Abstract 1704P
Background
Value frameworks such as American Society of Clinical Oncology Value Framework (ASCO-VF) and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) can be sensitive to control arms of randomized trials (RCTs). Standard of care changes over time. We explored clinical value based on appropriateness of control arm and its change over time.
Methods
We searched Drugs@FDA to identify new cancer drugs approved based on RCTs between January 2012 and December 2021, excluding trials with overlap between experimental and control arm. Appropriateness of control arm was based on published ESMO and NCCN guidelines both 1 year prior to start of accrual (as defined by ClinicalTrials.gov) and at time of drug approval. Control arm was defined as suboptimal if the strength of recommendation was 2B-3 for NCCN, III-V / C-E for ESMO guidelines and/or if prior RCT data showed that the control arm was inferior to an available alternative. Substantial clinical benefit was defined as ASCO-VF threshold score ≥45 and ESMO-MCBS grade A or B (curative intent) and 4 or 5 (palliative intent).
Results
We identified 55 RCTs supporting the approval of 32 drugs. Substantial benefit was observed in 65% and 55%, using ESMO-MCBS and ASCO-VF. Prior to accrual, appropriate control arm therapy was associated with non-significantly higher odds of clinical benefit using ESMO-MCBS with NCCN (OR 3.19, P=.23), but not ESMO guidelines (OR 1.29, P=.79). At the time of drug approvals associations were similar (OR 3.03, P=.06 and 1.17, P=.80). For ASCO-VF, appropriate control arm therapy was associated with non-significantly increased odds of clinical benefit (OR 5.52, P=.14) for both guidelines. These associations were not observed at the time of drug approval (OR 0.81, P=.69 for NCCN and 0.81, P=.72 for ESMO). Results were similar for RCTs performed exclusively in palliative intent.
Conclusions
ASCO-VF appears sensitive to change in appropriateness of control group therapy over time. ESMO-MCBS appears more consistent. Heterogeneity was observed based on scale, guideline and timepoint. Further exploration of impact of control group therapy on value frameworks is warranted.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.
Resources from the same session
1579P - Gender differences and worse metastatic survival outcomes in young adult patients with oesophagogastric cancer: 12-year data from a Czech comprehensive cancer center
Presenter: Tomás Sokop
Session: Poster session 22
1580P - Early onset oesophageal adenocarcinoma: A separate biological entity?
Presenter: Dharmesh Valand
Session: Poster session 22
1581P - Total neoadjuvant chemotherapy with FOLFIRINOX regimen in patients with resectable locally advanced gastric and gastroesophageal junction cancer
Presenter: Maria Sedova
Session: Poster session 22
1582P - Gastroesophageal adenocarcinoma in young adults: Retrospective analysis of clinical and molecular features
Presenter: Daniel Acosta Eyzaguirre
Session: Poster session 22
1583P - Intraperitoneal chemotherapy for peritoneal metastases of gastric origin: A systematic review and meta-analysis
Presenter: Niels Guchelaar
Session: Poster session 22
1585P - The impact of platinum-induced peripheral neuropathy (PN) in first-line treatment on PN and efficacy in second-line paclitaxel (PTX)-based chemotherapy for unresectable advanced gastric cancer (AGC): A prospective observational multicenter study - IVY study
Presenter: Yoshiyasu Kono
Session: Poster session 22
1587P - Analysis of survival outcomes according to start timing of adjuvant chemotherapy in patients with gastric cancer: A retrospective nationwide cohort study
Presenter: Tae-Hwan Kim
Session: Poster session 22
1588P - Analysis of risk factors of anastomotic leakage after minimally invasive esophagectomy with circular cervical anastomosis
Presenter: ming lu
Session: Poster session 22