Remodeling an existing rare disease registry to be used in regulatory context: Lessons learned and recommendations.

Mordenti, Marina; Boarini, Manila; D'Alessandro, Fabio; Pedrini, Elena; Locatelli, Manuela; Sangiorgi, Luca

Mordenti, Marina; Boarini, Manila; D'Alessandro, Fabio; Pedrini, Elena; Locatelli, Manuela; Sangiorgi, Luca.

Afiliação

Mordenti M; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Boarini M; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
D'Alessandro F; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Pedrini E; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Locatelli M; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Sangiorgi L; Department of Rare Skeletal Disorders, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.

Front Pharmacol ; 13: 966081, 2022.

Article em En | MEDLINE | ID: mdl-36210847

Palavras-chave

EMA initiative; disease registry; multiple osteochondromas; orphan drug; rare diseases; real-world evidence (RWE)

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials / Guideline / Prognostic_studies Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google