One Rare Disease Drug’s Access Journey Across The Globe

Vertex Pharmaceuticals Inc., a company focused on developing therapies for rare diseases and the first to produce a medicine to treat the underlying cause of cystic fibrosis (CF), has a vested interest in improving market access and reimbursement procedures worldwide. As a drug developer, it has argued for fair, and high, prices for its medicines, that it says must reflect the significant value its drugs bring to patients as treatments for the disease and not just the symptoms. The company has sought several innovative reimbursement agreements across the globe for its portfolio of CF products – particularly for Orkambi (lumacaftor/invacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy approved for use in children and adults with CF.

Orkambi – the second to market of Vertex’s three available CF therapies – made history as the first combination drug priced below that of one of its component parts. When...