EGFR Tyrosine Kinase Inhibitors for the Treatment of Metastatic Non-Small Cell Lung Cancer Harboring Uncommon EGFR Mutations: A Podcast.

Supplementary file1 (MP4 21169 KB).

Real-world study of patients with locally advanced HNSCC in the community oncology setting.

Introduction: There is a need to understand the current treatment landscape for LA HNSCC in the real-world setting. Methods: This retrospective study assessed real-world outcomes and treatment patterns of 1,158 adult patients diagnosed with locally advanced (stage III-IVB) HNSCC initiating chemoradiotherapy (CRT) within the period January 2015 to December 2017 in a large network of US community oncology practices. Structured data were abstracted from electronic health records. Demographic, clinical and treatment characteristics were analyzed descriptively overall and stratified by index treatment (cisplatin + radiotherapy [RT], cisplatin + other chemotherapy + RT, or cetuximab + RT). Time to next treatment (TTNT) and overall survival (OS) were measured using the Kaplan-Meier method, and median duration of treatment was assessed. OS was compared across treatment cohorts using multinomial logistic regression with inverse probability treatment weighting. To identify covariates associated with OS, a multivariable adjusted Cox proportional hazard model was used. Results: This study examined 22,782 records, of which 2124 had stage III to stage IVB and no other cancers, and 1158 met all eligibility criteria. Among the treatment cohorts analyzed (cisplatin + RT, cisplatin + other chemotherapy + RT, or cetuximab + RT), cisplatin + RT was the most common concurrent chemotherapy (65.8%). Among 1158 patients, 838 (72.4%) did not initiate subsequent treatment and 139 (12.0%) died. The median TTNT and median OS were only reached by the cetuximab + RT cohort. Among patients with oropharynx primary tumor location, patients with human papilloma virus (HPV) positive status had the longest time on treatment and highest survival at 60 months. Covariates associated with improved survival were never/former tobacco use, HPV positive status, and overweight or obese body mass index. Covariates associated with poorer survival were age of 60+ years, primary tumor location of hypopharynx or oral cavity and Eastern Cooperative Oncology Group performance status score of 2+. Conclusion: These data describe real-world treatment patterns in locally advanced head and neck squamous cell cancer and sets the baseline to assess outcomes for future studies on the community oncology population.

Real-world relationship of early end points to survival end points in patients with resectable non-small-cell lung cancer.

Aim: Pathologic response has been shown to be a promising surrogate for survival in non-small-cell lung cancer. We examined the real-world relationship between these end  points in patients with resectable stage IB-IIIA non-small-cell lung cancer receiving neoadjuvant chemotherapy/chemoradiotherapy (CT/CRT). Methods: Electronic health records/medical charts were analyzed. Overall and event-free survival (OS/EFS) were assessed by Kaplan-Meier stratified by pathologic response. Associations between the end  points were assessed by Cox analyses. Results: A total of 425 patients were selected for the study; 147 and 278 received CT and CRT, respectively. Pathologic complete response (pCR) was associated with longer OS (adjusted HR = 0.50; 95% CI: 0.29-0.85) and EFS (adjusted HR = 0.44; 95% CI: 0.28-0.68) versus no pCR, and EFS was associated with OS (HR = 0.51, 95% CI: 0.38, 0.69). Conclusion: In patients receiving neoadjuvant CT/CRT, pCR and EFS were associated with improved survival in this real-world dataset.

Treatment patterns and outcomes in metastatic synovial sarcoma: a real-world study in the US oncology network.

Aim: To examine and understand patient characteristics, treatment patterns and outcomes for patients with metastatic synovial sarcoma (mSS) treated in a US community setting. Materials & methods: Retrospective observational study in adults with mSS in The US Oncology Network (diagnosed January 2012-December 2018). Results: Of 202 patients diagnosed with synovial sarcoma (SS), 71 had mSS. Of 39 patients with mSS who received first-line (1L) systemic treatment, 25 and 16 continued to 2L and 3L+ treatment, respectively. With each subsequent treatment line, time-to-treatment-discontinuation (1L-3L: 3.9-2.7 months) and time-to-next-treatment (1L-3L: 9.3-4.6 months) decreased. At 1L, median overall survival was 24.5 months. Conclusion: This study highlights the ongoing need for effective therapies for mSS.

Synovial sarcoma (SS) is a rare and aggressive type of soft tissue sarcoma (STS), a group of rare cancers that start in the soft tissues, such as muscle, tendons, fat, lymph and blood vessels and nerves. Usually STS presents in one location, and frequently spreads to other locations, referred to as metastatic SS (mSS). Many studies have explored the characteristics, treatments and outcomes of people with STS. Yet, a limited number of studies have been performed specifically for people with mSS. This study aims to describe characteristics, treatment patterns and clinical outcomes of people with mSS treated in a US community setting. The study showed that more than a third of people diagnosed with SS had disease that spread, mostly to the lung. Of the 71 people with mSS included in the analysis, 39 people received chemotherapy. Of these, 25 people with mSS needed second-line chemotherapy and a further 16 people with mSS required third-line treatment. People with mSS who did not respond well to chemotherapy received a variability of treatments in the US community setting. More lines of treatment were associated with shorter time-to-next-treatment and reduced survival time. Together, these findings highlight the burden of illness and the need for more effective treatments for people with this rare disease. Investigating the characteristics, treatment patterns and clinical outcomes of people with mSS can help to understand the unmet need in this population and pave the way to improving future treatment approaches.

Real-world patterns of biomarker testing and targeted therapy in de novo metastatic non-small cell lung cancer patients in the US oncology network.

BACKGROUND: This study investigated biomarker testing and biomarker-guided treatment among patients with metastatic NSCLC in a real-world setting. METHODS: This retrospective study examined adult patients diagnosed with de novo mNSCLC between 01-Jan-2016 and 30-Sep-2019, with follow-up through 31-Dec-2019 using The US Oncology Network structured electronic health records data, with chart review for a subset. RESULTS: Of 2257 patients, 76.3% had results for ≥1 driver mutation (DM) or programmed death ligand-1 (PD-L1) during the study observation period. The proportion with results for all 4 DM before 1L initiation increased from 2017 to 2019. Over 40% had results for anaplastic lymphoma kinase (ALK), epidermal growth factor receptor (EGFR), and c-ros oncogene 1 (ROS1) and 22% for B-Raf proto-oncogene (BRAF) before 1L initiation by structured data. In the chart review subset (n = 197), >70% had results for ALK, EGFR, or ROS1 with 44% for BRAF. Of the 42 ALK+ patients, 5 had results before 1L treatment and 3 received 1L ALK inhibitors. Similar, for the other biomarkers, not all who tested positive for a DM received 1L targeted therapy. The proportion of biomarker-positive patients receiving 1L targeted therapy was higher in chart review versus structured data. However, in both analyses, a substantial proportion did not have results for all 4 DM plus PD-L1 tests for appropriate biomarker-directed 1L treatment selection. CONCLUSIONS: Despite increasing biomarker testing rates, reduced turnaround times, and availability of promising biomarker-based therapies, inadequate testing in the community oncology setting means that not all eligible patients are receiving the most effective therapies up front.

Safety and Efficacy of Pembrolizumab in Combination with Acalabrutinib in Advanced Head and Neck Squamous Cell Carcinoma: Phase 2 Proof-of-Concept Study.

PURPOSE: Programmed cell death-1 (PD-1) receptor inhibitors have shown efficacy in head and neck squamous cell carcinoma (HNSCC), but treatment failure or secondary resistance occurs in most patients. In preclinical murine carcinoma models, inhibition of Bruton's tyrosine kinase (BTK) induces myeloid cell reprogramming that subsequently bolsters CD8+ T cell responses, resulting in enhanced antitumor activity. This phase 2, multicenter, open-label, randomized study evaluated pembrolizumab (anti-PD-1 monoclonal antibody) plus acalabrutinib (BTK inhibitor) in recurrent or metastatic HNSCC. PATIENTS AND METHODS: Patients received pembrolizumab 200 mg intravenously every 3 weeks, alone or in combination with acalabrutinib 100 mg orally twice daily. Safety and overall response rate (ORR) were co-primary objectives. The secondary objectives were progression-free survival (PFS) and overall survival. RESULTS: Seventy-six patients were evaluated (pembrolizumab, n = 39; pembrolizumab + acalabrutinib, n = 37). Higher frequencies of grade 3-4 treatment-emergent adverse events (AE; 65% vs. 39%) and serious AEs (68% vs. 31%) were observed with combination therapy versus monotherapy. ORR was 18% with monotherapy versus 14% with combination therapy. Median PFS was 2.7 [95% confidence interval (CI), 1.4-6.8] months in the combination arm and 1.7 (95% CI, 1.4-4.0) months in the monotherapy arm. The study was terminated due to lack of clinical benefit with combination treatment. To assess how tumor immune contexture was affected by therapy in patients with pre- and post-treatment biopsies, spatial proteomic analyses were conducted that revealed a trend toward increased CD45+ leukocyte infiltration of tumors from baseline at day 43 with pembrolizumab (monotherapy, n = 5; combination, n = 2), which appeared to be higher in combination-treated patients; however, definitive conclusions could not be drawn due to limited sample size. CONCLUSIONS: Despite lack of clinical efficacy, immune subset analyses suggest that there are additive effects of this combination; however, the associated toxicity limits the feasibility of combination treatment with pembrolizumab and acalabrutinib in patients with recurrent or metastatic HNSCC.

Treatment patterns and clinical outcomes in patients with advanced non-small cell lung cancer initiating first-line treatment in the US community oncology setting: a real-world retrospective observational study.

PURPOSE: Treatments for advanced non-small cell lung cancer (NSCLC) have evolved to include targeted and immuno-oncology therapies, which have demonstrated clinical benefits in clinical trials. However, few real-world studies have evaluated these treatments in the first-line setting. METHODS: Adult patients with advanced NSCLC who initiated first-line treatment with chemotherapy, targeted therapies (TT), or immuno-oncology-based regimens in the US Oncology Network (USON) between March 1, 2015, and August 1, 2018, were included and followed up through February 1, 2019. Data were sourced from structured fields of USON electronic health records. Patient and treatment characteristics were assessed descriptively, with Kaplan-Meier methods used to evaluate time-to-event outcomes, including time to treatment discontinuation (TTD) and overall survival (OS). Adjusted Cox regression analyses and inverse probability of treatment weighting (IPTW) were performed to control for covariates that may have affected treatment selection and outcomes. RESULTS: Of 7746 patients, 75.6% received first-line systemic chemotherapy, 11.7% received immuno-oncology monotherapies, 8.5% received TT, and 4.2% received immuno-oncology combination regimens. Patients who received immuno-oncology monotherapies had the longest median TTD (3.5 months; 95% confidence interval [CI], 2.8-4.2) and OS (19.9 months; 95% CI, 16.6-24.1). On the basis of multivariable Cox regression and IPTW, immuno-oncology monotherapy was associated with reduced risk of death and treatment discontinuation relative to other treatments. CONCLUSION: These results suggest that real-world outcomes in this community oncology setting improved with the introduction of immuno-oncology therapies. However, clinical benefits are limited in certain subgroups and tend to be reduced compared with clinical trial observations.

Real-world disease burden and outcomes of brain metastases in EGFR mutation-positive non-small-cell lung cancer.

Aim: To evaluate the real-world impact of brain metastases (BM) among patients with EGFR mutation-positive (EGFRm) metastatic non-small-cell lung cancer (NSCLC). Materials & methods: This retrospective, observational matched cohort electronic health record study assessed adults with EGFRm metastatic NSCLC with/without BM. Results: Among 402 patients split equally between both cohorts (±BM), the majority were Caucasian (69%), female (65%) and with adenocarcinoma (92%). Overall symptom burden and ancillary support service use were higher and median overall survival from metastatic diagnosis was significantly shorter in BM patients (11.9 vs 16 months; p = 0.017). Conclusion: BM in EGFRm NSCLC patients can negatively impact clinical outcomes. New targeted therapies that can penetrate the blood-brain barrier should be considered for treating these patients.

Treatment Patterns and Healthcare Resource Utilization among Patients with Advanced or Metastatic Soft Tissue Sarcoma in US Community Practices.

INTRODUCTION: This study was designed to describe demographic and clinical characteristics of patients diagnosed with advanced or metastatic soft tissue sarcoma (STS) and to examine treatment and healthcare resource utilization patterns of this patient population in a United States (US) community-based oncology practice setting over time. METHODS AND MATERIALS: A retrospective observational study was conducted within the US Oncology Network (USON). Patients were eligible if they were diagnosed with advanced or metastatic STS and were treated at a USON site between 01 July 2015 and 31 August 2018. Demographic, clinical, and treatment characteristics were described for the overall study population. Comparisons between patients by time period (prior to and after October 2016) were evaluated using the T test for continuous variables and chi-squared test for categorical variables. Data were available for analysis through 31 August 2018. RESULTS: Demographic and clinical characteristics of the eligible study cohort (N = 376) were similar between patients who initiated treatment before and after October 2016 (all p > 0.05). Forty-three unique regimens were observed in the first-line setting, with the predominant regimen (gemcitabine + docetaxel) received by 33.2% (n = 125) patients. Prior to October 2016, 45.4% of patients received first-line gemcitabine + docetaxel, while 29.0% received this regimen after October 2016. CONCLUSIONS: While demographic and clinical characteristics were similar, treatment patterns changed in 2016. Future research should evaluate the impact of changing drug approvals and clinical trial results on future treatment patterns.

Observational Study of Treatment Patterns in Patients with Epidermal Growth Factor Receptor (EGFR) Mutation-Positive Non-Small Cell Lung Cancer After First-Line EGFR-Tyrosine Kinase Inhibitors.

INTRODUCTION: Epidermal growth factor receptor (EGFR) mutations are observed in approximately 15% of patients with non-small cell lung cancer (NSCLC) in the USA. Little is known about treatment patterns in EGFR mutation-positive NSCLC following progression on or after first-line (1L) treatment with first- or second-generation EGFR tyrosine kinase inhibitors (EGFR-TKIs). Osimertinib, a third-generation EGFR-TKI, is a treatment option for patients with EGFR T790M-positive NSCLC following progression on 1L EGFR-TKIs. This study analyzed real-world treatment sequencing of EGFR-TKIs, EGFR T790M testing rates, and disposition of patients with EGFR mutations after 1L EGFR-TKI post-FDA approval of osimertinib in patients with EGFR mutation-positive NSCLC. METHODS: Adult patients with stage IV NSCLC and documented EGFR mutation-positive status were identified between December 1, 2015 and May 31, 2017 from the US Oncology Network iKnowMed™ electronic health record (EHR). Data were abstracted from the EHR database and supplemented by chart review. RESULTS: Of 308 patients, 302 (98%) received an EGFR-TKI overall, and 246 patients (80%) received a 1L EGFR-TKI. The most common 1L EGFR-TKI was erlotinib (66%); the remaining 1L regimens were predominantly combination chemotherapies with or without an EGFR-TKI. Only 80 patients (26%) received any 2L therapy. The most common EGFR-TKIs used as 2L monotherapy in patients who received 1L EGFR-TKI were afatinib and osimertinib (n = 7 for both). Among all patients treated with 1L EGFR-TKI (n = 246), 47 (19%) were tested for EGFR T790M [16 patients (34%) were positive], 48 (20%) remained on 1L EGFR-TKI, 29 (12%) received subsequent therapy, 38 (15%) had died on or after their 1L EGFR-TKI therapy, and 131 (53%) stopped their EGFR-TKI with no recorded evidence of having received subsequent therapy at follow-up end. CONCLUSION: Following 1L EGFR-TKI treatment, 19% of patients were tested for EGFR T790M, and most (69%) had no record of receiving any subsequent therapy.

Treatment patterns and outcomes among patients with recurrent/metastatic squamous cell carcinoma of the head and neck.

AIM: Cetuximab was approved in 2008 for treating recurrent/metastatic (R/M) head-and-neck squamous-cell carcinoma (HNSCC), and this study assessed the utilization of cetuximab for R/M-HNSCC in a real-world setting. MATERIALS & METHODS: Adult patients with R/M-HNSCC, who initiated systemic therapy between 1 September 2011 and 31 December 2014 and followed through 31 December 2015, were identified from iKnowMed electronic-health-records database (McKesson Specialty Health) supplemented with manual chart-abstraction. RESULTS: For 325 R/M-HNSCC patients; median age 62 years; 82% males, 67% had oropharyngeal cancer, most common first-line (1L) regimen was platinum-based combinations (76%), of whom only 8% received platinum + cetuximab +/- 5-fluorouracil. CONCLUSION: Despite US FDA approval and National Comprehensive Cancer Network guidelines recommending use of cetuximab for palliative treatment of R/M-HNSCC, our study demonstrates low utilization in 1L and 2L settings, underscoring the need to understand reasons for low utilization.

Treatment Patterns by EGFR Mutation Status in Non-Small Cell Lung Cancer Patients in the USA: A Retrospective Database Analysis.

INTRODUCTION: Targeted therapies, including tyrosine kinase inhibitors (TKIs) that target the sensitizing epidermal growth factor receptor (EGFR) gene are recommended for patients with non-small cell lung cancer (NSCLC). Most patients with NSCLC who test positive for the EGFR mutation and receive TKIs develop resistance to these drugs. Questions remain regarding which treatment sequence is optimal for patients with EGFR-mutant NSCLC, and few studies have evaluated patterns of TKI treatment use in NSCLC, irrespective of EGFR mutation status, in a real-world setting. This population-based study aimed to evaluate treatment patterns at a national level in the USA. METHODS: This retrospective observational study used data from the US Oncology Network's iKnowMed database. Patients with advanced NSCLC who initiated first-line therapy with erlotinib and/or intravenous chemotherapy between January 1, 2012 and June 30, 2015 and met all other study criteria were included. Descriptive analyses assessed demographic and clinical characteristics and treatment patterns among the overall study cohort, as well as for specific erlotinib treatment subgroups, stratified by EGFR status. RESULTS: Among the 3108 patients identified, 18.5% were EGFR positive, 49.8% were EGFR negative, and 31.7% were EGFR documented unknown. For the overall cohort, 18.4% received first-line erlotinib monotherapy, fewer than 1% received first-line combination therapy (erlotinib plus chemotherapy), 4.7% received second-line erlotinib monotherapy, and 3.3% received second-line combination therapy. First-line erlotinib monotherapy was used in 77.8% of all EGFR positive patients. Almost two-thirds of the overall cohort were not observed to have advanced to second-line therapy. CONCLUSIONS: As treatment options evolve, this study provides real-world treatment patterns that suggest concordance with NCCN guidelines and confirm the remaining need to understand sequencing of therapies and related outcomes. FUNDING: Eli Lilly and Company.

Treatment Patterns and Clinical Outcomes Among Metastatic Non-Small-Cell Lung Cancer Patients Treated in the Community Practice Setting.

INTRODUCTION: Multiple therapeutic options now exist for metastatic non-small-cell lung cancer (mNSCLC). In this study we evaluated treatment patterns and outcomes in mNSCLC patients who received first-line (1L), second-line (2L), and third-line (3L) therapy. PATIENTS AND METHODS: A retrospective, observational cohort study was conducted using an electronic health record database of mNSCLC patients who received initial treatment from January 2012 through April 2016, with follow-up through June 2016. Patient characteristics and treatment patterns were characterized. Overall survival (OS) was assessed using the Kaplan-Meier method. RESULTS: We identified 10,689 1L patients. Median age was 68 years, and 5816 (54%) were male. Most patients (6337; 59%) had a performance status of 1, and 8282 (77%) had nonsquamous histology. 1L treatment was chemotherapy in 9969 (93%) patients, and targeted therapy in 685 (6%). Median OS (mOS) for all patients in 1L was 12.3 months (95% confidence interval [CI], 11.9-12.7), and 24.3 months in 1L patients receiving targeted therapy. Among patients who received 2L therapy (n = 4235), 2790 (66%), 718 (17%), and 727 (17%) received chemotherapy, targeted therapy, and immunotherapy, respectively. mOS from 2L therapy was 9.6 months (95% CI, 9.1-10.1). In patients receiving 3L therapy (n = 1580), 921 (58%), 355 (22%), and 304 (19%) received chemotherapy, targeted therapy, and immunotherapy, respectively. mOS from 3L therapy was 8.2 months (95% CI, 7.3-8.7). CONCLUSION: Targeted therapy and immunotherapy was most frequently used in the 2L and 3L setting during the study time frame. Survival differences observed according to treatment types are likely because of biologic differences, and suggest that patients with actionable mutations have a survival advantage.

Clinical Pathways and the Patient Perspective in the Pursuit of Value-Based Oncology Care.

The art of practicing oncology has evolved substantially in the past 5 years. As more and more diagnostic tests, biomarker-directed therapies, and immunotherapies make their way to the oncology marketplace, oncologists will find it increasingly difficult to keep up with the many therapeutic options. Additionally, the cost of cancer care seems to be increasing. Clinical pathways are a systematic way to organize and display detailed, evidence-based treatment options and assist the practitioner with best practice. When selecting which treatment regimens to include on a clinical pathway, considerations must include the efficacy and safety, as well as costs, of the therapy. Pathway treatment regimens must be continually assessed and modified to ensure that the most up-to-date, high-quality options are incorporated. Value-based models, such as the ASCO Value Framework, can assist providers in presenting economic evaluations of clinical pathway treatment options to patients, thus allowing the patient to decide the overall value of each treatment regimen. Although oncologists and pathway developers can decide which treatment regimens to include on a clinical pathway based on the efficacy of the treatment, assessment of the value of that treatment regimen ultimately lies with the patient. Patient definitions of value will be an important component to enhancing current value-based oncology care models and incorporating new, high-quality, value-based therapeutics into oncology clinical pathways.

Time of day effects on railroad roadway worker injury risk.

INTRODUCTION: The purpose of this study is to examine how time of day affects injury risk of railroad maintenance of way employees and signalmen (roadway workers). Railroads reported 15,654 serious roadway worker injuries between 1997 and 2014. Roadway workers primarily work outdoors on or near railroad tracks and frequently encounter hazardous conditions. To avoid closing an active rail line during peak hours, railroads sometimes require roadway workers to work at night. Previous studies of roadway worker injury have not adequately accounted for exposure to time of day effects, nor have they investigated the human factors issues contributing to roadway worker injury. METHOD: The Federal Railroad Administration (FRA) database of injury reports provided data for circadian rhythm models of the odds of fatal and nonfatal injuries. The FRA database and fatal injury investigation reports also permitted an analysis of the circumstances and the human factors issues associated with injuries that occur at different times of day. RESULTS: Odds of injury increased during nighttime work. The odds of nonfatal injury for both roadway worker crafts rose above 9:1 in the early morning hours. The relative odds of a fatal injury also increased significantly at night. A human factors analysis suggested that during all three shifts most nonfatal injuries involve workload, but workload was not identified as a factor in fatal injuries. CONCLUSIONS: Nighttime work is more hazardous for roadway workers than daytime work. Several factors related to fatigue and other conditions appear to increase the risk of injury during the outdoor, nighttime work required of roadway workers. PRACTICAL APPLICATION: For practical reasons, nighttime roadway work is sometimes unavoidable. Therefore, new practices for nighttime work must be developed to adequately address fatigue and protect roadway workers from harm.

Epidemiology, treatment patterns, and outcomes of metastatic soft tissue sarcoma in a community-based oncology network.

Purpose. To assess epidemiology, treatment patterns, and outcomes of metastatic soft tissue sarcoma (mSTS) patients in USA community oncology practices. Methods. This retrospective, descriptive study used US Oncology's iKnowMed electronic health records database. Adults (≥18 years) with mSTS and at least two visits between July 2007 and June 2010 were included. Key outcomes were practice patterns, overall survival (OS), and progression-free survival (PFS). Results. 363 mSTS patients (174 treated and 189 untreated) met the prespecified exclusion/inclusion criteria. The most common subtypes were leiomyosarcoma (n = 104; 29%), liposarcoma (n = 40; 11%), and synovial sarcoma (n = 12; 3%); the remainder (n = 207; 57%) comprised 27 histologic subtypes. Treated patients were younger and had lower ECOG scores; 75% and 25% received first-line combination or monotherapy, respectively. Median OS of treated and untreated patients was 22 and 17 months, respectively, and 29 months in patients with the three most common subtypes. Before controlling for effects of covariates, younger age and lower ECOG scores were associated with better OS and PFS. Conclusion. This study provides insights into mSTS epidemiology, treatment patterns, and outcomes in a large community-based oncology network. These results warrant further studies with larger cohorts.

Does framing of cancer survival affect perceived value of care? A willingness-to-pay survey of US residents.

AIMS: To investigate the relationship between the framing of survival gains and the perceived value of cancer care. METHODS: Through a population-based survey of 2040 US adults, respondents were randomized to one of the two sets of hypothetical scenarios, each of which described the survival benefit for a new treatment as either an increase in median survival time (median survival), or an increase in the probability of survival for a given length of time (landmark survival). Each respondent was presented with two randomly selected scenarios with different prognosis and survival improvements, and asked about their willingness to pay (WTP) for the new treatments. RESULTS: Predicted WTP increased with survival benefits and respondents' income, regardless of how survival benefits were described. Framing therapeutic benefits as improvements in landmark rather than median time survival increased the proportion of the population willing to pay for that gain by 11-35%, and the mean WTP amount by 42-72% in the scenarios we compared. CONCLUSION: How survival benefits are described may influence the value people place on cancer care.

In a survey, marked inconsistency in how oncologists judged value of high-cost cancer drugs in relation to gains in survival.

Amid calls for physicians to become better stewards of the nation's health care resources, it is important to gain insight into how physicians think about the cost-effectiveness of new treatments. Expensive new cancer treatments that can extend life raise questions about whether physicians are prepared to make "value for money" trade-offs when treating patients. We asked oncologists in the United States and Canada how much benefit, in additional months of life expectancy, a new drug would need to provide to justify its cost and warrant its use in an individual patient. The majority of oncologists agreed that a new cancer treatment that might add a year to a patient's life would be worthwhile if the cost was less than $100,000. But when given a hypothetical case of an individual patient to review, the oncologists also endorsed a hypothetical drug whose cost might be as high as $250,000 per life-year gained. The results show that oncologists are not consistent in deciding how many months an expensive new therapy should extend a person's life before the cost of therapy is justified. Moreover, the benefit that oncologists demand from new treatments in terms of length of survival does not necessarily increase according to the price of the treatment. The findings suggest that policy makers should find ways to improve how physicians are educated on the use of cost-effectiveness information and to influence physician decision making through clinical guidelines that incorporate cost-effectiveness information.