Two years on from reforms to the Scottish Medicines Consortium’s processes for evaluation drugs for end-of-life and rare conditions implemented in 2014, Dr Brian Montgomery was asked by the Scottish Government to review their impact on access. The result was a wide-ranging report covering multiple aspects of drug access, with recommendations for action based on consultation with a broad range of stakeholders.
One of the recommendations relates to drugs to treat ultra-orphan conditions. These drugs are evaluated against a separate, multi-criteria non-scored framework, which has an analogue in NICE’s Highly Specialised Technologies (HST) route; it includes the cost-per-QALY where it’s available, but also considers additional factors. Montgomery finds that positive funding decisions for drugs for ultra-orphan conditions have not risen in the same way as drugs for less rare conditions. As a result, there is a recommendation is to take ultra-orphan funding decisions out of the SMC process, with SMC providing only the assessment; the intention appears to be to increase the number of these drugs accepted for funding.
CASMI’s own data finds the same result in an analysis of the past 2 years’ funding decisions in Scotland. Only 14 of 21 ultra-orphan drugs were recommended for funding, compared to over 85% for all drugs. This is specifically an issue outside cancer – 13 of 15 cancer ultra-orphans were funded (87% recommended or restricted) compared to 1 of 6 non-cancer ultra-orphans (17%). Perhaps unsurprisingly, ultra-orphans are under-represented in the ‘restricted’ category (equivalent to NICE ‘Optimised’) – further stratification would be challenging in an already small population.
In contrast, NICE has appraised and approved 3 of the six non-cancer ultra-orphans for use in England. NHS England (NHSE) accepted a fourth via Specialised Commissioning, but did not fund the other two. For the 3 with NICE and SMC figures available, the costs per QALY were estimated at over £500, 000 (from the published appraisals, and CASMI estimates). Hence it would appear that in these cases, the funding regime in England has been more permissive than in Scotland. Confusingly, NHS England did not accept the one drug that was accepted by Scotland, despite a low base case ICER of under £6000 per QALY (with upward uncertainty); perhaps if this drug had gone through NICE’s HST programme the decision would have been different.
The story continues to evolve. NICE and NHSE are consulting on a de facto threshold for Highly Specialised Technologies in England: under the proposal, a drug for these ultra-rare conditions with a cost-per-QALY under £100 000 will automatically receive routine funding, with treatments above this range being assessed for funding by NHSE. Whilst this can be interpreted as a noticeably higher threshold for these ultra-orphan drugs than the standard £20 000-£30 000, we note that none of the three HST drugs described above would have been accepted for funding, and would have been considered by NHSE’s prioritising process instead. It is not clear whether NHSE will be more or less permissive than NICE, although given the remits of the two organisations we might expect budget impact to be given more weight by NHSE.
So Scotland appears to be aiming to increase access, whilst there are concerns that England’s proposals could work in the opposite direction. Will the two countries diverge? The answer depends on who Scotland hands the decision-making power to, and the parameters considered in that decision. If that choice rests with NHS Scotland, we may in fact see convergence between the two countries, as the decisions then reside in two national healthcare providers with (we assume) similar philosophies, objectives, and budget constraints. We will continue to follow developments as each government develops its response to their various consultations.
– Dr Liz Morrell