A Meeting With The FDA On Adding CCA To The Disease List

On behalf of my Mom and myself, thank you so much for what you are doing Percy.

Until Mom’s diagnosis I had never even heard of this disease. I began to research and was so disheartened on the lack of information and resources out there

I am so glad I found this board and all of you awesome people!

Warmly
Christine

I agree with everything mentioned above. We are fortunate to have Percy appointed a FDA Patient Representative.
Here is a little background on the developments leading to his appointment.

On March 1, 2012, the Food and Drug Administration (FDA) celebrated the fifth annual Rare Disease Day by hosting a “FDA Rare Disease Patient Advocacy Day” to engage and educate the rare disease community on regulatory processes related to rare diseases. This meeting was held in conjunction with the NIH Rare Disease Day, but had been staggered allowing for attendance of both, NIH and FDA meetings.

http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/OOPDNewsArchive/ucm286797.htm

The position of FDA patient representative is a newly developed position and was officially announced at this meeting. The FDA, as many other federal institutions, recognizes the importance of patient input, in this case in regards to approval of newly developed drugs, biologics and devices.

The application process was difficult, lengthy and very time consuming for all that applied. It required completion of a rigorous interview followed by numerous, in-depth training sessions.

Yet our Percy prevailed.

Thank you, Percy. We can’t thank you enough for what you do for us and for the Cholangiocarcinoma patient community at large.
The Cholangiocarcinoma Foundation has representation in NCI disease specific clinical development of Phase II/III however; we now also enjoy the benefit of representation at the FDA level.

Below is a recap of the events leading to Percy’s appointment:
Please note that Dr. Stephen P. Spielberg, FDA Deputy Commissioner for Medical Products and Tobacco, focused on the new targeted treatment for cystic fibrosis.
Although an impressive achievement for those patients with a specific gene (one I believe to be about 80% of patient group) however; following gene discovery, the drug development leading to approval of drug required an additional 18 years. It is something I mentioned to Dr. Spielberg following his presentation. He agreed with my comments and pointed out that the Cystic Fibrosis Foundation played an important role in the process of research and development something, we (the Cholangiocarcinoma Foundation) should mirror in our efforts and involvement with our disease.

On March 1, 2012, the Food and Drug Administration (FDA) convened its first “FDA Rare Disease Patient Advocacy Day” to give rare disease patient advocates the opportunity to meet with FDA staff and learn more about how FDA works. Experts gave informative presentations that were supplemented with interactive discussions in front of 216 patients, their families, industry and advocacy group representatives, with an additional 181 participants joining via live webcast.
Highlights from the day’s agenda included a commentary from the FDA Commissioner, Dr. Margaret Hamburg who reinforced the FDA’s dedication to rare diseases. The keynote address by Dr. Stephen P. Spielberg, FDA Deputy Commissioner for Medical Products and Tobacco, addressed the impressive progress made in the rare disease arena in the last few decades, including gene sequencing and the approval of a new targeted treatment for cystic fibrosis.
Dr. Gayatri R. Rao, Acting Director of the Office of Orphan Products Development, as well as each Center within the FDA, representing drugs, biologics and devices, presented their perspectives on products for rare diseases. This prompted participants to ask questions on what they could be doing to help researchers bring their discoveries to clinical trials and to prepare the rare disease community for participation. The ensuing discussions touched upon patient registries, natural history studies, and adverse event rates.
Hugs,
Marion

Percy;

Thank you for all you do and I hope God continues to bless you.

Oh, Percy, your introduction was fabulous! And you are awesome! Thank you so very much! I would venture a guess that most of these meetings you go to, you are the first CC survivor that many of them have seen! You are one of a kind, we love you!

Bless you, Percy for being our voice.