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Small biopharmaceutical companies are becoming increasingly important as drivers of innovation in drug development. It has recently been estimated that the majority of drugs currently in development are in the hands of small biopharmaceutical companies.1 Such companies range in size from virtual companies with no commercial products and no revenue to those with only a few commercial programs.
Small biopharmaceutical companies often encounter important challenges in designing and implementing clinical development programs. In a context in which only approximately 10% of clinical programs result in drugs that achieve regulatory approval, small-company clinical programs may have an even lower rate of success than that of large companies2 owing to limited internal experience in clinical development and limited infrastructure, which may also affect manufacturing and clinical supply. However, these challenges are largely overshadowed by limited resources and funding, which in turn fuel demand for short timelines owing to the need to demonstrate progress to investors. As such, these companies must focus their resources on small, less-costly development programs for very specific targets and often must spearhead new approaches to testing new products in order to survive.
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, or the use of trial designs that take advantage of the specific market they hope to enter. Other companies develop products that are spun off from or licensed from large companies. In fact, many small companies may choose to partner with larger companies to add resources and experience.
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. 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. Table 1 illustrates some examples of approaches used by small companies that achieved Food and Drug Administration (FDA) approval for their drugs in 2014 and 2015.
It is beyond the scope of this article to provide a comprehensive review of all the challenges and strategies...