Drug Target Identification and Validation: Global Pharmaceutical Industry Experts on Challenges, Best Strategies, Innovative Precompetitive Collaboration Concepts, and Future Areas of Industry Precompetitive Research and Development

Ther Innov Regul Sci. 2016 Nov;50(6):769-776. doi: 10.1177/2168479016651298. Epub 2016 Jul 10.


Focused interviews were conducted with global pharmaceutical company representatives in order to derive a consistent view on drug target identification/validation challenges, collaborative strategies, and future developments in a precompetitive space. Analysis revealed translation into clinical utility as a major hurdle of novel drug target validation, originating from lack of biological understanding, irreproducibility of published results, and lack of valid animal models. Direct and close collaborations with academia are the preferred model to tackle basic research on novel drug targets in high-risk projects. Efforts to conduct target identification in large precompetitive consortia are acknowledged with some doubts about the pace of progress and data-sharing policies, while concept to extend the precompetitive space to target validation in phase II trials was curtailed to niche indications together with a revision of current intellectual property (IP) practice. Public-private partnerships in established areas are forecasted to increase. Novel emerging themes are toxicology data sharing, joint genetic patient data analysis, and reimbursement concepts.

Keywords: drug target identification and validation; pharmaceutical industry; precompetitive collaboration concepts; public-private partnerships.