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Changes to the HST programme

NICE in close collaboration with NHS England have decided to propose a dynamic upper-limit for costs per QALY above which they will not recommend funding for highly specialised technologies (HST) treatments.

We welcome NICE’s willingness to make changes to their proposals following the consultation process. That this process remains independent from the NHS England decision making process is an improvement to proposals. The proposed upper threshold has also risen to a maximum of £300,000 per QALY gained for treatments which bring a truly life-changing outcome for patients.

However, the changes are not enough to prevent these proposals from drastically changing the landscape for innovative treatments for very rare diseases in England. Actual prices paid by the NHS are confidential, so it is impossible to be certain, but we do not believe any of the four medicines that have gained funding since the HST programme began in 2013 would have been approved under these proposals.

This is a clear message from NICE and NHS England that they are unwilling to continue to pay for innovation treatments for rare diseases to the extent that they do now.

This would make a bad situation worse: decisions on rare disease treatments in England are already slow and restricted; these proposals further narrow the window for funding of life saving treatments.

The upper-limit will vary according to the life-long impact of the technology on the patient, varying from £100,000 per QALY per year for treatments that deliver less than 10 QALYs to the patient in their lifetime (additional QALYs); up to a maximum of £300,000 for treatments that deliver more than 30 additional QALYs to the patient in their lifetime.

So far the programme has approved life-changing treatments for four conditions. None of these treatments would have been able to significantly raise the lower threshold.

  • Atypical haemolytic uremic syndrome – eculizumab. List price £327-340k per year – additional QALYs: 10.14
  • Fabry disease – migalastat List price £210k per year – additional QALYs: 0-0.98 (though there is already a treatment for this condition).
  • Mucopolysaccharidosis type IVa – elosulfase alfa List price £398k per year – additional QALYs: 5.04-10.03
  • Nonsense mutation Duchenne muscular dystrophy – ataluren List price £220k per year – additional QALYs: 1.9-8.6

Though actual prices paid are confidential, we have previously been told by NICE that these treatments would not have been approved at a £100,000 threshold.

Each one of the treatments recommended by NICE for these four conditions adds a great deal to both the quality and length of the patients’ life. We do not think that the new process will be able to continue to bring treatments to patients and families that are able to deliver the same transformative impact as the first treatments delivered through the NICE evaluation process.

The threshold, which varies between £100,000 and £300,000 per QALY gained, is an arbitrary selection. We are uncomfortable with a decision which will potentially exclude a significant proportion of our members from accessing innovative therapies being based on a number plucked out of the air.

We encourage NICE to carry out further analysis on the likely outcome of drawing the threshold at this point, so that the impact of this choice can be assessed to patients and families currently living with untreated rare diseases.

We are concerned that the proposal for an upper limit on cost per QALY gained for highly specialised technology evaluations will be over-reliant on health economic calculations that are prone to large subjective variations and that do not reflect the value of treatments to families affected by rare diseases. 

This is very technical proposal and we are concerned with the reliance on QALY calculations that this brings to the HST programme. The programme was designed to be able to operate without using QALY calculations as there are significant challenges relating to the estimation of these health economic measures in the field of rare diseases due to the small data sets used. More importantly, the programme was designed to be able to take into account a much broader set of evidence that a QALY calculation is capable of representing.

The committee are able to take into account the broad range of affects on families that a rare disease can have, and which may be solved by a treatment under consideration. This ability might be moot with the implementation of these proposals.

There are 6,000 rare diseases affecting 3.5 million people in the UK. There is an effective treatment for only a handful of these, and many of those treatments have not been commissioned for delivery in the NHS. These measures further reduce the likelihood of innovative treatments becoming available for the patients and families we represent.

The restrictive proposals, alongside an already restrictive regime at NHS England for innovative rare disease treatments, will mean that more patients and families in the UK will not receive the life changing treatments that they need. Alongside proposals for potential delays to implementation of NICE guidance for treatments which fail the £20 million budget impact test, this sends a negative message regarding the UK’s attitude to innovation in healthcare.

These actions are contradictory to the positive recommendations made in the Accelerated Access Review, and will be restrictive to any attempt through the Industrial Strategy to position the UK as a centre for the development of innovative medicine.

UK patients currently benefit from clinical trials and other research programmes sponsored by industry here. In a time of political uncertainty, when some of the key positive attributes that the UK has to offer industry are being eroded, the UK should not be restricting its investment in innovative industry. If we do not maintain the positive environment for clinical research in the UK, patients will have reduced access to potentially life-changing clinical trials.

The ultimate aim of the measures announced today is to assist the NHS which is struggling to deal with costs in the face of multiple health challenges in the UK. A great deal of the focus to solve this issue has been on restricting investment in new proposals and innovative technology. We believe at least as much energy, thought and effort should be put into assessing the potential of disinvestment in NHS activity that may not bring significant benefit to patients.

We expect patients and patient representatives to be involved in the development and implementation of these recommendations.

NICE have admitted that the threshold chosen was arbitrary. We would like to see analysis of the impact of this choice, and facility to adjust this, up or down, in the future to react to those findings.

NICE have observed that the introduction of this cap brings the methodology of the HST evaluation closer to the methodology of their Technology Assessments. In light of this we would like to see serious consideration of an expansion of the scope of the HST evaluation process. This could allow there to be more than three HST evaluations a year, and for the process to consider rare disease treatments for populations larger than 1,000 patients in England.

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