Monday, 2 November 2015

Drugs, Mucus and Money

Cystic fibrosis (“CF”) causes thick, sticky mucus to build up in the airways and people with CF work very hard each day to clear the mucus from their airways using a variety of inhaled medications and physiotherapy techniques. None of these treatments fixes the problem they just buy you time and space for bits of life.

CF mucus is sticky stuff, hard to shift. It doesn't come up by itself, it's not like saliva, more like glue, it grips whatever it touches, it can take several flushes to get it down the toilet. It is nasty stuff, you think to yourself “surely this must have industrial applications or maybe it could be used as a form of renewable energy/biomass. Perhaps I could sell it!”, then crackles and gurgles in your airways demand an end to the day dream and it’s back to work, it's like wrestling with a sticky bear. This work takes energy, patience and skill (deliberate breathing, huffing, coughing techniques), it soaks up energy, it causes headache and nausea (especially if you have the signature CF bloated stomach. You have to be tuned in, listening/feeling for sounds (crackles, wheezes) vibrations to give you clues and direct your efforts, you have to proactively hydrate your airways “from above” with inhaled therapies like hypertonic saline and “from below” by drinking lots of water, just to give yourself a chance. 

This is never ending, your work is never finished - there is always more gunk down there, crackling away and trying to sop you breathing. So when you stop for a rest you feel like you should be doing more and you start to feel guilty. It can feel pointless - like Sisyphus trying to push the rock up the hill. 

You can’t meet others with CF because there is a real risk of cross-infection so you have to get on with it by yourself. You used to be able to meet others with CF because nobody knew about the risks then but it is different now and most of them have passed away now anyway. It can be a lonely battle. Hours of treatments and clearing 50-100 ml of mucus each day, even when well, with no chance of a day off, can be gruelling. It can feel like a cruel form of punishment.

If mucus is crackling and gurgling away in your airways, it is impossible to sleep. So even though you are exhausted you can't sleep. You have to do more work just to try to clear a bit so you can have a chance of a little sleep. But as you sleep, it builds up again because you are not clearing! And after a while you're too exhausted to do all your treatments, or eat but if you don't do that you will get worse. You are exhausted to work but you get no real help from the government because you can walk 50m and “look healthy” so then you have no money to support yourself or buy food or pay the rent or get to the hospital. This is normal life, it is worse when you are unwell. It is like this everyday, 7 days a week, 365 days a year.

Eventually you will drown in your own mucus unless you get a lung transplant. And you know this from the start. And there is nothing you can do to prevent it, you can only delay it. Unless someone develops a cure.

And then a company called Vertex comes along with a pill which is not a cure but which looks like it might actually start to address the underlying problem (like 10% of a cure) but they want $300,000 per patient per year for this pill and there is no way you will get it because the money is not there to pay for it. And the company says if they don't charge such a high price, they won't be able to afford to do more research to finish the job. But nobody really knows how much it costs to develop these pills. It's a secret. We all know it is risky business and most drugs fail; and that the winners have to pay for the losers. It’s like you can't point at a lottery winner and say they shouldn't get the jackpot because they only paid $1 for the ticket! Obviously you have to think about all the losing tickets too. Understood. But that shouldn’t mean they can charge whatever they like. They say they it costs the same to develop a drug for 1,000 people as it does to develop a drug for 1 million people. So in a rare disease like CF they have to charge more per patient to recover this big inflexible, mysterious lump of cost. They say the price also reflects the "transformational" effects for patients (i.e. how much like a cure it is). But then they charge basically the same for a drug which is like 10% of a cure with a market of 30,000 people as the earlier one they made for 2,000 people which is like 40% of a cure for those people. So the logic doesn't hold. And the executives who work for the company who are not the people who did the science and developed the pill) get paid tens or even hundreds of millions of dollars a year in salary, bonus and equity and this tells us the company has a lot of excess money so it makes even less sense. It just looks like they are charging what they can get away with. Because there aren't very many people with CF and we have no political clout, nobody with power cares and nothing changes. Then the mucus starts gurgling away and it's back to reality, time to try to clear it out again. Because you like being alive and that means you need to be able to breathe.