FDA’s Office of Orphan Products Development Is Dedicated to Rare Diseases Every Day

Debra Lewis, O.D., M.B.A.
Acting Director, Office of Orphan Products Development

Debra Lewis, the deputy director and current acting director of FDA’s Office of Orphan Products Development (OOPD), knew when she came to FDA that she wanted to tackle public health needs.  She started as a clinical reviewer, and after gaining new skills and insights at FDA, she decided she wanted to be a part of the OOPD's team committed to advancing products that treat the one in ten Americans living with a rare disease. A Doctor of Optometry, and a former officer in the Public Health Service and Air Force, Lewis had seen her share of global medical challenges, and she knew she wanted to engage in the special challenges posed by rare diseases. Later, her mission became even more personal, after learning that a member of her family had a visual rare disease without a cure.

FDA’s OOPD plays an integral role in in fostering the development of drugs, biologics, medical devices and medical foods that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. It is OOPD that evaluates scientific and clinical data submissions from sponsors to identify and designate orphan status to drugs and biologics and those medical devices that qualify for a special path to market for Humanitarian Use Devices (HUDs).

Over the past five years, the number of orphan drug designation requests at FDA have increased, with 527 in 2017, nearly doubling since 2012. Given this record volume, it is not surprising that 2017 saw 77 drug and biologic indications approved for rare diseases, a record number. 2017 was also noteworthy for the HUD program – with FDA receiving its 400th HUD request. The agency has now approved over 70 HUDs through the Humanitarian Device Exemption marketing pathway.

The OOPD also administers extramural grants programs to encourage the develop of promising treatments for rare diseases. Last year, for instance, the FDA Orphan Products Grants Program issued $17 million in grants to fund 15 new and 65 on-going clinical trials studying products to treat rare diseases.  Since the inception of the program, these grants have helped bring to market over 60 approved products for treatment of rare diseases/conditions.  In addition, OOPDA administers the Pediatric Device Consortia Grants Program to advance pediatric device development and has recently launched a grants program for targeted natural history studies for rare diseases which collect data on how specific rare diseases progress in individuals over time so that knowledge can inform and support product development. In 2017 FDA funded 6 natural history grants for rare diseases, including two in partnership with the National Institutes of Health, and funds the Pediatric Device Consortia Grants Program to advance pediatric device development.

Lewis now finds herself on the cusp of an exciting effort to modernize FDA’s program for designating which drugs qualify for orphan status. This includes eliminating the agency’s backlog of requests for orphan drug designation and ensuring continued timely response to all new requests for designation.  

In addition, her office is initiating an Orphan Drug Designation Modernization pilot which includes a new orphan drug designation application form for sponsors and a new webinar tutorial on preparing orphan drug requests. Both are designed to improve and clarify the designation request process and assist and support drug sponsors, as well as to make the submission much easier to review, potentially speeding the development process for patients with rare diseases and conditions.

Since the effort was begun last spring, the orphan drug designation team already has eliminated the backlog of requests older than 120 days, and is putting in place additional policies to further improve the efficiency of that review process to allow FDA to meet a new 90-day mandate for review and to address new scientific challenges for rare disease therapies.