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