NICE Appraisals: Rare Diseases Treatments Speech
21 March 2019
I am delighted to support the motion standing in the names of the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon). This is a life-changing issue for thousands of our fellow citizens who simply do not have time on their side. A number of my constituents, including the parents of my young constituents battling with cystic fibrosis, have brought to my attention the weaknesses in the current NICE appraisal model. I look forward to reinforcing the arguments of the hon. Member for Blaydon with some details that they have provided.
NICE’s appraisal model has led to a horrendous block on life-changing cystic fibrosis drugs being made available to those young people. Vertex’s three approved medicines and investigational triple regimen may be able to treat the underlying cause of cystic fibrosis for up to 90% of patients. There is currently no cure for cystic fibrosis, and half of people with the disease will die before they are 31. I recommend that my hon. Friend the Minister instructs NICE to review its current single technology appraisal, which is used to appraise inherited rare diseases, in order to come to a solution that can work best for all parties. The current model, which specifically affects Vertex drugs such as Orkambi, is fundamentally flawed. It directly affects the lives of not only my young constituents suffering from cystic fibrosis, but those with spinal muscular atrophy, Batten disease and PKU, to name but a few.
NICE’s single technology appraisal has been used for the past 20 years, and although it served as an important new way to assess the cost-effectiveness of new treatments, it has failed to keep pace with advances in science. No model should be biased towards favouring specific medicines, but there remains an unwillingness to accept that new precision medicines that treat the underlying cause of disease and have the potential to extend life are fundamentally different from the medicines that existed when NICE’s processes were first developed. The idea of working on an innovative new model for appraising rare diseases is also supported by the Genetic Alliance.
When performing a single technology appraisal, NICE applies the same methodology and cost-effectiveness criteria regardless of whether it is appraising a single-use medicine for an acute condition or a lifetime medicine for an inherited, progressive, incurable, life-limiting disease. The current evaluation process turns on the incremental cost-efficiency ratio, measured in quality-adjusted life years. With acute conditions resulting from shorter-term treatment, the ICER is moderated even if the drug is very costly. Conversely, with chronic and lifelong conditions, the drug must be taken every day for life, and the cost of lifelong treatment prevents downward moderation of the ICER. That means that, when evaluating medicines that extend life, those that treat conditions from which patients would die within a short period are favoured over those that would extend life far into the future.
That unfairness is doubly compounded by the fact that, when computing the number of quality-adjusted life years attributable to a treatment, NICE usually applies a “discount rate” of 3.5% per annum, based on the Treasury’s Green Book, on both the costs and health effects of the medicine by reference to how far into the future those life years will be added to the patient’s life. In essence, the longer a patient lives, the more expensive they are to the system and the higher the cost per quality-adjusted life year.
Let me give an example. If a treatment were projected to extend the life expectancy of a six-year-old cystic fibrosis patient from 47 to 57 years, the present value of those additional 10 years would be less than two once they were discounted. By comparison, an oncological orphan medicinal product may add five life years, starting immediately, to a patient’s life expectancy, so discounting would reduce those five years to 4.66 for the purpose of calculating quality-adjusted life years. That approach cruelly fails to account for the fact that every year of additional survival, regardless of whether it is gained in the short or the long term, will be valued equally in the mind of a cystic fibrosis patient and his or her family.
To add insult to injury, NICE currently does not take into account the fact that when medicines lose their market exclusivity after patent expiry, their cost to the NHS falls dramatically, typically by 80% to 90%. It is unrealistic to assume that a medicine would remain at its currently listed price over the entire model horizon, particularly when that can be upwards of 40 years. There is no reason why NICE could not model the effect of a post-patent expiry price reduction by reference to available evidence from the pharmaceutical market. That is yet another example of NICE’s discrimination against treatments for chronic and incurable conditions in favour of those for acute conditions.
Finally, while NICE recognises that medicines for very rare diseases—ultra-orphan medicines—need a higher threshold and more discretion in the way in which they are appraised, it does not allow cystic fibrosis medicines to be judged against that threshold. That is because, although cystic fibrosis is a rare disease globally, its prevalence in England is such that NICE insists that it is appraised via the conventional approach.
Vertex is not the only manufacturer of precision medicines for rare diseases to experience challenges with NICE. Both the Bioindustry Association and the Association of the British Pharmaceutical Industry have, on behalf of their members, repeatedly highlighted the need for NICE to be reformed, to take a broad view of the value of new treatments and innovations to the health service, and to incorporate a wider range of factors and flexibilities, beyond the standard cost per quality-adjusted life year gained. It is right for us to ensure that NICE processes are modern and up to date with the evolution of precision medicines.
My constituent Sharon Cranfield is a bit disappointed with a letter sent to the Minister by the Chairman of the Health Committee, outlining the conclusions of the Committee’s public hearing on 8 March. She says that the points that I had raised on her behalf appear not to have been considered and the findings of the Committee seem to lie with continuing to defend the NICE model that has been used for the last 20 years and an unwillingness to accept that they need to be re-evaluated to reflect the current and near term developments in precision medicines.
I understand that the Committee may have advisers who were associated with the setting up of NICE. I think that Ministers, NICE, and everyone who is engaged in this should look forward to a model that will actually work for the people whom we represent.
NICE must re-evaluate the way in which it values rare disease medicines. I sincerely hope that following today’s debate, it will do more to achieve alignment on value, evidence and price for the sake of patients, and will address, once and for all, the limitations of the current NICE STA process for diseases such as cystic fibrosis. That would also benefit all the other patients who already suffer enough after being diagnosed with a rare disease.