1702P - Real-world evidence contributions to European medicines agency’s safety and efficacy evaluations of oncology targeted therapies between 2018-2022

Background

While Real-world Evidence (RWE) has documented value for safety monitoring and disease epidemiology, its objective contribution to safety and efficacy evaluations for regulatory purposes is still unclear. Here, we aim to describe the prevalence and type of RWE considered by European Medicines Agency (EMA) as contribution to efficacy and safety-related evidence generation among approved oncology targeted therapies.

Methods

On March 10, 2023, we screened the medicines listing of EMA to identify all anti-cancer targeted therapies for solid malignancies with a decision date (initial marketing authorizations and extension of indications) between 2018-2022. We screened the European public assessment reports (EPARs) using a standardized approach to collect data on RWE. When generated pre-authorization, the RWE contribution to the final regulatory decision was classified as definitive, supportive, or non-supportive. For published RWE studies, we collected their main characteristics.

Results

Out of a total of 1976 medicines, we identified 55 oncology targeted therapies, corresponding to 75 EPARs (indications), which are described in the table. The use of RWE in regulatory deliberations occurred in 24/75 (32%) EPARs, increasing from 30% in 2018-2020, to 34% in 2021-2022. Pre-authorization RWE was described in 20/24 (83%) EPARs, among which none were definitive, 8 RWE studies (in 7 EPARs) non-supportive, and 20 RWE studies (in 15 EPARs) were supportive of the decision. Published RWE (n=14) originated from data aggregators (n=5), patient registries (n=3), health records (n=3), standardized cohort data (n=2), or from combined sources (n=1).

Table: 1702P

Conclusions

Over the past 5 years, RWE involvement in the approval of oncology targeted therapies in Europe tends to increase, with the majority being supportive for EMA regulatory decision making complementary to traditional clinical trials.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

Project from ESMO Real-world Data and Digital Health working group.

Funding

Has not received any funding.

Disclosure

J.W.G. Derksen: Financial Interests, Institutional, Research Funding, Institutional funding for clinical research project: Nordic Pharma. 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. S.C.M.W. van Nassau: Financial Interests, Institutional, Research Funding, Unrestricted Institutional Grant for clinical research: Pierre Fabre. 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. 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 respresentative 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. 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. 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. 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. All other authors have declared no conflicts of interest.