Donanemab Approved by MHRA but Rejected by NICE for NHS Use Due to Cost Concerns
核心洞察
Donanemab, a new Alzheimer's treatment by Eli Lilly, has been approved by the MHRA but rejected by NICE for use in the NHS due to high costs.
NICE determined that donanemab does not offer sufficient value for NHS use, despite clinical evidence showing it could slow disease progression by 4-7 months.
Concerns over the drug's safety profile, including amyloid (搜索)-related imaging abnormalities (ARIA) in 33% of participants, and the need for monthly infusions contributed to the rejection.
The Alzheimer's treatment donanemab, manufactured by Eli Lilly, has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. However, the National Institute for Health and Care Excellence (NICE) has issued a draft decision rejecting its use within the National Health Service (NHS) due to concerns over cost-effectiveness.
Efficacy and Cost-Effectiveness Concerns
Donanemab, also known as Kisunla, is designed to slow cognitive decline in patients with early-stage Alzheimer’s disease by targeting beta-amyloid (搜索) plaques in the brain. Clinical evidence presented to NICE indicated that the drug could slow the progression of Alzheimer’s disease by 4 to 7 months. Despite this potential benefit, NICE's independent committee concluded that the cost-effectiveness estimate for donanemab was five to six times higher than what is typically considered acceptable for NHS resource allocation.
Helen Knight, director of medicines evaluation at NICE, stated, “While the drug offers some benefit, this is just not enough to justify the significant cost to the NHS.” She noted that the draft decision could be revisited if further evidence from Eli Lilly and NHS England becomes available.
Safety Profile and Monitoring Requirements
In addition to cost concerns, NICE also raised questions about the drug’s safety profile. Clinical trials revealed that 33% of participants experienced amyloid (搜索)-related imaging abnormalities (ARIA), including brain swelling and bleeding. The administration of donanemab requires monthly hospital infusions and intensive monitoring, adding to the overall cost and logistical challenges for the NHS.
With an estimated 70,000 patients in England potentially eligible for the treatment, the financial implications for the NHS were a critical factor in NICE’s decision. Total treatment costs for donanemab in the US, including monitoring and scans, average $78,000 per year per patient.
Industry and Advocacy Group Response
The Alzheimer’s Society responded to the news with mixed emotions. Professor Fiona Carragher, Chief Policy and Research Officer at Alzheimer’s Society, remarked, “Disease-modifying therapies like donanemab and lecanemab offer a new horizon of hope in the fight against dementia. However, we respect NICE's decision and hope to see improvements in the affordability and efficacy of treatments over time, as we have seen in other diseases like cancer.”
Professor Sir John Hardy, a leading neurological researcher at UCL, expressed disappointment with NICE's decision, stating that the drug was 'game-changing' and that Nice was 'coming down on the wrong side of the argument'. He added, 'These drugs can give people an extra two years at home, rather than in a nursing home. That is time enjoying their lives, having holidays – this is important stuff.'
Future Considerations
NICE has announced that it is monitoring around 27 new products in the Alzheimer’s disease pipeline. The organization also plans to revisit its guidance on Alzheimer’s care and diagnostics, including researching blood biomarkers for earlier and more accurate detection.
The consultation period for NICE’s draft guidance on donanemab will run until 20 November 2024, during which further evidence and responses will be reviewed. A final decision on donanemab’s NHS availability is expected in the coming months. The Alzheimer’s community also awaits the final recommendations for lecanemab, with its second committee meeting scheduled for November 2024.