It has been a remarkable summer of milestones and crises for high-technology medicine.
An FDA panel has unanimously approved a gene therapy : The patient’s own immune cells are taken from his or her body, genetically modified, and reinfused to attack cancer. While the treatment has some dangers, it can be worth trying when conventional therapy has failed, and it appears to be curative when it works. Final FDA approval is expected in September.
Scientists have also announced the first use of gene therapy to effectively treat a human embryo . They successfully replaced a defective gene with an engineered correction. The in vitro embryos were not implanted.
While those breakthroughs were occurring, the parents of Charlie Gard , an infant in England with a very rare and devastating mitochondrial disease, were seeking experimental therapy for their child. The medical staff disagreed with the parents: They recommended that the best thing for Charlie would be to stop the ventilator and allow him to die, rather than let him to continue to suffer. Three British courts reviewed Charlie’s case and concurred with the medical staff; on appeal, the European Court of Human Rights also denied the parents’ wishes.
End of life cases similar to Charlie’s are not rare. In modern medicine, parents sometimes must make the heart-wrenching decision to stop aggressive therapies and accept that death is imminent and unavoidable. Many factors go into making that decision. Both the courts and medical staff presume that parents are the best decision makers. Generally, medical staff provide emotional and spiritual support to the parents, along with a tincture of time. In the vast majority of cases, parents and physicians come to agree on the course of care, but sometimes, there are irreconcilable disagreements.
It is rare for courts to overrule parents. The government typically intervenes only when the harm from a parent’s choice exceeds some threshold. For instance, it is not in a child’s best interest to be put in a car during a blizzard and driven to the store to get cigarettes. But neither is it wise to have an intrusive government reviewing every choice a parent makes. The potential harm must be large enough, likely enough, and imminent enough before most judges will intervene. The law will insist the child be in a car seat at least.
In Charlie’s case, the medical staff and the judges all explicitly said that the cost of therapy did not factor into their decision making; they looked solely at what was best for Charlie. The focus was on whether the unproven potential benefits of experimental therapy outweighed the risk of suffering caused by the therapy and continued intensive medical care.
Even when a bedside decision ignores the financial impact, money often structures which therapeutic choices are available. There are also issues of equitable access to be raised and weighed. Expenditures impact other social choices.
Money influenced the actions of Martin Shkreli, who is best known as the pharmacy company executive who markedly increased the price of a drug. Mr. Shkreli was recently convicted on three of eight charges for securities fraud, and sentencing is pending; the convictions were not related to the price increase.
Money also appears to have played a key role in the tragic deaths of more than 30 infants in northern India , who died when the hospital’s oxygen tanks went empty. The company responsible for refilling the oxygen tanks didn’t do so because, it claimed, the hospital wasn’t paying its bills. Public outrage has the government investigating the situation.
The United States has created some amazing technologies to save individual, identifiable lives, but they come at a high price that often costs lives in ways more subtle than the incident in India. At some point, the government and the public are responsible for either financing or rationing care, but that doesn’t absolve the scientists completely. The Russell-Einstein (Pugwash) Manifesto established that scientists have a moral accountability for the negative consequences of creating new technology, and that includes the financial aspects.
Dr. Powell is a pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at pdnews@frontlinemedcom.com
