The Role of Real-World Evidence to Support Treatment Choices in Malignant Pleural Mesothelioma

In this issue of JTO Clinical and Research Reports, Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar report outcomes from patients diagnosed with having malignant pleural mesothelioma (MPM) who received at least one line of pemetrexed-based chemotherapy. They describe the prescribing patterns with specific interest in the following: the platinum analog used in combination with pemetrexed as first-line treatment (no difference observed between cisplatin and carboplatin); whether maintenance pemetrexed prolongs survival (also not observed); and finally, in patients who received subsequent lines of therapy, whether immunotherapy was more efficacious than chemotherapy (no statistically significant difference found here either).Although these three findings do not substantively alter current views of managing MPM, one can consider how the research platform used by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar can appraise and inform treatment paradigms.Real-world evidence (RWE) is gaining importance and credence in the medical literature. RWE reports outcomes from treatments or technologies in the clinical management of a disease, derived from single- or multi-institution chart review projects, specific RWE platforms, disease-specific registries, or health care administrative data. There are inherent constraints to applying the results of prospective controlled clinical trials, with very specific eligibility criteria, to general practice. RWE can consider clinical outcomes for unselected populations or selected subgroups of interest, potentially more applicable to everyday clinical decision-making, ultimately providing pragmatic support in the clinic, both in understanding efficacy and safety. RWE may provide supportive data in areas where there is a paucity of clinical trial data or in rare conditions. Increasingly, RWE may be incorporated into regulatory submissions to support approval of new treatments and can provide valuable information on health system utilization of treatments or services.2Di Maio M. Perrone F. Conte P. Real-world evidence in oncology: opportunities and limitations.Oncologist. 2020; 25: e746-e752Crossref PubMed Scopus (35) Google ScholarNevertheless, there are well-recognized limitations of RWE.2Di Maio M. Perrone F. Conte P. Real-world evidence in oncology: opportunities and limitations.Oncologist. 2020; 25: e746-e752Crossref PubMed Scopus (35) Google Scholar Foremost is that selection for interventions in routine practice raises the potential for bias, although it may not be feasible to adjust for confounding variables in interpreting outcomes owing to the lack of control data. There is also a basic issue about the quality of the data used for analysis. Each source of RWE will have its own benefits and challenges. Disease registries and health care administrative data have the benefit of including data on large numbers of patients, with minimal effort, as data already exist and are being used in research as a secondary objective. The obvious challenge is that the data were not created with research in mind, and therefore are often missing key components that are important to accurately answer a given research question. Single-center chart reviews can be time consuming to undertake and are often limited by small patient numbers. Nevertheless, there is an ability to collect and analyze significant patient-level detail specific to the research question at hand. Multicenter collaborations can achieve more meaningful patient numbers and help generate more robust RWE.With the emergence of electronic health records, the ability to curate RWE from patient charts has become more feasible. Standards are now described for the level of quality in RWE. For example, the Observational Medical Outcomes Partnership Common Data Model is designed to provide a standardized level of quality observational data using standardized vocabularies that enhance analysis and can lead to generation of reliable reports and evidence.3Stang P.E. Ryan P.B. Racoosin J.A. et al.Advancing the science for active surveillance: rationale and design for the Observational Medical Outcomes Partnership.Ann Intern Med. 2010; 153: 600-606Crossref PubMed Scopus (265) Google Scholar Similarly, in RWE evidence research in cancer, minimal Common Oncology Data Elements has been developed by the American Society of Clinical Oncology (ASCO) to provide a standardized set of cancer data points that can allow patient-level research and information to be curated from electronic health records.4Osterman T.J. Terry M. Miller R.S. Improving cancer data interoperability: the promise of the minimal common oncology data elements (mCODE) initiative.JCO Clin Cancer Inform. 2020; 4: 993-1001Crossref PubMed Scopus (22) Google Scholar Finally, the Clinical Data Interchange Standards Consortium is a global nonprofit organization that collaborates with groups such as the U.S. Food and Drug Administration and the European Medicines Agency to develop data standards for clinical research.5Facile R. Muhlbradt E.E. Gong M. et al.CDISC) standards for real-world data: expert perspectives from a qualitative Delphi survey.JMIR Med Inform. 2022; 10e30363Crossref Scopus (3) Google ScholarThe Real-World Evidence Alliance is a U.S.-based group of private companies that specialize in RWE and data analytics in health, seeking to engage with the U.S. Food and Drug Administration and the U.S. Congress to enhance the use of RWE to support regulatory decision-making.6The Real World Evidence Alliance.https://rwealliance.org/Date accessed: February 10, 2022Google Scholar Flatiron, a part of the RWE Alliance, is a health technology company founded in 2012 that specifically works to develop oncology RWE and claims to have patient-level data from more than 3 million patients with cancer in the United States from more than 280 cancer centers. Deidentified patient data are pulled from the electronic medical records by trained data abstractors, with as little as 30-day recency.7Ma X. Long L. Moon S. Adamson B.J.S. Baxi S.S. Comparison of population characteristics in real-world clinical oncology databases in the US: Flatiron Health, SEER, and NPCR. medRxiv.https://www.medrxiv.org/content/10.1101/2020.03.16.20037143v2Date accessed: February 10, 2022Google Scholar Most of the contributing centers are community cancer centers, who are perhaps less likely to participate in the same number of randomized controlled trials (RCTs) as major academic centers, and therefore able to provide a better picture of routine oncology practice in large parts of the United States.The use of the Flatiron database by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar showcases some of the strengths and weaknesses of the Flatiron platform. They were able to report relatively current information on 787 patients with MPM who were treated with platinum-based chemotherapy, a significant feat in a rare disease. Nevertheless, despite propensity matching, they lack significant high-quality data regarding confounding factors that may bias their results. Stage of disease is notably missing from their analysis, and several time-varying factors are included dating back to 180 days before the start of treatment. This is a significant duration of time for a disease with a median overall survival (OS), in their analysis, of approximately 1 year. Mortality data in Flatiron Health come from a composite variable, trading currency for the reliability of population-based Vital Statistics.7Ma X. Long L. Moon S. Adamson B.J.S. Baxi S.S. Comparison of population characteristics in real-world clinical oncology databases in the US: Flatiron Health, SEER, and NPCR. medRxiv.https://www.medrxiv.org/content/10.1101/2020.03.16.20037143v2Date accessed: February 10, 2022Google ScholarThe dataset for the study of Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar covered the period from January 2011 to July 2019. The study findings support the recommendations made in the ASCO guideline for treatment of MPM that was published in 2018.8Kindler H.L. Ismaila N. Armato 3rd, S.G. et al.Treatment of malignant pleural mesothelioma: American Society of Clinical Oncology clinical practice guideline.J Clin Oncol. 2018; 36: 1343-1373Crossref PubMed Scopus (230) Google Scholar The ASCO guideline was based on a comprehensive review that focused on the results of systematic reviews, meta-analyses, RCTs, and prospective and retrospective comparative observational studies published between 1990 and 2016, with greater weight given to those recommendations supported by higher levels of scientific evidence. In this instance, RWE lends support to an evidence-based practice guideline, providing reassurance that the guideline recommendations are relevant to day-to-day management in the clinic.Nevertheless, treatment of MPM has changed dramatically in the past few years. The MAPS study was published in 2016 and revealed an OS advantage when bevacizumab was added to platinum/pemetrexed chemotherapy.9Zalcman G. Mazieres J. Margery J. et al.Bevacizumab for newly diagnosed pleural mesothelioma in the Mesothelioma Avastin cisplatin Pemetrexed a 3 Full Text Full Text PDF Scopus Google Scholar The of this has been variable, perhaps owing to the OS benefit the of the the lack of regulatory approval for the and studies as a in have been S. J. for patients with malignant pleural Oncol. PubMed Scopus Google Scholar this has been by the emergence of immunotherapy has been the an in survival outcomes for with platinum/pemetrexed as first-line treatment of P. et in malignant pleural mesothelioma a 3 Full Text Full Text PDF PubMed Scopus Google Scholar from a advantage in median there was a in survival at years. Nevertheless, have whether the OS advantage is limited to the population and whether 1 may a S. R. et chemotherapy in patients with malignant pleural mesothelioma from Oncol. Full Text Full Text PDF Google the in or published the results of important the of one of the by the RWE data by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar the study randomized patients with MPM, treated with platinum-based chemotherapy, to or and revealed a OS with S. et in patients with malignant mesothelioma a 3 Oncol. Full Text Full Text PDF PubMed Scopus Google Scholar the study of active supportive care active supportive care and that of not this be an use of et al.A randomized trial of as therapy for patients with malignant pleural Clin Oncol. of interest is chemotherapy with The a study of revealed significant for the combination with differential based on the of et with first-line chemotherapy in malignant pleural mesothelioma a trial with a Oncol. 2020; Full Text Full Text PDF PubMed Scopus Google Scholar are the results of 3 trials this such as the Cancer trial platinum/pemetrexed with platinum/pemetrexed where RWE in the of randomized data published and in the MPM be to whether Kerrigan et K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar or provide RWE in MPM the start of the immunotherapy such data help to answer about the of and in a or having patient including help to patients with MPM benefit from immunotherapy and patients chemotherapy results of the 3 trials of chemotherapy immunotherapy are there will be interest in to an for these and the very RWE how have data from trials of the treatment being used in routine practice. RWE help for subsequent lines of including efficacy of platinum/pemetrexed as therapy or treatment such as and have clinical the by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar the strengths and weaknesses of by and care in these patients to be in line with current in this not being able to on that despite RWE will not the by and in the levels of scientific when using often standards such as the Observational Medical Outcomes minimal Common Oncology Data or Clinical Data Interchange Standards RWE can and provide and such research be and review and review and In this issue of JTO Clinical and Research Reports, Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar report outcomes from patients diagnosed with having malignant pleural mesothelioma (MPM) who received at least one line of pemetrexed-based chemotherapy. They describe the prescribing patterns with specific interest in the following: the platinum analog used in combination with pemetrexed as first-line treatment (no difference observed between cisplatin and carboplatin); whether maintenance pemetrexed prolongs survival (also not observed); and finally, in patients who received subsequent lines of therapy, whether immunotherapy was more efficacious than chemotherapy (no statistically significant difference found here these three findings do not substantively alter current views of managing MPM, one can consider how the research platform used by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar can appraise and inform treatment Real-world evidence (RWE) is gaining importance and credence in the medical literature. RWE reports outcomes from treatments or technologies in the clinical management of a disease, derived from single- or multi-institution chart review projects, specific RWE platforms, disease-specific registries, or health care administrative data. There are inherent constraints to applying the results of prospective controlled clinical trials, with very specific eligibility criteria, to general practice. RWE can consider clinical outcomes for unselected populations or selected subgroups of interest, potentially more applicable to everyday clinical decision-making, ultimately providing pragmatic support in the clinic, both in understanding efficacy and safety. RWE may provide supportive data in areas where there is a paucity of clinical trial data or in rare conditions. Increasingly, RWE may be incorporated into regulatory submissions to support approval of new treatments and can provide valuable information on health system utilization of treatments or services.2Di Maio M. Perrone F. Conte P. Real-world evidence in oncology: opportunities and limitations.Oncologist. 2020; 25: e746-e752Crossref PubMed Scopus (35) Google Scholar Nevertheless, there are well-recognized limitations of RWE.2Di Maio M. Perrone F. Conte P. Real-world evidence in oncology: opportunities and limitations.Oncologist. 2020; 25: e746-e752Crossref PubMed Scopus (35) Google Scholar Foremost is that selection for interventions in routine practice raises the potential for bias, although it may not be feasible to adjust for confounding variables in interpreting outcomes owing to the lack of control data. There is also a basic issue about the quality of the data used for analysis. Each source of RWE will have its own benefits and challenges. Disease registries and health care administrative data have the benefit of including data on large numbers of patients, with minimal effort, as data already exist and are being used in research as a secondary objective. The obvious challenge is that the data were not created with research in mind, and therefore are often missing key components that are important to accurately answer a given research question. Single-center chart reviews can be time consuming to undertake and are often limited by small patient numbers. Nevertheless, there is an ability to collect and analyze significant patient-level detail specific to the research question at hand. Multicenter collaborations can achieve more meaningful patient numbers and help generate more robust RWE. the emergence of electronic health records, the ability to curate RWE from patient charts has become more feasible. Standards are now described for the level of quality in RWE. For example, the Observational Medical Outcomes Partnership Common Data Model is designed to provide a standardized level of quality observational data using standardized vocabularies that enhance analysis and can lead to generation of reliable reports and evidence.3Stang P.E. Ryan P.B. Racoosin J.A. et al.Advancing the science for active surveillance: rationale and design for the Observational Medical Outcomes Partnership.Ann Intern Med. 2010; 153: 600-606Crossref PubMed Scopus (265) Google Scholar Similarly, in RWE evidence research in cancer, minimal Common Oncology Data Elements has been developed by the American Society of Clinical Oncology (ASCO) to provide a standardized set of cancer data points that can allow patient-level research and information to be curated from electronic health records.4Osterman T.J. Terry M. Miller R.S. Improving cancer data interoperability: the promise of the minimal common oncology data elements (mCODE) initiative.JCO Clin Cancer Inform. 2020; 4: 993-1001Crossref PubMed Scopus (22) Google Scholar Finally, the Clinical Data Interchange Standards Consortium is a global nonprofit organization that collaborates with groups such as the U.S. Food and Drug Administration and the European Medicines Agency to develop data standards for clinical research.5Facile R. Muhlbradt E.E. Gong M. et al.CDISC) standards for real-world data: expert perspectives from a qualitative Delphi survey.JMIR Med Inform. 2022; 10e30363Crossref Scopus (3) Google Scholar The Real-World Evidence Alliance is a U.S.-based group of private companies that specialize in RWE and data analytics in health, seeking to engage with the U.S. Food and Drug Administration and the U.S. Congress to enhance the use of RWE to support regulatory decision-making.6The Real World Evidence Alliance.https://rwealliance.org/Date accessed: February 10, 2022Google Scholar Flatiron, a part of the RWE Alliance, is a health technology company founded in 2012 that specifically works to develop oncology RWE and claims to have patient-level data from more than 3 million patients with cancer in the United States from more than 280 cancer centers. Deidentified patient data are pulled from the electronic medical records by trained data abstractors, with as little as 30-day recency.7Ma X. Long L. Moon S. Adamson B.J.S. Baxi S.S. Comparison of population characteristics in real-world clinical oncology databases in the US: Flatiron Health, SEER, and NPCR. medRxiv.https://www.medrxiv.org/content/10.1101/2020.03.16.20037143v2Date accessed: February 10, 2022Google Scholar Most of the contributing centers are community cancer centers, who are perhaps less likely to participate in the same number of randomized controlled trials (RCTs) as major academic centers, and therefore able to provide a better picture of routine oncology practice in large parts of the United The use of the Flatiron database by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar showcases some of the strengths and weaknesses of the Flatiron platform. They were able to report relatively current information on 787 patients with MPM who were treated with platinum-based chemotherapy, a significant feat in a rare disease. Nevertheless, despite propensity matching, they lack significant high-quality data regarding confounding factors that may bias their results. Stage of disease is notably missing from their analysis, and several time-varying factors are included dating back to 180 days before the start of treatment. This is a significant duration of time for a disease with a median overall survival (OS), in their analysis, of approximately 1 year. Mortality data in Flatiron Health come from a composite variable, trading currency for the reliability of population-based Vital Statistics.7Ma X. Long L. Moon S. Adamson B.J.S. Baxi S.S. Comparison of population characteristics in real-world clinical oncology databases in the US: Flatiron Health, SEER, and NPCR. medRxiv.https://www.medrxiv.org/content/10.1101/2020.03.16.20037143v2Date accessed: February 10, 2022Google Scholar The dataset for the study of Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar covered the period from January 2011 to July 2019. The study findings support the recommendations made in the ASCO guideline for treatment of MPM that was published in 2018.8Kindler H.L. Ismaila N. Armato 3rd, S.G. et al.Treatment of malignant pleural mesothelioma: American Society of Clinical Oncology clinical practice guideline.J Clin Oncol. 2018; 36: 1343-1373Crossref PubMed Scopus (230) Google Scholar The ASCO guideline was based on a comprehensive review that focused on the results of systematic reviews, meta-analyses, RCTs, and prospective and retrospective comparative observational studies published between 1990 and 2016, with greater weight given to those recommendations supported by higher levels of scientific evidence. In this instance, RWE lends support to an evidence-based practice guideline, providing reassurance that the guideline recommendations are relevant to day-to-day management in the Nevertheless, treatment of MPM has changed dramatically in the past few years. The MAPS study was published in 2016 and revealed an OS advantage when bevacizumab was added to platinum/pemetrexed chemotherapy.9Zalcman G. Mazieres J. Margery J. et al.Bevacizumab for newly diagnosed pleural mesothelioma in the Mesothelioma Avastin cisplatin Pemetrexed a 3 Full Text Full Text PDF Scopus Google Scholar The of this has been variable, perhaps owing to the OS benefit the of the the lack of regulatory approval for the and studies as a in have been S. J. for patients with malignant pleural Oncol. PubMed Scopus Google Scholar this has been by the emergence of immunotherapy Most has been the an in survival outcomes for with platinum/pemetrexed as first-line treatment of P. et in malignant pleural mesothelioma a 3 Full Text Full Text PDF PubMed Scopus Google Scholar from a advantage in median there was a in survival at years. Nevertheless, have whether the OS advantage is limited to the population and whether 1 may a S. R. et chemotherapy in patients with malignant pleural mesothelioma from Oncol. Full Text Full Text PDF Google Scholar In the in or published the results of important the of one of the by the RWE data by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar the study randomized patients with MPM, treated with platinum-based chemotherapy, to or and revealed a OS with S. et in patients with malignant mesothelioma a 3 Oncol. Full Text Full Text PDF PubMed Scopus Google Scholar the study of active supportive care active supportive care and that of not this be an use of et al.A randomized trial of as therapy for patients with malignant pleural Clin Oncol. Scholar of interest is chemotherapy with The a study of revealed significant for the combination with differential based on the of et with first-line chemotherapy in malignant pleural mesothelioma a trial with a Oncol. 2020; Full Text Full Text PDF PubMed Scopus Google Scholar are the results of 3 trials this such as the Cancer trial platinum/pemetrexed with platinum/pemetrexed where RWE in the of randomized data published and in the MPM be to whether Kerrigan et K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar or provide RWE in MPM the start of the immunotherapy such data help to answer about the of and in a or having patient including help to patients with MPM benefit from immunotherapy and patients chemotherapy results of the 3 trials of chemotherapy immunotherapy are there will be interest in to an for these and the very RWE how have data from trials of the treatment being used in routine practice. RWE help for subsequent lines of including efficacy of platinum/pemetrexed as therapy or treatment such as and have clinical In the by Kerrigan et al.1Kerrigan K. Jo Y. Chipman J. et al.A real-world analysis of the use of systemic therapy in malignant pleural mesothelioma and the differential impacts on overall survival by practice pattern.JTO Clin Res Rep. 2022; 3: 100280Abstract Full Text Full Text PDF Scopus (6) Google Scholar the strengths and weaknesses of by and care in these patients to be in line with current in this not being able to on that despite RWE will not the by and in the levels of scientific when using often standards such as the Observational Medical Outcomes minimal Common Oncology Data or Clinical Data Interchange Standards RWE can and provide and such research be and review and review and review and review and Real-World of the of in Mesothelioma and the on by Clinical and Research pleural mesothelioma (MPM) is an that with real-world treatment This study the overall survival of patients with MPM by of first-line platinum chemotherapy immunotherapy chemotherapy, and by of maintenance therapy PDF

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