Generating and using real-world data: A worthwhile uphill battle.

The precision oncology paradigm challenges the feasibility and data generalizability of traditional clinical trials. Consequently, an unmet need exists for practical approaches to test many subgroups, evaluate real-world drug value, and gather comprehensive, accessible datasets to validate novel biomarkers. Real-world data (RWD) are increasingly recognized to have the potential to fill this gap in research methodology. Established applications of RWD include informing disease epidemiology, pharmacovigilance, and healthcare quality assessment. Currently, concerns regarding RWD quality and comprehensiveness, privacy, and biases hamper their broader application. Nonetheless, RWD may play a pivotal role in supplementing clinical trials, enabling conditional reimbursement and accelerated drug access, and innovating trial conduct. Moreover, purpose-built RWD repositories may support the extension or refinement of drug indications and facilitate the discovery and validation of new biomarkers. This perspective explores the potential of leveraging RWD to advance oncology, highlights its benefits and challenges, and suggests a path forward in this evolving field.

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Déclaration de conflit d'intérêts

Declaration of interests E.E.V. is founder and current member of the supervisory board of the Hartwig Medical Foundation, independent non-executive director of Sanofi, co-founder of Mosaic Therapeutics, and board member and founder of the Center for Personalized Cancer Treatment. He has received clinical study grants from Amgen, AstraZeneca, BI, BMS, Clovis, Eli Lilly, GSK, Ipsen, MSD, Novartis, Pfizer, Roche, and Sanofi.