Navigate regulatory uncertainty with strategy, early engagement, and thoughtful communication to build trust, reduce risk, and drive rare disease programs forward.

RareMoon exists to navigate rare disease drug development, overcoming regulatory hurdles to bring life-changing therapies to patients. #RareDiseaseDay

RareMoon expert Mette Due Theilade Thomsen to lead DIA Global’s virtual Paediatric Investigation Plans (PIP) training in November, offering key regulatory insights and practical skills.

The FDA’s RPD PRV Program has led to 569 RPD designations in 10 years, boosting treatments for rare diseases like Duchenne muscular dystrophy, neuroblastoma, and sickle cell.

Time flies. In July 2019 the FDA released revised draft guidance describing their thinking on the Rare Pediatric Disease Priority Review Voucher program, originally set forth in Section 529 of the FD&C Act and revised by the Advancing Hope Act of 2016.

It’s officially day one of #BIO2018 for Boston, the industry, and RareMoon. Co-founders, Chris and Sabrina, will be taking on #BIO2018 together this year to divide, conquer, and make the best of the week. This year has already been a fantastic year for RareMoon and our growth both as a company and in the intimate support we’ve provided to global biotech & pharma companies developing orphan medicines.

A Breakthrough Therapy (BTD) is an FDA designation that was introduced in 2012 as a means to permit the FDA to grant priority review to a drug candidate under development for a serious or life-threatening disease, when clinical evidence indicates the therapy offers substantial treatment advantages over existing therapies for patients.

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. Question #2 – When is the right time to submit a PIP? This depends on a lot of variables. In general, it can be submitted prior to initiating trials and up until initial proof-of concept trials, definitely before pivotal or confirmatory trials.

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.