The regulator issued recommendations for stakeholders seeking meetings with the OOPD, in a bid to provide consistent procedures that promote well-managed meetings.
The guidance document is aimed at assisting stakeholders with requesting, preparing, scheduling, conducting and documenting meetings with the OOPD to address issues related to orphan drug designation requests, humanitarian use device (HUD) designation requests, rare pediatric disease designation requests, funding opportunities via the Orphan Products Grants Program and the Pediatric Device Consortia Grants Program, and orphan product patient-related topics of concern.
Every year, the OOPD staff takes part in meetings with stakeholders who want guidance or clarification. The meetings can be “informal” or “formal,” and help build a common understanding of the FDA’s thinking concerning orphan products, which may include drugs, biological products, devices, or medical foods for a rare disease or condition.
In its guidance, the regulator:
- clarifies what constitutes an “informal” or “formal” meeting;
- addresses program areas within the OOPD that may be affected by this draft guidance;
- discusses procedures for requesting and scheduling meetings with the OOPD;
- describes what constitutes a meeting package; and
- goes over procedures for the conduct and documentation of meetings with OOPD.
The document notes informal meetings typically take the form of a brief phone conversion, and meeting packages don’t need to be provided because the information contained in the meeting request is adequate. During informal meetings, the OOPD can address a number of general questions, including those regarding its policies and procedures, definitions of basic designation terms, and patient group initiatives relating to orphan products, among others.
Formal meetings, which usually consist of in-person meetings or teleconferences, allow the OOPD to address more specific matters, including questions about designation requests and denied requests and questions concerning orphan drug exclusivity, among others. The document says meeting packages must be provided to the OOPD prior to meetings, and notes the meetings aren’t forums for stakeholders to get feedback on product development protocols or planned studies. The meetings rather serve to provide stakeholders with clarification from the OOPD and allow them to discuss disagreements about policies, positions and statements.
Programs for which meeting with the OOPD can be requested include the following OOPD designation programs: orphan drug designation, HUD designation and rare pediatric disease designation. The FDA says many questions regarding designations can be answered during informal meetings; those related to exclusivity are usually best addressed at formal meetings. Further, questions concerning orphan drug products grants, pediatric device consortia grants and patient-related issues can also usually be tackled at information meetings.
In discussing procedures for requesting and scheduling meetings, the document refers to the OOPD’s website for information and resources that should be consulted prior to requesting a meeting. The FDA goes over several ways stakeholders can go about this, listing a number of components that should be included in requests, such as a brief statement of the meeting purpose, the type of meeting preferred, and suggested dates and times for the meeting.
The FDA notes the OOPD aims to respond to requests within five working days of receipt, upon which it will determine the appropriate meeting type and find a date.
If a meeting is scheduled, the OOPD should get its meeting package at least two weeks prior. The guidance describes what information should be contained in a package, listing elements such as basic information about the product at issue, a proposed meeting agenda and any information, data or material necessary to support the discussion.
The guidance also covers meeting protocol, noting the stakeholder will take the lead following introductions and a statement of the meeting purpose, and closes by addressing documentation, calling for stakeholders to provide a draft summary of meeting minutes to the OOPD within 15 working days after the meeting.