Plans unveiled to rid FDA of orphan designation backlog

The US Food and Drug Administration (FDA) has revealed its strategic plan to eliminate its orphan designation request backlog and to ensure there is continued timely response to new requests.

Currently, the agency has about 200 orphan drug designation requests that are pending review, as noted in a recent release. This number has steadily increased during the past five years and is expected to continue to expand with the increasing interest in the development of drugs and treatments for diseases affecting fewer than 200,000 people.

The Orphan Drug Modernization Plan is the first element of the FDA’s efforts to ensure its regulatory tools and policies are modern, risk based and efficient. It is aimed at facilitating the development of safe, effective and transformative medical innovations that have the potential to significantly impact disease and reduce overall healthcare costs.

As a part of the plan, the agency will deploy a Backlog SWAT team comprised of senior, experienced reviewers with expertise in orphan drug designation. This team will focus solely on the backlogged applications, starting with the oldest. Additionally, the FDA will employ a new Designation Review Template to streamline the review process moving forward.

Other plans to ensure future requests receive a response within 90 days will include a reorganisation of the review staff to maximise expertise and improve workload efficiencies, using the expertise across the FDA’s medical product centres and establishment of a new FDA Orphan Products Council that will help address scientific and regulatory issues so that the agency can approach orphan designations in a consistent and efficient manner.

“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,” said FDA commissioner Dr Scott Gottlieb. “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.”