Skip to main content

NICE Methods consultation

Final consultation, will it work for rare conditions?

After two years of hard work the last chance to input into the changes NICE are proposing to improve their decision making on medicines has come.


The consultation page is now live and ends on 13 October 2021. Engagement events coming up are:

HST Criteria proposals

NICE has two current pathways that can be used to assess treatments for rare conditions: their standard Single Technology Appraisal (STA) pathway, and the Highly Specialised Technology (HST) evaluation pathway. HST has been designed for treatments for rare conditions that might be disadvantaged by the STA process, so it is crucial that the right treatments are selected.

The old criteria were confusing and applied inconsistently. NICE proposes four criteria to replace the previous seven. There is more clarity here, but the route is still too narrow to be able to assess all treatments for rare conditions that might be disadvantaged by the normal STA pathway, which was designed for all medicines.

The key changes here are available in the topic selection proposal paper in paragraph 47 onwards.

The top lines for the new proposed criteria are:

I. The condition is very rare defined by 1:50,000 in England

II. Normally no more than 300 people in England are eligible for the technology in its licensed indication and no more than 500 across all its indications

For one off treatments generally a small prevalent population of up to 50 patients and an incidence of no more than 40 patients a year would normally be considered acceptable

III. The very rare condition significantly shortens life or severely impairs its quality

IV. No satisfactory treatment options exist, or, if it does the technology is likely to be of significant additional benefit to those affected

Changes to STA

The other half of the picture is how STA might be changed to become fairer to medicines for rare conditions. If this ambition is reached, the current imperative to be selected for HST is reduced. The changes here fit into a few different boxes.

Severity modifier – this will allow NICE to assign greater value for treatments for severe conditions – this is a positive for rare conditions, as they are often severe. This replaces an end of life modifier which never benefited rare conditions.

Managing uncertainty – the idea here is that committees will have more flexibility to deal with uncertainty. If this is done right then treatments for rare conditions would benefit, as there is usually uncertainty about the value of these treatments due to small populations for trialing them.

Accepting more evidence – this will allow NICE to accept more evidence from people living with the condition concerned.

Health inequalities – this is an important issue that needs to be addressed to counter some of the challenges described in our APPG meeting on access to medicines by representatives of the Sickle Cell Society and UK Thalassemia Society. We are disappointed an inequality modifier has not been possible at this stage, but NICE have committed to taking this forward in the future.

Discounting rate – This is a technical issue, but a crucial one relating to how valuable long term benefits are seen to be. Treatments for rare conditions might deliver benefits beyond thirty years or more, and so we are disappointed that there has not been progress made on this issue. We understand that this is because of wider government influence against this change. NICE are clear that there is evidence to reduce the discount – which would be a positive change for our community – and that they want to make the change, but must wait until the wider issues are resolved

Preliminary thoughts

The middle of August is an unfortunate time to launch such a crucial consultation, and the timing has meant we have not had as much time to review this in advance as we would have liked. We will carry on our analysis and bring you more thoughts as they come. This consultation will be a key topic in our Policy, Policy, Policy! check in meeting on 7 September at 11:00 (Note different time for one week only!)

Our top line reaction now is that the work by NICE and stakeholders does look like it will bring meaningful change, but probably not to the extent that we might have hoped in Autumn 2020, when there was a strong sense of ambition. We therefore need to give a robust response to NICE restating our ambition and the imperative for a step change in improvements.

We will want to be robust in the areas of managing uncertainty, the new HST criteria and on health inequalities, we will want to call for the best possible outcomes for rare conditions in the evidence base changes and the severity modifier, and we will need to keep the pressure on to drive the change of the discount rate.

Please join us at 11:00 on 7 September for the ‘Policy, Policy, Policy!’ members check-in meeting. Email: [email protected]create new email.

Join our alliance of 200+ SUPPORT ORGANISATIONS AND charities