Spinal muscular atrophy sufferers are a step closer to accessing vastly life-improving treatment following the formal National Institute for Health and Care Excellence (NICE) invitation to Biogen to submit SPINRAZA (nusinersen) for assessment via the Single Technology Appraisal route. Nusinersen is the first and currently only disease-modifying treatment for 5q spinal muscular atrophy.
Spinal muscular atrophy is a rare autosomal recessive neuromuscular disease, and is the leading genetic cause of infant death. As spinal muscular atrophy is progressive, patients decline over time, with increasingly debilitating symptoms, affecting sufferers’ ability to move, swallow, and ultimately breathe. Many type one patients — who experience symptoms within six months of birth — do not live to see their second birthday.
Terry O’Regan, vice president and managing director of Biogen UK and Ireland, comments, “Up until nusinersen came onto the market, there was no disease-modifying treatment for this horrific disease. We are incredibly excited about the fact that we are now able to bring the first and only therapy for these patients.”
“We now have an opportunity for spinal muscular atrophy patients in England and Wales who deserve access to nusinersen, a medicine which has the potential to address unmet medical need in a devastating disease.”
The choice of route to bring this product to market has been a potential point of concern for Biogen, however. There are two main available routes for NICE to assess a product: the highly specialised technology appraisal route, and the standard technology appraisal route. This latter route encompasses most treatment options.
The highly specialised technology appraisal route, in contrast, is designed for ultra orphan therapies. To qualify, there needs to be fewer than 100 patients living with the disease at any one time. Between 650 and 1,300 children and adults currently live with spinal muscular atrophy in the UK, so this specialised route was deemed inappropriate following the initial scoping meeting between Biogen and NICE last year.
Speaking to Spinal News International, O’Regan says, “For us, we have been clear throughout the scoping process that both of these routes, to some degree, were the extreme cases, and therapies such as nusinersen are destined to fall between the cracks.”
The Biogen MD cites his initial reservations about the single technology appraisal route as the lack of comparators on the market (the STA route typically requires demonstrable clinical improvements and increased cost-effectiveness over existing treatments), and the heavy weighting towards quantitative quality of life measures, saying “To ask a two month old if their quality of life has improved is always going to be quite difficult.”
However, O’Regan remains optimistic about future patient access to nusinersen, noting that “there was a recognition” from both NICE and NHS England during a recent meeting. O’Regan elaborates, “We can understand why NICE has chosen the single technology appraisal route, because it enables us to assess a broader group of patients, which is good news for patients.”
“Whilst we have been disappointed by the delays in the NICE process so far, we have been encouraged by a recent meeting with NICE and NHS England, where the need to respond to specific STA and access challenges in appraising nusinersen was expressly acknowledged, and an immediate plan for a Managed Access Agreement that can work alongside the STA process was agreed. Now is the time for all stakeholders to work together to ensure that eligible patients who may benefit get rapid access to this life saving medicine.”
Specifically, this openness demonstrated by NICE and NHS England to commence development of a Managed Access Agreement straight away reassured Biogen that there is still a viable, long-term reimbursement path for the significant therapeutic advancement in England.
O’Regan elaborates: “It’s not an ideal situation, but as long as there is an adherence to what was intimated in that meeting – that there needs to be more flexibility, there needs to be collaboration, and these two processes need to run in parallel – I think that could be good news. Good news in terms of eligible patients getting access to this therapy, but also this could be good news in terms of trying to make up some of the lost time that we’ve had since nusinersen got its marketing authorisation in Europe.”
Nusinersen received European marketing authorisation following accelerated assessment in May 2017. So far, nusinersen is reimbursed in Italy and Sweden with ongoing negotiations taking place across Europe.
Due to the significant clinical improvements shown with nusinersen in clinical trials and the urgent need for a treatment where before there was none, Biogen opened one of the largest global expanded access programmes in rare disease (since Autumn 2016) as an interim measure for those with infantile-onset spinal muscular atrophy (consistent with type one). To date, all eligible children in the UK have been treated with nusinersen following collaboration between Biogen and the NHS in which it was agreed that Biogen would provide the medicine free of charge, and the NHS would devise an urgent policy to administer the treatment.
Biogen aim to ensure that every eligible patient living with spinal muscular atrophy has access to nusinersen. O’Regan is hopeful: “Our focus now is to make sure that patients with later symptom onset SMA where clinical benefit has been demonstrated, also have access. I think that’s what this means. The progress being made with NICE and NHS England – I think it’s good news.”