News and Blog

News and Blog



NICE approach to assessing orphan and ultra-orphan drugs – details are now emerging

NICE approach to assessing orphan and ultra-orphan drugs – details are now emerging

The National Institute for Health and Care Excellence (NICE) is now the agency with the tricky task of assessing and appraising orphan and ultra-orphan drugs. The change was announced back in July 2012, and in May 2013 details were set out on their Interim Process in Board Papers. 

Making decisions on drugs that tend to be very expensive (they can cost upwards of £100,000 but not all are this expensive) yet treat only a small number of patients is not new and nor is it easy.  NICE has already looked at some orphan drugs and set out their thoughts in 2006 (but this was not implemented).  Back then NICE felt that their usual approach, including the cost per Quality Adjusted Life Year (QALY), was successful for many orphan drugs. The challenge came from ultra-orphans to treat really rare diseases (defined by NICE as treating diseases with a prevalence of less than 1:50,000).  Their solution at the time was to not to modify their approach but to set out separate ‘decision rules’; that meant a higher cost effectiveness threshold based on what was already on the UK market.  That translated to £200,000 to £300,000 per QALY.

Not everyone was happy about the move to NICE. This is especially because the cost per QALY can be very high for ultra-orphans and orphan drugs leading to concerns that they would simply fall foul of the NICE threshold of between £20,000 to £30,000 per QALY (although higher thresholds can sometimes be allowed for end of life).  Others are concerned that separating out decision making for services and drugs will fragment care for those with rare diseases, or that NICE just doesn’t have the expertise that’s needed when dealing with diseases that simply don’t occur often and hence few clinicians and others really know and understand them and their treatment.

Some may be a little relieved that the Interim Process from NICE includes a range of criteria:

• Nature of the condition including extent and nature of current treatment options
• Impact of the new technology from clinical effectiveness to robustness of the evidence base
• Costs to the NHS and Personal Social Services including patient access agreements
• Value for money
• Impact of the technology on the delivery of the specialised service

NICE also says that they; ”will take into account what could be considered a reasonable cost for the medicine in the context of recouping manufacturing, research and development costs from sales to a limited number of patients.”

But in developing the approach feedback from invited experts has stressed the need to avoid just taking the cost-utility approach. As a result of testing the Interim Process NICE has also added more on the particular context for these drugs, acknowledging that; “Given the very small numbers of patients living with these very rare conditions a simple utilitarian approach, in which the greatest gain for the greatest number is valued highly, is unlikely to produce guidance which would recognise the particular circumstances of these vary rare conditions. These circumstances include the vulnerability of very small patient groups with limited treatment options, the nature and extent of the evidence, and the challenge for manufacturers in making a reasonable return on their research and development investment because of the very small populations treated.”  

NICE also has patient engagement and involvement running throughout its work, so it may give more patient organizations a chance to give their views.  But that could be limited; some of these diseases may not actually have a patient organization and many patients may never had had to get involved with NICE before.

The Interim Process is not the last word though, as NICE is expected to conduct a fuller consultation.  That’s everyone’s chance to input and have their say on how best to assess and appraise medicines that can change and save lives for those with rare conditions.

Leela Barham is an independent health economist and can be reached at She is the author of Orphan drugs: Special Treatment Required? available at:


    Add your comment

    Please enter the word you see in the image below:


    Recent Articles

    Latest Tweets

    Press Enquiries

    Please contact James Hargrave at JBP PR or telephone 0203 267 0074 for any press or event related enquiries.