News & Posts

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 In July 2019 the FDA released revised draft guidance describing their thinking on the Rare Pediatric Disease Priority Review Voucher program, originally set forth in Section 529 of the FD&C Act and revised by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

Believe it or not, it’s that time of year to start thinking about making the most out of your JP Morgan 2018 experience. Given what we know and have come to anticipate, each year investors and partners expect to see various (and promising) regulatory milestones achieved, and in the past three years all eyes have been on ORPHANS.

While the both the EMA and FDA (Office of Orphan Products) has seen a substantial and consistent rise in orphan designation requests, and consequently an increase in the orphan designations for which they approve, the figures are not equivalent. In the United States for instance, the OOPD designated 354 of the 466 applications received in 2015. In 2016, the OOPD received 110 more applications, setting a new record from the previous year, yet designated fewer (333). These numbers show designated applications dropping a whopping 20%.

RareMoon is in full preparation mode and enthusiastic about planning for a successful three days in San Francisco for the industry's biggest events of the year. Why do we attend? Considering what we've seen in the past few years, we are anticipating a lot of buzz around rare diseases, orphan drugs and novel therapies at the JP Morgan Healthcare Conference this upcoming January. For RareMoon, we consider JPM a time to tap into investors, bankers, and biotech companies investing both research efforts and time into the rare disease arena.

The President signing off on “Advancing Hope Act of 2016,” which modifies the FDA’s priority review voucher program for rare pediatric diseases means an extension of the program through the end of the year, for now. Even more importantly, this means that between now and December 31st, Congress will examine and deliberate ways to amend the program and extend it past 2016. The sign off also clarifies one of the more controversial aspects of the bill - the definition of “rare pediatric diseases."