Today, news broke that baby Arthur Morgan is the first to receive ground-breaking gene therapy treatment Zolgensma®, reimbursed by the NHS.
This is not only life changing for those who suffer with various
chronic and life shortening illnesses but is a positive sign to the
pharmaceutical industry, who are the pioneers behind these
treatments. Furthermore, this occasion marks the second medical
treatment now available for children with SMA, after Spinraza
became available on the NHS to eligible patients in May 2019.
Zolgensma® is an advanced therapy medicinal products, which
could allow healthcare providers globally to shift from a paradigm
of illness management to one of cure. However, the route to market
for many of these treatments has been quite challenging,
particularly across Europe. One of the key issues in getting these
treatments to patients is that cell and gene therapies may look
costly from the outset, Zolgensma® is deemed to be the most
expensive drug in the world with a list price of £1.79m per
dose. However, there is a significant cost saving to healthcare
providers over time. In this case, NHS England have managed to
strike a confidential discount.
In the case of Zolgensma®, the treatment received marketing
authorisation back a year ago while the UK was still in the EU.
However, the drug has only been given 'conditional
authorisation'. This means that more clinical evidence is
needed about the medicine's safety and efficacy, which the
company is required to provide. Once further data is submitted to
the EMA, the marketing authorisation may be converted into a
standard marketing authorisation over time. It is also a regulatory
requirement that this medicine continues to undergo additional
monitoring, so monitored even more intensively than other
medicines.
The deal between Novartis and NHS England will pave the way for the
National Institute for Health and Care Excellence (NICE) to publish
draft guidance recommending treatment with Zolgensma®.
Overall, this is exciting news for the future of medicine. It's
also good to see that pharmaceutical companies are open to
negotiating prices to get treatments to patients. Moreover, the use
of another treatment by the NHS, is a clear signal to the UK life
sciences industry, particularly those operating within the cell and
gene therapy space that the pathway to market for innovative
treatments is getting clearer.
Summary of the facts:
- On 18th May 2020, the European Commission issued a conditional marketing authorisation for the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) to Novartis for treating spinal muscular atrophy (SMA) a serious condition of the nerves that causes muscle wasting and weakness.
- Zolgensma has been given 'conditional authorisation'. This means that there is more clinical evidence to come about the medicine's safety and efficacy, which the company is required to provide. The marketing authorisation is valid across all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom. Once further data is submitted to the EMA, the marketing authorisation may be converted into a standard marketing authorisation over time. It is also a regulatory requirement that this medicine continues to undergo additional monitoring, meaning that it is monitored even more intensively than other medicines.
- On 8th March 2021, NHS England struck a deal with Novartis Gene Therapies, to secure the drug for NHS patients in England at a substantial confidential discount and paves the way for the National Institute for Health and Care Excellence (NICE) to publish draft guidance recommending treatment with Zolgensma.
- Zolgensma, deemed to be the most expensive drug in the world with a list price of £1.79m per dose, and is now available through the NHS at an undisclosed discounted price that has been agreed between NHS England and Novartis.
- Today 1st June 2021, Baby Arthur become one of the first patients to be treated on the NHS with Zolgensma. The approval of the drug marks the second medical treatment now available for youngsters with SMA, after Spinraza became available on the NHS to eligible patients in May 2019.
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