Delivering Solutions and Clinical Benefits for Diseases with Small and Intermediate-Size Patient Populations

Complementary skill sets, talent, capabilities, resources, infrastructure, and knowledge available in academic and pharmaceutical organizations have all greatly contributed to the delivery of novel medicines, innovative solutions, and clinical benefits to patients with major and more common diseases (. defined as diseases with large patient populations). In sharp contrast, patients with less common diseases (. defined as diseases with small and intermediate-size patient populations, including autoimmune diseases such as type 1 diabetes, multiple sclerosis, and celiac disease) have not profited to the same extent. The lack of industry involvement in less common diseases and the absence of critical resources and R&D capabilities in academia have severely hampered progress in this area. Here, the reasons for these greatly different outcomes are analyzed. Strategies that reduce risk, cost, and timelines are proposed to advance novel concepts, implement innovative solutions, and deliver clinical benefits for diseases with small and intermediate-size patient populations.