Gaucher's Tags a Facebook Friend

A few weeks ago I signed up for a Facebook account, not really knowing what to expect. In a very short time I found a world that was, and still is, somewhat foreign to me.

I have a Twitter account but so far have not seen the value in reading about what someone had for breakfast or knowing that their dog pooped on the carpet.

I thought Facebook would be pretty much the same except with pictures and I really didn’t want to go there. But on a lark I signed on and connected not only with current friends and business clients but also with folks I have not seen or communicated with in 40 years. Hard to believe it has been that long since high school.

Some of the stories are fun, some are interesting, and some are quite touching. I found out through a good friend I have known since elementary school that several of our mutual friends had died in the last few years. That was quite a shock.

But I have also connected with someone that was always a friend but in a sisterly kind of way. She reached out to me in a way that was totally unexpected.

In reviewing my profile she decided to click through to my business website and read a bit about my work. Her first correspondence was very intriguing to me. She revealed she has two daughters who are now adult’s living with Gaucher’s disease.

I was only casually familiar with the illness but something in her note raised my awareness even more. She said the medication required to keep her daughter’s stable runs $250,000 per year for each of them.

That is astounding even to me. I have clients with various types of cancer and a few with RA and MS. Their medication runs anywhere from $2,000 to $5,000 per month. Certainly expensive but not even close to $250,000 per year.

When clients want to purchase major medical coverage the only thing I really push is full Rx coverage. Sadly, there are quite a few plans out there with no Rx coverage or annual limits as low as $2,000.

Most folks feel like they will never get sick and need expensive medication. As a whole, we are really out of touch with how much medical treatment can cost. That is caused by having plans with a copay for practically everything.

The line I hear most often is, “I don’t plan on getting sick”.

How silly is that? No one PLANS on getting sick any more than planning an accident.

In the case of Gaucher’s, it is an inherited disease. For children to contract Gaucher’s both parents must be carriers. Due to the rarity of the illness (1 in 40,000 births) it is not something normally tested for unless there is a history somewhere in the family line.

One of the meds used to treat Gaucher’s is Cerezyme. Not only is the drug expensive but there have been recalls of the drug due to contamination. After a 6 month void the girls were finally able to get a new dosage this week. Without the drug, symptoms of the disease start to manifest. The drug does not cure but will alleviate some of the effects of the illness.

Illnesses that only affect a small percentage of the population are sometimes classified as orphan illnesses. The research that goes into finding a treatment is time consuming and expensive, often running in the billions of dollars and taking years.

Some will argue that Genzyme, the company that developed and manufactures Cerezyme, is becoming wealthy on the backs of those who are desperate due to health. The profit margin on Cerezyme is reported to be 30% which some view as excessive.

To a Gaucher’s patient, having access to the medication is a difference in quality of life.

The Orphan Drug Act was signed into law in 1983 under Ronald Reagan. Under the law, companies that develop drugs for orphan diseases are granted special tax incentives and exclusive marketing rights for several years. It should be noted that not all research pans out and some of the drugs developed never hit the market.

In the 10 years prior to 1983 less than 10 drugs for orphan illnesses hit the market. Since passage of the ODA, almost 2,000 drugs have been given orphan status and of those roughly 340 actually reached the market under exclusive arrangements.

No doubt, without this law many illnesses would go untreated and many would die.

If you want to argue against protectionism and profits then you must accept the reality that many, perhaps even someone you know, could be adversely affected without proper treatment. In the case of Cerezyme, if the manufacturer was prohibited from making a profit you need to consider the cost would still exceed $150,000 per year per patient.

Of course without the profit incentive and tax breaks, the drug most likely would never have been developed.

You can’t have it both ways.

Comments

  1. Nancy Masters says:

    Thanks Bob!
    What a nice article – great information and personally touching!
    Keep up the good work!

  2. I am forever thankful that Genzyme took the leap into incredibly rare drugs and created Cerezyme. Unlike cancer and leukemia and even autism, the amount of money needed to research these diseases that affect so few are so hard to come by. All children should have a chance at life.

    Mom to Hannah, 17 months old with neuronopathic Gaucher’s disease

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