The Haemophilia Society responds to NICE Consultations

Written by Aaron Dennis, March 11, 2021

Starting in 2020, NICE has been running a series of consultations looking at their processes for health technology evaluations. While new technologies, such as Emicizumab for Haemophilia A, have been adopted recently we have concerns with how NICE reviews treatments and worry that their processes unnecessarily delay access to new treatments.

NICE is a government body that reviews newly licensed medical technologies and decides whether they should be used by the NHS in England. They review how effective a new treatment is and decide whether it is cost effective. If they make a positive recommendation then there is an expectation that the technology will be made available to patients, however, sometimes access will be delayed while the NHS works out how to affordably roll out access to the new treatment.

There are several different routes to approval that new technologies can take, but recently NICE has been involved in reviewing Emicizumab (Hemlibra) for Haemophilia A, Coagadex for Factor X deficiency and recombinant VWF for treatment of VWD. While all these treatments have eventually been given positive decisions, new treatments may only be approved for certain groups of patients or be given other restrictions on access, such as we saw for extended half-life factor VIII and IX products.

The first consultation, which started last year, was on what issues they need to consider, then on the methods they use and finally, the processes they will use to put these methods into practice. In general, the consultations were initially on what needs to change and then moved on to how they can achieve those changes.

As well as patient groups such as The Haemophilia Society they have also been seeking the view of Government, the NHS, Academia and the pharmaceutical industry.

The need for this work is driven by increased need for NICE to review medical technologies as well as new types of technologies such as Gene Therapies likely to be licensed in the coming years.

The latest stage of the consultations close in mid-April with NICE committed to publishing and beginning implementation of the new methods and processes from September 2021.

So far, The Haemophilia Society have commented on how the methods could better consider new treatments for rare diseases, which we feel the current systems disadvantage. We call for better use of disease-specific quality of life measures, more reliance on patient reported outcomes and real-world evidence and a need to have methods that are more efficient and scalable. You can read our full response to the methods part of the consultation here.

NICE have also been consulting on their guidance on Shared Decision Making. The Haemophilia Society also responded to this consultation. Our comments focus on ensuring patients are provided with detailed yet accessible information on which to base their decisions. Also, in light of previous failings, we also emphasised the importance of appropriately making patients aware of the risks of treatments, including risks that are as yet unquantified or only understood theoretically.