New rules at NICE may block treatments for those with rarest conditions

Having spent six years developing processes for evaluating treatments for rare diseases, leading the Highly Specialised Technologies (HST) programme at NICE and developing Health Technology Assessments with the Advisory Group for National Specialised Commissioning, I am all too familiar with the challenges in assessing high-cost, low-volume treatments with a limited evidence-base. Like many, I was eagerly awaiting the latest proposals from NICE and NHS England setting out changes to the HST programme.

Previous attempts at evaluating treatments aimed at very rare conditions have looked at several criteria, in part due to the difficulty of generating a reliable cost per quality-adjusted life year (QALY) on the basis of limited data, about both the disease and the treatment. Indeed, so far NICE has avoided publishing cost per QALY figures, though it has been moving towards this.

Even supposing methodological hurdles can be overcome, is the proposed £100,000 per QALY an appropriate figure for treatments aimed at very rare conditions?

That may depend on which treatments are included in the HST programme going forward. The HST programme and its predecessor have typically looked at life-long treatments for very rare, disabling, often life-limiting conditions with no more than a few hundred patients in England. These treatments can be both lifesaving and life transforming but do not typically restore a patient to full health. The cost per QALY is therefore far higher than the cost of treatment and the proposed £100,000 threshold would likely exclude the products evaluated to date, even if generous confidential discounts are in place.

The proposals are clear that there will be another route for funding these treatments through NHS England’s prioritisation process. In theory, reuniting decisions about treatments with decisions about services could enable more rounded decision-making. However there remain concerns about NHS England’s ability to give fair consideration to treatments for those with rare diseases when they are competing against often cheaper treatments for more prevalent conditions. NHS England will need to work hard to provide reassurance that patients with rare diseases will not find themselves at the back of a very long queue as priority is given to more cost-effective technologies.

In addition to the QALY threshold for automatic funding, NICE and NHS England are proposing an affordability threshold of £20 million, beyond which NHS England and the company would be forced to negotiate. In some ways this formalises the already commonplace use of managed access agreements for HST, giving clarity on when such agreements would be expected. However it is concerning that there is no mention of a role for patients and clinicians in developing such agreements.

As companies come to prepare submissions to the HST programme, most will be anxious about what this will mean for their prospects for reimbursement at sustainable prices. It is likely that evidence submissions and committee meetings will come to more closely mirror those found in Technology Appraisals.

The devil, as always, is in the detail and inevitably at this stage there are more questions than answers. The rare disease community will be hoping that, as more details emerge from the consultation, we see a system striving to support the timely and sustainable introduction of effective new treatments for rare diseases that retains the belief that the NHS should leave nobody behind.

Josie Godfrey was formerly Associate Director, Highly Specialised Technologies at NICE and led the establishment of the HST programme. Prior to joining NICE, Josie was Head of Policy at AGNSS.

Written By

Josie Godfrey