In 2025, the UK's healthcare landscape witnessed a pivotal shift in drug approvals, reflecting a careful balance between innovation and cost-effectiveness. The spotlight shone on treatments for endometriosis, haemophilia, rare diseases, HIV prevention, and cancer, with a critical eye on affordability and clinical impact. The National Institute for Health and Care Excellence (NICE) played a pivotal role, recommending or rejecting treatments based on their value proposition. Here's a breakdown of the year's significant decisions and the stakeholders who benefited from them.
Endometriosis Treatment: A Step Forward
A major breakthrough occurred in women's health with the approval of the first long-term daily oral treatment for endometriosis. This decision by NICE marked a significant advancement, covering adults of reproductive age whose symptoms had not responded to previous medical or surgical interventions. The treatment, a combination of relugolix, estradiol, and norethisterone, offers a more rapid and convenient approach compared to injectable gonadotropin-releasing hormone agonists. This approval not only addresses a critical unmet need but also empowers women with a more manageable treatment regimen.
Haemophilia Care: Weekly Relief
NICE recommended marstacimab, a weekly subcutaneous injection, for individuals with severe haemophilia B. This drug targets tissue factor pathway inhibitor, enhancing clotting activity and reducing bleeding episodes. While it showed promise in haemophilia A, NICE deemed it cost-ineffective for this group. The approval of efanesoctocog alfa for severe haemophilia A further solidified the commitment to improving bleeding management in this rare genetic disorder.
Rare Diseases and Devolved Decisions
The Scottish Medicines Consortium (SMC) made a landmark decision by accepting givinostat for patients with Duchenne muscular dystrophy aged 6 and older. This approval, despite the lack of disease-modifying treatment options, sparked calls for NICE to follow suit. Additionally, maralixibat received approval for cholestatic pruritus caused by Alagille syndrome in young children, highlighting the importance of tailored healthcare solutions for rare conditions.
HIV Prevention: Injectable Innovation
Cabotegravir, an injectable form of HIV pre-exposure prophylaxis (PrEP), gained approval for use in England and Wales, following its earlier endorsement in Scotland. This move aligns with global efforts to end new HIV transmissions by 2030, offering a convenient and effective prevention method administered every two months by healthcare professionals.
Cancer Approvals and Firsts
Cancer treatment saw significant advancements with the approval of tisotumab vedotin for recurrent or metastatic cervical cancer, providing a much-needed treatment option for patients with limited alternatives. NICE's recommendation of durvalumab for limited-stage small-cell lung cancer marked a 20-year breakthrough, with clinical trials demonstrating improved survival rates. Enfortumab vedotin combined with pembrolizumab emerged as a first-line treatment for advanced bladder cancer, addressing a decades-long gap in treatment options.
Reversals of Fortune
Pembrolizumab, when combined with carboplatin and paclitaxel, was recommended for advanced endometrial cancer, marking the first time immunotherapy and chemotherapy were combined as a first-line treatment. This decision reversed NICE's earlier rejection due to cost concerns. Similar reversals occurred for sparsentan in primary immunoglobulin A nephropathy and the combination of nivolumab and ipilimumab in advanced colorectal cancer.
Cost-Effectiveness Rejections
Despite the approvals, affordability remained a critical challenge. NICE rejected orasidenib for certain low-grade brain tumours, citing insufficient cost-effectiveness. Cerliponase alfa, intended for neuronal ceroid lipofuscinosis type 2, was also turned down due to its high annual cost. Two Alzheimer's disease treatments, donanemab and lecanemab, faced rejection for their limited clinical benefits and high prices.
Technology and Devices
Regulators approached new technologies with caution. NICE conditionally recommended an AI system for assessing suspicious skin lesions, emphasizing the need for further evidence. Leadless cardiac pacemakers were provisionally recommended for bradyarrhythmias, reducing infection risks. Additionally, 11 robotic surgical systems were approved for NHS use, with applications in soft tissue and orthopaedic procedures.
Safety Reassurance
The MHRA reassured the public that paracetamol remains safe during pregnancy, addressing claims by former US President Donald Trump. Health Secretary Wes Streeting advised pregnant women to disregard the claims, emphasizing the trustworthiness of British medical professionals and the NHS.
In summary, the year's decisions underscored the ongoing challenge of balancing innovation and cost-effectiveness in healthcare. NICE's role as a gatekeeper highlights the need for rigorous evaluation to ensure that NHS patients receive the most valuable treatments available.
