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A welcome intervention for cystic fibrosis

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New breakthroughs for cystic fibrosis New breakthroughs for cystic fibrosis

January's Final Appraisal Determination (FAD) by NICE recommending TOBI Podhaler (tobramycin inhalation powder) as a cost-effective therapy is to be welcomed by people with cystic fibrosis (CF). This innovative device presents a less time-consuming option for treating chronic pulmonary infection due to Pseudomonas aeruginosa (Pa), the most common respiratory illness affecting these patients.

People with CF can spend up to two hours a day actively managing their condition, including physiotherapy and nebulised treatments. As a specialist CF physiotherapist, I am acutely aware of the challenges adherence to routines can create for patients and their families. Reducing effort needed for administration of just one therapy can generate a significant time-saving.

In this case, switching from a nebuliser to a dry powder device for the twice-daily delivery of tobramycin reduces treatment time from 20 minutes (plus additional time needed for nebuliser dismantling and cleaning) to just five or six minutes for each administration, without compromising efficacy of treatment. My patients say this may be the difference between having breakfast in the morning before rushing to work or school and not missing the bus in.

The inhaler is pocket-sized and light-weight and because it doesn't need batteries or an external power source and the drug doesn't require refrigeration (as the nebuliser does), it makes it more portable to use on the go. All these attributes contribute to greater treatment satisfaction and quality of life improvements when compared with nebulised therapy.

CF affects more than 9,000 people in the UK and is one the most common life-threatening inherited diseases in the UK. NICE's decision to make an inhaled dry powder device more readily available to suitable patients acknowledges the burden people with CF face on daily basis and will definitely contribute to improvements in quality of life for many.

Penny Agent, deputy director of rehabilitation and therapies, Royal Brompton and Harefield NHS Foundation Trust

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