Health: Rare Diseases

Baroness Thomas of Winchester Excerpts

Tuesday 3rd March 2015 Lords Chamber

[Baroness Thomas of Winchester (LD)]

My Lords, I acknowledge at the outset that the two matters I am mainly going to speak about tonight are not drug treatments for rare diseases, but they are certainly treatments in the wider sense of that word. I, too, am very grateful to the noble Lord, Lord Turnberg, for asking this Question, which can never be asked too often. I am also very pleased that the noble Lord, Lord Walton of Detchant, spoke about the new drugs coming on to the market for some Duchenne muscular dystrophies. I should at this point declare an interest as I have a rare disease. The two matters that I wish to raise are cough assist machines and hydrotherapy.

Last week, Muscular Dystrophy UK published a new report called Right to Breathe, highlighting the vital need for access to specialist respiratory care for people with muscle-wasting conditions. The report found that respiratory infections have been a primary factor in deaths for certain muscle-wasting conditions with, shockingly, a third of families being repeatedly turned down for equipment by local NHS commissioners, even when it has been requested by consultants or specialist physiotherapists.

Each cough assist machine costs in the region of £5,000, which is equivalent to a 48-hour stay in an intensive care unit. They are considered to be of vital importance by respiratory specialists and are routinely used during hospital stays for patients. An example of what can happen is the case of Freddie Kemp, who had Duchenne muscular dystrophy. He was turned down by his local NHS for a cough assist machine, which would have helped keep his lungs clear. Tragically, in November he died, weeks after leaving hospital following a serious chest infection. This essential piece of equipment may not be a treatment in the way that a new drug is, but it can still save lives. What assurances can my noble friend give that people with muscle-wasting conditions who require a cough assist machine will be provided with one by their clinical commissioning group? This matter of spending a relatively small amount of money on the right equipment for vulnerable people in order to save an expensive hospital stay later on crops up time and again. Surely, something should be done to point this out to CCGs.

Hydrotherapy is a highly effective form of therapeutic exercise in a warm water pool for people with muscle-wasting conditions. For many, particularly boys with Duchenne or anyone with serious mobility problems, it is the only exercise they might be able to manage. The benefits are perhaps obvious, but I will spell them out. The first include a sense of freedom from the confines of a wheelchair, a greater range of movement with the relaxation that very warm water gives and, very often, the alleviation of pain. Secondly, the psychological effect on a person’s well-being should not be overlooked. Perhaps the provision of hydrotherapy should be partly assigned to the mental health budget because of its effect on a patient’s sense of well-being. One young woman with congenital muscular dystrophy told the all-party group some time ago that she felt much better for days after a hydrotherapy session.

However, accessing hydrotherapy is ridiculously hard. If a local hospital does have a pool—many have closed or are in danger of closing in order to save money—patients are told that they are entitled only to a block of six sessions. If you have a progressive condition and this is the best way of keeping you well, a block of six sessions gets you only so far. It is fine for a broken leg, but those of us with progressive conditions will never have what is called “an outcome”. It is not easy to measure the effect of hydrotherapy on those with progressive conditions, but we all know that it is good for us. A study into the provision of these pools in the south-west of England a few years ago by Khurm Arshad, whose brother Auzair has Duchenne muscular dystrophy, found that there were more hydrotherapy pools for horses than for people. Muscular Dystrophy UK is undertaking an audit into hydrotherapy provision for people with muscle-wasting conditions across the country. Will my noble friend encourage the NHS to work in partnership with Muscular Dystrophy UK to compile this audit in order to improve access to hydrotherapy pools?

Muscular Dystrophy UK’s Fast Forward campaign is looking at potential new drug treatments, in particular to ensure that cutting-edge, high-cost potential treatments are not being held up due to lack of funding. That will be the leitmotiv throughout this debate this evening. I am sure that I know the answer to this, but I must just ask whether there are any plans to re-establish a ring-fenced fund for rare disease drugs.