PMU100 - MAPPING BENEFIT-RISK DECISION-MAKING PROCESSES AND IDENTIFYING DECISION POINTS WITH THE POTENTIAL TO INCLUDE PATIENT PREFERENCE INFORMATION THROUGHOUT THE MEDICAL PRODUCT LIFECYCLE

Abstract

Patient preference information (PPI) has not been effectively integrated in decision-making throughout the medical product lifecycle (MPLC). A first step requires an understanding of existing processes and decision points to know how to incorporate PPI to improve patient-centric decision making. The aims were to: 1) identify the decision-making processes and decision points throughout the MPLC for stakeholders, and 2) determine which decision points have potential to include PPI. A 3-step approach was conducted, including a scoping literature review identifying relevant white and grey literature, validation meetings with stakeholders to confirm decision-making processes, and semi-structured interviews with representatives of 3 stakeholder groups (industry n=24, regulatory n=22, HTA n=24). The literature review was conducted using five scientific databases, and interviews were conducted within seven different European countries and the US. Six decision points were identified for industry decision-making process: 1) Select & prioritize targets and leads; 2) Prioritize studies; 3) Prioritize assets; 4) Optimize & Prioritize assets; 5) Regulatory Submission & Launch; and 6) Manage MPLC & Prioritize opportunities. Four decisions points for the regulatory decision-making process: 1) Submission and validation; 2) Scientific opinion; 3) Orphan designation; and 4) Commission decision. Six decision points for HTA decision-making: 1) Filtration; 2) Prioritization; 3) Appraisal; 4) Filtration II; 5) Prioritization II; and 6) Appraisal II. PPI is currently not considered required information to be submitted for decision-making, but has the potential to be included at most key decision points. Currently, PPI is not considered as obligatory information to submit for any of the MPLC decision points, nor is it a pre-set criterium for decision-making. PPI is considered an important component by most stakeholders to inform future decision-making across the MPLC. Acknowledgement: This work received support from the EU/EFPIA Innovative Medicines Initiative [2] Joint Undertaking PREFER grant n° 115966