Overcoming reimbursement hurdles will allow Orkambi to reach its entire target patient population.
By Daniel Chancellor, Lead Analyst
28 June 2016
I am a Lead Analyst covering the CNS and rare disease markets at Datamonitor Healthcare. I joined in early 2010, having ...
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Despite having a large addressable population market for a CFTR-targeted therapy, Vertex’s biggest challenge for Orkambi is to make sure that all eligible patients with two copies of the F508del mutation are able to receive the drug. During its Q1 2016 investor call, Vertex revealed that 65% of eligible patients in the US were receiving Orkambi and that its goal for 2016 was to convert the vast majority of remaining patients. In the US, this is driven by formulary coverage, where Orkambi is now covered by all major commercial insurers and Medicaid programs.
Vertex also needs to improve access to the drug in the EU, although the drug is still in its infancy and country-specific market access decisions tend to be made in the year following approval. The vast majority of ex-US Orkambi sales are in Germany, which provides access before pricing negotiations are finalized. The National Institute for Health and Care Excellence (NICE) has already issued guidance recommending against the reimbursement of Orkambi in the UK. An independent appraisal committee deemed that the drug was not cost-effective, noting that despite the reduced incidence of acute exacerbation requiring hospitalization, overall benefits to lung function were modest. With new clinical data unlikely to change NICE’s assessment of Orkambi’s efficacy, Vertex will need to negotiate discounts in order for the 2,7500 F508del homozygous patients in England to gain access to the drug.
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