What’s Happening with UK SMA Patient Databases?

14 May 2018

Over the past few years, dramatic progress has been made in establishing various SMA patient databases – initially with SMArtnet and the SMA Patient Registry and more recently with the addition of a valuable natural history database, SMA REACH. This has now received ‘The National Institute of Health Research (NIHR) portfolio adoption’. The NHIR Portfolio of studies are high-quality clinical research studies that are eligible for consideration for support from the Clinical Research Network in England.

This has all been made possible by generous funding and commitment from Muscular Dystrophy UK, SMA Support UK and the SMA Trust. More recently, considerable work has taken place to link these initiatives to form a more comprehensive resource to help facilitate clinical trial readiness in the UK and standardisation of care.

In addition, the SMA REACH network has expanded into an international collaboration (involving the Italian Telethon Network and the PNCR Network in the US) which, amongst other things, has enabled the development of sensitive outcome measures and scales that can be used to assess patient responses and to model disease progression within different age groups and SMA Types.

Now with the advent of nusinersen, the first SMA treatment, there is an additional urgent requirement to include the facility for continued monitoring of the effects of the drug in patients (post-marketing surveillance). 

Biogen, as the first pharmaceutical company to have an approved treatment, has announced an investment of £450,000 over 3 years to further facilitate / accelerate the ability to bring together the various databases and to expand the programme to include more UK centres. This development will also enable the collection of more in-depth clinical data. The focus would initially be on children but there are already plans to include adults and to establish an adult neuromuscular clinical network, along the lines of the existing North Star paediatric group which deals with Duchenne Muscular Dystrophy. Biogen is also working with other networks such as TREAT NMD in order to capture anonymous data from as many SMA patients as possible.

However, whilst Biogen is providing significant investment, it has been important to agree that they will not own the data, which will be independently owned by SMA REACH. Work is being done to assess what summary data might be made available, with patient consent, to Biogen and potentially, in the future, to other companies involved in individual drug development programmes for SMA.

This SMA REACH / Biogen collaborative development will need to follow a strict formal process (including ethics approval), part of which will include a steering committee, in which patient advisory groups will be involved.

This initiative represents important progress in the world of SMA drug development and one which wouldn’t have been possible without initial funding and commitment from SMA charities.