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

J 2022

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

DEL ÁLAMO, Marta, Christoph BÜHRER, Dirk FISHER, Matthias GRIESE, Paul LINGOR et. al.

Basic information

Original name

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

Authors

DEL ÁLAMO, Marta (724 Spain, guarantor), Christoph BÜHRER, Dirk FISHER, Matthias GRIESE, Paul LINGOR, Giovanni PALLADINI, Nicolas SIREAU, Virginie HIVERT, Luca SANGIORGI, Florence GUILLOT, Juliane HALFTERMEYER, Lenka SOUČKOVÁ (203 Czech Republic, belonging to the institution), Kristýna NOSKOVÁ (203 Czech Republic, belonging to the institution) and Regina DEMLOVÁ (203 Czech Republic, belonging to the institution)

Edition

TRIALS, ENGLAND, LONDON, 2022, 1745-6215

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30104 Pharmacology and pharmacy

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 2.500

RIV identification code

RIV/00216224:14110/22:00126687

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1186/s13063-022-06713-y

UT WoS

000854766200006

Keywords in English

Randomized clinical trials; Rare diseases; Drug repurposing; Academic-sponsored; Barriers; Challenges

Abstract

V originále

Background Academic-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. Methods Six 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. Results Administrative 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. Conclusion A 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.

Links

LM2018128, research and development project

Name: Český národní uzel Evropské sítě infrastruktur klinického výzkumu (Acronym: CZECRIN)

Investor: Ministry of Education, Youth and Sports of the CR

825575, interní kód MU

Name: European Joint Programme on Rare Diseases (Acronym: EJP RD)

Investor: European Union, Health, demographic change and wellbeing (Societal Challenges)

Citovat

DEL ÁLAMO, Marta, Christoph BÜHRER, Dirk FISHER, Matthias GRIESE, Paul LINGOR, Giovanni PALLADINI, Nicolas SIREAU, Virginie HIVERT, Luca SANGIORGI, Florence GUILLOT, Juliane HALFTERMEYER, Lenka SOUČKOVÁ, Kristýna NOSKOVÁ and Regina DEMLOVÁ. Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases. TRIALS. ENGLAND: LONDON, 2022, vol. 23, No 1, p. 1-10. ISSN 1745-6215. Available from: https://dx.doi.org/10.1186/s13063-022-06713-y.

@article{2218057,
   author = {del Álamo, Marta and Bührer, Christoph and Fisher, Dirk and Griese, Matthias and Lingor, Paul and Palladini, Giovanni and Sireau, Nicolas and Hivert, Virginie and Sangiorgi, Luca and Guillot, Florence and Halftermeyer, Juliane and Součková, Lenka and Nosková, Kristýna and Demlová, Regina},
   article_location = {ENGLAND},
   article_number = {1},
   doi = {http://dx.doi.org/10.1186/s13063-022-06713-y},
   keywords = {Randomized clinical trials; Rare diseases; Drug repurposing; Academic-sponsored; Barriers; Challenges},
   language = {eng},
   issn = {1745-6215},
   journal = {TRIALS},
   title = {Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases},
   url = {https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-022-06713-y},
   volume = {23},
   year = {2022}
}

TY  - JOUR
ID  - 2218057
AU  - del Álamo, Marta - Bührer, Christoph - Fisher, Dirk - Griese, Matthias - Lingor, Paul - Palladini, Giovanni - Sireau, Nicolas - Hivert, Virginie - Sangiorgi, Luca - Guillot, Florence - Halftermeyer, Juliane - Součková, Lenka - Nosková, Kristýna - Demlová, Regina
PY  - 2022
TI  - Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases
JF  - TRIALS
VL  - 23
IS  - 1
SP  - 1-10
EP  - 1-10
PB  - LONDON
SN  - 17456215
KW  - Randomized clinical trials
KW  - Rare diseases
KW  - Drug repurposing
KW  - Academic-sponsored
KW  - Barriers
KW  - Challenges
UR  - https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-022-06713-y
N2  - Background Academic-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. Methods Six 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. Results Administrative 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. Conclusion A 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.
ER  -

DEL ÁLAMO, Marta, Christoph BÜHRER, Dirk FISHER, Matthias GRIESE, Paul LINGOR, Giovanni PALLADINI, Nicolas SIREAU, Virginie HIVERT, Luca SANGIORGI, Florence GUILLOT, Juliane HALFTERMEYER, Lenka SOUČKOVÁ, Kristýna NOSKOVÁ and Regina DEMLOVÁ. Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases. \textit{TRIALS}. ENGLAND: LONDON, 2022, vol.~23, No~1, p.~1-10. ISSN~1745-6215. Available from: https://dx.doi.org/10.1186/s13063-022-06713-y.

Detailed Information on Publication Record