It looks like the FDA is going to announce their decision regarding approval of Cinryze, the C1 Inhibitor that I have been receiving as part of a drug trial for treatment of my Hereditary Angio Edema (HAE), on the 14th of October, 2008.
Since the committee who advises the FDA on such matters voted 100% to recommend approval, it is highly unlikely that the FDA will make a decision against their recommendation. This means that Cinryze will be commercially available in the United States about 4-6 weeks afterwards (but I won’t be surprised if that lags into the New Year).
As part of their approval, it is anticipated that the FDA is going to require that patients be required to receive this treatment from an approved practitioner. It was hoped that, although this treatment must be injected intravenously, patients with medi-ports or capable family members would be allowed to do home infusions. The decision to require patients to go to a medical facility for this treatment presents several concerns for HAE sufferers:
1. Immediate Availability: For those sufferers, such as myself, who (without Cinryze) suffer nearly constant attacks of HAE, immediately availability of Cinryze is of utmost concern. Having to transport a patient to a medical facility for treatment can be life threatening. Further, if practitioners are not available 24/7 (as in my case, where my physician is only available during office hours and has no backup while on vacation), treatment is not just not timely, it isn’t even available.
2. Financial Imposition: Again, for sufferers like myself who need infusions every 2-3 days, the cost of office visits (even if paying only a co-pay of $15) which were covered by the drug company now become the burden of the patient. For people on fixed incomes (like –you guessed it– me!), this can become a deal breaker. For people without insurance, the deal is broken before it even gets on the table.
3. Personal Inconvenience: It takes about 15 minutes to do an infusion (5 minutes to reconstitute the blood product and 10 minutes to do the infusion). It takes about an hour and a half to get a doctor’s office to do it, and you spend all the time in excess of the 10 minutes the doctor sits there with you slowly depressing the syringe plunger just waiting — usual doctor’s office bull****. No one who had to give up their 9-to-5 job because of HAE-related absence is now going to be able to jump back into the job market if they are spending a great deal of their time sitting in their doctor’s office.
This is all in addition to the cost of the treatment itself. When I was purchasing C1 Inhibitor for my personal consumption and bringing it in from abroad, it cost $800/vial. I need 2 vials every 2-3 days. That’s approximately $16,000/$24,000 a month! And that’s what it costs in Europe, where drugs are generally cheaper. The drug company is going to want to recoup all of its research costs and I have no idea how much Cinryze is going to cost on the US market. I have good insurance and I hope to hell that I’ll be able to buy this under the “mail order” program, where it will only cost either $35 for a 3 month supply or at the most $35 per month. But if they insist that I foot 25% of the cost, I’ll be out of the race. This is the ONLY treatment in the WORLD that works for me and if I don’t have access or can’t afford it, I won’t survive. Period.
I’m very excited that this treatment is going to be made available to everyone who needs it in the United States…finally.
I’m very afraid of what is going to happen to me when it is. Will I be able to afford it? Will I be able to get it when I need it? Will my Grandmother die before I can have it at home, to give to myself and take with me wherever I want to go?
I needed to tell you, friends, about this mess, because I am trying very hard not to freakout. Too much stress is very bad for anyone, but stress in even small amounts can be deadly for people with HAE; it just aggravates our condition. I don’t think I’m handling my fears about this upcoming transition very well; it’s probably why I can’t seem to get my feet under me for any substantial length of time. Sometimes my life just seems to suck.