Blog RSS Blog.

Author Archive: Practical Bioethics

About Practical Bioethics


Group-Level Expanded Access to Unapproved Medicine: The realistic solution for patients

Guest Blog Author: Jess Rabourn*, Managing Director, ALS – Emergency Treatment Fund

Since 1962, Federal law prohibits open sales of any new pharmaceutical product until that product goes through years of clinical research and is granted approval by the FDA.  

Yet unapproved new drugs are provided to thousands of patients daily.  They’re not openly sold, but instead made available by drug companies to study safety and effectiveness in humans.    FDA allows such managed uses of unapproved drugs through authorized Investigational New Drug (IND) exemptions, which are filed by drug companies, physicians, or other program sponsors.

INDs are filed for clinical trials that are intended to gather data and to support marketing approval.  Additionally, INDs for Expanded Access may be filed when seriously ill patients lack access to traditional clinical trials and there is no other effective treatment available.  Expanded Access programs were used extensively in the late 1980s and early 1990s to provide exploratory treatment to persons living with AIDS. As new antiretroviral drugs were studied in research trials they were simultaneously made available in much larger Expanded Access programs for the many patients who were too sick to take part in the research trials.

Between 2002 and 2012, a new class of lifesaving TK-1 inhibitors was made available to tens of thousands of cancer patients through several Expanded Access programs.

FDA understands the need for treatment options and has a track record of liberally authorizing Expanded Access through group-level “Intermediate Size INDs” and larger group “Treatment INDs”.  A third kind of Expanded Access IND exists for exceptional single-patient cases, but this channel is inappropriate for most diseases, provides no useful data, and is almost never agreed to by the drug companies.  True Expanded Access programs enroll on a group level just like regular clinical trials and require no patient-level FDA filings.

Despite the easy regulatory pathway, drug companies struggle to justify the costs and burdens of providing their product to patients before commercialization.    THIS factor, more than any other, is what keeps dying patients from accessing safe exploratory medicines.  As a consequence, there aren’t nearly as many access programs as there are eligible drugs in deadly diseases.

To address this gap, a nonprofit company called ALS Emergency Treatment Fund (ALS-ETF) was formed in 2012.  ALS-ETF was built to sponsor Expanded Access programs as an independent third party, for ALL patients who otherwise have no access to investigational medicine.  Through FDA-authorized cost recovery, ALS-ETF can engage healthcare payers, patients, and charitable assistance funds to ensure enrollment is both scalable and fair.  Participating clinics work in partnership with the platform –not as service providers- allowing ALS-ETF to manage large, centrally-coordinated Expanded Access programs at little or no cost to drug companies.  Well-designed Expanded Access programs can generate vast amounts of patient response data, which is especially valuable in poorly understood diseases.

This responsible solution works under the laws that already exist and has been enthusiastically received by FDA, drug companies, and research organizations in multiple disease areas.

* Mr. Rabourn spent the first 15 years of his career in institutional asset management and was awarded the Chartered Financial Analyst designation in 2004. He held a senior position at a New York hedge fund until 2008, when his dad, Frank Rabourn, showed the first symptoms of ALS. From that point onward, he has made it his full time career and personal mission to change the treatment landscape for current and future rare disease sufferers.  Mr. Rabourn is involved in multiple drug development ventures and, in 2012, joined three ALS families to launch ALS Emergency Treatment Fund, a professional service provider and a leading authority on Expanded Access programs.

Full Article

This entry was posted in Health Care and tagged . Posted by Practical Bioethics. Bookmark the permalink.


The Center for Practical Bioethics Weighs In on Proposed “Right to Try” Legislation in Kansas

A perspective from John G. Carney’s (President, Center for Practical Bioethics) based on testimony before the House Committee on Health and Human Services (HB 2004, Right to Try)

Most of us would agree that the “Right to Try” legislation proposed in the State of Kansas is appealing in the world of ideas and the realm of rhetoric where personal freedom and the pursuit of limitless aspirations are admirable ideals. But the world of reality bends its merits to questionable decisions that may disrupt the safe delivery of care to the most vulnerable population that healthcare professionals are called to serve.

In bioethics we recognize the deep-seated human impulse to rescue the imperiled, which is what this legislation seeks to do. However, we also grudgingly acknowledge that we could not run our businesses, public and private services or health care systems while indulging this impulse without limits. Furthermore, it is also widely accepted that “spectacle ethics” that turn individual cases into cause célèbre should not dictate public policy – no matter how heart-breaking they are. In the real world, there are good reasons to think that the “Right to Try” legislation should not be pursued despite our natural impulse to rescue.

While the popular framing of this issue characterizes interventions as miraculous and life-saving, there is little to no evidence that the interventions actually result in a good or “hoped for” outcome. We are not bound to provide a treatment that offers no benefit. In fact, it is a violation of our moral duty to do so.

Proponents may argue, if there is some evidence in the first phase of the clinical trial process that the patient may benefit from this treatment, then we should allow them access in the face of their impending death. Urgency is a given in these instances and experts argue that impending death is a criterion that allows for special consideration in these cases. But we are also obliged to consider the facts before arguing for new legislation to provide that consideration. 

Recent efforts within the FDA clearly demonstrate the agency’s attention to this issue. The FDA has updated and expanded its expedited processes for accommodating requests for access to drugs under development called Investigational New Drug (IND) Application. FDA Commissioner Dr. Margaret Hamburg has reported that in FY 2013, the most recent year for which data are available, nearly 100% of all applications submitted were approved (974/977) and many of those requests processed within hours of submission.

FDA has further pledged to continue to streamline its efforts despite its mandate to ensure safety and efficacy. For individual states to adopt legislation that circumvents the process of safety and efficacy places undue burdens on private business and manufacturers. Drug and medical device companies have repeatedly expressed concerns about their ability to handle and process the applications diverted from the FDA.

Furthermore, drug manufacturers are under no obligation to provide access to their products. The effects of unlimited access to drugs that have only been through Phase One clinical trials cannot be known and subverts the scientific process. In turn, accommodating appeals by individual patients ultimately diverts private business interests on the basis of questionable practices.

The substantive ethical and practical problems associated with this bill, coupled with the fact that these instances are exceedingly rare (estimated to be about 5 cases a year in Kansas), raise serious doubts about its merits. Significant and promising collaborative efforts are underway, such as the ALS Emergency Treatment Fund, that offer far more hope to patients than “Right to Try.”

The agencies that provide for our safety and ensure the efficacy of treatments operate in the real world, as does the legislature. Despite our natural need to rescue the imperiled, this committee and this body retain the responsibility to legislate with prudence and wisdom.

John G. Carney, President and CEO
Center for Practical Bioethics

Full Article

This entry was posted in Health Care and tagged . Posted by Practical Bioethics. Bookmark the permalink.


Chronic Pain — The Invisible Public Health Crises

Chronic Pain -- The Invisible Public Health Crises

A Call for Moral Leadership

“I am an invisible man. No I am not a spook like those who haunted Edgar Allen Poe: Nor am I one of your Hollywood movie ectoplasms. I am a man of substance, of flesh and bone, fiber and liquids- and I might even be said to possess a mind. I am invisible, simply because people refuse to see me.”
- Ralph Ellison

Ralph Ellison’s famous novel, The Invisible Man, starts with this passage, which also reminds me of the problem of chronic pain. The Institute of Medicine (IOM) report, Relieving Pain in America, documented the more than 100 million Americans (almost 1 in 3 and surely someone whom you know and love) suffers from chronic pain, at an economic cost of $6 billion and an incalculable psychological cost. We named pain as a “disease” because of its profound effects on the brain and its interference with multiple domains of the quality of life of sufferers. The committee identified chronic pain as a public health problem, given the sheer numbers affected, and the opportunities to intervene to prevent acute pain from becoming chronic pain. However, the report is now almost four years old, and it is fair to say has not really moved the needle in doing what we implored in the IOM report—“changing the way in which pain is judged, managed and perceived.” Why is that?

Because pain is subjective -- and therefore difficult to measure by the usual medical tests -- it is often doubted. As someone once said, my pain is real, your pain is in doubt. Also, we live in profound cultural ambivalence about pain. Cultural icons such as  Julius Caesar and Albert Schweitzer have been quoted as saying that pain is worse than death, but there is also an ethos of “no pain, no gain.” Medical interventions, particularly powerful opioid drugs such as morphine and oxycodone, although essential to manage acute and persistent pain, come with a cost of many side effects and may induce psychological dependence in some. Persons in pain and their doctors fear addiction, although we do not truly know the risk of addiction in persons taking opioids who have not abused recreational or illicit drugs. For these and other reasons, on an individual and societal level, we prefer to ignore the problem of chronic pain, unless confronted by it in our personal lives.

So how do we advance the moral imperative to address pain and suffering in contemporary medical practice, as required by our ethical codes and professional oaths? How do we bring the invisible suffering of so many to light and work to alleviate it? I think we commit ourselves to five big goals:

1. We advocate for more and better science to understand the underlying neuroscience of pain production and modulation. This requires advocacy at the NIH and other federal agencies to fund worthy science related to pain mechanisms and clinical trials of pain treatments.

2. We advocate for more and better drug development, including the creation of abuse deterrent opioid formulations and novel non-opioid based analgesics. This will require advocacy for effective public-private partnerships between the pharmaceutical industry, academia and federal agencies.

3. We advocate and demand better education of health care professionals to live up to their obligations to be competent and to attend to pain and suffering in their patients. We also advocate for better public education so that persons suffering with chronic pain understand that this is a disease, and not subject to quick fixes.

4. We advocate and demand better policy solutions to provide sustainable and patient-centric interdisciplinary pain treatment programs that truly address patient and provider needs. 

5. Finally, we need effective collaboration on a shared policy agenda between pain specialists and substance abuse specialists to advocate for comprehensive, rehabilitation-focused care for chronic pain, and greater access to substance abuse treatment for those persons who have a dual diagnosis of chronic pain and addiction.    

These are my thoughts. What do you think?

Richard Payne, MD
John B. Francis Chair, Bioethics
Center for Practical Bioethics

Esther Colliflower Professor of Medicine and Divinty
Duke University

Full Article

This entry was posted in Health Care and tagged , , , , . Posted by Practical Bioethics. Bookmark the permalink.


Privacy and Whole Genome Sequencing

-->Privacy is a big topic that’s getting bigger and more complicated every day. People increasingly accept that just about anyone can find information about them on the Internet. Indeed, millions make it easier by sharing details on social media.

With respect to health and healthcare, it’s thrilling to look forward to a time when cures are found and people who have banked their genetic code can be contacted to learn about and benefit from them.  

At the same time, many are concerned about potentially negative consequences of the availability of genetic information. What if, in the wrong hands, it could affect health insurance, employment, loans, a divorce or custody case, even the chance of finding a spouse – not only for the individual but other living and future relatives?

The Center for Practical Bioethics’ 2013 Fall Symposium, “Ethical Frontiers in the Brave New World of Genomics and Learning Health Systems” on November 8 will explore the ethical implications of whole genome sequencing in light of its rapid growth and importance to Learning Health Systems, where knowledge is drawn from every encounter, both research and clinical, yet policies regarding the handling of data differ substantially. 

The Presidential Commission and Beyond

The Presidential Commission for the Study of Bioethical Issues’ report submitted to President Obama in October 2012, “Privacy and Progress in Whole Genome Sequencing,” made recommendations in six areas:

·      Strong baseline protections while promoting data access and sharing
·      Data security and access to databases
·      Informed consent process
·      Facilitating progress in whole genome sequencing
·      Public benefit from medical advances that result from whole genome sequencing

“The Presidential Commission’s work and the Center’s Symposium,” said Alan Rubel, JD, PhD, “are first steps in the process of tailoring the apparatus we already have in place to deal with the problems inherent in whole genome sequencing.” Dr. Rubel served as an advisor to the Presidential Commission, an assistant professor at the University of Wisconsin School of Law, and will join the faculty at the Symposium.

Procedures to Drive Benefit & Minimize Risk

“The potential for people to benefit and suffer negative consequences from whole genome sequencing is substantial enough that we need to develop procedures to ensure that we derive the most benefit without jeopardizing people’s well being,” said Dr. Rubel. “How information is used isn’t always clear. There ought to be some way – some standard language – to convey what the possibilities are so that people donating material or having their genome sequenced can be said to properly consent. There are issues of data security as well.”

Dr. Rubel is a strong proponent of providing more and better information to the public about what whole genome sequencing can and cannot do. “Many conditions have to do with chronic and environmental disease that can be prevented by changes in lifestyle, independent of genomics, and may be better addressed without getting into genomics.”

He also believes that concerns about privacy often mask other worries that may be best understood as issues of justice. “If, for example, our worry is that genetic information being disclosed will harm our employment, it may be that the real problem is how vulnerable people are in terms of employment, and if we deal with that, the genetic issue goes away.”

About the Symposium

At this Symposium, you will:

·      Hear updates on progress in the brave new world of genetic science.
·      Gain insight into ethical issues around data collection, privacy and informed consent, especially as they apply to genomics and Learning Health Systems.
·      Explore what type of ethical framework is needed to advance research while protecting human subjects in Learning Health Systems.

Register now at For more information, contact Cindy Leyland at 816-979-1357 or

Full Article

This entry was posted in Health Care and tagged , , , . Posted by Practical Bioethics. Bookmark the permalink.


How Future Doctors Think

How Future Doctors Think
Flanigan Lecture Explores How Medical Students Make Sense of Their World

 What kind of physician do you want? Do you want someone who, out of respect for your autonomy, explains treatment options but makes no recommendations, leaving the decision up to you? Or do you want something more?

If you want something more, the research conducted by Felicia Cohn, Ph.D., who presented the 19th Annual Rosemary Flanigan Lecture on July 30, 2013, underscores the importance of knowing how your doctor thinks and helping him or her to understand your story.

Patterns in Their Stories

When Dr. Cohn taught medical ethics at the University of California-Irvine School of Medicine, she noticed patterns in the stories medical students shared concerning conflicts they encountered between their personal values and professional obligations and what they did about them. Her colleague at UCI, Humanities Director Johanna Shapiro, wondered if there might be a way to analyze and identify themes from these stories as told in 299 papers Dr. Cohn had collected.

“The themes of the students’ narratives really did fall quite neatly into six categories,” said Dr. Cohn.

• Restitution (38%)  - Appealed to a moral norm or ethical principle.
• Compromise (16%) – Conceded core values.
• Journey (16%) – Grew through experience.
• Witnessing (13%) – Felt empathy but stood by and watched.
• Resistance (9%) – Rejected professional ethics in favor of personal views
• No Problem (2%) – Never experienced any conflict.

“I thought the papers would tell a lot more stories about witnessing and compromise, which were the type of stories that the students mostly shared in class,” said Dr. Cohn. “But it turned out that restitution – appeal to principle – was the most common story they told. And more often than not the principle the students appealed to was autonomy. In other words, it’s okay for me to do this because it’s what the patient said he wants.”

Autonomy or Guidance?

Dr. Cohn explained that this emphasis on autonomy in modern medical culture has important implications for both healthcare professionals and their patients.

“If physicians really think what a patient is doing is wrong but feel that respecting autonomy takes precedence, they’re going to be spending a lot of time doing things that they think are wrong. Then they’re miserable and we go and ask them to be nice to patients. I can’t help but think that’s where a lot of the dissatisfaction and even burnout from healthcare professionals comes from.”

Conversely, for the patient who wants more than options from their physician – who wants their physician to consider how their illness and treatment will integrate into their life and make honest recommendations based on that – then the doctor who tells a restitution story probably isn’t the doctor for them.

Learn more at

Full Article

This entry was posted in Health Care and tagged , , . Posted by Practical Bioethics. Bookmark the permalink.


A Primer on Learning Healthcare Systems

Healthcare systems and all who participate in them – providers, patients and administrators – have an obligation to pursue continuous quality improvement in order to quicken the pace of medical discovery, reduce harm to patients and improve the efficiency of care. To achieve these goals, traditional healthcare systems must transform into Learning Healthcare Systems that more seamlessly incorporate research into clinical care, continuously learn from daily patient-provider encounters, and use ever-expanding databases to inform patient assessment and treatment.

These activities raise thorny issues concerning the under and overprotection of human subjects, especially in healthcare settings where research has traditionally and formally been distinguished from treatment. Overprotective research ethics protocols can stall progress. Underprotection of human subjects exposes patients to significant risk without oversight.

The challenge faced by the LHS is to develop policies and procedures that maintain respect for human subjects and, at the same time, do not unnecessarily encumber the aggregation of data that allow investigations that lead to quality improvement.

The Center’s 30-year history in working with healthcare providers and consumers uniquely positions it to:

   · Develop consensus and common definitions as to what constitutes “low risk, low-burden” research that may not require intense oversight;

   · Raise public awareness of patient and provider responsibility to participate in research with great potential to improve quality; and

   · Implement a communications strategy to engage providers and patients in a dialogue that results in their mutual understanding of roles they play in improving health outcomes and quality.

The Center has always served as an honest broker and convener in contentious cutting-edge bioethics issues and regional health care initiatives and stands poised and ready to help communities meet the obligations of Learning Healthcare Systems.

Learn more at

Full Article

This entry was posted in Health Care and tagged , , . Posted by Practical Bioethics. Bookmark the permalink.


Medical Interpreters and Patient Communications

Medical interpreters help patients, doctors communicate Kirsti Marohn and Stephanie Dickrell USA Today December 9, 2012Health care regulations require medical providers who receive federal funding to provide interpreters. There's al...

Full Article


A Book on Pain 2013

A 2011 Report on Pain by the Institute of Medicine indicates some 100 million Americans suffer from chronic pain.Author Judy Foreman will publish a book on pain in 2013 and talks about it in this edition of The Bioethics Channel with Lorell LaBoub...

Full Article


Science/Spirituality/Good Mix?

David O. Wiebers

Washington Post

December 6, 2012

Throughout my scientific training and subsequent career, I found myself consistently gravitating not only to the science and medical sections of bookstores but also to the metaphysical, New Age, self-help and spiritual sections of such stores in search of answers to many of the ongoing mysteries of science and to many of life’s most profound and perplexing questions.

Link to Podcast: The Legacy of Father Kevin O'Rourke, Sister Rosemary Flanigan and Bill Colby, JD, The Bioethics Channel, May 9, 2012

Full Article


Medical interpreters

Medical interpreters help patients, doctors communicateKirsti Marohn and Stephanie Dickrell USA TodayDecember 9, 2012Link to Podcast: The Ethics of Cultural Competency, Michael Brannigan, PhD, The Bioethics Channel, May 4, 2012

Full Article