Rare Diseases Strategy

Greg Mulholland Excerpts

Tuesday 28th March 2017 Westminster Hall

[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.