Since the Orphan Drug Act was passed in 1983, developing drugs to treat rare diseases has become a more common practice among pharmaceutical manufacturers.
At the core of this legislation was the Orphan Drug Designation Program, which incentivized the development of drugs to treat rare diseases by awarding tax credits, grants, market exclusivity, fast-track approval, and greater access to the Investigational New Drug Program.
While this act has been a catalyst for powerful new treatments for decades, an amendment that eliminated user fees under PDUFA caused an explosion in designation requests, prompting FDA to make some changes to how these requests are approved.
Longer review times for orphan drug designation requests
Until 2016, FDA's stated goal was to review 75% of orphan drug designation requests within 90 days of receipt. However, an FDA Voice blog post published earlier this July announced the Agency would be expanding its review timeline due to growth trends and competing for workload priorities.
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To ensure officials can review requests “with the appropriate level of care and consideration,” the new goal is to review an average of 75% of designation requests within 120 days of receipt.
Keeping orphan drug review cycles to a minimum
Manufacturers can work to avoid delays in review times by reducing the number of review cycles needed—for example, when OOPD needs additional information from the sponsor prior to determining the outcome of an orphan drug designation request.
FDA has stated that the average request for designation typically goes through two review cycles. However, sponsors can eliminate one of these by making sure their requests are complete and address all requirements in full. We recommend keeping in close contact with OOPD to ensure your review process goes as quickly as possible.
Tips for meeting with OOPD
OOPD engages with sponsors in both formal and informal meetings. Informal meetings are used to ask general questions about policies, procedures, the designation process, rare diseases and other general advice matters.
Formal meetings are used to address questions about a particular product, address a denied orphan product designation request, determine periods of market exclusivity, or any other matters which would require a more extensive discussion between sponsors and the OOPD.
When requesting a meeting, either formal or informal, your request should include the following:
- A brief overview of the purpose of the meeting, identifying the product in question and any relevant application numbers
- Suggested date and time for the meeting to take place
- Preference for a formal or informal meeting
- A preferred meeting format (in-person or teleconference)
- Contact information
When a meeting is scheduled, put together a meeting package with the following items:
- A more detailed description of the purpose of the meeting
- Basic information about the product to be discussed (product name or identifier, designation or application number, the relevant rare disease or condition, and background about the product and its intended use)
- A proposed meeting agenda, including a list of questions
- Relevant data, information, or presentation materials to support the discussion
- A list of everyone, with their titles and affiliations, who will be participating
Once your meeting is completed, stakeholders should provide OOPD with any documentation detailing the outcomes, agreements, and items for further action. Once reviewed, officials will return these items to the stakeholder with any revisions they deem necessary.
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