Promoting vaccination with a not-too-heavy hand

This week’s Nature has a worthwhile read, “Mandate Vaccination with Care.”    The recent rise in the number of cases of measles is well-documented in the general press, and there is a strong argument that it is a social good that sufficient numbers of children be vaccinated for a range of infectious diseases.  Your correspondent considers it unfortunate, to put it mildly, that there is a persistent belief that vaccines for the standard childhood diseases are harmful.  Although some cases of vaccine harm occur, they are rare—rarer than many in the general public believe—and the cost of under-vaccination is great.  I, for one, never want to see an infant with pertussis (whooping cough) again, and, although I recall having had measles and chicken pox when I was a kid, it’s best to prevent them.  Some can even be eradicated (see: smallpox—which we should fervently hope is never purposely re-introduced, now that we don’t routinely vaccinate for it). 

In brief, the authors in this case argue for promoting vaccination in the public with such steps as ensuring supply and access, providing information and allowing public forums, monitoring safety carefully, and tracking vaccination rates.  They argue, reasonably, that mandatory vaccination that carries the wrong kind of penalties—such as, fines or even jail sentences imposed in some countries—for non-compliance actually can harm poorer, medically underserved people, and as such be counterproductive and, frankly, unfair.  They comment that harsh mandates can unnecessarily prompt a backlash, with increased resistance.

They say, further, that if mandates are deemed “politically appropriate,” then the procedures should be just, with constraints on choice as limited as possible; any penalties must be proportionate; those who do suffer complications should be adequately compensated.   They speak favorably of creating administrative hurdles to getting exemptions from mandates.  They also argue against governments mandating only some vaccines while excluding others.  They claim that making some vaccines only “recommended” can limit the uptake of all. 

This last point may be the most questionable of all in this article.  It is easier to justify mandating vaccination for highly contagious diseases that can have devastating effect (e.g. measles, rubella, diptheria, and others), than, for example, vaccination for human papilloma virus (HPV), infection with which predisposes to certain kinds of cancer but transmission of which is through sexual activity.  In this last case, the argument for a mandate is substantially weaker; vaccination at a fairly young age might be wise, but one might still reasonably accept, for oneself or one’s child, the less certain and more remote risks of the consequences of infection, and therefore reasonably object to mandated vaccination.

Again, a worthwhile read. >

Should Abortion be a Trivial Event?

A recent NEJM Perspective by Drs. Jane Henney and Helene Gayle is calling for reduction in the current restrictions on Mifepristone, previously better known as RU-486. Mifepristone, a progesterone blocker, which effectively opens the cervix of the uterus, when combined with misoprostol, which promotes contractions of the uterus, forms the two-pill combination marketed as Plan C – an effective, safe, self-administered home medical abortion. Arguing that the drug has been shown to be far safer than originally thought, and because many US women are accessing foreign internet sources of potentially suspect quality Mifepristone, Drs. Henney and Gayle are encouraging the FDA to make the drug available from regular US pharmacies rather than at special medical/surgical clinics from a restricted set of doctors as it is presently distributed.

At the time of its initial FDA approval in 2000, it was unknown whether mifepristone would have a high risk of excessive bleeding or incomplete abortions so it was approved under FDA Subsection H, which required that the drug be dispensed directly from a physician (rather than a pharmacy) who can provide blood transfusion or surgical abortion. Drs. Henney and Gayle argue that numerous studies since 2000 support that this level of restriction is unnecessary. This is not to say that the risks of Mifepristone are zero but rather, as one of their arguments goes, there are many other medications available from a pharmacy via prescription that have higher risks so why single out Mifepristone? They are not specifically calling for the medication to become “over-the-counter” (meaning available without prescription) but they list other countries where this is the case.

Their concern about the ease of obtaining Mifepristone (or Plan C) over the Internet is not exaggerated. A quick search of the Internet shows one advocate website called plancpills.org providing a report card where their own secret shoppers grade various Internet sites offering Mifepristone for sale. Another popular site called AidAccess.org offers an informative video, telemedicine consultation as well as foreign sourced Mifepristone and this site is presently one of the least expensive sources of the drug.

One of the statistics offered in support of abortion, especially medication-based abortion such as Plan C, is that these processes are safer for women from a morbidity and mortality standpoint than actually being pregnant, particularly the labor and delivery of birthing a baby. Of course, it (always) goes without saying that the same processes are nearly universally fatal for the baby. But, for arguments where the morbidity and mortality of the child are excluded, abortion, particularly medication-based abortion, has become a relatively safe process.

I have no doubt that the present restrictions by the FDA on access to Mifepristone will be reduced for the reasons Drs. Henney and Gayle have provided, perhaps eventually to point of making Plan C available over the counter.

Abortion is becoming a trivial event.

Abortion kills a human being.

Killing a human being should not become a trivial event.

Parental responsibility in childhood immunizations

Last week I wrote about the issue of parental responsibility and medical decision-making for children. We have good reasons for having parents be the primary decision makers for children who are not capable of making their own medical decisions. However, as I discussed in the last post, there are some situations in which parents make decisions that are not in the best interest of their child. When those decisions reach the point at which the child’s life is in danger, society clearly has a moral obligation to intervene to save the life of the child.

Parental refusal of recommended immunizations for their children raises similar issues but is more complex. We can clearly demonstrate that it is in a child’s best interest to receive at least most of the commonly recommended immunizations for children. Those immunizations help to prevent a child being affected by diseases that can be serious and have a very low risk for serious adverse effects. However, the chance of an individual child being affected by many of these diseases is relatively small and the situation is quite different from a child whose life is in immediate danger due to a parental refusal of treatment. Since the risk to an individual child is relatively small and there is some possible risk to the child from receiving the immunization, it is not clear that a parent who refuses immunizations for a child is being negligent in the same way as a parent who refuses treatment for a life-threatening illness. It can be argued that the best way to deal with this type of situation is education. Many times, parents refuse immunizations for their children because of misconceptions about the risks and a lack of understanding of the benefits of doing the immunizations. It can be difficult to counter widespread misconceptions, but physicians have the responsibility of trying to do that the best that we can. However, this is not the only issue involved in determining whether society has an obligation to intervene and mandate childhood immunizations when parents refuse.

Immunizations are unique in the way that they work. They help protect the individual who is immunized, but they also help protect the community as a whole when we can achieve sufficient levels of immunity to halt the spread of an infectious disease within the community. This is sometimes called herd immunity. We have an excellent example of how this works with the current measles outbreak in the United States. Measles happens to be one of the most easily transmittable infectious diseases that we know of. It also can be deadly in a small percentage of the people who acquire the disease. It is also primarily a disease of children and is transmitted by children. When about 97% of children have been immunized in a community the disease will not be transmitted through that community and those who have not been immunized or cannot be immunized are protected from acquiring the disease. This level of immunity was accomplished in the United States about 20 years ago and for some time the only cases of measles that were seen in the United States were cases that originated elsewhere. However, because of parents refusing to have their children immunized for measles in significant numbers in some parts of the country, herd immunity is no longer present and we are having outbreaks of measles this year that are more than we have seen in 20 years.

That raises a different question. Should we mandate immunization of children for diseases such as measles and override parental decision-making not solely for the benefit of the children whose parents refuse to have them immunized but for the common good of the community? There are some children and other individuals who cannot be immunized or for whom immunizations would not be effective who are put at increased risk when herd immunity is not achieved in the community. This makes the decision about whether to immunize a child not just a decision about what is best for that child but also a decision about what is best for the community. For Christian parent it makes the decision about whether to immunize a child a decision about love of neighbor. Immunizing one’s own child helps to protect the child, but it also helps to protect the most vulnerable in the community. If we truly love our neighbor, we should do that even if there is a small risk to our own child. The final question is whether we as a society should require children to be immunized for these diseases when a parent is not willing to authorize it out of either concern for the best interest of the child or concern for those who are vulnerable in the community.

Measles: When does Screening, Quarantining and/or Vaccination become Mandatory?

As this linked PBS NewsHour interview between Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, and Judy Woodruff reports, the number of new cases of measles in the US has reached nearly 700, which is the highest number of new cases since measles was supposedly eliminated in 2000. The video is short and provides a quick review of the current causes of the outbreak and suggested steps one can take to mitigate future personal and family risks of contracting the disease. The reason for this being the subject of this bioethics blog is to touch on the ethical tensions between personal autonomy and utilitarian public health calculations regarding the recent measles outbreak.

So what is the problem with getting measles in the first place? Measles is a highly contagious viral respiratory disease. Simply being in the same room with someone with the disease can lead to becoming infected. In its milder form, the disease results in fever, runny nose, ear infection and a classic spotted red skin rash. In its more severe form, it can cause a severe pneumonia requiring hospitalization, deafness, lifelong brain damage and even death. Children under 5 years of age are at particular risk. A common two-stage vaccine called MMR is available that successfully immunizes 97% of those that receive it. It is given at age one and again around age 5.

The benefits of the immunization are two-fold. The first is direct personal protection against contracting the disease if you receive the vaccination and are one of the 97% of individuals who gain future immunity against the measles virus. The second is something called herd immunity. If enough people are immunized (experts estimate “enough” to be between 95-97% of the population), then even people who cannot be immunized, such as infants less than one year of age or individuals whose immune systems are compromised, are still somewhat protected from contracting the disease. This is because new measles cases from “outside the herd” are severely limited in their ability to spread to the small number of non-immunized people within the largely immunized herd. The immunized people effectively act as a physical barrier to protect the non-immunized people. Problem solved, right?

To quote my favorite ESPN College Gameday commentator coach Lee Corso: “Not so fast, my friend”. The measles vaccine is not completely risk-free. Minor side effects include fever, rash and local injection site infections. Much less common but more severe reactions include seizures and rare deaths from severe allergic reactions. In the late 1990s, the British medical journal Lancet published a study by Andrew Wakefield positing a link between the MMR vaccine and autism. This study was later proven to be a completely falsified claim and Wakefield was completely discredited, though some parents still use the original study to argue against vaccinating their children.

If the vaccine were completely risk-free, there would be no logical or ethical reason not to receive the vaccination. If everyone who could take the vaccine did so, herd immunity from a public health standpoint would be at its maximum, protecting the remainder of individuals unable to receive or benefit from the vaccine. The current measles outbreak argues that either we are not properly screening or quarantining new cases of measles at the point of entry to the US or our herd immunity may be breaking down (or some combination of the above).

So, at your next social function, after you have debated your usual political concerns or dismay at your favorite NFL football team’s shocking choice in the recent NFL draft, settle in to a potentially more meaningful discussion around the ethics of personal autonomy versus public health policy regarding mandatory measles screening, quarantining and vaccination. Suggested sub-topics might include:

• Is it fair for those who are able to vaccinate but choose not to vaccinate to freely rely on the herd immunity of those that do vaccinate?
• How public should one’s vaccination or immunity status be to avoid quarantine?
• What reasons are reasonable to choose not to immunize?
• Would it be fair to deny public (or private) insurance coverage for treating the measles if one chose not to take the vaccine?

Are pharmaceutical companies responsible for the opioid crisis?

Recently a major pharmaceutical company settled a lawsuit with the state of Oklahoma for $270 million. The state had alleged that the company’s marketing of OxyContin had helped to fuel the opioid epidemic in the state. Pharmaceutical companies in general do some things that are very good and have many times had some questionable practices. Some of their pricing and marketing practices are morally questionable, but it seems to me that it is the role of the FDA to evaluate those marketing practices and discipline pharmaceutical companies when they market inappropriately.

It does not seem to me that states suing pharmaceutical companies is an appropriate way to deal with the opioid crisis. The problem of what we used to call narcotic addiction has been around for centuries. It has been a problem long before any modern pharmaceutical companies existed. Whether the narcotic being abused was opium, morphine, heroin, or prescription pain pills the primary driver of narcotic addiction has always been hopelessness and despair. This is true whether it involved the opium dens in China or the slums of London, heroin addiction in the inner cities of the US or opioid abuse by the rural poor of states like Oklahoma or Indiana (where I practice). Supply plays a role in which narcotics are abused, but the underlying problem is a social and spiritual one.

There are many factors that go into the hopelessness and desire to escape that underlies narcotic addiction. One factor is economic. People who are unable to find work to support themselves and have no hope of being able to do so may turn to narcotics to escape. Those who are wounded by broken families and have no hope of being able to find the wholesome family relationships they desire frequently turn to alcohol and drug abuse. It would make as much sense to sue those who have contributed to these economic and social conditions as it would to sue pharmaceutical companies. Should states sue manufacturers who have yielded to economic pressures and have left empty factories scattered around our country while they profit from manufacturing goods overseas? Should they sue musicians who glorified drug abuse in their songs and modeled that in their behavior? Should they sue the entertainment industry that has promoted sexual immorality and the breakdown of families? Should they sue both state and federal legislators who have created a welfare system that promotes dependence and generational poverty?

I do not think that this is the answer. There are many things in our society that have helped to promote the increase in drug abuse that we are dealing with today. It will take all of us working together voluntarily to impact this crisis. Churches, businesses, physicians, hospitals, pharmaceutical companies, and government at the local, state, and federal level will all need to work together to help reduce the hopelessness and despair that underlies the current opioid epidemic. Research and treatment like what will be funded by the settlement of the Oklahoma lawsuit is needed, but working on the underlying problem of hopelessness and despair is essential. Local churches have the potential to impact that most effectively without needing to sue anyone.

Summarizing ethical issues with heritable human gene editing

By Jon Holmlund

A brief recap of reasons why we should not pursue heritable human gene editing:

It seems unlikely that risks to immediately-treated generations can be predicted with the accuracy we currently and reasonably expect from human subject research and medical practice.

Risks to later generations, that is, to the descendants of edited people, would be incalculable, and the informed consent of those later generations would be unobtainable.

To allow heritable gene editing even in the uncommon cases of untreatable, devastating genetic illness is to place too much faith in the ability of human providence to identify, and human behavior to observe, firm boundaries on its eventual use. 

Eventual use will become unavoidably subject to a eugenic approach in which the key decision will be what sort of people do we want, what sort of people should be allowed to receive life.

There will be no end to the disagreement over what edits should be permitted, and to the vilification of those considered to have been illegitimately edited, from those who object to their existence, perceived unfair advantage, or other characteristic.

Human populations will become stratified into the “edited” and the natural, introducing deep new justice concerns.  The main issue will be not will humans be gene-edited, but what should be the social status of those who are. 

To reduce heritable human gene editing to a reliable practice requires submitting it to the paradigm of manufacturing, as in drug development, with children seen as quality-controlled products of choice, not gifts of procreation.   To develop the practice, a “translational model,” again analogous to drug development, is necessary, with human embryos serving as raw materials, and, of necessity, a large, indeterminate number created and destroyed solely for development purposes, for the benefit of other humans yet to be born, and of those who would raise them.

Quite possibly, the translational model will demand great license on the extent to which embryos and fetuses may be experimented on; to wit, longer and longer gestations, followed by abortion of later and later stage, to further verify the success of the editing process.

In the extreme situation, the degree of editing may change the human organism in ways that will create a “successor” species to homo sapiens whose nature and desirability cannot be reasonably envisioned at this time.  In the extreme situation.

Even granting that this last scenario may never really arrive in ways that fiction writers can easily imagine, the other reasons should be enough that we simply don’t move heritable gene editing forward.

National Public Radio recently reported on the gene editing of human embryos—one day old—in the laboratory, in an attempt to correct and eliminate the inherited cause of blindness, retinitis pigmentosa.  The end is laudable.  The means is not.  We should not race ahead without considering why, first.  Then, we should not move ahead, but seek alternate means to the medical ends.

Edited embryos should not be created and brought to term—certainly not now, and I would say, not ever.  To be outraged over the former but not the laboratory creation of edited embryos is insufficient.  Both are outrages, although outrage over the recently-claimed birthing of edited babies in China is real, not “faux,” as one reaction held.  Still, the authors of that reaction are correct that one’s condemnation of the China event somehow justifies the laboratory work.  It does not. One last point: The Economist carried an essay decrying the birth of the edited twins in China as a case of “ethical dumping,” the practice of carrying out human subject research that would be disallowed in the West in other, perhaps less advanced (although China is certainly not backward), nations with fewer ethical scruples.  A valid point—but not one to cloak oneself in, while trying to justify the efforts to edit humans in ways that can be passed on from generation to generation.

Is More “Ruining” of Medicine on the Way?

By Mark McQuain

Ask older medical doctors their opinion on the current state of the practice of medicine and I suspect the majority will give you an earful, generally along the lines of “How [blank] has ruined the practice of medicine”, filing in [blank] with any number of things, including the government, insurance companies, pharmaceutical companies or doctors themselves. “Ruined” is a strong claim and even if true, I certainly don’t know how to assign blame as there is probably plenty to go around. Regardless, new initiatives by any one of these groups warrants watching. So, a recent September 20 NEJM editorial about a proposed change in reimbursement by the Centers for Medicare and Medicaid Services (CMS) made me wonder if more “ruining” is on the way.

The NEJM article nicely summarizes the current state of affairs (additional summary for those without subscription below):

“Medicare pays for office visits using five levels of codes based on clinical complexity, medical decision-making complexity, and time. For visits with established patients, physicians are currently paid $22, $45, $74, $109, and $148 for levels 1, 2, 3, 4, and 5 visits, respectively; for new patients, they receive $45, $76, $110, $167, and $172. This pricing structure in the Medicare Physician Fee Schedule, established by Congress in 1989, is the basis for physician payment by both public and private payers.”

CMS is proposing to collapse levels 2-5 reimbursements into a single payment of $93 for established patients and $135 for new patients. Documentation requirements would also be reduced to level 2 requirements thus arguably reducing some of administrative bureaucracy physicians say interferes with patient care, allowing them to spend more of this freed-up-time with patients. As an “older medical doctor”, I am certainly happy to reduce my administrative burdens so this sounds good. What could possibly go wrong with: “CMS is from the government and they are here to help”?

The authors of the NEJM article applaud CMS for their efforts to reduce administrative burden but then go on to list some potential unintended consequences. The biggest is that physicians lose the financial incentive to care for more complex patients. They hypothesize that this could result in some physicians reducing office visit times and bringing patients back more frequently, thus fragmenting the care of more complex problems and patients. They also worry that this payment policy will further maintain disparities between physicians who spend practice time on so-called cognitive evaluation and management issues versus time on the portions of their practice that receive reimbursement from procedures, imaging or laboratory fees. Lastly, if private payers don’t follow suit, the authors point out that physicians may shift further away from providing care for Medicare and Medicaid patients in favor of private insurance that does reimburse better for the complex problems.

Reimbursement is only one factor affecting today’s practice of medicine though certainly one that cannot be ignored. Many Christian physicians consider their practice of medicine as more of a calling than simply their occupation. I pray that external factors be kept from “ruining” that calling.

The Sad Case of Daniel Te’o-Nesheim

BY NEIL SKJOLDAL

The New York Times  recently published a lengthy article on Daniel Te’o-Nesheim, a former NFL player who died at age 30 after playing football for half his life.

With all the recent information about the dangers of football-related head trauma, it is not surprising to learn that upon his death, Te’o-Nesheim’s family donated his brain to the Boston University CTE center, where it was found to have chronic traumatic encephalopathy.  The Times article states that Te’o-Nesheim had suffered approximately 100 concussions.  His is another tragic story of someone who dies much too early, in part because CTE drove him to erratic behavior.

The Times article also details Te’o-Nesheim’s other football related injuries. According to his attorney, Sam Katz, an orthopedist told him that “he had one of the worst cases of degenerative arthritis in his ankles that he had ever seen.  He also found damaged knees and shoulders, pinched nerves in his neck, and a tendon tear in his biceps.”

What is particularly troubling about this situation is that a day after his death, the NFL denied his earlier claim for “line of duty” benefits, where his attorney had listed all of his ailments and injuries from playing football.  According to The Times, his claims were denied because“his injuries were not severe and numerous enough.”  His family contested the denial, and months later they were finally awarded $17,500 posthumously.

While denying people benefits the first time around might seem to be best practices within a ‘for-profit’ business model, it hardly seems like it addresses the much more important issue of promoting the health and wellness of the patient.  This seems especially true in this case, as a former employee who used the means available to him to ask for assistance for injuries that obviously were a result from his employment within the NFL.

It is cases like Te’o-Nesheim’s that makes me think that bioethics should speak clearly not only to those who provide frontline care, but also to the business side of the equation.

Reducing Abortion Regardless of Roe v. Wade

By Mark McQuain

The selection of the next Supreme Court Justice has perhaps naturally unleashed a flurry of op-eds describing the post-apocalyptic world that will result from any partial or complete reversal of Roe v. Wade. In the July 18th, 2018 Perspective in the NEJM, Dr. Julie Ingelfinger offers the tragic case of a foreign nursing student she befriended while both were training in New York in the late 1960s. The student was finishing her final nursing year and was engaged to be married when she became pregnant despite the use of contraceptives. Per Dr. Ingelfinger, neither the student nor fiancé had “the means to provide for a baby, so they reluctantly decided that terminating the pregnancy was the only choice.” The only abortion option available at that time, pre-Roe v. Wade, was a “back-alley abortion.” After the abortion, the student developed sepsis, resulting in a hysterectomy and kidney failure. Dr. Ingelfinger oversaw the dialysis and despite appropriate medical care, the student died suddenly from complications of the dialysis. Dr. Ingelfinger’s reason for sharing this story now is to remind us that back-alley abortions resulted in similar complications in many other young women pre-Roe v. Wade and warn that if Roe v. Wade is overturned in the future, young women seeking abortion will again suffer the same fate as her nursing student friend.

In a similar vein to Dr. Ingelfinger’s editorial, there is a second op-ed on CNN website on May 5, 2018 by Danielle Campoamor entitled “Why Supporting Abortion is a Pro-Life Position”. She fears any future restrictions in Roe v. Wade will result in the suffering or death of young women seeking an abortion and wants everyone to have the “safe, affordable and relatively easy abortion” that she experienced:

“I wasn’t subjected to mandatory waiting periods, forced counseling or an abortion provider required to regurgitate state-mandated, inaccurate information. I didn’t have to travel long distances, worry I was getting there too late in the pregnancy, find money to pay for child care or walk past angry or intrusive protesters. Instead, I went in pregnant and, a few hours later, came out with my future back in my control.”

In both articles, the focus is unilaterally on the health and life of the mother. Ms. Campoamor’s position is easily challenged, if not decimated, by including the health and life of the baby in her calculus. Dr. Ingelfinger’s premise requires more unpacking.

Her position appears to be that all future unwanted pregnancies in an overturned-Roe v. Wade world would require a pre-Roe v. Wade “back-alley” surgical abortion. Many Latin American countries have never legalized abortion yet their illegal abortion fatalities have dropped as medical abortifacients (morning after pills) have replaced surgical abortion methods. Interestingly, both the author of the previously linked article on the Latin American experience and Dr. Ingelfinger cited economics (and not legality) as a main reason for choosing abortion. Analysis of the statistics on why women in the US choose to abort challenges this assertion. A clear understanding of these statistics might help identify strategies that lead to a voluntary reduction in the number of abortions, absent changes in the legal status of abortion.

There is a nearly 15-fold increased risk to carry a baby to full-term than it is to have an elective abortion. We have “successfully” divorced sexual activity from the risk and responsibility of bearing and rearing a child, as long as we are willing to use abortion as the definitive stop gap in maintaining our birth control. From my standpoint, this success and this control has come at a terrible price, namely the deaths of over 60 million babies in the US alone. Sadly, I pessimistically do not believe that there will be a meaningful change in the Federal law regarding abortion, regardless of who becomes our next Supreme Court Justice (link requires subscription). There are simply too many women and men who have come to rely upon the type of control of their future activities that abortion provides. Therefore, I ask Dr. Ingelfinger, Ms. Campoamor and all of those on the other side of the abortion divide: must all unwanted pregnancies end in abortion (medical or surgical), regardless of the status of Roe v. Wade?

Mumbling orphans—a bit more

Mark McQuain has raised the persistent, vexing issue of the pricing of drugs for rare diseases—in the case at hand, Sarepta’s eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy, the disease over which the late comedian Jerry Lewis lost sleep every Labor Day weekend for years.

Mark provided an excellent summary (he calls it “crude,” but it’s anything but that).  In this case, the concern is not just price for a truly rare disease, but whether the drug showed sufficient evidence that it worked for FDA to approve it.  In the absence of alternative treatments, that was the truly tempestuous issue for Sarepta.  (Recall that under the 1962 Kefauver-Harris amendments to the Federal Food, Drug, and Cosmetic act, drug manufacturers in the U.S. may not sell a drug unless the FDA finds it not only safe, but effective—a standard that generally applies worldwide.)  It’s one thing for a drug to have a high price, but rather another if it doesn’t work, or doesn’t work very well.  (I decline to comment publicly about the Sarepta data; outside my expertise.  Those seeking a case in point may wish to consider Avastin for breast cancer.)

And to be sure the high price concern dogs other treatments that appear to work quite well—such as high-profile ones for cystic fibrosis or for cancer.  A case can be made that such drugs are worth the price, that too much government heavy-handedness risks stifling innovation, and that a search for the “just price” is misguided, but also, for sure, that society should share the costs of some of these drugs, that measures should be taken to limit out-of-pocket costs to disease sufferers, and that reimbursement approaches are ripe for overhaul.  In that last bucket: if drugs work only some of the time, only pay for the cases in which they do work; foster true competition (rather than having the costs of all drugs in a class go up when a new one is introduced, as if drugs were houses); eliminate the middle man (i.e., pharmacy-benefit managers that take a cut—that appears on the horizon); and the “biggie,” having government payers push back harder on prices.  At least some of these measures seem likely, and at least some seem warranted.

But overall, high costs for truly innovative treatments are justifiable, where no alternatives existed previously and especially when other, more expensive and quite possibly less effective medical treatments may be obviated (see: drug treatment for hepatitis C vs liver transplantation).  This is not to endorse price gouging for existent, cheap drugs that fall into an incidental monopoly (in which case, BTW, elimination of said monopoly, through regulatory facilitation of alternative sources, is warranted).