Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©).
Karen KaiserSusan E YountChrista E MartensKimberly A WebsterSara ShaunfieldAmy SparlingJohn Devin PeipertDavid CellaScott T RottinghausBonnie M K DonatoRichard WellsIoannis TomazosPublished in: Patient preference and adherence (2020)
The PNH-PPQ provides a patient-centered approach for evaluating preferences for the treatment of PNH. The PNH-PPQ has subsequently assessed patient preference in the clinical trial sub-study ALXN1210-PNH-302s.