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

Summary

Require the Secretary to set up ways for the FDA to get outside help and to hold talks with stakeholders about new drugs and biologics for rare diseases and genetically targeted treatments. The Secretary must keep a list of external experts who have scientific or medical training the agency lacks and may call them in when that expertise is needed. The Secretary must also give chances for stakeholder consultation consistent with certain PDUFA letters for Fiscal Years 2013–2017, at times the Secretary thinks are right. Consultations may cover things like what a rare disease is and how severe it is, unmet medical needs, whether patients can or will join trials, benefit‑risk tradeoffs, how to design trials for small or special groups, patient demographics and clinical descriptions, and methods for small population studies. Outside experts may be treated as special government employees under title 18, section 202. Confidential commercial or trade secret information cannot be shared with an expert without the sponsor’s written consent unless the expert is a special government employee or the law allows it. These rules do not create a legal right to a meeting, change agreed goals or review cycles, limit other consultations used before July 9, 2012, or delay reviews. For INDs, NDAs, or BLAs, a Center Director must decide the consultation will help finish the review and fix outstanding problems or that the sponsor agreed to it; that decision rule applies only when the consultation is done only under this authority.