Real-World Evidence to Contextualize Clinical Trial Results and Inform Regulatory Decisions: Tofacitinib Modified-Release Once-Daily vs Immediate-Release Twice-Daily for Rheumatoid Arthritis.
Claims analysis
Clinical effectiveness
Drug approval process
Registry
Rheumatoid arthritis
Rheumatology
Tofacitinib
Journal
Advances in therapy
ISSN: 1865-8652
Titre abrégé: Adv Ther
Pays: United States
ID NLM: 8611864
Informations de publication
Date de publication:
01 2021
01 2021
Historique:
received:
28
07
2020
accepted:
05
09
2020
pubmed:
10
10
2020
medline:
15
4
2021
entrez:
9
10
2020
Statut:
ppublish
Résumé
Tofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). To provide additional clinical evidence in regulatory submissions for a modified-release (MR) once-daily (QD) tofacitinib formulation, we compared real-world adherence and effectiveness between patients initiating the MR QD formulation and patients initiating an immediate-release (IR) twice-daily (BID) formulation. Two noninterventional cohort studies were conducted. First, adherence and two effectiveness proxies were compared between patients with RA who newly initiated tofacitinib MR 11 mg QD or IR 5 mg BID in the IBM In each study, approximately two-thirds of patients initiated the MR formulation. In the claims database study, improved adherence and at least comparable effectiveness were observed with tofacitinib MR vs IR over 12 months, particularly in patients without prior advanced therapy. In the registry study, the noninferiority of tofacitinib MR vs IR was demonstrated for both CDAI outcomes at ~6 months; this finding was robust across multiple sensitivity analyses. These results demonstrate the value of real-world evidence from complementary data sources in understanding the impact of medication adherence with a QD formulation in clinical practice. These analyses were suitable for regulatory consideration as an important component of evidence for the comparability of tofacitinib MR 11 mg QD vs IR 5 mg BID in patients with RA. Claims database study: ClinicalTrials.gov identifier NCT04018001, retrospectively registered July 12, 2019. Corrona US RA Registry study: ClinicalTrials.gov identifier NCT04267380, retrospectively registered February 12, 2020.
Identifiants
pubmed: 33034006
doi: 10.1007/s12325-020-01501-z
pii: 10.1007/s12325-020-01501-z
pmc: PMC7854470
doi:
Substances chimiques
Antirheumatic Agents
0
Piperidines
0
Protein Kinase Inhibitors
0
Pyrimidines
0
Pyrroles
0
tofacitinib
87LA6FU830
Banques de données
ClinicalTrials.gov
['NCT04267380', 'NCT04018001']
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Pagination
226-248Références
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