Words by Jade Williams
The National Institute for Health and Care Excellence (NICE) has recommended an oral therapy for adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH) with haemolytic anaemia – marking the 1000th medicine evaluated by NICE since its inception in 1999.
Novartis’ iptacopan offers a more convenient treatment alternative for over 200 patients in the UK, which, unlike traditional therapies requiring intravenous administration in a hospital setting, is taken at home twice daily in pill form.
PNH, a rare haematological disorder, leads to persistent anaemia due to the immune system’s destruction of red blood cells. Novartis’ treatment targets both intravascular and extravascular haemolysis, reducing red blood cell destruction in the blood vessels, liver and spleen.
Clinical trial data indicates that the treatment elevates haemoglobin levels, decreasing the need for blood transfusions and offering a more cost-effective solution for the NHS. The current standard for care traditionally includes eculizumab and ravulizumab – both administered in hospital as infusions.
Over the past 25 years, NICE has issued positive recommendations for 84% of the 1000 therapies reviewed, significantly impacting patient outcomes across the UK. This latest recommendation from the institute conveys the industry’s commitment to advancing patient care in rare diseases and optimising treatment strategies, paving the way for greater patient autonomy.
