The hesitation of the National Institute for Health and Care Excellence (NICE) in sanctioning a “miracle” drug for Cystic Fibrosis, as recently reported in this paper, might seem harsh and unfair. Still the watchdog’s decision is certainly illustrative of an ever more pressing problem – the NHS simply can’t afford to pay the inflated prices charged by pharmaceutical companies for new medicines.
To clarify. The prospects for those born with the genetic disorder rank amongst the major medical successes of modern times – an improvement in average life expectancy from just five years in the postwar years to nowadays their late forties.
Several factors have contributed to this, but mostly it has been brought about by relatively straightforward measures to mitigate the effect of those thick, sticky bronchial secretions that are the hallmark of the condition – vigorous daily chest physiotherapy, aerosol inhalations and prompt antibiotic treatment of infective exacerbations.
It was hoped cystic fibrosis might be curable with “gene therapy” – replacing the defective gene in the cells lining the airways with a normal copy. When this failed to materialise, researchers turned their attention to finding a chemical that might fix the abnormal protein that the faulty gene gives rise to. This entailed screening more than a million compounds, just three of which were found to coax the protein into functioning normally. Taken together these are the ingredients of the “miracle” drug Kaftrio.
It certainly works – reducing, for example, by almost two-thirds those infective exacerbations warranting treatment with intravenous antibiotics. But multiply the price tag of £160,000 per year by the 10,000 people with Cystic Fibrosis and a lifetime treatment of around 50 years leaves the NHS facing a total bill for £80billion, enough money to rebuild many of the UK’s hospitals.
NICE’s hesitation is thus understandable, though it could be a negotiating tactic to persuade the pharmaceutical company to reduce the price to the figure that the NHS offered: around £10,000 a year. Still expensive but affordable.
There is no mistaking the characteristic rash and intense itchiness of the commonest of all skin conditions, eczema, that has become commoner still in recent times. This has been attributed, speculatively, to the “hygiene hypothesis”, the notion that being now so much less exposed to dirt and germs than in the past, the immune system has become more sensitive to environmental allergens. That may be true, but gratifyingly the rash and itch usually improves with a combination of a moisturiser and steroid cream such as Betnovate or hydrocortisone – the latter being deemed sufficiently safe and free of side effects as to be available from the local pharmacy without the need for a prescription.
Topic of the moment
There is a fly in the ointment (as it were) much commented on recently, the phenomenon of Topical Steroid Withdrawal Syndrome. Here, those who discontinue their steroid cream after several years may not only experience a flare-up of their original eczema but also be afflicted with a quite different and grievous skin complaint. This manifests initially as a diffuse burning redness of face and limbs that become swollen and may be studded with papules. The skin subsequently becomes dry and itchy with profuse flaking of the outer layer that may take weeks or months to resolve.
There is currently no explanation as to why the skin should react so violently in this way or indeed how it might be remedied. The wider recognition of TSWS has however prompted a call for greater caution: “We have to critically review their long-term safety”, notes Dr Chantal Cotter in the British Journal of Dermatology. Meanwhile, steroid creams should be applied as sparingly as possible and preferably no more than twice weekly.
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A wonder drug has given me back my musical career – and my life