It is a pleasure to serve under your chairmanship, Mrs Miller. I draw attention to my entry in the Register of Members’ Financial Interests.
NICE was established to determine cost-effectiveness, and it quickly became the best at this in the world. Given the pandemic, the importance of the life sciences sector to our country has never been clearer. If we want to retain our global reputation, however, NICE needs to alter the way it works. The rare diseases framework should be seen in this context. Its themes of pioneering research and being a global player are the right ones. I am encouraged that NICE recognises the challenges we face. That said, I am not yet convinced that NICE has got to the right place on the detail. Unless we get it right, we risk reducing the number of new medicines arriving to treat patients.
Within the framework, priority 4 is all about improving access to specialist treatments. The review has identified the need to change and update the discount rates, but less helpful are the suggestions in the current process review consultation on how we evaluate new health technologies. Essentially, there are two routes: the regular single technology appraisal process; and the highly specialised technology option, which is far more flexible. Medicines for rare diseases need that flexibility and the higher threshold for cost-effectiveness. If they do not meet the HST criteria, new technologies are stifled. NICE is risking needlessly complex and convoluted criteria that will not allow for transparency on why particular medicines are put into the programme. I hope it will register the concerns expressed by the ABPI and others.
The Medicines and Healthcare Products Regulatory Agency is talking about new pathways to licences. Such work needs to be joined up and supported by NICE’s processes. Early engagement is positive, but NICE must avoid premature decisions, including about the commercial aspects and pricing.