In October 2016, I was honoured to present some research at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congress in Vienna. HJCL collaborated with RJW & Partners to conduct a comparative study of recent NICE and SMC decision-making. We wanted to understand to what extent, if at all, the two organisations arrived at different recommendations about a medicine when presented with the same evidence base, and then to explore what the reason for those differential decisions might be. Our research was well received, winning an award for Best General Podium Presentation!

We reviewed a total of 184 SMC decisions published between January 2013 and May 2016. 54 were not recommended due to a non-submission and were not reviewed further. Where the product involved a resubmission (10 in total), the outcome of the resubmission was assumed to supersede the earlier “not recommended” advice. The remaining published decisions were then cross-referenced to the relevant NICE Single Technology Appraisal (STA) or Highly Specialised Technology (HST) guidance.

After applying the exclusion criteria, 120 decisions were analysed, of which 19 (~16%) resulted in substantively different recommendations between NICE and the SMC. All 19 involved full submissions to the SMC. NICE recommended products in 15 of 18 decisions, while the SMC rejected 10 of those 18. Three of the 10 products rejected by the SMC have all been recommended under the NICE HST process for highly specialised technologies. A nineteenth decision resulted in the product being recommended by both organisations, but for different patient populations.

Our research showed that NICE and SMC decision-making wass broadly aligned, but that substantively different recommendations could result for products licensed for specialised, rare or orphan indications. Through the HST programme, NICE had more flexibility than the SMC in its decision-making methodology to recommend highly-specialised, ultra-orphan technologies at that time. The results also highlighted the challenges of applying PACE, disease modifiers and end of life criteria to products that blur the lines of orphan/ultra-orphan definitions or which are targeted at niche therapy areas.

NICE and SMC methodologies and pathways have evolved since our original research was conducted in 2016; in particular, the SMC has introduced a designated pathway for ultra-orphan medicines.  So, we’re planning a five-year update to ascertain what, if anything, has changed in terms of the decisions reached. Watch this space!