Implementing the UK Strategy for Rare Diseases in Parliament

There was an informative but rather disappointing debate on the UK Strategy for Rare Diseases on 28 March 2017 in Westminster Hall, which is used as an alternative to the House of Commons chamber. The debate was initiated by Ben Howlett MP who is chairman of the All-Party Parliamentary Group on rare, genetic and undiagnosed conditions.


Central to Ben Howlett’s speech were the deficiencies of the HST system:

“NICE’s recent decision to implement an upper funding threshold for its HST programme, [was] made despite widespread condemnation from the rare diseases community….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…. 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.”

The Minister of State for Health, Philip Dunne, did not dispute what Ben Howlett had said, but gave no ground. He referred to the new thresholds as being ten times greater than what NICE used for mainstream appraisals, that it was not possible to predict how likely any individual new drug would be recommended by NICE under the changes in the future and that “even when NICE is not able to recommend a drug for the full patient population, NHS England may still be able to fund a drug for a subgroup of patients who will most benefit from treatment.”

UK Strategy for Rare Diseases

The one positive main point that the Minister announced was in respect of the 2013 UK strategy for rare diseases:

“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.”

Although No 13 of these commitments is to “Ensure that there are appropriate procedures for evaluating the costs and benefits of treatments for patients”, the wording looks sufficiently broad to allow the HST changes to remain unchanged.

The transcript of the Rare Diseases Strategy Debate is now available online.

To read more about this, please click here.

Tagged with

Published on 29. March 2017 in News UK