Alpha-1 Antitrypsin Deficiency

Part of the debate – in Westminster Hall at 3:59 pm on 31st October 2018.

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Photo of Mark Pawsey Mark Pawsey Conservative, Rugby 3:59 pm, 31st October 2018

Part and parcel of our efforts today are to achieve precisely that. I am delighted that the hon. Lady’s constituent comes to Coventry and is being treated by the excellent services at University Hospitals Coventry and Warwickshire.

Unfortunately, alpha-1 is not yet curable, and no specific treatment for the disease is freely available in the UK; it is a matter of treating only the symptoms with the appropriate therapeutic methods. Jim Shannon referred to intravenous AAT protein augmentation therapy, which involves replacing the missing AAT protein. That treatment is available in the United States, Spain, Germany and Italy, for example, but it is not yet available in the UK.

The National Institute for Health and Care Excellence—the Government body that produces guidelines on which treatments to make available—only last month published draft guidelines that rejected the use of the only licensed augmentation therapy product in the UK, Respreeza. It has had a UK licence since 2015, but was unfortunately deemed by NICE to be too expensive to be made available on the NHS. We acknowledge that it is expensive; lifelong therapy costs around £60,000 per patient per year. NICE continues to evaluate that and will make its final recommendations next year. The entire alpha-1 community has been heavily involved in pressing the case for patients across the country to be prescribed the treatment.

Only the symptoms of alpha-1 sufferers are treated, often by inhaled medications developed for people suffering from asthma and COPD, rather than specific treatments for the lung damage caused by alpha-1. The other issue is that those who suffer from alpha-1 become susceptible to chest infections, which was certainly the case for Stephen Leadbetter, and it is vital that they are treated quickly with antibiotics at the first sign of infection and are vaccinated every year against flu.