My Lord’s, I shall speak to Amendments 178 and 240, and I remind the Committee of my interest as chair of the Specialised Healthcare Alliance.
Amendment 178 deals with innovative medicines and medicinal products. The debate on this amendment is very timely, given the recent conclusion of NICE’s review of its methods and processes and the current consultation on the innovative medicines fund. Both the review and the IMF consultation are to be welcomed. It is clear that they will result in improvements in the system of assessing medicines and medicinal products, particularly in respect of analysing and addressing uncertainty and incorporating more real-world evidence into decision-making. However, it is also clear that both the IMF proposals and the outcome of NICE’s review are at risk of falling short of the hopes of many patients, clinicians and the life sciences sector more generally in a number of important ways.
First, there is still a lacuna in NICE’s approach to considering treatments for rare disease. For ultra-rare diseases—those affecting fewer than 1,000 people—NICE retains its highly specialised technologies process. For more common conditions—those affecting more than 25,000 people—NICE has its separate technology appraisal process. But for patients with rare diseases—those affecting between 1,000 and 25,000 people—there is no process, and so treatments for these patients have to be considered instead through the unsuitable technology appraisal process. This gap sets us apart from other countries, such as the more generous ASMR system in France and the AMNOG system in Germany for evaluating rare disease treatments.
It was therefore very disappointing to see that the case for the rare disease modifier was again rejected in NICE’s review. It was rejected on the grounds that society does not value treatments for rare disease more highly than those for more common diseases. Those representing rare-disease patients would contend that the fact is that these treatments are inherently costly. The Government accept this in relation to ultra-rare disease, so why do they not do so for rare disease treatments? I would be grateful if the Minister could address that specific question when he replies.
Secondly, NICE’s own consultation looked favourably on reducing the discount rate at which NICE assesses the future costs and benefits offered by a treatment, saying that such a change
“could make a particularly big difference to some treatments, like gene therapies.”
However, NICE has now said that this change would not be possible, due to the views of “system stakeholders”, and this has disappointed many people. When the Minister replies, I would be grateful if he could expand on what “system stakeholders” really means in this context. Who is NICE talking about and why did it assign conclusive weight to their views?
Thirdly, the system in England still fails to formalise the input of patients and clinical experts in the way that, for example, the SMC in Scotland does through its patient and clinical engagement process.
Finally, proposals for the innovative medicines fund now move far beyond the originally planned narrow focus on autoimmune and rare diseases. This causes some SHCA members to worry that rare diseases will get less attention than originally envisaged.
These proposals fall short of the hoped for bridge between the MHRA’s licensing process—which reforms are speeding up in some cases—and NICE’s reimbursement process. Without such a bridge, earlier licensing will not deliver benefits to NHS patients, and ultimately companies will lose interest in making bespoke licensing applications to the MHRA. The Government’s own figures—the life sciences competitiveness indicators, published by the Office for Life Sciences—demonstrate that it is already the case that the per capita uptake of new medicines remains lower and slower in this country than in comparable countries.
Our Amendment 178 suggests that the Government review the situation by the end of the year, when we will have a good half year of experience of the changes to NICE and the operation of the IMF, and when we will be able to see that the hoped for improvements have materialised. I hope that the Minister will consider this suggestion.
I now turn to Amendment 240, which seeks to probe the Government’s actions to improve awareness of rare diseases among healthcare professionals. There are more than 7,000 rare diseases, and it would clearly be impossible for every healthcare professional to receive training on every single one of them. However, as the Government’s rare disease framework notes, healthcare professionals can improve their awareness of rare diseases more generally, be more alert to considering them and be provided with the educational resources that help them recognise rare diseases in patients. Healthcare professionals can also be better supported to help signpost patients with rare disease to information about their condition and to help them understand it.
In a 2016 survey by Rare Disease UK, it was found that 70% of patients were not provided with sufficient information on their condition following diagnosis, and that 35% of patients given information did not understand the information that they were given.
More recent surveys demonstrated that these challenges continue. The Government’s national conversation on rare diseases in 2019 found that almost one in five
people living with a rare condition reported that a lack of healthcare professional awareness of their disease was the number one challenge that they faced, and healthcare professionals themselves identified it as the second biggest challenge they faced behind only the well-known difficulties in obtaining an accurate diagnosis. I accept that healthcare professional regulators can do only so much to make improvements, but it would be helpful to understand from the Minister what steps they might be able to take to help better embed rare disease content in training frameworks.
Finally, there is a wider question of how the Government currently track progress in increasing awareness of rare diseases among healthcare professionals. How do the Government do that? I look forward to the Minister’s reply.