FDA Adjusts Timeline for Reviewing Orphan Drug Applications

FDA Revises Review Timeline Due to Increasing Number of Orphan Drug Applications

FDA Adjusts Review Timeline For Orphan Drug Designation (ODD) Requests

In 1983 Congress passed the Orphan Drug Act, which created FDA’s Orphan Drug Designation (ODD) program.  The goal of this program was to encourage pharmaceutical companies to develop drugs and biologics to treat rare diseases; it does this by providing financial incentives such as major tax credits, no user fee, and seven years of market exclusivity for products approved with an orphan designation.

Since the program was enacted more than 33 years ago, the number of ODD requests that FDA receives each year has drastically increased.  In 2014, FDA saw a 30% increase in the number of requests that were received as compared to 2013, which was also a record breaking year for the Agency.  Since then, the numbers have only continued to rise, reaching 470 requests in 2015, a number that is expected to increase by another 30% in 2016 (based on the number of requests received so far).  Due to this upward trend, on July 18th, Gayatri Rao, MD, JD, FDA’s Director of the Office of Orphan Products Development (OOPD) announced that the Agency is adjusting its timeframes for reviewing ODD requests.

Although there is no regulatory review deadline for these products, the FDA currently aims to review 75% of all orphan designation requests within 90 days of receipt.  In her recent blog post, Dr. Rao states that the Agency strives “to review these requests in an efficient and timely manner because we understand how critical designation can be for companies to move forward with their drug development plans.”  Dr. Rao notes that reviewing ODD applications in both an efficient and timely manner is still a top priority within the OOPD, “but to ensure we continue to conduct these reviews with the appropriate level of care and consideration, our current goal is to review on average 75% of designation requests within 120 days of receipt.”

Many of the requests that are submitted to the FDA today go through two review cycles with the OOPD.  To compensate for the increased review time, FDA recommends that sponsors take measures to ensure that their request is complete and that it fully addresses all requirements before being submitted for review.  Furthermore, Dr. Rao suggests that sponsors review the information provided on the FDA’s website for helpful information and FAQs regarding the development of ODD requests.

Are you in the process of developing an orphan drug designation or other type of application for submission to FDA?  We can help you achieve a successful interaction with the FDA.  To learn more about our services and how we can help you, contact us today.