Kuvan (phenoptin) is a newly approved drug for the treatment of phenylketonuria (PKU). PKU has been screened for and treated through diet in the United States for the past 40 years. Without treatment, PKU results in mental retardation. With treatment, intelligence is normal. Kuvan is the only drug approved for treating PKU.... and it costs approximately $180,000 per year per person. Before you start bashing BioMarin, the company that makes Kuvan, consider that they had to invest millions of dollars to develop a drug that will be used by very few people. Thus, the cost for each person has to be high in order for the company to stay in the business of developing and producing new drugs.
But there is a major problem with Kuvan, and it's not the cost. The problem is that the benefit it provides is MINIMAL.
Currently PKU diets consists of fruits and vegetables, low protein foods, and a special phenylalanine (phe) deficient metabolic formula. The problem with this diet is that the main source of nutrition, the formula, tastes terrible. Imagine having to drink a product that tastes worse than baby formula every day as you main source of nutrition. In addition, PKU diets have no meats (fish, etc.) and very restricted amounts of bread, pasta, etc. (which contain protein).
Kuvan increases the amount of protein a person with PKU can eat. The problem with Kuvan is that the increase is minimal.
A person with PKU on Kuvan still has to drink the metabolic formula, they still can not eat meat. They are able to eat regular bread or pasta instead of low protein bread or pasta. (Low protein bread and pasta actually taste similar to regular bread and pasta.) A person with PKU can also eat more of some foods, such as french fries. (Yes, french fries contain protein and are restricted for people with PKU.)
So let's review the situation:
An adult with PKU is started on Kuvan. This costs the insurance company $180,000/year. The person with PKU can now eat regular pasta instead of low protein pasta and can have a large order of french fries instead of a small order of french fries with dinner.
Let me repeat that:
$180,000/year so the person can have a large order of french fries (or other equivalent amount of protein) with dinner. He must still drink the poor tasting metabolic formula. (Which, by the way, costs the insurance company $30,000/year.)
This illustrates a common problem in medicine today. The situation where the cost of treatment is very high and the benefit very low.
I would venture to say that most of us wouldn't pay anything close to that amount of money annually to be able to eat a large order of fries with dinner instead of a small order. Yet, we expect our insurance company to pay. And where does the insurance company get the money? From us directly or from us indirectly through our employer. (or as economist say... TANSTAAFL).