Rare Disease Report

Removing the Orphan Drug Backlog a Top Priority of the FDA

JUNE 29, 2017
James Radke
In 2016, the U.S. Food and Drug Administration (FDA) received 568 new requests for Orphan Drug Designation and there are still currently an estimated 200 requests under review for the special designation.
 
The numbers reflect the pharmaceutical industry’s interests in developing orphan drugs for rare disease, and as a champion for the rare disease community, new FDA Commissioner Scott Gottlieb, MD, is making efforts to ensure that those drugs are reviewed in a more efficient and timely manner.


 
Today, Gottlieb announced a 2-part plan that will remove the backlog of requests waiting for review and expedite responses moving forward.
 
An orphan drug designation means that the FDA has determined that the drug in development is for a rare disease and the designation provides various development incentives, including tax credits for clinical trial costs, relief from prescription drug user fee, and eligibility for 7 years of marketing exclusivity upon approval. 
 
First, in the coming 90 days, the FDA will complete the review of all Orphan Drug Designation requests that are currently backlogged (requests older than 120 days).
 
Next, the FDA plans to have a strategy in place to ensure that all new Orphan Drug Designation requests will have received a response from the agency within 90 days of receipt.
 
In a statement, Gottlieb said, “People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations.” Dr. Gottlieb, added, “Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”
 
1. To achieve the first goal of addressing the backlog of orphan drug designation requests in the next 90 days, the agency has put in place the following procedures:
 
  • Create a Backlog SWAT Team of senior, experienced, proficient Office of Orphan Products Development (OOPD) reviewers to focus solely on reviewing orphan drug designation requests, starting with the oldest ones first
  • Create and implement a new streamlined Designation Review Template to increase consistency, efficiency, and predictability of orphan designation reviews
  • Minimize discretionary work – i.e., FDA will reduce non-designation and non-grant-specific duties and assignments – for all other reviewers to enable the review teams to focus on core activities
  • OOPD will collaborate with FDA’s Medical Product Centers to complete a CDER-CBER Orphan Designation Pilot Project – CDER and CBER reviewers will conduct preliminary primary reviews of a subset of drug designation requests, with OOPD conducting secondary reviews
  • OOPD will collaborate with the Office of Pediatric Therapeutics (OPT) to jointly review rare pediatric disease (RPD) designation requests. In these cases, OPT will conduct the pediatric review and OOPD will conduct the rare disease review. This policy began as of May 15, 2017.
  • OOPD transitioned secondary review of FOIA requests to the FOIA office, as of May 18, 2017
  • Continue to track weekly progress, adjust as necessary, and report on progress to the public
 
2. To achieve the second goal of making sure all future orphan drug designations requests will receive a response within 90 days, the agency will take the following steps:
 
  • FDA will establish an “FDA Orphan Products Council” to address scientific and regulatory issues related to orphan products to ensure a consistent approach to regulating these products
  • FDA will work to establish and implement a future state including the below changes. They will report on a full timeline of the progress on these activities within the next two months.
  • Organizational re-structuring to maximize expertise and improve workload efficiencies
  • Leverage the inter-center consult process, involving the medical product centers, that was developed for combination products to develop a streamlined process for consistent and timely orphan consults
  • Designation and Exclusivity Programs (Orphan Drug, RPD, Humanitarian Use Device (HUD))
    • Centralize orphan exclusivity review and determinations
    • Continue to enhance the information technology infrastructure, e.g., automating more of the administrative processes for designation reviews
    • Improve and implement streamlined “Designation Review Template” across all designation programs to bring more efficiency, consistency, and predictability to these activities
    • Complete development of web-based training for sponsors to enhance quality of submissions
  • Grant Programs: With respect to the Orphan Products (OPD) Grant Program (Clinical Trials + Natural History) and Pediatric Device Consortia (PDC) Grant Program; FDA will:
    • Revise grant monitoring processes by increasing utilization of desk-top and virtual tools and by implementing a new risk-based approach for conducting in-person site visits to grant recipients
    • Modify and modernize reporting requirements so that FDA can continue to give a high assurance related to appropriate monitoring of federal funds and efficiently measures program success
    • Continue to enhance IT infrastructure for continued efficiency and better monitoring
  • Reduce OOPD office-wide workload
    • Modify Orphan Cluster meetings with EMA from monthly to quarterly       
      • The impact of the reduction in frequency of meetings with EMA is mitigated by our well established and long-standing relationship with our EMA counterparts, which will allow us to have ad hoc meetings should they become necessary in the intervening months.
    • Modify FDA Rare Disease Council meetings from monthly to quarterly
      • The RDC was established in 2012 to serve as a forum to communicate and collaborate across the agency on rare disease issues. It is chaired by OOPD and includes representatives CDER, CBER, CDRH, OHCA, OL, and OOPD. Quarterly meetings would ensure continuity of cross-agency communication but would help reduce workload in administering monthly meetings. The implementation of more joint reviews and closer regular, ongoing collaboration should reduce the need for the larger, RDC meetings.
    • Minimize outreach activities and discretionary projects to only those deemed most meaningful
  • OOPD will create a new “Tracking Dashboard” to monitor and facilitate its efforts to meet the new designation goals and FDA will report on overall workload and progress more regularly.
 
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