Orkambi, made by Vertex Pharmaceuticals, is quite literally a life changing drug for the thousands of UK and Channel Island CF sufferers with the deltaF508 gene mutation. Along with Ivacaftor, it’s the first of a new line of precision drugs that targets the cause of CF rather than just reducing symptoms. New precision drugs like Orkambi stabilise lung function, improving and extending the quality of life.
The Westminster debate was the result of a petition started earlier this year by parents Gemma Weir and Michelle Frank, which attracted a staggering total of more than 100,000 signatures in just 10 days. The overwhelming support for this petition truly demonstrates how the entire CF community and their friends and families have come together united to simply say: enough is enough.
Whilst protesters gathered outside, inside Westminster Hall 22 MPs shared the experiences of those who live with cystic fibrosis. Some of those stories came from a lucky few who have access to Orkambi and are thriving, but most came from those who are less fortunate and are still awaiting to access to the drug.
Lynsey Beswick, Public Affairs Manager at the CF Trust was sat in the public gallery. Her story is one of many in the latter group. Ian Austin MP told of the deterioration of her lung function over the past two years since the drug was licensed. He explained that while many of her friends are getting married and starting families, at 35 she instead is potentially facing the end of her life as her health plummets, helpless to do anything despite knowing there is a drug that could help slow this decline. This was in contrast to Ian’s own constituent, Carly Jeavons, who he explained had thrived since being on Orkambi through ‘compassionate’ use, getting married and even starting her own business.
Unfortunately, Lynsey’s story does not exist in isolation – the debate highlighted the cruel reality for the thousands affected by this devastating genetic condition. Yet Orkambi is still not available here in the UK or Channel Islands. The debate was a vital opportunity for the government to acknowledge and respond to this pressing issue.
The key themes within the debate included MPs urging the Secretary of State for Health and Social Care to look at current NICE appraisal systems, and a strong call for government to look at the potential cost savings around implementation of the drug. However the key message many parliamentarians impressed was the urgent need for government to apply pressure on all parties to find a workable solution.
In his response, Health Minister Steve Brine MP said that “The government whole heartedly support efforts to ensure prescion medicines we have heard about are made widely available to CF patients” and that he agrees they should “get the same quality, safety and efficacy in medicines as those who have more common conditions” and that “there is more talking to do”.