NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE

Scope to inform NHS England’s specialised commissioning of:

Susoctocog alfa for treating episodes in adults with acquired caused by antibodies to factor VIII

FINAL

August 2017

Background Acquired haemophilia caused by antibodies to factor VIII is a rare disorder that occurs when the immune system produces autoantibodies that inactivate factor VIII. Factor VIII is one of the proteins needed for normal clotting of the . The disorder generally develops later in life. Approximately half of people having an underlying condition, including cancer, autoimmune conditions such as rheumatoid arthritis and systemic lupus erythematosis, or inflammatory bowel disease. In 50% of cases the condition is idiopathic with no specific associated underlying condition. Increasingly because of advanced age, patients with acquired haemophilia have significant co-morbidities that impact on treatment choices and increase the risk of adverse events. The pattern of bleeding differs from that seen in congenital haemophilia. In contrast with congenital haemophilia, subcutaneous bleeding including extensive sheet bruising and other soft tissue bleeding are common features. Mucosal bleeding such as gastrointestinal bleeding may occur and muscle bleeds are also common. Bleeding may be life or limb threatening and severe bleeding episodes commonly occur, requiring emergency treatment with haemostatic agents. However, the severity of the condition can vary, with approximately 25%-30% of patients not requiring therapy to control bleeding. In the UK, around 120 people are diagnosed with acquired haemophilia caused by antibodies to factor VIII each year (National Haemophilia Database).

Susoctocog alfa is a recombinant porcine derived DNA derived factor VIII replacement therapy that works by replacing the depleted factor VIII in a person’s body and enabling the blood to clot. Its efficacy is based upon lack of cross reactivity between antibodies against human factor VIII and recombinant porcine factor VIII. Additionally, it provides clinicians an ability to measure patient plasma levels to titrate dosing and thus optimise efficacy. The scope of this work is specifically focusing on the treatment of bleeding episodes in adults with acquired haemophilia caused by autoantibodies to factor VIII.

Remit To support the development of an NHS England specialised commissioning policy on susoctocog alfa within its marketing authorisation for treating bleeding episodes in adults with acquired haemophilia caused by antibodies to factor VIII

NHS URN 1703 / NICE ID006 Page 1 of 2 NICE commissioning support draft scope for susoctocog alfa for acquired haemophilia caused by antibodies to factor VIII Intervention Susoctocog alfa (Obizur)

Population Adults with acquired haemophilia caused by antibodies to factor VIII who require a haemostatic agent to manage a bleeding event

Company Shire

Regulatory status On 11 November 2015, susoctocog alfa received a marketing authorisation and licensed from the European Medicines Agency for the treatment of bleeding indication episodes in patients with acquired haemophilia caused by antibodies to Factor VIII Comparator(s) • Activated prothrombin complex concentrate (FEIBA) • Recombinant activated factor VII (NovoSeven)

Outcomes • Percentage of patients achieving complete resolution of a bleed • Frequency of dosing • Time taken to achieve resolution of a severe bleed The following outcomes are included as standard and will be considered where evidence allows: survival; progression free survival; health related quality of life (including mobility; self-care; usual activities; anxiety/ depression); replacement of more toxic treatment; dependency on care giver/supporting independence; safety (including adverse effects specifically related to the incidence of thrombotic events and development of inhibitory antibodies to the porcine product); and delivery of intervention Other Evidence for the following will be noted in the Clinical Evidence Review: considerations • presence of cross reacting antibodies • the correlation of response to anti-porcine FVIII antibody titre • dose required to achieve resolution of a bleed • the use of susoctocog in related acquired haemophilia caused by antibodies to factor VIII

If evidence allows, the following subgroups will be considered: • patients for whom a bypassing agent is not a suitable treatment • those who are at high risk of thrombotic event

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NHS URN 1703 / NICE ID006 Page 2 of 2 NICE commissioning support draft scope for susoctocog alfa for acquired haemophilia caused by antibodies to factor VIII