The possibility to appeal against a negative funding recommendation for technologies evaluated by the National Institute for Health and Care Excellence (NICE) in England was established to ensure fairness in their decision-making and priority-setting. However, more than three years after an appeal lodged during the appraisal proceedings of afamelanotide for treating erythropoietic protoporphyria (EPP), the points raised by concerned stakeholders, including the IPPN, and upheld by the appeal panel have yet to be addressed and, regrettably, patients with EPP in England remain without access to the only treatment for their condition.
To raise awareness in the scientific community about this issue, the IPPN has published their experience with NICE in a peer-reviewed, open access article in the medical journal Disability & Society:
‘… they had interpreted “disability” as referring to a patently visible disability’: experience of a patient group with NICE:
With this publication we hope to promote a more fair, consistent, and respectful evaluation process for patients with EPP and other conditions at NICE.