NYULMC: Compassionate Use Could Impact Long-Term Medical Benefits

A new working group at the NYU Langone Medical Center has issued preliminary findings from their studies on the research ethics of compassionate use. Among their findings include:

  • Biotechnology companies have no legal or regulatory obligation to provide access to unapproved treatments on the grounds of compassionate use. Some companies allow access under the guidance of well thought out policies; some companies decline to allow access; some companies grant access but have no set guidelines; and some companies change their practices midstream as a result of public pressure. This lack of uniform policy is confusing to those seeking unapproved treatments.
  • Contrary to widespread perception, the U.S. Food and Drug Administration (FDA) is not an obstacle to those seeking compassionate use. In fact, the FDA almost always defers to the company that is developing the unapproved treatment to decide whether to grant compassionate use acces.
  • The “human impulse” to help patients facing insurmountable odds motivates both the general public’s support for compassionate use and so-called “right to try” laws to help gain access to unapproved treatments. However, increasing access to unapproved therapies may prove detrimental in the long run to longstanding and effective research and clinical trial systems through which interventions are proven effective and safe, and given regulatory approval.

You can learn more about the working group and read more of their findings here.

Art Caplan: Is It Fair That Americans Received the Ebola Treatment?

Art Caplan has a new video on Medscape laying out the principles behind rationing limited supplies of experimental ebola treatments. As he explains:

I believe the answer to the question of who should receive the drug is: people we can both learn from and potentially help the most. I believe those are the 2 values we use when trying to ration access to an experimental drug. If we do not learn whether something is safe and effective, then we have missed an opportunity, even in the middle of an epidemic, to find out whether it is worth giving out drugs that are new, untested, and unapproved. People who should be included are those who can be observed and kept under surveillance — not for a day or a week but probably for months and years. That favors people who are not in rural villages. That favors people who will have access to hospital facilities. Those criteria will drive the selection of who receives a new, unapproved drug.

Click here to see the video and read more.

Art Caplan: WHO Ethics Committee on Ebola Just a Start

Art Caplan has a series of new opinion pieces out on the WHO ethics advisory committee meeting that approved the use of experimental drugs to treat patients ill with Ebola.

He suggests deeper exploration of issues of informed consent, corporate responsibility, and resource allocation in this blog post for The Health Care Blog. As he writes in his piece in NBC News Health:

It is important that the WHO committee affirmed the morality of compassionate use. This addresses the concern that any use of unapproved drugs is inherently exploitative. But there are huge ethical issues that still remain unaddressed and unanswered regarding experimental interventions.

In the wake of the Canadian government’s offering 1,000 doses of an experimental Ebola vaccine to the stricken nations, he also extends the argument from allocation of treatment to allocation of prophylaxis in this opinion piece in NBC News Health:

It is ethically appropriate in the midst of a deadly contagious epidemic to try both untested treatments and experimental preventative vaccines that have shown some promise in animals and no safety issues. But with only 1,000 doses of vaccine available, who should get them? And what do they need to be told?

The most ethical way to distribute limited experimental vaccine, is, as the WHO ethics group noted, with an eye toward collecting information on safety and efficacy. Rather than just handing out vaccine to a small group of people in countries that have seen Ebola outbreaks, it is important to learn as much as possible about whether the vaccine has any efficacy in humans and is safe.

You can read more at the links above.

Art Caplan: Ebola Treatment Distribution is Troubling

Amidst news from Spain that a 75-year-old Catholic priest has received the experimental treatment ZMapp for Ebola, Art Caplan critiques what he describes as the “bad science” behind choosing its recipients:

ZMapp is not the answer to the Ebola epidemic ravaging West Africa. There is no chance of getting a significant amount of this drug made for many months. Deploying more health care workers, face guards, moon suits, gloves and antiseptic, along with restrictions on travel and burying the dead, is the only way to get the epidemic under control. [...]

The fact that a 75-year-old has been given the scarce drug is especially disturbing, not because he is 75 but because 75-year-olds do not have strong immune systems — something very important in battling a virus like Ebola. Moreover 75-year-olds often have other medical problems that complicate the ability of scientists to figure out if the drug is safe and if it is really working.

In testing unapproved, highly risky drugs like ZMapp, it is crucial that recipients not be so sick that they may well die regardless of whether they get the drug or not. Indeed, the recipients ought not be very sick so that side-effects can be seen and efficacy determined. To do that, doctors need to be able to monitor experimental subjects for months to make sure the drug does not damage their livers or cause any other fatal side-effect. So not every person infected with Ebola makes for the best recipient — younger, those more recently infected and those who can be closely monitored are among the “best” candidates.

You can read more of Art Caplan’s perspective on NBC News Health here.

Art Caplan: Why do two white Americans get the Ebola serum while hundreds of Africans die?

As the WHO announced today that medical ethicists will convene next week in New York to discuss the use of experimental medicines in the West African Ebola outbreak, Art Caplan has a timely new opinion piece in the Washington Post asking why only white American victims of the Ebola outbreak have been treated with an experimental serum. Caplan argues that the decision was a question of economics:

The reasons for different treatment are partly about logistics, partly about economics and, partly about a lack of any standard policy for giving out untested drugs in emergencies. Before this outbreak, ZMapp had only been tested on monkeys. Mapp, the tiny, San Diego based pharmaceutical company that makes the drug stated two years ago: “When administered one hour after infection [with Ebola], all animals survived…Two-thirds of the animals were protected even when the treatment, known as Zmapp, was administered 48 hours after infection.”

But privileged humans were always going to be the first ones to try it. ZMapp requires a lot of refrigeration and careful handling, plus close monitoring by experienced doctors and scientists—better to try it at a big urban hospital than in rural West Africa, where no such infrastructure exists. [...]

But it’s about more than logistics. Drugs based on monoclonal antibodies usually cost a lot—at least tens of thousands of dollars. This is obviously far more than poor people in poor nations can afford to pay; and a tiny company won’t enthusiastically give away its small supply of drug for free. It is likely that if they were going to donate drugs, it would be to people who would command a lot of press attention and, thus, investors and government money for further research—which is to say, not to poor Liberians, Nigerians or Guineans. [...]

To get Caplan’s full perspective, read the full article.

Art Caplan: The Real Reasons for Worrying About Ebola

Art Caplan has a new opinion piece on NBC News responding to the recent media coverage of Ebola. He makes the case that although this has been the worst recorded outbreak of the disease, citizens of developed countries have little reason to panic:

Ebola is not going to run amok in downtown Boston, Cape May or Myrtle Beach or anywhere else in the U.S. It is running amok in poor African nations because local authorities did not have the will or the resources to respond quickly, because no one confronted local funeral customs that expose people to Ebola, mainly because the world did not care much if hundreds died in poor, politically insignificant nations.

The harsh ethical truth is the Ebola epidemic happened because few people in the wealthy nations of the world cared enough to do anything about it.

Read the full article.

Art Caplan: Everyday Ethics

Art CaplanBill of Health Contributor Art Caplan and ethicist Kelly McBride have launched a new twice-weekly podcast, Everyday Ethics, in which they explore the moral dimensions of our everyday lives. Recent topics have included conflicts of interest on Dr. Oz, recent “Right to Try” laws, and Jenny McCarthy’s anti-vaccination campaigns.

You can access the podcast online here, or subscribe to it on iTunes.

Art Caplan New Op-Ed: When Does Human Life Begin?

Art Caplan has a new Op-Ed on The Council for Secular Humanism about when human life begin.

From the piece:

“When does human life begin? For those in the “personhood” movement in the United States, there is no doubt about when that happens—it is at conception, when the sperm meets the egg. The personhood movement has gained a foothold among antiabortion activists who are looking to pass laws that define embryos as people with full rights. Personhood advocates aim to outlaw all abortions, along with in vitro fertilization, embryonic stem-cell research, and emergency contraception. Granting embryos personhood would also mean that someone who killed a pregnant woman at any stage in her pregnancy would be at risk of prosecution for a double homicide. And in those states that restrict a woman’s right to utilize a living will if she is pregnant, no living will could apply from the moment of conception..”

Read the full article.

Art Caplan: Facebook Experiment Used Silicon Valley Trickery

Art Caplan has a new opinion piece on NBCNews about a recently published study in The Proceedings of the National Academy of Sciences, where a  Facebook scientist teamed up with two academics to subtly tweak the news feeds of nearly 700,000 Facebook users.

From the piece:

“The question of whether or not an experiment is ethical hinges upon the question of “informed consent.” Generally, this means that a subject in a study needs to have basic information about the study he’s participating in, understand the nature of the experiment and its risks and benefits, and have the ability to withhold his consent without fear of harm or retribution.

The authors of the study argue that they obtained subject consent: Their manipulation of Facebook users’ emotions was “… consistent with Facebook’s Data Use Policy, to which all users agree prior to creating an account on Facebook, constituting informed consent for this research.” This is nonsense; it’s not informed consent. It is an old Silicon Valley trick for systematically eliminating the legal rights of its customers.”

Read the full article.

Art Caplan Says Vasectomy Has No Place in Plea Deal

Art Caplan has a new opinion piece on NBCNews on the controversy over the case of Jessie Herald, in which he was offered a plea bargain that involved sterilization for a reduced sentencing. From the piece:

Jessie Lee Herald was facing five years or more in prison after a crash in which police and prosecutors said his 3-year-old son was bloodied but not seriously hurt. But Herald cut a deal. Or more accurately, the state agreed to reduce his sentence if he would agree to be cut. Shenandoah County assistant prosecutor Ilona White said she offered Herald, 27, of Edinburg, Virginia, the opportunity to get a drastically reduced sentence if he would agree to a vasectomy. It may not be immediately clear what a vasectomy has to do with driving dangerously and recklessly. It shouldn’t be. There is no connection.

Read the full article.

Caplan on Teens and Vaccines

Art Caplan, and his coauthor Dorit Rubinstein Reiss, have a guest column over at Daily Kos on the NY bill that would allow adolescents to receive treatment – including preventive treatment like vaccines for HPV – against a sexually transmitted disease without their parents’ or guardians’ knowledge or consent. Take a look.

Chip and Fish: Inadvertent Spies

Art Caplan has authored a new opinion piece on Bioethics.net on the issue of “chipping” human beings. From the piece:

There has been a great deal of fingerpointing, second-guessing and recrimination over the decision by the President to exchange five former Taliban leaders for the American soldier, Bowe Bergdahl.  “You’ve just released five extremely dangerous people, who in my opinion … will rejoin the battlefield,” Senator Marco Rubio, R-Fla., and likely Presidential candidate told Fox News.  Senator John McCain, R-AZ, told ABC news and many other outlets that he would never have supported the swap if he’d known exactly which prisoners would be exchanged given their former high roles in battling the U.S. in Afghanistan.

Put aside for a second whether the five Taliban leaders that were flown to Qatar for Bergdahl are now too old and too long removed from Taliban affairs to resume anything close to their old roles.  Presume, instead, they will eagerly resume where they left off prior to their capture, attacking Americans and others they see as hindering Taliban goals for Afghanistan.  Is it possible that the U.S. did something to these men before letting them go in the swap—surreptitiously implanting them with microchips so that they could be tracked or traced?

Read the full article.

Art Caplan: “Was Sarah Murnaghan Treated Justly?”

Art Caplan has coauthored a new opinion piece in the journal Pediatrics on the controversy over the case of Sarah Murnaghan, in which a federal judge ordered the Secretary of Health to place a pediatric patient on the adult transplant list to increase her chances of receiving a donor organ. From the piece:

Lung transplantation is a potentially life-saving procedure for patients with irreversible lung failure. Five-year survival rates after lung transplantation are >50% for children and young adults. But there are not enough lungs to save everyone who could benefit. In 2005, the United Network for Organ Sharing developed a scoring system to prioritize patients for transplantation. That system considered transplant urgency as well as time on the waiting list and the likelihood that the patient would benefit from the transplant. At the time, there were so few pediatric lung transplants that the data that were used to develop the Lung Allocation Score were inadequate to analyze and prioritize children, so they were left out of the Lung Allocation Score system. In 2013, the family of a 10-year-old challenged this system, claiming that it was unjust to children. In the article, we asked experts in health policy, bioethics, and transplantation to discuss the issues in the Murnaghan case.

Read the full article.

Art Caplan on ‘Right to Try’ Law: More Cruel Than Compassionate

Art Caplan has a new opinion piece up at NBC News on Colorado’s new “right to try” law, which “expands access to experimental medications, making it legal for the terminally ill to seek access to drugs that have not yet received FDA approval.” From the article:

Colorado has become the first state to enact a so-called “right to try” law that expands access to experimental medications, making it legal for the terminally ill to seek access to drugs that have not yet received FDA approval. Many others are considering doing the same thing.

Politicians know a surefire winning issue when they see one. Sounds like a great idea? The truth is, such laws are utterly inadequate for helping the dying and the desperate. They would better be described as “right to beg” laws — a right which, sadly, the terminally ill already have and already use in social media campaigns.

Read the full article.

Art Caplan on handshakes in medical settings

Art Caplan has a new opinion piece up at NBC News on the suggestion, in a recent JAMA article, that handshakes should be banned in healthcare settings. From the article:

Now, the handshake ban might make sense if it were not for the fact that the constant touching of microbe-laden things by providers and patients is likely to go on and, handshake or not, they are still likely to fist bump, shoulder pat, rub noses or whatever else they think shows love, care and concern for one another.

Health care has gotten very sterile and impersonal as more technology appears, less time is set aside for talking and more health providers find themselves chained to their computers or handheld medical devices. While not every culture values a handshake, many do, and putting the kibosh on grip and grinning just adds to the perception that caring and curing are heading down different highways.

Read the full article.

Art Caplan: Condoms Should Be Encouraged, Not Used as Evidence

Art Caplan has a new opinion piece up at NBC News on the increasing use of condoms as evidence of a crime by police in the United States. From the article:

Why do we shoot ourselves in the foot when it comes to public health? The latest example of what not to do in the war against disease comes from the world of sex: All over the United States, police and prosecutors are discouraging safer sex by using the possession of condoms as evidence of a crime.

For decades, police officers have been regularly confiscating condoms from people they believe are engaged in prostitution to either justify an arrest or to use as evidence at trial. District Attorneys routinely mention the number of condoms a person had on them when arrested to help cinch prostitution charges.

Prostitutes are far more likely to be infected with HIV and other sexually transmitted diseases than are the general population. That is why health departments spend a lot of money distributing condoms and trying to convince sex workers to use them.

Read the full article.

Art Caplan on “Pediatric Euthanasia in Belgium: Disturbing Developments”

Art Caplan has coauthored a new piece in JAMA on problems with Belgium’s new law allowing terminally-ill children and their families to choose euthanasia. From the article:

The Belgian pediatric euthanasia law seeks to respect the moral status of children as agents who possess the nascent capacity for self-determination. Specifically, the law requires the medical team to demonstrate a patient has the “capacity for discernment,” indicating that he or she understands the consequences of a choice for euthanasia.

What the law does not consider, however, is that adults choose euthanasia for reasons that go beyond pain. For adults, the decision to end their life can be based upon the fear of a loss of control, not wanting to burden others, or the desire not to spend their final days of life fully sedated. These desires might be supported by the experience they have had witnessing a loved one express a loss of dignity or because they understand what terminal sedation is and wish to refuse it. Children, however, lack the intellectual capacity to develop a sophisticated preference against palliative interventions of last resort. Instead, in the case of the new Belgian law, children seem to be asked to choose between unbearable suffering on the one hand and death on the other.

This possibility causes the Belgian euthanasia law to fall short of the standard required for valid assent. The criterion related to the “capacity for discernment” runs the risk of ignoring the fact that children and adolescents lack the experiential knowledge and sense of self that adults often invoke—rightly or wrongly—at the end of their lives.

Read the full article.

It’s Doctors’ Duty to Promote Gun Safety With Patients

Art Caplan has a new opinion piece up at Medscape: “It’s Doctors’ Duty to Promote Gun Safety With Patients.” From the article:

There is a huge problem with guns in the United States, and there are many things that public health can do, and many things that doctors should do to try to minimize the threats that guns pose to children, families, and all of us. One of the leading problems with guns is that we don’t educate children to know what to do when they find a gun or see a gun. So, if a 5- or 6-year-old sees a gun, we need a program that teaches that child that if you see a gun, walk away, get out of the area, and then tell an adult. That is a public health program. It’s something that school nurses could do. It’s something that pediatricians should talk about with their child patients. It’s something that families should hear about. We need a campaign. Vivek Murthy, the Surgeon General candidate, thinks that it is an appropriate thing to do. Apparently, the NRA doesn’t.

You can watch or read the full piece on the Medscape website (access requires a login and password, but registration is free).

Art Caplan: #SaveJosh? Maybe, but What about the Rest?

Art Caplan has a new opinion piece up at MSNBC on the #SaveJosh social campaign that is seeking to get a young cancer patient access to an experimental drug under compassionate use policies. From the article:

If Josh were 67 instead of 7, he would already be out of luck. Those who are not very cute get less attention in their pursuit of unproven drugs. If Josh had parents who did not understand how to use social media, he would already be out of luck. If Josh did not have sharp, well-connected doctors, he would already be out of luck. But he is not in any of these categories, so he may yet get the drug.

All of which is to say, this is no way to handle requests from desperate patients, parents or families to try to save themselves or their loved ones from imminent death by giving them access to unproven, experimental drugs. We need an equitable compassionate use policy for everyone in this country.

Read the full article.

Caplan: Three-Parent Babies Are an Ethical Choice

Art Caplan has a new op-ed out on the three-parent baby issue.  Here’s an excerpt:

In my view, trying the technique to fix a terrible disease even with risks of failure makes ethical sense. The FDA may ask for more studies in monkeys, but that really wont settle the safety issue in humans. Given the severity of mitochondrial diseases it is worth trying the technique.

The big worry is not so much safety, but where will allowing this form of genetic engineering lead. If we let doctors try to repair defective eggs today, who is to say they won’t be trying to make superbabies or designer babies tomorrow by transferring other genes into eggs?

The answer to that is that how far we go in engineering future generations through genetic manipulations is up to us. We can enact laws and treaties that say yes to gene therapies but no to cosmetic genetic engineering. Holding families hostage by saying they cannot try to repair broken genes to treat diseases because we worry that we cannot put steps or handrails on the slippery slope to designer babies seems wrong to me.

Take a look here.