Patient-reported outcomes and realistic clinical endpoints for JAK inhibitors in rheumatoid arthritis

ABSTRACT

Introduction

Despite many effective therapeutics, health-related quality of life in RA remains low. Patients describe impacts of their disease in differing terms than health-care providers, stressing importance of pain, fatigue, poor sleep, and restrictions in work and social participation.

Areas covered

Patient-reported outcomes (PROs) across the phase 3 randomized controlled trials (RCTs) in RA with the JAK inhibitors (JAKis) are summarized. Patient populations, whether conventional synthetic disease modifying anti-rheumatic drug incomplete responders (csDMARD-IR) or biologic DMARD incomplete responders (bDMARD-IR) or csDMARD-naive, report differing baseline scores and placebo responses, generally lower in more treatment experienced patients. Improvements with all the approved JAKis in RA occur rapidly and are often maximal by 12–14 weeks, continuing thereafter. The rapidity of benefit reported by patients and convenience of oral administration often lead to increased adherence.

Expert Opinion

A broad variety of PROs utilized in the RA RCTs with the JAKis confirm the clinical meaningfulness of their efficacy across treatment-experienced and naive populations. A majority of patients report statistically significant as well as clinically meaningful (≥ minimum clinically important differences, MCID) improvements, with numbers needed to treat (NNTs) ≤ 10 and scores ≥ normative values at endpoint, despite ≤ 12% reporting such scores at baseline.

KEYWORDS:

• JAK inhibitors
• patient-reported outcomes
• health-related quality of life
• disease impact
• rheumatoid arthritis

Declaration of interest

V Strand serves or has served as a consultant for the following sponsors with approved JAKis including Abbvie, Galapagos, Gilead, Incyte, Eli Lilly, and Pfizer, but they have had no input/influence into the preparation of this manuscript. The work presented represents V Strand’s analyses [as well as others], published previously and analyzed here in a unique way. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.