Debate on the UK Strategy for Rare Diseases

28th March 2017

Philip Dunne responds to a debate on implementing the UK Strategy for Rare Diseases.

Mr Pritchard, I am grateful for your generosity and guidance. You are chairing the debate admirably, as usual. I congratulate my hon. Friend the Member for Bath (Ben Howlett) and the other members of the all-party group on rare, genetic and undiagnosed conditions on securing the debate and on producing a timely and informative report. My hon. Friend rightly pointed out that the Under-Secretary of State for Public Health and Innovation would have been the Minister responding to the debate, but I am sure he will understand why she cannot be here: she was invited to accompany the Prime Minister elsewhere today. I regret, for him, that that proved an invitation hard to resist. It has, however, given me the opportunity to learn more about rare diseases and I am grateful to my hon. Friend for that. I hope to be able to address some of the comments that he and other hon. Members have made.

As has been acknowledged, the UK strategy for rare diseases was published in 2013 and set out a high-level framework that aimed to improve the lives of those affected by rare diseases. The Government remain committed to implementing the strategy by 2020 and are aware that the real test of success will be tangible improvements experienced by patients with rare diseases and their families. Last month’s publication of the all-party group report is a timely reminder that there is still much to do, as has been mentioned by all speakers today. However, it is important not to forget just how much we have achieved.

NHS England has been actively working to raise the priority given to rare diseases. In particular, advances in genomics are already having a positive impact on patients with rare diseases. NHS England has embarked on a world-leading project to establish genomics in mainstream healthcare, establishing a network of 13 genomic medicine centres that will underpin the delivery of genetic medicine over the years to come. The Government have also made a clear commitment to developing genomics, and that is best demonstrated by the £500 million investment in the 100,000 genomes project, which is the biggest national sequencing project of its kind in the world.

The full potential for genomics can be realised only with continued research into rare diseases, and the National Institute for Health Research has established a rare disease translational research collaboration to make maximum use of its significant research infrastructure. I welcome the support given by the Opposition spokesperson, the hon. Member for Washington and Sunderland West (Mrs Hodgson), to that initiative. It is supported by a £20 million investment and has already recruited more than 15,000 patients to support 56 projects related to rare diseases.

I am pleased to say that the UK is a recognised leader on many rare diseases. We are an active member of the International Rare Diseases Research Consortium and have been actively involved in the establishment of 24 European reference networks—ERNs—for rare disease, six of which we are leading on. I was at an informal meeting of EU Ministers early last week, where our role in punching above our weight for the ERNs was widely acknowledged.

An important element of our plans will be to continue to look for ways in which the UK can work with international partners. The all-party group, along with hon. Members present in the debate and a number of external groups in the rare diseases community, have raised concerns about the perceived impact of the planned changes to the methods used by the National Institute for Health and Care Excellence for the evaluation of highly specialised technologies. That is one of a set of proposals being introduced by NHS England and NICE following the recommendation in last year’s Public Accounts Committee report on specialised services that the Department of Health and our arm’s length bodies should better consider affordability when making decisions, including on rare diseases. NICE and NHS England have consulted on the proposals, and I can assure hon. Members that they have listened carefully to the responses and made substantive changes.

The planned changes to NICE’s methods, as amended following the consultation, recognise the unique position of patients with very rare diseases and the need to pay a premium for their treatments. The changes introduce a clearer framework for the assessment of drugs for very rare diseases, which will better enable commercial discussions between NHS England and the pharmaceutical companies, in line with the recommendations of the accelerated access review. The consultation originally proposed the introduction of a threshold of £100,000 per quality-adjusted life year. In response to feedback, that single threshold has been replaced by a sliding scale, which means it will be possible for transformative treatments that offer significant health gains to be approved up to £300,000 per QALY. That is 10 times greater than NICE’s threshold for treatments considered by its mainstream technology appraisal process.

My hon. Friend the Member for Bath can speculate, but I gently say to him that it is not possible to predict how likely any individual new drug is to be recommended by NICE under the changes in the future, and we cannot retrospectively apply the new framework to past decisions. Furthermore, it is important to stress that even when NICE is not able to recommend a drug for the full patient population, NHS England may still be able to fund a drug for a subgroup of patients who will most benefit from treatment.

The hon. Member for Linlithgow and East Falkirk (Martyn Day) referred to the co-operation of Scotland, along with the other devolved nations, in collaborative work. We acknowledge and welcome that, and we acknowledge that Scotland has published an implementation plan. My hon. Friend the Member for Bath referred to four examples of drugs that NICE had approved for use for rare diseases in England, and they are available for use in Northern Ireland. The hon. Member for South Down (Ms Ritchie) asked about that. In particular, Translarna was approved for Duchenne muscular dystrophy, which she referenced. I gently say to the hon. Member for Linlithgow and East Falkirk that those four drugs are not yet available in Scotland—at least, not according to my briefing—other than one that is available for restricted use. Although we wish to continue co-operating with Scotland, the system in England has some advantages thus far.

I am confident that the planned changes create a framework that will enable truly transformational new drugs for patients with rare diseases to be made available where companies are willing to set prices that fairly reflect the added benefit they bring.


I congratulate the hon. Member for Bath (Ben Howlett) on securing this debate. I welcome the progress that the Minister has described, but one thing that has been touched on in several speeches is the difficulty people have as a result of the nature of rare diseases. When they are trying to get a diagnosis in the first instance, medical practitioners often have no experience of the disease. As he develops his speech, or as the Department develop the policies and framework for delivering more services, will consideration be given to how that knowledge can be more widely spread?


The right hon. Gentleman anticipates something that I will come to shortly in my speech, but we acknowledge that challenge and we recognise that there will continue to be concerns among patient groups and Members. I hope that my hon. Friend the Member for Bath in particular will understand that the proposed changes are intended to put in place a fairer, more transparent framework for the evaluation of technologies for very rare diseases as they are developed.

I want to respond specifically to the challenge posed by my hon. Friend and the shadow Minister on the real need to ensure that the commitments set out in the UK rare disease strategy are fully realised. It is right that last week in the House, my hon. Friend the Under-Secretary of State for Public Health and Innovation made a personal commitment to that effect in her response to my hon. Friend’s oral question, and I am happy to reiterate that commitment today. I can confirm to the House that I have agreed with the chief executive of NHS England that by the end of this year he will deliver an implementation plan for those of the 51 commitments of the UK strategy for rare diseases for which NHS England has lead responsibility. For those commitments that fall outside NHS England’s remit, the Department will work collaboratively across stakeholders to contribute to the implementation plan. I am sure my hon. Friend will agree that the development of that plan will be a significant step in the journey.


indicated assent.


I welcome my hon. Friend’s acknowledgement of that. We are absolutely clear that we need to ensure that the proposals are used to drive real action and make tangible improvements for patients affected by rare diseases. It not only about having a plan but about ensuring that the plan has effect for sufferers of these conditions. That is why we have recently reconfigured and strengthened the governance arrangements and formed a more streamlined UK rare disease policy board to monitor and co-ordinate progress in implementing the strategy.

In addition, we have strengthened the patient voice with the appointment of two patient representatives to the policy board. To make our work more transparent, a broad online stakeholder forum will operate in collaboration with the policy board to allow a more meaningful dialogue with the rare disease community. The rare disease policy board will be supported by clearly defined task and finish groups to examine progress objectively and to consider in particular the diagnostic odyssey that my hon. Friend referred to, which can be so frustrating for those who are still unable to determine their condition. That issue is a considerable worry for anyone affected by a rare disease, who will be familiar with the problem.

I will conclude slightly ahead of schedule by confirming that the lives of patients with rare diseases can be improved only by means of concerted and co-ordinated action. I take this opportunity to reaffirm the Government’s aims to drive real improvements in the care and treatment of those affected by rare diseases by working with stakeholders to deliver the standard of care and treatment that all patients deserve and to ensure that the implementation plan for England is delivered by the end of this year.

| Hansard




Click here to signup to my newsletter.


Advice Surgeries

All are welcome at my advice surgeries, but an appointment is recommended.

To make an appointment please telephone the constituency office on 01584 872187.
Monday 25th June
54 Broad Street, Ludlow
Friday 27th July
Market Hall, Cleobury Mortimer