Recombinant Products Over Plasma-Derived

UK Government

Recommendation 9e: NHS England, working with clinical advisors, has reviewed the existing gaps in the availability of recombinant factors, and other blood product alternatives, and is currently developing clinical commissioning policies for these indications. As a rapid response to this recommendation, in August 2024, NHS England commenced funding recombinant Von Willebrand factor (VWF), for all patient age groups, to manage bleeding episodes and surgical pre-treatment (but not regular prophylaxis). Further clinical policy work, which includes reviewing the clinical evidence base, cost effectiveness and service implementation factors, is required relating to the use of recombinant products for prophylaxis. Work is currently underway on the following:
Recombinant VWF: currently licensed for prophylaxis in adults (regular treatment for those with the severest bleeding) but not currently commissioned for this indication.
Emicizumab as prophylaxis in people with moderate congenital haemophilia A without factor VIII inhibitors.

Funding will be required to implement these clinical policies, and this has not yet been identified.

Scottish Government

These recommendations have largely been implemented in Scotland. The great majority of bleeding disorders patients in Scotland are already provided with recombinant products rather than plasma-based ones. However, in relation to provision of the recombinant product vonicog alpha (known as Veyvondi) for children under 18 years old with von Willebrand disease, given the medicine is not currently licensed for use for under 18s, the Chief Pharmaceutical Officer for Scotland wrote to Scottish Health Boards in October 2024 to ask them to ensure it is prescribed for children where this is appropriate for them in line with recommendation 9e).

Welsh Government

The recombinant coagulation factor Vonicog alfa is routinely available in Wales for the treatment of haemorrhage and surgical bleeding, and for prevention of surgical bleeding, in people with a confirmed diagnosis of von Willebrand disease (VWD) in accordance with the NHS Wales Joint Commissioning Committee policy.

The All Wales Medicines Strategy Group (AWMSG) endorsed One Wales interim decision which extends routine use to children up to 17 years with VWD.

The IBI Oversight Group has recommended Vonicog alfa should also be made available for long term prophylaxis against bleeds in people with VWD. Officials are considering the implications of implementing this recommendation and are working with the Joint Commissioning Committee and All Wales Therapeutics and Toxicology Centre (AWTTC) to prepare advice for consideration by Ministers later this spring.

Northern Ireland Executive

In relation to Recommendation 9e), the Department of health has a formal link with the National Institute for Health and Care Excellence (NICE) under which NICE Technology Appraisals are reviewed locally for their legal and policy applicability in Northern Ireland, and where applicable, they are endorsed for implementation within HSC organisations. As such, in practice, treatments that have been recommended by NICE for routine use in the NHS in England are also routinely available in Northern Ireland.

The Health and Social Care (HSC) Managed Entry of New Medicines process applies NICE recommendations as policy. In the absence of a NICE recommendation, guidance from the Scottish Medicines Consortium (SMC) or All Wales Medicines Strategy Group (AWMSG) advice can be applied on a discretionary basis.

Where such guidance is not available as described above, an Individual Funding Request (IFR) process will provide a mechanism to consider requests from clinical consultants for treatments for individual patients that are not routinely commissioned, and which are deemed as clinically exceptional. Funding will be on an individual patient basis only, but where a cohort of 3 or more patients may potentially benefit from a treatment, this then will fall out of scope of the IFR process.

In the specific case of vonicog alfa (Veyvnodi) for long-term prophylaxis of haemorrhage in adults (licensed indication) or children (unlicensed indication) with von Willebrand disease, in the absence of NICE, SMC or AWMSG guidance, there is no route in Northern Ireland under extant arrangements to make this treatment routinely available within the HSC. However, the Department of Health will remain abreast of any developments arising from England, Scotland or Wales and give their advice due consideration.