Tag: placebos

Bioethics News

Novel system to get dying patients an experimental cancer drug raises hopes — and thorny questions

The drug was still experimental, but clinical trials suggested it could be a lifesaver for patients with a lethal form of blood cancer called multiple myeloma.

And those patients were clamoring to get it. They overwhelmed drug maker Janssen Pharmaceuticals with requests for the medication.

Most companies don’t know how to handle such requests. Often, it’s the richest patients, or the best connected, or those who run the most compelling social media campaigns who end up getting the drug. Everyone else is out of luck.

Janssen’s parent company, Johnson & Johnson, decided on a unique approach: Rather than try to deal with the barrage of requests itself, it asked a leading bioethicist to create an independent committee to determine which desperate patients could get access to the limited supplies of its experimental drug, known as daratumumab.

A year and a half later, the company and patient advocates deem the process a success — and the bioethicist, Arthur Caplan, is looking to replicate it with other drugs, starting perhaps with a psychiatric therapy.

But the novel system has also raised some thorny questions.

One ethicist who strongly supports the concept in general nonetheless asks whether it is truly wise to eliminate all personal lobbying and decide who gets access to drugs based only on anonymous medical records. What if someone like scientist Stephen Hawking is in the mix, he asked: Shouldn’t he get priority access to a lifesaving drug, for the good of humanity?

“Imagine if he wasn’t saved, what a difference in the world there might have been,” said Dr.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Research Ethics Roundup: New Clinical Trial Transparency Tool, Returning Genetic Data, Genetically Modified Animals in Research, and Placebos as Treatment

This week’s Research Ethics Roundup examines the new online transparency tool from AllTrials, returning data to participants in genetic research, concerns about overuse of genetically modified animals, and the potential for placebos as treatments for non-urgent pain.

The post Research Ethics Roundup: New Clinical Trial Transparency Tool, Returning Genetic Data, Genetically Modified Animals in Research, and Placebos as Treatment appeared first on Ampersand.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

Is a Placebo a Sham If You Know It’s a Fake And It Still Works?

October 28, 2016

(NPR) – So placebos have pretty much been tossed in the “garbage pail” of clinical practice, says Ted Kaptchuk, director of the Program for Placebo Studies and the Therapeutic Encounter at Beth Israel Deaconess Medical Center. In an attempt to make them more useful, he has been studying whether people might see a benefit from a placebo even if they knew it was a placebo, with no active ingredients. An earlier study found that so-called “open-label” or “honest” placebos improved symptoms among people with irritable bowel syndrome. And Kaptchuk and his colleagues found the same effect among people with garden-variety lower back pain, the most common kind of pain reported by American adults.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Prescribing the Placebo Effect

By Sarika Sachdeva
This post was written as part of a class assignment from students who took a neuroethics course with Dr. Rommelfanger in Paris of Summer 2016. 

Sarika Sachdeva is an undergraduate junior at Emory studying Neuroscience and Behavioral Biology and Economics. She is involved with research on stimulant abuse and addiction under Dr. Leonard Howell at Yerkes National Primate Research Center. 
In 2006, Dr. Ted Kaptchuk designed a clinical drug trial to evaluate a new pain pill in patients with severe arm pain. Participants in the study were assigned to receive either the pill or an acupuncture treatment for several weeks. Dr. Kaptchuk found that the people who received acupuncture ended up with more pain relief than those who had taken the pain pill. This difference was surprising, not because the pain pill was expected to be more effective, but because neither treatment was real- the pain pills contained cornstarch and the acupuncture was done with false needles that never pierced the skin.

Placebos are often considered baseline measurements, used as the standard scientific method to determine if a drug is actually making a biological difference or if its effects are just ‘inside the head’ and no better than a sugar pill (Anderson 2013). Utilizing the placebo effect as a form of treatment carries a stigma: only 0.3% of physicians admit to regularly prescribing them, in contrast with data that indicates around 50% of physicians actually do (Rommelfanger 2013).
Image courtesy of Pixabay
Recently, however, there has been a growing body of evidence that placebos produce real physiological changes, making them an active treatment not unlike ibuprofen, aspirin, or other traditional pharmaceuticals.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Placebo as Therapy: Context, Ethics, and Recommendations

By Somnath Das
This post was written as part of a class assignment from students who took a neuroethics course with Dr. Rommelfanger in Paris of Summer 2016.

I am a Senior at Emory University and am currently pursuing a double major in Neuroscience and Chemistry. Currently, I am applying to medical school. My interest in healthcare lies primarily in understanding the behavioral motivations of patients as they navigate through various healthcare systems. I also wish to study how to effectively translate innovations powered by biomedical research into accurate health information for patients and optimized healthcare delivery. Neuroethics allows me to focus these interests onto patient dignity and rights when considering the role novel therapeutics and interventions in treatment. Studying this fascinating field has given me a perspective on the role deontological considerations play in both neuroscience and medicine as a whole. It is with this perspective that I hope to approach my patients with a balanced worldview, taking into account both individual rights as well as stakeholders and developers participating in a rapidly changing field. 
Placebo therapy is broadly characterized as the administration of an agent that possesses a physiologically inert effect. However, current research suggests that placebo in fact has observable therapeutic outcomes across a wide spectrum of disorders. Thus, placebo’s efficacy should be investigated thoroughly by researchers, ethicists, and physicians in order to evaluate and develop protocols to implement placebo therapy in an effective manner. It is necessary that researchers communicate to physicians and clinicians about the efficacy and rigor by which research has quantified placebo’s effect.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Babies and unnecessary pain in research

A Reuters news service article this week reported that many babies are subjected to excessive or unnecessary pain in the course of medical research.  The article provided a link to an online report from the journal Acta Paediatrica.

The authors of the article in question (freely available through the link above) summarized their review of 46 recent (previous 2 ½ years) clinical trials testing analgesic treatments for pain caused by medical procedures performed on neonates—babies in the first month of life.  As anyone who has visited the doctor knows, medical procedures can cause pain, sometimes more, sometimes less.

The article’s authors found that a majority of the studies used a concurrent control group that did not receive any analgesia, or placebo, to compare with an experimental analgesic drug or method.  The problem with that is that there are numerous standard pharmacologic and non-pharmacologic analgesic methods that may be used for neonates and older infants.  By denying those to some subjects, the investigators did a disservice to these neonates, the authors argue.  Their argument is entirely reasonable.  They further find fault with ethics committees that approve such research, journals that publish the results, and parents of the infants, who provided consent either out of ignorance or for some other unknown reason.  All of this should stop, they argue, in favor or research designs in which all subjects receive at least standard-of-care analgesia.

This looks like an easy one: a fundamental criterion for ethical research on human subjects is that risks be minimized to the extent possible and reasonable, and consistent with good scientific and medical practice. 

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Targeting Cystic Fibrosis: Are Two Drugs Better than One?

Caption: Doctor with a child with cystic fibrosis who is taking part in clinical research studies. Credit: Colorado Clinical and Translational Sciences Institute

To explain the many challenges involved in turning scientific discoveries into treatments and cures, I often say, “Research is not a 100-yard dash, it’s a marathon.” Perhaps there is no better example of this than cystic fibrosis (CF). Back in 1989, I co-led the team that identified the cystic fibrosis transmembrane conductance regulator (CFTR) genethe gene responsible for this life-shortening, inherited disease that affects some 70,000 people worldwide [1]. Yet, it has taken more than 25 years of additional basic, translational, and clinical research to reach the point where we are today: seeing the emergence of precise combination drug therapy that may help about half of all people with CF.

CF is a recessive diseasethat is, affected individuals have a misspelling of both copies of CFTR, one inherited from each parent; the parents are asymptomatic carriers. The first major advance in designer drug treatment for CF came in 2012, when the Food and Drug Administration (FDA) approved ivacaftor (Kalydeco™), the first drug to target specifically CF’s underlying molecular cause [2]. Exciting news, but the rub was that ivacaftor was expected to help only about 4 percent of CF patients—those who carry a copy of the relatively rare G551D mutation (that means a normal glycine at position 551 in the 1480 amino acid protein has been changed to aspartic acid) in CFTR. What could be done for the roughly 50 percent of CF patients who carry two copies of the far more common F508del mutation (that means a phenylalanine at position 508 is missing)?

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

What’s Wrong With Giving Treatments That Don’t Work: A Social Epistemological Argument.

Let us suppose we have a treatment and we want to find out if it works. Call this treatment drug X. While we have observational data that it works—that is, patients say it works or, that it appears to work given certain tests—observational data can be misleading. As Edzard Ernst writes:

Whenever a patient or a group of patients receive a medical treatment and subsequently experience improvements, we automatically assume that the improvement was caused by the intervention. This logical fallacy can be very misleading […] Of course, it could be the treatment—but there are many other possibilities as well.

So we decide to hold a Randomised Control Trial (RCT). An RCT takes account for the non-specific effects of the treatment—these misleading possibilities Ernst is so interested in. The RCT has three arms to the experiment: the first arm receives the active treatment (in our case, drug X); the second arm receives a placebo, a treatment with mimics the drug X except in that it is devoid of the active ingredients (in our case, it’ll be a sugar pill); the third arm receives no treatment whatsoever. Following the RCT, we’ll know: first, whether the treatment is doing anything at all; and second, whether or not it is the active elements of the treatment that are doing the work.

The results of our RCT say that the first and second arms of the treatment are effective (that is, those patients receiving drug X and the sugar pill); however, there doesn’t appear to be any difference between those patients receiving drug X and those receiving the sugar pill.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Hype, Money and Stem Cells

Michael Hiltzik of the Los Angeles Times used newly published research by Kalina Kamenova and Timothy Caulfield [abstract] to write a scathing article largely focused on the California Institute for Regenerative Medicine (CIRM):

New study: Stem cell field is infected with hype

This conclusion is scarcely novel, but both the academic analysis and the polemical journalistic commentary are welcome. The researchers examined “the portrayal of translational stem cell research in major daily newspapers in Canada, the United States, and the United Kingdom between 2010 and 2013” and found that optimistic perspectives outnumbered pessimistic ones by more than five to one, with less than a third of the reports being neutral.

Kamenova and Caulfield conclude by essentially pinning the responsibility on scientists for “authoritative statements … regarding unrealistic timelines.” Hiltzik, on the other hand, “wouldn’t give journalists this much of a pass,” noting their complicity in exaggerating promises of breakthroughs and cures.

In fact, there is plenty of criticism to spread around.

There are also long-term effects of stem-cell hype. Many people are still predisposed to believe hucksters — and why wouldn’t they be? Weren’t we promised cures a decade ago?

Blog comments reveal that some people now think that the pharmaceutical multinationals are in cahoots with the FDA to prevent treatments from becoming available. (The discussion at this post, in which Paul Knoepfler explains the need for regulation, is one of the better threads.) It is therefore hardly surprising that desperate patients are willing to travel to Mexico, or the Philippines, or the Bahamas, or elsewhere, and to spend thousands of dollars on what are almost certainly placebos at best.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.