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