NICE Appraisals: Rare Diseases Treatments

Liz McInnes Excerpts

Thursday 21st March 2019 Commons Chamber

[Liz McInnes (Heywood and Middleton) (Lab)]

It is a pleasure to contribute to this important debate. I thank my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon) for obtaining this debate, and the Backbench Business Committee for granting it.

Before I was elected to this House I worked for more than 30 years as a clinical scientist in our NHS. During that time, I developed a healthy scepticism for politicians advising on medical treatments, as it is a field that is best left to clinicians. However, I did want to take part in this particular debate because I know that issues around the availability of life-saving drugs and treatments for rare diseases are important to many people. My constituents have made me particularly aware of the unavailability of Orkambi to cystic fibrosis sufferers and of Kuvan to those affected by phenylketonuria.

Today’s debate is also extremely valuable because it focuses on the way NICE operates and questions whether that operation is appropriate for those suffering from rare diseases. A rare disease is generally defined as one affecting fewer than five people in 10,000, but many people are affected by rare conditions. The UK strategy for rare diseases estimates that, in the UK alone, more than 3 million people will suffer from a rare disease at some point in their life.

Given, however, that relatively few people are affected by a particular rare disease, there are specific challenges in ensuring speedy diagnoses and access to appropriate services and treatments. NICE’s technology appraisal process involves looking at evidence from clinical trials and peer-reviewed research showing how well a medicine or treatment works, including its likely impact on mortality and quality of life; at the economic evidence of how much it costs the NHS; and at the views of clinicians, patients and other stakeholders.

As well as looking at the clinical effectiveness of a treatment, single technology appraisals and highly specialised technology evaluations also assess its cost effectiveness, which, as many speakers have said, is usually measured in terms of the cost per additional quality-adjusted life year, and this is assessed by looking at how many extra months or years of life of a reasonable quality a person might gain as a result of treatment.

Following changes introduced in April 2017, NICE set a maximum additional quality-adjusted life year threshold of £300,000 for highly specialised treatments. Under that threshold, they would automatically be approved for routine commissioning. This is 10 times higher than the standard NICE threshold of £30,000 for non-specialised treatments. Owing to the nature of lifelong genetic diseases, however, the required quality of life improvements are likely to be unobtainable. The charity Genetic Alliance UK highlights that no ultra-orphan treatments—drugs used to treat extremely rare diseases—currently used by the NHS would pass this test.

It is clear that an urgent rethink is needed on these policies, which are focused almost exclusively on price, to the detriment of patient outcomes. Genetic Alliance UK has argued that these policies will halt future access to innovative treatments for rare genetic conditions in England and that they contrast with the stated aim of the UK strategy for rare diseases to ensure appropriate procedures for evaluating the costs and benefits of treatment for patients.

The problem with the current NICE appraisal process is that certain treatments, particularly those designated as orphan medicines, are neither eligible under the narrow criteria of the highly specialised technology process nor appropriate for the single technology appraisal process. The reason could be that the treatment’s patient population is marginally higher than the maximum size considered through the highly specialised technology process, while the single technology appraisal process is inappropriate for most orphan drugs because of limited trial data.

The accelerated access review, which aims to speed up access to innovative drugs, devices and diagnostics for NHS patients, recommends that NICE undertake a review of its methods and processes to ensure that they are fit for purpose, which I think everyone in the Chamber would agree is long overdue. The review warns that

“it is important that no groups of products can ‘fall between the cracks’ and struggle to find a decision-making process”.

We have heard heartfelt speeches today from my hon. Friends that have illustrated that some products are falling through the cracks and that families and young children are suffering.

The views of the accelerated access review are in line with those of the NHS five year forward view, which includes a recognition of a broad measure of value that goes beyond price alone. I have combed through the latest NHS long-term plan, and I cannot find any reference to this important issue. When the Minister responds, I would be grateful if he pointed out to me where the issue is mentioned in the most recent iteration of the plan.

NHS England and NICE need to reconsider how they account for rarity in their assessment process to support the NHS in its mission to provide a comprehensive service that is available to all at the point of need, including to those with rare conditions.