A new draft guidance document published by the US Food and Drug Administration (FDA) aims to clarify the processes and procedures of meetings with its Office of Orphan Products Development (OOPD).
OOPD's mission is to help facilitate the development of drugs, devices, biologics and other medical products to treat patients with rare diseases. Those diseases, as defined by the Orphan Drug Act of 1983, affect fewer than 200,000 patients in the US in any given year. As a result of their small market size, rare diseases have historically been neglected by the life sciences industry-hence, the "orphan products" name.
Starting in 1983, FDA began to offer help and other incentives to sponsors of orphan drug products, including tax incentives and special meetings with regulators meant to reduce bottlenecks, provide feedback and accelerate approvals.
FDA's latest draft guidance document, Meetings with the Office of Orphan Products Development, focuses on these meetings.
Uncommonly, the guidance isn't solely targeted at the usual audience (industry), but is rather addressed to a broad spectrum of stakeholders, including industry, researchers, patient groups and "other stakeholders."
As FDA explains in the document, many of these stakeholders wish to meet with OOPD, either to request orphan drug status, humanitarian use device (HUD) status, obtain funding or work through patient-related issues.
"These meetings vary from general information-gathering meetings, where stakeholders ask basic questions about the designation or grants process, to more specific and complicated discussions involving, for example, questions related to orphan drug exclusivity," FDA wrote. These meetings, the agency adds, can be "critical" to the development process, and may have an impact on the ability of a product to make it to market.
"It is therefore important that these meetings be scheduled within a reasonable time, conducted effectively, and documented where appropriate," it said.
Meeting with OOPD
The guidance therefore delves into what stakeholders can expect when they meet with OOPD, including the various types of meetings, OOPD's scope of authority, how to request a meeting with OOPD, content that must be submitted to OOPD in advance of a meeting, how meetings will be conducted, and how meetings will be documented for future reference.
OOPD conducts two types of meetings: formal and informal. The latter is used to ask general questions about policies, procedures, the orphan drug status designation process, rare diseases and to ask for general advice. In contrast, formal meetings are used to address product-specific questions, address denied orphan product designation requests, determine periods of orphan drug exclusivity, or any other matters which would require "extensive discussion" and preparation between a sponsor and OOPD.
OOPD's other general advice to all stakeholders: Read its website (www.fda.gov/orphan), which it says contains the answers to many questions they might have. If, after consulting the website, there are still questions, then they should consider sending a meeting request to the office.
Those meeting requests should include the following:
- a brief statement of the meeting purpose, including identification of the product to be discussed and any applicable designation request or grant application number
- whether the stakeholder prefers an informal or formal meeting
- suggested dates and times for the meeting
- preferred format of the meeting (i.e., teleconference or in-person meeting)
- the e-mail address and telephone number at which the stakeholder may be reached
Once a meeting is scheduled, a meeting package should be put together by the stakeholder, to include (at a minimum):
- the date, time, and subject of the meeting
- a detailed explanation of the meeting purpose
- basic information about the product to be discussed (product name or identifier, designation or application number, the relevant rare disease or condition, brief background about the product and its intended use
- a proposed meeting agenda, including a detailed list of questions that the stakeholder hopes to have answered
- any data, information, or presentation materials to support the discussion, if needed
- a list of all individuals, with their titles and affiliations, who are expected to participate in the meeting
After the meeting, the stakeholder should provide OOPD with documentation of the meeting, including any outcomes, agreements, disagreements and action items. OOPD will then either revise or accept the meeting minutes and return them to the stakeholder.
Meetings with the Office of Orphan Products Development