The National Institute for Health and Care Excellence (NICE) has recommended a treatment for advanced Parkinson's disease that it said could benefit hundreds of patients in England.
The drug foslevodopa–foscarbidopa (Produodopa, AbbVie) should be an option for treating advanced levodopa-responsive Parkinson's disease in adults whose symptoms include severe motor fluctuations and hyperkinesia or dyskinesia, the NHS spending regulator said in final draft guidance.
Treatment with foslevodopa–foscarbidopa was an option when available medicines were not working well enough, and only if patients could not have apomorphine or deep brain stimulation, or these treatments no longer controlled symptoms, an appraisal committee stipulated.
Approximately 153,000 people live with Parkinson's disease in the UK, according to figures from the charity Parkinson's UK.
NICE acknowledged that advanced Parkinson's disease "severely affects the quality of life of people with the condition and their family and carers". It estimated that because of its decision, about 900 adults with advanced Parkinson's could benefit.
'More Reliable' Symptom Management
The treatment, which is administered subcutaneously via an infusion pump, works by releasing the drugs foslevodopa and foscarbidopa into the body. Foslevodopa is converted into dopamine, and foscarbidopa increases the availability of foslevodopa in the brain.
Helen Knight, director of medicines evaluation at NICE, said: "Foslevodopa–foscarbidopa represents an important new treatment for people with advanced Parkinson's, providing an easy-to-use option that can help them manage their symptoms more reliably and effectively."
Go-Ahead Given Despite Uncertainties
Evidence from a clinical trial suggested that foslevodopa–foscarbidopa improved motor symptoms compared with standard care. However, some people in the trial had previously had apomorphine, so appraisers said it was "uncertain how well foslevodopa–foscarbidopa works for people who cannot have apomorphine". The regulator added that an indirect comparison suggested that foslevodopa–foscarbidopa worked as well as levodopa–carbidopa intestinal gel, but that the results were, again, "uncertain".
"There is some uncertainty in the treatment effects, which could lead to the benefits of foslevodopa–foscarbidopa being overestimated," NICE pointed out. It also considered that the cost-effectiveness estimates for foslevodopa–foscarbidopa were uncertain.
However, NICE acknowledged the magnitude of the unmet need, and the treatment's many potential benefits. Therefore, despite some uncertainties, NICE decided that the most likely cost-effectiveness estimates were within the range usually deemed acceptable for use of NHS resources, and the treatment was recommended.
Under the terms of a confidential agreement, the manufacturer must also provide the drug to the NHS at a discount, NICE said.
Potential for a 'Big Impact'
This was the first technology appraisal from NICE to look at a treatment for Parkinson's disease. The regulator had previously only published clinical guidelines and medtech innovation briefings on the condition.
Commenting for Medscape News UK, Laura Cockram, head of campaigns at Parkinson's UK, welcomed the NICE recommendation and said that the treatment could be a "life-changing" option for those whose symptoms were not well controlled by oral medication, and that the charity was keen to see NHS bodies approve and use the treatment.
She said that the NICE decision would offer hope to many families, and it could potentially have a "big impact".
NICE assured patients already in receipt of the treatment that they could continue without any change to the funding arrangements in place before publication of the guidance until they and their NHS clinician considered it appropriate to stop.
Final guidance is expected on 29 November, 2023.