Orphan Insights, Part 1: Orphan Drug Designations

Orphan Insights is a series of Q&A assembled from the many discussions I have with companies developing medicines and therapies for rare diseases who are seeking regulatory advice. These questions are related to when, why and how to engage with the regulators, and take advantage of the various designations applicable to their orphan program. Each “Part” in this series will pose 3-4 questions and offer strategic and thought-provoking responses.

If you want one of your questions to be answered in this series, send me a message with your question or POST it below and I’ll answer!

Question #1 – What are the disadvantages, if any, of filing for Orphan Drug Designation?

There are really no true disadvantages in filing for orphan designation.

Considerations:

When you do receive a designation in the US or EU, this information will be made public by the Agency.

In Europe:

• The EMA will make public the active ingredient.
• Sponsors have plenty of opportunities to withdrawal an application before the opinion is made public.

This may be seen as a disadvantage to Sponsors.

Question #2 – When is the right time to apply for an ODD?

You CAN apply at the nonclinical stage, and with the right data.

Considerations:

The “right” time to apply depends on your program stage and development strategy. Often times your “exit strategy” should also be considered.

What stage is your program?

Why do you want an Orphan Designation?

Is one of your most pressing needs to identify a partner, or to fundraise? Or to obtain clinical proof-of-concept?

Is there a milestone tied to capital?

Also consider the incentives linked to this program. The first tangible incentive tied to an ODD in the US starts at human trials (i.e. the tax incentive). In Europe, the first incentive applied is the reduced/waived fees associated with Scientific Advice and Protocol Assistance.

Taking these points into consideration should help you identify the right time for your specific program.

Question #3 – Should we be applying for orphan designation in the EU at the same time as the US?

Not necessarily. While this is an efficient use of time, the timing for each should be more of a strategic consideration and should be in line with your program development goals.

Considerations:

Rare disease drug development should and often does transpire on a global level. Take into consideration where you plan to initiate your clinical developments and which agency to you wish to engage first. If the answer is both, then you may consider applying at the same time or successively.

Summary

A successful regulatory affairs program requires carefully calculated and creative solutions to global regulations. There are some regulations you cannot get around. And there are some regulations that you don’t want to miss out on. Every package is unique and for this, I encourage you to consider some of these points and questions, as the beginning of your regulatory road map.