Regulatory Focus™ > News Articles > Unique Public Meeting Set to Give One-on-One Advice to Orphan Drug, Device Developers

Unique Public Meeting Set to Give One-on-One Advice to Orphan Drug, Device Developers

Posted 20 August 2013 | By Alexander Gaffney, RAC

Usually when the US Food and Drug Administration (FDA) meets with the public, and in particular members of industry, the meetings taking something of a group-centric approach. With lots of people likely to share similar views or benefit from hearing similar questions, the agency invites the public to listen to short presentations from public speakers before opening the floor to those who have additional, unanswered questions.

But in a new and somewhat unusual announcement, FDA says it plans to take a slightly different approach for an upcoming meeting-an approach that eschews the group approach in favor of one far more personal.


Under the Orphan Drug Act of 1984, sponsors were given a variety of incentives to bring drug products intended for smaller populations to market. These populations were sometimes referred to as "orphan" populations, as the population size was too small to create a viable market for companies to recoup their investment. To reverse this trend, the ODA granted manufacturers enhanced market exclusivity, tax incentives and expedited review processes, all intended to make the process less risky and (presumably) more profitable.

While the law's effects were slow to catch on, recent years have seen a veritable explosion in orphan drug products brought to market as companies realize that small populations can be just as profitable for their bottom line as traditional populations, if not more so.

That doesn't mean that traditional drug development problems aren't still a challenge for companies to surmount. Because the populations-and often the companies working on orphan drugs-remain small, companies must be able to support product applications with different types and scales of data. One obvious example: It's hard to run a 3,000-person clinical trial if the entire US population for your disease is just 1,200 people.

And unlike diseases with larger populations, there often aren't guidance documents specific to the particular development problems associated with a given product, making the clinical testing process inherently riskier.

A Unique Meeting

It's little wonder then that FDA tries to meet early and often with products granted Orphan Drug Designation status by its review teams, affording those products and their sponsors more help in designing and validating their clinical programs.

Now, it seems, FDA is extending that same one-on-one logic to an upcoming meeting on Orphan Product Designations and grants.

The one-day workshop on 4 October 2013 is set to bring together regulators from FDA and the European Medicines Agency (EMA), both of which will be presenting at the meeting. The first half of the meeting is fairly routine, with regulators providing an overview of the Orphan Drug Designation program for drug products and the Humanitarian Use Device Designation Program/Pediatric Device Consortia Grant program for medical devices.

The second half of the meeting, however, is where things take a turn for the unique. "The afternoon session (no Webcast), provides an opportunity for appropriately registered participants to have one-on-one meetings with FDA staff members onsite, to discuss the specifics on how to apply for an orphan product grant, a HUD designation, or orphan drug designation," FDA explains in a Federal Register notice. EMA staff will also be available for the same purpose.

Candidates interested in the opportunity-and FDA notes that just 50 teams will be given the opportunity to meet with regulators-are expected to bring information on "at least one candidate orphan drug or device that holds promise for the treatment of a rare disease or condition in order to discuss the process for putting together an application." Working draft submissions are also highly encouraged to be brought to the meeting "in order to maximize the utility of the one-on-one meetings," FDA explained.

No word yet on whether meeting participants will be given private meeting spaces, which some might find beneficial given the sensitivity of their intellectual property.

Federal Register Notice

Regulatory Focus newsletters

All the biggest regulatory news and happenings.