Consultation response specialised comm 27 04 15

April 27th 2015 NHS England Full consultation response-‘Investing in Specialised Commissioning’ 1. Do you have any comments on the proposed process described above in paragraphs 20-24 for making investment decisions about specialised services?

There is not enough detail in the consultation document about this process to comment. The descriptions of the parts of the process involved are very broad and do not really pin down who or how decisions about which treatments and services should be funded are reached. We understand that some SOPS have now been published for developing services specification, however, we are not able to access these, although some other stakeholders have been given access to these. This makes this consultation incomplete and non-transparent.

Additionally, the proposed process still has inherent roadblocks for patients with rare diseases such as Rett Syndrome as the cost per QALY system/proposed scorecard methodology will obviously favour the majority with prioritisation of common conditions and acute situations where cost per QALY is lower. As a patient group representing approximately 2400 patients with Rett Syndrome and related MECP2 disorders in the UK, we do not have confidence that this proposed process is either sound, fair or transparent. Because of the lack of transparency, it is not possible for patients, parents or carers, process.

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

It is difficult to answer this without any clarification of what happens at each stage of the process, which is being proposed.

3. Are there any additional stages in the process where engagement with patients and the public should take place?

Patient representatives should be an implicit part of the decision-making process with patient representatives involved at every stage and on every level. Engagement means tell me what you think is best.

Involvement means we will work out what is best together - at the proposed ‘decision making events,’ with all parties’ interests at the heart of the decision-making process and yes, this includes the patient.

4. 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 we have described.

As a patient group representing a progressive condition for which there is currently no treatment, but for which there are a great deal of treatments on the horizon, we call on NHS England to enable interim access to new and emerging medications for patients with immediate clinical need. Although we are pleased to see that the Cancer Drugs Fund remains outside these arrangements, we ask why NHS England does not ring-fence funds for new and emerging treatments, which will slow the progression of other serious and life-altering conditions. It is possible that placing drugs for a specific condition outside other arrangements negatively impacting patients with (non-cancerous) but similarly serious, life altering and threatening diseases, could be seen as discriminatory.

We also call upon NHS England to address inadequacies of assessment.

When looking at deliverable and measurable benefits, we ask NHS England to also incorporate impact: i.e. Increase in stationary weightbearing for 5 seconds might not seem a drastic change in quality of life to NHS England but for a young woman with Rett Syndrome, this could be the difference between her being able to continue to live in her own home with her family and having to live in a care facility where she can be transferred from bed to wheelchair by hoist with two carers. The impact of a new therapy on a patient should be given equal weighting to clinical measures.

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

In this document, NHS England specifies that you will prioritise treatments and interventions that demonstrate the greatest value for money. There is an inherent prejudice against rare disease groups here because orphan drugs are likely to be much more expensive than drugs for common diseases. This consultation has also requested the views of stakeholders on the principles underpinning the decision making process without providing adequate detail about what the decision making process is. There is important information missing here, which has been, published which could provide more information to stakeholders, but which has not been made publicly available. The consultation therefore seems incomplete and non transparent. 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 should be delivered. If you have any views on which services should be prioritised for a service review in 2015/2016, please tell us.

6. Declaration: Reverse Rett is a registered charity. We do not now and have never received any direct or indirect funding from and companies that manufacture drugs or treatments used in the treatment of specialised services.