Approaches for Enhanced Extrapolation of Long-Term Survival Outcomes Using Electronic Health Records of Patients With Cancer.

OBJECTIVES: This study aimed to demonstrate enhanced survival extrapolation methods using electronic health record-derived real-world data (RWD). METHODS: The study population included patients diagnosed of ER+/HER2- metastatic breast cancer who started first-line treatment with anastrozole or letrozole between November 18, 2014, and November 18, 2015. Two patient cohorts were constructed: a clinical trial cohort from digitized MONARCH-3 clinical trial results and a RWD cohort from a deidentified electronic health record-derived database. RWD patients were weighted to trial baseline covariate distributions. Standard parametric approaches were applied to trial data and a "best-fit" model was selected. We demonstrate traditional and enhanced hybrid (pooling with weighted RWD at start, 75%, or end of trial) extrapolation approaches. RESULTS: Observed and estimated 5-year progression-free survival (PFS) rates in extrapolating the trial control arm (n = 165) were comparable across all methods. Compared with the observed 5-year mean PFS in the RWD cohort (n = 118) of 20.4 months (95% confidence interval [CI] 16.9-23.8), there was some variation among studied methods. Best-fit standard parametric model (log-normal) had 5-year mean PFS of 21.3 months (95% CI 18.2-24.9), and for the hybrid methods in order of estimate conservativeness was start of trial (20.8 months; 95% CI 18.5-23.2), 75% of trial (21.3 months; 95% CI 18.1-24.5), and end of trial (21.8 months; 95% CI 18.8-25.2). CONCLUSIONS: Our study leverages RWD to enhance long-term survival extrapolation. Future use cases should include applying patient eligibility criteria, weighting on baseline characteristics, and choice of time window to add RWD to trial data.

Retrospective Database Analysis: Dose Escalation and Adherence in Patients Initiating Biologics for Ulcerative Colitis.

BACKGROUND: Biologic therapies are often used in patients with ulcerative colitis (UC) who are nonresponsive to conventional treatments. However, nonresponse or loss of response to biologics often occurs, leading to dose escalation, combination therapy, and/or treatment switching. We investigated real-world treatment patterns of biologic therapies among patients with UC in the USA. METHODS: This study analyzed data from the IBM® MarketScan® Commercial and Medicare Supplemental Databases (medical/pharmacy claims for >250 million patients in the USA) to identify patients with UC initiating a biologic therapy (adalimumab, infliximab, golimumab, or vedolizumab) with 12 months of follow-up post-initiation. Key measures were patient baseline characteristics, dose escalation (average maintenance dose >20% higher than label), adherence (proportion of days covered), and ulcerative colitis-related healthcare costs in the 12 months following biologic therapy initiation. RESULTS: Of 2,331 patients included in the study (adalimumab [N = 1,291], infliximab [N = 810], golimumab [N = 127], and vedolizumab [N = 103]), 28.1% used concomitant immunosuppressant therapy within 12 months post-initiation. Overall, 23.6% (adalimumab), 34.8% (infliximab), 9.9% (golimumab), and 39.2% (vedolizumab) of patients dose escalated within 12 months. Patients who dose escalated incurred USD 20,106 higher total UC-related healthcare costs over 12 months than those who did not. Adherence (covariate-adjusted proportion of days covered) ranged from 0.63 to 0.73, and 39.3% of patients discontinued within 12 months (median treatment duration = 112 days). CONCLUSION: Dose escalation was common, and incurred higher costs, in patients with UC initiating biologic therapies. Suboptimal adherence and/or discontinuation within 12 months of initiation occurred frequently, highlighting the challenges in managing these patients.

The association between disease activity and patient-reported outcomes in patients with moderate-to-severe ulcerative colitis in the United States and Europe.

BACKGROUND: Patients with ulcerative colitis (UC) experience periods of recurring and episodic clinical signs and symptoms. This study sought to establish the association between disease activity and health-related quality of life (HRQoL) and other patient-reported outcomes. METHODS: United States (US) and European Union 5 ([EU5]; i.e., France, Germany, Italy, Spain, and the United Kingdom) data from the 2015 and 2017 Adelphi Inflammatory Bowel Disease-Specific Programme (IBD-DSP) were used. The IBD-DSP is a database of retrospective patient chart information integrated with patient survey data (EuroQoL-5 Dimensions [EQ-5D], Short Quality of Life in Inflammatory Bowel Disease Questionnaire [SIBDQ], and Work Productivity and Activity Impairment-Ulcerative Colitis [WPAI-UC] questionnaire). Using available chart information, physicians classified their moderate-to-severe patients into one of the following categories: remission with a Mayo endoscopic score = 0 ("deep remission"), remission without a Mayo endoscopic score = 0 ("remission"), or active disease. Differences among disease activity categories with respect to patient-reported outcomes were analyzed using generalized linear models, controlling for confounding variables. RESULTS: N = 289 and N = 1037 patient charts with linked surveys were included from the US and EU5, respectively. The disease activity distribution was as follows: active disease = 40.1% (US) and 33.6% (EU5); remission = 48.0 and 53.0%; deep remission = 11.9 and 13.3%. Patients with active disease reported significantly lower levels of EQ-5D health state utilities (adjusted mean [AdjM] = 0.87 [US] and 0.78 [EU5]) compared with remission (AdjM = 0.92 and 0.91) and deep remission (AdjM = 0.93 and 0.91) (all P < 0.05 compared with active disease within each region). Similar findings were observed with the scores from the SIBDQ and the WPAI-UC. No significant differences were observed between remission categories. CONCLUSIONS: Among patients with moderate-to-severe UC in the US and EU5, active disease was associated with significant impairments in HRQoL, work and leisure activities. These results reinforce the importance, to both the patient and society, of achieving some level of remission to restore generic and disease-related HRQoL and one's ability to work productively.

Axitinib, cabozantinib, or everolimus in the treatment of prior sunitinib-treated patients with metastatic renal cell carcinoma: results of matching-adjusted indirect comparison analyses.

BACKGROUND: In the absence of head-to-head trials comparing axitinib with cabozantinib or everolimus, the aim of this study was to conduct an indirect comparison of their relative efficacy in patients with metastatic renal cell carcinoma (mRCC), using data from the AXIS and METEOR trials. METHODS: Progression-free survival (PFS) and overall survival (OS) in prior sunitinib-treated patients with mRCC were compared by conducting matching-adjusted indirect comparison (MAIC) analyses, including base-case and sensitivity analyses. Individual patient-level data from prior sunitinib-treated patients who received axitinib in AXIS were weighted to match published baseline characteristics of prior sunitinib-treated patients who received either cabozantinib or everolimus in METEOR. RESULTS: There was no statistically significant difference in PFS (aHR [adjusted hazard ratio] = 1.15 [CI: 0.82-1.63]) and OS (aHR = 1.00 [CI: 0.69-1.46]) between axitinib versus cabozantinib in the base-case analysis. In the sensitivity analysis, PFS (aHR = 1.39 [CI: 1.00-1.92]) and OS (aHR = 1.35 [CI: 0.95-1.92]) were shorter for axitinib compared with cabozantinib; however, the OS difference was not statistically significant. Axitinib was associated with significantly longer PFS compared with everolimus in the base-case (aHR = 0.53 [CI: 0.36-0.80]) and sensitivity analyses (aHR = 0.63 [CI: 0.45-0.88]), respectively. Results suggested an OS benefit for axitinib versus everolimus in base-case analyses (aHR = 0.63 [CI: 0.42-0.96]); however, the difference in OS in the sensitivity analysis was not statistically significant (aHR = 0.84 [CI: 0.59-1.18]). CONCLUSIONS: MAIC analyses suggest PFS and OS for axitinib and cabozantinib are dependent on the Memorial Sloan Kettering Cancer Center definition used; in the base-case analysis, there was no significant difference in PFS and OS between axitinib and cabozantinib. In the sensitivity analysis, PFS in favour of cabozantinib was significant; however, the trend for prolonged OS with cabozantinib was not significant. For axitinib and everolimus, MAIC analyses indicate patients treated with axitinib may have an improved PFS and OS benefit when compared to everolimus. Disparities between the base-case and sensitivity analyses in this study underscore the importance of adjusting for the differences in baseline characteristics and that naïve indirect comparisons are not appropriate.

Preference-based measures to obtain health state utility values for use in economic evaluations with child-based populations: a review and UK-based focus group assessment of patient and parent choices.

BACKGROUND: No current guidance is available in the UK on the choice of preference-based measure (PBM) that should be used in obtaining health-related quality of life from children. The aim of this study is to review the current usage of PBMs for obtaining health state utility values in child and adolescent populations, and to obtain information on patient and parent-proxy respondent preferences in completing PBMs in the UK. METHODS: A literature review was conducted to determine which instrument is most frequently used for child-based economic evaluations and whether child or proxy responses are used. Instruments were compared on dimensions, severity levels, elicitation and valuation methods, availability of value sets and validation studies, and the range of utility values generated. Additionally, a series of focus groups of parents and young people (11-20 years) were convened to determine patient and proxy preferences. RESULTS: Five PBMs suitable for child populations were identified, although only the Health Utilities Index 2 (HUI2) and Child Heath Utility 9D (CHU-9D) have UK value sets. 45 papers used PBMs in this population, but many used non-child-specific PBMs. Most respondents were parent proxies, even in adolescent populations. Reported missing data ranged from 0.5 to 49.3%. The focus groups reported their experiences with the EQ-5D-Y and CHU-9D. Both the young persons' group and parent/proxy groups felt that the CHU-9D was more comprehensive but may be harder for a proxy to complete. Some younger children had difficulty understanding the CHU-9D questions, but the young persons' group nonetheless preferred responding directly. CONCLUSION: The use of PBMs in child populations is increasing, but many studies use PBMs that do not have appropriate value sets. Parent proxies are the most common respondents, but the focus group responses suggest it would be preferred, and may be more informative, for older children to self-report or for child-parent dyads to respond.

Quantifying the healthcare costs of treating severely bleeding major trauma patients: a national study for England.

INTRODUCTION: Severely bleeding trauma patients are a small proportion of the major trauma population but account for 40% of all trauma deaths. Healthcare resource use and costs are likely to be substantial but have not been fully quantified. Knowledge of costs is essential for developing targeted cost reduction strategies, informing health policy, and ensuring the cost-effectiveness of interventions. METHODS: In collaboration with the Trauma Audit Research Network (TARN) detailed patient-level data on in-hospital resource use, extended care at hospital discharge, and readmissions up to 12 months post-injury were collected on 441 consecutive adult major trauma patients with severe bleeding presenting at 22 hospitals (21 in England and one in Wales). Resource use data were costed using national unit costs and mean costs estimated for the cohort and for clinically relevant subgroups. Using nationally available data on trauma presentations in England, patient-level cost estimates were up-scaled to a national level. RESULTS: The mean (95% confidence interval) total cost of initial hospital inpatient care was £19,770 (£18,177 to £21,364) per patient, of which 62% was attributable to ventilation, intensive care, and ward stays, 16% to surgery, and 12% to blood component transfusion. Nursing home and rehabilitation unit care and re-admissions to hospital increased the cost to £20,591 (£18,924 to £22,257). Costs were significantly higher for more severely injured trauma patients (Injury Severity Score ≥15) and those with blunt injuries. Cost estimates for England were £148,300,000, with over a third of this cost attributable to patients aged 65 years and over. CONCLUSIONS: Severely bleeding major trauma patients are a high cost subgroup of all major trauma patients, and the cost burden is projected to rise further as a consequence of an aging population and as evidence continues to emerge on the benefits of early and simultaneous administration of blood products in pre-specified ratios. The findings from this study provide a previously unreported baseline from which the potential impact of changes to service provision and/or treatment practice can begin to be evaluated. Further studies are still required to determine the full costs of post-discharge care requirements, which are also likely to be substantial.

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study.

OBJECTIVES: Examine whether data from early access to medicines in the USA can be used to inform National Institute for Health and Care Excellence (NICE) health technology assessments (HTA) in oncology. DESIGN: Retrospective cohort study. SETTING: Oncology-based community and academic treatment centres in the USA. PARTICIPANTS: Patients present in a nationwide electronic health record (EHR)-derived deidentified database. INTERVENTIONS: Cancer drugs that underwent NICE technology appraisal (TA) between 2014 and 2019. PRIMARY AND SECONDARY OUTCOME MEASURES: The count and follow-up time of US patients, available in the EHR, who were exposed to cancer drugs of interest in the period between Food and Drug Administration (FDA) approval and dates relevant to the NICE appraisal process. RESULTS: In 59 of 60 TAs analysed, the cancer therapy was approved in the USA before the final appraisal by NICE. The median time from FDA approval to the publication of NICE recommendations was 18.5 months, at which time the US EHR-derived database had, on average, 269 patients (SD=356) exposed to the new therapy, with a median of 75.3 person-years (IQR: 13.1-173) in time-at-risk. A case study generated evidence on real-world overall survival and treatment duration. CONCLUSIONS: Across different cancer therapies, there was substantial variability in US real-world data accumulated between FDA approval and NICE decision milestones. The applicability of these data to generate evidence for HTA decision-making should be assessed on a case-by-case basis depending on the intended HTA use case.

Improving patient-clinician communication following nephrectomy in renal cell carcinoma: Development, content validation and pilot testing of a conversation aid tool.

OBJECTIVES: This study developed, and established the content validity, of a conversation aid tool (CAT) for use in clinical practice with renal cell carcinoma (RCC) patients who receive a curative nephrectomy and are at high-risk of recurrence. The CAT was pilot tested in a sample of RCC patients to establish whether the CAT increases knowledge of RCC, treatment options (such as adjuvant therapy), and care options. METHODS: A cross-sectional, mixed methods design was used involving initial, exploratory interviews with RCC patients, RCC specialists and a steering group. Further content validation interviews were conducted with RCC patients and specialists. A web-based survey was conducted with RCC patients (N = 60), to compare the CAT versus a standard of care (SOC) consultation comparator tool on patient knowledge. RESULTS: Findings from exploratory interviews were used to develop the CAT. Content validation interviews demonstrated that the CAT was well understood and relevant to RCC patients. The web-based survey demonstrated that viewing the CAT significantly improved participants knowledge of RCC, and care options, when compared to the SOC. CONCLUSION: The findings highlight that the CAT is a relevant, comprehensive and well-understood tool for use in the post-nephrectomy consultation. PRACTICE IMPLICATIONS: Use of the CAT may increase patient knowledge of RCC and care options.

Living with Ulcerative Colitis in Japan: Biologic Persistence and Health-Care Resource Use.

OBJECTIVE: The aim of the study was to improve understanding of adherence and persistence to biologics, and their association with health-care resource utilization (HCRU), in Japanese patients with moderate to severe ulcerative colitis (UC). METHODS: Data were from Medical Data Vision, a secondary care administrative database. A retrospective, longitudinal cohort analysis was conducted of data from UC patients initiating biologic therapy between August 2013 and July 2016. Data collected for 2 years prior (baseline) and 2 years after (follow-up) the index date were evaluated. Patients completing biologic induction were identified, and adherence/persistence to biologic therapy calculated. HCRU, steroid, and immunosuppressant use during baseline and follow-up were assessed. Biologic switching during the follow-up was evaluated. Descriptive statistics (e.g., means and proportions) were obtained and inferential analyses (from Student's t tests, Fisher's exact tests, χ2 tests, the Cox proportional hazard model, and negative binomial regression) were performed. RESULTS: The analysis included 649 patients (adalimumab: 265; infliximab: 384). Biologic induction was completed by 80% of patients. Adherence to adalimumab was higher than that to infliximab (p < 0.001). Persistence at 6, 12, 18, and 24 months was higher with infliximab than with adalimumab (p < 0.05). Overall, gastroenterology outpatient visits increased, and hospitalization frequency and duration decreased, from baseline to follow-up. UC-related hospitalizations were fewer and shorter, and endoscopies fewer, in persistent than in nonpersistent patients, although persistent patients made more outpatient visits than nonpersistent patients. Hospitalization duration was lower in persistent than nonpersistent patients. Approximately 50% of patients received an immunosuppressant during biologic therapy; 5% received a concomitant steroid during biologic therapy. Overall, 17% and 3% of patients, respectively, received 2nd line and 3rd line biologics. CONCLUSIONS: Poor biologic persistence was associated with increased non-medication-associated HCRU. Effective treatments with high persistence levels and limited associated HCRU are needed in UC.

Real-world Effectiveness of Advanced Therapies Among Patients With Moderate to Severe Ulcerative Colitis in the United States.

BACKGROUND: Ulcerative colitis (UC) treatment aims to induce response and maintain steroid-free remission. For patients with moderate to severe UC and/or nonresponse to conventional treatment, advanced therapies (immunosuppressants and biologics) are available. We assessed real-world effectiveness of advanced UC therapies. METHODS: This retrospective analysis of claims data included adult patients with UC initiating immunosuppressant or biologic therapy, with 12 months' continuous enrollment pre- and postinitiation. Patients had no prescription for biologic therapy (and/or immunosuppressant if initiating immunosuppressant) in the previous 12 months. Proportion of patients remaining steroid-free (excluding 14-week tapering period), hospitalizations, and costs in the 12 months postinitiation were assessed. RESULTS: In total, 3562 patients were included in the analysis. Most patients (83.0%) used steroids in the 12 months before initiating advanced therapy. Overall, 47.8% remained steroid-free after 12 months (excluding tapering). After adjusting for patient characteristics, remaining steroid-free was significantly more likely with infliximab (43.9%) than with adalimumab (39.4%; P < 0.05); golimumab (38.2%) and vedolizumab (41.4%) were not significantly different vs adalimumab. Overall, 12.2% of patients had a UC-related hospitalization within 12 months of initiation, with a mean (SD) total length of stay of 8.2 (8.9) days and no significant differences between biologic therapies. Mean, unadjusted, UC-related costs in the 12 months postinitiation were $42,579 and were similar between therapies. CONCLUSIONS: Patients with UC initiating advanced therapy frequently continued using steroids for at least a year. Some patients experienced extended UC-related hospitalizations, with high UC-related costs overall. This suggests an ongoing challenge in managing patients with moderate to severe UC.

Using electronic health record data to identify comparator populations for comparative effectiveness research.

Electronic health records (EHRs) can define real world patient populations with high levels of clinical specificity, potentially addressing some of the shortcomings of other types of real world data (RWD) when informing decisions about the comparative effectiveness of medical technologies. An important but under-recognized concern for EHR-derived RWD, however, is that the rich clinical data permits creation of very homogenous subpopulations from the larger group of eligible patients, thereby reducing the representativeness of the cohort relative to clinical practice. In this article, we discuss the tradeoffs between choosing clinical specificity versus representativeness in population sampling for comparative effectiveness research. Using EHR-derived RWD, we provide an example in non-small cell lung cancer to illustrate the concepts, showing wide variation in outcomes among potential comparator cohorts. We close with several recommendations for selecting comparator populations from EHRs that address the balance between matching clinical guidelines and capturing practice variability in comparative effectiveness research.

Treatment patterns among patients with moderate-to-severe ulcerative colitis in the United States and Europe.

OBJECTIVE: The aim of the present study is to examine how moderate-to-severe ulcerative colitis (UC) is currently managed in real-world clinical practice across the United States (US) and European Union Five (EU5; France, Germany, Italy, Spain, and the United Kingdom). METHODS: Data from the 2017 Adelphi Inflammatory Bowel-Disease Specific Programme (IBD-DSP) were used. The IBD-DSP is a database of patient chart information abstracted by selected gastroenterologists across the US and EU5. Eligible gastroenterologists who agreed to participate were asked to complete patient record forms for the next seven consecutive eligible adult patients with UC. Only charts from patients with moderate-to-severe UC were included in the analysis (defined as those with documented administration of either an immunosuppressant [IM] or a biologic). Treatment patterns were reported descriptively. RESULTS: 411 and 1191 patient charts were included in the US and EU5 (mean ages 44.2 and 39.6 years; 53.0% and 43.5% female), respectively. For those with complete treatment history, 40.7% and 52.9% used either an IM or biologic as their first treatment (with or without steroids). Usage of these therapies increased in subsequent lines. The percentage of patients treated with combination therapy (i.e., biologic therapy with a concomitant IM) in first line generally varied between 10-20% (e.g., US: adalimumab (ADA), 10.8%; infliximab (IFX), 18.2%; EU5: ADA, 12.5%; IFX, 19.9%), though increased in later lines in the EU5. Among patients currently using a biologic therapy, between 10-40% of patients used a higher than indicated dose or greater than indicated dosing frequency during maintenance (e.g., US: IFX, 37.1%; ADA, 13.4%; EU5: IFX, 39.1%; ADA, 36.1%). In both the US and EU5, the primary reason for switching therapy was efficacy-related. CONCLUSIONS: In this analysis, many patients with moderate-to-severe UC use an IM or biologic as their first therapy after diagnosis. Combination therapy and dose escalation are also common, and underscore the challenges with managing this patient population.

Living with Ulcerative Colitis Study (LUCY) in England: a retrospective study evaluating healthcare resource utilisation and direct healthcare costs of postoperative care in ulcerative colitis.

OBJECTIVE: Ulcerative colitis (UC) is a lifelong, relapsing-remitting disease. Patients non-responsive to pharmacological treatment may require a colectomy. We estimated pre-colectomy and post-colectomy healthcare resource utilisation (HCRU) and costs in England. DESIGN/METHOD: A retrospective, longitudinal cohort study indexing adult patients with UC undergoing colectomy (2009-2015), using linked Clinical Practice Research Datalink/Hospital Episode Statistics data, was conducted. HCRU, healthcare costs and pharmacological treatments were evaluated during 12 months prior to and including colectomy (baseline) and 24 months post-colectomy (follow-up; F-U), comparing baseline/F-U, emergency/elective colectomy and subtotal/full colectomy using descriptive statistics and paired/unpaired tests. RESULTS: 249 patients from 26 165 identified were analysed including 145 (58%) elective and 184 (74%) full colectomies. Number/cost of general practitioner consultations increased post-colectomy (p<0.001), and then decreased at 13-24 months (p<0.05). From baseline to F-U, the number of outpatient visits, number/cost of hospitalisations and total direct healthcare costs decreased (all p<0.01). Postoperative HCRU was similar between elective and emergency colectomies, except for the costs of colectomy-related hospitalisations and medication, which were lower in the elective group (p<0.05). Postoperative costs were higher for subtotal versus full colectomies (p<0.001). At 1-12 month F-U, 30%, 19% and 5% of patients received aminosalicylates, steroids and immunosuppressants, respectively. CONCLUSION: HCRU/costs increased for primary care in the first year post-colectomy but decreased for secondary care, and varied according to the colectomy type. Ongoing and potentially unnecessary pharmacological therapy was seen in up to 30% of patients. These findings can inform patients and decision-makers of potential benefits and burdens of colectomy in UC.

Living with ulcerative colitis in Germany: a retrospective analysis of dose escalation, concomitant treatment use and healthcare costs.

Aims: To investigate treatment of moderate-to-severe ulcerative colitis (UC) using real-world German health insurance claims data.Materials and methods: A retrospective, longitudinal cohort study was conducted from a German statutory health insurance database for adult patients with UC indexed on biologic therapy initiation (2013-2015). Anonymized data were evaluated for 12 months prior to (baseline) through 24 months after (follow-up) indexing. Biologic dose escalations, steroid and immunosuppressant use, healthcare resource utilization (HCRU) and direct healthcare costs were evaluated, with significant differences assessed across and between index biologics. Descriptive statistics, chi-square or Fisher's exact tests, and analysis of variance were performed.Results: The analysis included 304 patients (adalimumab, n = 125; golimumab, n = 47; infliximab, n = 114; vedolizumab, n = 18). Demographic and clinical characteristics were similar across biologics. Dose escalations occurred in 58% of patients (73% of patients receiving adalimumab), with 41% receiving subsequent de-escalation. Steroids were used during follow-up by 74% of patients; 25% received steroids >14 weeks after indexing. Overall, 41% of patients received an immunosuppressant during follow-up. Steroid and immunosuppressant use were similar across biologics. Total direct healthcare costs were higher during follow-up than baseline and differed significantly across treatments (p < .05), with highest costs for golimumab. Biologic costs contributed to a major portion of follow-up costs. HCRU and costs for most resources were higher in the first 12-month follow-up period than baseline. All resource use except gastroenterology visits returned to, or below, baseline levels 13-24 months post-index date.Limitations: There was potential for inappropriate inclusion/exclusion due to miscoding. Patients may have received biologics >12 months prior to the index date. Biologic originators and biosimilars could not be differentiated.Conclusions: These data suggest that control with current biologics is suboptimal. Further treatment options that provide sustained steroid-free remission for this patient population without the need for dose escalations or concomitant therapies may be warranted.

Sunitinib Treatment Modification in First-Line Metastatic Renal Cell Carcinoma: Analysis of the STAR-TOR Registry.

BACKGROUND/AIM: Sunitinib is the current standard of care for first-line (1L) treatment of metastatic renal cell carcinoma (mRCC). Previous studies suggest that a modified treatment schedule may benefit patients. Our aim was to evaluate efficacy and safety regarding sunitinib treatment modification in 1L treatment of mRCC. MATERIALS AND METHODS: Data were drawn from STAR-TOR, a German real-world registry to evaluate outcomes of patients with mRCC who received 1L sunitinib. Patients were divided into two groups: subsequent treatment modification (SM) or remaining on standard dose/schedule (SS). Time on treatment (TT), progression-free survival (PFS), and overall survival (OS) were estimated. RESULTS: Overall, 297 patients were analyzed; 33% underwent treatment modification. Significant baseline differences between groups were observed; SM patients were older and had a more favourable Karnofsky performance status. SM patients achieved better outcomes than SS patients for median TT (15.1 versus 3.9 months; p<0.0001), PFS (15.1 versus 6.0; p<0.0001), and OS (38.1 versus 13.7; p<0.0001). Diarrhoea (34%/17%), fatigue (30%/11%), hand-foot syndrome (28%/10%), and stomatitis (20%/6%) were more frequently reported in SM versus SS; incidence was reduced following schedule/dose modification (except diarrhoea). CONCLUSION: In addition to AE mitigation, sunitinib treatment modification may help improve efficacy outcomes in mRCC by prolonging treatment duration.

Utility values associated with advanced or metastatic non-small cell lung cancer: data needs for economic modeling.

INTRODUCTION: Cost-effectiveness analyses often inform healthcare reimbursement decisions. The preferred measure of effectiveness is the quality adjusted life year (QALY) gained, where the quality of life adjustment is measured in terms of utility. Areas covered: We assessed the availability and variation of utility values for health states associated with advanced or metastatic non-small cell lung cancer (NSCLC) to identify values appropriate for cost-effectiveness models assessing alternative treatments. Our systematic search of six electronic databases (January 2000 to August 2015) found the current literature to be sparse in terms of utility values associated with NSCLC, identifying 27 studies. Utility values were most frequently reported over time and by treatment type, and less frequently by disease response, stage of disease, adverse events or disease comorbidities. Expert commentary: In response to rising healthcare costs, payers increasingly consider the cost-effectiveness of novel treatments in reimbursement decisions, especially in oncology. As the number of therapies available to treat NSCLC increases, cost-effectiveness analyses will play a key role in reimbursement decisions in this area. Quantifying the relationship between health and quality of life for NSCLC patients via utility values is an important component of assessing the cost effectiveness of novel treatments.