Benefit-Risk Trade-offs and Patient Preferences for Therapy Selection in Ulcerative Colitis: a Multicountry Preference Study

Autores da FMUP

• Fernando José Magro Dias

  Autor

Participantes de fora da FMUP

• Gisbert, Javier P.
• Schreiber, Stefan
• Siegel, Corey A.
• Jus, Anna
• Whichello, Chiara
• Michaels-Igbokwe, Christine
• Heidenreich, Sebastian
• Oortwijn, Alessandra
• Vermeire, Severine

Unidades de investigação

• Biomedicina

• Laboratório Associado RISE- Rede de Investigação em Saúde

  

  Investigador

  Fernando Carlos De Landér Schmitt

• RISE-Health

  

  Investigador

  Fernando Carlos De Landér Schmitt

Abstract

Background To help navigate the complex treatment landscape of ulcerative colitis (UC), we quantified the benefit-risk trade-offs that patients were willing to make when choosing treatment.Methods Patients completed an online discrete choice experiment. Eligible patients had a UC diagnosis for >= 6 months, were aged >= 18 years, and resided in France, Germany, Italy, Spain, or the UK. Patients chose between 2 hypothetical treatments set up to ensure trade-offs were made. Clinical trial data, literature review, and patient interviews identified treatment attributes. Relative attribute importance (RAI) scores and maximum acceptable risks were generated. A patient-centric benefit-risk assessment of 200 mg of filgotinib was conducted as an example to show how measured trade-offs can be used.Results Overall, 631 patients participated; patients had a mean age of 42.2 years and were predominantly male (75.3%). Achieving and maintaining clinical remission was the most important factor for patients (RAI 32.4%); to achieve this, patients were willing to accept slightly higher risks of blood clots, serious infections, and malignancies compared with lower risk treatment profiles. Patients also valued the convenience of oral treatments, avoiding steroids, and the ability to attend school/work. The patient-centric benefit-risk assessment suggested patients are significantly more likely to prefer Janus kinase 1 preferential inhibitor filgotinib over placebo.Conclusions Achieving clinical remission was the highest treatment priority for patients. To attain this, patients were willing to accept some slightly higher risk treatment profiles. Patient choices in the benefit-risk assessment suggested patients were significantly more likely to prefer filgotinib over placebo. Patients were willing to accept slightly higher risk treatment profiles over lower risk treatment profiles for an increased chance of achieving and maintaining remission. A patient-centric benefit-risk assessment suggested 200 mg of filgotinib had an acceptable benefit-risk profile.

Keywords

• benefit-risk assessment; filgotinib; ulcerative colitis