One Rare Disease Drug’s Access Journey Across The Globe
Executive Summary
Infographic: Vertex, the first company to produce a drug to treat the underlying cause of cystic fibrosis, has fought for fair (and high) prices for its medicines to reflect their value as treatments for the cause of a genetic disease. The company has sought innovative reimbursement agreements for its CF products in various countries.
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 Vertex's first therapy, Kalydeco (ivacaftor), launched in the US in 2012 the company set a wholesale acquisition cost (WAC) of $294,000, which has since increased to $312,000. With Orkambi, it set the initial WAC lower than both of those price points, at $259,000 a year. (Also see "Why Pricing Orkambi Below Kalydeco Is A Savvy Strategy For Vertex" - Pink Sheet, 13 Jul, 2015.)
Strategic pricing is not the only unique play Vertex has used for its CF portfolio, the company has also negotiated different and somewhat creative reimbursement agreements for its products in various countries. It has innovative reimbursement arrangements in place in Denmark, Ireland and Sweden, so far.
Vertex’s novel “portfolio-based” approach to pricing and reimbursement allows for greater budget predictability and for an earlier launch with its newer therapies – particularly the third drug in its CF basket, Symkevi (tezacaftor/ivacaftor). In Denmark, for example, the company’s reimbursement deal with the local authority covers all Vertex’s current and future CFTR modulator medicines, including Orkambi and Kalydeco, as well as “future drugs” when they win marketing authorization. (Also see "Vertex’s Danish Reimbursement Deal Brings Budget Predictability" - Scrip, 16 Oct, 2018.) Regulators in Europe granted a positive recommendation in late-October for the approval of Symkevi. (Also see "Vertex Secures Hat Trick Of Europe CF Approvals With Nod For Symkevi" - Scrip, 2 Nov, 2018.)
Despite being approved in the US, EU, Canada, Australia, Switzerland, Israel, and Brazil, Orkambi has not yet been reimbursed in several countries. Places where pricing negotiations have started but not concluded with an access agreement include: Canada, France, Spain, Switzerland and the UK.
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