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Almost simultaneously with a request to prescribe any novel treatment for our patients, the bellowing voices of the Mr Bumbles, to be found inhabiting our drug and therapeutic committees and our local family health services authorities, will be heard to exclaim, ‘More! after he has prescribed the drugs already allotted by the pharmacy!’1 Thus those of us committed to the care of patients with cystic fibrosis should not have been surprised to encounter a wall of resistance to the introduction of recombinant human DNase (rhDNase) at an estimated cost of £7500 per patient per year. Even after multicentre, international studies conducted by most respected colleagues we were asked to make the case again, and to prove its efficacy and cost effectiveness.
Key messages
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rhDNase is an effective additional treatment for a subgroup of patients with cystic fibrosis
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These patients should be monitored for objective evidence of continuing response
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At least one third of the cost of treatment will be recouped from the decreased need for intravenous antibiotic courses
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Conclusive judgments on cost effectiveness depend on the results of longer term studies which are now in process
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All such judgments must be made in the context of the effects of rhDNase on a disease which is fatal, on average, at about 30 years of age
What is cost effectiveness in cystic fibrosis?
The concept of cost effectiveness is fraught with difficulties for clinicians committed to the care of patients with cystic fibrosis. There is no easily defined starting point and no clearcut finishing tape. The cost effectiveness of treatment for cystic fibrosis cannot be assessed in the same way as, for example, the immunisation campaign to eradicate poliomyelitis, which has a well documented preintervention scenario and easy to record end points, both economic (less decimation of the workforce, less money spent on longterm care, no need for …