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.

Základní údaje

Originální název

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

Autoři

DEL ÁLAMO, Marta (724 Španělsko, garant), Christoph BÜHRER, Dirk FISHER, Matthias GRIESE, Paul LINGOR, Giovanni PALLADINI, Nicolas SIREAU, Virginie HIVERT, Luca SANGIORGI, Florence GUILLOT, Juliane HALFTERMEYER, Lenka SOUČKOVÁ (203 Česká republika, domácí), Kristýna NOSKOVÁ (203 Česká republika, domácí) a Regina DEMLOVÁ (203 Česká republika, domácí)

Vydání

TRIALS, ENGLAND, LONDON, 2022, 1745-6215

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30104 Pharmacology and pharmacy

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 2.500

Kód RIV

RIV/00216224:14110/22:00126687

Organizační jednotka

Lékařská fakulta

DOI

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

UT WoS

000854766200006

Klíčová slova anglicky

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

Štítky

14110516, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 30. 11. 2022 14:06, Mgr. Tereza Miškechová

Anotace

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.

Návaznosti

LM2018128, projekt VaV

Název: Český národní uzel Evropské sítě infrastruktur klinického výzkumu (Akronym: CZECRIN)

Investor: Ministerstvo školství, mládeže a tělovýchovy ČR, Český národní uzel Evropské sítě infrastruktur klinického výzkumu (CZECRIN)

825575, interní kód MU

Název: European Joint Programme on Rare Diseases (Akronym: EJP RD)

Investor: Evropská unie, European Joint Programme on Rare Diseases, 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 a 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, roč. 23, č. 1, s. 1-10. ISSN 1745-6215. Dostupné z: 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 a 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, roč.~23, č.~1, s.~1-10. ISSN~1745-6215. Dostupné z: https://dx.doi.org/10.1186/s13063-022-06713-y.

Podrobný výpis o publikaci