The UK has scored lowest in a European comparison of six countries providing access to drugs that help prolong lives of cystic fibrosis (CF) suffers.
At the 29th European Cystic Fibrosis Society congress in Copenhagen, it was revealed two thirds of French patients receive Roche’s Pulmozyme (dornase alfa), compared to fewer than one in five in the UK as a whole and only 11% in Scotland. Scotland also has the lowest life expectancy for CF patients in Europe.
Pulmozyme breaks up the DNA of white blood cells in mucus that clogs lungs of CF sufferers enabling it to be coughed up. The drug is highly effective at preventing exacerbations associated with a decline of lung function and structural damage to lung tissue, say CF specialists. However, the market for CF drugs is small – only 60,000 potential users worldwide - so Pulmozyme is inevitably costly at around 12,000 euros ($15,000) or £5000 per patient per year.
Dr Ian Ketchell of Llandough Hospital, Wales said many health authorities in the UK are currently refusing to fund not only Pulmozyme but also Chiron’s TOBI (tobramycin), a highly effective but expensive antibiotic therapy for CF.
“This is another postcode lottery where getting the drugs you need depends on where you live” he noted. “Pulmozyme has not been subjected to a NICE appraisal leaving a huge variation in health authorities’ attitudes to funding its use. What funding is provided for expensive new CF drugs in the UK is limited so that physicians tend to give them to the sickest patients rather than patients whose lung function is better and which the right drugs could help preserve for longer.”
Dr Ketchell is one of a group of prominent specialists in CF who are leading ‘Back the kid’, a campaign launched in the UK at the same time as the ‘Back the bid’ Olympics campaign. Back the kid, which has an educational grant from Roche, aims to improve access to the best care possible for children with CF. “Sadly, many parents don’t even know their children are receiving suboptimal care,” he commented.
James Chmiel, paediatric pulmonologist at Case Western Reserve University, Cleveland, USA, said in Copenhagen: “Therapies such as Pulmozyme which enhance secretion removal, benefit patients by reducing inflammation. Limiting the inflammatory process is important in slowing the decline in lung function and prolonging survival.”
Almost all cystic fibrosis patients in the US would be considered for the drug and around 85% used it, he added.
Progressive bouts of lung disease lead to patients dying on average in early adulthood. Although internationally median survival from birth has risen dramatically from a mere 8 years in the 1970s, it is still only 31 today with a projected 40 years for patients born in the 1990s. Median survival of US patients with CF however is around seven years longer than in Europe, said Dr Chmiel.
Aggressive use of drugs aimed at keeping airways clear and free of infection are attributed with playing a major role in extending survival, said CF specialist Profesor Felix Ratjen, of University of Toronto, Canada.
From Olwen Glynn Owen in Copenhagen
