1 (edited by marions Sat, 15 Dec 2012 23:00:09)

Topic: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

On behalf of the Cholangiocarcinoma Foundation I participated in the Faster Cures – New York Partnering for Cures – Conference, Nov. 28 – 30.

This investor-style conference sets a platform for nontraditional allies to share ideas and to collaborate with leaders from every sector to help transform promising scientific discovery into treatments and cures. It provides the venue for focus on strategic planning, collaboration of structuring, attracting capital, interacting with the FDA and communications and marketing. 

It has been noted that within the next two years the US is loosing its grip on research and development for new drugs.   Venture capitalists have decreased investments within the last three years and they will continue to shift their interest to Europe and Asia.  The main factors are related to the lengthy regulatory approval (FDA) and the lesser financial return.
To better understand: the cost of single clinical trial can add up to $100 million at the high end, and the combined cost of manufacturing and clinical testing for some drugs can reach up to $1 billion, and when calculating in the risk of failure (fewer than 1 in 10 medicines tested in human clinical trials succeed) the rationale of the investors makes sense.
The time from drug development to drug approval and for that drug to ultimately reach the patient clearly necessitates acceleration of this process.
Additionally, collaboration and transparency amongst researchers, agencies and academia has to be improved upon. For example: up to 40% of clinical trials conducted by the NIH are not published.  This is similar to what is happening in cancer centers where clinicians’ trials may have had a positive response and yet we never hear of it. 

For our patient community in particular, emphasis has to be placed on acceleration of the process of discovery and development of new medical solutions. 

The biggest danger, said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, is “where public criticism and controversy outweigh science.”
The agency represents the Gold Standard for drug approval globally, is trying to become more innovative and approve new treatments more quickly and efficiently, but there are obstacles. 
When AIDS activists basically started the fast track of orphan drugs they were willing to take any risk because there was nothing that could help them.  They had no other options and in order to live they choose to risk the possible side effects or non-response to drugs.

Today’s drugs are safer than those distributed in the 80th and much of this is contributed to the complex benefit and risk information analyses adapted by the FDA.
 
The FDA wants to hear from patient community what type of risk they are willing to take on and jointly develop a methodology.  They have chosen 20 diseases which will set the standard for the entire methodology.  Other diseases are planned to be added in 2017.

The average length of approval has decreased substantially since the advent of PDUFA in 1992. The median approval for applications received in FY 1993 was 19.0 months, compared to 9.9 months for applications received in FY 2011.

Rare disease advocates joined Jimmy Lin, MD, PhD, in a round-table discussion.  Dr. Lin is the lead computational biologist for the ground-breaking cancer genome sequencing efforts, Vogelstein Lab, John Hopkins.  Their sequencing of the first 100+ cancer exomes in 5 different tissue types has helped lay the foundation for a revolution in cancer genomics.
Jimmy and his group at The Rare Genomics Institute http://www.raregenomics.org/
believe that most rare diseases are genetic in nature and genome sequencing has the power to help these patients in a way that conventional diagnostics cannot. 

Representing the largest and most effective rare disease foundation of this group, I shared my understanding of the process of developing and building a patient community port via a website; the challenges encountered and experiences gained. We already have the necessary infrastructure and communications venues in place in order to participate in the current shift from proprietorship to translucency.

As the “veil of secrecy” is lifting and the NCI and FDA and other stakeholders are actively incorporating advocacy into the efforts of fighting diseases, we see foundations partnering with biotechnology and pharmaceutical companies. 

It also is comforting to know that “finally” recognition is given to disease specific foundations such as ours, that we can have an impact by using sincere advocacy for the patient community, networking and guiding patients through the process so that they are not lost in information overload, and ultimately infuse money accordingly. 

Toward the end of the conference I agreed to a taped interview with Timeequalslive.org.  Here I made an appeal to investors and emphasized that in comparison to other, larger cancers our patient base is small, but that our collective, global patient community combined would provide adequate return on their money. 

Personally, I continue to struggle with the crucial question: should financial return involve the basic right of a human – that is to receive treatments for his/her diseases?

Ultimately though, I hope for our patients to benefit from the rapidly changing research and development environment and that the  accelerated drug development will continue to speed up the process of bringing life saving drugs to our patient community. 

Hugs to all,
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

Re: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

WHEW!, MARION. As usual many thanks for this report. I know you don't like me to say this but thanks again for being our Advocate! Knowing you as I do, we WILL be heard! Again, well done!

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.

3 (edited by PCL1029 Sat, 15 Dec 2012 23:16:39)

Re: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Hi, Marion,
Our disease definitely will provide much more patients ( customers) globally than just in the North America alone , China, Japan and Korea and Far East  and Europe countries. So it will a big selling to the investors ESP. With the FDA,USP Agencies' reputation behind it.
For our CCA, if we get chosen to be the first 20 rare disease study,and with the recent 1.7 million grant to 10 medical institutions to study our disease by NCI, and if the venture capital companies see this happen ALL at the same time and make financial backing on it,then our hope to fine a cure is not impossible within the next 3-5 years.
Thanks, Marion for you hard work.
God bless.

Below is another population data for Europe. By another journal.
1. GENERAL INFORMATION
1.1 Epidemiological Data
1.1.1 Incidence
Cholangiocarcinoma is an uncommon adenocarcinoma which arises from the epithelial cells of bile ducts, anywhere along intrahepatic and extrahepatic biliary tree, excluding the papilla of Vater and the gall bladder. In Europe, approximately 50,000 new cases of primary liver cancer are diagnosed every year (Parkin 2002). Data from the Cancer Incidence in Five Continents initiative (Parkin 2002; Curado 2007), indicate that approximately 20% of those cases are attributed to cholangiocarcinoma. The annual age-standardised incidence rate in Europe is less than 1.5 cases per 100,000 population. Cholangiocarcinoma is more common in males because of the predominant occurrence of primary sclerosing cholangitis (PSC) in men. Figure 1 shows the incidence rates in those European and Asiatic populations covered by cancer registries ( Parkin 20022). There is a marked geographic variability, largely due to variations in regional environmental risk factors. The majority of patients are older than 65 years, with the peak incidence occuring in the seventh decade of life (Parkin 2002; Curado 2007). The incidence of intrahepatic cholangiocarcinoma has been increasing recently in Western industrialized countries (Patel 2001; Taylor-Robinson 2001; Khan 2002).

Figure 1. Age-standardised incidence rates in European and Asian populations by gender (Parkin 2002)

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: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Percy… according to Janet Woodcock, the originator of this concept, the FDA has chosen the 20 diseases out of the more than 25 thousand patient and representatives requests received.  I strongly believe that the methodology developed within this group will spill over to our disease also and benefit us accordingly. 
Given the fact that you have qualified for the Patient Representatives to the FDA Advisory Committees and I am a member of the Alliance for a Stronger FDA, we are in a good position for our patient community to benefit from the forthcoming, positive developments.
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

Re: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Hi,Marion,
As far as I know, the 20 rare diseases selection are not finalized yet. They may revise and may add to that presumed list once they have made up their minds.
As an outsider looking into the FDA operation, I find them more or less like the other agencies of the government ,slow and cumbersome and too many layers to work it through. You can say the FDA is very cautious and methodological in doing business to protect the consumers . But that feeling is not mutual as far as I am concern. One of the reason may be I am the patientand feel more urgent than most of them who are not.
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.

6 (edited by marions Sun, 16 Dec 2012 15:54:20)

Re: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Hmmm.... Janet Woodcock and I spoke briefly at this conference.  I will try to reach her and will get to back to you. 
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

Re: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Thank you for this Marion. The $ numbers and time scale you talk of are quite frightening with regards to drug development, clinical trials and also failure rate of new drugs. With less than 1 in 10 trials succeeding, that is not a good rate at all is it. Grrrr....... But lets hope that much will change and much good will come from everything that is being done. Positive thoughts!

My thanks and hugs to you,

Gavin

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: FASTER CURES/PARTNERING FOR CURES CONFERENCE NEW YORK, 2012

Percy...according to Janet Woodstock's office, the twenty diseases chosen cover a broad area of requirements for this project.  It is not set in stone and there might be a chance for some of these diseases to be replaced with others, but it is highly unlikely for our disease to be chosen. 

On another note:  I so much agree with you.  Things are not moving fast enough.  We need accelerated research and we need it yesterday.  I see all the more reason for us to continue to challenge the current system and for our advocating for the global Cholangiocarcinoma patient community at large. 
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