Is NICE losing its standing as a trusted source of guidance?
BMJ 2023; 383 doi: https://doi.org/10.1136/bmj.p2571 (Published 08 November 2023) Cite this as: BMJ 2023;383:p2571
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Dear Editor
Professor Michaels summarises the issues facing organisations developing guidelines well. The NICE guideline development committee on AAA (referenced in his article and of which I was a member) was heavily influenced by NICE's 'Social Value Judgements' policy document when developing its recommendations following literature review and HE analysis produced by NICE's technical team. A different set of social value judgments would have likely resulted in different recommendations.
It is essential that decisions made by NICE reflect the societal values and that there are mechanisms to ensure that social value judgement policies remain relevant and contemporary. A balance between a strict utilitarian approach (embodied in HE analysis) and wider libertarian considerations of fairness, equity, dignity and autonomy is essential. Population level cost effectiveness decisions will always conflict with patients' personal priorities at times of healthcare need unless there is a wider public discourse about what it is reasonable for the NHS to provide and how it might provide this.
Competing interests: Member of NICE AAA Guideline Development Committee 2015-20
Dear Editor
Of course, Santhanam Sundar is correct in stating that in a taxpayer funded system “NICE cannot allow healthcare professionals to prescribe the best available treatment that would suit the patients' preferences all the time.”
Healthcare professionals are well versed at providing the best possible advice within the limited resources available to them, and it is appropriate for those limitations to be based upon evidence-based assessments of value-for-money. However, this comes with a couple of caveats.
The first is that implementing restrictions through clinical guidelines, as part of a shared decision-making process, is likely to be both ineffective and inequitable. [1]
The second is that the values embodied in such value-for-money calculations should align with those of the taxpayer, rather than commercial values or political priorities. Unfortunately, there is a growing suspicion that the pharmaceutical industry may have disproportionate influence on NICE decision making [2], and, judging from the many fines imposed [3] the industry may not restrict itself to ‘all legal means’ in maximising profits.
1. Michaels JA, Maheswaran R. Conflicting perspectives during guidelines development are an important source of implementation failure. Health Policy2023;131:104801. doi:10.1016/j.healthpol.2023.104801
2. Calnan M. Rationing, regulating and big pharma. Health policy, power and politics: Sociological insights: Emerald Publishing Limited 2020:55-72.
3. Greenman C, Zupancic K, Davis K, Healy C. Penalties for fraud in the pharmaceutical industry: A financial and reputational analysis. Journal of Accounting, Ethics & Public Policy 2023;24(1) doi: 10.60154/jaepp.2023.v24n1p21
Competing interests: No competing interests
Dear Editor
Every individual patient naturally want the best available treatment that makes them better. In self funded systems and insurance based health systems, people make value based judgements and accept the risks that come with their choices.
In tax payer funded systems, individuals do not make a financial choice beforehand and when they have an illness , they do expect the the best available treatment irrespective of the cost.
Pharmaceutical companies have a primary duty to maximise shareholder value by all available legal means. Hence they are duty bound to maximise revenue for their existing and new products.[1].
On the other hand, Tax payers, in general prefer to pay the lowest possible tax. Tax paid voluntarily in the UK is very very miniscule. [2] [3].
Hence , in a resource constrained, tax payer funded healthcare system such as the NHS, the overall societal values and preferences is in direct conflict with market driven, new drug development .
Hence NICE cannot allow healthcare professionals to prescribe the best available treatment that would suit the patients' preferences all the time. [4].
References
1. Perkins L. Pharmaceutical companies must make decisions based on profit. Western Journal of Medicine 2001;175:422–3. doi: https://doi.org/10.1136/ewjm.175.6.422
2. Voluntary payments / donations to government. GOV.UK. https://www.gov.uk/guidance/voluntary-payments-donations-to-government (accessed 8 Nov 2023).
3. Is the system for voluntary tax payments due an overhaul? | RSM UK. www.rsmuk.com. https://www.rsmuk.com/insights/weekly-tax-brief/is-the-system-for-volunt... (accessed 8 Nov 2023).
4. Michaels JA. Is NICE losing its standing as a trusted source of guidance? BMJ 2023;383:p2571. doi: https://doi.org/10.1136/bmj.p2571
Competing interests: 1. Advisory board work and multiple conference sponsorships from various Pharmaceutical companies 2. Member of various NICE committees (NICE prostate cancer update, NICE Trans perineal biopsy , NICE MRI fusion biopsy , NICE Audit panel and Ext ref group)
Dear Editor
Michaels is right to refer to the financial opportunity cost of funding expensive treatments. However there is another opportunity cost that NICE ignores- the opportunity cost of clinician time.
No matter how much money the NHS might have, it has a finite amount of clinician time. Clinicians spending time doing one thing are unavailable to do other things. Interventions such as treatment for primary prevention of CVD at "low" (10%) 10-year risk may make sense if one treats clinician time as a "free" resource, but perhaps not if one acknowledges that the clinicians involved could more productively be doing something else with their finite time.
NICE needs to find a way to factor this opportunity cost into its calculations.
Competing interests: No competing interests
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
References:
(1) NICE (2018): Advice on Afamelanotide for treating erythropoietic protoporphyria [ID927]. Decision of the panel. https://www.nice.org.uk/guidance/hst27/documents/appeal-decision
(2) NICE (2023): Advice on Afamelanotide for treating erythropoietic protoporphyria (ID927): Decision of the panel. https://www.nice.org.uk/guidance/hst27/documents/appeal-decision-2
(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. https://www.nice.org.uk/guidance/hst27/documents/final-evaluation-determ...
(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