BACKGROUND

—  MND is the name given to a group of related diseases affecting the motor neurones (nerve cells) in the brain and spinal cord. As the motor neurones die, the muscles stop working.

—  MND is a rapidly progressive and fatal disease that can affect any adult at any time. The cause of MND is unknown and there is no known cure.

—  Every year 1,600 people die from MND in the UK. It affects over 5,000 people in this country at any one time, with a prevalence of around seven per 100,000.

—  On average it takes 17 months from first symptoms to diagnose MND. Half of those with the disease die within 14 months of diagnosis.

—  MND leaves people unable to walk, talk or feed themselves, but the intellect and the senses usually remain unaffected.

EXECUTIVE SUMMARY

  1.  The MND Association welcomes the Health Committee's inquiry into the National Institute for Health and Clinical Excellence (NICE). We have a particular concern about the length of time it takes for topics to be selected for appraisal by NICE.

  2.  The MND Association suggested non-invasive ventilation (NIV) for MND to NICE as a topic in January 2006. However, we are concerned at the length of time it is taking for NIV to be selected for appraisal. The Association has estimated that if NIV is selected as a topic, guidance on its use would not be issued until 2009 at the earliest—three years after we suggested it. Nearly 5,000 people with MND will have died in this time, many without access to this beneficial treatment.

NON-INVASIVE VENTILATION FOR MND

  3.  During the course of the disease, most people with MND develop respiratory muscle weakness and respiratory failure is frequently the cause of death.

  4.  Research published in 2006 showed that NIV can significantly improve quality of life and extend survival for people with MND.(1) For people without severe bulbar dysfunction, NIV extended life by a median of 205 days (around six months). This is significant on a median survival of 14 months.

  5.  A survey of consultant neurologists carried out in 2002 showed that only 5.5% of people with MND under review were using NIV (an estimated 2.6-3.5% of all people with MND).(2) The survey showed that most neurologists (172 out of 265) had referred no patients for NIV in the preceding year, while three neurologists made 30% of all referrals nationally. This variation in clinical practice is clearly unacceptable.

  6.  The only drug treatment licensed to treat MND—riluzole—was recommended by NICE in January 2001. It has shown to extend life by an estimated three to six months, so NIV compares favourably to it.

NICE SELECTION PROCESS

  7.  We formally suggested NIV to NICE in January 2006. This was about the same time that the topic selection procedure was being reviewed, ironically to make it faster.

  8.  Fourteen months later, we understand that NIV may be considered at the Consideration Panels in July. If it is successful at this stage and later stages of the process, it will be at least another nine months before any appraisal would start (spring 2008). This would mean that guidance would be issued sometime in 2009—three years after the Association suggested it as a topic. Sixteen hundred people die from MND every year, so over this period of time nearly 5,000 people with MND will have died, many without access to NIV.

REFERENCES

(1)  Bourke SC, Tomlinson M, Williams TL, Bullock RE, Shaw PJ, Gibson GJ Effects of non-invasive ventilation on survival and quality of life in patients with ALS: a randomised controlled trial. Lancet Neurol 2006; 5:140-47.

(2)  Bourke SC, Williams TL, Bullock RE, Gibson GJ, Shaw PJ. Non-invasive ventilation in motor neuron disease: current UK practice. ALS and other motor neuron disorders: official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases 2002 Sep; 3 (3); 145-9.

Alison Railton

MND Association

21 March 2007