Pubblicazioni

Autori:

Ingrasciotta, Ylenia; Spini, Andrea; L'Abbate, Luca; Fiore, Elena Sofia; Carollo, Massimo; Ientile, Valentina; Isgrò, Valentina; Cavazzana, Anna; Biasi, Valeria; Rossi, Paola; Ejlli, Lucian; Belleudi, Valeria; Poggi, Francesca; Sapigni, Ester; Puccini, Aurora; Ancona, Domenica; Stella, Paolo; Pollina Addario, Sebastiano; Allotta, Alessandra; Leoni, Olivia; Zanforlini, Martina; Tuccori, Marco; Gini, Rosa; Trifirò, Gianluca

Titolo:

Comparing clinical trial population representativeness to real-world users of 17 biologics approved for immune-mediated inflammatory diseases: An external validity analysis of 66,639 biologic users from the Italian VALORE project

Anno:

2024

Tipologia prodotto:

Articolo in Rivista

Tipologia ANVUR:

Articolo su rivista

Lingua:

Inglese

Formato:

Elettronico

Referee:

No

Nome rivista:

Pharmacological Research

ISSN Rivista:

1043-6618

N° Volume:

200

Intervallo pagine:

1-9

Parole chiave:

Biological drugs; Biologics; External validity; Generalizability; Immune-mediated inflammatory disease; Pivotal randomized clinical trial; Real-world setting; Representativeness

Breve descrizione dei contenuti:

: To date, no population-based studies have specifically explored the external validity of pivotal randomized clinical trials (RCTs) of biologics simultaneously for a broad spectrum of immuno-mediated inflammatory diseases (IMIDs). The aims of this study were, firstly, to compare the patients' characteristics and median treatment duration of biologics approved for IMIDs between RCTs' and real-world setting (RW); secondly, to assess the extent of biologic users treated for IMIDs in the real-world setting that would not have been eligible for inclusion into pivotal RCT for each indication of use. Using the Italian VALORE distributed database (66,639 incident biologic users), adult patients with IMIDs treated with biologics in the Italian real-world setting were substantially older (mean age ± SD: 50 ± 15 years) compared to those enrolled in pivotal RCTs (45 ± 15 years). In the real-world setting, certolizumab pegol was more commonly used by adult women with psoriasis/ankylosing spondylitis (F/M ratio: 1.8-1.9) compared to RCTs (F/M ratio: 0.5-0.6). The median treatment duration (weeks) of incident biologic users in RW was significantly higher than the duration of pivotal RCTs in almost all indications for use and most biologics (4-100 vs. 6-167). Furthermore, almost half (46.4%) of biologic users from RW settings would have been ineligible for inclusion in the respective indication-specific pivotal RCTs. The main reasons were: advanced age, recent history of cancer and presence of other concomitant IMIDs. These findings suggest that post-marketing surveillance of biologics should be prioritized for those patients.

Id prodotto:

137373

Handle IRIS:

11562/1117854

ultima modifica:

12 dicembre 2024

Citazione bibliografica:

Ingrasciotta, Ylenia; Spini, Andrea; L'Abbate, Luca; Fiore, Elena Sofia; Carollo, Massimo; Ientile, Valentina; Isgrò, Valentina; Cavazzana, Anna; Biasi, Valeria; Rossi, Paola; Ejlli, Lucian; Belleudi, Valeria; Poggi, Francesca; Sapigni, Ester; Puccini, Aurora; Ancona, Domenica; Stella, Paolo; Pollina Addario, Sebastiano; Allotta, Alessandra; Leoni, Olivia; Zanforlini, Martina; Tuccori, Marco; Gini, Rosa; Trifirò, Gianluca, Comparing clinical trial population representativeness to real-world users of 17 biologics approved for immune-mediated inflammatory diseases: An external validity analysis of 66,639 biologic users from the Italian VALORE project «Pharmacological Research» , vol. 200 , 2024 , pp. 1-9

Consulta la scheda completa presente nel repository istituzionale della Ricerca di Ateneo