Saturday, July 13, 2024

Gene Therapy, Cystic Fibrosis Treatments, and Obesity Injection Included in Pre-Election Guidance

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Gene therapy advancements were a key focus during the pre-election period from 22 May to 5 June 2024. Despite restrictions preventing the National Institute for Health and Care Exellence (NICE) from publicizing its work, the organization continued developing essential guidance to ensure patients received the best treatments promptly. More than 20 pieces of draft and final guidance on various medicines and medical conditions were published during this time.

On 4 June, NICE issued final guidance recommending the once-a-day tablet ivosidenib with azacitidine (Tibsovo, by Servier Laboratories) for untreated acute myeloid leukemia with an IDH1 R132 mutation, benefiting around 130 patients. On the same day, a consultation was launched on draft recommendations for tirzepatide, a weight loss medication for individuals with a BMI of 35+ and at least one weight-related comorbidity, to be used alongside a reduced-calorie diet and increased physical activity.

NICE Recommends Voxelotor and Tafamidis for SCD and ATTR-CM

On 12 June, final guidance was published recommending voxelotor (Oxbryta, by Pfizer) for treating haemolytic anemia caused by sickle cell disease (SCD), potentially benefiting around 4,000 patients. This recommendation followed a price reduction, making the treatment accessible on the NHS. On 19 June, tafamidis (Vyndaqel, by Pfizer) was recommended for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a progressive, life-limiting condition affecting around 800 people in the UK. Tafamidis, taken as a once-a-day tablet, is the first medicine aiming to treat ATTR-CM directly, moving beyond symptom management and supportive care.

Final draft guidance for cystic fibrosis (CF) treatments Kaftrio, Symkevi, and Orkambi was published on 20 June, following a new commercial agreement between NHS England and Vertex. This agreement ensures that around 8,200 current and future CF patients in England will continue receiving these treatments routinely on the NHS. Additionally, it includes access to any future license extensions and a commitment to rapid access for the next-in-class triple combination treatment for CF, subject to MHRA authorization.

gene therapy

First Gene Therapy for Haemophilia and Outcomes-Based Pilot

On 27 June, NICE recommended etranacogene dezaparvovec (Hemgenix, by CSL Behring), the first gene therapy for haemophilia, via the Technology Appraisal program. This recommendation also marked the first haemophilia treatment and the first pilot for the outcomes-based scheme under the 2024 VPAG commitments. The one-off infusion is intended for around 260 adults with moderately severe or severe haemophilia B and is included in the Innovative Medicines Fund while further data on its long-term effects are collected.

On 18 June, a public consultation was opened on the draft joint guideline for asthma: diagnosis, monitoring, and chronic asthma management. This collaboration between NICE, the British Thoracic Society (BTS), and the Scottish Intercollegiate Guideline Network (SIGN) aims to produce new UK-wide guidance for diagnosing and managing chronic asthma in adults, young people, and children. The consultation will remain open until 30 July, with the final guideline expected later this year.

These developments during the pre-election period highlight NICE’s commitment to advancing healthcare through timely and effective guidance, ensuring that patients receive the most appropriate and innovative treatments available.


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Resource: National Institute for Health and Care Exellence, July 08, 2024

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