US Cancer Population Research Articles

Association of patient socioeconomic status with outcomes after palliative treatment for disseminated cancer.

Palliative treatment has been associated with improved quality of life and survival for a wide variety of metastatic cancers. However, it is unclear whether the benefits of palliative treatment are uniformly experienced across the US cancer population. We evaluated patterns and outcomes of palliative treatment based on socioeconomic, sociodemographic and treating facility characteristics. Patients diagnosed between 2008 and 2019 with Stage IV primary cancer of nine organ sites were analyzed in the National Cancer Database. The association between identified variables, and outcomes concerning the administration of palliative treatment were analyzed with multivariable logistic regression and Cox proportional hazard models. Overall 238,995 (23.6%) of Stage IV patients received palliative treatment, which increased over time for all cancers (from 20.7% in 2008 to 25.6% in 2019). Palliative treatment utilization differed significantly by region (West less than Northeast, OR: 0.55 [0.54-0.56], p < 0.001) and insurance payer status (uninsured greater than private insurance, OR: 1.35 [1.32-1.39], p < 0.001). Black race and Hispanic ethnicity were also associated with lower rates of palliative treatment compared to White and non-Hispanics respectively (OR for Blacks: 0.91 [0.90-0.93], p < 0.001 and OR for Hispanics: 0.79 [0.77-0.81] p < 0.001). There are important differences in the utilization of palliative treatment across different populations in the United States. A better understanding of variability in palliative treatment use and outcomes may identify opportunities to improve informed decision making and optimize quality of care at the end-of-life.

Increasing diversity in clinical trials: demographic trends at the National Cancer Institute, 2005-2020.

We described participant demographics for National Cancer Institute (NCI) clinical trials at the clinical center (NCI-CC participants) of the National Institutes of Health to identify enrollment disparities. We analyzed NCI-CC data from 2005 to 2020, calculated enrollment fractions, compared with the US cancer population represented by the Surveillance, Epidemiology, and End Results cancer incidence data (2018) and the Cancer in North America database (2018), and compared further with clinical trial disparities data from the NCI Community Oncology Research Program and National Clinical Trials Network (2005-2019), and from ClinicalTrials.gov (2003-2016). NCI-CC (38 531 participants) had higher enrollment fractions for older adults (8.5%), male (5.6%), non-Hispanic (5.1%), and Black or African American (5.3%) participants; lower women proportion across race and ethnicity; and fewer female sex-specific cancer (6.8%) than male sex-specific cancer (11.7%) participants. NCI-CC had lower median age than Surveillance, Epidemiology, and End Results (54.0 vs 65.4); more Black or African American participants (12.0% vs 11.1%); and fewer women (41.7% vs 49.5%), White (76.1% vs 80.5%), Asian or Pacific Islander (4.6% vs 6.0%), American Indian or Alaska Native (0.3% vs 0.5%), and Hispanic participants (7.1% vs 13%). NCI-CC had more Black or African American and Asian or Pacific Islander participants; fewer Hispanic participants than the NCI Community Oncology Research Program and National Clinical Trials Network; more Black or African American and Hispanic participants; fewer Asian or Pacific Islander participants than ClinicalTrials.gov data. Improvement was noted for NCI-CC (older adults, Black or African American, Asian or Pacific Islander, Hispanic participants). We found lower representation of older adults, women, Asian or Pacific Islander, American Indian or Alaska Native, and Hispanic participants vs the US cancer population and higher representation of Black or African American vs US cancer population and oncology clinical trials. Multifaceted efforts are underway to reduce disparities in cancer clinical trials at the NCI-CC.

Data transparency and demographic representation in approval packages of FDA-approved oncology drugs from 2012-2021.

1594 Background: Over the past decade, the U.S. Food and Drug Administration (FDA) has leveraged regulations and guidance documents to accelerate improvements in demographic reporting and encourage adequate representation of minorities in clinical trials. This cross-sectional study aims to assess the impact of these interventions by characterizing demographic data reporting and representation by sex, age, and racial and ethnic identity in pivotal trials supporting novel cancer therapeutics approved by the FDA from 2012 through 2021. Methods: Study demographic data were abstracted from FDA drug approval packages and US cancer population demographic data was abstracted from US Cancer Statistics. The percentages of trials reporting sex, age, racial and ethnic identity of participants in drug approval packages were calculated and participation to prevalence ratios (PPRs) were calculated by dividing the percentage of participants in each demographic by the percentage of the US cancer population in each group. PPRs were constructed for each pivotal trial by cancer type. Adequate representation was defined as PPR 0.8-1.2 and the proportion of trials which were adequately representative of each demographic was calculated and trended over time. Results: From 2012 through 2021, the FDA approved 118 cancer therapeutics, based on 131 pivotal trials. For drugs approved 2012-2016, the majority of associated pivotal trials reported participation based on sex (100% of trials), age (100%) and White (98%), Black (75%) or Asian (84%) racial identity, but not American Indian or Alaskan Native racial identity (18%) or Hispanic ethnic identity (41%). For drugs approved 2017-2021, there were no significant improvements in demographic data reporting, although there was a trend towards increased reporting on Hispanic ethnic identity from 2012-2016 (41%) to 2017-2021 (65%) (p= 0.06). For drugs approved 2012-2016, most pivotal trials adequately represented female participants (78% of trials) and participants identifying as White (87%), but few pivotal trials adequately represented older adults (7%), participants identifying as Black (11%), American Indian or Alaskan Native (0%), Asian (18%) or Hispanic (5%). Representation in pivotal trials did not significantly improve for any demographic for drugs approved 2017-2021 (p&gt;0.05). Conclusions: This study found that the last decade’s efforts to improve demographic data transparency and representation in clinical trials have not yet borne fruit for novel cancer therapeutics. More consequential FDA regulations may be necessary. Beyond the FDA, demographic data transparency and adequate minority representation in oncology clinical trials is an all-stakeholder responsibility. A more just clinical trial infrastructure requires greater collaboration between the FDA, sponsors, enrollers, contract research organizations and cooperative groups.

Race and Ethnicity Reporting and Enrollment Disparities in Clinical Trials Leading to FDA Approvals for Breast Cancer Between 2010 and 2020

BackgroundWe determined the race and ethnicity demographics and reporting trends of clinical trials leading to Food and Drug Administration (FDA) approvals for breast cancer. MethodsWe collected enrollment and reporting data from clinical trials leading to FDA novel and new use approvals for breast cancer from 2010 to 2020 from Drugs@FDA, ClinicalTrials.gov, and associated journal manuscripts. Enrollment demographics were compared to the US cancer population estimates obtained using National Cancer Institute-Surveillance, Epidemiology, and End Results and 2010 US Census databases. ResultsSeventeen drugs received approval based on 18 clinical trials with a total enrollment of 12,334. For approvals from 2010 to 2015 and from 2016 to 2020, there was no significant difference in race (80% vs. 91.6%, P = .34) or ethnicity reporting (20% vs. 33.3%, P = .5) on ClinicalTrials.Gov, manuscripts, and FDA labels. For trials that reported race and ethnicity, White, Asian, Black, and Hispanic patients represented 73.8%, 16.4%, 3.7%, and 10.4% of trial participants. Relative to their US cancer incidence, Black (31% of expected) patients were underrepresented compared with White (90% of expected), Hispanic (115%), and Asian (327% of expected) patients. ConclusionWe observed no significant difference in race and ethnicity reporting in pivotal clinical trials leading to FDA approval for breast cancer from 2010 to 2020. Black patients were underrepresented in these pivotal trials relative to White, Hispanic, and Asian patients. Ethnicity reporting remained low throughout the study period. Innovative approaches are needed to ensure equitable benefit of novel therapeutics.

Metrics, baseline scores, and a tool to improve sponsor performance on clinical trial diversity: retrospective cross sectional study

ObjectiveTo develop a measure for fair inclusion in pivotal trials by assessing transparency and representation of enrolled women, older adults (aged 65 years and older), and racially and ethnically minoritized...

Population, Clinical, and Scientific Impact of National Cancer Institute's National Clinical Trials Network Treatment Studies.

In the United States, the National Cancer Institute National Cancer Clinical Trials Network (NCTN) groups have conducted publicly funded oncology research for 50 years. The combined impact of all adult network group trials has never been systematically examined. We identified randomized, phase III trials from the adult NCTN groups, reported from 1980 onward, with statistically significant findings for ≥ 1 clinical, time-dependent outcomes. In the subset of trials in which the experimental arm improved overall survival, gains in population life-years were estimated by deriving trial-specific hazard functions and hazard ratios to estimate the experimental treatment benefit and then mapping this trial-level benefit onto the US cancer population using registry and life-table data. Scientific impact was based on citation data from Google Scholar. Federal investment costs per life-year gained were estimated. The results were derived through December 31, 2020. One hundred sixty-two trials comprised of 108,334 patients were analyzed, representing 29.8% (162/544) of trials conducted. The most common cancers included breast (34), gynecologic (28), and lung (14). The trials were cited 165,336 times (mean, 62.2 citations/trial/year); 87.7% of trials were cited in cancer care guidelines in favor of the recommended treatment. These studies were estimated to have generated 14.2 million (95% CI, 11.5 to 16.5 million) additional life-years to patients with cancer, with projected gains of 24.1 million (95% CI, 19.7 to 28.2 million) life-years by 2030. The federal investment cost per life-year gained through 2020 was $326 in US dollars. NCTN randomized trials have been widely cited and are routinely included in clinical guidelines. Moreover, their conduct has predicted substantial improvements in overall survival in the United States for patients with oncologic disease, suggesting they have contributed meaningfully to this nation's health. These findings demonstrate the critical role of government-sponsored research in extending the lives of patients with cancer.

Association of medical uninsurance with sociodemographic attributes in US cancer population: A cross-sectional study of NHANES data 2013 to 2018.

Medical uninsurance (MU) is associated with cancer disparities, particularly among underprivileged and minority sections of the United States. In this cross-sectional study of National Health and Nutritional Examination Survey (NHANES) data from 2013 to 2018, we evaluated sociodemographic attributes of MU disparity in the US cancer population. Those aged ≥20 years with a history of cancer and disclosed MU status were included. We calculated the descriptive statistics of the population stratified by insurance type and performed bivariate and multivariate logistic regression models to assess the association of sociodemographic attributes and MU and reported unadjusted (UOR) and adjusted odds ratios (AOR). Among the 1681 participants (US estimated, 25,982,352), 4.3% ± 0.62 were uninsured. Uninsured individuals were 13.5-year younger, largely female, less educated, and non-US born compared to insured individuals. Age (UOR: 0.94, 95% CI: 0.93–0.96), female sex (UOR: 3.53, 95% CI: 1.73–7.19), Hispanics (UOR: 4.30, 95% CI: 2.45–7.54), <high school education (UOR: 7.41, 95% CI: 2.51–21.86), and non-US born with <20-years-stay in US (UOR: 7.69, 95% CI: 3.32–17.82) were associated with MU. In the multivariate model, age (AOR: 0.95, 95% CI: 0.93–0.96), female sex (AOR: 2.88, 95% CI: 1.25–6.62), <high school education (AOR: 4.02, 95% CI: 1.24–13.00), and non-US-born status with <20-years stay (AOR: 3.42, 95% CI: 1.44–8.11) were independent predictors of MU. Income was not a predictor of MU. The US cancer population has unique determinants of MU. Ethnicity alone is not a predictor of MU, whereas income is not correlated with MU. Public health interventions focusing on the attributes of MU are needed.

MP19-03 COMPARISON OF FACT-G SCORES WITH CANCER AND GENERAL POPULATION NORMS IN CANADIAN PATIENTS UNDERGOING RADICAL CYSTECTOMY

You have accessJournal of UrologyHealth Services Research: Practice Patterns, Quality of Life and Shared Decision Making II (MP19)1 Sep 2021MP19-03 COMPARISON OF FACT-G SCORES WITH CANCER AND GENERAL POPULATION NORMS IN CANADIAN PATIENTS UNDERGOING RADICAL CYSTECTOMY Julie Trudel, Ryan McLarty, and Conrad Maciejewski Julie TrudelJulie Trudel More articles by this author , Ryan McLartyRyan McLarty More articles by this author , and Conrad MaciejewskiConrad Maciejewski More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002004.03AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: The Functional Assessment of Cancer Therapy-General (FACT-G) is often used as a generic measure in Health-Related Quality of Life (HRQOL) assessment among cancer patients. Its reference values relating to the general population and patients with different diseases are useful for the interpretation of HRQOL scores and allow a better understanding of the patients needs and HRQOL. No study to date has yet compared FACT-G normative data scores to those of Canadian patients undergoing radical cystectomy (RC) for bladder cancer. The objective of this study was to compare our patients pre-cystectomy FACT-G scores with US cancer and general population norms. METHODS: Patients were recruited from the Diana Wood Cancer Survivorship Clinic at The Ottawa Hospital between January 2019 and August 2020. HRQOL was evaluated by the FACT-G and raw scores were obtained for each FACT-G subscale, higher scores indicating better HRQOL. Effect sizes (ES-Hedge's g statistics), confidence intervals and minimal clinical significant differences (MCIDs) were used to compare pre FACT-G subscale scores with normative data. As suggested by current literature, a change of at least 5% to 10% within the instrument range was used as an estimate calculation of the MCID. RESULTS: 55 patients completed the FACT-G questionnaire before RC. The mean age was 67 years (SD: 10 years), 71% were male, 80% were in a married/common law partnership and 84% lived with someone. Overall, ES between pre-surgery FACT-G subscale scores and US cancer norms were small (ES≤0.2) and the MCIDs were less than 5% of change in the FACT-G measurement scale except for the dimension “Physical Well-Being” (ES=0.3, reported MCID value of 1.77, 6% of change). ES calculated between FACT-G scores and those from the US general population showed small effect sizes (ES≤0.2) for all FACT-G subscales except for the dimensions “Emotional Well-Being” (Medium ES=-0.56, reported MCID value of 2.7, 11% of change) and “Social Well-Being” (Medium ES=0.37, reported MCID value of 2.5, 9% of change). CONCLUSIONS: In our survivorship clinic, patients undergoing RC reported broadly similar FACT-G scores to US cancer population norms, with a notable exception of increased perceived “Physical Well-Being”. When comparing our data to US population norms, we once again showed similar scores on FACT-G subscales with the exception of decreased “Emotional Well-Being” and higher “Social Well-Being”. These results emphasize unique aspects of the bladder cancer survivorship experience, and highlight the value of patient reported assessment of HRQOL in RC patients. Source of Funding: The Diana Wood Bladder Cancer Survivorship Program © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 206Issue Supplement 3September 2021Page: e328-e328 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.MetricsAuthor Information Julie Trudel More articles by this author Ryan McLarty More articles by this author Conrad Maciejewski More articles by this author Expand All Advertisement Loading ...

Adolescent and young adult enrollment to a National Cancer Institute-sponsored National Clinical Trials Network Research Group over 25 years.

Adolescent and young adult (AYA) patients with cancer have not seen the same improvements in survival as younger (pediatric) patients and older patients (adults 40 years old or older). This may be related to their lower participation in clinical trials. This study examined AYA patient accrual to SWOG Cancer Research Network phase 1 to 3 clinical treatment trials for 29 cancers over 25 years (January 1996 to December 2020). Trial enrollments for AYA patients (15-39 years old) were compared with trial enrollments for older patients (40 years old or older) in SWOG and with US AYA cancer population rates derived from US Census and National Cancer Institute/Surveillance, Epidemiology, and End Results data. In total, 84,219 patients were enrolled in SWOG treatment trials, including 7109 AYA patients (8.4%); in contrast, AYAs constituted 3.8% of the US cancer population. By histology, the highest proportions of AYA patients were in trials for Hodgkin disease (825 of 1220; 67.6%) and acute lymphocytic leukemia (350 of 678; 51.6%), whereas breast cancer trials had the greatest number of AYA patients (3032 of 32,693; 9.3%). SWOG AYA patients were more often female (68.8% vs 58.7%; P < .001), Black (10.1% vs 8.2%; P < .001), and Hispanic (10.6% vs 5.6%; P < .001) than SWOG patients who were 40 years old or older, and they were more often female (68.8% vs 65.1%; P < .001) but less often Black (10.1% vs 11.8%; P < .001) or Hispanic (10.6% vs 12.8%; P < .001) than AYA patients in the US cancer population. AYA patients with cancer were well represented in SWOG clinical trials in comparison with US cancer population patients with the same cancers. The SWOG AYA population was more racially/ethnically diverse than older SWOG patients, although it was less diverse than the US AYA cancer population. Adolescent and young adult (AYA) patients with cancer (aged 15-39 years) have not seen the same improvements in survival as younger (pediatric) patients and older patients (adults 40 years old or older). This may be related to their lower participation in clinical trials. This study evaluated the extent to which AYA patients were enrolled in a large, National Cancer Institute-sponsored network group over 25 years (1996-2020). Overall, 8.4% of the enrolled patients (7109 of 84,219) were AYAs; this was twice the corresponding rate of 3.8% in the US cancer population. AYA patients were also more racially/ethnically diverse than older trial patients, although they were less racially/ethnically diverse than the US AYA cancer population.

Healthcare access disparity among U.S. cancer population and evaluation of social determinants of health using NHANES data for 2013-18.

e18527 Background: Ethnic minorities in US have poorer outcomes due to poor socioeconomic determinants. We evaluated association between ethnicity and insurance status among US adults with cancer who participated in National Health and Nutrition Examination survey for 2013-18. Methods: This was a cross-sectional study involving participants with history of cancer; participants with missing insurance/ethnicity information were excluded. Outcome of interest was insurance coverage in minorities vs Whites. The comparison of baseline characteristics (Table) stratified by insurance (Yes vs No) was made using Chi-square and t-tests incorporating survey procedures in SAS v. 9.4. Logistic regression was used for adjusted and unadjusted odds ratio (OR). Results: Among 29,400 participants, 1,684 had cancer. After excluding 81 participants with missing data, 1,603 were included; 5.2% were uninsured. Table summarizes characteristics of participants stratified by insurance. The uninsured individuals were 14 years younger, predominantly females, Hispanics and non-US born, and were less educated compared to the insured. Unadjusted OR of no insurance vs insurance was 4.23 (95%CI: 2.40-7.47) for Hispanics, 1.23 (95%CI: 0.64- 2.38) for Blacks and 1.50 for others (95%CI: 0.48-4.74) when compared to Whites. After adjusting for age, sex, education, US born status and routine place for medical care, OR for Hispanics, Blacks and others were 1.31 (95%CI: 0.73-2.36), 1.00 (95%CI: 0.46-2.18) and 0.86 (95%CI: 0.26-2.78), respectively. Conclusions: Poor socioeconomic indicators among US cancer population may leave them being uninsured; thereby making them vulnerable to poor outcomes in particular in the ethnic minorities including Hispanics. [Table: see text]

Reporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics

Adequate representation of demographic subgroups in premarketing and postmarketing clinical studies is necessary for understanding the safety and efficacy associated with novel cancer therapeutics. To characterize and compare the reporting of demographic data and the representation of individuals by sex, age, and race in premarketing and postmarketing studies used by the Food and Drug Administration (FDA) to evaluate novel cancer therapeutics. In this cross-sectional study, premarketing and postmarketing studies for novel cancer therapeutics approved by the FDA from 2012 through 2016 were identified. Study demographic information was abstracted from publicly available sources, and US cancer population demographic data was abstracted from US Cancer Statistics. Analyses were conducted from February 25 through September 21, 2020. The percentages of trials reporting sex, age, and race/ethnicity were calculated, and participation to prevalence ratios (PPRs) were calculated by dividing the percentage of study participants in each demographic group by the percentage of the US cancer population in each group. PPRs were constructed for premarketing and postmarketing studies and by cancer type. Underrepresentation was defined as PPR less than 0.8. From 2012 through 2016, the FDA approved 45 cancer therapeutics. The study sample included 77 premarketing studies and 56 postmarketing studies. Postmarketing studies, compared with premarketing studies, were less likely to report patient sex (42 studies reporting [75.0%] vs 77 studies reporting [100%]; P < .001) and race (27 studies reporting [48.2%] vs 62 studies reporting [80.5%]; P < .001). Women were adequately represented in premarketing studies (mean [SD] PPR, 0.91; 95% CI, 0.90-0.91) and postmarketing studies (mean PPR, 1.00; 95% CI, 1.00-1.01). Although older adults and Black patients were underrepresented in premarketing studies (older adults: mean PPR, 0.73; 95% CI, 0.72-0.74; Black patients: mean PPR, 0.32; 95% CI, 0.31-0.32), these groups continued to be underrepresented in postmarketing studies (older adults: mean PPR, 0.75; 95% CI, 0.75-0.76; Black patients: mean PPR, 0.21; 95% CI, 0.21-0.21). This study found that older adults and Black patients were underrepresented in postmarketing studies of novel cancer therapeutics to a similar degree that they were underrepresented in premarketing studies. These findings suggest that postmarketing studies are not associated with improvements to gaps in demographic representation present at the time of FDA approval.

Representativeness of Black Patients in Cancer Clinical Trials Sponsored by the National Cancer Institute Compared With Pharmaceutical Companies.

BackgroundMany clinical trials supporting new drug applications underrepresent minority patients. Trials conducted by the National Cancer Institute’s National Clinical Trial’s Network (NCTN) have greater outreach to community sites, potentially allowing better representation. We compared the representation of Black patients in pharmaceutical company–sponsored cancer clinical trials with NCTN trials and with the US cancer population.MethodsWe established a large cohort of study publications representing the results of trials that supported new US Food and Drug Administration drug approvals from 2008 to 2018. NCTN trial data were from the SWOG Cancer Research Network. US cancer population rates were estimated using Surveillance, Epidemiology, and End Results survey data. We compared the proportion of Black patients by enrollment year for each cancer type and overall. Tests of proportions were used. All statistical tests were 2-sided.ResultsA total 358 trials (pharmaceutical company–sponsored trials, 85; SWOG trials, 273) comprised of 93 825 patients (pharmaceutical company–sponsored trials, 46 313; SWOG trials, 47 512) for 15 cancer types were analyzed. Overall, the proportion of Black patients was 2.9% for pharmaceutical company–sponsored trials, 9.0% for SWOG trials, and 12.1% for the US cancer population (P < .001 for each pairwise comparison). These findings were generally consistent across individual cancer types.ConclusionsThe poor representation of Black patients in pharmaceutical company–sponsored trials supporting new drug applications could result in the use of new drugs with little data about efficacy or side effects in this key population. Moreover, because pharmaceutical company–sponsored trials test the newest available therapies, limited access to these trials represents a disparity in access to potential breakthrough therapies.

FDA analysis: Demographic trends in bladder and renal cancer trials.

e18144 Background: Many publications report under representation of minorities in certain subgroups, which may limit the generalizability of clinical trial (CT) results. This analysis, investigates and reports enrollment trends in CTs submitted between 2006-2017 in support of marketing applications for drugs indicated for the treatment of urothelial (UC) and renal cancer carcinoma (RCC), and compares them to incidence rates of these diseases by Surveillance, Epidemiology, and End Results (SEER) registry and the US census bureau. Methods: We identified all marketing applications for the treatment of UC and RCC that provided the primary evidence of safety and efficacy and aggregated the demographic data across trials and disease. Using these two pooled datasets, we compared the patient proportions enrolled in each of the race, sex and age categories to the corresponding rates in US cancer population estimated based on the corresponding incidence rates reported by SEER and the US census bureau using a Chi-squared test. Results: The pooled seven UC and 14 RCC CTs provided 2035 and 6757 patients respectively. The results are summarized below for the 939 (46%) UC and 1489 (22%) RCC patients enrolled in the US. Conclusions: Our findings indicate that majority of the patients were enrolled outside of the US. There were lower proportion of Black patients (4% vs 8%), older patients, age ≥ 75 years (30% vs 48%) and males (74% vs 80%) enrolled in UC population in the US. Higher proportions were observed in both White (89% vs 85%) and Asian (4% vs 2%) patients in UC and in White (90% vs 79%) patients in RCC.[Table: see text]

Patient-Reported Outcomes in Systemic Light-Chain Amyloidosis

Background: Patients with systemic light-chain (AL) amyloidosis have a high symptom burden which can negatively impact their health-related quality of life (HRQoL). However, there is a lack of data on patient-reported outcomes (PROs) in this population in the current era of effective plasma cell directed therapies. The primary objectives of our study were to assess baseline HRQoL using FACT-G (Functional Assessment of Cancer Therapy-General) and PROMIS-GH (Patient Reported Outcomes Measurement Information System-Global Health), evaluate the degree of correlation between FACT-G and PROMIS-GH, and compare HRQoL by hematologic response to first-line therapy in systemic AL amyloidosis.Method: This was an observational study using the Cleveland Clinic Knowledge Program database, which contains HRQoL data captured at outpatient visits. FACT-G was administered every 90 days since its implementation on September 2012. PROMIS-GH was implemented in October 2015, initially administered every 30 days and subsequently every 90 days since July 2017.Results: A total of 81 patients with systemic AL amyloidosis diagnosed between September 2012 and December 2017 and ≥1 HRQoL measurement were included in our analysis. The median age at diagnosis was 64 years. Cardiac involvement at diagnosis was present in 49% of patients. The most common induction therapy was Bortezomib-Cyclophosphamide-Dexamethasone (86%). Autologous stem cell transplantation was performed in 38% of patients.Data on HRQoL at baseline (≤2 months from diagnosis) using FACT-G was available for 43 patients. The mean FACT-G total score at baseline was 74 (standard deviation [S.D.] ±15). In comparison, the mean FACT-G total score for general US population is 80.1 (±18.1) and that of US cancer patients is 79.3 (±17.0) [Normative data from Pearman et al; Cancer. 2014]. Maximal HRQoL deficit was seen in the functional well-being (FWB) domain of FACT-G, with the mean score being >0.5 S.D. below that of the general population (Table 1). Data on HRQoL at baseline using PROMIS-GH was available for 18 patients. There was a significant deficit in global physical health (GPH) compared to the general population, with a mean T-score of 37.7 (±7.8) [Mean T-score in general US population being 50±10]. The mean T-score for global mental health (GMH) was 44.4 (±6.7).A total of 72 patients had 128 outpatient visits where FACT-G and PROMIS-GH were captured concurrently (range, 1-4 visits per patient). Using Cohen's criterion, GPH domain of PROMIS-GH had a large and statistically significant correlation with FACT-G total (r=0.66), physical well being (PWB) (r=0.77) and FWB (r=0.66) scores. PROMIS GMH domain also had a strong and statistically significant correlation with FACT-G total (r=0.73), PWB (r=0.60), emotional well-being (EWB) (r=0.64) and FWB (r=0.73) scores. The scatterplots for correlation between PROMIS-GH domains and FACT-G total score is shown in Figure I.At follow-up, a total of 50 patients had data on best hematologic response and HRQoL assessment. Patients achieving a complete response (CR) to first-line therapy had a significantly superior FACT-G score at all domains compared to those with less than CR or no response. Maximal benefit in complete responders was noted in the FWB domain of FACT-G (Mean score ≥2 S.D. higher than patients with less than CR or no response; P=0.002).Conclusion: Patients with AL amyloidosis have a worse HRQoL at diagnosis compared to US cancer and general adult population. Deficit in HRQoL was most prominent in the FWB domain of FACT-G and GPH domain of PROMIS-GH. Most domains of FACT-G and PROMIS-GH had strong and significant correlation. Patients achieving a CR to first-line therapy had significantly superior HRQoL at all FACT-G domains. Clinical trials in AL amyloidosis should include patient-reported HRQoL as a key endpoint and focus on the domains of physical health and functioning to assess meaningful benefit of novel therapies. Psychometric validation of FACT-G and PROMIS-GH in AL amyloidosis would be helpful in generating robust PRO data in future studies. DisclosuresMajhail:Anthem, Inc.: Consultancy; Incyte: Honoraria; Atara: Honoraria.

Representation of Minorities and Women in Oncology Clinical Trials: Review of the Past 14 Years.

Many cancer clinical trials lack appropriate representation of specific patient populations, limiting their generalizability. Therefore, we determined the representation of ethnic minorities and women in cancer clinical trials. Enrollment data from all therapeutic trials reported as completed in ClinicalTrials.gov from 2003 to 2016 were analyzed. We calculated enrollment fractions (EFs) for each group, defined as the number of enrollees divided by the 2013 Surveillance, Epidemiology, and End Results (SEER) database cancer prevalence. Of 1,012 clinical trials, 310 (31%) reported ethnicity with a total of 55,689 enrollees. Participation varied significantly across ethnic groups. Non-Hispanic whites were more likely to be enrolled in clinical trials (EF, 1.2%) than African Americans (EF, 0.7%; P < .001) and Hispanics (EF, 0.4%; P < .001). A decrease in African American (6% v 9.2%) and Hispanic (2.6% v 3.1%) enrollment was observed when compared with historical data from 1996 to 2002. Younger patients (age younger than 65 years) were more likely to be enrolled in clinical trials than the elderly (64% v 36%; P < .001). Low recruitment of female patients was observed in clinical trials for melanoma (35%), lung cancer (39%), and pancreatic cancer (40%). We observed a decrease in recruitment of minorities over the past 14 years compared with historical data. African Americans, Hispanics, and women were less likely to be enrolled in cancer clinical trials. Future trials should take extra measures to recruit participants that adequately represent the US cancer population.

The Effect of Positive SWOG Treatment Trials on Survival of Patients With Cancer in the US Population

Recently, tremendous prominence has been given to the investigation of the effect of different research processes as part of the Cancer Moonshot. More than half a century ago, the National Cancer Institute (NCI) established a network of publicly funded cancer cooperative research groups to systematically evaluate new treatments for efficacy and safety. To examine the extent to which positive NCI-sponsored cancer treatment trials have benefited patients with cancer in the US population. This investigation used study data from SWOG, an NCI-sponsored network cooperative research group. All treatment trials during SWOG's 60-year history (1956-2016) were identified for which the new, experimental therapy provided a statistically significant improvement in overall survival. It was assumed that the new, proven treatments from these trials established new standards for cancer care in the treatment community. Twenty-three positive SWOG treatment trials were identified from a variety of different disease settings. This study estimated population life-years gained from the 23 treatment trials through 2015 by mapping the effect of the new treatments onto the US cancer population using an area under the Kaplan-Meier survival curve approach that combined trial-specific hazard function and hazard ratio results, along with Surveillance, Epidemiology, and End Results program and life table data. Calculations were age adjusted. The US dollar return on investment was estimated as the ratio of the total investment by the NCI in the treatment trial program divided by the estimate of life-years gained. In total, 12 361 patients were enrolled to the 23 positive trials from 1965 to 2012. The study estimated that 3.34 million (95% confidence limit, 2.39-4.15 million) life-years were gained from these 23 trials through 2015. Estimates were greater than 2 million life-years gained under most model simulations. The US dollar return on investment was $125 per life-year gained. SWOG treatment trials have had a substantial impact on population survival for patients with cancer over 60 years. The NCI's investment in its cancer cooperative group research program has provided exceptional value and benefit to the American public through its research programs generating positive cancer treatment trials. clinicaltrials.gov Identifiers: NCT00004001, NCT00075764, and NCT00644228.

FDA analysis of enrollment of older adults in clinical trials for cancer drug registration: A 10-year experience by the U.S. Food and Drug Administration.

10009 Background: Older adults are a growing segment of our oncology population, with an expected increase in cancer incidence of 67% from 2010 to 2030 in people age 65 and older. However, older adults have been proportionally underrepresented in clinical trials. We sought to analyze the age-related enrollment of cancer patients onto trials supporting registration of new drugs or new indications approved by the US Food and Drug Administration from 2005 to 2015. Methods: This study involved retrospective analyses of demographic data of cancer patients enrolled onto trials supporting registration from 2005-2015. The data on 224,766 cancer patients supporting 105 drug applications were analyzed according to age distributions of &lt;65, 65-69, 70-74, 75-79, and ≥80 years. The rates of enrollment were compared with the corresponding rates in the US cancer population. The age distributions of the US cancer population were derived from the Surveillance, Epidemiology, and End Results Program of the National Cancer Institute for the year 2013 based on the 2010 US Census. Conclusions: Older adults were under-represented in the registration trials of new cancer therapies, especially those over age 75. Various strategies may be needed to evaluate cancer therapies for older adults in prospective clinical trials and to improve cancer care in adults over age 75. These include re-evaluating what may be considered restrictive eligibility criteria so as not to exclude older adults. Incorporating elements from geriatric assessment tools may help identify older adults most likely to benefit from treatment. More detailed labeling information that reflects the clinical experience with older adults could be considered. The FDA encourages drug sponsors as well as clinical trial cooperative groups to devise strategies to recruit patients that are reflective of their intended population. [Table: see text]

Outcome of Myelodysplastic Syndromes over Time in the US: A National Cancer Data Base Study from 2004-2013

Background: Over 10,000 new cases of myelodysplastic syndrome (MDS) are diagnosed in the US annually (MA X, Cancer 2007). Hypomethylating agents (azacitidine and decitabine), immunomodulating drugs (lenalidomide) and erythropoietin stimulating agents (epoetin α and darbepoetin) have been approved for more than a decade for the treatment of MDS. However, changes in overall outcome after the approval of these treatments are unknown. We used the National Cancer Data Base (NCDB), a nationwide oncology registry covering 70% of the US cancer population to answer these questions.Methods: We identified patients with MDS diagnosed from 2004-2013 using ICD-O-3 codes 9980-9983 and 9985-99912. Overall survival (OS) was estimated using the Kaplan-Meier method. We compared OS using the log-rank test according to demographic subgroups (sex, age [<40, 41-65, and ≥65 years], race [White, Black, Native American, Asian/Pacific Islander, and other], and ethnicity) across years of diagnosis (Period 1: 2004-2006, Period 2: 2007-2009, and Period 3: 2010-2013).Results:i) Characteristics: There were 54,953 MDS patients in our study. The median age at diagnosis was 76 years (78.7% older than 65) and 44% were females. White race (88.9%) was the most common, followed by black (7%). Four percent were Hispanics. A third (30.8%) of all patients were treated in academic centers. Three quarters (75.8%) had either Medicaid or Medicare insurance.ii) MDS Subtypes: 5q Syndrome was seen in 2008 cases (3.7%) while the most common subtype was MDS-Unclassifiable (U) (55.6%). Therapy related MDS (t-MDS) was seen in 1125 cases (2%), however coding for t-MDS was discontinued in 2009. Refractory anemia (RA), refractory anemia with ring sideroblasts (RARS), refractory cytopenia with multilineage dysplasia (RCMD), and refractory anemia with excess blasts were seen in 9%, 7.6%, 6%, and15.6%, respectively.iii) OS: Compared to their counterparts, patients who were male, older, had a diagnosis of RAEB, covered by Medicare insurance, and treated at non-academic centers had the worse OS (all P <0.001). Across Periods 1-3, we did not see improvement in OS among demographic subgroups except for those who were <40 years old (P=0.03).Conclusions: In the past decade, the overall outcome of MDS did not improve despite the advent of new therapies. More than half of the cases were classified as MDS-U reflecting the difficulty of subtyping MDS based on World Health Organization classification. We found disparity in outcomes by demographics and type of treatment facility. This is the largest study of MDS outcome to-date and should help in understanding the gaps and areas that need future research. [Display omitted] DisclosuresAl-Kali:Onconova Therapeutics, Inc.: Research Funding; Celgene: Research Funding.

Impact of the Type of Treatment Facility on the Outcome of Acute Myeloid Leukemia in the Adolescents and Young Adults: A National Cancer Data Base (NCDB) Study from 2003-2011

Background: Unlike acute lymphoblastic leukemia, data on acute myelogenous leukemia (AML) in the adolescents and young adults (AYA) are limited and generally derived from clinical trials, which are mostly conducted in academic centers. It is unclear whether the choice of treatment facility influences clinical outcome independent of disease biology and socio-demographic factors. This study used the National Cancer Data Base, a nationwide oncology database covering 70% of the US cancer population, to address this question.Methods: We identified 11,712 AYA patients who were diagnosed with AML from 2003-2011. We excluded patients who were: a) 15-17 years of age (n = 3,104) as treatment facility data were not available for age <18 years; b) not treated at the reporting facilities (n = 415) as follow-up might be incomplete; c) treated at treatment facilities with ≤100 new cancer cases/year (n = 18); and d) those without follow-up data (n = 47). Treatment facilities were classified as academic, comprehensive community (>500 new cancer cases/year), or community (101-500 new cancer cases/year) cancer programs. AMLs harboring t(15;17), inv(16), or t(8;21) were defined as good-risk, while the rest were considered non-good risk. Overall survival from the time of AML diagnosis was the primary outcome. We used multivariate imputation by chained equation to impute missing values, weighted Cox regression to obtain average hazard ratios (HRs), and pooled Wald c2 statistics to detect interaction.Results: We included 8,128 AYA patients with AML in the study and 25.2% were good risk. Among the good risk AMLs, 11.6%, 12.3%, and 76.1% had t(8;21), inv16, and t(15;17), respectively. The distribution of patients by treatment facility were academic (64.1%), comprehensive community (32.9%), and community (3.0%). After detecting an interaction between AML risk and type of treatment facility (P=0.038), we stratified treatment facility by AML risk and found significant differences in overall survival among good risk (P=0.02) but not non-good risk AML (P=0.19). This survival difference persisted even after adjusting for demographic (age, sex, race/ethnicity), socioeconomic (income, education, medical insurance), geographic (area of residence, travel distance), co-morbidities (Charlson-Deyo score), and treatment (receipt of chemotherapy) factors. Patients with good risk AML treated in comprehensive community cancer programs had a higher risk of death compared to those treated in academic programs (adjusted HR: 1.41, 95% CI: 1.11-1.79). For community cancer programs, the risk of death was also higher but did not reach statistical significance (adjusted HR: 1.22, 95% CI: 0.68-2.21). ). Subset analysis showed that the survival difference among those with good risk AML was observed in t(15;17) or acute promyelocytic leukemia (APL; P=0.02) but not in t(8;21) or inv16 (P=0.79). The disparity occurred primarily during the first month of APL diagnosis, wherein the risk of death was two-fold higher in community programs (Figure).Conclusions: Among AYA patients with AML, treatment at academic cancer programs was associated with a better survival, but only for the APL subgroup. Because APL comprises a fifth of all AMLs in the AYA and is a highly curable cancer, health care delivery research in APL should become a priority in this age group. [Display omitted] DisclosuresAl-Kali:Celgene: Research Funding.

The Influence of Treatment Facility Volume on Survival of Non-Hodgkin Lymphoma: Determining the Volume-Outcome Relationship

Background: Non-Hodgkin lymphoma (NHL) is a relatively uncommon cancer with annual incidence of ~70,000 cases but with over 50 distinct subtypes. The goal of this study was to determine the extent to which the number of NHL patients treated annually in a facility (facility volume) affects overall survival (OS). This study used the National Cancer Data Base (NCDB), a nationwide oncology database covering 70% of the US cancer population, to address this question.Methods: We used the NCDB to identify patients with NHL diagnosed from 1998 to 2006. Year 2006 was used as a cut-off in order to allow a minimum of five years of follow-up for all patients. Only patients treated at facilities with continuous annual reporting to NCDB were included. We classified treatment facilities by quartiles based on facility volume (mean patients/year): Quartile 1 (Q1: 2-13), Quartile 2 (Q2: 14-20), Quartile 3 (Q3: 21-32) and Quartile 4 (Q4: ≥33). We used Pearson correlation methods to examine collinearity, unadjusted Kaplan-Meier methods to estimate OS rates, log rank test to compare survival distributions, and multivariable Cox proportional hazards model to examine the associations between hospital volume and OS adjusting for other covariates of interest. We also included random effects for hospital to more fully adjust for clustering of outcomes within hospitals. To examine non-linear effects of hospital volume, we utilized smoothing splines.Results: There were 278,985 NHL patients cared for at 1,151 facilities. The distribution of patients according to facility volume was Q1 (10.7%), Q2 (13.5%), Q3 (23.3%) and Q4 (52.5%) and according to facility type was academic (31.2%), comprehensive community (55.9%), community (10.6%) and other (2.3%) centers. The unadjusted median OS by facility volume was: Q1: 61.8 months, Q2: 65.9 months, Q3: 71.4 months and Q4: 83.6 months. After multivariable analysis adjusting for demographic (sex, age, race, ethnicity), socioeconomic (income, insurance type), geographic (area of residence), disease-specific (NHL subtype, stage) and facility-specific (type and location) factors, we show that facility volume remains an independent predictor of all-cause mortality. Compared to patients treated at Q4 facilities, patients treated at lower quartile facilities had a worse OS (Q3HR: 1.05 [95% CI, 1.04-1.06]; Q2HR: 1.08 [1.07-1.10]; Q1HR: 1.14 [1.11-1.17]). We adjusted for hospital as a random effect, performed sensitivity analyses removing primary payor and facility type (due to collinearity with age and facility volume, respectively), and adjusted for Charlson-Deyo co-morbidity score (available only for patients diagnosed after 2003) in secondary models and found similar results. Using smoothing splines, we found a significant non-linear effect of hospital volume on OS (P <0.001). This is depicted in the Figure wherein the hazard ratio of 1.0 corresponded to the average predicted hazard, which occurred at a hospital volume of 59 patients per year.Conclusions: Patients who were treated for NHL at higher volume facilities had longer OS than those who were treated at facilities with a lower volume. This is the first study in the US using a national sample to show that a volume-outcome relationship exists in the medical management of cancer. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.