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e18824
Background: Recent advances in NGS testing have revolutionized the scope for personalized medicine. The goal of this study was to describe historic trends in the average allowed ...cost for NGS testing. Methods: This was a retrospective database study evaluating biomarker testing claims in patients undergoing tumor and/or germline sequencing tests between 2016-2019. Molecular testing claims data and marketplace data of tests currently sold in the U.S. were analyzed in the proprietary Concert Genetics Medical Claims Data Warehouse. Results: Analysis of data highlighted high variability in utilization rates as well as allowed amounts for NGS testing. The average allowed amounts for NGS Tests varied from $1269 to $2058 per test during the study period. Higher variability in billing codes and price was also observed when analysis was done for subgroups, which included region, age, gender and cancer type. Similar trends were also observed for panels containing NGS-derived tests such as Tumor Mutation Burden (TMB), an upcoming biomarker. Average allowed amounts for NGS tests that included TMB testing varied from $438 to $3700 per test. To gain a better perspective on evolution of coding, utilization and pricing trends, additional analyses were done in hereditary cancer, given longer availability of these tests in the US Market. For Hereditary cancers, the average allowed amounts varied from $1722 to $2249 per test during the study period. Greater consistency in price was observed in hereditary cancer. Utilization is also more consistent over time and price showed a gradual decreasing trend over time. Coding was also less variable per claim for hereditary cancer and showed a converging trend. Conclusions: Understanding and quantifying the trends in cost of NGS testing for novel biomarkers is critical to predict evolution of testing costs and provide insights essential for minimizing barriers to availability and affordability of these tests. NGS testing can play an important role in improving quality of care and making the clinical decision-making process more efficient. Currently, coverage and reimbursement of NGS tests is one of the key policy challenges that needs to be addressed. Hence, development and adoption of policies is needed to ensure efficient integration of NGS testing in the US health care system.
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Background: Curative intent therapy with resection or ablation for early-stage HCC is associated with high incidence of recurrence. Effective adjuvant therapy is needed to improve outcomes. While ...OS is the preferred endpoint for demonstrating efficacy of new adjuvant therapies, recurrence-free survival (RFS) may better reflect the benefits associated with adjuvant therapy due to being independent of subsequent therapies upon recurrence. Additionally, RFS may provide insights into patient (pt) outcomes while OS data are immature. We assessed the correlation between OS and RFS in pts with early-stage HCC after treatment with curative intent. Methods: This study used the SEER-Medicare database (2007–2017). RFS was defined as time from curative intent therapy, by local ablation or surgical resection, until death or disease recurrence (first of: new HCC treatment, liver transplant, metastatic disease). OS was defined as time from curative intent therapy until death. Kaplan-Meier analyses were used, censoring on end of Medicare Parts A/B/D continuous eligibility or end of data availability. RFS/OS were summarized overall and by type of prior local therapy and risk of recurrence. Pts with a solitary tumor with histologic grade 3 or 4, solitary tumor >3cm, or multiple tumors were classified as high to very high risk. Pts with a solitary tumor not histologic grade 3 or 4 or a solitary tumor ≤3 cm were classified as low to intermediate risk. Correlation between RFS and OS was assessed using the Kendall’s τ rank correlation. Results: 611 HCC pts met the inclusion criteria (mean follow-up 3.3 years; mean age 73.3 years). Average (median) primary tumor size at diagnosis was 4.8 (4) cm. 91.6% of pts had a solitary tumor at diagnosis. 66.1% of pts experienced death or recurrence (Table). Median IQR OS and RFS post-curative intent therapy were 4.6 1.8, NR and 1.8 0.8, 6.3 years, respectively. OS and RFS were poorer for those in the ablation (median OS 3.2 years; RFS 1.2 years) and high to very high risk of recurrence group (median OS 4.3 years; RFS 1.4 years). Kendall’s τ rank correlation model in the overall group demonstrated a statistically significant positive correlation between RFS and OS (τ = 0.66; 95% CI: 0.60–0.70; P < 0.001). Conclusions: Based on real-world data, a significant positive correlation between RFS and OS supports RFS as an efficacy marker and appropriate endpoint for pts in adjuvant HCC setting when OS data are immature. Table: see text
Introduction:While the majority of patients (pts) with newly diagnosed classical Hodgkin's lymphoma (cHL) achieve remission following first-line therapy, approximately 30-40% will relapse or become ...refractory (R/R). Despite R/R status, the 5-year relative survival rate in this population is high (ranging from 78% to 92%) (American Cancer Society 2020). While overall survival (OS) is an unambiguous measure of efficacy in clinical trials, using it as a primary end point requires a long duration of follow-up and may prolong the process of identifying novel and potentially beneficial therapy. Surrogate outcomes, such as progression-free survival (PFS) and time to progression (TTP) are sometimes regarded as valid alternatives when assessing efficacy of a treatment in the absence of mature OS data. The objectives of this study were to assess the conditional survival (CS) given prior PFS among R/R cHL pts treated in the real-world setting.
Methods:This retrospective cohort study used electronic medical record (EMR) data of US pts from a network of oncology practices, including practices affiliated with CancerLinQ, maintained in the Definitive Oncology Dataset. Eligible adult (≥18 years) pts who had a confirmed diagnosis of cHL and ≥1 R/R event that occurred between 2000 to 2019 were included. Refractory disease was defined as not achieving a complete or partial response (CR or PR) during a line of therapy. Relapsed disease was defined as the reappearance of cHL at any time after the end of treatment after having achieved a CR or PR. PFS and OS were measured from the start of second-line 2L therapy to the earlier or disease progression or death; pts without evidence of disease progression or death were censored at the last observed visit. The association between PFS and OS was quantified through the nonparametric Kendall tau rank correlation for bivariate censored data. CS was defined as the Kaplan-Meier probability of surviving an additionalymonths (from the start of 2L), given no OS or PFS event in the previousx(< y) months.
Results:A total of 286 R/R cHL pts were included. Most pts were Caucasian (77.3%, n=221) with a median age of 35 years (range: 19-86) at the first R/R event. Median length of follow-up was 12 months from the first R/R event. The most common B symptoms reported were night sweats (38.8%, n=111), weight loss (36.4%, n=104), and fatigue (27.3%, n=78). More than half of pts (54.9%, n=157) received autologous stem-cell transplant (SCT) and 6.6% (n=19) received allogenic SCT. Two-hundred twenty-six pts (79.0%) had documented 2L therapy (first R/R event). From the first R/R event, among these 226 pts, median PFS was 21.0 months (95% confidence interval CI: 15.1, 52.5) and median OS was 146.7 months (95% CI: 119.9, not achieved NA). CS rates among pts alive without disease progression increased with time: 1-year and 6-year survival rates were 95.7% (95% CI: 91.5, 97.8) and 79.3% (95% CI: 70.5, 85.7), respectively, in pts alive without progression at 3 months, but increased to 100.0% (95% CI: 100, 100) and 94.6% (95% CI: 79.9, 98.6) in pts alive without progression at 3-years (Table). The calculated Kendall tau correlation was 0.42 (p<0.0001), which suggested a statistically significant dependence between PFS and OS.
Conclusion:In the real-world setting, R/R cHL pts with longer time without disease progression had lower rates of death and better prognosis. Results from this study support a relationship between PFS and OS; however, further validation and acceptance of PFS as a surrogate endpoint in cHL is required. Establishing these relationships may inform future clinical trial design and interpretation of interim trial data.
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Desai:Merck & Co., Inc:Current Employment, Current equity holder in publicly-traded company.Yang:Merck & Co, Inc.:Current Employment.Gilligan:ConcertAI:Current Employment;Merck & Co., Inc.:Research Funding.Li:Merck & Co., Inc.:Current Employment, Current equity holder in publicly-traded company.Raut:Merck & Co., Inc.:Current Employment.Nahar:Merck Sharp & Dohme, Corp., a subsididary of Merck & Co., Inc., Kenlworth, NJ, USA:Current Employment.
Introduction: Limited real-world studies exist on the management of relapsed/refractory (R/R) classical Hodgkin's lymphoma (cHL) patients (pts) who failed autologous stem transplant (auto-SCT) and ...their subsequent healthcare resource utilization (HRU) and cost. Current treatment options include chemotherapy, a second auto-SCT, allogenic (allo-) SCT, palliative care, or newer therapies like brentuximab vedotin (BV) or programmed death-1 (PD-1) blocking antibodies. Pts eligible for treatment post auto-SCT failure may consume significant resources and using real-world data may inform the place of therapy of newly approved agents. Therefore, the objectives of this study were to compare HRU and cost among R/R cHL pts who received auto-SCT by transplant success and failure.
Methods: This retrospective cohort study used electronic medical record (EMR) data of US pts from a network of oncology practices, including practices affiliated with CancerLinQ, maintained in the Definitive Oncology Dataset. Eligible adult (≥18 years) pts who had a confirmed diagnosis of cHL and ≥1 R/R event that occurred between 2000 to 2019 were included. Treatment patterns included any systemic anti-cancer therapy received post auto-SCT failure. Descriptive analyses examined differences by auto-SCT success vs failure. Auto-SCT failure was defined as having a R/R event or disease progression after receipt of auto-SCT. HRU included hospitalization rates, emergency department (ED) visits, and infused supportive care drugs. Costs (inflated for 2020$) were based on matched Health Care Utilization Project coded events. HRU and costs were reported per patient per month (PPPM) from initial cHL diagnosis (first-line 1L therapy) through the second R/R event (third-line 3L therapy) and for 3L among a subset of pts who failed auto-SCT in second line (2L). PPPM was calculated by dividing the total HRU or cost during the observation period by the number of months of the observation period and then averaged across all pts (regardless of being flagged for a specific service).
Results: A total of 157 pts (54.9%) received auto-SCT among the R/R cHL cohort (n=286). Most pts were Caucasian (77.7%) with a median age of 31 years (range: 19-73) at the first R/R event. Median length of follow-up was 11 months from the first R/R event. Nearly all pts (91.7%) received auto-SCT after the start of 2L (68.2%, n=107) and 3L (23.6%, n=37). Approximately 9.6% (n=15) also received allo-SCT in later lines. Among auto-SCT pts, 62.4% (n=98) had a transplant success vs 37.6% (n=59) with a transplant failure. Across these 59 pts, 46 (78.0%) received treatment post auto-SCT failure. Treatment post auto-SCT failure consisted of 21 different anti-cancer regimens (monotherapy or in combination) and included BV (alone or in combination) (37.3%, n=22), chemotherapy (30.5%, n=18), PD-1 therapy (alone or in combination) (6.8%, n=4), other (5.1%, n=3), and allo-SCT (1.7%, n=1). The 59 pts with auto-SCT failure primarily failed in 2L (66.1%, n=39) and 3L (27.1%, n=16). HRU and costs for the 39 pts who failed auto-SCT in 2L were substantial in 3L. Approximately 92.3% of pts had a hospitalization, 30.8% had an ED visit, and 51.3% received infused supportive care treatment in 3L. Monthly costs in 3L were high: hospitalization $3,903, ED visit $130, infused supportive care $279, anti-cancer therapy $64,572, and $69,186 total.
From the start of 1L through the end of 3L, the proportion of pts with a hospitalization was significantly higher for pts who failed auto-SCT (Table). Subsequently, costs were also higher and average length of stay longer. While HRU did not differ, infused supportive care costs were higher for auto-SCT pts. No significant differences in HRU and cost were observed across the two groups for ED visits and oncology setting outpatient visits. Anti-cancer therapy costs were significantly higher for pts who failed auto-SCT. Total monthly costs were higher for pts who failed auto-SCT.
Conclusion: In the real-world setting, almost 40% of R/R cHL pts failed auto-SCT. There appears to be no clear standard of care post auto-SCT failure and using real-world data may inform the place in therapy of newer therapies. The HRU and cost of managing post auto-SCT failure was substantial and highlights the significant unmet need in this population. These findings add to the scarce real-world data on treatment patterns, utilization, and cost among R/R cHL pts who receive auto-SCT.
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Yang:Merck & Co, Inc.: Current Employment. Desai:Merck & Co., Inc: Current Employment, Current equity holder in publicly-traded company. Gilligan:ConcertAI: Current Employment; Merck & Co., Inc.: Research Funding. Raut:Merck & Co., Inc.: Current Employment. Nahar:Merck Sharp & Dohme, Corp., a subsididary of Merck & Co., Inc., Kenlworth, NJ, USA: Current Employment.
The objectives of this study were to describe the characteristics and initial treatment patterns, healthcare resource use (HCRU), and costs of patients newly diagnosed with neurofibromatosis type 1 ...(NF1)-related plexiform neurofibromas (PN).
This was a retrospective study of individuals enrolled in the IBM MarketScan Multi-State Medicaid database from 1 October 2014 to 31 December 2017. Patients aged ≤18 years at the index date (first diagnosis of NF1 or PN, whichever occurred later) with at least 1 ICD-10-CM diagnosis code for both NF1 and PN were included. All-cause HCRU and the associated direct costs during the follow-up period were calculated per patient per year (PPPY) in 2018 USD.
A total of 383 patients were included with a mean follow-up of 448 days. 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 PPPY inpatient, outpatient, ER, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ± SD (median) total PPPY healthcare costs were $17,275 ± $61,903 ($2889), with total medical costs of $14,628 ± $56,203 ($2334) and pharmacy costs of $2646 ± $13,303 ($26).
This study showed that many pediatric patients newly diagnosed with NF1 and PN were initially 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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e18069
Background: Historically, recurrence in ovarian cancer (OC) following first-line (1L) chemotherapy (CT) occurs in up to 80% of patients within 2 years. The Clinical ...Classification Software (CCS) systematically classifies thousands of ICD-9 codes into a smaller number of clinically meaningful categories. We sought to use CCS and other routinely collected variables to differentiate the clinical and demographic profiles of patients with good prognosis (GP) versus poor prognosis (PP) in the United States (US). Methods: This was a retrospective cohort study of newly diagnosed (FIGO stage II - IV), treatment-naïve patients, ≥ 66 years, who received 4-10 cycles of platinum-based 1L CT between Jan 2009 - Dec 2015 using the SEER-Medicare database, a nationally representative cancer registry. Patient were assumed to have progressed to a subsequent line of therapy following a gap between consecutive CT cycles ≥ 63 days. Patients were classified as GP if alive ≥4 years with no further treatment following 1L CT; PP was defined as receipt of ≥2L CT within 12 months of initial 1L CT. Demographic and prognostic characteristics were assessed during a 6-month baseline period prior to initiation of 1L CT. We assessed clinically meaningful differences in baseline characteristics with absolute standardized differences (ASD) using a threshold of 0.1 (indicating negligible difference between two cohorts). Results: There were a total of 2,262 patients (mean age: 74.6 ±6.2 years) including 251 GP (11%) and 209 PP (9%) patients (table below). PP patients were significantly more likely to be older than 70 years, and present at stage IV, liver disease and ascites, and anemia at diagnosis. PP patients were also less likely to have primary debulking surgery. Conclusions: Approximately one tenth of OC patients received no further treatment 4 years after the initial treatment with contemporary standard of care. GP may be differentiated from PP on the basis of commonly used clinical characteristics such as stage and also specific comorbidities such as liver disease and ascites. Table: see text
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e18087
Background: A critical question in determining long-term prognosis for women with newly diagnosed ovarian cancer (OC) is whether or not their risk of death changes with time. The ...emergence of large, well-populated real-world datasets permits assessment of conditional survival (CS) given prior overall survival (OS) or progression-free survival (PFS). Methods: The Tempus EMR clinical dataset consists of patients from both National Cancer Institute designated centers and a sample of community oncology centers in the U.S. This study included adult women with a primary diagnosis of ovarian, fallopian tube, or peritoneal cancer from 1982 to 2018; women treated with a poly-ADP ribose polymerase (PARP) inhibitor were excluded due to low numbers & limited follow-up (final n = 2,031). The effects of patient attributes on OS were estimated using Cox regression. We calculated CS as the Kaplan-Meier probability of surviving an additional y years (from first line chemotherapy initiation), given no OS or PFS event in the previous x (< y) years. Results: Median age was 61 years and 68% of patients were Caucasian. The majority (92%) had epithelial histology, 58% were stage 3 or 4, and 49% were ECOG 0 or 1. Median OS was 37 months (95% CI: 36-39), and OS differed by age, stage, and performance status; adjusted hazard ratios (HRs) for OS were 1.3 (95% CI: 1.0, 1.5) for age > 65 versus < 45, 2.4 for stage 4 versus stage 1 (95% CI: 1.7, 3.4), and 1.4 for ECOG 2 to 4 versus 0 or 1 (95% CI: 1.2, 1.7). Conditional 1- or 5-year survival rate did not vary based on prior OS. However, CS rates among women alive without disease progression increased with time: 1-year and 5-year survival rates (with 95% CI) were 86% (84-87) and 28% (26-31), respectively, in women alive without progression at 6 months, but increased to 94% (89-97) and 53% (44-62) in women alive without progression at 3 years. Conclusions: Long-term prognosis, as shown by conditional survival rates, did not improve based on time alive since initiation of chemotherapy. However, women with longer time without disease progression had lower rates of death and a better prognosis. Table: see text
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