Objective: There is a growing sense of urgency to integrate the voice of the patient in drug development in Parkinson’s disease (PD). Public private partnerships are uniquely poised to align stakeholders and enable innovative patient-focused drug development paradigms. Strategy and initial progress will be presented on the development of a unique Quantitative Systems Pharmacology (QSP) model for PD therapeutics.

Background: QSP uses mathematical models to characterize pathophysiology and drug pharmacology in the development of new pharmaceuticals. The heterogeneous nature of PD pathophysiology, however, can lead to unanticipated side effects, off-target effects, pharmacodynamic drug interactions, and poor patient response in general. A fully validated QSP model for will require PD patient engagement in designing the model and identifying the appropriate targets.

Method: An initial QSP model framework will be generated using the LIFE approach developed by Rutgers’ QSP group and in collaboration with the QSP Steering Committee. An extensive literature review will be harmonized using artificial intelligence/machine learning querying to form the base for the QSP model. Software tools for the simulation of the QSP model will also be developed. A platform (QTIPS) will be created that will serve as a conduit for PD patients’ real-world experiences in the development of the model.

Results: Identification of the target literature for the initial QSP model development has been initiated. An R/Shiny-based prototype QTIPS platform has been developed (barrettkyle.shinyapps.io/SwarmPrototype) as a proof-of-concept tool to facilitate patient engagement. Input from people living with PD has been ongoing to assure that the QSP model reflects concepts and potential new targets that are aligned with patient needs.

Conclusion: The development of a QSP model for PD drug development complements important principles of the Critical Path for Parkinson’s Consortium by engaging the PD patient’s voice, both on the developmental side and as a contributor of real-world data. This will allow to perform virtual trials informed by the final, validated QSP model, to generate patients (clinical avatars) that better resemble real-life patients. QTIPS may have tremendous impact for drug development and provide a tool for PD patients to make more informed individualized drug choices.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/model-innovation-in-parkinsons-disease-drug-development-linking-quantitative-systems-pharmacology-to-the-patient-voice/