£ 1.8m per gene therapy approved for NHS use in Scotland

One-time treatment for babies with rare hereditary diseases is the first gene therapy accepted by the Scottish Medical Consortium (SMC) for use in NHS Scotland and is being approved by the National Laboratory. Can be the most expensive treatment in. Health and Care Excellence (NICE) in England and Wales.

Onasemnogene Abeparbobeck (Zolgensma; Novartis Gene Therapies) costs £ 1.79 million per dose and is a potential treatment for infants with spinal muscular atrophy (SMA). A rare progressive neuromuscular condition caused by a mutation in the SMN1 gene on chromosome 5.

Affecting about 65 babies born in the United Kingdom each year, SMA affects the nerves in the spinal cord that control movement, causing weakness, progressive lack of exercise, and difficulty breathing and swallowing.

In a draft guidance published on March 8, 2021, NICE was given to infants up to 12 months of age with type 1 SMA, one of the most severe conditions occurring in about 60% of SMA cases. It is recommended to use.

NICE also recommends use in presymptomatic infants of SMA, which has up to three copies of the SMN2 gene, as part of a controlled access arrangement while further data are being collected.

According to the NICE, despite the high cost of treatment, infants have extraordinary benefits and may reach milestones in normal infancy development, so their use in the NHS is recommended. If left untreated, infants with type 1 SMA have a life expectancy of about 2 years.

The drug is offered to the NHS at a fixed price discount, but the actual price paid by the NHS is not disclosed for commercial reasons.

Meindert Boysen, Deputy Chief Executive Officer and Director of NICE’s Center for Health Technology Assessment, said the Commission concluded that Onasemnogene Aveparbovec was an important development in the treatment of SMA. It may allow babies to earn important exercise milestones such as sitting and walking independently, and for some babies diagnosed before symptoms appear to be closer to healing. There is a possibility.

“As with many new treatments for very rare diseases, limited evidence means that there is uncertainty about the long-term benefits of Onasemnogene aveparbovec,” he said. Told.

“With the help of everyone involved in the evaluation, it is now possible to recommend a treatment that is the most expensive drug NICE has ever evaluated. This allowed him to die before his second birthday. The baby’s life may be transformed. “

SMC Chair Mark MacGregor said the decision to accept the drug was “incredibly difficult.”

“The drug can change the lives of patients and their families, but even with the discounts on patient access schemes offered by the company, the single dose required is very expensive,” he said. Said.

Onasemnogene aveparbovec is the second gene therapy recommended by NICE using an adeno-associated virus (AAV) vector. In this case, the AAV vector has been modified to carry a functional copy of the SMN1 gene to the motor neurons with the aim of promoting survival and function in the motor neurons. It is given as a one-time infusion and its effects are believed to last a lifetime.

This treatment was considered under the NICE Highly Specialized Technologies program. The program reflects the difficulty of raising cost-effectiveness thresholds and developing treatments for very rare conditions that affect only a few patients per year.

NICE currently recommends disease-modifying therapy nusinersen to some people with presymptomatic SMA and type 1, 2, or 3 SMA as part of a controlled access contract.

The NICE Draft Guidance is open for consultation until April 6, 2021.