New clotting products approved for haemophilia patients

Extended half-life (EHL) recombinant Factor VIII and Factor IX will be added to the National Blood Authority list of clotting factor concentrates this year

The decision by the Authority to add them to the next national tender for clotting factors and related products follows an assessment of the products by the Medical Services Advisory Committee (MSAC) in 2018.

MSAC recommended that the EHL products would replace standard half-life (SHL) Factor VIII and Factor IX for the prevention of bleeding in patients with haemophilia A or B.

“However, MSAC noted that for on-demand use and surgical prophylaxis, there is insufficient evidence to support a clinical claim for EHL relative to SHL products,” the Committee’s Public Summary Document said.

The Committee said the longer half-life particularly for Factor IX meant higher trough levels could be maintained for longer with the corollary of a meaningful reduction in dosing frequency for patients and improved adherence to prophylaxis.

There was also some but limited evidence that bleeding rates were improved with both EHL Factor VIII and Factor IX compared to their SHL counterparts.

MSAC noted there was no data to suggest that EHL products were associated with a higher rate of adverse events than SHL products in either haemophilia A or B.

“For prophylactic use, MSAC concluded from the evidence available that there was little clinically-important improvement in patient outcomes between the two types of products, but accepted that EHL products would reduce dosing frequency.”

Feedback during public consultation suggested that the benefits of fewer infusions and fewer bleeding episodes could have a large impact on both patients and their parents or carers.

The National Blood Authority advised that interim arrangements for temporary access to EHL products for about 200 haemophilia patients with high priority needs remain in place for now.

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