Background/Purpose:  Given the increasing number of available treatments for RA with varying efficacy and safety profiles, it is critical to understand the level of trade-offs that patients are willing to make between benefits and risks. This study quantified the thresholds of benefit-risk trade-offs that patients are willing to accept in the treatment of RA.

Methods: Adult patients with moderate to severe RA were invited to participate in a discrete choice experiment that solicited their preferences for hypothetical RA treatments. These hypothetical RA treatments consisted of 9 attributes, including 3 efficacy measures (reduction in number of swollen joints, reduction of RA-related pain, and improvement of physical function), 3 adverse events (AEs; abnormal laboratory results [including abnormal liver function tests, blood count, and lipid profile], cancer, and serious infection), and 3 process-related features (route of administration, dose frequency, and out of pocket cost). Each participant completed 14 choice cards, and on each card was asked about their preference between two hypothetical RA treatments with varying levels of the 9 attributes. A multivariable logistic regression model was estimated to assess the association between the attributes and patient preference. Using the model, benefit-risk thresholds were calculated for the efficacy measures and AEs.

Results:  510 eligible patients with moderate to severe RA completed the experiment. The average age of the participants was 56.4 years, 64.7% were female, 38.4% were employed, 43.1% had RA for more than 10 years, and 45.1% received biologic agents. To achieve a 50% improvement in physical function, patients were willing to accept risk-increases of 91.1%, 4.7%, and 18.4% for abnormal laboratory results, cancer, and serious infection, respectively. Similarly, to achieve a 50% reduction in RA-related pain, patients were willing to accept risk increases of 70.6%, 3.7%, and 14.2% for each AE. Moreover, patients were willing to trade risk-increases of 42.0%, 2.2%, and 8.5% for each AE to obtain a 50% reduction in the number of swollen joints. Physical function affects patients’ preference for the treatment the most (odds ratio [OR]=4.03 for a 50% improvement), followed by RA-related pain (OR=2.95 for a 50% reduction) and number of swollen joints (OR=1.90 for a 50% reduction). Increased risk of cancer affects patients’ avoidance of the treatment the most (OR=0.74 for a 1% increase), followed by serious infections (OR=0.93 for a 1% increase) and abnormal laboratory results (OR=0.98 for a 1% increase). In addition, patients preferred oral and subcutaneous treatments (OR=2.30 for oral vs. intravenous and OR=1.69 for subcutaneous vs. intravenous), treatments with less frequent dosing (OR=1.69 for monthly vs. daily dosing), and lower out of pocket costs (OR=0.48 per $100 increase).

Conclusion:  Patients with moderate-to-severe RA are willing to accept increased treatment risks to achieve improved physical function and disease control.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/thresholds-of-benefit-risk-trade-offs-from-the-patient-perspective-for-treatment-decisions-in-moderate-to-severe-rheumatoid-arthritis/