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 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 are impossible to perform, yet clinical data and other supportive data present a compelling picture that the new agent would provide some benefit to the patient.  A proposed regulatory paradigm would be to require demonstration by the drug sponsor to the FDA that indeed clinical trials cannot be done, and are even incapable of utilizing the accelerated approval mechanisms that exist to help foster some types of drugs approvals.  The language in this legislative initiative, now being drafted, sets very strict criteria that the drug company and FDA must follow, and has safeguards preventing abuse of approval based on more "relaxed" standards than normally seen.  NOARC appreciates how sensitive some advocacy groups and other bodies are to the perception of "lower" standards for efficacy approval for new agents for life-threatening diseases.  This unique pathway is envisioned for only extraordinary circumstances, but NOARC feels that the pathway might occasionally provide some hope to some patients who otherwise would die of their ailment without this pathway. 

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.

Conquer Childhood Cancer Act of 2007
NOARC endorses all of the provisions of the Conquer Childhood Cancer Act of 2007 introduced in mid-2006.