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The National Institute for Health and Care Excellence (NICE) has recommended faricimab as treatment option for adults with wet age-related macular degeneration or diabetic macular oedema. Thousands of people in England could benefit from the recommendation of a new drug that helps to treat two leading causes of sight loss and visual impairment. Faricimab is administered as an eye injection and is being recommended as an option for treating some adults with wet age-related macular degeneration (AMD) or with diabetic macular oedema (DMO). In the key clinical trials, aflibercept, another eye injection drug used to treat AMD and DMO, was administered every 8 weeks, while faricimab dosing, based on assessments of the disease activity, allowed for an interval of up to 16 weeks between doses, and was found to be equally effective. Up to 300,000 people in England with wet age-related macular degeneration (AMD) could be eligible to receive faricimab as part of their treatment alongside just over 28,000 people with diabetic macular oedema.

The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.

The National Institute for Health and Care Excellence (NICE) has recommended Enhertu for people with advance breast cancer. "There is not enough evidence yet to show how much longer people live with Enhertu compared with trastuzumab emtansine because the clinical trial is still ongoing. This means the cost-effectiveness estimates are highly uncertain and Enhertu cannot be recommended for routine use in the NHS," said NICE. The independent appraisal committee concluded that Enhertu could be cost-effective if further evidence from the ongoing trial and from NHS practice can show how much longer people live with treatment. Helen Knight, director of medicines evaluation at NICE, said: "Today's draft guidance is good news for people with this type of advanced breast cancer, who often experience severe and debilitating symptoms.

The Medicines and Healthcare products Regulatory Agency (MHRA) made its new International Recognition procedure (IRP) fully operational from 1 January 2024, inviting developers of new medicines to submit their applications for marketing authorisation. Beginning this year, the EC Decision Reliance Procedure (ECDRP) has been replaced by IRP, and the Mutual Recognition/Decentralised Reliance Procedure (MRDCRP) has come under its umbrella. The move is expected to further improve access to life-saving medicines for UK patients following the UK's departure from the European Union.