Use of nivolumab (N) and cabozantinib (C) for treatment of the metastatic renal cell carcinoma (mRCC) in the Veneto region: Results of AMOUR study.

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 290-290
Author(s):  
Marco Maruzzo ◽  
Alberto Bortolami ◽  
Dario Palleschi ◽  
Andrea Zivi ◽  
Maurizio Nicodemo ◽  
...  
Keyword(s):  
Real World ◽  
Treatment Options ◽  
Standard Of Care ◽  
Tumor Burden ◽  
Regulatory Agencies ◽  
Veneto Region ◽  
Opposite Sequence ◽  
Retrospective Nature ◽  
Third Line ◽  
The Cost

290 Background: Second (2L) or third-line (3L) treatment options for mRCC have dramatically changed in the last years. The standard of care as per Italian Regulatory Agencies approvals is N or C. To date, there are no criteria for the choice between N and C, which both demonstrated OS gain in the pivotal trials. Methods: We planned a retrospective, real world analysis of the use of N and C as 2L and 3L treatment in 17 Oncology Units of Veneto Region. All consecutive patients (pts) with mRCC treated in advanced setting in 2017-2018 were included. Results: We identified 170 pts, 73% males, median age 68.4 years. All pts started a 2L treatment while only 59% received a 3L treatment. In our cohort, patients with NLR > 3 at treatment start had a shorter OS (43 vs 90 months (mos), p < 0.0001); IMDC classification maintained its prognostic role. In 2L, N was administrated in 108 pts (63%), C in 29 pts (17%); in 3L N was administrated in 42 pts (25%), C in 49 pts (29%). Reported oncologists’ reasons for 2L choice were: change of mechanism of action compared to first line (28%), response to previous TKI (21.2%), intolerance to TKI (17.6%), previous toxicity (12.9%), tumor burden (11.2%), age of the patient (4.1%). Median OS and PFS in 2L were 28.4 and 6.6 mos for N, 16.8 and 6.6 mos for 2L C. Median OS and PFS in 3L were 27 and 5.2 mos for N, 16.6 and 7.5 mos for C. 46 pts received the sequence of drugs N > C, 12 the opposite sequence C > N. Median OS for N > C vs C > N were 96.6 vs 36 mos (p > 0.0001); median PFS for both the sequences were similar at 5.7 mos (p = ns). The cost per patient of the sequence N > C is 51.606 € while for the sequence C > N is 31.480,00 €. Between the two sequences a cost effectiveness per month of survival analysis was performed: the cost per month of OS for the sequence N > C was 534,18 € while for the sequence C > N was 874,46 €, heavily higher. Conclusions: In our real-world setting cohort, most of the pts received N as 2L treatment and a minority received C. Outcome of single drug are superimposable to published literature. With the limits of the retrospective nature of the study, with a cost per month of OS lower a much longer OS, the sequence N > C appear to be a better treatment strategy.

scholarly journals Real-world efficacy of docetaxel plus nintedanib after chemo-immunotherapy failure in advanced pulmonary adenocarcinoma

2021 ◽  
Author(s):  
Martin Metzenmacher ◽  
Filippo Rizzo ◽  
Kato Kambartel ◽  
Jens Panse ◽  
Diana Schaufler ◽  
...  
Keyword(s):  
Disease Control ◽  
Real World ◽  
Overall Response Rate ◽  
Response Rate ◽  
Treatment Options ◽  
Pulmonary Adenocarcinoma ◽  
Disease Control Rate ◽  
Overall Response ◽  
Clinical Benefits ◽  
Third Line

Aim: This real-world analysis evaluated docetaxel plus nintedanib in patients with advanced pulmonary adenocarcinoma after chemotherapy and immune checkpoint inhibitor failure, for whom treatment options are limited. Methods: Data were sourced retrospectively from seven German centers. Results: Of 93 patients, overall response rate was 41.4% (disease control rate: 75.9%). Of 57 patients given third-line docetaxel plus nintedanib, overall response rate was 50.0% (disease control rate: 82.7%). Median overall survival following third-line docetaxel plus nintedanib was 8.4 months. Adverse events were consistent with the known safety profile of docetaxel plus nintedanib. Conclusion: To date, this was the largest retrospective, real-world analysis of docetaxel plus nintedanib after chemotherapy–immunotherapy failure, indicating that docetaxel plus nintedanib offers meaningful clinical benefits in this setting.

Treatment patterns and characteristics in the second and third-line therapy setting for real-world small cell lung cancer patients.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e20576-e20576
Author(s):  
Patricia Prince ◽  
Adina Estrin ◽  
Dylan Supina ◽  
Rami Ben-Joseph ◽  
Anne Boccuti ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Real World ◽  
Medical Records ◽  
Treatment Options ◽  
Treatment Patterns ◽  
Small Cell ◽  
Small Cell Lung ◽  
Line Therapy ◽  
Third Line Therapy ◽  
Third Line

e20576 Background: With emerging treatment options for small-cell lung cancer (SCLC) patients after initial treatment failure, the real-world management of this disease should be assessed. This study aims to describe treatment patterns of real-world SCLC patients in the second-line therapy (2L) and third-line therapy (3L) setting. Methods: This was a descriptive study of real-world data sourced from ConcertAI electronic medical records, including data from CancerLinQ, an initiative of the American Society of Clinical Oncology of adults (age ≥ 18 years) with confirmed SCLC between January 1, 2016 and December 31, 2018. Patients were excluded based on evidence of other primary cancer at baseline, missing stage, death prior to diagnosis, or clinical trial participation. Patients entered the cohort on their SCLC diagnosis date and were followed through three tumor progressions to assess treatment patterns. Treatment patterns were assessed in 2L and 3L. Baseline characteristics were assessed in the 6 months prior to each line. Patients were classified as having unknown treatment if they had a gap in medical records > 90 days or no recorded tumor response or death within 180 days. Results: Overall, 538 patients were identified; 131 with unknown treatment, 108 with no treatment, 299 with 1st line therapy (1L), 124 with 2L, and 44 3L. The majority (76%) of patients were diagnosed in a community hospital setting. The most common comorbidities were chronic obstructive pulmonary disease (2L 25%, 3L 16%), diabetes (2L 15%, 3L 14%), congestive heart failure (2L 7%, 3L 9%), and myocardial infarction (2L 3%, 3L 2%). Radiation therapy was commonly used in 2L and 3L (2L 48%, 3L 43%) as were immunotherapies (2L 23%, 3L 23%). The most common systemic therapy in 2L and 3L was nivolumab (19% and 16%). Topotecan accounted for 11% of patients treated in 2L and 7% of those in 3L. Conclusions: During this treatment era (pre-immunotherapy 1L approval) there were limited treatment options available for SCLC patients. Radiation therapy and immunotherapies were frequently used as 2L and 3L therapy.[Table: see text]

scholarly journals KarMMa-RW: comparison of idecabtagene vicleucel with real-world outcomes in relapsed and refractory multiple myeloma

2021 ◽  
Vol 11 (6) ◽  
Author(s):  
Sundar Jagannath ◽  
Yi Lin ◽  
Hartmut Goldschmidt ◽  
Donna Reece ◽  
Ajay Nooka ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Real World ◽  
Treatment Options ◽  
Progression Free Survival ◽  
Standard Of Care ◽  
Eligibility Criteria ◽  
T Cell Therapy ◽  
Refractory Multiple Myeloma ◽  
Single Data ◽  
Line Of Therapy

AbstractPatients with relapsed and refractory multiple myeloma (RRMM) who are triple-class exposed (to an immunomodulatory agent, proteasome inhibitor, and anti-CD38 antibody) have limited treatment options and there is no standard of care. Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T-cell therapy, demonstrated efficacy in triple-class exposed RRMM patients in the KarMMa trial (NCT03361748). In this retrospective study (KarMMa-RW), patient-level data from triple-class exposed RRMM patients were merged into a single data model and compared with KarMMa using trimmed stabilized inverse probability of treatment weighting. Endpoints included overall response rate (ORR; primary), rate of very good partial response or better (≥VGPR), progression-free survival (PFS), and overall survival (OS). Of 1949 real-world triple-class exposed RRMM patients, 190 received subsequent (index) line of therapy and met KarMMa eligibility criteria (Eligible RRMM cohort). With a median follow-up of 13.3 months in KarMMa and 10.2 months in Eligible RRMM, ORR, and ≥VGPR were significantly improved in KarMMa versus Eligible RRMM (ORR, 76.4% vs 32.2%; ≥VGPR, 57.9% vs 13.7%; both P < 0.0001) as were PFS (11.6 vs 3.5 months; P = 0.0004) and OS (20.2 vs 14.7 months; P = 0.0006). This study demonstrated that ide-cel significantly improved responses and survival compared with currently available therapies in triple-class exposed RRMM.

scholarly journals Peg-interferon plus ribavirin in chronic hepatitis C: cost-efficacy and pharmacoutilization in clinical practice

2008 ◽  
Vol 9 (4) ◽  
pp. 173-181 ◽  
Author(s):  
Luisa Cavalletto ◽  
Elisabetta Bernardinello ◽  
Giulio Diodati ◽  
Enzo Raise ◽  
Angelo Gatta ◽  
...  
Keyword(s):  
Chronic Hepatitis ◽  
Hepatitis C ◽  
Standard Of Care ◽  
Hcv Infection ◽  
Veneto Region ◽  
Ifn Alpha ◽  
Study Population ◽  
Ifn Therapy ◽  
The Cost ◽  
Adjusted Dose

The current standard of care for the treatment of chronic hepatitis C virus (HCV) infection is combination therapy with Peg-interferon (IFN) alpha-2a or alpha-2b type plus ribavirin (RBV). This antiviral schedule can in fact avoid the three fold mortality rates associated to untreated cases with HCV infection, by inducing viral eradication and liver damage regression, so as to define the patients “definitively cured” from liver disease. This analysis describes the modalities of antiviral treatment in the Veneto region, in particular the therapy-schedule mainly used and compares the cost-effectiveness of treatment with the 2 available Peg-IFNs with strategies proposed as in the every day practice. Twelve on line hepatologic units, centralized by a network program on “Surveillance and Control of HCV Infection in the Veneto Region”, prospectively collected data and, of these, we evaluated 450 subjects that underwent antiviral therapy for chronic hepatitis or cirrhosis. A post hoc retrospective analysis of cases treated from January 2003 to December 2005 was performed, grouping the study population in 166 cases treated with Peg-IFN alpha-2a (Pegasys®, Roche, fixed-dose of 180 μg/weekly) and 284 that received Peg-IFN alpha-2b (Peg-Intron®, Schering-Plough, weight-adjusted-dose from 50 to 150 μg/weekly), both in combination therapy with ribavirin (Copegus®, Roche, or Rebetol®, Schering-Plough, weight-adjusted-dose of 15 mg/kg/daily). Epidemiological characteristics and cumulative rate of end-of-therapy response and Sustained Virological Response (SVR) were similar in the 2 groups, but 78% of cases treated with Peg-IFN alpha-2b and RBV received a significantly lower dose with respect to the weight-adjusted dose. This event conditioned efficacy to therapy as demonstrated in cases that received a < 1 μg/kg dose with respect to those treated with > 1 μg/kg (respectively SVR: 49% vs 66%, p < 0,01), particularly in genotype HCV-1 (SVR: 29% vs 51%, p = 0,01), known to be more resistant to IFN-therapy. The overall cost of antiviral therapy in this study population was about € 3,528,000/450 treated cases and considering the 269 that reached SVR (98 and 171, respectively by Peg-IFN alpha-2a or 2b and RBV therapy), the cost/SVR was € 15,632 and € 11,672 in the 2 groups. In conclusion, the optimization of Peg-IFN therapy, that is the use of the full dose, particularly in cases treated with a weight-adjusted Peg-IFN alpha-2b and RBV, at the standard of care dosage of 1.5 μg/kg/week and 15 mg/kg/day respectively, will allow a better efficacy, especially in genotype HCV-1 with an increase of 11% in SVR (43% to 54%) at a lower cost.

scholarly journals Systematic Literature Review on Global Real-World Treatment Patterns of Mantle Cell Lymphoma (MCL)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5881-5881
Author(s):  
Keri Keri Yang ◽  
Beth Lesher ◽  
Eleanor Lucas ◽  
Tony Caver ◽  
Boxiong Tang
Keyword(s):  
Literature Review ◽  
Adverse Events ◽  
Real World ◽  
Treatment Options ◽  
Treatment Patterns ◽  
Second Line ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Third Line

Introduction: MCL is an aggressive type of non-Hodgkin's lymphoma, and was reported associated with early relapse and poor long-term survival. Treatment options include chemotherapy, immunotherapy, and molecular targeted therapies. As of 2019, molecular targeted therapies available in the United States indicated for the treatment of MCL include the proteasome inhibitor bortezomib, the immunomodulatory drug lenalidomide (following two previous lines of therapy), and the Burton's tyrosine kinase inhibitors (BTKIs) ibrutinib and acalabrutinib (following at least one previous line of therapy). Objective/Methods: To examine the real-world treatment patterns of patients with MCL globally, a systematic literature review was performed (2010-2019) with predefined methodology and inclusion and exclusion criteria. Embase and Medline were searched via ProQuest and the Cochrane Controlled Register of Trials (CENTRAL) via the Cochrane Library. Results: Of the 2207 publications identified, 6 publications (US, n = 4; EU, n = 2) provided information on the first-line treatment of MCL (Table). The most commonly administered first-line treatments were bendamustine-rituximab; high dose cytarabine ± rituximab; and cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone (CHOP) ± rituximab although differences were noted across studies. Most patients received rituximab first-line either in combination with chemotherapy (54.2%-87.5%) or as monotherapy (12.9%-28.9%); although in some studies, rituximab maintenance therapy could not be excluded. The most commonly administered second-line therapies were cytarabine, rituximab monotherapy, and ibrutinib while third-line therapies were rituximab monotherapy, ibrutinib, and temsirolimus. Nine studies provided data on the real-world treatment of MCL with the BTKI ibrutinib (EU, n = 3; US, n = 5; EU/US, n = 1; Table); no real-world studies were identified for acalabrutinib. Six studies enrolled patients only with relapsed or remitting MCL; 3 studies enrolled patients (≤7.5%) who received ibrutinib as first-line therapy. Ibrutinib second-line therapy was administered to 13%-20% of patients and third-line therapy to 21% of patients. Ibrutinib discontinuation rates in 7 studies varied from 38.7%-83.6%. Non-response, including relapse or progression (34.6%-100%), was the main cause of discontinuation, followed by toxicity/adverse events (8.1%-25.6%). Across studies, toxicity/adverse events causing ibrutinib discontinuation included atrial fibrillation, bleeding/hemorrhage, chronic obstructive pulmonary disease, diarrhea, herpes zoster, infection, leukocytosis/ lymphocytosis, lung cancer, myelodysplastic syndrome, and thrombocytopenia. Two studies provided information on ibrutinib dose reductions (16.4% of patients) and ibrutinib dose interruptions (7.8%-30.2% of patients). Treatment options administered post-ibrutinib included rituximab (52.7%), hyper-CVAD + rituximab (16.7%-25.8%), lenalidomide-based regimens (9.7%-41.5%), and bortezomib-based regimens (8.4%-34.4%). Conclusion: Our analyses showed that most patients with MCL received first-line chemoimmunotherapy, although regimens varied across studies. Approximately 13%-21% of patients received ibrutinib following first-line therapy. Most ibrutinib discontinuation was due to progression followed by toxicity/adverse events. Upon discontinuation of ibrutinib, considerable variation in treatments was seen and no standard therapy identified. Given the limitations of current therapies, there is a need for additional second- and third-line treatments for patients with MCL. Quantitative assessments of clinical endpoints from real-world studies evaluating BTKI therapies are also warranted. Disclosures Yang: BeiGene, Ltd.: Employment. Lesher:Pharmerit: Employment. Lucas:Pharmerit: Employment. Caver:BeiGene, Ltd.: Employment. Tang:BeiGene, Ltd.: Employment.

Using real-world cohorts to assess the generalizability and relevance of randomized clinical trials (RCTs).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6540-6540 ◽  
Author(s):  
Caroline Savage Bennette ◽  
Nathan Coleman Nussbaum ◽  
Melissa D. Curtis ◽  
Neal J. Meropol
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Treatment Options ◽  
Metastatic Breast ◽  
Standard Of Care ◽  
Real World Data ◽  
Eligibility Criteria ◽  
Relevance Score ◽  
World Data

6540 Background: RCTs are the gold standard for understanding the efficacy of new treatments, however, patients (pts) in RCTs often differ from those treated in the real-world. Further, selecting a standard of care (SOC) arm is challenging as treatment options may evolve during the course of a RCT. Our objective was to assess the generalizability and relevance of RCTs supporting recent FDA approvals of anticancer therapies. Methods: RCTs were identified that supported FDA approvals of anticancer therapies (1/1/2016 - 4/30/2018). Relevant pts were selected from the Flatiron Health longitudinal, EHR-derived database, where available. Two metrics were calculated: 1) a trial’s pt generalizability score (% of real-world pts receiving treatment consistent with the control arm therapy for the relevant indication who actually met the trial's eligibility criteria) and 2) a trial’s SOC relevance score (% of real-world pts with the relevant indication and meeting the trial's eligibility criteria who actually received treatment consistent with the control arm therapy). All analyses excluded real-world pts treated after the relevant trial’s enrollment ended. Results: 14 RCTs across 5 cancer types (metastatic breast, advanced non-small cell lung cancer, metastatic renal cell carcinoma, multiple myeloma, and advanced urothelial) were included. There was wide variation in the SOC relevance and pt generalizability scores. The median pt generalizability score was 63% (range 35% - 88%), indicating that most real-world pts would have met the RCT eligibility criteria. The median SOC relevance score was 37% (range 15% - 74%), indicating that most RCT control arms did not reflect the way trial-eligible real-world pts in the US were actually treated. Conclusions: There is great variability across recent RCTs in terms of pt generalizability and relevance of SOC arms. Real-world data can be used to inform selection of control arms, predict impact of inclusion/exclusion criteria, and also assess the generalizability of the results of completed trials. Incorporating real-world data in planning and interpretation of prospective clinical trials could improve accrual and enhance relevance of RCT outcomes.

scholarly journals Assessing social preferences in reimbursement negotiations for new Pharmaceuticals in Oncology: an experimental design to analyse willingness to pay and willingness to accept

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Dominik J. Wettstein ◽  
Stefan Boes
Keyword(s):  
Willingness To Pay ◽  
Real World ◽  
Cognitive Biases ◽  
Social Preferences ◽  
Standard Of Care ◽  
Added Value ◽  
Amazon Mechanical Turk ◽  
Patient Organisation ◽  
Patient Benefit

Abstract Background Price negotiations for specialty pharmaceuticals take place in a complex market setting. The determination of the added value of new treatments and the related societal willingness to pay are of increasing importance in policy reform debates. From a behavioural economics perspective, potential cognitive biases and other-regarding concerns affecting outcomes of reimbursement negotiations are of interest. An experimental setting to investigate social preferences in reimbursement negotiations for novel, oncology pharmaceuticals was used. Of interest were differences in social preferences caused by incremental changes of the patient outcome. Methods An online experiment was conducted in two separate runs (n = 202, n = 404) on the Amazon Mechanical Turk (MTurk) platform. Populations were split into two (run one) and four (run two) equally sized treatment groups for hypothetical reimbursement decisions. Participants were randomly assigned to the role of a public price regulator for pharmaceuticals (buyer) or a representative of a pharmaceutical company (seller). In run two, role groups were further split into two different price magnitude framings (“real world” vs unconverted “real payoff” prices). Decisions had real monetary effects on other participants (in the role of premium payers or investors) and via charitable donations to a patient organisation (patient benefit). Results 56 (run one) and 59 (run two) percent of participants stated strictly monotone preferences for incremental patient benefit. The mean incremental cost-effectiveness ratio (ICER) against standard of care (SoC) was higher than the initial ICER of the SoC against no care. Regulators stated lower reservation prices in the “real world” prices group compared to their colleagues in the unconverted payoff group. No price group showed any reluctance to trade. Overall, regulators rated the relevance of the patient for their decision higher and the relevance of their own role lower compared to sellers. Conclusions The price magnitude of current oncology treatments affects stated preferences for incremental survival, and assigned responsibilities lead to different opinions on the relevance of affected stakeholders. The design is useful to further assess effects of reimbursement negotiations on societal outcomes like affordability (cost) or availability (access) of new pharmaceuticals and test behavioural policy interventions.

The Cost-Effectiveness of Pre-school Peanut Oral Immunotherapy in the Real World Setting

2021 ◽  
Author(s):  
Marcus Shaker ◽  
Edmond S. Chan ◽  
Jennifer LP. Protudjer ◽  
Lianne Soller ◽  
Elissa M. Abrams ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Real World ◽  
Oral Immunotherapy ◽  
The Real ◽  
Real World Setting ◽  
The Cost
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