§360bbb–8 Consultation with External Experts on Rare Diseases, Targeted Therapies, and Genetic Targeting of Treatments

Summary

Requires the Secretary to make sure the FDA offers chances to talk with outside experts about rare diseases and genetically targeted drugs. The agency must keep a list of outside experts who have scientific or medical training the FDA lacks and may ask them for advice when needed. Topics for advice include how serious a rare disease is, unmet medical needs, whether patients can join trials, benefits and risks of treatments, how to design trials for small groups, who the patients are, and the science of small studies. Outside experts can sometimes be treated as special government employees. Confidential business or trade secret information stays protected. The FDA cannot share such information with an expert without the drug sponsor’s written consent unless the expert is a special government employee or another law allows it. The rule does not change earlier consultation practices that existed before July 9, 2012. It does not create a legal right to a meeting or force the FDA to meet any specific person, and it does not change agreed goals, procedures, or the number of review cycles in effect before July 9, 2012. Before using an outside expert on an investigational new drug application (section 355(i)), a new drug application (section 355(b)), or a biologics license application (section 262 of title 42), the Center director must decide the consultation will help finish the review and fix remaining problems, or the sponsor must have allowed the consultation. Those director-review rules apply only when the consultation is done only under this authority.