This week’s news includes the FDA approval for two treatments for Sickle Cell Anemia.
This is wonderful news, and a result of years of government-financed research that has provided tools for correcting genetic abnormalities.
Some background: Sickle Cell anemia is a genetic disease in which an error in the DNA coding for hemoglobin (the protein that allows red blood cells in your blood to carry oxygen). This error also results in a change to the normal shape of the red blood cells from round to “sickle” shape. Those sickle cells are stiffer, sometimes resulting in them getting stuck in blood vessels and causing strokes, and more brittle, meaning that the life span of those cells is shorter than normal and because they die off faster and are not replaced quickly enough, there is a shortage of those cells. A shortage of red blood cells is called “anemia”.
SCA has many corollary issues including, multiple yearly trips to the ER, many days of work lost, lower self-esteem, depression, and poor nutrition. The sum of these issues results in lower economic success, lower home ownership and decreased earnings. The net result is that those with SCA are far less able to handle expenses associated with the disease.
The cause of the disease itself is particularly interesting.
As you may remember from previous posts, the DNA in your cells codes for proteins. In the string of the four building blocks of the DNA (bases A, T, C and G), each sequential trio of bases (a “codon”) codes for one amino acid. When the cell builds a protein from the mRNA, it uses that codon instruction sheet to construct the protein, one amino acid at a time.
In Sickle Cell Anemia, there is an error in the DNA coding for the hemoglobin molecule. It occurs in the seventh codon and is a substitution of a “T” base for an “A” base, resulting in a change in that codon from GAG to GTG. This single change modifies the instruction sheet for Hemoglobin to substitute a Valine amino acid at the seventh point in construction for what should have been a Glutamic Acid amino acid.
That’s it! That one “point mutation” in the DNA results in a faulty hemoglobin molecule that modifies the structure of the red blood cell and causes all the resulting problems of the individuals who suffer from this painful, life-threatening disease. If both parents carry this mutation, the resulting child will have the disease.
Now, two separate companies have developed techniques, approved by the FDA for treating people with SCA. The techniques are not simple, are extremely expensive (between $2-4 million per patient), and utilize extensive assets in hospitals.
The basic concept of the treatment is to modify the DNA in the stem cells of the bone marrow that are the precursors to the development of red blood cells.
First, remove the stem cells from the bone marrow of a patient.
Second, modify the DNA in those stem cells in a laboratory.
Third, kill all the remaining stem cells in the bone marrow of the patient.
Fourth, repopulate the bone marrow of the patient with the new, modified stem cells.
100,000 Americans suffer from this disease.
What should we do?
If we authorize treatment for all 100,000, and IF the hospitals can find beds, support staffs and medical supervision to treat them all so that they can lead normal lives, the costs will total between $200-400 Billion and the ongoing costs would be $20-40 Billion per year to treat the 10,000 babies born with the disease in the US each year.
How should we pay for this, or how can we triage treatment to a large fraction of these individuals?
If we want to pay for it, then each individual in this country would have to pay between $1,300 and $2,500, either in a single tax, or by increasing their healthcare insurance policy, and then the 150 million policies issued in the US would have to increase by increase of $150-300 per year to cover the new cases each year.
If we make the co-pay even 10%, then the out-of-pocket cost for each SCA patient would be between $200,000 and $400,000. This would put it out of range for almost all the patients that need to be covered.
As I have pointed out in the past, who is the “black hat” here? Is it the companies who created the solution? Is it the government that cannot afford the costs? Is it the public that can’t afford the increases in their insurance coverage? Is it the patient who could actually lead a normal life? Is it the attorneys that will sue to provide those patients with the care that allows them to lead a normal life?
The truth here, I believe, is that the cost of medical care keeps increasing. Research leads to advances. Advances lead to new treatments. New treatments allow patients to live longer. Living longer results in higher long-term health care costs.
The only way to control those costs is to nationalize the health care system so that it does not rely on private, for-profit companies to decide who gets care and what care you can receive. For-profit hospitals, for-profit providers (doctors, clinics, nurses, and health care groups) make more money when they perform more tasks.
It remains true that the government is neither a good administrator nor a cost-effective organization; however, until the profit motive is removed from those who deliver health care, these “problems” will continue to create inflation in our healthcare delivery system.