There are 1200 sufferers of CF in Ireland but they have a very loud collective shout. I guess it's the esprit de corps in the cohort: they are all young because there are no old people with CF; they have been nobbled by one or other of the many bowel obstructions or pneumonias which the gloopy CF mucus fails to discourage.
I've set out my stall on drugs to ameliorate the symptoms of cystic fibrosis CF. The two recently developed drugs Kalydeco and Orkambi are both ruinously expensive at €300K/yr and €140K/yr respectively: no normal family can afford them; so Vertex, the company which developed the drugs, is banking on insurance companies (US) or governments (in countries which put collective caring above individual enterprise) to foot the bill. The purse-strings will not be loosened unless some sort of cost-benefit analysis is carried out. A democracy is not going to stop people buying snake-oil if it makes them feel better - the placebo effect can be really powerful magic - but they won't accept mere assertions of Widow Twankie that her snake-oil cures boils, the pox or the dropsy.
In Ireland we have a bunch of knowledgeable and experienced people who work in the National Centre for Pharmacoeconomics NCPE. They are tasked to read the literature and do further statistical analysis as required to see if new drugs work well enough to justify the government paying for them. Kalydeco only works for a small group [N ~= 40 in Irelandof those who have a particular mutation that causes CF, and even then gives only a marginal increase in the quality of life. Reluctantly - nobody wants to deny children the chance of being fit and healthy - NCPE refused to allow the Health Service Executive HSE to pay for the drug. The then Minister of Health, James Reilly, over-ruled this evidence based decision by the governments own experts. tsk! that is a lot of money (my money, I pay taxes) which is now no longer available for other things, for other people who are not so articulate.
When I wrote about CF evidence-based economics in December 2016, the NCPE had refused to countenance paying for Orkambi. Although it is targetted at the deltaF508 mutation, which is the most common cause of CF in Ireland, and although there was a compelling rational drug design argument behind the drug's development, it only improved symptoms for about 3% of those who could possibly benefit. Those who did benefit, seemed to do really well under that therapeutic regime and were not above shouting their wellness from the rooftops. It made a very effective feel-good story in the media and these happy few got a lot of air-time. In the Spring of this year he Minister agreed to meet CF representatives to assure them that His People were negotiating hard with Vertex to get the best possible deal. Vertex were interested in helping people with CF, true dat: they had devoted hundreds of person-years in researching and developing the best available CF therapeutics. But they also owed their employees and shareholders and needed to recoup their R&D costs; so they were playing hard-ball too.
Then at the beginning of April Simon Harris the current Minister of Health caved in to get a positive political photo-opportunity struck a deal with Vertex over Orkambi. Cystic Fibrosis Ireland were happy although they had to big up the benefits "improved their lung function by a very dramatic 6.8 percentage points". Last time I used a peak-flow meter, my doctor asked me to try again because my puff was so woeful, but I don't think I'd notice a 7% increase in lung-function. I wouldn't pay €380 a day for it in any case. If I wanted to improve my QALY, I'd put it towards a mobility scooter so I could get out and about and meet [both] my pals. Minister Pot Reilly called Minster Kettle Harris black over this foolish subversion of a fair and agreed method for making the hard decisions in health care. Nobody denies that making decisions in modern health care is hard - a former incumbent called the Health portfolio "Angola" It's hard to say No to the CFistas but let's not shy away from the maths that by paying for CF drugs you-the-minister are saying No to people waiting for a hip-replacement or psychiatric help or a bed in a ward rather than another night on a trolley.
You can say that decision making is hard and give the responsibility to an objective protocol or you can say it's easy because you give in to the most articulate, the most pathetic or the most closely related to the Minister of Health. James Reilly qualified in medicine and spent 25 years at the coal-face. Simon Harris may be qualified in Journalism and French, (or may have dropped out to pursue a career in politics) and I have to doubt whether he knows what evidence is, let alone how to evaluate it for the best possible outcome for the majority of the people in his care [that is essentially all 4.7 million of us in the Republic. If he threw the cheque book [€70 million /yr!] at Vertex over Orkambi, what do you think he will do when the nurses come out on strike later this year? He has been called 'Icarus' but that high-flyer was the son of Daedalus an archetype for a canny craftsman and inventor - a sort of proto-scientist - and I don't see that in young Harris.