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Letter to Jeremy Hunt MP

Last Reviewed 29/03/2017

On Monday 27 March 2017, together with the help of our members we sent a letter to Jeremy Hunt MP asking for a halt to proposed changes to the highly specialised technologies programme. The full text of the letter, sent on behalf of 198 patient organisations, is below. 

Dear Secretary of State,

Urgent call to pause changes to NICE’s Highly Specialised Technologies Programme

I am writing to you in your role as Secretary of State for Health to ask for a halt to the implementation of NICE and NHS England’s proposed dynamic upper-limit for highly specialised technologies (HST), which will have a devastating impact on patients and work against the Government's goal of accelerating access to innovative medicines.

We oppose these proposed changes because:

  • England already has extremely slow and limited access to treatments for rare genetic conditions. Further narrowing of access routes will in practice 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 are announced. We call for a pause in implementation, for an impact assessment on the proposals and for a comprehensive consultation with stakeholders.
  • These changes are contradictory to the positive recommendations made in the Accelerated Access Review, and will limit the impact of any attempt through the Industrial Strategy to position the UK as a centre for the development of innovative medicine.

Fourteen days ago, NICE announced measures to limit recommendations by its Highly Specialised Technologies Evaluation programme. These proposals will halt future access to innovative treatments for rare genetic conditions in England, and send a chilling message to the life sciences sector, that runs contrary to messages from other parts of Government. We are calling for a pause in the implementation of these proposals, planned for 1 April 2017, to ensure that NICE’s and NHS England’s approaches are in step with what UK patients and families affected by rare genetic conditions need.

Genetic Alliance UK is the national charity working for all those affected by genetic conditions. We are an alliance of 198 patient organisation members. There are more than 6,000 genetic conditions, affecting more than three million people in the UK. Very few of these conditions have effective cures or treatments. Our members look towards research and the innovative treatments that follow as the route to a better future for people affected by genetic conditions.

If implemented, NICE’s latest proposal for a cost per quality adjusted life year (QALY) gained threshold for the highly specialised technology (HST) evaluation programme will severely limit access to innovative treatments for rare diseases in England.

NICE’s original proposals were criticised because none of the four treatments recommended by the pathway to date would have been funded under the proposed threshold of £100,000 per QALY gained. NICE’s response has been to define a new system where the threshold could in theory be raised. We believe NICE could have been clearer when presenting the latest recommendations, specifically stating that none of the four treatments evaluated to date would have been judged sufficiently valuable to significantly raise the threshold above the originally proposed £100,000 per QALY gained.

The sliding threshold only begins to expand when treatments deliver additional QALYs above 10. The most additional QALYs demonstrated so far by any of the four treatments assessed to date has been 10.14, far below the 30 additional QALYs required to take the threshold to its maximum of £300,000 per QALY gained. In practice therefore, the threshold has not been increased at all, and the position remains that none of the four treatments recommended by the HST programme to date would be funded under the new proposals.

The proposed methodology deserves scrutiny and discussion. In previous HST evaluations, companies’ estimates of additional QALYs have varied dramatically from those of NICE’s Evidence Review Group (ERG). In the first HST evaluation, the company estimate of 25.22 additional QALYs would have given a threshold of £252,200 per QALY gained; the ERG estimate of 10.14 would have set the threshold at £101,400 per QALY gained. Clearly these are differences large enough to make or break access. NICE has never before based thresholds for decision making on additional QALYs.

The QALY gains delivered by the four medicines that NICE has evaluated are already substantially higher than those for more common indications, where the additional QALYs delivered are frequently less than 1.0. These innovative therapies are already outperforming the rest by up to an order of magnitude. Raising the bar further is unjust and unjustifiable.

The four topics approved to date bring tremendous benefit to the patients eligible to receive them. These are life-saving, life-lengthening treatments which have the potential to lift the burden of a rare genetic condition from a whole family. They are some of the most powerful and effective treatments for rare diseases ever seen and, under previous methodology, the HST evaluation committee was able to recommend them for funding in England.

Under the new methodology, the committee will no longer be able to exercise such judgement, leaving patients in England with no route to access through the NHS.

Access routes for medicines for rare genetic conditions are already beset by delays and limitations. These proposals take us from a bad situation to crisis point.

To date 143 medicines have been authorised by the European Medicines Agency for rare diseases. England provides routine funding for just 68 of these. This is fewer than five new treatments a year during the fifteen years of the Orphan Medicinal Product Regulation in Europe. England is far behind Germany (routine funding for 133), France (for 116), Italy (for 84) and Spain (for 75) in both the number of treatments funded and in the speed of those funding decisions.

The capacity of the HST programme has already proven insufficient leading to long delays in access. The programme had its first meeting in December 2013, and with a supposed capacity of three topics per year has only published guidance on four topics to date. Since January 2015 alone, there have been 16 technologies referred to scoping for an HST evaluation. Even more technologies that fit the scope for HST evaluation, in terms of population size and innovative treatment method, are being referred for single technology appraisal.

The alternative routes to funding for rare disease medicines not evaluated by NICE are governed by NHS England. They are the annual specialised services commissioning round, individual funding requests, and the clinically critically urgent use process, amongst others. None of these processes are run in a transparent way, nor do they function in a predictable or timely manner. The most likely source of a consistent commissioning decision in NHS England, the annual specialised services commissioning round, has never yet delivered its recommendations on time (i.e. before the beginning of a commissioning year) yet, and in 2016 delivered its funding recommendations eight months late.

England already has extremely slow and limited access to treatments for rare genetic conditions. Further narrowing of access routes will in practice shut the door to innovation for our community of patients and families.

Our members work hard to fund, support and participate in research studies aimed at understanding and treating more rare genetic conditions. We want to be able to benefit from investment in the UK’s life sciences sector, and to participate in the development of new innovative treatments in the UK.

The existing environment for funding of innovative treatments in the UK is already deterring investment from biotech firms and pharmaceutical companies. The uncertainty surrounding Brexit poses a further threat to the attractiveness of the UK as a place to perform research and to develop innovative cures and treatments for rare genetic diseases.

To reverse this trend, the Accelerated Access Review has made a series of welcome recommendations, supported by the Green Paper: Building Our Industrial Strategy. We need to be working to preserve and improve the UK’s position as a centre for innovative life science. We have a broad range of rare genetic condition patient communities in the UK, who are already engaged in research, experts in their conditions, and in an excellent position to collaborate on new innovative approaches to deliver treatments. The NHS is the perfect environment for this work to happen.

NICE’s proposals send a clear message contradicting the Accelerated Access Review’s recommendations and the Government’s vision for the UK life sciences sector as outlined in the Industrial Strategy.

It is unacceptable to implement such drastically damaging proposals just 18 days after they are announced. We call for a pause in implementation and for a consultation and impact assessment on the proposals.

These proposals vary significantly from those proposed in NICE’s original consultation. In practice, they are no less damaging than the original proposals which were so strongly rejected by the patient community during the consultative process.

In their original proposal, NICE did not transparently state the impact of their proposals – that no technology previously recommended by an HST evaluation would meet the new criteria. Again, with their new proposals, NICE have not transparently indicated that despite the changes made to the proposals following the consultation, still no technology previously recommended by an HST evaluation would have been recommended under the new regime.

The HST process was designed to evaluate the effectiveness of treatments for very small patient populations without relying on calculations of cost per QALY, recognising that these health economics metrics do not adequately capture the benefit a treatment can offer to patients and families affected by a rare condition.

Such a large change in scope, output and design of this important process deserves a more detailed conversation than has been possible in the 14 days since this announcement was made.

We need to align NHS England and NICE’s negative messages to the innovative life sciences sector with the Government’s work to nurture the sector. We believe some time to reconsider these proposals is a first step towards a coordinated approach to building a positive future for patients and families affected by rare genetic conditions in the UK.

Yours sincerely,

Alastair Kent OBE



This letter was sent to: Rt Hon Jeremy Hunt, Secretary of State for Health; Professor Sir John Bell, leading on the life science component of the Industrial Strategy and was Chair of the Accelerated Access Review’s Expert Advisory Group; Nicola Blackwood MP, Parliamentary Under Secretary of State for Public Health and Innovation; Sir Andrew Dillon, Chief Executive, National Institute for Health and Care Excellence; Lord O'Shaughnessy, Parliamentary Under Secretary of State for Health; Lord Prior of Brampton, Parliamentary Under Secretary of State for Business, Energy and Industrial Strategy; John Stewart, Acting Director of Specialised Commissioning, NHS England; and Sheela Upadhyaya, Associate Director of the Highly Specialised Technology programme, National Institute for Health and Care Excellence.  

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