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Why has therapy development for dementia failed in the last two decades?
Gauthier S, Albert M, Fox N, Goedert M, Kivipelto M, Mestre-Ferrandiz J, Middleton LT.. Alzheimer's & dementia : the journal of the Alzheimer's Association, 2015-12-19
Topic: Risk, prevention and care in population studies
The success rate of the pharmaceutical research and development (R&D) for dementia drugs has been abysmally low, in the last two decades. Also low has been the number of pipeline drugs in development, compared to other therapy areas. However, the rationale of early terminations has not been reported in the majority of trials.
Key findings
- Also low has been the number of pipeline drugs in development, compared to other therapy areas.
- However, the rationale of early terminations has not been reported in the majority of trials.
- These are key findings of the recently published pharmaceutical pipeline analysis by the UK-based Office of Health Economics (OHE).
- Our understanding of main challenges include (1) the significant gaps of knowledge in the nosology and complexity of the underpinning biological mechanisms of the commonest, not familial, forms of late onset dementias; (2) low signal-to-noise ratio, notwithstanding the lack of validated biomarkers as entry and/or end-point criteria; (3) recruitment and retention, particularly in the asymptomatic and early disease stages.
Why it matters
This analysis reports that dementia drug development has had a very low success rate over two decades, with few pipeline drugs and reasons for early trial terminations often unreported. It identifies challenges in therapy development and does not offer a treatment, cure or prevention.
Study details
- Evidence strengthLow
How this study scored 51/100
Published in a leading specialty journal. Scored zero for study design, sample size, effect reported, studied in people and randomised / blinded because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 9 / 10 | Tier 2 — leading specialty journal (Alzheimer's & dementia : the journal of the Alzheimer's Association) |
| Journal's citation record | 2 / 4 | Alzheimer s & Dementia: journal h-index 228 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 1 / 5 | Rated “low” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 1 / 5 | 2016 — within 20 year(s) of the newest study here |
| Sample size | 0 / 4 | Participant count not stated — scores zero (not stated in the record — scored 0) |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 0 / 3 | Not stated whether this was studied in people — scores zero (not stated in the record — scored 0) |
| Randomised / blinded | 0 / 2 | No randomisation or blinding stated — scores zero (not stated in the record — scored 0) |
| Times cited | 5 / 7 | Cited 237 times (OpenAlex, 2026-10-10) |
| Citations per year | 3 / 5 | 19.8 citations a year over 12 year(s) since 2016 |
| Impact for its field and year | 4 / 6 | Cited 10.6× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 3 / 6 | Cited by 19 other studies in this library |
| Still being cited | 3 / 4 | Cited 50 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 3 / 4 | Last-listed author Lefkos T. Middleton: h-index 66 (OpenAlex, whole career) |
| Institution's track record | 2 / 3 | Johns Hopkins University: institution h-index 2,306 (OpenAlex) |
| Multi-centre collaboration | 3 / 3 | OpenAlex lists 12 institution(s) in 4 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 26710325) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 51 / 100 | — |
What each of these 20 components measures, and what this score is not →
Read the original
May need a subscription OpenAlex found no free copy of the full text (checked 6 Feb 1020), so the publisher may charge for it. The PubMed abstract is free.
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. The record names 7 authors; APA and Vancouver show the first six, BibTeX and the downloads carry all of them. Check the citation against the original before you use it.
APA 7th
Gauthier, S., Albert, M., Fox, N., Goedert, M., Kivipelto, M., Mestre-Ferrandiz, J., et al. (2016). Why has therapy development for dementia failed in the last two decades? Alzheimer's & dementia : the journal of the Alzheimer's Association. https://doi.org/10.1016/j.jalz.2015.12.003
Vancouver
Gauthier S, Albert M, Fox N, Goedert M, Kivipelto M, Mestre-Ferrandiz J, et al. Why has therapy development for dementia failed in the last two decades? Alzheimer's & dementia : the journal of the Alzheimer's Association. 2016. doi:10.1016/j.jalz.2015.12.003. PMID: 26710325.
BibTeX
@article{gauthier2016-26710325,
author = {Gauthier, S. and Albert, M. and Fox, N. and Goedert, M. and Kivipelto, M. and Mestre-Ferrandiz, J. and Middleton, L. T.},
title = {{Why has therapy development for dementia failed in the last two decades?}},
journal = {Alzheimer's \& dementia : the journal of the Alzheimer's Association},
year = {2016},
doi = {10.1016/j.jalz.2015.12.003},
pmid = {26710325},
url = {https://doi.org/10.1016/j.jalz.2015.12.003}
}
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