Ed McIntosh takes a look at the past, present and future of the Highly Specialised Technologies programme
NICE’s Highly Specialised Technology (HST) programme has just turned five years old. A less distinguished milestone than NHS 70 – but, given the life-expectancy of an acronym in the health service these days, merely surviving for this long is an achievement in itself.
There have been other achievements too. NICE can point to a higher rate of approvals compared to the STA programme, including a UK-first recommendation for a gene therapy, and a good record on patient and clinical involvement.
However, birthday celebrations at Piccadilly Plaza are likely to have been somewhat muted, as despite these achievements, the HST programme has not lived up to expectations, either from NICE itself or from patient organisations and industry.
Only seven pieces of final guidance have been published in five years, a glacial pace of publication, even in the ultra-rare disease space. And in almost every case, the development of guidance has been dogged by disruption and delays.
Incisive Health has a uniquely informed perspective on the HST process, having worked on four of the seven products which have successfully navigated it – and working now on many of the products which are progressing through it today. As a result, we can draw six lessons from how it has worked in the first five years of its operation – and which help us understand how it may evolve in the future.
1. HST is not purely an assessment of cost-effectiveness, despite recent reforms
Prior to 2013, recommendations on the adoption of ultra-rare disease treatments were made the health ministers’ Advisory Group for National Specialised Services (AGNSS). AGNSS used a form of multi-criteria decision analysis (MCDA), of which cost formed one element, alongside efficacy, societal value and efficiency of delivery.
When legislation in 2012 formally removed ministers from the decision-taking processes, AGNSS’s functions passed to NICE. NICE initially retained aspects of the existing approach, but in 2017 this was replaced with an approach that is ostensibly based on a ‘floating’ cost-per-QALY threshold of between £100,000 and £300,000.
Although these reforms may suggest that health economic calculations are now the sole test in the HST process, this is not the case. In practice, because clinical trials for ultra-rare treatments involve such small patient populations, black and white assessments of cost-effectiveness cannot be made – and subjective opinion is still an important factor in NICE’s decisions. The failure in practice of a definitive cost-effectiveness test for HSTs is something which will not be lost on either the Government’s or the industry’s side during the current PPRS negotiations (or, indeed, in Scotland, which has rejected crude cost-effectiveness tests in its new ultra-orphan pathway).
2. NHS England’s role is constantly evolving
When the HST process was set up, NHS England had only just come into being. In the face of significant capacity and capability challenges, it adopted a passive role in the HST process: indeed, NHS England’s involvement in the earliest HSTs was notable only for its absence. Following the appointment of Simon Stevens as its Chief Executive in early 2014, however, NHS England has become increasingly, but inconsistently, involved.
Initially, it sought an active role throughout individual HST evaluations. Then, it turned its attention to more systemic changes – triggering the reforms in 2017 which introduced the cost-effectiveness test. Latterly, it has elected to stand aside until NICE seeks its involvement at a late stage. In the future, NHS England’s approach may change again: indeed, it is fair to say that with each new HST evaluation to date, NHS England’s approach has shifted.
There is a reason why NICE doggedly maintains ‘interim’ processes for HST evaluations: five years in, it believes that it is still too early for these to be finalised.
3. Complex commercial deals are the norm, and slow down the process
The small patient populations which create such trouble when NICE is trying to determine the cost-effectiveness of ultra-rare treatments nonetheless lend themselves to NHS England’s desire to agree what can be very complicated commercial arrangements with manufacturers. These deals often come in the form of a ‘managed access agreement’ informed in part by the views of patients and clinicians.
NHS England’s starting point for commercial discussions with manufacturers is therefore that such offers will be put on the table. Small patient populations mean that it is easier to track patients for the purposes of ‘outcomes-based’ commercial deals – but NHS England’s appetite for such arrangements is at risk of outstripping its capacity to agree them. This can cause frustrating delays for manufacturers
4. A positive evaluation is not necessarily the conclusion of the process
Manufacturers may be forgiven for thinking that positive final guidance from NICE will deliver the automatic NHS funding of their therapy that it should – but this is not always the case, even with a commercial deal agreed by all parties. It has always been possible for the NHS to argue successfully that the implementation of NICE guidance should be delayed whilst the necessary services are put in place, and the HST process is not immune to this
5. Mis-scoping happens and can be rectified
The criteria for determining entry to the HST process are not simple, and are open to interpretation – leading to sometimes questionable scoping decisions. For those manufacturers of treatments for ultra-rare treatments who find themselves in the standard ‘STA’ process, this is an understandable problem. However, NICE has proven itself capable of changing its opinion when provided with the appropriate evidence before the all-important ministerial referral.
6. A successful HST process requires multi-faceted engagement
If the HST process was truly a crude test of cost-effectiveness, then the arena would be one monopolised by health economists. Health economic perspectives are undoubtedly important, but there are other arguments that manufacturers can marshal to make the case for funding. In a typical HST evaluation, manufacturers need to be prepared to make arguments that (i) ensure ministers make a referral into the HST process in the first place, (ii) expedite the decision-taking process on both NHS England’s and NICE’s side once the evaluation has commenced, (iii) ensure NHS England’s prioritises its scarce commercial negotiating capacity and adopts a flexible approach to commercial negotiations when these commence, and (iv) implements positive HST guidance once it arrives as rapidly as possible.
Where next for HST?
The future of the HST programme is uncertain. NHS England and NICE may insist that further reforms are unlikely in the short term, however the fact that it exists only in ‘interim’ form, and that the crude cost-effectiveness test is so manifestly incapable of supporting decision-taking, means that pressure to review the current approach may become impossible to resist. In the meantime, manufacturers and the NHS will need to muddle through, and prepare themselves for every eventuality they may encounter on the journey to a positive decision.
Once the dam bursts, and substantive reform is a likely prospect, we might hope for further changes along the lines of that taking place in Scotland – where a system based around granting conditional access to all new ultra-orphan medicines will be introduced from October this year. The inclusion of reforms in a ‘final’ methods and process guide might provide manufacturers with some much-needed certainty on how the HST process works for the next five years of its existence at least. Expecting any reform to fix the HST process for the next 70 years is, perhaps, more ambitious.
Published HST guidance is available on NICE’s website:
- Strimvelis (Autologous CD34+ enriched cell fraction transduced to encode for human adenosine deaminase (ADA)) for treating adenosine deaminase deficiency–severe combined immunodeficiency
- Strensiq (asfotase alfa) for treating paediatric-onset hypophosphatasia
- Cerdelga (eliglustat) for treating type 1 Gaucher disease
- Galafold (migalastat) for treating Fabry disease
- Translarna (ataluren) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
- Vimizim (elosulfase alfa) for treating mucopolysaccharidosis type IVa
- Soliris (eculizumab) for treating atypical haemolytic uraemic syndrome