The Scottish Medicines Consortium (SMC) has accepted Novartis Gene Therapies’ Zolgensma and Genentech’s Rozlytrek for use by NHS Scotland as part of its March 2021 decisions.
Zolgensma (onasemnogene abeparvovec) is the first gene therapy accepted by the SMC for use by NHS Scotland and is intended to treat spinal muscular atrophy (SMA).
The SMC reviewed Zolgensma through its Patient and Clinician Engagement (PACE) process, which is used for medicines for the treatment of rare diseases and end of life.
The PACE meeting for Zolgensma highlighted that SMA has a ‘devastating’ impact on families and patients and that the gene therapy – which is intended to be a one-time treatment – would be less disruptive to life compared to ongoing hospital visits required for the other available treatment for SMA.
“This is the first gene therapy the committee has accepted for use by NHS Scotland using our process for medicines for rare conditions, which allows us greater flexibility in relation to cost-effectiveness,” said Mark MacGregor, chairman of the SMC.
“This medicine has the potential to be life changing for patients and their families. However, it is extremely expensive for the single dose required, even with the Patient Access Scheme discount offered by the company.
“These are incredibly difficult decisions. Allocating resource to new innovations diverts that resource from other important needs. We expect the pharmaceutical industry will work on improving its efficiency to deliver such high cost innovations at lower cost, which is essential for the sustainability of healthcare systems around the world,” he added.
The SMC also accepted Rozlytrek (entrecitinib) for the treatment of solid tumours with NTRK gene fusion.
Rozlytrek is a tumour agnostic treatment, meaning that it can be effective in tumours with specific genetic features, regardless of where that tumour is situated in the body.
The SMC also reviewed Rozlytrek through its PACE review process. In the PACE meeting, participants highlighted that tumours of this kind can often affect teenagers and young adults and have a significant impact on patients, family and friends.
Rozlytrek is also taken orally, meaning patients can avoid frequent hospital visits for other potential treatments, according to the PACE meeting participants.
