Rare Cancers RESEARCH PARADIGM
NOARC will give guidance to all coalition member organizations in ways that their organizations can best work to increase therapeutic options for their respective rare cancer/exceptionally rare disease. Our approach is to advise for the formation of patient registries and tissue banks.  For rare cancer groups, once your registry and tissue bank is established, it can be used to create a clinical trials consortium within the framework of the NCI Cancer BioInformatics Grid (CaBIG). It can also be integrated into the recent initiative between FDA, NCI and Center for Medicare Services called the "Oncology Biomarker Qualification Initiative."  A demand by our community has to be that similar biomarker qualification projects begin for all Rare Cancers.

"PROJECT 53" 
The U.S. Food and Drug Administration has established what they refer to as the "Critical Path Initiative" to speed development of new agents for patients with life-threatening diseases.  They have published a "Critical Path Opportunities List" of over 70 areas of focus that will help realize this vision for the country.  Item number 53 on this list is focused on rare diseases:  

53. Natural History Databases for Rare Diseases. Many rare diseases are hard to study due to both the difficulty in enrolling subjects and the long duration of clinical trials. Databases recording the natural history of patients with rare diseases, incorporating observations on clinical progression and biomarkers, could assist in creating disease models and better designing clinical programs and, possibly, contribute virtual historical control groups

NOARC believes that establishing natural history databases will result in the ability to perform clinical trials more easily and efficiently through the application of the data towards the historical control groups mentioned in Item 53.  One of our founding organizations, the Sarcoma Foundation of America (SFA), is beginning its registry / tissue bank project, and will try to do so in sync with the FDA Critical Path Initiative.  The SFA is willing to let any other coalition member know of its "lessons learned" so that other coalition groups can streamline their own efforts at establishing natural history databases.  

The Exceptionally Rare Disease "Advantage" in Biomarker Validation 
As a result of the Humanitarian Device Exemption Act,  pharmaceutical industry members working to co-develop molecular diagnostics with targeted new drugs have a decided "edge" if they aim their development plans towards patient populations that number less than 4000 United States prevalence. The HDE Act, by design of Congress, allows for extraordinary leeway in the amount of validation work necessary for approval for an exceptionally rare disease-targeted molecular diagnostic.  We feel that current validation standards for molecular diagnostics for larger disease populations will severely inhibit introduction of new agents in the "targeted therapy" era that we have now entered.  While waiting years and perhaps decades for the proper legislative relief in this area, pharmaceutical/molecular diagnostic manufacturers would be well-advised to take advantage of the HDE reform success and consider the added value of initial co-development of their targeted agent + diagnostic in disease populations less than 4000.