NHS England Consultation

NHS England Consultation

NHS England are consulting on its process following our legal challenge on their discriminatory process back in November 2014.

The consultation closes on 27 April 2015 and you can find the document here: https://www.engage.england.nhs.uk/consultation/investing-in-specialised-commissioning/consultation/intro/view We are keen for as many people to respond so please follow this link and use our answers (see below) as a guide.

Please feel free to use our responses, listed below, which highlights our concerns about the process and lack of transparency.

The outcome of the consultation is having an immediate effect on those with MPS IVA Morquio who have been in clinical limbo since Vimizim was licensed on 28th April 2014. However, for any future treatments that are being trialed the outcome of this consultation will affect the likelihood of the treatment being funded following licensing so please do respond.

If you have any questions. Please don’t hesitate to contact Charlotte at c.roberts@mpssociety.org.uk or 0345 389 9901.


1. Do you have any comments on the principles that we have proposed to underpin the process for making investment decisions about specialised services?

This long overdue consultation process which follows our legal challenge of November 2014 on the previous process still lacks transparency and fails to provide any information about the detailed processes and criteria behind these principles. It is therefore impossible as a patient, parent or carer to know or understand or trust NHS England that they will arrive at a decision through a fair and transparent process that reflects the needs of all health service users including those with ultra-rare diseases.
Whilst in principle there is nothing wrong with the principles per se the proof is in the detail and the consultation provides no detail. Therefore it is impossible as a representative of 750 living patients across 25 ultra-rare diseases to have confidence in the principles as prioritisation will always favour the common conditions and acute situations where cost per QALY is much lower and favours the majority.
We understand that there may be standard operating procedures for developing service specifications and clinical commissioning policies but we have not been able to access these therefore making this consultation incomplete and far from transparent.

2. Are there any other principles that you think NHS England should adopt as part of its process for making investment decisions about specialised services?

NHS England has no record of normal good practice in making prioritisation decisions in a transparent way and documenting the outcomes at all stages of the process. This is proved by the debacle with the scorecard. Before we can accept the principles we would want to see the transparent policy which sets out good practice and is fair to people with ultra rare diseases. We do not believe that the consultation was fairly represented by stakeholder involvement of people with ultra-rare diseases and have been very disappointed by the lack of appropriate representation by Rare Diseases UK and the Genetic Alliance. We call upon NHS England to be transparent and publish details of the people participating in the consultation.

Our experience of NHS England’s assessment of adequate clinical reliable evidence to demonstrate clinical effectiveness worries us enormously. It does not take into account the complexities of assessing ultra-rare diseases like the ones our members suffer from and we believe the current principles will discriminate against people with ultra-rare diseases when it comes to prioritisation decisions.

It is a travesty of justice that ultra-rare diseases will be judged only on peer reviewed public papers. By their nature there will always be a shortage of published evidence for ultra-rare diseases so the process already sets out to discriminate against these patients.

NHS England must enable interim access for patients with immediate clinical need without prejudice to future decision making on those therapies. Our members with MPSIVA have been in clinical limbo for an unacceptably long time and an interim policy is as still as far off as it was when Vimizim was licensed by the European Medicines Agency on the 28th April 2014. We ask that urgent steps are taken to allow the treatment of MPS IVA children and adults affected by the delays of NHS England’s previous illegal process and their failure to consult.

The question of deliverable and measurable benefits to patients requires a very different approach than that of rare, common and chronic conditions. Ultra-orphan diseases are defined by NHS England as affecting no more than 500 living patients in England. In truth all our conditions except one have less than 100 surviving patients.

In turning to the evidence we give you some examples of some of our members’ experience.

• The increase in a 6 minute walk test might not seem much to NHS England but to someone with ultra-rare diseases it is the difference to them being able to get out of the house, live independently and not be a recluse.

• The difference demonstrated in a 3 minute stair climb is life changing as it means they can now climb the stairs to the toilet instead of relying on a commode or having to have an expensive downstairs extension or move home.

It is vital that the impact on patients of a new therapy is given equal weighting to the clinical measurements. Remaining stable in these diseases is a huge deliverable for parent carers as it means they can continue to work / pay taxes and have a life. Without treatment patients would get steadily worse and be confined to a wheelchair and die early.

In the case of our members’ children’s diseases the treatment is far superior to any other form of care available in the NHS because the only other option is palliative care and care of the dying. Are these decisions really down to a child or adult with ultra-rare diseases being cheaper dead than alive?

It is shocking to think that based on price per person rather than total cost impact, NHS England will deny children and adults treatment for ultra orphan diseases where the clinical trial has shown significant benefit under the European Medicines Agency appraisal.

The perceived view of our members in regards to prioritisation by NHS England is – too expensive to treat cheaper dead.

3. Do you have any comments on the proposed process for making investment decisions about specialised services?

We are relieved to see that NHS England may accord priority for ultra rare conditions even where there is limited published evidence on clinical effectiveness and recognising that the rarity of the condition may make the data unavailable. However, everything is in the detail and the word MAY should be replaced with WILL. We note, that you will only prioritise treatments where these can be offered to all patients within the same patient group and you will accord priority to treatments that are likely to reduce health inequalities. Therefore why won’t you have a commitment to people with ultra rare diseases and get rid of the MAY and insert WILL?
Surely it is unfair to judge ultra rare disease against other conditions where there is impact of any prioritisation on the wider health and care system including societal impact.

4. Are there any additional stages in the process that we should consider introducing?

We accept that NHS England should take into account all relevant guidance but we remain unconvinced by the vagueness and lack of detail of this statement. What is all relevant guidance?

5. Are there any additional stages in the process, in addition to those described, where engagement with patients and the public should take place?

Your first statement prioritised the innovations which provide the greatest value for money. We ask the question who for? Are you saying that people with ultra rare diseases where the production of treatments is expensive and therefore the drug is expensive are disposable? This suggests ethnic cleansing by rare disease.

When our members’ children were born they hoped for and expected a healthy child – most mother’s did not drink, smoke, managed their weight but despite all these factors the parents could not safeguard themselves against the randomness of giving birth to a child with an ultra-rare disease. How can it be right that people with morbid obesity, drug and alcohol addictions, self-inflicted hepatitis C who put their health at risk through their life style benefit from massive NHS England resources when if they took responsibility for themselves they could release funds to people with ultra-rare diseases who can’t help their condition.

6. Please provide any comments that you may have about the potential impact on equality and health inequalities which might arise as a result of the principles and process that we have described.

This consultation is not fit for purpose within the ultra-rare disease community because the medications for these conditions will never fit into the desired cost-cutting budgets of NHS England.

No patient is more diligent and conscious of money than those receiving high cost therapy for ultra-rare diseases. There is virtually no non-compliance or wastage in taking their medicines and they travel hundreds of miles at their own expense to attend rigorous medical reviews to ensure that the medicine is still working for them and necessary. Compare this with the huge wastage of prescription drugs left in medicine cupboards or delivered back to pharmacies to be destroyed up and down the country.

The MPS Society has a 30 year registry with more than 95% of every single patient with an MPS disease entered. We have been able to provide anonymised data on evidence of drug take up and dosage to ensure that the budget is worked out on actuality rather than theoretical eligibility.

NHS England needs to focus on putting back into place a fair transparent and equitable highly specialised commissioning service which is cost conscious, not driven by budget but by principles of compliance and stop start criteria.

At present it is difficult to see how fair the highly specialised technology programme will be to ultra-rare diseases but early signs through our experience of the drug Vimizim leaves us very worried. Whilst we welcome the focus given to the cancer drugs fund we have to ask ourselves why people with non-cancerous ultra-rare diseases are treated like lepers.

7. Are there any other considerations that you think we should take into account when developing the principles and process about investing in specialised services?

Since we started to follow NHS England and delve further into the structure we have to conclude that we cannot understand why there needs to be so many committees and senior managers absorbing millions of pounds and that this money could be put back better into health care.

Before NHS England the AGNSS framework worked well and had particular regard for the urgency and severity of the ultra-rare disease patient needs which appear virtually absent from the proposed principles and process.

We have heard that the standard operating procedures for service specifications includes more than 40 steps and seven decision making points and the standard operating procedure for commissioning policies includes again over 40 steps and nine decision making points. This proves our point that NHS England is not about making streamlined and timely decisions within a process and therefore the process is unfit for purpose. What are these steps and when will these be consulted upon? The only conclusion that we can reach is that NHS England is hiding behind bureaucracy and this costs lives.

8. As well as hearing your views on which treatments and services NHS England should prioritise for investment, we are also keen to hear your views on NHS England’s rolling programme of service reviews on how specialised services are delivered. If you have any views on which services should be prioritised for a service review in 2015/16, please tell us:

Review the cost of treating lifestyle diseases and conditions. If health tourism was addressed NHS England could fund new therapies for all ultra-orphan diseases. How can it be right that when UK citizen’s travel abroad they need to have insurance and have to produce this and guarantee treatment will be paid before moving into the clinic room?

The money spent on health tourism denies British citizens the treatments that they or their family members have paid in to for many decades.

Talking to pharmaceutical companies who are developing treatments for ultra-rare disease the change in climate with NHS England is clearly focusing minds and it is quite likely that clinical trials for ultra-orphan diseases will going forward not take place in England or elsewhere in the UK. The additional money made by clinical trial centres in England help to prop up the National Health Service.

9. Before completing the survey you must declare any financial or other interests in any specialised services. For example, if you are responding on behalf of a voluntary organisation and your organisation received any funding within the last two years (including sponsorship or grants) from companies that manufacture drugs or treatments used in the treatment of specialised services, you must declare this. If you are a commercial supplier to the NHS of specialised services this should also be specified.

The Society for Mucopolysaccharide Diseases has received unrestricted educational grants from six pharmaceutical companies. The highest grant received did not exceed 3% of the Society’s total income.