Alexion Pharmaceuticals (Nasdaq:ALXN) is pleased that the National Institute for Health and Clinical Excellence (NICE) Evaluation Committee has acknowledged that eculizumab (Soliris
) is a very effective treatment option for patients with aHUS, and that the use of eculizumab would be of significant value to patients with this life-threatening disorder who have no other treatment options.
In an Evaluation Consultation Document (ECD) released today, the NICE Evaluation Committee confirmed that currently available supportive care for patients with aHUS has limited impact on disease morbidity and mortality, but a substantial negative effect on a patient’s quality of life. Importantly, the Committee underscored that substantial restoration of health for a very long period is achieved with ongoing treatment with eculizumab. The Committee also concluded that eculizumab produced a substantial gain in quality-adjusted life years (QALY, a measure of the number of high-quality years of life gained) of a magnitude rarely seen for a new drug treatment – let alone one for an ultra-rare disorder.
Today’s very positive assessment by NICE of the clinical value of eculizumab for patients with aHUS aligns with the two prior positive recommendations from Government – AGNSS and NHS England. In addition, NICE confirmed today that the broad interim NHS England commissioning policy for eculizumab treatment of existing and new patients with aHUS will continue.
However, Alexion is concerned that nearly 3 years after Government commenced its evaluation, and more than 1 year after eculizumab was referred to NICE, the Committee was still unable to provide a formal recommendation whether eculizumab should be nationally commissioned. Additionally, NICE has now stated that it will seek further information from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken into account. NICE has also asked the company to provide additional information related to budget impact. NICE has not requested any additional clinical effectiveness information from Alexion.