Recombinant Products Over Plasma-Derived

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.

In response to 9e, all diseases or conditions where a non-plasma treatment exists to replace a plasma/blood-derived treatment are now addressed by commissioning policies or funding agreements. There are some specific regimens which are subject to ongoing clinical policy development but in each case there are alternative regimens or treatment approaches which negate the use of plasma-derived medicines. Of particular interest to the clinical and patient community is the development of a clinical commissioning policy for recombinant Von Willebrand factor, which is currently licensed for prophylaxis in adults, as regular treatment for those with the severest bleeding, but which is not currently commissioned for this indication. Funding will be required to implement this clinical policy for all ages, and this has not yet been identified.