Evidence mapPaperPMID 41174993Full record

SynthesisInquiry : a journal of medical care organization, provision and financing

Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.

N Ferizović, P Lorgelly, C S Clarke, R M Hunter, R Plackett, W Abbas, N Freemantle

Erratum issuedAbstract readSystematic Review
In one paragraph

Synthesis in Inquiry : a journal of medical care organization, provision and financing. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. 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

5 · Who and what money

Authors and funding

7 authors.

N FerizovićUniversity College London, UK.ORCID 0000-0002-9631-0470
P LorgellyUniversity of Auckland, New Zealand.
C S ClarkeUniversity College London, UK.ORCID 0000-0002-4676-1257
R M HunterUniversity College London, UK.
R PlackettUniversity College London, UK.
W AbbasUniversity College London, UK.
N FreemantleUniversity College London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Advanced therapy medicinal products (ATMPs) are ground-breaking genetic and cell/tissue-based therapies with the potential to treat and even cure myriad health conditions. However, they have faced multiple challenges in decision making with relatively low success in achieving reimbursement. The aim of this study was to review the published preference-based research on ATMPs to identify value attributes as a starting point in a research workstream aimed at understanding the true value of these innovative therapies. A literature review was used to identify studies across MedLine, Embase, Allied and Complementary Medicine Database, Global Health, ProQuest Central, Social Sciences Premium Collection, Applied Social Sciences Index and Abstracts, Econ Lit, Web of Science and SCOPUS from inception to 3rd March 2024. Search terms for genetic and cell/tissue-based therapies were conducted separately. Studies were included if they were a peer-reviewed primary research study or systematic literature review using stated preference measures to elicit preferences for ATMPs. Thirteen studies were included with 8 pertaining to genetic therapies and 5 to cell/tissue therapies. Attributes of value were similar across the studies and ATMP categories. Attributes were grouped into the following for genetic therapies: clinical benefits, uncertainty, risk, treatment burden, and quality of life; for cell/tissue therapies clinical benefit, uncertainty, risk and treatment burden. There is limited research on the preferences for ATMPs; but these findings suggest that the value attributes of ATMPs are consistent across gene and cell/tissue therapies with clinical benefit, uncertainty, risk, treatment burden, and quality of life being important.

Indexed as

Cell- and Tissue-Based TherapyGenetic TherapyPatient PreferenceDecision MakingHumansadvanced therapy medicinal productattributespreferencesreimbursementvalue

Identifiers

PMID41174993
PMCPMC12580532

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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 Socratic graph.