Drug safety has understandably been much in the press recently -- but speeding good new drugs to those who need them most is equally critical.
On September 18, 2007, a number of patient advocacy groups gathered before the FDA's Headquarters in Rockville, Maryland, to rally on their designated "A Right to Life Day." Participants raised their signs and shouted, passionately arguing that FDA often slows access to experimental drugs for the terminally and very seriously ill (specifically the new drugs Provenge, for advanced prostate cancer; and Juvenon, for osteosarcoma in children). The Huntington's community may itself need access to experimental drugs soon, with many promising HD agents now in the research pipeline. At HDDW, we are looking into this issue now to help ensure that the best new drugs reach our community just as fast as they can.
The demonstrators rallying before the FDA wanted to bring attention to what they see as FDA's glacial drug approval process and other problems with FDA's clinical trial procedures. In particular, some argued that conflicts of interest had also played a part in FDA's recent failure to approve the promising drug Provenge. (One FDA review committee member who voted against approval in this case is a lead researcher and investor in a competing prostate cancer drug.) Above all, the groups criticized FDA's overcautious approval process for drugs.
Members of the demonstrating groups have already helped write legislation - the proposed ACCESS Act - to broaden access to experimental drugs for seriously ill patients, and they have brought lawsuits against FDA. Though a lower court's decision ruled in their favor, the case was lost on appeal to a higher court. One of the participating patient advocacy groups, Care to Live, is now bringing another lawsuit, claiming that the FDA acted arbitrarily and capriciously in denying patients approval of and access to Provenge.
FDA has acknowledged the importance of speeding and increasing access to experimental drugs for severely ill patients through several measures it has taken, including the Expanded Access Rule, which the agency proposed in December of 2006, and the agency's Critical Path Initiative (see weblinks below). The proposed Expanded Access Rule is designed specifically to make experimental drugs more widely and easily available to seriously ill patients with no other treatment options and to clarify when and how manufacturers can charge for these drugs. FDA is currently reviewing comments received on this proposed rule and should issue its next statement on the rule in coming months. FDA's second effort, the Critical Path Initiative, aims to modernize the technical tools through which scientific breakthroughs are brought to fruition through the drug approval process.
Soon enough, the HD community will also be focused on securing the earliest appropriate access to experimental drugs. Some of the many promising lines of current HD research could lead to better therapeutics, perhaps even effective cures, in not too very long. The HD community must be organized when the time comes to promote the availability of promising new HD drugs and therapeutics.