What NOT to Do in an FDA Meeting

Many Sponsors come out of a positive-response FDA meeting thinking they have gotten everything they wanted. Unfortunately, the FDA is not obligated to tell you what you missed. Did you get permission to conduct a trial, or did you get confirmation your drug discovery program is on track to receive marketing approval? I’ve seen topContinue reading “What NOT to Do in an FDA Meeting”

Pediatric Rare Disease Vouchers Sell for an Average of $100M. Can You Qualify?

The Pediatric Rare Disease Voucher Program was just reinstated by an act of Congress. This gives us until September 2024 to gain Pediatric Rare Disease Designation (PRD). While this program is not as important to large drug development companies, who can simply purchase a voucher if they think it will improve their balance sheet, it is mission critical for smaller companies in the rare disease space.

Getting the ODD: The Art of Orphan Subsetting

Why is the term ‘orphan subsetting‘ important for you to understand, and how does it affect your Orphan Drug Designation (ODD) application? This important consideration can make the difference in whether your ODD is approved. Let’s break it down.

Fundamental Concept: Know Your Audience!

The FDA Office of Orphan Products Development (OOPD), the EMA Committee for Orphan Medicinal Products (COMP), and your FDA review division are unique entities with individual characteristics. Care should be taken to ensure any submissions are written with your particular audience in mind.

The Pediatric Rare Disease Voucher Program Is Set To Sunset

While I do not have a crystal ball, I can tell you this: the program has been renewed before and is one of the most popular mechanisms by which small pharma/biotech (like most of my clients) can enter the drug development space. In my experience, getting Pediatric Rare Disease designation early in development makes it much more possible to fund clinical trials.