Orphan Drug Designations Skyrocket

Orphan Drug Designations (ODD) at the FDA has soared over the past few years with yearly jumps of 30% not uncommon.  In a blog posted today on FDA’s website, Dr. Gayatri Rao, Director for the Office of Orphan Products Development (OOPD) notes that: “In 2014, we saw a 30% increase over the prior year’s record number. Yet, that record was broken the very next year when we received close to 470 requests. And the pace does not seem to be slowing. In fact, comparing the number of new requests received so far in 2016 with the corresponding date in 2015, there appears to be yet another 30% increase.”

Clearly, this points to what appears to be an increased interest in orphan drug designation and is a good sign for the patients suffering from many rare diseases or disorders that currently have no treatment.  The rise in the number of ODD requests places enormous pressure on OOPD to act on those burgeoning requests in a timely manner.  Since the Orphan Drug Program is not a fee-based program, there are limited resources available to meet the demand.  Dr. Rao noted that, while there are no formal goals for review, the OOPD had an information target goal of reviewing 75% of requests within 90 days.  However, due the significant increase in requests, OOPD has recently revised that goal to 75% review within 120 days from date of receipt.

She also noted that most reviews for ODD undergo 2 review cycles and, to improve efficiency, she implores sponsors to fully review the requirements for ODD and to include a complete submission that addresses all requirements in the first cycle.

Just as a reminder, an Orphan Drug Designation is granted when a sponsor demonstrates that a specific drug product will be used to treat a specific indication for a rare disease (less than 200,000 patients in the US).   Dr. Rao’s remarks can be found here and the requirements for ODD can be found here.

With the number of requests increasing and the funding remaining the same, hopefully review timelines will not extend further in the future.