1.1
Efgartigimod is not recommended, within its marketing authorisation, as an add-on to standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies.
1 Recommendation
1.2
This recommendation is not intended to affect treatment with efgartigimod that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.
Why the committee made these recommendations
Standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies includes surgery, acetylcholinesterase inhibitors, immunosuppressants, intravenous immunoglobulin or plasma exchange. Efgartigimod would be used as an add-on to standard treatment.
Clinical trial evidence suggests that efgartigimod plus standard treatment improves symptoms and people's ability to carry out their normal activities compared with standard treatment alone. But it is uncertain if the people in the trial reflect the people who would have efgartigimod in the NHS because the company has proposed a target population with more severe disease.
The economic model does not accurately capture how efgartigimod would be used in the NHS; that is, as an additional treatment in the treatment pathway. The most likely cost-effectiveness estimates are substantially above what NICE considers an acceptable use of NHS resources. This is because while the company's model suggests there is a modest gain in quality-adjusted life years, this is at a substantial additional cost, since efgartigimod is an additional treatment in the treatment pathway. So, efgartigimod is not recommended.