Small Companies Use Smart Strategies to be Successful with FDA
[Posted on: Thursday, February 23, 2017] Small drug companies with limited resources have become increasingly smart at using innovative strategies to get market approval for their products by FDA according to a review published earlier this month. The review, co-authored by FDA personnel, lists four major strategic approaches that lead to positive NDA or BLA decision for the product being reviewed. This information should not come as a surprise to industry insiders but they present an interesting summary for all developers of new drugs, irrespective of the size of their enterprise. Small companies usually go for easier, shorter steps to find development projects that fit the resources and time available. Companies more frequently use new technical platforms, and creative trial design. New formulations for previously approved drugs are also popular due to their lower perceived risk of failure. Of the 19 companies reviewed by the authors, 9 went for 505(b)(2) products. Other companies took over products that were not being pursued by larger companies for any reason, developed them to a predefined milestone and then partner with a larger company to add resources and experience. In addition to these business strategies, there are four regulatory strategies that helped specific cases. The first was using a historical control group when using a placebo was not feasible or ethical. In case of a rare life-threatening rapidly progressing condition (Pompe disease) with only one previously approved drug, FDA accepted a clinical trial using historical control group since using a placebo would be unethical. Using a surrogate marker to define the clinical endpoint was another successful regulatory strategy. The third regulatory approach was enriching trial population using genetic screening to identify subjects with higher potential of benefit. FDA has talked about clinical trial enrichment strategies and even has a guidance document on the subject since 2012. The fourth regulatory strategy was to address high placebo response in an indication using creative trial designs. By some estimates more than 70% of drug companies are small businesses with limited resources and extreme pressures to succeed. Only about 10% of new drugs that enter the clinical phase succeed in market approval by the FDA. And this number includes products developed by both large and small companies. So, any measures that increase the odds of success are welcome.
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