Home › Research › Health systems and care delivery
Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability
Emond Sarah K, Goettsch Wim G, Ollendorf Daniel A. Clinical pharmacology and therapeutics, 2025-02-20
Topic: Risk, prevention and care in population studies
Conducting high-quality health technology assessments requires high-quality evidence. With evolving regulatory standards for faster approval of new pharmaceutical products, health technology practitioners often find that the evidence base available to inform their work is lacking. This review article provides case examples of how health technology assessors have grappled with this tension, from the United States and European perspective, including experiences with new therapies for large populations, such as Alzheimer's disease, and gene therapies for ultra-rare conditions. The article concludes by offering some potential policy solutions that can meet the goals of robust evidence generation, patient access, and system affordability, including reimbursement with evidence development, outcomes-based contracts, and other types of managed entry agreements.
Key findings
- The article concludes by offering some potential policy solutions that can meet the goals of robust evidence generation, patient access, and system affordability, including reimbursement with evidence development, outcomes-based contracts, and other types of managed entry agreements.
Why it matters
This review discusses how health technology assessors handle limited evidence, using Alzheimer's disease therapies as one example. It offers policy solutions such as reimbursement with evidence development and outcomes-based contracts, but does not test any treatment, cure, or prevention.
Study details
- Evidence strengthLow
How this study scored 40/100
Published in a peer-reviewed 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 | 7 / 10 | Tier 3 — peer-reviewed journal (Clinical pharmacology and therapeutics) |
| Journal's citation record | 3 / 4 | Clinical Pharmacology & Therapeutics: journal h-index 271 (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 | 4 / 5 | 2025 — within 3 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 | 2 / 7 | Cited 6 times (OpenAlex, 2026-10-10) |
| Citations per year | 1 / 5 | 2.0 citations a year over 3 year(s) since 2025 |
| Impact for its field and year | 3 / 6 | Cited 5.8× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 1 / 6 | Cited by 1 other studies in this library |
| Still being cited | 1 / 4 | Cited 6 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 2 / 4 | Last-listed author Daniel A. Ollendorf: h-index 38 (OpenAlex, whole career) |
| Institution's track record | 1 / 3 | Utrecht University: institution h-index 1,157 (OpenAlex) |
| Multi-centre collaboration | 2 / 3 | OpenAlex lists 4 institution(s) in 2 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 39972486) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 40 / 100 | — |
What each of these 20 components measures, and what this score is not →
Read the original
- DOI: 10.1002/cpt.3579
- PubMed: 39972486
- Original source →
- Free full text: Clinical Pharmacology & Therapeutics →
Full text: free to read — OpenAlex lists this copy as free to read: the link above (checked 6 Feb 1020).
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. Check the citation against the original before you use it.
APA 7th
Emond Sarah, K., Goettsch Wim, G., & Ollendorf Daniel, A. (2025). Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability. Clinical pharmacology and therapeutics. https://doi.org/10.1002/cpt.3579
Vancouver
Emond Sarah K, Goettsch Wim G, Ollendorf Daniel A. Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability. Clinical pharmacology and therapeutics. 2025. doi:10.1002/cpt.3579. PMID: 39972486.
BibTeX
@article{emondsarah2025-39972486,
author = {Emond Sarah, K. and Goettsch Wim, G. and Ollendorf Daniel, A.},
title = {{Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability}},
journal = {Clinical pharmacology and therapeutics},
year = {2025},
doi = {10.1002/cpt.3579},
pmid = {39972486},
url = {https://doi.org/10.1002/cpt.3579}
}
Later studies that cite this one
1 study in the alzhub library lists this one in its references.
Related studies in Health systems and care delivery
Also in Health systems and care delivery
172 studies in the library are filed under Health systems and care delivery with no topic tag they share with another; these are this study's neighbours in a fixed tour of them. The Health systems and care delivery hub.