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Vertex faces rejection of its CF medicines in Scotland

Another rejection over cost-effectiveness


The Scottish Medicines Consortium (SMC) has not recommended Vertex’s cystic fibrosis drugs Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) for routine NHS use in Scotland. 

Despite recognising that the decision regarding the drugs “will be disappointing” for patients and families of those living with CF, the Scottish NHS watchdog maintained there was “significant uncertainty around their overall health benefits in the long term in relation to their costs”.

The drugs were reviewed through the Patient and Clinician Engagement (PACE) process, which the SMC uses to evaluate end of life medicines and medicines which treat rare conditions. According to the SMC, the PACE process allows patient groups and clinicians to be more involved in its decision making.

However, even with strong testimonies from both patient groups and clinicians, the SMC decided that the evidence submitted by Vertex did not sufficiently prove the efficacy of both medicines in relation to their costs.

“In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we will continue to work with the company to achieve that,” said Alan MacDonald, SMC Chairman.

Even though the medicines were not recommended for routine use, the SMC has said that it can consider requests from healthcare professionals who make a case for use for a particular patient. This is made possible through the PACS Tier 2 process and according to Vertex approximately 65 patients in Scotland who are eligible for its medicines have been approved through this scheme.

In response to the SMC decision, Vertex has said that it has been working with the Scottish Government to come to an agreement for broad access for all eligible patients. CF is an inherited disease which has profound effects on lung function and the digestive system, with around 900 people in Scotland living with the condition.

The SMC’s decision follows a history of rejections of Vertex’s CF drugs, with NHS England labelling the American pharma company an “extreme outlier” in its pricing and behaviour. Currently, Orkambi is listed at $104,000 per year of treatment, which has caused a continuing row over access between NHS England, the English cost-effectiveness watchdog NICE and Vertex.

Most notably, patients have set up a ‘buyers’ club’ to access Orkambi through a legal loophole, which allows private individuals to import generic copies from abroad where there is no patent covering the drug.

The CF Buyers’ Club has sourced a generic copy of Orkambi from Argentinian pharma company Gador. It has asked for £23,000 per patient per year, which it will reduce to £18,000 if the buyers’ club can get at least 500 members.

The pricing disagreements are likely to continue, with Vertex recently filing its new CF triple therapy with the FDA in the US. This breakthrough treatment could transform the treatment of the disease, but if it follows in the footsteps of its predecessors, access to its potential benefits could prove tricky.

Article by
Lucy Parsons

13th August 2019

From: Regulatory



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