry in the United States (FDA approval November 2015). Medicare patients, diagnosed with MM, and with existing claims for DAR, an immunomodulatory agent, and a PI (tri-exposure) were eligible for inclusion. Index tri-exposure was achieved once a patient had been exposed to all 3 MM treatments, regardless of their sequence. The index date was the first observed claim for any MM regimen (index line of therapy [LOT]) following tri-exposure. Patients were required to have ≥6 months of continuous enrollment prior to the index date (baseline period). Therapies given after the index LOT were defined as post-index therapy. Patient data were assessed until health plan disenrollment, death, or end of study period, whichever occurred first. Results: There were 1336 Medicare patients with MM who met the inclusion criteria. Of these patients, the mean age (standard deviation [SD]) at the index date was 71 (8.5) years and 705 (52.8%) were male. The Southern United States showed the largest representation of patients in this population (n=471, 35.3%). The top baseline comorbidities included respiratory infections (98.3%), osteoarthritis (86.8%), anemia (86.9%), hypertension (78.1%), dyslipidemia (66.5%), chronic pain/fibromyalgia (51.9%), acute or chronic kidney disease (44.5%), cardiac arrhythmia (45.81%), and neutropenia (47.98%). The mean (SD) number of days from the index date until the end of index LOT was 48.7 days (12.0). Among 949 patients (71.0%) who had post-index therapy, the mean (SD) time from the last observed claim for index LOT to the beginning of the post-index therapy and duration of post-index therapy was 51.8 (44.2) days and 57.8 (16.0) days, respectively. The median (range) duration of follow-up time from the index date was 223 (3─886) days. During the study follow-up period, 571 patients (42.7%) died, and the median (range) number of days from the index date until death was 173 (3─873) days. While there was variation in treatment sequencing leading to tri-exposure, the most frequent tri-exposure sequence was an immunomodulatory agent < PI < DAR (33.2% [See Table for overview of treatment sequencing]). Among patients who received index LOT following tri-exposure, 49.4% had triplet therapy and 29.8% had doublet therapy. The most common index LOT regimens were triplet: DAR/pomalidomide/dexamethasone (DEX; 7.6%), elotuzumab/lenalidomide/DEX (5.2%), and DAR/bortezomib/DEX (4.9%). The most common post-index LOT regimens were triplet (39.9%): DAR/pomalidomide (6.1%), carfilzomib/cyclophosphamide/DEX (3.7%), DAR/bortezomib/DEX (3.5%), and DAR/lenalidomide/DEX (3.5%). During follow-up, 52.8% of patients were retreated with DAR, 79% with PI, and 77.3% with immunomodulatory drugs. Conclusions: This study suggests wide variation in treatment sequencing and regimens after tri-exposure to DAR, an immunomodulatory agent, and a PI. Triplet regimens were predominant treatment after the tri-exposure and following the index LOT. Retreatment with the same agents was common. Among those who died, survival was often less than 1 year following tri-exposure. These results highlight the need for new treatment options in triple-class refractory MM settings. Funding: GSK (Study 213462) Figure 1 Figure 1. Disclosures Boytsov: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Cyhaniuk: STATinMED Research: Current Employment. Leung: STATinMED Research: Current Employment. Wang: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Hogea: GlaxoSmithKline, paid employee: Current equity holder in publicly-traded company, Ended employment in the past 24 months. Mudumby: STATinMED Research: Current Employment.

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Real-world treatment patterns, healthcare use and costs in triple-class exposed relapsed and refractory multiple myeloma patients in the United States

Future Oncology ◽

10.2217/fon-2020-1003 ◽

2020 ◽

Author(s):

Deepu Madduri ◽

May Hagiwara ◽

Kejal Parikh ◽

Corey Pelletier ◽

Thomas E Delea ◽

...

Keyword(s):

Multiple Myeloma ◽

Median Time ◽

Proteasome Inhibitors ◽

The United States ◽

Healthcare Resource Utilization ◽

Treatment Patterns ◽

Refractory Multiple Myeloma ◽

Kaplan Meier ◽

Line Of Therapy

Aim: To estimate treatment patterns and healthcare costs among triple-class exposed relapsed and refractory multiple myeloma (RRMM) patients. Materials & methods: Eligible patients had ≥1 line of therapy (LOT) each of proteasome inhibitors, immunomodulatory drugs and daratumumab in December 2015–September 2018 and received a new LOT. Results: A total of 154 patients were included with a median follow-up of 6.2 months. Median time from diagnosis to new LOT was 41.0 months. Kaplan–Meier estimate of median time to therapy discontinuation was 4.2 months. Mean per-patient, per-month MM-related costs were 35,657 US dollars. Most frequently observed regimens were lenalidomide or pomalidomide + daratumumab (18.2%), lenalidomide or pomalidomide + proteasome inhibitors (15.6%) and lenalidomide or pomalidomide monotherapy (11.0%). Conclusion: Triple-class exposed RRMM patients receive heterogeneous treatments for a short duration with high healthcare resource utilization and costs.

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Healthcare Resource Utilization and Costs of Patients with Relapsed/Refractory Follicular Lymphoma Receiving 3 or More Lines of Therapy

Blood ◽

10.1182/blood-2021-145407 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 1923-1923

Author(s):

Matthew J. Matasar ◽

Sheila Shapouri ◽

Jamie T. Ta ◽

Tu My To ◽

Mei Wu ◽

...

Keyword(s):

Healthcare Costs ◽

Research Funding ◽

Healthcare Resource ◽

Healthcare Resource Utilization ◽

Subsequent Treatment ◽

Publicly Traded ◽

The Past ◽

The Mean ◽

Anti Cd20

Abstract Background: Follicular lymphoma (FL) is indolent and typically incurable, and patients (pts) often receive multiple lines of therapy (LOTs) throughout their lifetime (Batlevi et al. Blood Cancer J 2020). Options at relapse following first- or second-line anti-CD20 monoclonal antibody (MoAb)-containing regimens remain limited, although approved third- and later-line therapies (3L+) have become available. Few studies have estimated the real-world economic burden of pts with relapsed/refractory (R/R) FL requiring 3L+ treatment. The aim of this study was to examine real-world healthcare resource utilization (HRU) and costs among pts receiving FL therapies in the 3L+ setting. Methods: This retrospective cohort study used administrative claims data from the IQVIA PharMetrics ® Plus, a US commercial claims database. Adult pts who had ≥1 inpatient claim or ≥2 outpatient claims with an FL diagnosis from January 1, 2011 to September 30, 2020 were included. The final 3L+ population was identified by combining two groups: (1) pts newly initiating FL treatment (defined as systemic anti-cancer therapies listed in the National Comprehensive Cancer Network [NCCN] guidelines) between January 1, 2012 and September 30, 2017 and receiving any subsequent 3L FL treatment during the study period; and (2) pts who received a NCCN-recommended 3L+ FL phosphatidylinositol-3-kinase inhibitor (PI3Ki) between January 1, 2012 and March 30, 2020. Group 1 captured pts who received 3L FL therapies by a proxy algorithm for LOT (Optum 2018) based on NCCN guideline-listed therapies, and group 2 captured pts who received newer available PI3Kis, which are only approved in pts who have received ≥2 previous systemic FL therapies. The index date was the 3L treatment initiation date or the initial PI3Ki claim date. All pts had ≥12 months of pre- and ≥6 months of post-index continuous enrollment in medical and pharmacy benefits. Pts with other primary cancers or evidence of histological transformation during the pre-index period, or clinical trial participation during the study period were excluded. All-cause HRU and all-cause and FL-related (i.e. claim with a FL diagnosis in any position) costs (2020 USD) were annualized during the 3L+ treatment period (defined as the period from index 3L+ treatment until the end of the LOT) to mitigate the effects of different follow-up times. Results: Overall, 100 pts who initiated 3L+ FL treatment were included. Of these, 51% of pts were male, and the mean (standard deviation [SD]) age at index and Charlson Comorbidity Index (non-cancer) at baseline were 62 (10.1) years and 0.9 (1.5), respectively. Mean follow-up time was ~2.3 years, and the mean duration of index 3L+ FL treatment was 273 days. Overall, 44 pts (44%) received subsequent treatment. The most common therapy classes received for index 3L+ FL treatment were oral PI3Kis (n=45, 45%), anti-CD20 MoAb monotherapy (n=19, 19%), and chemoimmunotherapy (CIT; n=18, 18%). A summary of all-cause annualized HRU in pts receiving index 3L+ FL treatment is provided by visit type (Table). For all 3L+-treated pts, mean (SD) all-cause annualized total healthcare costs in the 3L+ treatment period were $193,207 ($148,702), and 83% of total healthcare costs were FL-related costs ($159,815 [$138,477]; Figure). Of the most common 3L+ FL therapy classes, CIT had the highest FL-related mean annualized costs ($214,631 [$120,799]), followed by oral PI3Kis ($131,208 [$86,712]), and anti-CD20 MoAb monotherapy ($105,061 [$73,445]). Conclusions: The economic burden of pts with R/R FL requiring 3L+ FL treatment is substantial, with FL-related costs comprising the majority of total healthcare costs. More than 40% of the pts in this analysis needed subsequent treatment, further compounding the challenges faced by this high-risk population. This analysis provides an initial benchmark for ongoing and future evaluations of the economic value of currently available and emerging therapies for multiple relapsed FL, though future studies with larger sample sizes and longer follow-up are warranted. Figure 1 Figure 1. Disclosures Matasar: Pharmacyclics: Honoraria, Research Funding; Memorial Sloan Kettering Cancer Center: Current Employment; Merck Sharp & Dohme: Current holder of individual stocks in a privately-held company; Genentech, Inc.: Consultancy, Honoraria, Research Funding; Merck: Consultancy; GlaxoSmithKline: Honoraria, Research Funding; F. Hoffmann-La Roche Ltd: Consultancy, Honoraria, Research Funding; Juno Therapeutics: Consultancy; Janssen: Honoraria, Research Funding; Bayer: Consultancy, Honoraria, Research Funding; Teva: Consultancy; Takeda: Consultancy, Honoraria; Seattle Genetics: Consultancy, Honoraria, Research Funding; IGM Biosciences: Research Funding; Daiichi Sankyo: Consultancy; Rocket Medical: Consultancy, Research Funding; ImmunoVaccine Technologies: Consultancy, Honoraria, Research Funding; TG Therapeutics: Consultancy, Honoraria. Shapouri: F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment. Ta: Genentech, Inc.: Current Employment. To: F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months; Genentech, Inc.: Current Employment. Wu: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Wang: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Aurinia Pharmaceuticals Inc.: Current equity holder in publicly-traded company; Novavax, Inc.: Current equity holder in publicly-traded company; Oragenics, Inc.: Current equity holder in publicly-traded company; The SPHERE Institute: Ended employment in the past 24 months; TG Therapeutics, Inc.: Current equity holder in publicly-traded company.

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Treatment Patterns and Healthcare Resource Utilization in Patients with Multiple Myeloma and Baseline Renal Impairment

Blood ◽

10.1182/blood-2020-134597 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 17-18

Author(s):

Parameswaran Hari ◽

Lita Araujo ◽

Dominick Latremouille-Viau ◽

Peggy Lin ◽

Mikhail Davidson ◽

...

Keyword(s):

Multiple Myeloma ◽

Overall Survival ◽

Renal Impairment ◽

Resource Utilization ◽

Real World ◽

Economic Burden ◽

Healthcare Resource ◽

Healthcare Resource Utilization ◽

Treatment Patterns ◽

Analysis Group

Background: Renal impairment (RI) is associated with substantial clinical and economic burden in patients with multiple myeloma (MM), but real-world data reporting on healthcare resource utilization (HRU) and outcomes in these patients are lacking. We assessed treatment patterns, overall survival (OS), HRU and associated costs across lines of therapy (LoT) in patients with MM who had baseline RI. Methods: We identified patients (aged ≥18 years) with continuous Part A, B and D coverage who initiated pharmacologic therapy for MM between January 1, 2012 and December 31, 2016. Baseline demographics, disease characteristics, and treatment patterns from first-line to fourth-line (1L-4L) were reported for all eligible patients (main cohort). Within this cohort, a subgroup of patients diagnosed with RI at baseline (RI subgroup) were identified using appropriate International Classification of Diseases (ICD)-9 and ICD-10 codes. Treatment regimens were identified during the first 60 days following start of each LoT; stem cell transplantation (SCT) in 1L was considered part of the 1L regimen. The end of each LoT was indicated by treatment augmentation, treatment switching (after >60 days), discontinuation of all agents (for >90 days), or death. Overall survival (Kaplan-Meier analysis) was defined as time from start of each LoT until death or censoring (end of data/Medicare coverage). All-cause HRU categories were identified during each LoT and reported as incidence rate per patient per month (PPPM); associated all-cause healthcare costs during LoT were reported in 2017 US$. Results are presented using standard descriptive statistics. Results: A main cohort of 10,026 patients was identified; of these, a RI subgroup of 714 patients with baseline RI was identified (7.1% of main cohort). At 1L initiation, the RI subgroup was generally younger (71.9 vs. 74.6 years), had a lower proportion of females (47.8% vs. 53.1%) and had a higher proportion of Medicare coverage for end-stage renal disease (62.9% vs. 6.3%) than the main cohort. Patients with RI had a higher mean Charlson Comorbidity Index score (excluding MM; 4.8 vs. 3.3) and a higher proportion of patients with comorbidities (anemia: 72.5% vs. 57.9%; diabetes with chronic complications: 38.7% vs. 27.1%; cardiovascular diseases: 97.2% vs. 82.5%) than the main cohort. In the RI subgroup, among patients who received SCT in 1L (n=76), bortezomib-dexamethasone (Vd) was the most frequent 1L regimen (39.5%), followed by bortezomib-lenalidomide-dexamethasone (VRd; 17.1%) and bortezomib-cyclophosphamide-dexamethasone (VCd; 15.8%). In patients who had no SCT in 1L, Vd was the most frequent 1L regimen (59.5%), followed by VCd (12.7%) and lenalidomide-dexamethasone (Rd; 12.1%). Among patients in the RI subgroup who progressed to 2L therapy, 61.7% received lenalidomide-based regimens in 1L. Newer MM therapies such as carfilzomib, pomalidomide, ixazomib, daratumumab, and elotuzumab were used more frequently in later LoTs (2L: 25.6%; 3L: 50.0%; 4L: 68.8%). Median OS from start of 1L was shorter in the RI subgroup than in the main cohort (29.9 vs. 46.5 months; Table), and this difference was consistent across each subsequent LoT. Incidence of HRU during 1L (Table) was generally higher in the RI subgroup than the main cohort, particularly for inpatient days (1.3 vs. 0.7 PPPM) and home health services (0.9 vs. 0.5 PPPM); this pattern was consistent between cohorts across each subsequent LoT. Total costs in the 1L RI subgroup vs. main cohort (Table) were $14,782 vs. $12,451; the cost differential was maintained across each subsequent LoT. The key driver of this difference was the additional medical service costs ($12,047 vs. $7,459 in 1L) incurred by patients with RI. Conclusion: Patients with MM who had baseline RI were shown to experience higher clinical and economic burden in real-world clinical practice than the overall MM population. This burden was maintained across LoTs. Efficacious regimens that help improve renal function with minimal toxicity would enable patients with MM and RI to persist with treatment and may help address unmet need in this subgroup of patients. Table Disclosures Hari: BMS: Consultancy; GSK: Consultancy; Janssen: Consultancy; Amgen: Consultancy; Takeda: Consultancy; Incyte Corporation: Consultancy. Araujo:Sanofi Genzyme: Current Employment. Latremouille-Viau:Sanofi Genzyme: Consultancy, Other: Dominique Latremouille-Viau is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.; Novartis Pharmaceutical Corporation: Consultancy, Other: Dominique Latremouille-Viau is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Lin:Sanofi Genzyme: Current Employment. Davidson:Sanofi Genzyme: Other: Mikhail Davidson is an employee of Analysis Group, Inc which received consultancy fees from Sanofi Genzyme.. Guerin:Sanofi Genzyme: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.; Abbvie: Consultancy, Other; Novartis Pharmaceuticals Corporation: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Sasane:Sanofi Genzyme: Current Employment.

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Evolving Unmet Need in Patients Refractory to Lenalidomide: Overview of the Clinical Trials in Relapsed/Refractory Multiple Myeloma

Blood ◽

10.1182/blood-2020-142642 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 22-23

Author(s):

Maria-Victoria Mateos ◽

Rohan Medhekar ◽

Istvan Majer ◽

Mehmet Turgut

Keyword(s):

Multiple Myeloma ◽

Clinical Trials ◽

Literature Review ◽

Board Of Directors ◽

Unmet Need ◽

Publicly Traded ◽

First Line ◽

Advisory Committees ◽

Line Of Therapy

Introduction: The majority of newly diagnosed multiple myeloma (NDMM) patients are currently treated with lenalidomide-based regimens as their first line of therapy. This trend is likely to continue in the coming years. Typically, lenalidomide is administered until disease progression and has significantly contributed to better outcomes in these patients. However, most patients relapse, and prognosis worsens with each relapse. The choice of optimal treatment for patients who relapse while receiving lenalidomide as first line of therapy is unclear. Moreau et al (Blood Cancer J. 9, 38 [2019]) concluded that there is limited data on approved combinations for treating these patients and are restricted by the low number of lenalidomide-refractory patients enrolled in the pivotal trials. Results from the ongoing clinical trials of the combination of carfilzomib and anti-CD38 antibodies were not available at the time of the Moreau et al publication. The aim of this targeted literature review was to include this new data and to summarize currently available evidence on progression-free survival (PFS) for the treatment of RRMM patients who progressed on lenalidomide-based regimens. Methods: A targeted literature review was conducted to identify registrational clinical trials in patients with RRMM reporting PFS outcomes. PubMed, congress proceedings, and product labels were searched between Jan 2014 to July 2020. In addition to PFS, demographic, disease characteristics and treatment history were extracted for the trial populations to contextualize potential variations in study outcomes. The regimens studied in these trials were classified as lenalidomide-based, proteasome inhibitor (PI)-based and pomalidomide-based. Number of prior lines of therapy, prior exposure and refractoriness to lenalidomide and bortezomib were reported. Results: Twelve registrational trials were identified based on the search criteria (Table 1). Most pivotal trials assessing lenalidomide-based regimens (POLLUX, ELOQUENT-II, TOURMALINE-MM1) except the ASPIRE trial excluded patients who were refractory to lenalidomide. Trials evaluating PI-based regimens (e.g., CANDOR) or pomalidomide-based regimens (e.g., OPTIMISMM) included these patients, with more recent studies enrolling a larger proportion. Percentage of lenalidomide-exposed (and lenalidomide refractory) ranged from 40% (32%) in CANDOR to 98% (90%) in ELOQUENT III. These studies also enrolled a larger proportion of patients who were bortezomib-exposed, although most of these patients were at first relapse, with the exception of ELOQUENT III and ICARIA where most patients were at third relapse. Among lenalidomide-refractory patients, the median-PFS (mPFS) observed for the pomalidomide-based regimens ranged from 9.5 to 10.1 months and that observed for PI-based regimens ranged from 4.9 to 25.7 months. PFS in the lenalidomide-refractory subgroup was considerably shorter than in the ITT population. The mPFS for patients receiving carfilzomib/daratumumab/dexamethasone (KDd; CANDOR) and isatuximab/carfilzomib/dexamethasone (IsaKd; IKEMA) was not reached at median follow-up of 16.9 and 20.7 months respectively. While the mPFS for (KDd) for lenalidomide-refractory patients in CANDOR trial was not yet reached at median follow up of 16.9 months; the mPFS of 25.7 months for KDd in the MMY-1001 trial appears to be the longest among the assessed regimens. Conclusion: Patients refractory to lenalidomide have shorter PFS and represent a population with high unmet need. This targeted literature review suggests that the PI-based KDd regimen provides longer PFS compared to other lenalidomide-sparing regimens in lenalidomide-refractory populations. Heterogeneity across trial populations may limit the comparability of these treatments. Disclosures Mateos: Regeneron: 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; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees; Oncopeptides: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Honoraria, Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Janssen-Cilag: Consultancy, Honoraria; Adaptive Biotechnologies: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Abbvie/Genentech: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; PharmaMar-Zeltia: Consultancy; GlaxoSmithKline: Consultancy. Medhekar:Amgen Inc.: Current Employment, Current equity holder in publicly-traded company. Majer:Amgen (Europe) GmbH: Current Employment, Current equity holder in publicly-traded company.

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Analysis of Healthcare Resource Utilization and Costs after the Initiation of Biologic Treatment in Patients with Ulcerative Colitis and Crohn’s Disease

Journal of Health Economics and Outcomes Research ◽

10.36469/9791 ◽

2018 ◽

Vol 6 (1) ◽

pp. 96-112 ◽

Cited By ~ 1

Author(s):

Sue Perera ◽

Shibing Yang ◽

Marni Stott-Miller ◽

Joanne Brady

Keyword(s):

Ulcerative Colitis ◽

Crohn’S Disease ◽

Crohn's Disease ◽

Resource Utilization ◽

Healthcare Resource ◽

Healthcare Resource Utilization ◽

Small Sample ◽

Outpatient Visits ◽

Ed Visits

Background: This retrospective cohort study aimed to describe and quantify healthcare resource utilization and costs for patients with ulcerative colitis (UC) and Crohn’s disease (CD) following initiation of biologic therapy. Methods: Resource utilization and costs were analyzed at baseline and 1- and 2-years after initiating a biologic. Data were extracted from a US administrative health insurance claims database for adults ≥18 years. Eligible patients were continuously enrolled in a health plan with medical and pharmacy benefits for ≥12 months prior to, and 12 months (primary analysis) or 24 months (secondary analysis) after index date (biologic initiation). Results: In total, 4864 and 2692 patients with UC, and 8910 and 5227 patients with CD were identified in the 1- and 2-year follow-up cohorts, respectively. Of 1-year follow-up cohort patients, 45% received the same biologic initiated at index for ≥1 year. Infliximab and adalimumab were the most commonly initiated biologics in patients with UC or CD. The highest proportion of patients who continued with the same biologic after 1-and 2-years had initiated therapy with infliximab for both indications (although at the 1-year follow-up for CD, the highest proportion continued to use natalizumab, but this was a small sample [n=15]). Generally, the proportion of patients having inpatient admissions and emergency department (ED) visits decreased after receiving the same biologic for 1 year compared with baseline, although the proportion having outpatient visits did not change. Mean per patient all-cause costs for inpatient hospitalizations, ED visits and outpatient visits decreased for patients with UC or CD who received the same biologic for 1 year, while mean pharmacy costs per patient increased. Conclusions; This descriptive analysis shows that although biologics effectively reduced inpatient and ED resource utilization and corresponding costs in patients with UC and CD, total management costs increased, driven by increased pharmacy costs.

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Characteristics, Treatment Patterns, Healthcare Resource Use, and Costs among Pediatric Patients Diagnosed with Neurofibromatosis Type 1 and Plexiform Neurofibromas: A Retrospective Database Analysis of a Medicaid Population

10.21203/rs.3.rs-98302/v1 ◽

2020 ◽

Author(s):

Xiaoqin Yang ◽

Kaushal Desai ◽

Neha Agrawal ◽

Kirti Mirchandani ◽

Sagnik Chatterjee ◽

...

Keyword(s):

Resource Use ◽

Neurofibromatosis Type 1 ◽

Pediatric Patients ◽

Healthcare Resource ◽

Neurofibromatosis Type ◽

Treatment Patterns ◽

Healthcare Resource Use ◽

The Mean

Abstract Background: Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN) can cause substantial morbidity by disfigurement and compression of vital structures. Real-world data on the burden and cost of disease among pediatric patients with NF1 and PN is limited. The objectives of this study were to describe the characteristics, treatment patterns, healthcare resource use (HCRU), and costs of these patients.Results: A total of 383 patients were included in the retrospective analysis of patients aged ≤18 with at least 1 ICD-10-CM diagnosis code for both NF1 and PN enrolled in the MarketScan® Multistate Medicaid database from October 1, 2014 to December 31, 2017. The mean follow-up was 448 days. The mean age was 11.4 years and 52.0% of patients were male. Most patients were diagnosed by a specialist (63.5%). During the follow-up period, pain medications were used by 58.5% of patients, 25.1% were treated with chemotherapy, 7.1% received surgery for PN, 1.6% received MEK inhibitors, and 0.8% received radiation. Mean per patient per year inpatient, outpatient, emergency room, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ±SD (median) total per patient per year healthcare costs (2018 USD) were $17,275 ±$61,903 ($2,889), with total medical costs of $14,628 ±$56,203 ($2,334) and pharmacy costs of $2,646 ±$13,303 ($26). Inpatient costs were the largest drivers of medical cost, with a mean per patient per year cost of $6,739.Conclusions: This study showed that many pediatric patients diagnosed with NF1 and PN were treated with supportive care only, highlighting a substantial unmet medical need. This study also highlights the considerable economic burden among patients with NF1 and PN.

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Healthcare Resource Utilization and Sickness Absence in Newly Diagnosed Multiple Myeloma Patients in Sweden: Trends in a Changing Treatment Landscape

Blood ◽

10.1182/blood-2020-137498 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 17-18

Author(s):

Fredrik Borgsten ◽

Xenia Gatopoulou ◽

Marta Pisini ◽

Magnus Tambour ◽

Frida Schain ◽

...

Keyword(s):

Multiple Myeloma ◽

Sickness Absence ◽

Healthcare Resource ◽

Productivity Loss ◽

Healthcare Resource Utilization ◽

Publicly Traded ◽

The Third ◽

The Past ◽

Over Time

Background In the last decades the introduction of novel drugs has greatly improved the prognosis of multiple myeloma (MM) patients. We have investigated healthcare resource utilization and sickness absence-associated productivity loss over time in a population-wide, retrospective registry study in Sweden. Methods 8,693 patients were identified in the National Cancer Register with a MM diagnosis from July 2001 to December 2015 and followed until 2016. Specialized healthcare usage (inpatient admissions and outpatient visits) were obtained from the Patient Register and costs were estimated by weighted DRG codes. For patients under 66 years of age, sickness absence and salary information were obtained by linkage to the LISA Register. Analyses were performed separately on patients who underwent autologous stem cell transplantation (ASCT) (n=1,425) and on non-transplanted patients (n=7,012) and stratified by diagnosis periods 2001-2005, 2006-2010 and 2011-2015 to reflect increased introduction of effective drugs into clinical care. Median age was 60 years in the ASCT group and 75 years in the non-ASCT group. Results The number of MM patients that underwent ASCT increased over time (n= 282 in 2001-2006 to n= 592 in 2011-2015). MM patients diagnosed most recently had improved overall survival (OS), with five-year OS rate increasing from 52% to 58% to 62% for patients diagnosed in 2001-2005, 2006-2010 and 2011-2015, respectively (p<0.0001). Patients diagnosed during 2011-2015 spent on average 20% and 9% less total time in specialized healthcare than patients diagnosed during 2001-2005 and 2006-2010, respectively (adjusting for sex, age at ASCT, weighted comorbidity score at ASCT and per follow-up year and education at ASCT). This decrease was driven by less usage and time in both inpatient and outpatient care. Adjusted sickness absence time decreased by 41% and 38% in the third follow-up year for patients diagnosed during 2011-2015 compared to patients diagnosed during 2001-2005 and 2006-2010, respectively. Productivity loss costs represented about 45% of total costs (healthcare resource costs ~55%) in the first two follow-up years, but decreased over time. The cumulative median per person cost (healthcare- and productivity-related) over the three follow-up years post-diagnosis decreased by 21% in 2011-2015 (€52,273) compared to 2001-2005 (€66,182), despite an 8% increase in three-year OS over the same period. The number of non-ASCT MM patients also increased over time (n=2,053 in 2001-2005 to n= 2,587 in 2011-2015). Median survival increased from 2.5 years to 3.4 years for patients diagnosed during 2001-2005 compared to 2011-2015. Average total time spent in specialized healthcare was reduced by 29% and 12% for patients diagnosed during 2011-2015, compared to patients diagnosed during 2001-2005 and 2006-2010, respectively (adjusting for sex, age at diagnosis, weighted CCS at diagnosis, weighted CCS per follow-up year and education at diagnosis). This was associated with decreased need for inpatient care and a shift towards more outpatient usage. By the third follow-up year, the adjusted sickness absence time in patients diagnosed during 2011-2015 was reduced by 44% and 23% compared to patients diagnosed in 2001-2005 and 2006-2010, respectively. Productivity loss accounted for approximately 15% of total costs (healthcare resource costs ~85%) and was stable over follow-up years. The cumulative median per-person cost (healthcare- and productivity-related) over three follow-up years was similar for patients diagnosed in 2001-2005 (€25,621) and 2011-2015 (€26,592), despite a 12% increase in three-year OS over the same period. Conclusion The availability of new treatment options for MM patients in Sweden over time was associated with less healthcare usage, less time spent in healthcare and lower productivity loss due to sickness absence for both ASCT and non-ASCT-treated patients. These improved clinical and economic outcomes provide policy makers, healthcare providers and physicians with invaluable real-world insights for cost-benefit considerations in the continued development and introduction of effective treatments for MM. Figure 1 Disclosures Borgsten: Janssen: Current Employment. Gatopoulou:Janssen: Current Employment. Pisini:Janssen: Current Employment. Tambour:Janssen: Current Employment, Current equity holder in publicly-traded company. Schain:Schain Research: Current Employment, Current equity holder in private company, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Divested equity in a private or publicly-traded company in the past 24 months, Ended employment in the past 24 months. Jones:Schain Research: Current Employment. Kwok:Schain Research: Other: Internship . Hjortsberg:Janssen: Current Employment.

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