Abstract A085: Investigating real-world molecular testing within both clinical trial selected sites and surrounding sites to identify eligible study patients - a model for streamlined precision medicine clinical trial operations in oncology
Authors: Cushion Michael G , Rosenthal Michael Bennett , Benson Donald , Hodge Jeffrey , Brayer Marion , Wu Chang-I 
Published: March 2020

Experimental Procedures: A growing number of oncology clinical trials require a somatic gene mutation/alteration for eligibility. Without prior and timely knowledge of the patient’s tumour molecular status, current practice is to determine this from a screening test during trial procedures. This can often lead to lengthy recruitment timelines, particularly for populations with rare selective biomarkers. Our approach was to investigate and quantify real-world genomic testing data both within and local to sites selected for two ongoing studies, in order to i). understand molecular testing trends within these sites and ii). investigate the feasibility of a data-driven approach to facilitate the referral of eligible patients from surrounding sites to study sites - through the quantification of patient potential based on relevant real-world molecular testing results. A one year data look-back period was applied, the target populations were i). patients diagnosed with advanced cholangiocarcinoma and testing positive for somatic FGFR2 rearrangements and ii). patients diagnosed with advanced bladder cancer and testing positive for somatic FGFR3 mutations or translocations. These parameters were selected as they represent the target patient populations for two ongoing clinical trials. Summary Results: For simplicity, selected results only in France are given in the table below - data on multiple other geographies also exists. Study population/molecular biomarkerNo. of selected trial sitesNo. of real-world molecular testing sitesNo. of patients with a positive result - all testing sites combinedNo. of matching (identical) sites - trial & molecular testing sitesNo. of non-matching (non-identical) sites within 50 kilometres of each other - trial & molecular testing sites Advanced Cholangiocarcinoma/FGFR21315332215 Advanced Bladder Cancer/FGFR316191,063423

Conclusions: These data have provided invaluable insights into real-world FGFR2/FGFR3 molecular testing within sites, giving an enhanced understanding of molecular testing trends. Additionally, new sites in relatively close proximity (50 kilometres) to trial sites with patient potential have been identified. Strategies are underway to consider the best approach to action these findings, i.e. develop a referral network from surrounding sites to trial sites and ultimately accelerate the recruitment of eligible patients into these ongoing clinical trials, which they otherwise may have been unaware of. Whilst the one year look back period applied was informative as a first step, the key to our future approach will be to identify new patients from prospective real-world molecular testing, and to recruit these patients into the relevant trial before they become otherwise ineligible, e.g. through changing tumour molecular status or death. This nascent approach has the potential to be expanded upon and become a more widespread – and necessary – approach to targeted therapy trials, leading to accelerated clinical development timelines and bringing efficacious new medicines to patients in need of these. With the emergence of broad genomic profiling, the availability of actionable, real-world molecular data to support clinical trial enrolment is also expected to grow.

Citation Format: Michael G Cushion, Michael Bennett Rosenthal, Donald Benson, Jeffrey Hodge, Marion Brayer, Chang-I Wu. Investigating real-world molecular testing within both clinical trial selected sites and surrounding sites to identify eligible study patients - a model for streamlined precision medicine clinical trial operations in oncology [abstract]. In: Proceedings of the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics; 2019 Oct 26-30; Boston, MA. Philadelphia (PA): AACR; Mol Cancer Ther 2019;18(12 Suppl):Abstract nr A085. doi:10.1158/1535-7163.TARG-19-A085