Table 1.
Validation Exercise Meeting Dates | RWE Proposal | Research Question(s) |
---|---|---|
18 August 2021 | Polatuzumab vedotin for relapsed/refractory diffuse large B-cell lymphoma | What is the real-world comparative effectiveness (overall survival) of polatuzumab vedotin in combination with bendamustine and rituximab in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplantation (ASCT) and have received at least one prior therapy, as compared to standard systemic therapy? |
22 November 2021 | Dabrafenib and Trametinib for BRAF V600E mutation Positive Metastatic Non-Small Cell Lung Cancer |
What is the real-world progression-free survival (RQ1) of dabrafenib and trametinib as first line-line systemic therapy in patients with BRAF V600E mutation positive metastatic non-small cell lung cancer (NSCLC), as compared to standard platinum-doublet chemotherapy +/− pembrolizumab? |
What is the real-world overall survival (RQ2) of dabrafenib and trametinib as first line-line systemic therapy in patients with BRAF V600E mutation positive metastatic non-small cell lung cancer (NSCLC), as compared to standard platinum-doublet chemotherapy +/− pembrolizumab? | ||
What is the real-world time to next treatment (RQ3) of dabrafenib and trametinib as first line-line systemic therapy in patients with BRAF V600E mutation positive metastatic non-small cell lung cancer (NSCLC), as compared to standard platinum-doublet chemotherapy +/− pembrolizumab? | ||
What is the real-world duration of treatment (RQ4) of dabrafenib and trametinib as first line-line systemic therapy in patients with BRAF V600E mutation positive metastatic non-small cell lung cancer (NSCLC), as compared to standard platinum-doublet chemotherapy +/− pembrolizumab? | ||
23 February 2022 | Nivolumab in relapsed/ Refractory Classical Hodgkin’s lymphoma |
What is the real-world overall survival of nivolumab in patients with classical Hodgkin’s lymphoma with evidence of disease progression following autologous stem cell transplantation (ASCT) and brentuximab vedotin (BV), as compared to standard single-agent chemotherapy or pembrolizumab immunotherapy? * |
Atezolizumab in combination with bevacizumab for unresectable/metastatic hepatocellular carcinoma | What is the real-world overall survival of atezolizumab in combination with bevacizumab as first-line treatment for patients with unresectable or metastatic hepatocellular carcinoma (HCC), as compared to either sorafenib or lenvatinib? | |
23 June 2022 | Durvalumab in Combination with Platinum Etoposide for Extensive-Stage Small Cell Lung Cancer |
What is the real-world overall survival of durvalumab in combination with platinum-etoposide as first-line treatment for patients with extensive-stage small cell lung cancer, as compared to platinum-etoposide alone? |
Nivolumab in Combination with Fluoropyrimidine and Platinum-containing Chemotherapy for Metastatic Gastric Adenocarcinoma |
What is the real-world overall survival of nivolumab in combination with fluoropyrimidine and platinum-containing chemotherapy as first-line systemic therapy for HER-2 negative locally advanced or metastatic gastric adenocarcinoma, as compared to fluoropyrimidine and platinum-containing chemotherapy alone? | |
5 December 2022 | Nivolumab in Combination with Fluoropyrimidine and Platinum-containing Chemotherapy for Metastatic Gastric Adenocarcinoma † | What is the real-world overall survival of nivolumab in combination with fluoropyrimidine and platinum-containing chemotherapy as first-line systemic therapy for HER-2 negative locally advanced or metastatic gastric adenocarcinoma, as compared to fluoropyrimidine and platinum-containing chemotherapy alone? |
Note: Endpoint(s) of interest are bolded. * RWE Proposal, including the research question, was evaluated previously in an MCDA pilot study reported by Parmar et al., 2023 [13]. † Previously reviewed during the June 2022 meeting.