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Is NICE losing its standing as a trusted source of guidance?

BMJ 2023; 383 doi: (Published 08 November 2023) Cite this as: BMJ 2023;383:p2571

Rapid Response:

We have already lost our trust in NICE – Our real-life case

Dear Editor

Unfair, illegal, unreasonable, and unfair again. These are the findings of the two appeals against the decision of NICE`s Highly Specialised Technologies (HST) Committee not to recommend afamelanotide, the only treatment for the rare genetic light intolerance erythropoietic protoporphyria (EPP). (1,2) Patients with EPP suffer from painful phototoxic burn reactions on all parts of the body exposed to visible light. Symptoms occur within minutes of exposure and last up to 10 days, without effective pain relief. As a result, patients with EPP must avoid light exposure as best as possible, which has a negative impact on all aspects of their lives, including social isolation and mental health issues. In contrast, the amount of time patients on afamelanotide treatment spend in sunlight is in the same range as that of healthy indoor workers, and patients can lead a near-normal life for the first time. (3,4) Apparently, this is not good enough for NICE. In its initial assessment of afamelanotide, the HST Committee concluded that the results of the clinical trials "suggest small benefits with afamelanotide". (5)

Our patient organisation, the International Porphyria Patient Network (IPPN), is a not-for-profit organisation with no industry funding. The IPPN is a stakeholder in the appraisal process of afamelanotide at NICE. Our members live in countries where afamelanotide is reimbursed and we all have first-hand experience of the benefits of treatment with afamelanotide. Not only was our experience of the effects of treatment brushed aside by the HST Committee, but the Chair publicly stated during the appeal hearing that the Committee did not consider EPP to be a disability because there were no "patently visible" signs of disease. (1,6) While not even true - severe phototoxic reactions cause swelling, redness and second-degree burns to skin exposed to light - visible signs of disease are not a prerequisite for a condition to be considered disabling. Accordingly, the Appeal Panel emphasised in its decision that "EPP very clearly meets the definition of a disability under the Equality Act 2010." (1)

In addition, the Appeal Panel concluded that the Committee's finding that the trial results showed a small benefit of afamelanotide was unreasonable and raised concerns about whether the HST Committee had discriminated against people with EPP. (1) The appeal was upheld on all possible grounds, i.e., that NICE has failed to act fairly (ground 1a), exceeded its powers in making the assessment that preceded the recommendation (ground 1b), and that the recommendation was unreasonable in the light of the evidence submitted to NICE (ground 2). As far as we are aware, no other appeal has ever been upheld on all three possible grounds. (6)

After the appeal, the evaluation was referred to NICE with the expectation that the upheld appeal points would be addressed. The IPPN had expected that after such a clear outcome, NICE would be keen not to repeat the same mistakes and at least exercise more oversight over future interactions between the HST Committee and stakeholders. However, our request to at least replace the Chair after such an inappropriate comment revealing his attitude towards patients fell on deaf ears, and the unfairness continued: despite no other treatment option being available to patients with EPP, the HST Committee delayed the assessment of afamelanotide for several years, with a final decision not published until July 2023 – an unfair act as assessed during the second appeal hearing in May 2023. (2) Unsurprisingly, the decision to fund afamelanotide has remained negative. And, after this disappointing ordeal, our trust in NICE has completely dissolved.

Jasmin Barman-Aksözen, PhD. Vice-President International Porphyria Patient Network
Rocco Falchetto, PhD. President International Porphyria Patient Network

(1) NICE (2018): Advice on Afamelanotide for treating erythropoietic protoporphyria [ID927]. Decision of the panel.

(2) NICE (2023): Advice on Afamelanotide for treating erythropoietic protoporphyria (ID927): Decision of the panel.

(3) Langendonk JG, Balwani M, Anderson KE, et al. Afamelanotide for Erythropoietic Protoporphyria. N Engl J Med. 2015;373(1):48-59. doi:10.1056/NEJMoa1411481

(4) O'Reilly M, McGuire VA, Dawe RS. Erythropoietic Protoporphyria and Afamelanotide: A Patient's Perspective. Clin Exp Dermatol. Published online October 13, 2023. doi:10.1093/ced/llad346

(5) NICE (2018): Final evaluation determination – Afamelanotide for treating erythropoietic protoporphyria.

(6) Barman-Aksözen J, Granata F, Aksözen, MH, et al. ‘… they had interpreted “disability” as referring to a patently visible disability’: experience of a patient group with NICE, Disability & Society, 37:7, 1239-1245, DOI: 10.1080/09687599.2022.2060804

Competing interests: No competing interests

02 December 2023
Jasmin Barman-Aksözen
Rocco Falchetto
International Porphyria Patient Network
Zurich, Switzerland