Thread by @VincentRK

1/ During my Grand Rounds talk @MayoClinic on cost of drugs I had a question on #LEMS drug #Firdapse that was recently priced at a mindboggling price of $350,000 per year by @CatalystPharma - I answered it. What happened next was very very powerful and moving. Read on 👇

2/ Unknown to me, a patient with #LEMS was in the audience. She raised her hand and spoke up. She had received the drug almost for free from Jacobus Pharma for many years. But now because of @CatalystPharma this program was done. She had to buy Firdapse: at $3800/month co pay! 👇

3/ She had written to several Congressmen with no answers. She said she was on Medicare Part D and cannot afford the co pays. Her symptoms from this disease were well controlled for years. But not she must go without meds and suffer. There was no choice coz of @CatalystPharma 👇

4/ Some background -that will make most of you upset. First, #LEMS Lambert Eaton Myasthenic Syndrome is a serious muscle disease first described @MayoClinic by neurologists Lambert & Eaton. Second, the drug for treatment 3,4 DAP was given away for almost free by Jacobus Pharma👇

5/ @CatalystPharma makes a tiny modification to the drug, runs 2 small trials, gets @US_FDA approval, calls it Firdapse & sets the price at $350,000 per year (or $375,000 - does it really matter?). Jacobus was no longer permitted to send their drug to patients. 👇

6/ This story illustrates that Pharma is the #1 root cause of the cost of drugs problem. This price was not based on innovation. Not due to middlemen. You decide what it’s due to. But we have a serious problem. Its really sad to see patients suffer. @aholdenj @DavidP4AD @mtmdphd

I also don’t like stupid policies that as soon as there is an “FDA” approved drug all previous compassionate use programs must end. If some company wants to help patients why should the @US_FDA or any other agency prevent this. Change the regulations please!! cc: @peterbachmd

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