TY - JOUR AU - Chandra, Amitabh AU - Garthwaite, Craig AU - Stern, Ariel Dora TI - Characterizing the Drug Development Pipeline for Precision Medicines JF - National Bureau of Economic Research Working Paper Series VL - No. 24026 PY - 2017 Y2 - November 2017 DO - 10.3386/w24026 UR - http://www.nber.org/papers/w24026 L1 - http://www.nber.org/papers/w24026.pdf N1 - Author contact info: Amitabh Chandra John F. Kennedy School of Government Harvard University 79 JFK Street Cambridge, MA 02138 Tel: 617/496-7356 E-Mail: amitabh_chandra@harvard.edu Craig Garthwaite Kellogg School of Management Northwestern University 2211 Campus Drive Evanston, IL 60208 Tel: 847/491-2509 Fax: 847/467-1777 E-Mail: c-garthwaite@kellogg.northwestern.edu Ariel Dora Stern Harvard Business School Morgan Hall 433 Boston, MA 02163 Tel: 617-495-2332 E-Mail: astern@hbs.edu M1 - published as Amitabh Chandra, Craig Garthwaite, Ariel Dora Stern. "Characterizing the Drug Development Pipeline for Precision Medicines," in Ernst R. Berndt, Dana P. Goldman, and John W. Rowe, editor, "Economic Dimensions of Personalized and Precision Medicine" University of Chicago Press (2019) AB - Precision medicines – therapies that rely on the use of genetic, epigenetic, and protein biomarkers – create a better match between patients with specific disease subtypes and medications that are more effective for those patients. This heterogeneity in response has implications for the decision to develop therapies, their pricing, the design of clinical trials, and the relative importance of smaller biotech companies versus more traditional companies in pursuing early stage R&D. To understand the scope of these effects, we use a comprehensive database of over 130,000 global clinical trials and describe the drug development pipeline for precision medicines over the past two decades. We identify clinical trials for likely precision medicines (LPMs) as those that use one or more relevant biomarkers. We then further segment trials based on the nature of the biomarker(s) used and other trial features with economic implications. Since cancers represent a disease setting in which precision therapies are already successfully used, and since cancer applications of precision medicine are expected to grow rapidly over the coming years, we separately characterize cancer LPMs. Finally, we consider the types of firms pursuing R&D in precision medicines, considering how LPM R&D activities have evolved over recent years. ER -