Patrick F K Yong, Jolanta Bernatoniene, Rachel Annals, Charu Chopra, Tanya Coulter, Sarah Denman, Anthony Dorr, Tariq El-Shanawany, Alexandros Grammatikos, Padmalal Gurugama, Rashmi Jain, Sorena Kiani-Alikhan, Lucy Leeman, Lorena Lorenzo, Ania Manson, Sadia Noorani, Smita Patel, Arthur Price, Ravi Sargur, Cathal Steele, Catherine Stroud, Cassim Akhoon, Ariharan Anantharachagan, Emily Carne, Samuel Chee, Joanne Miller, Stephen Owens, Christine Symons, Ky-Lyn Tan, Michael D Tarzi, Scott Hackett, Angela Metcalfe, Sinisa Savic, Tomaz Garcez
Hereditary angioedema (HAE) is a rare genetic disorder characterised by recurrent episodes of swelling, which can potentially be life-threatening, resulting in substantial physical, psychological, educational and occupational burden. Treatment of HAE is divided into on-demand treatment for acute attacks and long-term prophylaxis for prevention of attacks, and there are now several effective therapies available. However, in the UK, use of on-demand therapy is intended for attacks defined as clinically significant, and access to long-term prophylaxis is determined primarily by frequency-based criteria. In this policy-focused review, we compare UK access criteria with international guidelines on best practice. We conclude that UK access criteria result in a group of HAE patients with persisting unmet need, and this may have a greater impact in children and young people. In view of this, we recommend greater flexibility in access criteria, allowing clinical judgement, shared decision-making, and broader measures of disease burden to inform treatment eligibility, so we can individualise care and fully meet the needs of these patients.