§360bbb–8 Consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments

Summary

Makes the Food and Drug Administration set up chances, at the right time, to get advice from outside experts and stakeholders about rare diseases and genetically targeted drugs and biologics. The FDA must follow the PDUFA 2013–2017 guidance letters under the 2012 Prescription Drug User Fee Amendments when planning these consultations. The agency must keep a list of external experts who have scientific or medical training the FDA needs and may use them when that expertise is needed. Those experts can be treated as special government employees. Topics for advice include things like how rare or severe a disease is, unmet medical needs, whether patients can join trials, benefits and risks of treatments, trial design, who the patients are, and how to study very small groups. Trade secrets and other confidential business information stay protected the same way they were before July 9, 2012, and the FDA generally cannot share such information with an expert without the sponsor’s written consent unless the law allows it or the expert is a special government employee. The law does not create a right to a consultation, force the FDA to meet anyone, change agreed PDUFA goals, or increase review cycles. For consultations tied to an IND, NDA, or BLA, the CDER or CBER director (or division director) must find the meeting will help finish the review and fix application problems, or that the sponsor allowed it; that requirement only applies when the meeting is held only under this rule.