Table 3 Clinical study design and methodology for early benefit assessment in Germany (G-BA/IQWIG) and single technology appraisal in England (NICE) – a comparison
From: Requirements for benefit assessment in Germany and England – overview and comparison
Methodological element | Benefit assessment in Germany (G-BA/IQWIG) | Single Technology Appraisal in England (NICE) |
|---|---|---|
Study design features | ||
Description | According to international standards. | According to international standards. |
Study type | RCTs clearly preferred; case-series acceptable if dramatic therapeutic effect. | RCTs preferred; non-RCTs and other evidence also desired (adjustment of data through modeling). |
Subgroup analysis | Always done (effect modifier; interaction tests). | Always done (statistical tests). |
Study duration | Important criterion for relevance of evidence (guidelines). | Data extrapolation through modeling. |
Comparator and comparison | ||
Choice of comparator | Preferably found beneficial in previous assessments; most economic (if alternatives); preferably reference priced; licensed; can be non-drug intervention; consultation procedure request (advice from G-BA); the comparator is determined by G-BA; often more than one comparator. | Best standard care (most commonly used, most cost-effective, also non-licensed or no intervention); input from manufacturer and other stakeholders during scoping; often more than one comparator. |
Direct and indirect comparisons | Direct comparison preferred; indirect comparison possible. | Direct comparison preferred; Indirect comparison possible. |
Endpoints/outcomes | ||
Clinical endpoints | Relevance to patients (mortality, morbidity, quality of life); reporting by patients (e.g. HRQL, symptom scores). | Relevance to patients (mortality, morbidity, quality of life) or carers; reporting by patients (e.g. HRQL); translatable into utilities (e.g. QALYs); ease of use of the technology; experience with use of the technology. |
Surrogate endpoints | Validation study applicable to the disease, its severity, the intervention, and the comparator required (exception: very serious diseases). | Accepted if correlation with final endpoint is strong or outcome measures are large. |
Composite endpoints | Accepted if components patient-relevant and also reported separately. | Accepted. |
Safety | Analysis of relevant adverse events. | Analysis of relevant adverse effects. |