Is access to unproven, experimental drugs a right-to-life issue?

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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. 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.

In early 2008, the U.S. Supreme Court declined to hear an appeal in the case of Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach. The case was brought on behalf of 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 Abigail Alliance argued 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.

Background

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?

Articles and publications

National sources

Organizations

  • Frances Visco

    Frances Visco is the president of the National Breast Cancer Coalition, an advocacy organization in Washington, D.C.

    She issued a statement in 2003 against the proposals of the Abigail Alliance.

    Contact: 800-622-2838, 202-296-7477.

  • Cancer Patients Alliance

    Cancer Patients Alliance is a 501(c)(3) non-profit organization led by a Medical Director and a five-member scientific board of highly regarded specialists in oncology from around the world. Their mission is to promote awareness, increase education and further pancreatic cancer research.

    They have supported wider participation in clinical trials and have given support to wider access to drugs for dying.

  • Society for Clinical Trials

    The Society for Clinical Trials is a unique group of representatives from government, academia, industry, for-profit and non-profit sectors that share is a common desire to learn, while advancing the field of clinical research. Contact Elizabeth Franks.

    They have issued a position paper opposing wider access to unproven drugs.

Bioethics institutes

Legal, medical and disease experts

  • Dr. Michael Grodin

    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.

  • Richard A. Merrill

    Richard A. Merrill is Daniel Caplin Professor of Law Emeritus 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 editor and author of multiple books on food and drug law.

  • Richard Cooper

    Richard Cooper is a partner at the Washington, D.C., law firm of Williams & Connolly, specializing in food and drug law. Formerly, he was general counsel at the Food and Drug Administration.

  • Frank Palumbo

    Frank Palumbo is a professor and director of the Center on Drugs and Public Policy at the University of Maryland School of Pharmacy in Baltimore.

  • Scott Ballenger

    Scott Ballenger is founder of the Trial Acceleration Institute in Spring House, Pa., which assists stakeholders in speeding up clinical trials.

    Contact: 610-526-0717.

  • Kenneth Kaitin

    Kenneth Kaitin is director of the Tufts Center for the Study of Drug Development in Boston.

  • Dr. Ezekiel J. Emanuel

    Dr. Ezekiel J. Emanuel is the Diane Levy and Robert M. Levy University Professor in the department of medical ethics and health policy at the University of Pennsylvania.

    He has written about the implications of the Abigail Alliance case.

  • Sylvia A. Law

    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 was formerly on the board of Compassion & Choices.

  • Dr. Jerome Groopman

    Dr. Jerome Groopman is the Dina and Raphael Recanati Professor of Medicine at Harvard Medical School, Chief of Experimental Medicine at Beth Israel Deaconess Medical Center and one of the world’s leading researchers in cancer and AIDS. Contact Dr. Groopman through his website.

    He wrote an article on the Abigail Alliance case titled “The Right to a Trial,” published Dec. 18, 2006, in The New Yorker (see above).

    Contact: 617-667-0070.

  • George Annas

    George Annas is professor and chairman of the health law department at the Boston University School of Public Health and an expert on abortion policy, embryo research, stem cells and end-of-life research.

    He is the author of American Bioethics: Crossing Human Rights and Health Law Boundaries (Oxford University Press, 2005).

  • Dr. Sidney Wolfe

    Dr. Sidney Wolfe is a senior adviser and co-founder of the Health Research Group, one of Public Citizen’s five policy groups. Public Citizen is a nonprofit public interest group in Washington, D.C. Contact Angela Bradbery, director of communications.

    Wolfe has expressed concern that weakening rules of access to drugs will be harmful.

Regional sources

In the Northeast

  • Frances Miller

    Frances Miller is a professor of law emerita at Boston University School of Law. Her interests include health law, food and drug law, biotechnology and genetics, comparative health systems, competition vs. regulation in health care and trusts and estates.

  • Carl H. Coleman

    Carl H. Coleman is director of the Health Law and Policy Program at Seton Hall Law School in Newark, N.J. Professor Coleman served as Bioethics and Law Adviser at the World Health Organization (WHO) in Geneva, Switzerland from 2006-2007. He has written on assisted suicide and was a member of the New York State Attorney General’s Commission on Quality of Care at the End of Life. He co-wrote The Ethics and Regulation of Research With Human Subjects (Lexis, 2005).

  • Lewis Grossman

    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, 3rd ed. (Foundation Press, 2007). His interests include civil procedure, food and drug law and legal history. He is of counsel at the law firm of Covington & Burling.

  • Mary Ann Chirba

    Mary Ann Chirba is a professor of legal reasoning, research & writing at the Boston College Law School in Newton, Mass.

    She has taught pharmaceutical product safety at the Harvard School of Public Health.

In the South

  • Lars Noah

    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, 2nd ed. (Foundation Press, 2006).

  • Kenneth W. Goodman

    Kenneth W. Goodman is a professor of medicine at the University of Miami and the founder and director of the University of Miami Bioethics Program and its Pan American Bioethics Initiative. He is also the co-director of the university’s Ethics Programs.

  • Dr. David Doukas

    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. He is a co-author of Planning for Uncertainty: Living Wills and Other Advance Directives for You and Your Family.

  • Daniel S. Goldberg

    Daniel S. Goldberg is a faculty member at the Department of Bioethics & Interdisciplinary Studies at East Carolina University in Greenville, N.C. His research interests include clinical ethics as well as ethical issues in clinical and pharmaceutical research.

In the Midwest

  • Maxwell Mehlman

    Maxwell Mehlman is the Arthur E. Petersilge Professor of Law and Director of the Law-Medicine Center at the Case School of Law and Professor of Biomedical Ethics in the Case School of Medicine at Case Western Reserve University in Cleveland. He is co-author of Genetics: Ethics, Law and Policy and author of Wondergenes: Genetic Enhancement and the Future of Society.

In the West

  • Dr. Leonard Deftos

    Dr. Leonard Deftos is a physician, medical researcher and lawyer. He is a 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.

  • Marsha Cohen

    Marsha Cohen is a law professor at Hastings College of Law of the University of California, San Francisco. Her expertise include food and drug law, torts and administrative law.

    She has taught courses on food and drug law.

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