Rare Diseases Strategy

Daniel Zeichner Excerpts

Tuesday 28th March 2017 Westminster Hall

[Daniel Zeichner (Cambridge) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Pritchard. I congratulate the hon. Member for Bath (Ben Howlett) on securing this debate and on his excellent work as chair of the all-party group.

My interest in this subject has been stimulated by visiting a number of key research sites in and around Cambridge, where vital work is being done to develop new treatments. The Sanger Institute has the world-famous and world-leading 100,000 Genomes Project and works closely with the adjoining European Bioinformatics Institute. Because of Brexit, both institutes face major problems with retaining key staff and ensuring data flows, although that is a debate for another day. We also have the National Institute for Health Research’s biomedical research centre, which is led by Dr John Bradley and based at Addenbrooke’s hospital.

I recently joined Dr Bradley and Professor Lucy Raymond, professor of medical genetics at Cambridge, to celebrate Rare Disease Day at an illuminating roundtable with patients and practitioners. We heard case studies that highlighted some of the very difficult challenges that patients with rare diseases face, including practical difficulties with the benefits system. I was particularly struck by meeting a patient who suffered from lupus. She explained that she had good days and bad days—something that the welfare system just cannot cope with, making her already difficult life even harder. The message that I came away with—apart from the important links to the genetic sequencing work being done at the Sanger and the need to identify the right people to test—was that research into rare diseases not only helps with those diseases, but leads to discoveries with a much wider application.

I will focus my comments on new treatments and on how to ensure ready access to them. As we have heard, this debate is very timely because it comes hot on the heels of recent NICE guidance that introduces a financial cap for access to new medicines. NICE is proposing an upper limit for costs per quality-adjusted life year, above which it will not recommend funding for highly specialised technology treatments. That decision could have a profound impact on people affected by rare diseases in the UK. On Thursday, when I asked the Leader of the House for a debate on the matter, he replied that

“decisions about individual medicines are taken by a body such as NICE, where clinical judgment is to the fore, rather than being subject to political pressure”.—[Official Report, 23 March 2017; Vol. 623, c. 966.]

None the less, I am glad to have the opportunity to raise the matter today. I agree that it should be for NICE to decide, but this is a significant change from medical efficacy to financial rationing. Such a highly political change should be debated here, not outsourced.

We must listen to what charities such as Alzheimer’s Research UK and Cancer Research UK are saying. They warn that lives will be lost because of this new rationing system. I was particularly struck by the words of the MPS Society, which provides support to people affected by mucopolysaccharide diseases. It said that the decision by NICE and NHS England

“will affect the most vulnerable in UK Society and confirms that children and young adults with ultra-rare diseases going forward are economic pawns in a failing NHS and cheaper dead than alive.”

Those are very strong words. They are not mine, but they show how people feel, and I am sure that the Minister will have noted them.

Genetic Alliance UK said:

“NICE, in close collaboration with NHS England, are sending a clear message that they are unwilling to fund new innovative treatments for rare diseases. This is a real slap in the face for patients and families with rare diseases, and contrary to the founding principles of the NHS.”

It has also suggested that, as we have already heard, none of the four rare disease medicines that have so far been approved would have been approved under the new proposals, which it believes will cause a

“drastic reduction in access to innovative treatments for very rare diseases in England”.

It is also worth mentioning that the guidance will have an impact not only on people affected by rare diseases, by closing off their access to innovative treatments, but on the UK’s vital life sciences sector more broadly, as it risks curtailing investment in new medicines. As the BioIndustry Association says, it sends

“an immediate, stark, negative signal to the global life science investors and companies that the UK needs to attract in the Brexit era.”

The reaction from patient groups, as well as from industry, demonstrates the broad concern about the adverse impact that NICE and NHS England’s decision will have on patients. The UK strategy for rare diseases published in 2013 has the laudable aims of raising awareness of rare diseases and improving diagnosis, research and access to services for people affected, but there is a real risk that those aims will be undermined by the new guidance.

I echo the calls from the hon. Member for Bath and other hon. Members for the Department of Health to publish an implementation plan for the UK strategy for rare diseases, because until we see a coherent plan, progress on the strategy’s objectives will continue to stall. As the all-party group has rightly summarised:

“An implementation plan…is a tool for delivery, coordination, collaboration, communication and monitoring.”

Such a plan would also enable stakeholder groups to have a clear idea of which institutions they could work with to influence the implementation of the strategy.

We have already heard today that the Minister responsible for public health and innovation, the hon. Member for Oxford West and Abingdon (Nicola Blackwood), has made a personal commitment that the UK rare diseases strategy should be translated into an implementation plan. I applaud her for that commitment, but I hope the Minister will confirm that the Department will follow through on it, because last year the then Health Minister, the hon. Member for Mid Norfolk (George Freeman), stated in a written answer:

“There are no plans to consult on the implementation plan”.

The Government have described the UK strategy for rare diseases as

“a long term strategic vision for improving the lives of all those with rare diseases and conditions”

and have committed

“to ensuring that patients with rare and ultra-rare diseases in England are able to access effective treatments.”

I fear that recent decisions will mean that access to those effective treatments will be shut off. The Government must act urgently if they want their strategy to move beyond paper and reach the patients and people it aims to help.