Monday, July 15, 2024

Guidance and Treatments Released by NICE During Pre-Election Period Highlighting Healthcare Dedication

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During the pre-election period, restrictions on public bodies prevented the National Institute for Health and Care Excellence (NICE) from publicizing its work, but despite this, NICE continued to develop and release guidance, publishing over 20 pieces of draft and final guidance to ensure patients received the best treatments quickly. This commitment to maintaining the momentum of their work, even under restrictive conditions, highlights the organization’s dedication to improving healthcare access and outcomes. By focusing on the critical task of developing and disseminating guidance, NICE ensured that patients could benefit from the latest advancements in medical treatments without delay.

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. This new treatment option can benefit around 130 people, providing a crucial new avenue for those affected by this specific type of leukemia. The availability of ivosidenib represents a significant step forward in the treatment of acute myeloid leukemia, offering patients a novel therapeutic option that targets the underlying genetic mutation driving their disease.

On the same day, NICE launched a consultation on draft recommendations to offer the weight loss medication tirzepatide alongside a reduced-calorie diet and increased physical activity to people with a BMI of 35+ and at least one weight-related comorbidity. This proposal aims to provide an effective and comprehensive treatment strategy for individuals struggling with obesity and related health issues. By addressing the multifaceted nature of weight management through a combination of medication, dietary changes, and increased physical activity, NICE hopes to support sustained weight loss and improved health outcomes for this population.

NICE Issues Final Guidance on New Treatments for Sickle Cell Disease and Transthyretin Amyloidosis

On 12 June, NICE recommended voxelotor (Oxbryta by Pfizer) for treating hemolytic anemia caused by sickle cell disease (SCD) in the final guidance. This decision allows around 4,000 people with SCD to benefit from the new treatment following a price reduction. Voxelotor represents a significant advancement in the management of SCD, offering patients a treatment that directly addresses the underlying cause of their anemia. By improving the oxygen-carrying capacity of red blood cells, voxelotor can help reduce the frequency and severity of sickle cell crises, ultimately enhancing the quality of life for individuals living with this chronic condition.

On 19 June, NICE published final guidance recommending tafamidis (Vyndaqel by Pfizer) for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This once-a-day tablet treats the disease rather than just managing symptoms and is expected to help around 800 people in the UK. Tafamidis offers a much-needed treatment option for ATTR-CM, a progressive and debilitating condition that affects the heart. By stabilizing the transthyretin protein and preventing its accumulation in the heart tissue, tafamidis can slow the progression of the disease and improve patients’ overall prognosis.


NICE Issues New Guidance on Cystic Fibrosis, Hemophilia B Gene Therapy, and Joint Asthma Management

On 20 June, NICE recommended the cystic fibrosis treatments Kaftrio, Symkevi, and Orkambi following a new commercial deal between NHS England and Vertex. This deal ensures continued access for around 8,200 people already receiving these treatments, along with future patients. The agreement also includes access to future license extensions of these medicines. The collaboration between NHS England and Vertex marks a significant milestone in the treatment of cystic fibrosis, ensuring that patients will continue to benefit from these life-changing therapies. Additionally, the deal paves the way for future advancements in cystic fibrosis treatment, with potential new therapies becoming accessible as they are developed and approved.

Etranacogene dezaparvovec (Hemgenix by CSL Behring) became the first gene therapy recommended by NICE via the Technology Appraisal programme. This treatment for haemophilia B, which affects around 260 adults, is also the first to enter the Innovative Medicines Fund. The one-off infusion was recommended while further data on its long-term effects is collected. Etranacogene dezaparvovec represents a groundbreaking advancement in the treatment of haemophilia B, offering the potential for a long-lasting solution to the bleeding disorder. By delivering a functional copy of the gene responsible for producing clotting factor IX, this gene therapy can significantly reduce the frequency of bleeding episodes and improve the quality of life for patients with haemophilia B.

On 18 June, NICE, the British Thoracic Society (BTS), and the Scottish Intercollegiate Guideline Network (SIGN) opened a public consultation on the draft joint guideline for asthma diagnosis, monitoring, and chronic asthma management. This is the first UK-wide joint guidance for managing chronic asthma in adults, young people, and children. The consultation will be open until 30 July, with the final guideline expected later this year. This collaborative effort aims to standardize asthma care across the UK, ensuring that all patients receive consistent and evidence-based treatment. By bringing together expertise from NICE, BTS, and SIGN, the new guideline will provide comprehensive and up-to-date recommendations for healthcare professionals managing asthma, ultimately improving patient outcomes and quality of life.

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Resource: National Institute for Health and Care Excellence, July 09, 2024

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