FDA’s willingness to be flexible in clinical trial designs for rare diseases and other hard-to-study conditions does not eliminate the need for biopharma companies, particularly small ones, to be cautious in interpreting results from early trials. That was one message from a New England Journal of Medicine article on drug development challenges for small biopharma companies.
Small Biopharmas Should Be Wary Of Early Results – US FDA Official
Two former Genzyme execs, one of whom now works at CDER, warn small companies to avoid over-emphasizing results from early, uncontrolled trials in their planning and business decision-making.
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