OBJECTIVES: Decision-analytic models assessing the value of emerging Alzheimer's disease (AD) treatments are challenged by limited evidence on short-term trial outcomes and uncertainty in extrapolating long-term patient-relevant outcomes. To improve understanding and foster transparency and credibility in modeling methods, we cross-compared AD decision models in a hypothetical context of disease-modifying treatment for mild cognitive impairment (MCI) due to AD. METHODS: A benchmark scenario (US setting) was used with target population MCI due to AD and a set of synthetically generated hypothetical trial efficacy estimates. Treatment costs were excluded. Model predictions (10-year horizon) were assessed and discussed during a 2-day workshop. RESULTS: Nine modeling groups provided model predictions. Implementation of treatment effectiveness varied across models based on trial efficacy outcome selection (clinical dementia rating - sum of boxes, clinical dementia rating - global, mini-mental state examination, functional activities questionnaire) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6 to 5.2 years for control strategy and from 0.1 to 1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0 to 0.6 and incremental costs (excluding treatment costs) from -US$66 897 to US$11 896. CONCLUSIONS: Trial data can be implemented in different ways across health-economic models leading to large variation in model predictions. We recommend (1) addressing the choice of outcome measure and treatment effectiveness assumptions in sensitivity analysis, (2) a standardized reporting table for model predictions, and (3) exploring the use of registries for future AD treatments measuring long-term disease progression to reduce uncertainty of extrapolating short-term trial results by health-economic models.
- MeSH
- Alzheimer Disease * economics drug therapy MeSH
- Cost-Benefit Analysis * MeSH
- Models, Economic MeSH
- Cognitive Dysfunction * economics MeSH
- Quality-Adjusted Life Years MeSH
- Humans MeSH
- Decision Support Techniques MeSH
- Disease Progression MeSH
- Treatment Outcome MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Comparative Study MeSH
- MeSH
- Health Services Accessibility MeSH
- Health Care Economics and Organizations * MeSH
- Healthcare Financing MeSH
- Humans MeSH
- Health Promotion MeSH
- Fees, Medical MeSH
- Telemedicine MeSH
- Influenza Vaccines MeSH
- Physician-Patient Relations MeSH
- Quality Assurance, Health Care MeSH
- Employer Health Costs MeSH
- Healthy Lifestyle MeSH
- Check Tag
- Humans MeSH
- Publication type
- Comment MeSH
- Newspaper Article MeSH
Pozvolna, ale jistě nastává nová éra inovativních léčebných možností Alzheimerovy nemoci. Objevují se nové cíle léčby a vznikají různé monoklonální protilátky zasahující cíleně molekuly uplatňující se v patogenezi Alzheimerovy nemoci. Cílem přehledové práce je seznámit čtenáře s novými potenciálními léčivy, přičemž většina uvedených v tomto přehledovém článku je právě ve 3. fázi klinické studie.
A new era of innovative treatment options for Alzheimer‘s disease is emerging. New goals of treatment are being developed with various monoclonal antibodies targeting potentially pathological molecules involved in the pathogenesis of Alzheimer‘s disease. The aim of this review is to acquaint the reader with new potential drugs, while most of those mentioned in this review are in the third phase of clinical trials.
- Keywords
- aducanumab,
- MeSH
- Alzheimer Disease * drug therapy MeSH
- Antibodies, Monoclonal, Humanized pharmacology therapeutic use MeSH
- Clinical Trials, Phase III as Topic MeSH
- Humans MeSH
- Antibodies, Monoclonal pharmacology therapeutic use MeSH
- Drug Approval MeSH
- Check Tag
- Humans MeSH
- Publication type
- Research Support, Non-U.S. Gov't MeSH
- Review MeSH
