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

Hi, everyone,

I attended the "public meeting on the patient-focused drug development " at FDA in Silver Springs in MD. The FDA officials included John Jenkins, MD ,director ,office of new drugs; Theresa Mullin, PhD, director ,Office of planning and informatics; Donna Griebel MD, director of Gastroenterology ; Patricia Keegan MD ,director ,division of oncology products II among others;  FDA want patients to have more involvement in helping FDA in
1. - Assessment of a drug's benefits and risks involves analysis of the severity of the condition and current state of treatment armamentarium.(This will help experts to evaluate the efficacy and safety data and the expected impact of efforts to reduce and further characterize risks of the drug.)
2.- Patients who live with a disease have a direct stake in drug review process and are in a unique position to provide input on analysis of the disease condition and current treatment options.(This will provide regulators with the clinical context for weighing benefits and risks of the new drugs).
3.-Review process would benefit from systematic approach to obtaining patient perspective on disease severity or unmet medical need.

This "patient-focused  drug development" by FDA is funded under the Prescription Drug User Fee ACT (PDUFA V) just passed by the congress, and FDA will hold 4 public workshops each year over the next 5 years. Each meeting will focus on a different disease area, reviewing the armamentarium(the resources available) for that indication, and identifying areas of unmet need.

The goal of today's meeting is to get public input on the  FDA's published preliminary list or nominate disease areas for the "patient-focused drug development" initiative and criteria used for nomination.
Of course , cholangiocarcinoma is not on the preliminary disease area list that FDA provided ; and there are a lot of  patient advocate organizations and individuals do not  see their diseases(most are related to rare diseases)  either on the list. Therefore about 50 individual and organizations signed up to speak out  to the FDA officials and requested their disease to add to the list for consideration. Each of us were allowed 2 minutes to present our case.

Below is the statement that I spoke to the FDA panel officials for CCA.

" I am representing The Cholangiocarcinoma.org to request  cholangiocarcinoma to be added to the list of diseases that  are under consideration. There are about 8,000 new cases/yr in the States and 3,000 new cases/yr in UK and there are a lot more new cases/yr around the world especially in the Far East.   You all are looking at a lucky patient of this disease who have survived for almost 4 years; but for most of our patient population it may not due to  the late discovery of their advance disease state. Survival time is between a few weeks to a couple years. Currently there are no FDA approved effective chemotherapy and targeted therapy protocol for treatment of the advance stages of cholangiocarcinoma patients; Most of the treatment plans are borrowed from other diseases like pancreatic and colon cancer. There are no accurate and useful biomarkers for both diagnosis and prognosis purposes. And most of all, this cholangiocarcinoma disease put tremendous psychological and physical pressure  on the caregivers due to the uncertainty of the treatment outcome and the severity of the side effects. So please consider to add  cholangiocarcinoma to your preliminary disease area list. thank you. "

I will continue to update on the development on this area of interest and will let all of you know .
God bless.

Please know that my personal opinion is not intended nor implied to be a substitute for professional medical advice. If  provided, information are for educational purposes.Consult doctor is a MUST for changing of treatment plans.

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

Thanks for all the hard work in putting CC out there!!
Lots of prayers-Cathy

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

Bless you, Percy for being our voice.

My beautiful daughter, Lauren Patrice, will live on in my heart forever.

My comments, suggestions, and opinions are based on my experience as a caretaker for my daughter, Lauren and from reading anything I can get my hands on about Cholangiocarcinoma. Please consult a physician for professional guidance.

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

Thanks Percy!!

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

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!

Teddy ~In our hearts forever~ATTITUDE is EVERYTHING
Any suggestion I offer is intended as friendly advice based solely on my own experience. Please consult your doctor for professional guidance.

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

Thank you Percy!!!!! Thank you so very very much indeed for this! You rock big time!!!! After reading your statement to the FDA panel they will have to listen to what you said to them.

Go Percy!!!

Any advice or comments I give are based on personal experiences and knowledge and are my opinions only, they are not to be substituted for professional medical advice. Please seek professional advice from a qualified doctor or medical professional.

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

Percy;

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

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

Thank you Percy. In a very concise statement, I think you've summarized the unique challenges of treating this disease. I know we all appreciate your efforts. Let me also take this opportunity to express my own appreciation for the breadth of knowledge you share on this board. When fighting this disease, information is power, and you've empowered a lot of people!

Best, Mark

9 (edited by marions Sat, 27 Oct 2012 06:40:04)

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

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/Developi … 286797.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

THIS INFORMATION IS NOT INTENDED NOR IMPLIED TO BE A SUBSTITUTE FOR PROFESSIONAL MEDICAL ADVICE. YOU SHOULD ALWAYS SEEK THE ADVICE OF YOUR PHYSICIAN OR OTHER QUALIFIED HEALTH CARE PROVIDER

10 (edited by PCL1029 Sat, 27 Oct 2012 08:04:29)

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

Hi,everyone,
Thanks for allof your compliment. As I mentioned before, we all become ONE ENTITY, one voice and united in one spirit for finding a cure for all of us,patients as well as caregivers, so we will never be suffer again. That is the only purpose for me to join this board voluntarily without reservation.

The road ahead is rough and long ; it requires the collaboration of the researchers of the academia,the pharmaceutical companies,the regulatory agents , investors and patient advocates to work together ,using the most uptodate methods and equipment to increase the effectiveness of finding new drugs,improving  the efficacy and decreasing the adverse drug reactions of the new drugs ; and therefore shorten the time to get new drugs on the market . As you know ,COLLABORATION is not easy. We may not get there in time to find the cure for all of us who now having this disease and relieve the emotional sufferings for both patients and  caregivers. But it is a start anyway toward the right direction. That means we,the patients and caregivers, have to work harder, ,raising more funds,doing more research,organize better,and increase the awareness of this disease to anyone you know . Compare to the cystic fibrosis foundation's research and organizational development, we still have a lot of catch up to do.
But for my part,I will try my best as long as my energy level up to the challenge.
God bless.

Please know that my personal opinion is not intended nor implied to be a substitute for professional medical advice. If  provided, information are for educational purposes.Consult doctor is a MUST for changing of treatment plans.

Re: 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

Caring for my Mom and helping her win this battle.

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

Percy,
You are the best !!!!!!
Thank you for your hard work
Lisa

This Information Is Not Intended Nor Implied To Be A Substitute For Professional Medical Advice. You Should Always Seek The Advice Of Your Physician Or Other Qualified Health Care Provider