Services to deliver Onasemnogene abeparvovec/Zolgensma

Spinal muscular atrophy, or SMA, is a rare genetic disorder that causes muscle weakness and progressive loss of movement. It is most commonly caused by defects in the gene SMN1.

Onasemnogene abeparvovec (Zolgensma, AveXis) is a single-use gene replacement therapy made of a viral vector that has been modified to contain the primary gene for the human survival motor neuron (SMN) protein, which is lacking or mutated in people with SMA. When injected, the vector is expected to carry the gene into the nerve cells, enabling production of sufficient amounts of SMN. It is administered intravenously.

NHS England is seeking to commission a service to deliver Onasemnogene abeparvovec/Zolgensma from four providers in England. It is currently anticipated that there may be one provider to cover the North of England, one to cover the Midlands, one to cover the South of England plus one other. The commissioner is by no means fixed on this geographical spread as the quality of the service provision is the most important factor.

As well as having a neuromuscular MDT and experience in treating children with SMA, the successful providers will need to have the capability to safely manage the product itself in line with it being classified as 'containment level 1' and ensure that all elements of the patient pathway (pre-treatment, during treatment and post-treatment) can be put into place. The providers will also be expected to operate as a national-MDT to ensure that eligible patients can be treated within appropriate timescales.

It is anticipated that the number of children requiring treatment each year is between 25 and 35, although this number may be subject to change, depending on the outcome of the NICE evaluation and, for example, if new-born screening for SMA was introduced.

Novartis Gene Therapies is the company who produce this gene therapy and providers will be required to comply with the company's contractual documentation. This is currently in draft so providers will be expected to have contacted Novartis Gene Therapies prior to submitting a tender response to discuss their requirements in respect of providing a service to deliver Onasemnogene abeparvovec/Zolgensma.

Given the significant amount of preparatory work that needs to be undertaken to deliver a gene therapy service, it is not feasible for commissioners and providers to wait until NICE has made its recommendation. To do so would risk the NHS not being ready to provide access within the timeframe set out in the Mandate requirements on NICE approved treatments and not being able to make the treatment available as soon as possible after the NICE decision has been made. Providers are therefore submitting bids to deliver a service 'at risk' and it may be the case that the product does not receive a positive NICE recommendation. Bidders are responsible for their own costs incurred throughout each stage of the Procurement process.