National Institute for Health and Care Excellence (NICE) guidance on monitoring and management of Barretts oesophagus and stage I oesophageal adenocarcinoma

Barrett’s oesophagus is the only known precursor to oesophageal adenocarcinoma, a cancer with very poor prognosis. The main risk factors for Barrett’s oesophagus are a history of gastro-oesophageal acid reflux symptoms and obesity. Men, smokers and those with a family history are also at increased risk. Progression from Barrett’s oesophagus to cancer occurs via an intermediate stage, known as dysplasia. However, dysplasia and early cancer usually develop without any clinical signs, often in individuals whose symptoms are well controlled by acid suppressant medications; therefore, endoscopic surveillance is recommended to allow for early diagnosis and timely clinical intervention. Individuals with Barrett’s oesophagus need to be fully informed about the implications of this diagnosis and the benefits and risks of monitoring strategies. Pharmacological treatments are recommended for control of symptoms, but not for chemoprevention. Dysplasia and stage 1 oesophageal adenocarcinoma have excellent prognoses, since they can be cured with endoscopic or surgical therapies. Endoscopic resection is the most accurate staging technique for early Barrett’s-related oesophageal adenocarcinoma. Endoscopic ablation is effective and indicated to eradicate Barrett’s oesophagus in patients with dysplasia. Future research should focus on improved accuracy for dysplasia detection via new technologies and providing more robust evidence to support pathways for follow-up and treatment.

Read More
Maggio 2024

Assessment of quality of data submitted for NICE technology appraisals over two decades

Background
The National Institute for Health and Care Excellence (NICE) pioneered the Health Technology Assessment (HTA) processes and methodologies. Technology appraisals (TAs) focus on pharmaceutical products and clinical and economic data, which are presented by the product manufacturers to the NICE appraisal committee for decision-making. Uncertainty in data reduces the chance of a positive outcome from the HTA process or requires a higher discount.

Objective
To investigate the quality of clinical data (comparator, quality of life (QoL), randomised controlled trials (RCTs) and overall quality of evidence) submitted by the manufacturers to NICE.

Design
This retrospective evaluation analysed active TAs published between 2000 and 2019 (up to TA600).

Methods
For all TAs, we extracted data from the Assessment Group and Evidence Review Group reports and Final Appraisal Determinations on (1) the quality of submitted RCTs and (2) the overall quality of evidence submitted for decision-making. For single TAs, we also extracted data and its critique on QoL and comparators. Each category was scored for quality and analysed using descriptive statistics.

Results
409 TAs were analysed (multiple technology appraisals (MTA)=104, single technology appraisal (STA)=305). In two-thirds of TAs, the overall quality of evidence was either poor (n=224, 55%) or unacceptable (n=41, 10%). In 39% (n=119) of the STAs, the quality of comparative evidence was considered poor, and in 17% (n=51) unacceptable. In 44% (n=135) of STAs, the quality of QoL data was considered poor, 15% (n=47) unacceptable, 33% (n=102) acceptable and 7% (n=21) as good. Over 20 years of longitudinal analysis did not show improvements in the quality of evidence submitted to NICE.

Conclusion
We found that the primary components of clinical evidence influencing NICE’s decision-making framework were of poor quality. It is essential to continue to generate robust clinical data for premarket and postmarket introduction of medicines into clinical practice to ensure they deliver benefits to patients.

Read More
Febbraio 2024