This post may be distressing to some readers. It discusses the medical care of young children. If you want to keep reading, at least grab a strong cup of tea.
The National Institute for Health and Care Excellence makes recommendations on what should and shouldn’t be funded on the NHS. Today is the close of their consultation on Nusinersen, a treatment which evidence shows helps very young children and school age children with SMA (a muscle-weakening condition I happen to have) to gain and keep muscle strength.
NICE are currently not recommending this treatment on the basis of cost.
But how have they made that decision? Where do you draw the line? Where do the figures come from?
There are children out there, not so different to me, who may not survive into adulthood without this treatment. Though we can’t fund every treatment ever invented, this is a serious decision, that means serious people need to make a serious case on both sides with serious evidence to back up what they’re saying.
This isn’t a “one person made a call we disagree with” issue. This is a question of how the right to life of these kids and others is respected and protected, not to mention the right to life of anyone who may one day need a novel treatment. It’s a big question.
Here is my consultation response.
To whom it may concern at NICE
I was dismayed but unsurprised by your proposal not to recommend the use of Nusinersen in adults with Spinal Muscular Atrophy (SMA), given the need for more studies to be undertaken to demonstrate its efficacy.
I was shocked by your proposal not to recommend the use of Nusinersen in children with Spinal Muscular Atrophy (SMA), given the body of evidence that shows the treatment has clear clinical benefits.
Looking deeper into your decision making process, I remain shocked.
Please find below a summary of my thoughts and some questions I would be keen to see answered. I look forward to your reply and would be happy to meet with you to discuss this further.
Adult with SMA type II
Question: By what processes does NICE take account of its duties as a public authority to act in a manner consistent with human rights?
These children, like all children, have a right to life.
I draw your attention to the UN Universal Declaration of Human Rights, Article 3: “Everyone has the right to life, liberty and security of person.” (UN, 1948)
And also to the UN Convention on the Rights of Persons with Disabilities, Article 10: “Right to life
States Parties reaffirm that every human being has the inherent right to life and shall take all necessary measures to ensure its effective enjoyment by persons with disabilities on an equal basis with others.” (UN, 2006)
These aspirations are realised through the Human Rights Act 1998, Article 2: “Right to life
Everyone’s right to life shall be protected by law. No one shall be deprived of his life intentionally save in the execution of a sentence of a court following his conviction of a crime for which this penalty is provided by law.” (Legislation.gov, 2018)
Publicly available information from the Equality and Human Rights Commission states “Public authorities should also consider your right to life when making decisions that might put you in danger or that affect your life expectancy.” (EHRC, 2018)
NICE recommendations are used to influence real-world decisions and patient pathways; NICE has a duty under the HRA to act in a way that protects life.
Nusinersen has been shown to protect the life of children with SMA. It is the first and only treatment of its kind designed to do so. Decisions which impact patient access to this drug affect these patients right to life. Despite this, the right to life does not appear to be not explicitly mentioned in NICE’s consultation.
If NICE were to not recommend a life-extending treatment without sufficient justification, NICE could be found to be not acting in accordance with the HRA.
The NICE website does show that NICE is aware of its duties under the HRA, though offers little detail as to how it ensures it discharges them.
From NICE’S EQUALITY OBJECTIVES AND EQUALITY PROGRAMME 2016-2020 (NICE, 2016)
“The Human Rights Act 1998
- When public authorities such as NICE carry out ‘functions of a public nature’, they have a duty under the Human Rights Act 1998 not to act incompatibly with rights under the European Convention for the Protection of Fundamental Rights and Freedoms. The Equality Act’s
public sector equality duty uses the same definition of functions of a public nature as the Human Rights Act 1998. The Human Rights Act places responsibility for ownership of human rights matters on every public body and employee and requires active consideration of whether decisions have any implications for human rights.”
“NICE’s compliance with the Human Rights Act
- NICE achieves compliance with human rights requirements primarily through:
- a robust procedural framework for developing guidance
- an equality analysis process that also looks at the situation of groups in addition to those who share the characteristics protected under the Equality Act
- asking advisory bodies to satisfy themselves that their decision making procedure is fair and transparent, that decisions do not discriminate against a group that is not a legally protected group,
and, if they do, whether that discrimination is legitimate
- obtaining legal advice when an issue arises that could potentially lead to challenge.”
Despite these assertions, the procedural frameworks (NICE, 2017, 2017, 2018) underpinning the Nusinersen decision do not appear to discuss how to make decisions which respect every individual’s human rights, including right to life.
Within the Nusinersen consultation document (NICE, 2018), there appears to be no explicit mention of human rights or right to life.
In all human rights discussions, the needs of the individual must be weighed against the needs of collective.
NICE’s approach to this is to calculate the cost relative to the benefit; incremental cost-effectiveness ratios (ICERs) per quality adjusted life year (QALY).
A very high cost treatment which did not have a significant effect on health related quality of life (HRQL) would have a large ICER per QALY. The inverse is also true.
Funding very high cost treatments which have very small benefits is unsustainable, and so the wider economic picture must be balanced against the needs of an individual or group of people. Both must be carefully assessed and considered in a robust decision making process.
In this case, we are establishing whether it would be financially sustainable for the NHS to give children with SMA access to the only life-protecting treatment of its kind.
NICE’s SOCIAL VALUE JUDGEMENTS, Principles for the development of NICE guidance, Second edition (NICE, 2008) states:
“NICE has never identified an ICER above which interventions should not be recommended and below which they should. However, in general, interventions with an ICER of less than £20,000 per QALY gained are considered to be cost effective.”
Question: What is the evidence based rationale behind this figure of £20,000 per QALY being used as a general guideline for cost-effectiveness?
Question: Does this figure change with inflation? Does it change according to the financial realities of the NHS at the time of decision making?
Given that the cost-effectiveness of Nusinersen is assessed through the ICER per QALY measure, it is important to ensure that the QALY measure is reflective of the individual experience of these patients and those around them.
However, there does not seem to be a consistent definition of a QALY used across all NICE decision making.
Measurement of quality of life of very young children has been pointed out in the Committee papers (NICE, 2018) as extremely difficult. Similarly, it was noted that the PedsQL may not be an appropriate tool to measure outcomes which matter to school-age children with SMA.
The approach to collecting data about health related quality of life of these children focuses on adverse events (such as respiratory infections), hospitalisations, motor milestones (rolling, sitting, lifting objects…), and interviews with a small number of clinical practitioners.
There is no publicly available summary of the interviews with the clinical practitioners. Though it is important to maintain doctor-patient confidentiality and to allow the clinical practitioners to feel they can speak freely without fear of public/media misinterpretation, the lack of transparency feels disconcerting.
Request: NICE seek the permission of the clinicians to publish a bullet pointed summary of the key points.
There appears to be no explanation of how the clinicians views and measured outcomes were converted to a numerical value; the QALY.
Given the importance of the QALY to the ICER per QALY measure, and thus to whether or not children will be treated with Nusinersen, it is important that this calculation is transparent.
Question: By what process was the information gathered converted into a QALY?
NICE has compared Nusinersen against best case usual care (henceforth best possible care) to give a value for the ICER.
This neglects to reflect the reality of the situation for children with SMA and their families. Many of the parent submissions talked of the difficulties associated with lifting a growing child, lack of sleep, getting the right equipment and making adaptations to the house. Best possible care would include steps to address these issues being taken by non-NHS bodies acting appropriately in a timely manner, providing and advising on equipment, housing, social care, respite and so on. The submissions you received reflect that this does not always happen smoothly. Best possible care is not the same as currently available care.
Similarly, best possible care is not always delivered by the medical team around the child. Within the company submission (Figure 34 and surrounding discussion), it is acknowledged that the UK standard of care in practice is not the same as that in other countries, highlighted by data from Italy. The UK does not presently deliver the best standard of care, which has a significant impact on patient survival. This must be addressed outside of the present discussion.
Given that best possible care is not uniformly experienced by children with SMA in the UK at present, it does not seem logical to use it as a direct comparator when looking at Nusinersen.
The “Green Book” from HM Treasury (HM Treasury, 2018) sets out a suggested process by which options of how to spend public money can be appraised. It recommends looking at an option compared to business as usual (BAU). In this situation, that would be a model of care which takes into account gaps in provision and waiting times for equipment, advice, and treatment, and the effects this has on children with SMA and their families.
Request: NICE produce a BAU model of care to use as comparator to Nusinersen.
The decision on whether or not to recommend Nusinersen and any medical technology must happen through a process which is fair, transparent, and uniformly applied.
I request that NICE not only review the decision to not recommend Nusinersen, but also review the process by which such decisions are made so to make it clearer how NICE carries out its duties.
A final word on quality of life
The submissions to NICE reflected the downsides of having SMA. However, it also seems important to mention that when health is stable, and the correct support is available, a good quality of life can be achieved.
Here are some examples from adults with SMA:
Fleur Perry (me): – 3 highlights from the past year: Seeing the Killers play live at the O2, meeting some of my heroes at London Comic-con in full cosplay with 2 of my best friends, meeting Baroness Jane Campbell at the House of Lords
– the highlight of the past month: Helping put together a last-minute surprise celebration for a family event complete with gold-edged invitations, and the looks on my rellys’ faces as we laughed and ate and drank altogether for the first time in 4 years.
– something you’re looking forward to?: Finishing my Physics degree and possibly starting a Masters
[Others redacted as permission granted to share with NICE only]
UN (1948) The Universal Declaration of Human Rights. Available at http://www.un.org/en/universal-declaration-human-rights/ (Accessed 1 September 2018).
UN (2006) Convention on the Rights of Persons with Disabilities (CRPD), Article 10 – Right to life. Available at https://www.un.org/development/desa/disabilities/convention-on-the-rights-of-persons-with-disabilities/article-10-right-to-life.html (Accessed 1 September 2018).
Legislation.gov (2018) Human Rights Act 1998. Available at https://www.legislation.gov.uk/ukpga/1998/42/schedule/1 (Accessed 1 September 2018).
Equality and Human Rights Commission (2018) Article 2: Right to life. Available at https://www.equalityhumanrights.com/en/human-rights-act/article-2-right-life (Accessed 1 September 2018).
NICE (2016) NICE’S EQUALITY OBJECTIVES AND EQUALITY PROGRAMME 2016-2020. Available at https://www.nice.org.uk/Media/Default/About/Who-we-are/Policies-and-procedures/NICE-equality-scheme/equality-objectives-and-equality-programme-16.pdf (Accessed 1 September 2018).
NICE (2017) Medical technologies evaluation programme process guide [PMG34]. Available at https://www.nice.org.uk/process/pmg34/chapter/introduction (Accessed 1 September 2018).
NICE (2017) Medical technologies evaluation programme methods guide [PMG33]. Available at https://www.nice.org.uk/process/pmg33/chapter/introduction (Accessed 1 September 2018).
NICE (2018) Guide to the processes of technology appraisal [PMG19]. Available at https://www.nice.org.uk/process/pmg19/chapter/acknowledgements (Accessed 1 September 2018).
NICE (2018) Appraisal consultation document, Nusinersen for treating spinal muscular atrophy. Available at https://www.nice.org.uk/guidance/GID-TA10281/documents/appraisal-consultation-document (Accessed 1 September 2018).
NICE (2008) SOCIAL VALUE JUDGEMENTS, Principles for the development of NICE guidance, Second edition. Available at https://www.nice.org.uk/media/default/about/what-we-do/research-and-development/social-value-judgements-principles-for-the-development-of-nice-guidance.pdf (Accessed 1 September 2018).
NICE (2018) Single Technology Appraisal, Nusinersen for treating spinal muscular atrophy [ID1069], Committee papers. Available at https://www.nice.org.uk/guidance/gid-ta10281/documents/committee-papers (Accessed 1 September 2018).
HM Treasury (2018) THE GREEN BOOK, CENTRAL GOVERNMENT GUIDANCE ON APPRAISAL AND EVALUATION. Available at https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/685903/The_Green_Book.pdf (Accessed 1 September 2018).