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Assessment of Whether the American Society of Clinical Oncology's Value Framework and the European Society for Medical Oncology's Magnitude of Clinical Benefit Scale Measure Absolute or Relative Clinical Survival Benefit: A Meta-analysis of Randomized Clinical Trials.

ABSTRACT: Importance:The American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) have independently published value frameworks. To date, whether the clinical benefit scoring algorithms from these framework were intended to measure absolute or relative survival benefit remains unclear. Objective:To empirically examine the measurement characteristics of these frameworks by comparing their survival efficacy components (ASCO clinical benefit score [CBS] and ESMO preliminary magnitude of clinical benefit grade [PMCBG]) with established measures of absolute (median survival difference and restricted mean survival time [RMST] difference) and relative (hazard ratios [HRs]) survival benefit. Data Sources:The US Food and Drug Administration (FDA)'s Hematology and Oncology Approvals and Safety Notifications database was retrospectively reviewed to identify phase 3 randomized controlled trials (RCTs) cited for clinical efficacy evidence in oncology drug approvals from January 1, 2006, through December 31, 2017. Study Selection:Two reviewers searched the database for initial trials cited for approval. Phase 3 trials with overall survival, progression-free survival, and/or time to progression as their primary or coprimary end points were included. Notifications for noncancer indications or presenting label changes and trials that did not report HRs for the required end points and/or did not publish survival curves with number-at-risk data were excluded. Of 269 notifications initially identified, 107 met the selection criteria. Data Extraction and Synthesis:Sensitivity analyses were conducted by calculating the scores using (1) the framework-defined end point, including tail-of-curve bonus points (ASCO) or long-term plateau adjustments (ESMO) (framework-defined end point plus tail-of-curve bonus), (2) overall survival data only, and (3) progression-free survival data only. For primary and sensitivity analyses, Spearman correlation coefficients were calculated to examine the relationships between (1) ASCO-CBS or ESMO-PMCBG and RMST difference, (2) ASCO-CBS or ESMO-PMCBG and median survival difference, and (3) ASCO-CBS or ESMO-PMCBG and HR. Data were analyzed from January 7 through April 30, 2018. Main Outcomes and Measures:In the primary analysis, ASCO-CBSs and ESMO-PMCBGs were calculated for the included trials using the framework-defined end point. Results:Compared with measures of absolute survival benefit, ESMO-PMCBGs showed low to moderate correlations with RMST difference (??=?0.44) and moderate to high correlations with median survival difference (??=?0.64). ASCO-CBSs showed low to moderate correlations with both measures of absolute benefit (??=?0.43 for RMST difference; ??=?0.44 for median survival). Compared with a relative measure of survival (HRs), ESMO-PMCBGs showed a low correlation (??=?0.47) and ASCO-CBSs showed a higher correlation (??=?0.76). Conclusions and Relevance:Neither framework consistently performed as an absolute measure of survival benefit. The incorporation of a direct measure of absolute clinical benefit, such as RMST difference, into the survival efficacy components of their algorithms should be considered.

SUBMITTER: Saluja R

PROVIDER: S-EPMC6537770 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature

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Assessment of Whether the American Society of Clinical Oncology's Value Framework and the European Society for Medical Oncology's Magnitude of Clinical Benefit Scale Measure Absolute or Relative Clinical Survival Benefit: An Analysis of Randomized Clinical Trials.

Saluja Ronak R Everest Louis L Cheng Sierra S Cheung Matthew M Chan Kelvin K W KKW

JAMA oncology 20190801 8

<h4>Importance</h4>The American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) have independently published value frameworks. To date, whether the clinical benefit scoring algorithms from these framework were intended to measure absolute or relative survival benefit remains unclear.<h4>Objective</h4>To empirically examine the measurement characteristics of these frameworks by comparing their survival efficacy components (ASCO clinical benefit score [CBS] ...[more]

PMID: 31095255

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Project description:BackgroundPrecision and matched cancer medicine has the potential to complement the existing biomarker approaches in cancer treatment. However, despite their promising potential, certain negative results have highlighted their limitations in molecular biology-driven treatment strategies. This study aimed to evaluate the clinical benefits of precision therapies.Materials and methodsThree reviewers independently identified and assessed precision and matched cancer treatment studies published between January 2015 and December 2020. Clinical benefits of the treatments included in our cohort were assessed using two established frameworks; the European Society of Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) and the American Society of Clinical Oncology Value Framework.ResultsOf the 290 eligible studies, 130 were for lung cancer, 51 for solid tumors, 24 for melanoma, and 24 for breast cancer. The common targets were: epidermal growth factor receptor (N = 66), serine/threonine-protein kinase B-Raf (N = 40), anaplastic lymphoma kinase (ALK) (N = 34), breast cancer protein (N = 26), phosphatidylinositol-3 kinase/protein kinase B/phosphatase and tensin homolog (PI3K/AKT/PTEN) pathway (N = 19), receptor tyrosine-protein kinase erbB-2 (HER2) (N = 19), mitogen-activated protein kinase (RAS/RAF/MAPK) pathway (N = 18), programmed death-ligand 1 (N = 12), fibroblast growth factor receptor (N = 8), and others (N = 43). The ESMO-MCBS scales ranged from 0 to 4. Based on the clinical benefit values, tumor mutational burden/mismatch repair-deficient/microsatellite instability-high for immunotherapy, anaplastic lymphoma kinase, and neurotrophic receptor tyrosine kinase therapeutic targets were considered high, whereas RAS/RAF/MAPK and PI3K/AKT/PTEN were considered low. Additionally, we found a significant difference between each average score (P < 0.001).ConclusionsThis study showed that precision and matched cancer therapies require further improvement. This is consistent with the views of the tumor board and of clinicians that precision strategies need to be revised to improve their therapeutic effects.

Project description:PurposeTo provide evidence-based recommendations to oncologists and others for the treatment of patients with metastatic pancreatic cancer.MethodsAmerican Society of Clinical Oncology convened an Expert Panel of medical oncology, radiation oncology, surgical oncology, gastroenterology, palliative care, and advocacy experts to conduct a systematic review of the literature from April 2004 to June 2015. Outcomes were overall survival, disease-free survival, progression-free survival, and adverse events.ResultsTwenty-four randomized controlled trials met the systematic review criteria.RecommendationsA multiphase computed tomography scan of the chest, abdomen, and pelvis should be performed. Baseline performance status and comorbidity profile should be evaluated. Goals of care, patient preferences, treatment response, psychological status, support systems, and symptom burden should guide decisions for treatments. A palliative care referral should occur at first visit. FOLFIRINOX (leucovorin, fluorouracil, irinotecan, and oxaliplatin; favorable comorbidity profile) or gemcitabine plus nanoparticle albumin-bound (NAB) -paclitaxel (adequate comorbidity profile) should be offered to patients with Eastern Cooperative Oncology Group performance status (ECOG PS) 0 to 1 based on patient preference and support system available. Gemcitabine alone is recommended for patients with ECOG PS 2 or with a comorbidity profile that precludes other regimens; the addition of capecitabine or erlotinib may be offered. Patients with an ECOG PS ≥ 3 and poorly controlled comorbid conditions should be offered cancer-directed therapy only on a case-by-case basis; supportive care should be emphasized. For second-line therapy, gemcitabine plus NAB-paclitaxel should be offered to patients with first-line treatment with FOLFIRINOX, an ECOG PS 0 to 1, and a favorable comorbidity profile; fluorouracil plus oxaliplatin, irinotecan, or nanoliposomal irinotecan should be offered to patients with first-line treatment with gemcitabine plus NAB-paclitaxel, ECOG PS 0 to 1, and favorable comorbidity profile, and gemcitabine or fluorouracil should be offered to patients with either an ECOG PS 2 or a comorbidity profile that precludes other regimens. Additional information is available at www.asco.org/guidelines/MetPC and www.asco.org/guidelineswiki.

Dataset Information

Assessment of Whether the American Society of Clinical Oncology's Value Framework and the European Society for Medical Oncology's Magnitude of Clinical Benefit Scale Measure Absolute or Relative Clinical Survival Benefit: A Meta-analysis of Randomized Clinical Trials.

Publications

Assessment of Whether the American Society of Clinical Oncology's Value Framework and the European Society for Medical Oncology's Magnitude of Clinical Benefit Scale Measure Absolute or Relative Clinical Survival Benefit: An Analysis of Randomized Clinical Trials.

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