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41/100

✓ Peer-reviewed alzhub usefulness score 41 out of 100. Costs and economics

Topic: Drug trials: efficacy and safety

Plain-language summary

Under the initial assumption that the hypothetical gene therapy grants a 30% risk reduction in disease progression and entry into institutional care, the maximum cost-effective price for gene therapy is $141,126 per treatment using the threshold of $150,000 per quality-adjusted life year (QALY). By increasing the treatment effectiveness to 50%, cost-effective price nearly doubled at each willingness-to-pay threshold (e.g., $260,902 at the $150,000/QALY threshold). Despite being cost-effective at a very high price, the hypothetical gene therapy for AD would still need to be priced considerably lower than other approved gene therapies on the market. Thus, a comprehensive pharmacoeconomic assessment remains critical in pricing innovative therapy and determining coverage for patients in need.

Cite this study APA, Vancouver, BibTeX · download .bib or .ris

APA 7th

Kieu, C., & Look, K. A. (2023). Cost-Effectiveness of a Hypothetical Gene Therapy for Alzheimer's Disease: A Markov Simulation Analysis. Innovations in pharmacy. https://doi.org/10.24926/iip.v14i3.5500

Vancouver

Kieu C, Look KA. Cost-Effectiveness of a Hypothetical Gene Therapy for Alzheimer's Disease: A Markov Simulation Analysis. Innovations in pharmacy. 2023. doi:10.24926/iip.v14i3.5500. PMID: 38487381.

BibTeX

@article{kieu2023-38487381,
  author  = {Kieu, Chinh and Look, Kevin A},
  title   = {{Cost-Effectiveness of a Hypothetical Gene Therapy for Alzheimer's Disease: A Markov Simulation Analysis}},
  journal = {Innovations in pharmacy},
  year    = {2023},
  doi     = {10.24926/iip.v14i3.5500},
  pmid    = {38487381},
  url     = {https://doi.org/10.24926/iip.v14i3.5500}
}

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How to read this study

Design: our record does not state a study design, so check the methods section of the original before weighing the result.
Size: the record gives no participant count. Look for one in the original before trusting a pattern.
Effect: the record gives no effect size, so the summary above cannot say how large any difference was.

What the evidence grade means here

This page is graded low. alzhub.org assigns the grade automatically from keywords in the abstract, not from reading the paper. Low means the abstract matched neither pattern. It is the default label, and it does not mean the work is poor; many sound lab and imaging papers land here. It can add at most 5 of the 100 points in the study score, and it is a label about design, not a verdict on whether the findings are true.

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Key findings

Under the initial assumption that the hypothetical gene therapy grants a 30% risk reduction in disease progression and entry into institutional care, the maximum cost-effective price for gene therapy is $141,126 per treatment using the threshold of $150,000 per quality-adjusted life year (QALY).
By increasing the treatment effectiveness to 50%, cost-effective price nearly doubled at each willingness-to-pay threshold (e.g., $260,902 at the $150,000/QALY threshold).
Despite being cost-effective at a very high price, the hypothetical gene therapy for AD would still need to be priced considerably lower than other approved gene therapies on the market.
Thus, a comprehensive pharmacoeconomic assessment remains critical in pricing innovative therapy and determining coverage for patients in need.

New to this subject? Start with our plain-English guide: Costs & economics →

Study details

Evidence strengthLow

How this study scored 41/100

Published in a peer-reviewed journal. Scored zero for study design, sample size, effect reported, studied in people and randomized / blinded because the record does not state it. We have no data on journal's citation record, times cited, citations per year, impact for its field and year, cited by studies here, still being cited, senior author's track record, institution's track record, multicenter collaboration and free to read, so those were left out: it earned 23 of the 56 points we could judge it on, which is 41 out of 100.

Component	Points	Why
Journal tier	7 / 10	Tier 3 — peer-reviewed journal (Innovations in pharmacy)
Journal's citation record	— / 4	No journal record from OpenAlex — left out of this score
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	3 / 5	2023 — within 6 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)
Randomized / blinded	0 / 2	No randomization or blinding stated — scores zero (not stated in the record — scored 0)
Times cited	— / 7	No citation count (not found in OpenAlex) — left out of this score
Citations per year	— / 5	No citation count or no year — left out of this score
Impact for its field and year	— / 6	No field-weighted citation impact (OpenAlex has none yet) — left out of this score
Cited by studies here	— / 6	Not yet checked against this library's citation graph — left out of this score
Still being cited	— / 4	No yearly citation counts — left out of this score
Senior author's track record	— / 4	Senior author's record not available — left out of this score
Institution's track record	— / 3	No institution listed in OpenAlex — left out of this score
Multicenter collaboration	— / 3	Institutions not listed — left out of this score
Indexed in PubMed	2 / 2	Indexed in PubMed (PMID 38487381)
Free to read	— / 2	Access not recorded — left out of this score
Points	23 / 56	Scored on 10 of 20 parts. We have no data for the rest, so they are left out: 23 of the 56 points we could judge it on is 41 out of 100.
Total	41 / 100	—

What each of these 20 components measures, and what this score is not →

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