Cystic Fibrosis

Edward Leigh Excerpts

Tuesday 8th December 2015 Westminster Hall

[George Freeman]

My right hon. Friend makes her point as powerfully as ever. I shall not add to it; it has been put on the record very clearly.

My right hon. Friend asked about contact with the company. It is not for Ministers to get actively involved—much as, at times, I would like to—in negotiating these deals, but I have made contact with the company, both on Vimizim and Translarna, to urge it to be as flexible as it can in discussions. I can only say that I am hopeful that it will have been able to reach a point where NICE feels able to make a recommendation.

Part of the reason why due process is important is that when NICE makes a recommendation, NHS England is bound in law to provide the drug in perpetuity, so it is a major cost undertaking. In some cases, these drugs cost £200,000 or £300,000 a year, so it is a commitment of several hundred million pounds from NHS England. Other patients would say, “We must make sure that when you make a decision like that, it is done properly.” However, I share my right hon. Friend’s frustration that a lot of these breakthroughs scientifically mean that we ought to be able to speed things up.

My right hon. Friend asked whether the Prime Minister is holding my feet to the fire. She need not worry; I am as passionate about this as ever and very impatient to make sure that the AAR is landed with some good recommendations.

My right hon. Friend made an excellent point about NIHR staffing. I am working with the chief medical officer and the NIHR on that at the moment. A number of our clinical research facilities could, with a few more staff, turn over more and do more trials work. There is an opportunity for us to get more people internationally to enrol in NIHR training—in clinical trials and translational research training—which would give us more capacity and allow us to move things along faster.

The hon. Member for York Central (Rachael Maskell) raised an important point about cost. I have touched on the work that we are doing on per-patient costing to try and make sure that we develop a system that more intelligently captures the real cost of disease.

I am grateful to the hon. Member for Denton and Reddish, the Opposition spokesman, for his comments. I congratulate him on the by-election victory. He asked about NICE reform, which I have touched on, through the AAR. We do not want to interfere with or undermine NICE’s independence and their “gold standard” reputation, but we want to create a place in which the accelerated access review gives them the freedoms that they are, indeed, helping to shape.

In conclusion, this debate has highlighted not only the challenges from the rising costs of new drug discovery—£200,000 to £400,000 a year for patients in the rare disease space—and the pressure on the one-size-fits-all model of assessment, but the opportunities for us to unleash our leadership in genomics and informatics to create a new landscape. That is why this week, the Association of Medical Research Charities conference and my summit this afternoon, and the accelerated access review work is creating momentum for a new landscape for accelerated pathways for patient-led innovation.

I think we will look back in two or three years at this as a crucial turning point at which the system that was set up to assess a very one-size-fits-all, 20th-century model was rapidly adapted, creating new opportunities for patient-led innovations and charities such as the CF Trust to bring through innovations that benefit their patients more quickly.

[Sir Edward Leigh]

Would you like to sum up, Mr Austin?