Drugs: Ultra-rare Diseases

Ben Howlett Excerpts

Tuesday 16th June 2015 Westminster Hall

[Ben Howlett (Bath) (Con)]

I add my congratulations to those that have been offered to the hon. Member for Leeds North West (Greg Mulholland) on tabling the debate. His passion and commitment on the subject are second to few.

Last week in Bath I met an incredibly impressive constituent of mine named Sarah Long. Many hon. Members will already know of her from the Twitter debate yesterday. She met me to discuss the benefits that she has received from Vimizim, the enzyme replacement therapy to address the cause of Morquio A. She is estimated to be one of just 88 people in the UK who need the drug. While she has been on Vimizim she has experienced dramatic changes, which have become gradually more apparent. She told me that since being on the drug her ability to use her lungs has been transformed. Before she started treatment she needed almost constant access to oxygen, and today she needs just four hours of nebulising. Pre-Vimizim, Sarah found it difficult to talk, but Vimizim has given her voice back to her. The idea of a return to the former days obviously fills her with dread.

Following recent conversations with the MPS Society, the National Institute for Health and Care Excellence announced on 4 June that it is leaning towards not recommending Vimizim for treating people with MPS IVA, or Morquio. That is only a preliminary recommendation and is not its final guidance; the decision may change after consultation. I hope in the interests of my constituent that it will change. I request my hon. Friend the Minister to lean heavily on NHS England before 25 June as it decides about reimbursement with respect to Vimizim on an interim basis, while NICE completes its decision making.

The date of 25 June is critical, as hon. Members have said. If NHS England announces a positive decision, all those who want treatment and who meet the criteria will be allowed access to treatment, regardless of whether they were on the clinical trial. If NHS England follows NICE’s current position and the decision is negative, BioMarin will immediately withdraw compassionate use from those in England who are receiving treatment.

As hon. Members will know, MPS can lead to reduced life expectancy. However, Sarah is in her forties. We have heard an awful lot in the debate about treatments that support young people, but Vimizim also supports adults, if they manage to get to such an age. If NICE gathered more evidence from people such as Sarah, the Minister would see that Vimizim has worked for her and for and others. The real injustice is that her quality of life has dramatically improved, but it appears that NICE is unable to conduct a peer review because of the lack of cases. I hope that the Minister will be able to look into that Catch-22 case.

Being on the drug has dramatically reduced the cost of my constituent’s care, because the amount of time on oxygen has fallen. That is also an obvious cost reduction to the taxpayer. NICE clearly needs to acknowledge the significance of clinical expertise in its processes, and to address its current expertise shortfalls to prevent other constituents with a rare disease from having to suffer the same problems. However, if a further extension to Vimizim is granted, it must be available to all ages and not just children.

I am delighted that the Government have published a strategy for rare diseases, but a strategy is only as good as its implementation. The strategy highlights a commitment to protecting patients with rare diseases and emphasises the need to improve and deliver effective interventions quickly, equitably and sustainably. I hope the strategy will put my constituent in a good place. I am pleased that the Government are leading the way on scientific and pharmaceutical research, but what good are those things if they do not reach those who most need them? The rare diseases strategy is excellent, but will the Minister provide the House with an update on an ultra-rare disease strategy? I would be most grateful if he were to have the time to meet my constituent in the coming days, given the urgency of the matter.