PMU55 HURDLES TO REGULATORY AND ACCESS (PRICING AND REIMBURSEMENT) OF 3D-PRINTED TUMOR ASSAYS FOR PERSONALIZED MEDICINE IN ONCOLOGY

Abstract

As the pharmaceuticals pipeline continues to focus on oncology, the number of approved companion diagnostic devices (CDx) tailoring treatment strategies is increasing. Using patient biopsies, 3D-printed tumor assays (TAs) testing drug response as an in vitro CDx are being developed. The objective is to identify hurdles to regulatory approval and market access (MA) for 3D-printed TAs in the US and EU5. Via analogue analysis, a scoring system was developed to identify the best comparators currently approved in each US and EU5 markets, as to anticipate the most appropriate assessment routes for future 3D-printed TAs. The final score X attributed is a weighed score out of 10, combining the PICO framework (Population, Intervention, Comparator, Outcome) and Technology aspects (Effectiveness of the test, Safety, R&D and Business Model). Sub-scores are defined based on expected results from a 3D-printed TA. Chosen analogues are fully mapped for their regulatory pathway, pricing and reimbursement model. As there are no approved 3D-printed TAs, more than 40 potential analogues have been evaluated. Three strong analogues (7 < X < 8) in the US (Next Generation Sequencing Panel, Somatic Gene Mutation Detection System & Recurrence Score), and 5 strong analogues in EU5 (1 per country) have been identified. Regulatory-wise, the biggest hurdle is the health technology assessment (HTA) conducted differently in the US (Pre-Marketing Approval by the Center for Devices and Radiological Health) and each EU5 country (National Notified Bodies). MA pathways are variable, with different reimbursement policies and discrepancies in funding as major hurdles to access. Although identified analogues tend to confirm the in vitro CDx pathway for 3D-printed TAs, limitations arise due to the novel technology used: scepticism around effectiveness, actual patient outcomes and value for payers. Companies willing to commercialize 3D-printed TAs should engage early-on with the different stakeholders involved (regulators, payers and physicians).