NORD Summit, a Rare Disease Regulatory Wonderland

Rare disease is one of our main areas of focus here at Premier, so you’ll often see my colleagues and me talking about rare, through our webinars and white papers, and basically, almost any phone conversation with us. Last week, I attended the NORD Summit in Arlington, VA, and as usual, I found myself inspired by the presenters and delegates at the meeting. I thought I’d share if you weren’t able to make it.

Day One
A high point was the keynote address by Dr. Robert Califf, the recently installed FDA Commissioner. Dr. Califf spoke on a number of topics, some of which were more generic in nature and some of which were of special interest to rare disease researchers and patient advocates.

He spoke in detail about the accelerated approval pathway and the necessity for researchers to follow-up on their post-marketing commitments after obtaining marketing approval under the accelerated approval process; he noted that compliance with these requirements is higher than he anticipated but that the FDA wants to see that number closer to 100 percent.

Dr. Califf also commented that not all academic research has to be considered “exploratory” and that sometimes a more rigorously designed academic study can still advance translation.  Additional comments touched on the adoption of the final rule regarding regarding publication of trial results.  Dr. Califf concluded his comments by discussing his view that Patient Advocacy Organizations should work together to develop and share best practices to advance rare disease research.

I elected to attend the track entitled, “Focus on Pediatric Diseases – Advancing Research and Treatments.” This session included speakers from FDA, NIH, NCATS, the National Cancer MoonShot Initiative, and a parent of a child with a rare disease that now works with the patient advocacy organization devoted to his son’s condition. The session included a review of pediatric drug development principles by the acting director of the Division of Pediatric and Maternal Health (CDER) as well as a review of the Rare Pediatric Disease Priority Voucher Review program which was recently extended until December 31, 2016, by President Obama.

Just as we have often heard from our own Vice President of Regulatory Affairs, Dr. Lisa Pitt, the FDA representatives reiterated that the agency is there to support ethical drug development and that they welcome interactions with researchers as development plans are being proposed and revised. It was an interesting and interactive session followed by an informative question and answer period.

I would be remiss, however, if I didn’t mention that the highlight of that first day was meeting my recent webinar co-presenter, Susan Stein, CEO of Connexion Healthcare and Global Genes Board Member!  Thanks for repping Philadelphia and bringing Tastykakes to the meeting, Susan!

Day Two
The next day at NORD was equally eventful.  One of the really attractive components of the NORD Summit is the significant presence of representatives from the FDA at the meeting.  In addition to the Califf keynote address, a number of other FDA personnel attended.

I really enjoyed the morning breakout session called “Trending Topics from FDA,” where all of the presenters were agency personnel.  This session included a very informative discussion about biosimilars as well as some case studies of how the FDA applies flexibility within their governing statutes to advance rare disease therapeutic development.  Of significant interest to the audience was the presentation on Patient-Focused Drug Development (PFDD) and how advocacy organizations can take the lead on creating and holding PFDD meetings with FDA.  Later in the afternoon, Drs. Woodcock and Marks, directors of CDER and CBER respectively, talked about the FDA’s commitment to rare disease research.

A few themes presented themselves throughout the day and throughout the meeting, the first being that the FDA welcomes early interactions with sponsor companies especially with regard to trial design discussions.  That’s exactly what our own Dr. Pitt, who recently worked at FDA, always says when talking with clients – reach out to the agency, they want to engage with clients to ensure everyone is on the same page.

Another theme was FDA’s desire to see patient advocacy groups collaborate amongst themselves, especially when considering pulling together an external PFDD meeting. Also highlighted in terms of collaboration was the joint FDA-EMA Rare Disease Cluster activities that were introduced earlier this year.

Of course, the NORD Summit attracts a number of patient advocates and having the opportunity to meet with some of them is always very rewarding. Their passion is inspiring, their stories always moving.

Looking forward to next year!