New England Journal of Medicine
May 4, 2017 Vol. 376 No. 18
The Changing Face of Clinical Trials
Jeffrey M. Drazen, M.D., David P. Harrington, Ph.D., John J.V. McMurray, M.D., James H. Ware, Ph.D., Janet Woodcock, M.D., Editors
Academic, Foundation, and Industry Collaboration in Finding New Therapies
Bonnie W. Ramsey, M.D., Gerald T. Nepom, M.D., Ph.D., and Sagar Lonial, M.D.
N Engl J Med 2017; 376:1762-1769 May 4, 2017 DOI: 10.1056/NEJMra1612575
Breakthroughs in the ability to probe and better understand biologic systems during the past 30 years1-3 have enabled the medical community to develop new therapeutic agents and change the course of many life-shortening diseases.4,5 Despite this success, bridging the gap between promising laboratory observations and the development of effective therapies remains risky and expensive, with fewer than 1 in 10,000 early translational programs successfully achieving Food and Drug Administration (FDA) approval, at a cost of nearly $1 billion.6 Most therapeutic development fails in the preclinical phase, which is sometimes described as the “valley of death.”7
For this reason and because therapies for some conditions will have a limited eventual market value, the pharmaceutical industry has been hesitant to initiate early-stage programs to treat so-called orphan diseases. In recognition of a critical need, federal agencies have developed programs to catalyze innovation and reduce barriers to early development of new therapies.8 In the past two decades, disease-focused foundations also have developed a new approach to bridging this preclinical gap. In a process known as venture philanthropy, such foundations have formed partnerships with industry and federal agencies to share the financial risk of therapeutic development, shorten the early translational pipeline, and advance research with “a focus on human, not financial, return.”9 In addition, foundations and their academic partners have accelerated early development by providing access to patient populations for clinical trials and assistance from disease-specific experts in study design, which has helped in bridging the gap in therapeutic development.
In this review, we will focus on three diseases — cystic fibrosis, multiple myeloma, and type 1 diabetes mellitus — to illustrate how collaborations among academic institutions, foundations, and industry partners have evolved to address the therapeutic challenges of these conditions.