Thursday, September 4, 2014


That would be a very nice salary wouldn't it? I wouldn't mind it myself. But its not a salary. Its the cost of a new drug to treat Gaucher's Disease from Genzyme.

Now scrape yourself off the floor for a second. Gaucher's disease is fairly rare. It is a genetic condition where if both parents are carriers there is a good chance their child will either have it or be a carrier. As it is genetic, there is no cure. But $310K/year? That's crazy.

"The cost of Cerdelga is not unusual for rare disease drugs that qualify for tax credits and patent extensions under the federal Orphan Drug Act, which covers treatments for diseases afflicting fewer than 200,000 patients. Gaucher affects only about 2,000 people in the United States, some of whom are on medications from competing drug firms. It affects fewer than 10,000 people worldwide."...

Okay so they cannot expect to earn that much back as there is a very small potential market for it. But if they get 1000 patients on it, that would be $310,000,000 annually in income.... No small potatoes there.

"“Gaucher is among the rarest of the rare diseases,” said Genzyme spokeswoman Lori Gorski. “The health care system already has been taking care of people with Gaucher disease. This is not a new burden on the system. This gives patients and physicians the ability to choose the therapy that is best for their circumstances without consideration of the price.”"

So previous drugs were just as expensive. Wouldn't a benefit be to make a lower cost drug?

"“It poses a challenge because a lot of these drugs are really breakthroughs and they’re drugs you want patients to have access to,” said Brendan Buck, vice president for America’s Health Insurance Plans, a trade group. “But they’re coming with increasingly high price tags that could become unsustainable for health plans, Medicaid, Medicare, and private businesses.”"

Breakthrough drugs are great but great big price tags are not great.

"Gorski acknowledged the manufacturing cost of the Cerdelga pill is less than that of biotech drugs such as Cerezyme, which are produced through an organic process in giant vats called bioreactors. But she said other factors come into play in pricing a drug, such as development costs, the clinical value it brings to patients, and the rarity of the condition."

The clinical value it brings to the patients. Hmmm... How much the companies think a patient will pay. That's the key. I can understand if its treating a very rare condition making a small potential market as I mentioned above. But by taking into consideration the clinical value, aren't they putting a dollar value on the patient's life?

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