4. Not having a regulatory strategy
Early on, get the latest and greatest in regulatory expertise. Make sure your chosen advisor is well-versed in current FDA requirements and issues so that he or she can recommend the best strategy for clinical development and regulatory approval. According to Ferruolo, it is important for biotechs to identify the best regulatory strategy for getting approval, including understanding alternative clinical designs and exploring other indications or endpoints that may be a more viable path to regulatory approval. "Too often companies pursue a regulatory path without critically assessing other possibilities that may be reasonable given the company's resources and objectives," says Ferruolo.
5. Not listening to the FDA
It's crucial for biotechs to engage the FDA throughout the course of drug development; not having a dialogue with the agency can delay or even kill a drug. Have a pre-IND meeting with the FDA, if possible. Ferruolo notes that the agency has been reluctant to have IND meetings recently, but companies should try to keep an open line of communication anyway.
If your company does manage to score a meeting, take to heart what the agency says. "When the FDA says ‘we think you ought to consider' something, that should not be viewed as a suggestion, but as a mandate," he explains. "Companies are foolish if they don't follow what the FDA has suggested." A biotech takes on significant and unnecessary risk if it doesn't follow an FDA suggestion or proactively address the FDA's concerns.
6. Designing trials poorly
"I once heard a very experienced biotech pioneer say, 'there's no such thing as a failed drug, but there are failed trials,'" recalls Ferruolo. "If you have a drug/biologic that has activity, when it fails, it's not because of the drug or molecule, it fails because you haven't designed the trial properly." Do you have the right endpoints? Are they appropriately defined? Are you pursuing the right indications? Know the answers to those questions before starting expensive clinical trials.
7. Not properly powering trials
Pivotal clinical trials must be properly powered based upon the level of activity shown in earlier trials and preclinical studies. Big Pharma typically powers their trials at no less than 80 percent. If you are an emerging biotech designing pivotal trials for the drug candidate that is going to make or break you, you should aim to power the trials at 90 percent. Admittedly, there may be reasons--above all limited financial resources--that will make that hard to do. But the alternative may be that tombstone press release where you have to disclose that while the data from the trials showed "positive trends," the results were not statistically significant and the endpoints were not met.
8. Relying heavily on third parties, especially CROs
No third party will put the urgency and care into your development program that you will. When it comes to working with CROs, often your trials will be of a much lower priority than those of bigger company that are repeat clients. There is a strong trend to outsourcing, but frequently the more a small company can do in-house the better, according to Ferruolo. Of course, going it alone is expensive and companies have to find a practical balance between in-house work and outsourcing. "There will be a variation in how much outsourcing is necessary," he explains. "But do as much as you reasonably can do with people who live, die, sleep and eat thinking about nothing but your program. What they lack in experience may be made up in commitment."