Opportunities and Challenges in Translational Therapeutics

While the number of new therapeutics approved by the FDA has remained relatively constant over the past 60 years, the cost of this endeavor has increased in a log linear fashion. This mainly reflects the exploding cost of failure of a model that is clearly unsustainable. Experience in the altruistic sector has illustrated the temporal and scientific efficiency of a globalized modular approach to drug discovery and development where (i) behavior is incented by altruistic ideals and a pot of money and (ii) intellectual property barriers are collapsed by the perception of there being no money to be made. Can this model be exported to the for-profit sector? There is some movement in that direction as Pharma seeks to outsource some of its activities, although still retaining most of the control -- a touch of perestroika with a whiff of glasnost. However, more radical reform is likely, driven primarily by crisis rather than the prospect of opportunity. This will require a fundamental revision of assumptions around intellectual property, an increased capability of academic medical centers to play in this space, a redeveloped workforce with skills in translational medicine and therapeutics, regulatory reform of incentives in drug development and a bioinformatic infrastructure capable of sharing heterogeneous information in a secure and compliant manner across distinct organizational structures and on a global scale.