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February 06, 2017
The temptation to use uncontrolled, early, small studies to support further development of products may prove problematic for emerging biopharmaceutical companies, and these firms require both innovative approaches and rigor for success, according to an article posted today (February 2nd) by the New England Journal of Medicine.
“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,” noted lead author Richard A. Moscicki, M.D., Deputy Director for Science Operations at the Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland (N Engl J Med 2017; 376:469-474).
Small biopharmaceutical companies range in size from virtual companies with no commercial products and no revenue to those with only a few commercial programs, and they are becoming increasingly important as drivers of innovation in drug development, he explained. These firms often encounter important challenges in designing and implementing clinical development programs.
Small companies use a variety of approaches to address these challenges, including the use of new technical platforms, the use of new formulations or technologies that enhance the actions of known drugs, and the use of trial designs that take advantage of the specific market they hope to enter. Other businesses develop products that are spun off from or licensed from large companies, according to Moscicki.
“Many small companies also repurpose drugs or pursue narrow niche markets - such as rare inherited diseases, uncommon cancers, or specific infectious diseases - to remain viable,” he wrote. “Furthermore, many companies turn to rare diseases for an opportunity to successfully negotiate many of the aforementioned issues, though such diseases present their own challenges - in particular, the small number of patients available for clinical trials.”
The article focuses on strategies used by these companies, with an emphasis on drugs developed for rare diseases. For further details, click here.