gms | German Medical Science

Background and state of research: NGS-based, molecularly guided therapy is becoming a pivotal part of precision oncology. Rare molecular cancer subtypes pose difficulties in traditional clinical trials; however, collecting patient RWD can help elucidate the effectiveness of targeted therapies, molecular tumor boards (MTBs) and NGS-based genomic profiling.

Research question and objectives, hypothesis: WAYFIND-R (NCT04529122) is a global registry [1], [2] that:

1.

characterizes the treatments and clinical course of patients with cancer;

2.

provides a data research platform to evaluate real world treatment decisions and outcomes;

3.

supports the design/conduct of epidemiological research and clinical trials.

WAYFIND-R will provide key learnings on data quality, sharing, and privacy for contemporary disease registries.

Method: Unlike many disease registries, WAYFIND-R is global, prospective, longitudinal, generalizable (all clinically validated NGS-based tests of any panel size are permitted), and includes NGS testing and standardized data collection to enhance data quality and minimize missing data. WAYFIND-R includes adults with any type/stage of solid tumor profiled with NGS no longer than 3 months prior to the enrollment date. NGS details, patient socio-demographics, cancer characteristics, relevant safety data, treatment decisions, and outcomes are collected at baseline and prospectively at least every 6 months. By 2026, WAYFIND-R aims to enroll ~15,000 patients in ≥35 countries from academic centers, community hospitals, and private clinics with regular MTBs.

Results: As of 15 March 2023, 1315 patients have been enrolled.

Discussion: A key component is the potential to explore global and local data within a secure analytics platform (also known as a trusted research environment), accessed through a visual dashboard to find treatment decisions for similar patients elsewhere. Researchers can access the patient-level dataset for correlative analyses, after independent data access committee approval. WAYFIND-R’s methodological/operational aspects were guided by external experts and the European Medicines Agency.

Implication for research: WAYFIND-R will inform on best practice for NGS-based treatment decisions by clinicians, foster global collaborations between cancer centers (enabling robust conclusions regarding outcome data to be drawn), aid understanding of disparities in patients’ access to advanced diagnostics and therapies, and ultimately drive advances in precision oncology.

Funding: Other funding; F. Hoffmann-La Roche Ltd, Basel, Switzerland