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Hundreds of children in England are set to benefit from a treatment for cystic fibrosis - Kaftrio, after the UK's Medicines and Healthcare products Regulatory Agency (MHRA) confirmed an extension to its licence. With the licence extension, more than 1,300 children in England with cystic fibrosis, aged six to 11, are newly eligible for this treatment, which improves lung function and improves overall quality of life of patients. Earlier, Kaftrio was only licensed for those aged 12 and above. British patients were the first in Europe to benefit from Kaftrio, when NHS England secured a landmark deal in June 2020. NHS chief executive Amanda Pritchard said: "Since NHS staff delivered one of the fastest rollouts of Kaftrio in the world just over a year ago, the lives of thousands of patients with cystic fibrosis have been transformed. "Innovative treatments like Kaftrio are life-changing for patients and their families, and that is why the NHS has done all it can since we secured the deal for Kaftrio to ensure patients benefit as soon as possible.

Modulator therapies, manufactured by Vertex Pharmaceuticals (VRTX.O), may no longer be available for new cystic fibrosis (CF) patients in the UK. A draft guidance recently published by the National Institute for Health and Care Excellence (NICE) stated that it would not recommend the use of Kaftrio, Symkevi and Orkambi on new CF patients. Kaftrio and Symkevi are currently recommended for the treatment of cystic fibrosis in patients aged 6 years and older, while Orkambi is used for patients aged 1 and older. The NICE draft guidance highlighted medicine costs and the strain on resources and finances faced by the National Health Service (NHS) as reasons for removing these drugs.

Pharmacy professionals to be included as key stakeholders in the implementation, delivery and evaluation of a wide range of genomic services, said the Royal Pharmaceutical Society (RPS). RPS's statement has been developed in collaboration with pharmacy organisations who have co-badged the report, such as the British Oncology Pharmacy Association, the UK Clinical Pharmacy Association, Association of Pharmacy Technicians and the College of Mental Health Pharmacy. It looks at current and future roles for pharmacy professionals in genomic medicine across many aspects of practice such as person-centred care and collaboration, professional practice, education, leadership, management and research. Pharmacists and pharmacy technicians in the UK have already established roles in the application of genomic medicine in some areas of practice, such as antimicrobial stewardship and infectious diseases, and the management of certain genetic conditions, such as cystic fibrosis. The society believes, the current role of pharmacy professionals in genomics can be expanded upon in the future to both lead and support many relevant aspects of genomic implementation. These are described across all healthcare sectors, within the Genome UK strategy produced by the UK Government, and within the implementation plans published in England, Scotland and Wales. Lead for Pharmacogenomics at RPS Sophie Harding said: "Pharmacy professionals are the gatekeepers of medication safety and efficacy across all areas of healthcare. They are skilled at interpreting complex scientific data and use evidence-based medicine to maximise the benefits of treatments for patients, whilst supporting shared decision-making with patients and the multidisciplinary team.