A new drug for Cystic Fibrosis manufactured by Vertex Pharmaceuticals, Boston, USA; promises to ease up the lives of about 3000 patients of the disease, in the UK alone, has now been deemed too expensive for testing. Named as Orkambi, the drug is a combination of two drugs Lumacaftor and Ivacaftor, which counteracts the effects of the specific genetic aberration, the F508del mutation on the CFTR gene.

Orkambi recently made headlines when the National Institute for Health and Care Excellence (NICE) in its new guidelines, discouraged public use of the drug. NICE affirmed that National Health Service (NHS) should not make the drug available as the drug could not show long-term improvements in patients and that the drug was not cost-effective. British Medical Journal (BMJ) published research news in this context on  June 20, 2016.

Orkambi is an oral drug which is the first of its kind to target the underlying cause of CF. It is noteworthy that it is only effective against CF cases that are specifically caused by F508del mutation. The drug works by rectifying the misfolding of the protein which in turn regulates chloride channel function.

Cystic fibrosis (CF) is a rare genetic condition caused by the defective CFTR gene. As the movement of water and chloride ions is disrupted across the cell membrane, accumulation of thick mucus clogs the lungs, pancreas and other organs. Additionally, CF severely affects the respiratory function, causing chronic lung infections and lung damage.

The FDA first approved the use of Orkambi in CF patients of 12 years or above in July 2015 in the US. Adding to another milestone for the drug manufacturer, It is to be noted that supplemental New Drug Application (sNDA) is a step, amongst many steps untaken prior to FDA’s final approval. FDA is likely to reach a decision later this year.

In the UK, Orkambi is licensed to be used by the people who have the F508del mutation. mutation. As far as the European market is concerned, the drug has gained approval in Germany and France.

According to NICE, although Orkambi was shown to reduce hospital admissions and the use of antibiotics for lung infections in patients of CF, no long-term benefit of the drug was observed. They claimed that adequate quantitative and qualitative data which would support long-term effectiveness of the drug was not presented.

Carole Longson, director of the NICE Centre of Health Technology Evaluation said, “We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high. We can only recommend treatments when we are certain they are both clinically effective and represent good value for money”.

The drug costs $147,000 for each patient for one year of treatment. NICE suggested the drug manufacturer reduce the drug price so that it could be accessed by the general population. It also implied that NICE will welcome the approval of this drug if the cost is reduced.

The Cystic Fibrosis Trust has responded to NICE discussion while opposing its claims of the drug’s short-term efficacy. The Trust took a strong stance and said that NICE failed to evaluate the additional data of clinical trials that showed long-term effectiveness of the drug.  The trust’s representative went on to say that in the two years since NICE conducted its appraisal, new data has shown that Orkambi has successfully confined the decline rate of lung function to a half. These multi-national trials recruited over 1,100 CF patients with the results favoring the new intervention for helping CF patients

Ed Owen, Chief Executive of the Trust, disappointed at NICE’s decision, said, It demonstrated the weakness of the current system and NICE has been able to confidently predict the long term benefits of the drug.”

However, in this discouraging situation, The Cystic Fibrosis Trust has come up with a solution in its open letter to Vextex and the NHS. The Trust, which contains the registry of 99% patients of CF in the UK, has extended its support to Vertex and NHS alike. The trust has also invited them  for collaboration and negotiations to solve all discord.

The trust has also recommended that Vertex and NHS can still reach an agreement to devise a mechanism to make this novel treatment available to patients despite the verdict from NICE.

One of the youngest participants of the drug trial, Clara Mann, aged 14, spoke in favor of the drug and said that the drug has changed her life for the better. Clara, who was unfit, underweight and weak for her age, now considers herself healthy.

NICE will issue the final guidelines by next month. Till then, the consultees have a right to make an appeal to review the decision. NICE representative also added that if the manufacturers submit a cost-effective proposal to NICE after this timeframe, NICE will still consider reviewing it on a priority basis.