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G551D mutation, only approximately 3000 patients might be able to benefit from the new drug. The costs for one year of treatment with KalydecoTM


$294,000 in the US or €306,000 (reference price in Germany) respectively, making it pricey. The time between the discovery of ivacaftor and the marketing authorisation was short, but filled with complexities. The development was partly sponsored by a charity. Even if we assume a total cost of development for KalydecoTM

of one

billion US$ and that only every third CF patient with a G551D mutation would in fact receive the branded drug, the return on investment would be reached within three years. Clearly, some of the sales returns will be used as royalties to the CFF and to help developing other compounds directed against the remaining 96% of mutations found in those CF patients who may not benefit from ivacaftor. But questions might be raised whether the public insurance systems are willing to support a lifelong therapy with orphan drugs such as KalydecoTM

. Countries such as Germany 22 and the UK require that an added benefit

for every newly approved drug over pre-existing therapies is demonstrated in order for it to be reimbursed through the public insurance system.


The future will tell whether ivacaftor is, in fact, the quantum leap innovation to justify the costs. However, from the perspective of someone who has spent half of his lifetime waiting for a drug that treats the basic defect in CF, ivacaftor is certainly a major leap forward. l

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