Rare Disease Awareness Day Entry #2: Orphan Drugs, Non-traditional Treatment, and the FDA (or, Why Healthcare is still Terrible)

Two (of many) drugs. Two stories. One really long post.

These are just two of my medications. The one on the left costs about $105 each month; the one on the right is up to about $125 each month, both including standard shipping. That is over ONE QUARTER of my total income - disability and social security - each month.

Before toilet paper, gas for the car, cleaning products, utilities - these need to be purchased or I will be nearly as sick as I was back when I had to live in a camper in my mother's driveway, bedridden.

Many (healthy) people assumed that the health care acts during the Obama years would make life so much better; everyone would have coverage, meds would be so much cheaper, happy utopia. For eight years I and many other chronically ill Americans knew that that simply would not be the case. Yes, there were absolutely improvements that so many people benefitted from - myself included toward the end there, and I continue to benefit - but healthy people as a whole were very quick to pat themselves on their backs for fighting for the poor, sick folks for eight years without seeing that there was still a very broken system in existence. The job was and is unfathomly big and is nowhere near done. Folk with pre-existing conditions cannot be denied coverage anymore, but those that are not disabled still have to pay way more than the healthy college student or adult does - and, at one point, were fined if we didn't have coverage. That's hard on a budget of a chronically ill patient, which is often significantly smaller than that of a healthy person due to their ability to perform while ill. I actually paid the fine one year because it was a lot cheaper than paying for the plan I was eligible for that wouldn't cover my specialist and some of my meds anyway. Medicare and Medicaid - the health care options for the disabled - are legally not accepted everywhere. Forward Health was a good step, but the fundamentals of a system based on averages and profit are untouched. I cannot even begin to discuss the current administration here. I do not have the answers. I write this part to try to let you, who is most likely at least healthy enough to work, know that many things are still quite broken for the chronically ill - and even more difficult for some with rare diseases. You need to understand and accept this for the rest of this entry to make sense.

Where getting care and meds is clearly still difficult for the chronically ill it can be even more of a struggle for those with rare diseases. I'm going to use the two medications pictured here to explain why it can be so much different for the latter group.

The first reason is the matter of something called "orphan drugs". The medication in the blue bottle is called ketotifen, and it is prescribed by my allergist and mast cell specialist and an approved, common treatment for the condition I have been diagnosed with. Ketotifen is a mast cell stabilizer that works in the intestine; it essentially tells the hyperactive mast cells in the intestine (rather than the ones everywhere else in the body) to STFU for about four hours so the individual can eat and digest without reaction. It is and has been freely available all over the world for treatment of mast cell disorders like MCAS or mastocytosis (a different, but much better-documented disorder, equally rare) for many years. It is not, however, available freely in the US; it is not FDA-approved, which ostensibly means that the US has not legally deemed it safe or theraputic for any known disease. As a result, no insurance will cover it, least of all disability insurance which is super-picky anyway.

FDA approval is a years-long, multi-phasic process that involves drug trials and data analysis and risk prediction. It is a good and necessary thing to have, don't get me wrong - that's why we don't have cocaine in children's cough syrup anymore. The problem comes when popularity and profit influence the process.

The issue of popularity is honestly a logical one. If the FDA has two applications for two different drugs - one that can help 50 million people and one that can help 50 thousand people - it really does make sense to pour more energy into helping 1/6th of the American population than it does to help 1/6000th. Medicine, like all sciences, is an art of averages and maximized utility. It's cold, but it's true. Equally cold (and probably far less excusable) is the fact that a drug with far more financial support or expected profitability will make it through the process faster than a drug for a disease that doesn't have as much American research (read: money) behind it yet. The FDA is like any other business or university; they need grants or other financial means to fund their research. The groups in charge of research of more well-known diseases are going to be able to provide more money faster for research. It's not all BIG PHARMA LOL; it is simply a function of a system based on helping as many people as quickly as possible. Look at it this way: if your favorite movie star wears that red or pink ribbon on that awards program and thanks their teacher that died from AIDS or their mother with breast cancer you are FAR more likely to donate to that cause than to a group researching a disorder that you've not heard of or cannot understand - especially one without an official ribbon like MCAS (they're working on it. Deep purple seems to be pulling ahead).

...so when there is a drug that will not benefit a large number of people AND doesn't have the current funding/future profitability, you get this problem of an orphaned drug that's kinda kicked around in the proverbial alley for years, slowly gathering studies while the other, richer drugs pass them by on their way to US approval. It's not a big deal to the vast majority of Americans, but for those of us who rely on, say, ketotifen, to literally eat, it is a very big deal indeed. I do know of people that cross borders to get it. Mine is made legally through a formulary in Oregon (the cheapest spot in the country) and sent to me via UPS. It is the medication we are currently changing the dose of; cost goes up by about $25 every time I'm increased. Despite the fact it is prescribed by my doctors and a legal, recognized treatment for the disorder I am diagnosed with I'll never have it covered by any insurance until it is FDA-approved. I actually do not know it's current status; I should remedy that.

The white bottle is a little different. It is a supplement, and it represents the often necessary step of using non-traditional substances for treatment of rare, not well-understood disorders. Supplements, of course, are seldom covered by anyone anyway (and especially not by the plans available to the disabled). This particular product is used to control histamine sensitivity - a condition where the body either releases too much or cannot metabolise extra histamine in the system. It is NOT a true allergic reaction in itself, but it definitely keeps more histamine around for someone with allergies like myself to react to. The supplement contains an enzyme that breaks down all that extra histamine. It is prescribed by my allergist and mast cell specialist and is discussed in the literature, although as more of a periphery than a mainstay like the ketotifen tends to be. As mentioned, it will not be covered due to it's status as a supplement. Why is it so expensive?

There is only one company in the country that offers this kind of supplement. They are based out of Michigan, and we're lucky to have them. Others have come and gone, mainly because supplements are a very big money game - more so, I'd say, than prescription drugs, at least in veracity. This is to say that if you have a large enough population that believes that your product helps them then sales will be OK. If your population is limited to those with one rare disease and you can't seem to diversify... well, someone at corporate is gonna shut that product down eventually. That's free market. There is no morality or rightness behind these decisions - and it is not at all likely that most people behind this or other products can really grasp how crucial the product is for some with rare diseases that need a cocktail of prescriptions and non-traditional means to keep their conditions under control. This is fully legal because, remember, supplements are not considered medically necessary or beneficial.

...so there you go: $125 because one drug just isn't big enough to hang with the cool kids and $105 because of a balance that needs to be struck between demand and production costs. $230 each month for two medications prescribed by my specialists for a diagnosed (rare) disorder that, as of now, have no coverage on the horizon. I cannot count these as medical expenses to get extra SNAP benefits as others can because - you guessed it - they aren't FDA-approved, and those SNAP benefits only go so far. My med expenses are not limited to this, of course, and I do take several medications each day. Eight of those are for MCAS, and of those only two are covered (and thank God they are; the other mast cell stabilizer I'm on is about $1300 cash price). The other six are comprised of the two I've discussed here and four OTC drugs that I've tried and failed to get coverage for because dissbility coverage is strict. All are prescribed by the same doctors for the same disorder and are acquired legally. That doesn't include the meds for my other, non-rare health issues.

That was a tirade. What do I want you to come away with? Medicating and care of a rare disease is different from even a more common chronic illness and MUCH different from the care most of those that read this can receive. The system can fail us in different ways, and it's hard to teach people that because rare disease affects or touches so few people outside of the odd interest piece online that's only there for the freak show thrill. I suppose that that's the overall point of this awareness day - and I hope that I've made you a bit more aware of one of the messier, scarier parts of a rare disease that we just don't tend to talk about.