scholarly journals Considerations for Developing a Reassessment Process: Report from the Canadian Real-World Evidence for Value of Cancer Drugs (CanREValue) Collaboration’s Reassessment and Uptake Working Group

2021 ◽  
Vol 28 (5) ◽  
pp. 4174-4183
Author(s):  
Wei Fang Dai ◽  
Vanessa Arciero ◽  
Erica Craig ◽  
Brent Fraser ◽  
Jessica Arias ◽  
...  
Keyword(s):  
Real World ◽  
Working Group ◽  
Decision Makers ◽  
Public Consultation ◽  
Cancer Drugs ◽  
Draft Report ◽  
Modified Delphi ◽  
Real World Evidence ◽  
Working Groups

The Canadian Real-world Evidence for Value in Cancer Drugs (CanREValue) Collaboration was established to develop a framework for generating and using real-world evidence (RWE) to inform the reassessment of cancer drugs following initial health technology assessment (HTA). The Reassessment and Uptake Working Group (RWG) is one of the five established CanREValue Working Groups. The RWG aims to develop considerations for incorporating RWE for HTA reassessment and strategies for using RWE to reassess drug funding decisions. Between February 2018 and December 2019, the RWG attended four teleconferences (with follow-up surveys) and two in-person meetings to discuss recommendations for the development of a reassessment process and potential barriers and facilitators. Modified Delphi methods were used to gather input. A draft report of recommendations (to December 2018) was shared for public consultation (December 2019 to January 2020). Initial considerations for developing a reassessment process were proposed. Specifically, reassessment can be initiated by diverse stakeholders, including decision makers from public drug plans or industry stakeholders. The reassessment process should be modelled after existing deliberation and recommendation frameworks used by HTA agencies. Proposed reassessment outcome categories include maintaining status quo, revisiting funding criteria, renegotiating price, or disinvesting. Overall, these initial considerations will serve as the basis for future advancements by the Collaboration.

scholarly journals Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration

BMJ Open ◽  
2020 ◽  
Vol 10 (1) ◽  
pp. e032884 ◽  
Author(s):  
Kelvin Chan ◽  
Seungree Nam ◽  
Bill Evans ◽  
Claire de Oliveira ◽  
Alexandra Chambers ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Healthcare System ◽  
Real World ◽  
Decision Makers ◽  
New Drugs ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Value For Money ◽  
Real World Evidence ◽  
Clinical Benefits

BackgroundOncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected.ObjectiveThe Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner.StrategyThe CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system.OutcomesThe framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money.

scholarly journals Utility of routinely collected electronic health records data to support effectiveness evaluations in inflammatory bowel disease: a pilot study of tofacitinib

2021 ◽  
Vol 28 (1) ◽  
pp. e100337
Author(s):  
Vivek Ashok Rudrapatna ◽  
Benjamin Scott Glicksberg ◽  
Atul Janardhan Butte
Keyword(s):  
Inflammatory Bowel Disease ◽  
Pilot Study ◽  
Disease Activity ◽  
Real World ◽  
Bowel Disease ◽  
P Value ◽  
Health Records ◽  
Real World Evidence ◽  
Inflammatory Bowel

ObjectivesElectronic health records (EHR) are receiving growing attention from regulators, biopharmaceuticals and payors as a potential source of real-world evidence. However, their suitability for the study of diseases with complex activity measures is unclear. We sought to evaluate the use of EHR data for estimating treatment effectiveness in inflammatory bowel disease (IBD), using tofacitinib as a use case.MethodsRecords from the University of California, San Francisco (6/2012 to 4/2019) were queried to identify tofacitinib-treated IBD patients. Disease activity variables at baseline and follow-up were manually abstracted according to a preregistered protocol. The proportion of patients meeting the endpoints of recent randomised trials in ulcerative colitis (UC) and Crohn’s disease (CD) was assessed.Results86 patients initiated tofacitinib. Baseline characteristics of the real-world and trial cohorts were similar, except for universal failure of tumour necrosis factor inhibitors in the former. 54% (UC) and 62% (CD) of patients had complete capture of disease activity at baseline (month −6 to 0), while only 32% (UC) and 69% (CD) of patients had complete follow-up data (month 2 to 8). Using data imputation, we estimated the proportion achieving the trial primary endpoints as being similar to the published estimates for both UC (16%, p value=0.5) and CD (38%, p-value=0.8).Discussion/ConclusionThis pilot study reproduced trial-based estimates of tofacitinib efficacy despite its use in a different cohort but revealed substantial missingness in routinely collected data. Future work is needed to strengthen EHR data and enable real-world evidence in complex diseases like IBD.

scholarly journals Connected health for growth hormone treatment research and clinical practice: learnings from different sources of real-world evidence (RWE)—large electronically collected datasets, surveillance studies and individual patients’ cases

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Nea Boman ◽  
Luis Fernandez-Luque ◽  
Ekaterina Koledova ◽  
Marketta Kause ◽  
Risto Lapatto
Keyword(s):  
Growth Hormone ◽  
Observational Study ◽  
Case Studies ◽  
Real World ◽  
Growth Disorders ◽  
Injection Device ◽  
Growth Outcomes ◽  
Real World Evidence ◽  
High Adherence

Abstract Background A range of factors can reduce the effectiveness of treatment prescribed for the long-term management of chronic health conditions, such as growth disorders. In particular, prescription medications may not achieve the positive outcomes expected because approximately half of patients adhere poorly to the prescribed treatment regimen. Methods Adherence to treatment has previously been assessed using relatively unreliable subjective methods, such as patient self-reporting during clinical follow-up, or counting prescriptions filled or vials returned by patients. Here, we report on a new approach, the use of electronically recorded objective evidence of date, time, and dose taken which was obtained through a comprehensive eHealth ecosystem, based around the easypod™ electromechanical auto-injection device and web-based connect software. The benefits of this eHealth approach are also illustrated here by two case studies, selected from the Finnish cohort of the easypod™ Connect Observational Study (ECOS), a 5-year, open-label, observational study that enrolled children from 24 countries who were being treated with growth hormone (GH) via the auto-injection device. Results Analyses of data from 9314 records from the easypod™ connect database showed that, at each time point studied, a significantly greater proportion of female patients had high adherence (≥ 85%) than male patients (2849/3867 [74%] vs 3879/5447 [71%]; P < 0.001). Furthermore, more of the younger patients (< 10 years for girls, < 12 years for boys) were in the high adherence range (P < 0.001). However, recursive partitioning of data from ECOS identified subgroups with lower adherence to GH treatment ‒ children who performed the majority of injections themselves at an early age (~ 8 years) and teenagers starting treatment aged ≥ 14 years. Conclusions The data and case studies presented herein illustrate the importance of adherence to GH therapy and how good growth outcomes can be achieved by following treatment as described. They also show how the device, software, and database ecosystem can complement normal clinical follow-up by providing HCPs with reliable information about patient adherence between visits and also providing researchers with real-world evidence of adherence and growth outcomes across a large population of patients with growth disorders treated with GH via the easypod™ device.

Comprehensive real-world evidence research in stage III and IV melanoma: Evaluation of a cohort of patients treated at a Portuguese institution.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18774-e18774
Author(s):  
Ivo Julião ◽  
Jose Luis Cunha ◽  
Patricia Redondo ◽  
Jessica Rodrigues ◽  
Tiago Figueiredo ◽  
...  
Keyword(s):  
Real World ◽  
Clinical Characteristics ◽  
Index Date ◽  
Systemic Treatment ◽  
Stage Iv ◽  
Stage Iii ◽  
Line Treatment ◽  
Adequate Treatment ◽  
Real World Evidence

e18774 Background: Malignant melanoma (MM) is one of the most aggressive skin cancers and its incidence has been increasing worldwide. Deep understanding of patient characteristics and the course of the disease, specially through the evaluation of real-world evidence, is extremely relevant for an adequate treatment approach and better outcomes. This study aims to comprehensively evaluate demographic and clinical characteristics and also treatment outcomes of patients with stage III and IV MM, treated at a Portuguese institution. Methods: Retrospective cohort study of patients with de novo MM stage III/IV or that evolved from earlier MM stages, between 2015 and 2017 (considered the index date). Patients were followed until 12/31/2019. Demographic, clinical and treatment characteristics were evaluated. Survival was assessed, from the index date, using the Kaplan Meier method and log-rank test to compare groups. Results: We included 215 patients with a median age of 66 years (20-96) and 50.2% (n = 108) were male. At index date, 63.7% (n = 137) were stage III. From those, 41.6% (n = 57) progressed to stage IV during follow-up. At diagnosis, the majority of patients had ulceration (53.3%; n = 119), normal LDH ( < 248 U/L; 56.3%; n = 121) and from 110 patients tested for BRAF, 45.4% (n = 50) had a mutation. In earlier stages, 41.8% (n = 81) performed sentinel LN only and from those 61.7% (n = 50) had latter metastatic disease. Complete LND was performed in 49% (n = 95) and 58.9% (n = 56) had a distant relapse. Brain metastasis were diagnosed in 28.4% (n = 61) of the patients, and 50.8% (n = 31) were not eligible for any treatment due to poor clinical status. Systemic treatment was performed in 70 patients with advanced disease. In 1st line, 34 (48.6%) patients underwent anti-PD-1, 28 (40.0%) BRAF/MEKi, 5 (7.1%) BRAFi and 3 (4.3%) chemotherapy. A 2nd line treatment was performed in 21 (30.0%) patients and 2 (9.5%) underwent 3rd line treatment. With a median follow-up of 29 months OS for all patients at 24 months was 54.9% (95% CI; 48.6-62.0): 69.3% (95% CI; 62.0-77.5) for stage III patients and 29.5% (95% CI; 20.9-41.6) for stage IV patients. OS was worst for known risk factors (ulceration, mitotic rate and LDH). OS at 24 months for patients under systemic treatment was 37.4% (95% CI; 26.9-52.0), with no differences between immunotherapy and targeted therapy. Finally, 22 patients were submitted to limb perfusion with an OS of 58.1% (95% CI; 41.2-81.9) at 24 months and a median PFS of 7.4 months (95% CI; 3.9-11.3). Conclusions: Analysis of real-world data is a solid tool in the evaluation, development and improvement of treatment strategies. Demographic and clinical characteristics are comparable to those of other studied cohorts. Longer follow-up of this population and the inclusion of new patients submitted to contemporary approaches will allow improving knowledge and care for melanoma patients in Portugal.

Real-world evidence (RWE) study of CAR-T agents in leukemia and lymphoma patients.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e19573-e19573
Author(s):  
Lincy S. Lal ◽  
Cori Blauer-Peterson ◽  
Stacey DaCosta Byfield ◽  
Jennifer Malin
Keyword(s):  
T Cell ◽  
Resource Utilization ◽  
Real World ◽  
Total Population ◽  
Long Term Results ◽  
Car T Cell ◽  
Real World Evidence ◽  
Car T ◽  
Study Population

e19573 Background: Chimeric antigen receptor T (CAR T) cell products are considered gene treatments, producing long term results, with just one infusion. Real world evidence on the two available CAR T cell products, tisagenlecleucel (T) and axicabtagene ciloleucel (AC) are limited in leukemia and lymphoma patients, specifically at the individual product level. This study presents treatment outcomes and resource utilization of these products from a payer perspective. Methods: Patients with evidence of CAR T administration per claims algorithm and from documentation from a prior authorization program from January 1, 2017 to May 30, 2020 were included; the CAR T administration was the index event. Baseline demographics and clinical characteristics, healthcare resource utilization (HCRU) for the CART T administration and pre and post CAR T administration for a fixed 6-month period, and previous treatments were captured and presented by product, using descriptive analytics. Results: The study population included 148 patients, mean age (SD): 57.4 (16.9), with 34% female, and 64% Commercial patients versus 36% Medicare patients, with a mean follow-up of 319 days (SD: 210). There were 15 leukemia patients, 119 lymphoma patients, and 14 patients with other indication in the study population; 71(48%) had evidence of being on a clinical trial during the study. The mean Charleson Comorbidity score at baseline was 3.9. Major comorbidities included anemia (71%), diseases of the heart (72%). 29(20%) patients were treated with T of which 24% were for leukemia and 76% for lymphoma and 67 (46%) were treated with AC of which 100% were for lymphoma, and 52 (35%) patients did not differentiate between products. Majority of the CAR T administration took place inpatient (84%). Baseline 6-month HCRU was 52% ER visits and 59% hospitalizations, compared to post 6-month utilization at 45% ER visits and 49% hospitalizations for the total population. 118 (80%) patients had evidence of prior treatment indicating that the CAR T was at least in the second line setting or higher. The most common priming chemotherapy was cyclophosphamide-fludarabine in 69 (47%) patients. Of the total population, 72% did not have any evidence of further treatment in the available follow-up time, specifically, 47% in the leukemia and 76% in the lymphoma populations, respectively. Conclusions: Majority of patients have evidence of prior treatments before the CAR T index date, indicating relapse. There is evidence of decrease in the HCRU subsequent to treatment, compared to pre period and 72% do not have subsequent treatment in the available follow-up time, indicating a high level of efficacy.

scholarly journals N15 A ‘real-world’ observational multi-centre study comparing the side effects and patient acceptability of budesonide MMX with prednisolone in patients with mild to moderate ulcerative colitis

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S664-S665
Author(s):  
J Kearns ◽  
L Scullion ◽  
C Masterson ◽  
N Kennedy ◽  
C Butcher
Keyword(s):  
Ulcerative Colitis ◽  
Side Effects ◽  
Real World ◽  
Additional Data ◽  
Patient Acceptability ◽  
Multi Centre Study ◽  
Real World Evidence ◽  
Randomised Controlled ◽  
The Impact

Abstract Background Budesonide MMX is indicated for the induction of remission in mild to moderate Ulcerative Colitis (UC) patients when 5-ASA treatment is not sufficient. Unlike traditional first-generation glucocorticoid steroids such as prednisolone, budesonide MMX has demonstrated a robust safety profile, comparable to placebo in several randomised controlled trials1,2,3. There is however limited real-world evidence to substantiate this safety claim in clinical practice. The aim of this observational analysis is to evaluate the tolerability and ease of administration of budesonide MMX in the real-world setting using prednisolone as a benchmark. Methods Patients receiving treatment for mild to moderate UC were identified in 3 treatment centres between April and October 2019. After providing privacy and data consent, patients completed a detailed nurse-led questionnaire regarding their experiences with prednisolone treatment. Following 6 weeks of therapy with budesonide MMX, patients were sent a follow-up questionnaire. Data from both the initial and subsequent questionnaires were entered by the nurse into a database for assimilation and analysis. Results Twenty-eight patients completed initial and follow-up questionnaires. Of these, 78.6% (n = 22) had experienced ≥1 prednisolone-related side effects. In comparison, following treatment with budesonide MMX, 21.4% (n = 6) reported ≥1 side effects. Instances of these side effects are shown in Figure 1. 46.4% of patients (n = 13) reported the impact of prednisolone-related side effects on daily life as moderate or severe vs. 7.1% (n = 2) following treatment with budesonide MMX. By week 2 of treatment with budesonide MMX, rectal bleeding was resolved in 32.1% of patients (n = 9) and stool frequency in 35.7% (n = 10). 93.1% (n = 27) found the instructions to take budesonide MMX given by the health care professional very easy to understand and of those expressing a preference, 71.1% of patients (n = 19) would take budesonide MMX again if prescribed. Additional data will be presented. Conclusion Data from this ‘real-world’ observational study appear to support the safely profile of budesonide MMX reported in clinical trials. The incidence of patients who experienced &gt; 1 side-effect was nearly 4 times lower for budesonide vs. prednisolone. In addition, budesonide MMX therapy was acceptable to the majority of patients and accompanying instructions easy to understand. Additional data will be presented. References

Real-world evidence of cabozantinib in metastatic renal-cell carcinoma (mRCC): Results from the Canadian Kidney Cancer Information System (CKCis).

2020 ◽  
Vol 38 (6_suppl) ◽  
pp. 682-682
Author(s):  
Hanbo Zhang ◽  
Naveen S. Basappa ◽  
Isaiah Joy ◽  
Sunita Ghosh ◽  
Aly-Khan A. Lalani ◽  
...  
Keyword(s):  
Real World ◽  
Kinase Inhibitor ◽  
Cancer Information ◽  
World Population ◽  
Treatment Algorithms ◽  
Real World Evidence ◽  
Time To Treatment Failure ◽  
Multitargeted Tyrosine Kinase Inhibitor ◽  
Sites Of Metastases

682 Background: Cabozantinib is a multitargeted tyrosine kinase inhibitor (TKI) that has demonstrated efficacy in mRCC randomized trials. Less is known about the activity of cabozantinib in patients (pts) exposed to immuno-oncology (IO) agents. We explored the real-world effectiveness of cabozantinib, including in pts who had progressed on IO therapy. Methods: Using CKCis, a prospective Canadian database, pts treated with cabozantinib monotherapy as second-line or later were identified. Baseline clinical and treatment characteristics were collected. Rates of partial response (PR), stable disease (SD), progressive disease (PD) and disease control (DCR, PR+SD) were determined along with median time to treatment failure (mTTF) and median overall survival (mOS). Results: A total of 156 pts were identified. Median age was 62 years (range 21-84), 74% of pts had clear-cell histology, and 54% had > 3 sites of metastases (12% in CNS, 47% in bone). At time of cabozantinib start, 34% had KPS score < 80. Outcomes are described below. Conclusions: The effectiveness of cabozantinib observed in this real-world population was consistent with results from clinical trials. Cabozantinib also appears to provide benefit to mRCC pts who have progressed on prior IO therapy, and should be incorporated into contemporary treatment algorithms. Further follow up is ongoing.[Table: see text]

scholarly journals Multidimensional Quantification of Exclusion Criteria to Reduce the Gap between Randomized Clinical Trial and Real-World Outcomes in Multiple Myeloma

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4003-4003
Author(s):  
Augusta Eduafo ◽  
Leland Metheny ◽  
Ravi Kyasaram ◽  
Farhad Sanati ◽  
James J. Driscoll ◽  
...  
Keyword(s):  
Real World ◽  
Research Funding ◽  
Advisory Board ◽  
P Value ◽  
Single Institution ◽  
Exclusion Criteria ◽  
Eligibility Criteria ◽  
Real World Evidence ◽  
Index Treatment

Abstract Randomized clinical trials (RCTs) are considered the highest level of evidence to define the efficacy of newly developed treatments before their adoption into clinical practice. RCTs incorporate exclusion criteria that eliminate specific patient populations in order to reduce the incidence of serious adverse events and enhance the efficacy of a given anti-cancer strategy. However, exclusion criteria may lead to a significant gap between patients (pts) enrolled on RCTs and real world pt populations, which represent the ultimate stakeholders in cancer treatment. The analysis of real-world evidence to answer clinical questions has recently gained increased interest. Assessing different dimensions of this gap may help overcome barriers in trial recruitment and enhance the applicability of RCTs in daily practice. There has been significant advancement in treating multiple myeloma (MM) over the past two decades bringing multiple new mechanisms of action to the bedside. We selected ten recent RCTs: ASPIRE, TOURMALNE-MM01, ELOQUENT-2, ENDEAVOR, POLLUX and CASTOR, OCEAN, ICARIA, APOLLO and ELOQUENT-3 studies, which are pharma-sponsored landmark trials that provided the basis for FDA approval of anti-myeloma agents. Our objective was to quantify the gap in eligibility criteria between the ten RCTs and real world populations by examining these trials using a single institution database. Methods: Pts with relapsed MM that were initiated on a second (or later line) of therapy that were recognized, retrospectively. Eligibility criteria of the ten landmark RCTs was applied during the 21 day period before the index treatment date. Pts that received Len-containing regimens were tested as to be enrolled on trials with Len/Dex control arm, patients that received Bor-containing regimens were examined to be enrolled on Bor/Dex trials and subjects who had Pom-containing regimen were screened for Pom/Dex trials. Pts were then classified as "Trial eligible" or "Trial ineligible", accordingly and were monitored longitudinally from the index treatment date until death, loss to follow-up, or end of the follow up period. Ten commonly used eligibility criteria were examined (Fig. 1). Any cancer in the three years prior to the index treatment date was counted as "history of other malignancies", i.e., skin and prostate cancer were excluded. Concurrent infection was defined as use of any antibiotic other than acyclovir, ciprofloxacin or bactrim. To calculate area under the curve of the polygon graphs Shoelace algorithm was used. Results : 516 pts were studied between 2010 and 2020 and 153 were excluded due to missing values. 224, 136 and 98 pts were treated with Len-, Bor- or Pom-containing regimens, respectively. Overall, the trial-eligible cohort was more likely to have autologous stem cell transplant and to have had longer treatment-free period before index treatment date (p-value: 0.009). There was a substantial variation in the ineligibility rate for these ten RCTs among the study population (Fig. 1). The most common items that excluded a patient from a RCT were: other malignancy, current infection and renal dysfunction. Differences between trial-eligible and trial-ineligible pts stratified by trial are listed in Tables 1, 2 and 3 for trials with Len, Bor and Pom as control arms, respectively. The median follow-up for the Len, Bor and Pom cohorts was 31, 30 and 22 months, respectively. Trial-ineligible pts displayed a significantly worse OS (2-year rate 58% vs. 78%, p-value: 0.001) and 49% higher chance of death (HR 1.69, 90%, CI: 1.17-2.62) compared with trial-eligible cohort. Conclusion: Here, we assessed the multidimensional gap that exists between patient cohorts enrolled on RCTs and real world cohorts for ten landmark MM trials. We present a quantitative deviation score as a tool to calibrate the generalizability of these landmark trials against a single institution. Importantly, we show that trial-eligibility alone significantly correlates with superior OS across a variety of MM clinical trials across all ten MM RCTs. Furthermore, our results reveal that ineligibility rates were quite different among the ten trials which significantly limit cross-trial comparisons. We propose a uniform methodology to assess patient exclusion criteria and narrow the efficacy gap observed between RCTs and real world evidence. Figure 1 Figure 1. Disclosures Metheny: Pharmacosmos: Honoraria; Incyte: Speakers Bureau. Malek: Medpacto Inc.: Research Funding; Amgen: Honoraria; Janssen: Other: Advisory board ; Takeda: Honoraria; BMS: Honoraria, Research Funding; Cumberland Inc.: Research Funding; Bluespark Inc.: Research Funding; Sanofi: Other: Advisory Board.

Real-world treatment patterns and overall survival in previously treated advanced renal cell carcinoma patients receiving nivolumab in the UK.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e16561-e16561
Author(s):  
Tom Waddell ◽  
Kate Fife ◽  
Richard Griffiths ◽  
Anand Sharma ◽  
Poonam Dhokia ◽  
...  
Keyword(s):  
Renal Cell Carcinoma ◽  
Overall Survival ◽  
Cell Carcinoma ◽  
Real World ◽  
Renal Cell ◽  
Treatment Patterns ◽  
Case Report Form ◽  
Real World Evidence ◽  
Previously Treated

e16561 Background: CheckMate 025 demonstrated favorable efficacy and safety results for nivolumab monotherapy in previously treated advanced or metastatic renal cell carcinoma (aRCC). However, real-world evidence on treatment patterns and clinical outcomes is limited. Methods: This multi-centre, retrospective cohort study examined treatment patterns and overall survival (OS) in aRCC patients treated with nivolumab monotherapy. Eligible patients who initiated nivolumab at second-line (2L) or beyond (index) between 01 March 2016 and 30 June 2018 were sampled from four UK centers. Data were extracted using an electronic case report form from index to earliest of: most recent visit; death; end of follow up (31 May 2019). Results: Overall , 151 patients were included in analyses (mean age at index 66.9 years, 72.2% male, median follow-up from index 15.2 months), with 109 (72.2%) and 42 (27.8%) receiving nivolumab at 2L and ≥ third-line (3L+), respectively. Key clinical characteristics are outlined in Table 1. All 2L nivolumab patients had received first-line (1L) tyrosine kinase inhibitors (TKI), pazopanib (57.8%), sunitinib (30.3%), or both in sequence (10.1%). After 2L nivolumab, 3L cabozantinib (36/52, 69.2%) was most common. Most 3L nivolumab patients received 2L TKI (31/36, 86.1%) - commonly axitinib (70.9%). After 3L nivolumab, most patients received fourth-line cabozantinib (8/12, 66.7%). Median time on line of therapy (LOT) decreased with LOT progression: from 7.8 months at 1L to 4.6 months at fifth-line (5L). The proportion of patients who discontinued treatment due to adverse events decreased by LOT, (28.6%, 22.7%, 16.0% and 0%, and 34.7%, 28.1%, 0% and 0% from 2L to 5L, overall and for nivolumab treatment, respectively). Overall, median OS from nivolumab initiation was 19.2 months [95% CI, 16.9-27.0]. Patients who received 2L nivolumab had longest median OS (23.0 months [95% CI, 17.2, not reached]), comparable to CheckMate 025 (25.8 months [95% CI, 22.2-29.8]). Median OS for 3L+ nivolumab patients was 12.4 months [95% CI, 8.8, 23.2]. Among 2L nivolumab patients, 73.9%, 46.2%, and 33.6% survived 12, 24, and 36 months, respectively. For the same respective timeframes, 52.4%, 24.7%, and 18.6% of 3L+ nivolumab patients survived. Conclusions: This study provides real-world evidence on the characteristics, treatment patterns and effectiveness of 2L or ≥ 3L nivolumab monotherapy in previously treated aRCC patients. OS results from UK routine clinical care were comparable to those found in CheckMate 025.[Table: see text]

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