Evidence map›Paper›PMID 36109818›Full record

ArticleTrials2022

Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases.

Marta Del Álamo, Christoph Bührer, Dirk Fisher, Matthias Griese, Paul Lingor, Giovanni Palladini, Nicolas Sireau, Virginie Hivert, Luca Sangiorgi, Florence Guillot and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Trials, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 1 pooled it
–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

16 citing papers in PubMed, 1 synthesis or guideline pooled it.

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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

14 authors.

Marta Del ÁlamoECRIN, European Clinical Research Network Infrastructure, Paris, France. marta.delalamo@ecrin.org.ORCID http://orcid.org/0000-0002-8254-9990
Christoph BührerDepartment of Neonatology, Charité Universitätsmedizin, Berlin, Germany.
Dirk FisherDivision of Developmental- and Neuropaediatrics, University Children's. Hospital Basel (UKBB), University of Basel, Spitalstrasse 33, Postfach, 4031, Basel, Switzerland.
Matthias GrieseDepartment of Pediatrics, Dr. von Hauner Children's Hospital, University. Hospital, LMU Munich, German Center for Lung Research (DZL), Lindwurmstraße 4, 80337, Munich, Germany.
Paul LingorDepartment of Neurology, Klinikum rechts der Isar, Technical University of Munich, Munich, Germany.
Giovanni PalladiniAmyloidosis Research and Treatment Center, Foundation "Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo," and Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Nicolas SireauAKU Society, Cambridge, UK.
Virginie HivertEURORDIS- Rare Diseases Europe, Paris, France.
Luca SangiorgiDepartment of Medical Genetics and Skeletal Rare Diseases, IRCCS Rizzoli Orthopaedic Institute, Bologna, Italy.
Florence GuillotANR, French National Research Agency, Paris, France.
Juliane HalftermeyerInserm Transfert, Paris, France.
Lenka SouckováECRIN, European Clinical Research Network Infrastructure, Paris, France.
Kristýna NoskováECRIN, European Clinical Research Network Infrastructure, Paris, France.
Regina DemlováDepartment of Pharmacology, Faculty of Medicine, Masaryk University, Brno, Czech Republic.

Funding

Horizon 2020 Framework Programme EJP RD COFUND-EJP N° 825575
6 · The paper itself

Abstract

backgroundAcademic-sponsored trials for rare diseases face many challenges; the present paper identifies hurdles in the set-up of six multinational clinical trials for drug repurposing, as use cases.

methodsSix academic-sponsored multinational trials aiming to generate knowledge on rare diseases drug repurposing were used as examples to identify problems in their set-up. Coordinating investigators leading these trials provided feedback on hurdles linked to study, country, and site set up, on the basis of pre-identified categories established through the analysis of previous peer-reviewed publications.

resultsAdministrative burden and lack of harmonization for trial-site agreements were deemed as a major hurdle. Other main identified obstacles included the following: (1) complexity and restriction on the use of public funding, especially in a multinational set up, (2) drug supply, including procurement tendering rules and country-specific requirements for drug stability, and (3) lack of harmonization on regulatory requirements to get trial approvals.

conclusionA better knowledge of the non-commercial clinical research landscape and its challenges and requirements is needed to make drugs-especially those with less commercial gain-accessible to rare diseases patients. Better information about existing resources like research infrastructures, clinical research programs, and counseling mechanisms is needed to support and guide clinicians through the many challenges associated to the set-up of academic-sponsored multinational trials.

Indexed as

Drug RepositioningRare DiseasesClinical Trials as TopicHumansOrganizationsAcademic-sponsoredBarriersChallengesDrug repurposingRandomized clinical trialsRare diseases

Identifiers

PMID36109818
PMCPMC9479412

What OpenQuestion holds

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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.