Small Biopharma Face Distinct Challenges: FDA Official and Sanofi Genzyme SVP Argue

Small biopharmaceutical companies, often known as the drivers of innovation, face a number of unique challenges in bringing new treatments, particularly for rare diseases, to market, Richard Moscicki, deputy director for science operations at the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research, and P.K. Tandon, senior vice president of Sanofi Genzyme, wrote.

In an article published Tuesday in the New England Journal of Medicine, the authors concluded: “Temptations to use uncontrolled, early, small studies to support further development of products may prove problematic. Small companies with limited resources require both innovative approaches and rigor for success.”

Moscicki and Tandon offer four examples of challenges for small biopharmaceutical companies: the use of a historical control group when the use of placebo is problematic; the use of a new biomarker endpoint that is likely to predict a clinical benefit; predictive enrichment with the use of a genetic marker; and a high placebo response rate and the importance of controlled trials.

“The unique challenges of clinical development by small companies are often addressed with the use of smaller clinical development programs than those used by large companies. Development of drugs for rare diseases may be used as a strategy, but the small size of the populations with such diseases and the small samples available for trials require approaches that can maximize the power to detect efficacy, which can include the use of historical controls, new surrogate end points, or enrichment for participants who are likely to have a response,” they wrote.

Also on Thursday, FDA offered a new webinar for small businesses to better understand best practices for communication between FDA and investigational new drug sponsors during drug development.