Covid-19’s impact on NICE’s evaluation of rare disease medicines

9 April 2020

Covid-19's impact on NICE's evaluation of rare disease medicines

NICE (National Institute for Health and Care Excellence) has had to prioritise elements of its workload due to the Covid-19 crisis. This is to allow for capacity issues arising from social distancing measures, because many members of NICE committees are key workers occupied by the Covid-19 response, and because NICE has a programme of specific work to support the NHS and social care to respond quickly to the challenges of the coronavirus pandemic, you can read about this here.

This means that NICE will be prioritising and only publishing work that is either therapeutically critical or relates to addressing Covid-19 diagnostic or therapeutic interventions. This means a change to the timelines for many rare disease treatment single technology appraisals and highly specialised technology evaluations. 

For rare disease treatments that are not judged therapeutically critical, NICE will continue developing guidance as much as possible where it does not require committee or engagement with those involved in the Covid-19 response. You can see the full list of those treatments judged therapeutically critical here, under the label Guidance topics that will publish as planned.

Genetic Alliance UK has received notification of some of the measures proposed where NICE must delay an assessment. The following list may not be comprehensive. For those assessments that are or may be paused, NICE has not been able to provide updated timelines at this point.

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene – There is a managed access agreement in place for this treatment. This Highly Specialised Technology evaluation has not been defined as therapeutically critical. NICE will continue to continue through to delivery of the ERG report at which point it is anticipated the evaluation will be paused. NICE and NHS England anticipate that this pause may require an extension to the duration of the MAA to allow more time for alaluren’s re-evaluation. Patients will continue to be monitored and receive treatment, where it is safe and appropriate to do so, in line with the funding arrangements established by the managed access agreement. 

Belimumab for treating active autoantibody-positive systemic lupus erythematosus – There is a managed access agreement in place for this treatment. This Single Technology Appraisal has not been defined as therapeutically critical. To proceed would require NICE to re-scope, which is not possible currently, and so the appraisal will be paused.

Caplacizumab for treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura – This appraisal is being prioritised. The only delay to this process has been the cancellation of all NICE Technology Appraisal Committee meetings for April and the first Committee meeting for this appraisal will therefore now be held on 13 May 2020 via video teleconference.

Crizanlizumab for preventing sickle cell crises in sickle cell disease – This Single Technology Appraisal has not been defined as therapeutically critical. Evidence review submissions will continue and the Evidence Review Group (ERG) report will be delivered. There may be a pause in the process at this point.

Metreleptin for treating lipodystrophy – This Highly Specialised Technology evaluation has not been defined as therapeutically critical. The evaluation will therefore be paused.

Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 -This Highly Specialised Technology evaluation has not been defined as therapeutically critical. The evaluation will therefore be paused.

OTL-200 for treating metachromatic leukodystrophy – This Highly Specialised Technology evaluation has not been defined as therapeutically critical. Even though this topic has not been formally referred by the Department of Health and Social Care, NICE will continue to continue through to delivery of the ERG report at which point it is anticipated the appraisal will be paused. 

Risdiplam for treating spinal muscular atrophy in children and adults – This Single Technology Appraisal has not been defined as therapeutically critical. The scoping for this topic will therefore be paused.

Volanesorsen for treating familial chylomicronaemia syndrome – This Highly Specialised Technology evaluation has not been defined as therapeutically critical. The evaluation will therefore be paused.