An overview of what’s involved when we advocate for access to a treatment:

In England:

• Scoping

The National Institute of Health and Care Excellence (NICE). NICE sets out information about the treatment that it’s considering assessing for access and poses initial questions that we answer when we submit our ‘scoping response’.

• Consultation Submission

This is the next stage. Submissions respond to questions posed by NICE, including ones about the impact of SMA and Community views of the treatment. We undertake community surveys – sort out what to ask, collect. collate and analyse the information. We make the results available to our partner SMA charities. Often we make joint consultation responses to inform what we say.

• Patient Experts

NICE asks us to nominate people to take part in the appraisal who can. Tell NICE about the impact of the condition on their / people’s lives and what difference the treatment could make. NICE make the final decision as to who they select – usually one or two people.

• Technical Engagement meetings

NICE ‘technical’ staff meet clinical and patient experts. NICE staff have at this stage reviewed all the evidence, including the pharma company’s economic cost effectiveness modelling and clinical trial evidence and ask detailed questions about the impact of the condition and any ‘uncertainties’ about the effectiveness of the treatment. These meetings are not public.

• Committee meetings

These meetings are public and are when NICE listens to all the evidence and makes a decision. Invariably this is at first a ‘no’ to recomendign the treatment for funding and potentially further consultations and committee meetings as has happened for all three SMA treatments to date.

In Scotland:

The Scottish system is similar to start with in terms of submitting a consultation response which includes any surveys results we have gathered. It is, though, simpler with automatic rights for Patient Group representatives who have made submissions to join the patient and clinical engagement meeting and the committee meeting.