1689O - Comprehensive mapping review of real-world evidence publications focusing on targeted therapies in solid tumors: A collaborative work from ESMO real-world data and Digital Health Working Group

Background

A growing body of real-world evidence (RWE) aims to better reflect outcomes of cancer patients treated in real-world settings. We aimed to conduct a first comprehensive mapping review of the RWE produced over the past 3 years in terms of tumor type, treatment strategies, setting, and data sources, focusing on targeted therapies (TT) in solid tumors.

Methods

We conducted a systematic review in PubMed of RWE studies published between 01/2020 and 12/2022. We identified non-interventional studies using observational data, focusing on solid tumors exposure to targeted therapies, excluding immunotherapies. Abstract and full-text screening were performed by 11 independent reviewers.

Results

A total of 7,774 publications were retrieved with 1,251 considered eligible and extracted. The number of publications per year progressively increased during this period (328 in 2020; 421 in 2021; 502 in 2022). Most studies (50%) were performed in Asia, followed by Europe (25%) and North America (17%). Only 8% of studies had patients treated in more than one country. Treatment effectiveness and safety were assessed in 71% and 42% of studies respectively. Main data sources were medical records (60%), different types of registries (12%), and standardized data (9%). The majority were retrospective cohorts (87%), while 8% were prospective cohorts and 16% could be classified as population-based. Small molecules (62%) were the most frequently type of TT investigated. Most studies (30%) assessed gastrointestinal tract tumors, followed by lung (23%) and breast tumors (21%). In 17% of cases, study funding was industry-based.

Conclusions

RWE publications on TT for solid tumors are heterogeneous and mostly rely on retrospective data such as medical records. Population-based and international studies are rare. Collaborative efforts towards international representativeness and the use of routinely collected and/or standardized data sources must be encouraged to increase the relevance and future quality of publications and their potential impact on oncology practice.

Dr Perrat and Dr Grinda contributed equally to this study.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

ESMO Real-world Data and Digital Health Working Group.

Funding

Has not received any funding.

Disclosure

A. Pellat: Financial Interests, Personal, Speaker, Consultant, Advisor: Servier; Non-Financial Interests, Personal, Speaker, Consultant, Advisor: IPSEN; Non-Financial Interests, Personal, Other: MSD, Ipsen, Sanofi. A. Prelaj: Financial Interests, Personal, Other, Training of personnel: AstraZeneca, Italfarma; Financial Interests, Personal, Invited Speaker, The HIVE Project: Discussant: Roche; Financial Interests, Personal, Advisory Board, Advisory board in Lung Cancer project: BMS; Financial Interests, Personal, Other, Travel Grant: Janssen. A. Valachis: Financial Interests, Institutional, Research Grant, Research Grant for a research project on Molecular profiling of triple negative breast cancer: Roche; Financial Interests, Institutional, Coordinating PI, PRODAT trial: Novartis; Financial Interests, Institutional, Coordinating PI, CHECKMATE-401 trial: Bristol Myers Squibb; Financial Interests, Institutional, Coordinating PI, CHECKMATE-76K trial: Bristol Myers Squibb; Financial Interests, Institutional, Coordinating PI, TELEPIK trial: Novartis; Financial Interests, Institutional, Coordinating PI, DESTINY-Breast09 trial: AstraZeneca. D. Martins Branco: Financial Interests, Personal, Advisory Board, (14/01/2021): Janssen; Financial Interests, Personal, Advisory Board, (22/03/2021): Merck Sharp & Dohme; Financial Interests, Personal, Invited Speaker, (03/02/2022 and 23/09/2022): AstraZeneca/Daiichi Sankyo; Financial Interests, Personal, Invited Speaker, (18/06/2021): Novartis; Financial Interests, Personal, Other, Meeting/travel grant (11/02/2021): Merck Sharp & Dohme; Financial Interests, Personal, Other, Meeting/travel grant (07/12/2021).: Novartis; Financial Interests, Institutional, Research Grant, Institutional funding for an observational research project.: Novartis; Financial Interests, Institutional, Research Grant, Institutional funding for an investigator-initiated clinical trial (NCT05075538).: F. Hoffmann-La Roche Ltd; Financial Interests, Institutional, Other, Work for Institut Jules Bordet as academic partner medical advisor in two industry-sponsored clinical trials (NCT01358877 and NCT03498716): F. Hoffmann-La Roche Ltd; Financial Interests, Institutional, Other, Institutional funding for an investigator-initiated clinical trial (NCT03339843): Eli Lilly; Non-Financial Interests, Member of Board of Directors: Associação de Investigação e Cuidados de Suporte em Oncologia - Portuguese MASCC affiliate; Non-Financial Interests, Leadership Role, Portuguese Young Oncologists Committee Chair: November 2020 - May 2022: Sociedade Portuguesa de Oncologia; Non-Financial Interests, Leadership Role, Oncology Committee Chair: January 2020 - January 2021: Health Parliament Portugal; Other, Other, Employment of non-household immediate family member: F. Hoffmann-La Roche Ltd. G. Nader Marta: Non-Financial Interests, Personal, Other: Roche, Bayer, AstraZeneca. B. Wilson: Financial Interests, Speaker, Consultant, Advisor: AstraZeneca; Financial Interests, Research Grant: BMS. F. Montemurro: Financial Interests, Personal, Other: Roche. L. Castelo-Branco: Financial Interests, Institutional, Full or part-time Employment, Full time employment with ESMO as Scientific and Medical Division Fellow: European Society for Medical Oncology; Non-Financial Interests, Advisory Role, Advisor for medicines policy (2016-ongoing): Portuguese Medical Board. G. Pentheroudakis: Financial Interests, Personal, Full or part-time Employment, Chief Medical Officer: ESMO; Non-Financial Interests, Member: ASCO, Hellenic Cooperative Oncology Group (HeCOG), Hellenic Society of Medical Oncology. S. Delaloge: Financial Interests, Institutional, Advisory Board: AstraZeneca, Novartis, Pierre Fabre, Orion, Sanofi, Rappta, Cellectis, Isis/Servier; Financial Interests, Institutional, Invited Speaker: Exact Sciences, Pfizer, Seagen, Lilly, AstraZeneca, MSD; Financial Interests, Institutional, Advisory Board, ad board: Besins Healthcare; Financial Interests, Institutional, Invited Speaker, ESMO symposium: Gilead; Financial Interests, Institutional, Advisory Board, scientific board: Elsan; Financial Interests, Institutional, Steering Committee Member: Roche Genentech, BMS, Puma, AstraZeneca, Sanofi, Pfizer; Financial Interests, Institutional, Local PI: Orion; Financial Interests, Institutional, Funding: GE; Financial Interests, Institutional, Coordinating PI, clinical research funding to my institution: Taiho; Non-Financial Interests, Member of Board of Directors, Société Française de Sénologie et Pathologie Mammaire: SFSPM; Non-Financial Interests, Principal Investigator, H2020 funding: European Commission. M. Koopman: Financial Interests, Institutional, Advisory Board, advisory board and speaker: Pierre Fabre; Financial Interests, Institutional, Advisory Board: MSD, Bayer; Financial Interests, Institutional, Advisory Board, Advisory Board, speaker: Servier; Financial Interests, Institutional, Invited Speaker: Merck, BMS; Financial Interests, Institutional, Trial Chair: Servier; Financial Interests, Institutional, Research Grant: Servier, Roche, Bayer, Bristol Myers Squibb, Merck, Personal Genomics Diagnostics, Sirtex, Pierre Fabre; Financial Interests, Institutional, Funding: Pierre Fabre, Amgen, Nordic Pharma, Novartis, Merck, Servier, BMS; Non-Financial Interests, Leadership Role, vice-chair of DCCG: Dutch Colorectal Cancer Group; Non-Financial Interests, Advisory Role, Member of KWF scientific board: KWF; Non-Financial Interests, Other, ESMO faculty member for the Gastro-Intestinal Tumours – colorectal cancer: ESMO; Non-Financial Interests, Advisory Role, expert member of committee “regie op registers dure geneesmiddelen” ZINNL: ZiNNL; Non-Financial Interests, Advisory Role, CRC expert on Kanker.nl platform for answering online CRC questions of CRC (non) patients: Patient representative organisation (Kanker.nl); Non-Financial Interests, Leadership Role, chair of RWD & DH working group: ESMO; Other, Other, PI of the Dutch Prospective Colorectal Cancer Cohort study: PLCRC project. All other authors have declared no conflicts of interest.