Objective: This presentation highlights a new precompetitive consortium based strategy for the successful development of regulatory-endorsed biomarkers and quantitative drug development platforms targeted at the early stages of PD.

Background: Critical Path for Parkinson’s (CPP) is a consortium funded by Parkinson’s UK and the pharmaceutical industry, and led by Critical Path Institute (C-Path). A coalition of industry members, regulatory agencies, academic experts and patient advocacy groups collectively aim to integrate data from international observational cohorts (CamPaIGN, OxfordPD, ICICLE, PRoBaND, PPMI) and randomized controlled clinical trials (ADAGIO and PRECEPT) into a unified database (Stephenson, 2015).

Methods: Regulatory agencies have identified quantitative disease models as valuable drug development platforms to accelerate drug development. The precedent for CPPs vision is based on the success of C-Paths Coalition Against Major Diseases (CAMD) in achieving the first-ever regulatory endorsement by the FDA and EMA for an Alzheimer’s disease (AD) clinical trial simulation tool (Romero, 2015). The clinical trial platform is publically available, and represents a milestone that serves to encourage the advancement of drug-disease-trial models, promising to increase the probability of success in future AD therapeutic trials.

Results: CPP plans to replicate the AD successful regulatory path in PD. Key to enabling successful data integration, C-Path in collaboration with NINDS and CDISC, has successfully developed publically available global consensus clinical data standards for Parkinson’s disease.

Conclusions: The goal of CPP is to qualify novel translational biomarkers and drug disease trial models for use in efficient clinical development programs designed to lead to the registration of new treatments for PD. The focus of CPP is on early stages of PD with the goal of advancing treatments that hold the most potential for delayed disease progression. Romero, K., et al. (2015) The future is now: model-based clinical trial design for Alzheimes disease. Clinical Pharmacology & Therapeutics, 97(3): 210-4. Stephenson D. T., et al. (2015) Precompetitive data sharing as a catalyst to addressing unmet needs in PD. Journal of Parkinson’s disease 5: 581-94. Disclaimer: The views expressed in this abstract are those of the authors and do not necessarily reflect the official views of FDA and EMA.

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