Rare Diseases Strategy

Ben Howlett Excerpts

Tuesday 28th March 2017 Westminster Hall

[Ben Howlett (Bath) (Con)]

I beg to move,

That this House has considered implementing the UK Strategy for Rare Diseases.

It is a pleasure to serve under your chairmanship, Mr Pritchard. Since taking over as chairman of the all-party parliamentary group on rare, genetic and undiagnosed conditions in 2015, I have had the great privilege of meeting and helping patients, children and their parents and families. To explain the enormity of the situation, one in 17 people will be affected by a rare disease at some point in their life. That equates to approximately 3.5 million people in the UK. There are currently between 6,000 and 8,000 rare diseases that have been identified, with patients affected at all ages. However, for those who have a known rare disease, the picture is slightly better than for those whose disease is not known. Many children and adults who I have met do not have a diagnosis. That is both deeply distressing and prevents adequate care from being provided fully. It results in a wide range of issues for patients and parents, who are often desperate to find the answers. Despite the scale of the issue, unfortunately a significant proportion of people with a rare, genetic or undiagnosed condition still do not have access to adequate care and treatment.

Given the enormity of the problem, the Government published their rare diseases strategy in 2013. It was heralded by the rare diseases and medical communities as a major breakthrough. While symptoms vary from condition to condition, there are a number of issues that patients and families affected by rare diseases face collectively—for example, the difficulties associated with accessing a timely and accurate diagnosis and the appropriate co-ordination of care. The publication of “The UK Strategy for Rare Diseases” should have heralded a new era of treatment and care for rare diseases patients in England, Scotland, Wales and Northern Ireland. Containing 51 commitments, the strategy aims to ensure that health and social care systems across the nations provide those living with rare conditions with the highest quality of evidence-based care and treatment, regardless of where they live in the UK.

[Jim Shannon (Strangford) (DUP)]

I thank the hon. Gentleman for bringing this important issue to Westminster Hall for consideration. There are some 60 different types of muscular dystrophy, and 1,000 children and adults for every 1 million of the population are affected—70,000 people are affected by a muscle-wasting condition in the UK. Does he share my concern that our current strategy helps those who live a life of suffering because of those terrible diseases, and does he believe that we are offering the best, or enough, support to their carers at this time?

[Ben Howlett]

The hon. Gentleman and I have discussed rare diseases many times in this Chamber. I pay tribute to his work and that of his party on pushing this agenda in Northern Ireland; that also has implications for England. I agree with what he said and I will come on to evidence given to the all-party group—he mentioned a range of different things—including that of Muscular Dystrophy UK. I hope hon. Members find that report and the evidence that we refer to beneficial.

Without wishing to go into too much detail about the 51 recommendations, given the limited time that we have, the UK strategy aims to

“ensure no one gets left behind just because they have a rare disease”.

Features include setting a personal care plan for every patient, bringing together health and social care services; ensuring that patients, their families and carers have the information that they need and are listened to and consulted; and improving diagnosis and intervention and strengthening research to improve personalised approaches to healthcare for those with a rare disease.

The four countries in the UK were given a deadline of 2020 to implement the commitments. Although the Health Departments in the devolved nations have all published country-specific implementation plans to reflect their respective health services, structures and priorities, the Department of Health in England has not yet co-ordinated a plan for England.

At this point, I want to pause to thank all the charities and organisations across the country—including the Centre for Rare Diseases in Birmingham, the Birmingham children’s hospital, Lupus UK and Muscular Dystrophy UK—that have not only submitted evidence to our report, but done so in advance of this debate. Although I cannot name a million and one organisations, given the limited time, I—and, I think, the whole Chamber—wish to thank them for all their work on helping patients with rare, genetic and undiagnosed conditions.

As has been referred to, between October 2016 and January 2017, the all-party group on rare, genetic and undiagnosed conditions conducted an inquiry into the implementation of the UK strategy for rare diseases in England. It held three hearings with the Department of Health and its arm’s length bodies. More than 300 patients, family members, patient organisations, clinicians and industry representatives also submitted evidence. I thank all those who did so and all colleagues who took part in the evidence sessions, which were very interesting. Overall, the findings were that the strategy is working well and being implemented effectively in some areas. However, where the strategy is failing to be implemented, the problems it was designed to fix are in some cases getting worse or, at best, failing to get better. I will detail the findings of our report and ask the Minister a number of questions.

I was pleased with the response to a question I asked during Health questions last Tuesday. The Under-Secretary of State for Health, my hon. Friend the Member for Oxford West and Abingdon (Nicola Blackwood)—she is sadly unavailable for this debate, but I thank the Minister here today for stepping in at short notice—said that

“the UK strategy for rare diseases needs to be translated into an implementation plan”.—[Official Report, 21 March 2016; Vol. 623, c. 772.]

She also said that that was one of her prime commitments, and I was pleased to hear that from her at the Dispatch Box. However, the evidence we received for the report suggested that the Department of Health does not intend to develop an implementation plan and believes that NHS England should assume responsibility for doing that, so will the Minister confirm who will introduce the implementation plan, as promised last week?

NHS England also suggested in evidence that it does not intend to develop an implementation plan and has neither the remit nor the capacity to influence all 51 commitments in the strategy. Given that evidence, will the Minister commit to finding a way either for the Department of Health to deliver that agenda or for NHS England to be given direction to do so?

[Jim Shannon]

I thank the hon. Gentleman for being gracious in giving way. He will be aware of the neuromuscular complex care centre that was set up in Queen Square in London in September 2014. Will he encourage the Minister to visit that centre to see the benefits of co-ordinated, specialist, multidisciplinary care delivery that could be replicated across the United Kingdom of Great Britain and Northern Ireland?

[Ben Howlett]

I am sure that the Minister heard that and will pass it on to the Under-Secretary of State for Health, my hon. Friend the Member for Oxford West and Abingdon.

We also saw evidence of poor communication between the organisations responsible for implementing individual commitments and other stakeholders involved in the strategy. What plans does the Minister have to better co-ordinate that communication? Many patients do not have access to the appropriate treatment or information about their condition, and the barriers to accessing information about a condition begin as soon as a diagnosis is made. Unfortunately, that leads to patient care continuing to be poorly co-ordinated.

I know that the Minister is a superb champion for those with rare diseases, and given the rare condition that the Under-Secretary of State for Health, my hon. Friend the Member for Oxford West and Abingdon, has, I hope that he provides more information about the Government’s commitment from last week. Hopefully they can commit to the Department of Health developing a comprehensive implementation plan that describes actions for its arm’s length bodies in particular. An implementation plan would offer direction to bodies involved in rare disease patients’ care, so that they can take action to improve the services that patients need. It would help patients to understand what progress is being made and to ask the right questions when it is not. It would also help to ensure that the needs of rare disease patients and their families are considered in decisions about patient care and access to treatments taken by bodies involved in the strategy.

[Chi Onwurah (Newcastle upon Tyne Central) (Lab)]

I congratulate the hon. Gentleman on securing this important debate. I welcome the recommendations in the strategy, although I am concerned that the recent announcement on capping NHS drug prices will influence implementation. Muscular Dystrophy UK has said that it would have a major impact on drugs for muscular dystrophy and other rare diseases. Newcastle is fortunate enough to be home to the John Walton Muscular Dystrophy Research Centre, but support could be improved further by providing specialist psychological support for people with muscle-wasting conditions. Does the hon. Gentleman agree that such support needs to be an integrated part of service provision for muscular dystrophy and other rare diseases?

--- Later in debate ---

[Ben Howlett]

I am really pleased that the hon. Lady raised that point, and I again pay tribute to the work done by Muscular Dystrophy UK to support patients. A key recommendation from our inquiry addressed the fact that part of the rare diseases strategy suggests that we should look at things on a much more integrated level. Patients are not just the medical condition that is attributed to them; they are also an entire person, who is part of a collective family. The rare diseases strategy has implications not just for those individuals, but for their families, carers and so on, across the board. We have seen a number of instances where the rare diseases strategy is simply not doing what it should have been doing, so I hope that in the Minister’s summation he will address taking a holistic approach, rather than just looking at the individual.

Further recommendations in the APPG report include that NHS England should be more proactive in implementing the commitments it can influence and dedicate more resources to improving the co-ordination of care, as the hon. Member for Newcastle upon Tyne Central (Chi Onwurah) said. It recommended that the Department of Health should improve its processes to both engage and communicate with stakeholders in the strategy. It also recommended that the Department of Health and the UK National Screening Committee should work together to establish robust programmes for identifying and preventing rare diseases, and that training for frontline medical staff on rare diseases and their impact on patients should become widely available and incentivised. The APPG is very much encouraged by the number of programmes that have been developed in response to the strategy that complement its aims, such as the 100,000 Genomes Project. Those programmes are amazing in themselves; none the less, those developments should not necessarily be considered as actions resulting from the UK strategy for rare diseases.

The time to act is now. I am sure that the Minister can guess my final point, which is about the changes to the National Institute for Health and Care Excellence’s highly specialised technologies programme. Commitment 13 of the strategy—lucky for some—is to ensure

“that there are appropriate procedures for evaluating the costs and benefits of treatments for patients.”

NICE’s recent decision to implement an upper funding threshold for its HST programme, made despite widespread condemnation from the rare diseases community, conflicts with that aim. The upper limit will vary according to the lifelong impact of the technology on the patient, varying from £100,000 per quality-adjusted life year for treatments that deliver less than 10 QALYs to the patient in their lifetime, up to a maximum of £300,000 for treatments that deliver more than 30 additional QALYs to the patient in their lifetime.

Unfortunately, the programme has been beset by delays. Only four medicines for the treatment of rare diseases have been evaluated since the HST programme began, averaging just one a year, despite a capacity for three treatments a year. The four medicines evaluated to date have been shown to bring tremendous value to the patients eligible to receive them. They are life-saving, life-lengthening treatments with the potential to lift the burden on whole families of a rare genetic condition.

[Greg Mulholland (Leeds North West) (LD)]

I thank the hon. Gentleman for securing this debate and for his leadership on the issue. On that particular point, does he agree that, as well as being a kick in the teeth for the rare and ultra-rare disease community—families, medical staff and charities—the plans make no sense? They have no bearing on the effectiveness of the drugs, which surely should be the basis for decisions, and they threaten new drugs that could change and save people’s lives, as well as some of the drugs that we joined families, medical staff and campaigners to get in the first place.

[Ben Howlett]

I thank the hon. Gentleman for his intervention and pay tribute to him for his work on rare diseases over the years. One of the first events I ever went to in Parliament was on rare diseases and was hosted by him. I could not have put it better myself. I will come later to my view that the issue needs to be thought about in a more timely way, potentially in a consultation and through some sort of implementation plan, which has been missing. It will impact not just people with muscular dystrophy, for example, but all those with the different conditions that he has championed in the past.

The four medicines evaluated to date have shown tremendous benefits to patients, and are life-saving and life-extending treatments. They are some of the most powerful and effective treatments for rare diseases ever seen, and the highly specialised technology evaluation committee recommended them for funding in England, but none of those life-changing medicines would have been able to raise the lower threshold significantly, and none would have been approved under the new regime.

If implemented, the plans will significantly affect patients with rare diseases and their ability to access life-changing treatment, at a time when we should be expanding access routes rather than limiting them further. The changes contradict the positive recommendations made in the accelerated access review and will restrict any attempt through the industrial strategy to position the UK as a centre for the development of innovative medicine. England already has extremely slow and limited access to treatments for rare genetic conditions; further narrowing of access routes will shut the door to innovation for our community of patients and families. It is unacceptable to implement such drastically damaging proposals just 18 days after they were announced. I therefore join the sector in calling for a pause in implementing the proposals and for a consultation and impact assessment.

[Chi Onwurah]

I thank the hon. Gentleman for his kindness in giving way once again. The John Walton Muscular Dystrophy Research Centre was named after a peer, now sadly deceased, who also founded Muscular Dystrophy UK and saw over a long lifetime the importance of the UK’s role in innovative science and research techniques, and its economic benefits to the research sector and, more widely, our international reputation. The changes proposed to NHS England will do much to undermine that.

[Ben Howlett]

I think the sector agrees on that point, from patient groups to the constituents whom the hon. Lady represents, as well as all the people with an undiagnosed condition who might access the services of that facility in future and many others throughout the country. The Government need to rethink the implementation and ultimately introduce an impact assessment on that basis.

I know that several hon. Members want to speak, so I will conclude by welcoming the commitment and dedication of this Minister and the Under-Secretary of State for Health, my hon. Friend the Member for Oxford West and Abingdon, in providing a solution to our long-standing problems with the implementation of the rare diseases strategy in England. Following the publication of our report, I hope that the Minister will be able to provide clarity on the questions that I have asked. The UK is doing superb work and leading internationally on rare diseases.

[Andrew Bingham (High Peak) (Con)]

I commend my hon. Friend on securing this important debate. My constituent Will Newman contacts me regularly about his granddaughter Ellie, who lives outside my constituency. He wanted me to come to this debate and thank my hon. Friend and the Minister for the work that they are doing. We think that rare diseases do not affect many people, but those whom they affect, they affect hugely. Does he, like me, take heart from the Prime Minister’s words in September? She said at Prime Minister’s questions:

“We are committed to ensuring that patients with rare conditions get access to the latest medicines”. —[Official Report, 7 September 2016; Vol. 614, c. 334.]

As she also said, we are taking the necessary steps to get those to them.

[Ben Howlett]

I thank my hon. Friend for that intervention. He is a great champion for his constituents. I share his hope in the Prime Minister’s commitment, made at an early stage in her office, and in a Minister who obviously understands rare diseases, having one herself, and who has made a clear personal commitment to resolve the issue. Having worked alongside the Department of Health for many years, I have been wanting to see this come to fruition. I am glad that we now have a leadership commitment in place to deliver it for the first time, at least in my memory of working alongside the NHS.

The UK is doing superb work and leading internationally on rare diseases. I hope that our all-party parliamentary group’s report will make a significant difference and help to steer the Department of Health to a place conducive to both the Minister’s requirements and ours. I look forward to hearing his response.

[Mark Pritchard (in the Chair)]

Before I call Margaret Ritchie, I will give some guidance. I am sure that hon. Members are aware that the spokesman for the Scottish National party will have five minutes, as will the shadow Minister. The Minister will have 10 minutes. This debate is due to finish at 5.30.

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[Mr Dunne]

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.

[Ben Howlett]

indicated assent.

[Mr Dunne]

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 is 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.

[Ben Howlett]

I will be brief. I thank the Minister for his response. The news about the implementation plan being brought in before the end of the year is incredibly welcome, and I thank him for taking leadership on that. That is a message to go back to my hon. Friend the Member for Oxford West and Abingdon (Nicola Blackwood) while she is with the Prime Minister. I appreciate that it is potentially a lot nicer to be with the Prime Minister than here. Nevertheless, the Government’s position is welcome.

I thank all Members for contributing to the debate. I particularly thank the hon. Members for South Down (Ms Ritchie), for Cambridge (Daniel Zeichner), for Linlithgow and East Falkirk (Martyn Day) and for Washington and Sunderland West (Mrs Hodgson) for their supportive comments. The time is now. The all-party parliamentary group, working with Genetic Alliance UK and Rare Disease UK, which provide the secretariat to the APPG, will be monitoring the progress that the Minister, the Department and NHS England make over the next few months. Progress will be incredibly welcome. The Minister referred to the highly specialised technology change and explained how the consultation has operated, and I appreciate that, but I reiterate that there is concern within the sector. The Ministers may be able to reassure people that that concern is being taken into consideration.

For a long time I have said that the accelerated access to medicines review might be a good pathway for rare diseases in the future. The work that the former Minister for Life Sciences, my hon. Friend the Member for Mid Norfolk (George Freeman)—he is now chair of the Prime Minister’s policy unit—has done and continues to do will be incredibly important. Thank you, Mr Pritchard, for chairing the debate. I thank the Minister for updating the House, and I also thank Members and all those who gave evidence and support to the APPG in its inquiry.

Question put and agreed to.

Resolved,

That this House has considered implementing the UK Strategy for Rare Diseases.