I have written in the past about the growing number of $1 million plus drugs in health care. Some are once-in-a-lifetime gene therapies, while others have to be repeated periodically. It is a good news / bad news situation depending on where you fall in the equation. Obamacare banned all annual and lifetime caps on benefits. The good news: that created a powerful incentive for drug makers to search for treatments for rare disorders. The bad news: that created a powerful incentive for drug makers to charge multimillion fees for treatments.
The New England Journal of Medicine published a report on a gene therapy used to treat an infant with a rare genetic disorder. The account was reported in New York Times:
He had a rare genetic disorder, CPS1 deficiency, that affects just one in 1.3 million babies. If he survived, he would have severe mental and developmental delays and would eventually need a liver transplant. But half of all babies with the disorder die in the first week of life.
The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation.
This is a happy story about a baby saved from death and disability caused by developmental delays. This type of technology could really transform health care, but at a huge cost. The baby was an inpatient in the hospital for nine months to control rising ammonia levels due to the genetic defect. The lead researchers collaborated with others to develop the technique that would be used to edit the precise gene that was lacking or malfunctioning. The race to develop the custom gene editing therapy was a herculean effort involving teams from numerous institutions.
To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be changed.
Not discussed was the cost for the treatment, or likely future costs once gene editing like this becomes commonplace.
The implications of the treatment go far beyond treating KJ, said Dr. Peter Marks, who was the Food and Drug Administration official overseeing gene-therapy regulation until he recently resigned over disagreements with Robert F. Kennedy Jr., the secretary of health and human services. More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need.
Thirty million people represents about 9% of the population of the United States. A chronic condition affecting 9% of the population would ordinarily be a lucrative target for drug makers, except for the fact that there are 7,000 target genetic diseases. If genetic diseases were distributed evenly across all 30 million patients (which of course they are not), that would mean less than 4,300 patients for any given genetic disease. What happens when the researchers in this article begin creating $1 million to $5 million therapies for 30 million people? Expect the cost to reach $30 trillion to $150 trillion for 9% of the population.
Read more at New York Times: Baby Is Healed With World’s First Personalized Gene-Editing Treatment