Nusinersen: wider PBS listing recommended
The Pharmaceutical Benefits Advisory Committee has recommended extending the existing PBS listing of nusinersen (Spinraza) to include the treatment of all paediatric patients with Type III Spinal muscular atrophy (SMA) who have symptom onset prior to 18 years of age.
At its July meeting the PBAC said this would provide equity of access across paediatric patients.
However the PBAC rejected an application by Biogen to extend the PBS listing of nusinersen to include adults with SMA. The committee said it recognised the clinical need for effective treatments for adult SMA but considered that the adult population most likely to benefit from treatment with nusinersen remained inadequately defined in the resubmission.
It advised that a listing for adults would require a substantial price reduction, a risk sharing arrangement (RSA), and a Managed Access Program (MAP).
At the July meeting the PBAC also recommended opicapone (Ongentys) capsules as a General Schedule, Restricted benefit listing for the treatment of Parkinson Disease, as adjunctive therapy to levodopa-decarboxylase inhibitor combinations in patients motor function fluctuations due to end-of- dose effects.
Nitrous oxide ‘nangs’ misuse leads to myelo-neuropathy
Nitrous oxide misuse can cause a severe subacute myelo-neuropathy that may be reversible with vitamin B12 therapy, a review of 20 cases presenting to Sydney hospitals has found.
A study led by Dr Grace Swart of the Department of Neurology, St Vincent’s Hospital, Sydney, found that the average age of patients was 24 years and they consumed an average of 148 canisters per day for nine months. At presentation paraesthesiae and gait unsteadiness were common. Serum and active B12 could be normal, while elevated homocysteine and dorsal column high T2 signal on MRI imaging strongly suggested the diagnosis, the investigators said. Nerve conduction studies showed a predominantly axonal sensorimotor neuropathy.
Treatment with intramuscular vitamin B12 resulted in variable functional recovery, with three of seven bedbound patients at baseline able to walk again. However, most patients persistent paraesthesiae and/or sensory ataxia, they reported in the European Journal of Neurology.
10 Choosing Wisely recommendations for COVID-19
An international taskforce on Choosing Wisely for COVID-19 has released its top ten recommendations for the general public and for physicians.
The 18-member taskforce encompassed the disciplines of public health, primary care, infectious diseases, respiratory medicine, critical care and more.
The five recommendations for the general public reinforce prevention messages, when to get tested and when to seek medical help.
The five recommendations for physicians focus on the management of COVID-19. They are:
- Do not use prescribe unproven or ineffective therapies for COVID-19
- Do not use drugs like remdesivir and tocilizumab except in specific circumstance where they may be of use
- Do use steroids prudently only in patients with hypoxia, and monitor blood sugar levels to keep them in normal range
- Do not routinely perform investigations that do not guide treatment, such as CT scans and inflammatory markers
- Do not ignore the management of critical non-COVID-19 disease during the pandemic.
Read more in Nature Medicine.