Johnson FR, Ozdemir S, Andrews E, Hass S, Miller DP, White J, Siegel C, Sands B. Patients' willingness to accept risk-benefit tradeoffs in treating crohn's disease. Poster presented at the 22nd ICPE International Conference on Pharmacoepidemiology & Therapeutic Risk Management; August 2006. Lisbon, Portugal.


BACKGROUND: All medical interventions carry risks that must be evaluated against clinical benefits.

 

OBJECTIVES: To evaluate the willingness of Crohn’s disease (CD) patients to accept treatment-related serious adverse event (SAE) risks in exchange for clinical improvements.

 

METHODS: An online panel of CD patients >18 years old in the US completed a carefully designed and tested questionnaire containing stated preference (SP) benefit-risk trade-off items. The SP method is used by health economists to quantify the relative importance of treatment processes and outcomes. The questionnaire asked patients to select between treatment alternatives with different levels of benefit and risk attributes. Attributes included severity of daily symptoms, rate of serious CD complications (fistulas, abscesses, bowel obstructions), time between flare-ups, oral steroid use, and varying levels of SAE risks (disability and death from Progressive Multifocal Leukoencephalopathy [PML], serious infection, or lymphoma). Information was collected on demographics, disease history, and functional status. The maximum acceptable 1-year risk (MAR) for SAE development was calculated for various levels of clinical benefit using logistic models.

 

RESULTS: Mean age of the 342 respondents was 45; 73% were female. Higher MAR, indicating greater risk acceptance, was observed for trade-off tasks involving higher levels of clinical benefit, among patients with lower current functional status, and among patients reporting a low level of worry about the potential adverse events. For PML, the mean (95% CI) MARs for an improvement from severe daily symptoms to remission and moderate daily symptoms to mild daily symptoms were 0.70% (0.60–0.80%) and 0.22% (0.14–0.30%), respectively. For serious infection and lymphoma, respectively, the mean (95% CI) MARs for an improvement from severe symptoms to remission were 0.73% (0.66–0.81%) and 0.82% (0.72–0.92%), and from moderate symptoms to mild symptoms were 0.37% (0.17–0.57%) and 0.42% (0.33–0.52%). The lowest observed MAR for any of the three studied SAEs is higher than observed rates of SAE occurrence with natalizumab and commonly used CD medications.

 

CONCLUSIONS: CD patients indicated they are willing to accept a defined risk of death or disability in exchange for defined levels of clinical effectiveness. This type of trade-off information can inform treatment and regulatory decisions.

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