FDA Primed to Help Dying Patients Gain Easier Access to Unapproved Drugs
By Beryl Lieff Benderly*

In 2007, terminally ill patients are expected to gain expanded access to unapproved medications. As soon as its proposed guidelines become final later this year, the Food and Drug Administration (FDA) will proactively assist physicians in getting experimental drugs in the hands of their dying patients, the Food and Drug Law Institute (FDLI) has learned.

FDA is taking action on the controversial issue of access to unapproved drugs as the landmark case of Abigail Alliance v. von Eschenbach heads toward a possible showdown in the U.S. Supreme Court. On March 1, the U.S. Court of Appeals for the District of Columbia will hear FDA's rehearing petition on its May 2006 ruling that terminally ill patients have a constitutionally based right to access to experimental drugs not fully approved by the agency.

In March 2006, two groups active in cancer treatment issues — the National Coalition for Cancer Survivorship and the American Society of Clinical Oncology — petitioned FDA to "clarify the circumstances" under which pharmaceutical companies may provide unapproved drugs to individuals with "life-threatening illnesses" who have "no other acceptable treatment options" and are not participating in clinical trials. Then, more than six months after the ruling by the three-judge appeals court panel, FDA published a proposal for "significant regulatory changes" to expand access to unapproved drugs.

The agency is currently evaluating public comment on those guidelines and is not expected to issue final rules for several months. However, after FDA publishes those final rules, it is committed to making sure that physicians who want to get unapproved medications for their patients have all the information they need to process their applications, FDA spokeswomen Susan Cruzan tells FDLI. "As we say in the preamble to the rules, FDA will make educational programs and materials available to help physicians and sponsors understand the expanded access use submission requirements in general, as well as the additional information necessary to justify the different types of expanded access."

"We plan to do a number of educational/outreach [efforts]," Cruzan explains. "This effort will coincide with publication of the final rule, and the details of this effort will be worked out by the time we publish the final rule. We also plan to engage external constituents [and] help identify the information needs and the target audiences as we develop this effort."

Although patients will be able to apply through their physicians for expanded access to unapproved drugs, the FDA has no authority to compel them to do so, emphasizes Cruzan. "As the preamble to the proposed rule says, 'FDA cannot compel a drug manufacturer to provide access to investigational drugs for treatment use.'" Whether pharmaceutical firms make such medications available will be their decision, Cruzan explains. FDA will attempt to balance the need of individual patients with its duty and responsibility to protect the clinical trial process that sets out rigid procedures for bringing drugs to market, Cruzan notes. "As the preamble states, the agency has tried in the proposed rule to strike the appropriate balance between authorizing access to promising drugs for treatment use and ensuring the integrity of the drug approval process," Cruzan explains. "The third criterion for all expanded access uses [as stated in the proposed rule] is that FDA must determine that providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use. In the proposed rule, various criteria, submission requirements, and safeguards were included in an attempt to protect the drug approval process," adds Cruzan.

No matter what happens with the final FDA rule and the Abigail Alliance case, the decades-long debate over expanded access is not likely to end soon. For David Welch, whose website (www.38lemon.com) has chronicled his experience seeking treatment for a "lemon-sized" brain tumor, the need to maintain the "integrity" of the clinical trials process is key. "It's my duty to be a solid data point in a clinical trial rather than me just grabbing for few extra months [of life]," he tells FDLI. "There's a larger battle going on than just me."

But on the other hand, says Welch, "in my overall treatment for brain cancer, my strategy is to plan for worst but hope for the best." In that spirit, he can imagine "a scenario where I have gone through standard protocols, perhaps through some clinical trials," and reached a point at which no further approved or trial treatments are available. Should he then be able to use an unapproved drug? He's hasn't reached a conclusion on that issue yet. "I want to hear both sides of the argument," he says, because "I could well be in that situation." (The Food and Drug Law Institute will sponsor a Colloquium on Access to Unapproved Drugs, Whose Life Is It, Anyway?, Feb. 27, 2007, in Washington, D.C. For more information on the Colloquium, visit FDLI's home page, www.fdli.org.)

*Beryl Lieff Benderly is a freelance healthcare writer based in Washington, D.C.