FDA Has the Final Say for What Studies Are Needed for Approval
[Thursday, February 20, 2020] Last year, Vanda Pharmaceuticals sued FDA for putting its clinical trial on partial hold for lack of nonclinical data from dog studies. This week, a federal court ruled that FDA has the authority to ask for any additional data that FDA’s reviewers deem necessary to lift the clinical hold. The court further ruled that Vanda should have used FDA’s internal dispute resolution process for arbitration rather than asking the court to decide the matter. After more than a year of litigation, the company is back to where it was, namely, conducting a dog safety study to support its clinical program. This should not be surprising for anyone as courts have repeated deferred to FDA’s opinions about the applications it regulates. This case highlights several commonly known but apparently ignored processes by Vanda. First, if there are potential safety issues raised in the small animal studies, there is no way one can get away without doing a large animal study to show safety. Second, FDA provides rationale for all its decisions. The rationale is developed and agreed upon by the entire review team along with FDA’s legal experts, particularly for issues that are controversial to the sponsor. So, arguing with the same team to change its mind rarely works. FDA teams can present you additional justification for their rationale ad nauseum. The sponsor can escalate the issue to senior management in a dispute resolution meeting or simple bite the bullet and do the work. Third, treatments for non-life-threatening conditions generally have a lower threshold for safety. Safety is a contextual concept. While higher level of risk may be acceptable for products intended to treat certain diseases, the general principle of a full battery of non-clinical safety to support human use still applies. Although FDA has stated that it wants to reduce non-clinical studies, particularly large animal studies like those done in dogs, don’t believe it till you see a written policy and even then expect FDA to ask you to do the extra work unless you have addressed the safety issue in other ways. Non-clinical safety studies still don’t have many alternates. Lastly, litigation with FDA should be the very last resort, after all other options have been exhausted and the studies proposed by FDA would be non-feasible. FDA loses very few court battles, and there is a good reason for that. Don’t litigate, just do it, and save yourselves a lot of pain. |
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