[ad_1]
NHS England has entered into an "access management" agreement with Biogen for its Spinraza drug for spinal muscular atrophy (SMA), which, according to the health services budget holder, is one of the most comprehensive in the world. world.
This announcement is the latest sign that the tough new negotiations at NHS England, but a quick approach to price negotiations, are helping to accelerate market access in the country – even though pharmaceutical companies need to make significant tariff concessions at home. behind closed doors.
Spinraza was first evaluated by NICE in August 2018, but uncertainties about its long-term clinical benefits and high catalog price – £ 450,000 for the first year and £ 225,000 for the following years – have been rejected.
Now the NHS England has stepped in again to negotiate – leaving both sides with the room for maneuver that was lacking when a NICE "no" marked the end of the discussion – a situation that now seems to be a thing of the past.
The managed access agreement means that the NHS will fund the treatment for a limited period, which will allow to collect additional data on its effectiveness. This approach is very similar to the Cancer Fund, managed by NICE, but it is currently limited to oncology treatments, although details of the Spinraza agreement have not yet been disclosed.
The agreement paves the way for NICE to formally approve the treatment to be used with eligible patients via its usual final guidelines.
The treatment will be made available immediately to Biogen for the youngest and most severely affected patients (ADM type 1), with NHS England providing funding for the release of the final guidelines by NICE.
For older babies, children and young adults with less severe symptoms (ADM types 2 and 3), the NHS will begin providing nusinursen shortly after the publication of NICE guidelines, once services for provide them will be established.
This is consistent with NICE's usual processes, and the NHS England said it would not take more than a few weeks.
Simon Stevens, CEO of NHS England, said, "This promising treatment has the potential to change the lives of children and their families.
"This is one of the most comprehensive offerings in the world, which means that all patients with AMS (AMS 1,2,3a and 3b), including adults and siblings who have not not yet presented with symptoms, will benefit from this treatment.
"This latest agreement, which follows a number of other recent and successful negotiations, shows that there is no reason why other companies do not show the same flexibility to benefit NHS patients, taxpayers and even themselves. "
Terry O'Regan, Vice President and General Manager of Biogen UK and Ireland, said:
"The NICE recommendation for infants, children, and adults with 5q ADM, including pre-symptomatic type I, type II, and type III ADMs, is a critical opportunity for patients and their families who have fought without release to have access to this revolutionary drug. drug.
"This positive result has been achieved through intensive and collaborative work between the SMA community, NICE, NHS England and Biogen."
He concluded: "We will continue to work with the health authorities to ensure that this welcome decision translates into immediate access to those awaiting treatment, which includes providing the NHS England with immediate access to nusinersen for treatment. type I patients. "
The breakthrough in England comes at a pivotal moment in the treatment of the SMA, while two competitors of Spinraza wait in the wings: the oral treatment of Roche, risdiplam and gene therapy Novartis, Zolgensma.
The arrival of these rivals in 2021 and 2020, respectively, is good news for payers. It gave new impetus to the discussions in England, both for Biogen and the NHS England.
Since its launch in 2016, Spinraza has become Biogen's main growth driver and generated $ 518 million in sales in the first quarter of this year.
NICE still needs a reform
Despite the relief resulting from a breakthrough – particularly for patients and their representatives – calls for reform of NICE's assessment system remain unresolved in order to avoid similar problems in # 39; future.
The activists said that one of the major flaws of the system was that Spinraza had been evaluated via NICE's unique technology evaluation method (STA) – this is because the disease is too prevalent to go through highly specialized technology (HST) of NICE, which is reserved for the rarest orphan and ultra-orphan treatments. Patient groups say that the economic aspects of a rare disease like ADM make it very unlikely that it will go through the NICE STA.
Muscular Dystrophy UK points out that Spinraza is only the second drug evaluated according to this pathway to be approved for a controlled access agreement.
UK and UK Atrophy Spinal Musclular Atrophy say they will continue to push for the process of evaluating drugs for rare diseases to be changed.
In the meantime, they are asking NICE to officially recommend treatment and Biogen and NHS England to implement the MAA urgently so that patients can access the drug quickly, and in Wales and Northern Ireland , which generally follows NICE guidelines, to follow his example.
Catherine Woodhead, Executive Director of British Muscular Dystrophy said:"Every day counts for people with ADMs and we need to make sure this decision is implemented as soon as possible and made available in the UK. Our work here is not over. "
She added, "The long and frustrating delays we observed throughout this process must not be repeated, and during this time, the health of ADM patients will have declined irreversibly. Evaluation of new treatments for rare diseases needs to be urgently revised to adapt to the needs of future treatments for rare conditions. "
<! –
->
Source link
