Evidence map›Paper›PMID 40989744›Full record

ArticleJournal of market access & health policy2025

European Health Technology Assessment Considerations Related to Gene Therapies in Eyecare: The Neovascular Age-Related Macular Degeneration Example.

Kevin Douglas, Gianni Pardhanani, Laetitia Mariani, Maria Chaita

Abstract read
In one paragraph

Article in Journal of market access & health policy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Kevin DouglasAbbVie, Chicago, IL 60064, USA.
Gianni PardhananiAbbVie, 00144 Rome, Italy.
Laetitia MarianiAbbVie, 6330 Cham, Switzerland.
Maria ChaitaAbbVie, Marlow SL6 4UB, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapies that induce the body to produce therapeutic anti-vascular endothelial growth factor (anti-VEGF) proteins are an emerging topic related to neovascular age-related macular degeneration (nAMD). Continuous delivery of anti-VEGF protein directly to the target tissue offers the possibility of lifelong efficacy without the need for repeated and frequent eye injections. This novel approach could revolutionize patient management through optimizing clinical outcomes while simplifying service delivery. However, such gene therapies are anticipated to face unique challenges related to patients' access and health technology assessment (HTA), and their integration into real-world eyecare practices. This article presents key elements raised at the European Access Academy (EAA) Fall convention (held in Rome in October 2024) regarding anticipated HTA challenges for gene therapies in nAMD. The important role of HTA and policymakers in ensuring that emerging gene therapies are accessible to all eligible patients is also highlighted. This article mainly focuses on the need for a fit-for-purpose EU HTA framework to address the widely varying utilization of standard of care in nAMD clinical practice, and to incorporate considerations about the long-term durability of gene therapies in nAMD. The importance of integrating real-world evidence (RWE) into the EU HTA framework is also discussed.

Indexed as

anti-vascular endothelial growth factor (anti-VEGF)European Access Academy (EAA)European Health Technology assessment (EU HTA)eyecaregene therapiesindirect treatment comparisons (ITC)neovascular age-related macular degeneration (nAMD)real-world evidence (RWE)

Identifiers

PMID40989744
PMCPMC12452758

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.