Evidence map›Paper›PMID 37868723›Full record

ArticleRegenerative therapy2023

Is it enough just to demonstrate that the advanced therapy medicinal products do work or we would prefer to keep walking on the Moon?

Dinko Mitrečić

Abstract read
In one paragraph

Article in Regenerative therapy, 2023. 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

1 author.

Dinko MitrečićLaboratory for Stem Cells, Croatian Institute for Brain Research and Department of Histology and Embryology, University of Zagreb School of Medicine, HR-10000 Zagreb, Croatia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

After several decades of continuous yet bumpy progress the advanced therapy medicinal products reached the stage when the first drugs with well documented efficacy started to be registered. However, in the disturbing chain of events, many of them were discontinued because of the lack of return on investment. By comparing this phenomenon to the fact that humans did not return to the Moon for already 50 years, primarily because of the lack of dedicated funds, this commentary proposes strategies how to avoid menace of the dead end threating to suffocate progress of the advanced medical therapies. While treatments for rare diseases can be defended by mixture of altruistic, inspiring and rational reasons, mostly covered by the fact that regardless of the price of the newly developed therapy, the total burden remains low, common diseases should be addressed in a different way. This needs to include precise modelling of the benefits which advanced therapy medicinal products bring for every condition, taking in account reduction of the costs of long, often life-long support of patients affected by such diseases. Without intention to steal romantic view on the scientific progress, powerful yet very expensive tools of advanced therapy medicinal products require urgent top-down decisions which include selection of priorities based on the financial modelling. Instead of spontaneous exploration in all directions, this commentary proposes an arranged marriage between scientific community and big investors sustained by combination of governmental requirements in the form of real time data sharing, reimbursement warranties according to demonstrated efficacy and clear recognition of the primary targets with accompanying pre-defined financial frameworks.

Indexed as

Advanced therapy medicinal productsCost of treatmentGene therapyHealth economicsStem cells

Identifiers

PMID37868723
PMCPMC10584667

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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 OpenQuestion graph.