ESTABLISH AN "OFFICE OF RARE CANCERS" WITHIN THE NATIONAL CANCER INSTITUTE
The election of President Obama, with his promise to focus on the plight of those with rare cancers (see Press Release for details), is a great opportunity for our community!  NOARC believes that one way to accomplish this goal is to establish an Office of Rare Cancers within the National Cancer Institute.

This Office could be the epicenter of the nation's focus on rare cancers, helping to expand and coordinate research and policy efforts to increase therapeutic options for those Americans suffering from rare cancers.

President Obama has similarly pledged to double the size of the NCI budget during his time in office.  Therefore the funds to create such an Office could easily be envisioned as part of upcoming Appropriations by the federal government.

Let us know what you think of this idea, and how we can all work together to help advance this agenda!

Improving Drug Approval Pathways for Ultra-Rare Diseases WITHOUT Surrogate Endpoints for Clinical Trials
An important legislative initiative for The National Organization Against Rare Cancers is to address the lack of clear FDA approval pathways if an ultra-rare disease has no established (validated) surrogate endpoints upon which to base the "accelerated approval" of a new drug. Legislation addressing this was introduced in the Congress on December 20, 2011 through the efforts of our like-minded brothers and sisters at the EveryLife Foundation for Rare Diseases. The bill, called the ULTRA Act, would result in modest reform of the Food, Drug and Cosmetic Act to result in a unique pathway for drug development for patients with extraordinarily rare diseases. This pathway could be utilized by a pharmaceutical company in situations where clinical trials to establish a surrogate endpoint are impossible to perform (i.e. the hundreds of patients required for validation studies simply do not exist), yet a biomarker or some other clinical endpoint exists that paints a compelling picture that an improvement in the surrogate would provide some benefit to the patient.

Improving Drug Approval Pathways for Ultra-Rare Diseases WITH Surrogate Endpoints for Clinical Trials
Another important legislative initiative of NOARC is passage of legislation that will help foster drug development for diseases that have established surrogate endpoints for clinical trials, but have patient populations too rare to receive full, final FDA approval.  As with the related legislative issues above, this legislation would also result in modest reform of the Food, Drug and Cosmetic Act to result in a much faster, common sense pathway for drugs approved for patients with Rare Cancers by the accelerated approval mechanisms currently in place at the FDA. This piece of legislation focuses on the esoteric, yet important phase of drug development that occurs AFTER accelerated approval, but BEFORE full, final approval of a drug for a life-threatening illness. The legislation addresses the so-called "fugue state," or "quagmire state" of post-marketing confirmatory trials in ultra-rare disease settings.  The legislative relief under development would allow a quality "body of data" to take the place of "adequate, well-controlled clinical trials" in exceptional circumstances where it is demonstrated to FDA that the usual pathway is not feasible due to rarity.  NOARC considers these reform efforts to be very mainstream since current standards for accelerated approval of new agents are not affected. Without reform like this, predictable access to helpful drugs approved conditionally by the accelerated approval mechanism, will never be assured for patients in continual need. One of the Advisory Committees to the FDA, the Oncology Drugs Advisory Committee, proposed this very reform at its November, 2005 meeting with FDA.  So in this case, NOARC is serving as the messenger to have FDA be allowed to carry-through on what its own advisory committee recommended.

A Citizen's Petition to the FDA to promote creation of a development path for approval of new therapies for rare cancers
View the submission to the FDA that occurred October 26, 2007 by NOARC members.

View the support NOARC's Citizen Petition is gaining from Congress