Terminally ill patients have the right to refuse treatment. Do they also have the right to demand treatment with unproven experimental drugs if no approved treatments exist? This question is gaining increasing attention as patient advocates press for a legal right to unapproved drugs and critics counter that such access would hamper and even halt lifesaving progress in medical research. Observers expect a lawsuit now under review to eventually land in the U.S. Supreme Court. The issue pits the hopes of individual patients against a government-regulated drug approval system that yields research benefiting large numbers of patients; it also has the potential to reshape end-of-life issues.

For decades, the Food and Drug Administration has tightly controlled access to medications, allowing only those proved safe and effective to be sold to the public and generally permitting use of experimental drugs only in strictly regulated clinical trials or under other, very limited, conditions. Changes that weaken that system could damage the long-term prospects for finding cures for currently fatal diseases. The system has, however, prevented desperately ill people from using drugs that might potentially save their lives. One such person was Abigail Burroughs, who died of cancer in her twenties in 2001 after trying unsuccessfully to obtain an experimental drug that was later shown to have benefit against her disease.

The D.C. Court of Appeals reheard the case of Abigail Alliance v. von Eschenbach on March 1 and will issue a decision, perhaps in the next few months. Some observers suggest that the case may affect the broader field of end-of-life law, which has raised contentious religious issues in the past. Although religious groups have not yet specifically addressed the issue, it has the potential to broaden the right-to-life debate in the United States.

In May 2006, a panel of the U.S. Court of Appeals for the District of Columbia declared, in a 2-1 ruling in the Abigail Alliance case, that patients with fatal diseases have a constitutional right to try unapproved treatments that have passed the first phase of FDA testing if a doctor advises it and no alternative, approved treatment is available. Critics assert that permitting patients to use any drug they wish would destroy the incentives to join clinical trials that generate scientifically valid information about which treatments work and which do not. Patients generally participate in trials in the hope of getting experimental drugs that are otherwise unavailable. Not all patients enrolled in trials actually get experimental treatments, however; those in control groups get standard treatment or placebos unless the experimental drug proves effective.

Double-blind comparisons of experimental and control groups are considered the “gold standard” of medical research because they provide the most reliable information about treatments’ efficacy and safety. They also find many experimental drugs useless and even harmful, critics note. Besides slowing scientific progress, open access outside the protections of the clinical trial system would, they argue, subject vulnerable individuals to the dangers of both untested substances and unscrupulous quacks purveying them.

What’s new

• The full nine-judge D.C. Court of Appeals reheard the case of Abigail Alliance v. von Eschenbach on March 1.

• On March 14, the FDA will end a period of public comment on a new rule, proposed in December 2006, that would expand access to unapproved drugs within the context of preserving clinical trials. The Court of Appeals’ decision could render the rule moot.

Why it matters

The issue of expanded access to experimental medications pits two important values against each other: the right of individuals to defend their lives against present threats versus potential progress in medical science, which could save many lives in the future. The issue also has the potential to affect the right-to-life debate.

Questions for reporters

• Have terminally ill patients in your area attempted to obtain experimental drugs? What has been the outcome of those efforts?
• Have patients’ groups, research scientists, bioethicists or legal experts in your area taken a position on this issue?
• Have clergy, hospital chaplains or hospital ethics boards encountered this issue?

Jump to background

National sources

ORGANIZATIONS

• The Abigail Alliance for Better Access to Developmental Drugs is the plaintiff in the case pending before the U.S. Court of Appeals for the District of Columbia. Frank Burroughs, father of the late Abigail Burroughs, is president. Contact 540-899-3766, frankburroughs@abigail-alliance.org.
• Compassion & Choices, which calls itself “the oldest and largest choice-in-dying organization in the country,” supports the efforts of the Abigail Alliance. It has headquarters in Denver and in Portland, Ore. Contact executive director Marsha Temple, Mtemple@compassionandchoices.org.
• The Washington Legal Foundation in Washington, D.C., is a public interest law firm that is representing the Abigail Alliance for Better Access to Developmental Drugs in federal court. Contact 202-588-0302, info@wlf.org.
• Carl Lundblad is an attorney who writes a blog for the Law of Life Project of the Christian Legal Society in Springfield, Va.,a national organization of lawyers. The Law of Life Project is run by the Society’s Center for Law and Religious Freedom and has the mission of “defending the sanctity of human life from conception to natural death.” Lundblad believes that the decision in the Abigail Alliance case may influence other cases concerning the end of life and the right to life. Contact lundblad@lawoflifeproject.com.
• Frances M. Visco, president of the National Breast Cancer Coalition, an advocacy organization in Washington, D.C., issued a statement Sept. 9, 2003, opposing Abigail Alliance proposals. Contact communications director Sue Ducat, 202-296-7477, media-request@stopbreastcancer.org.
• Abbey Meyers is president of the National Organization for Rare Disorders in Danbury, Conn., and has expressed concern that open access to experimental drugs will ultimately harm patients by slowing research. Contact Mary Dunkle, vice president for communications, 203-744-0100 or 800-999-NORD, mdunkle@rarediseases.org.
• Ellen Stovall is president of the National Coalition for Cancer Survivorship in Silver Spring, Md., which petitioned the FDA to clarify rules of access to unapproved drugs. Contact 301-650-9127, info@canceradvocacy.org.
• Lorenzen Cancer Foundation of Monterey, Calif., supports wider participation
  in clinical trials and maintains a database of pancreatic cancer clinical
  trials. It has also given support to wider access to drugs for dying patients.
  Contact Aaron Stoertz, media relations, 831-658-0600.
• The Society for Clinical
  Trials, a scientific society in Baltimore that fosters the improvement
  of clinical trials, has issued a position
  paper opposing wider access to unproven drugs. Contact administrative
  officer Mary Karpers-Burke, 410-433-4722, sctbalt@aol.com.

LEGAL, MEDICAL AND DISEASE EXPERTS

• Arthur Caplan is a professor and chairman of bioethics at the University of Pennsylvania and director of the university’s Center for Bioethics. The go-to ethicist is author and editor of more than 25 books and 500 journal articles. Caplan has written and spoken about the Abigail Alliance case as well as other end-of-life issues. A frequent media commentator on bioethics, he co-edited The Case of Terri Schiavo: Ethics at the End of Life (Prometheus Books, 2006). Contact 215-898-7136, caplan@mail.med.upenn.edu.
• Dr. Ezekiel J. Emanuel is a breast oncologist and chairman of the department of clinical bioethics at the Warren G. Magnuson Clinical Center at the National Institutes of Health. He wrote a July 3, 2006, New Republic article about the implications of the Abigail Alliance case. Contact eemanuel@nih.gov.
• Sylvia A. Law is Elizabeth K. Dollard Professor of Law, Medicine and Psychiatry and co-director of the Arthur Garfield Hays Civil Liberties Program at New York University Law School. She is on the board of Compassion & Choices, which supports the Abigail Alliance’s efforts. Contact 212-998-6265, sylvialaw@aol.com.
• William Schultz, a former FDA deputy commissioner for policy, calls the Abigail Alliance decision “devastating.” He has taught food and drug law at Georgetown University Law School. He is a partner in the Washington, D.C., law firm of Zuckerman Spaeder LLP. Contact 202-778-1820.
• David Welch runs the 38lemon.com Web site, which chronicles his experience with a brain tumor, for which he is receiving off-label treatment. Contact 701-786-9196.
• Dr. Jerome Groopman is the Dina and Raphael Professor of Medicine at Harvard Medical School and chief of experimental medicine at Beth Israel Deaconess Medical Center. He wrote an article on the Abigail case titled “The Right to a Trial,” published Dec. 18, 2006, in The New Yorker. Contact 617-667-0070, jgroopma@bidmc.harvard.edu.
• George Annas is the Edward R. Utley Professor and chairman of the department of health law, bioethics and human rights of Boston University School of Public Health. He is also a professor in the university’s School of Medicine and School of Law. He is the author of American Bioethics: Crossing Human Rights and Health Law Boundaries (Oxford University Press, 2005). Contact 617-638-4626, annasgj@bu.edu.
• Dr. Michael Grodin is professor of health law, bioethics and human rights at Boston University School of Public Health and a clinical medical ethicist for Boston Medical Center. Contact 617-638-4626, grodin.bu.edu.
• Richard A. Merrill is Daniel Caplin Professor of Law at the University of Virginia Law School in Charlottesville. He is a member of the Institute of Medicine of the National Academy of Sciences and is co-editor of Food and Drug Law: Cases and Materials, 3^rd ed. (Foundation Press, forthcoming 2007). Contact 434-982-2083, ram@virginia.edu.
• Richard Cooper was formerly general counsel at the Food and Drug Administration and is a partner in the Washington, D.C., law firm of Williams & Connolly, specializing in food and drug law. Contact 202-434-5466, rcooper@wc.com.
• Frank Palumbo is director of the Center on Drugs and Public Policy at the University of Maryland School of Pharmacy in Baltimore. Contact 410-706-2303, fpalumbo@rx.umaryland.edu.
• Scott Ballenger is founder of the Trial Acceleration Institute in Spring House, Pa., which assists stakeholders in speeding up clinical trials. Contact 215-646-1490.
• Kenneth Kaitin is director of the Tufts Center for the Study of Drug Development in Boston. Contact 617-636-2170, csdd@tufts.edu.
• Dr.
  Sidney Wolfe is editor of the Public Citizen Health Research Group Health Letter. Public Citizen is
  a nonprofit public interest group in Washington, D.C. Wolfe has expressed
  concern that weakening rules of access to drugs will be harmful. Contact
  202-588-7742 or 202-588-7703, rpleatman@citizen.org.

Background

• Read a March 2, 2007, FT.com story about the appeals court hearing, posted by MSNBC.com.
• Read a Feb. 18, 2007, Newhouse News Service story about the Abigail Alliance case. It’s posted by Masslive.com.
• Read a Dec. 18, 2006, New Yorker article, “The Right to a Trial,” about the Abigail Alliance case.
• Read a July 3, 2006, New Republic article about the implications of the Abigail Alliance case.
• Read a May 3, 2006, Washington Post story about an appeals court ruling in the Abigail Alliance case.
• Read a description of the drug development and approval process in the CDER Handbook, provided
  by the Center for Drug Evaluation and Research of the Food and Drug Administration.
• Read
  a Dec. 11, 2006, FDA news release on
  the proposed regulations that would expand access to unapproved drugs
  within the context of preserving clinical trials. Read the FDA’s proposed rule.

Regional sources

IN THE NORTHEAST

• Mary Ann Chirba-Martin is an associate professor at the Boston College Law School in Newton, Mass., and has taught pharmaceutical product safety at the Harvard School of Public Health. Contact 617-552-4381, chirbama@bc.edu.
• Frances Miller is a professor of law, public health and health care management at Boston University. Contact 617-353-4469, fmiller@bu.edu.

IN THE EAST

• Carl H. Coleman is a law professor and director of the Health Law and Policy Program at Seton Hall University Law School in Newark. He wrote The Ethics and Regulation of Research With Human Subjects (Lexis, 2005). Contact 973-642-8586, colemaca@shu.edu.
• Lewis Grossman is a professor at Washington College of Law at American University in Washington, D.C., and co-author of Food and Drug Law: Cases and Materials, 3^rd ed. (Foundation Press, forthcoming 2007). He is of counsel at the law firm of Covington & Burling. Contact 202-274-4363, lewisg@wcl.american.edu.

IN THE SOUTHEAST

• Lars Noah is a law professor at the Fred G. Levin College of Law of the University of Florida in Gainesville and author of Law, Medicine and Medical Technology: Cases and Materials, 2^nd ed. (Foundation Press, 2006). Contact 352-273-0923 noah@law.ufl.edu.
• Kenneth W. Goodman is co-director of the bioethics program of the University of Miami. Contact 305-243-5723, kgoodman@miami.edu.

IN THE SOUTH

• Edward P. Richards III is Harvey A. Peltier Professor of Law and director of the Program in Law, Science and Public Health at the Paul M. Hebert Law Center at Louisiana State University in Baton Rouge. Contact 225-578-7595, richards@lsu.edu.
• Dr. David Doukas is a faculty member in the Institute for Bioethics, Health Policy and Law, and he holds the William Ray Moore Endowed Chair of Family Medicine and Medical Humanism at the University of Louisville. Contact 502-852-1806, david.doukas@louisville.edu.

IN THE MIDWEST

• Michele Goodwin holds the Wicklander Chair in Ethics and is a professor of law at DePaul College of Law in Chicago. She directs the Health Law Institute as well as the Center for the Study of Race & Bioethics. Contact 312-362-8127, mgoodwin@depaul.edu.
• Maxwell J. Mehlman is director of The Law-Medicine Center and Arthur E. Petersilge Professor of Law and professor of Bioethics in the Case School of Medicine at Case Western Reserve University in Cleveland. Contact 216-368-3983, mjm10@case.edu.

IN THE SOUTHWEST

• Charles R. McConachie is an adjunct professor at the Dedman School of Law of Southern Methodist University in Dallas, where he teaches food and drug law. He is also a partner in the Dallas law firm of Simpson Woolley McConachie LLP. Contact 214-871-5083, mcconachie@swmdallas.com.
• Daniel S. Goldberg is research professor at the Health Law & Policy Institute at the University of Houston Law Center. He is also a doctoral student in ethics and medical humanities at the University of Texas Medical Branch. His research interests include clinical ethics as well as ethical issues in clinical and pharmaceutical research. Contact 713-743-2198, dgoldbe2@central.uh.edu.

IN THE WEST/NORTHWEST

• Dr. Leonard Deftos, a physician, medical researcher and lawyer, holds positions as professor of medicine at the University of California-San Diego School of Medicine, a physician at the VA San Diego Healthcare System and an adjunct professor of law at California Western School of Law in San Diego. Contact 858-552-8585 ext. 3715, ljdeftos@ucsd.edu.

• Marsha Cohen is a law professor at Hastings College of Law of the University of California, San Francisco. She teaches food and drug law and pharmacy law and has served on the California State Board of Pharmacy. She is the first non-pharmacist to be its president. Contact 415-565-4676, cohenm@uchastings.edu.