FDA Final Rule and Draft Guidance on Orphan Drug Designation

A final rule amending the 1992 Orphan Drug Regulations became effective on August 12, 2013. The main change from the proposed rule was to add the definition of “orphan subset”.  In particular, “Orphan subset of a non-rare disease or condition (‘orphan subset’) means that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of the drug outside of that subset (in the remaining persons with the non-rare disease or condition) would be inappropriate owing to some property(ies) of the drug, for example, drug toxicity, mechanism of action, or previous clinical experience with the drug.” Additional changes include:

• Removing the language in the proposed rule that, to demonstrate clinical superiority, the drug must provide safety and effectiveness comparable to the approved drug
• Clarifying that a designation request need include only ‘relevant’ in vitro laboratory data, as well as data from ‘clinical experience’ with the drug in the rare disease or condition
• Stating that FDA’s publicly available posting of designated drugs will include whether a drug is no longer designated if the drug loses designation
• Clarifying that the scope of orphan exclusive approval is limited to the indication(s) or use(s) for which the designated drug is approved
• Clarifying that a designated drug that is otherwise the same as a previously approved drug receives 7-years market exclusivity upon approval only if the sponsor of the second-in-time drug demonstrates upon approval that its drug is clinically superior to the previously approved drug

A draft guidance published in April 2014 describes procedures for meetings with the Office of Orphan Products Development (OOPD), including timing:

• OOPD will aim to respond to a request for an informal or formal meeting within 5 working days of receipt. Before scheduling the meeting, OOPD may request a brief list of questions that the stakeholder hopes to have answered.
• If a formal meeting is scheduled, OOPD should receive a meeting package at least 2 weeks before the meeting. If OOPD fails to receive a meeting package on time, OOPD may postpone the meeting and work with the stakeholder to reschedule.
• If meeting minutes are warranted, the stakeholder should provide a draft of summary meeting minutes within 15 working days after the meeting.

For further assistance on preparation of orphan designation applications, contact RPI.