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Kaiser K, Yount SE, Martens CE, et al. Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ. Patient Prefer Adherence. 2020;14:705-715doi: 10.2147/PPA.S233830.
Kaiser, K., Yount, S. E., Martens, C. E., Webster, K. A., Shaunfield, S., Sparling, A., Peipert, J. D., Cella, D., Rottinghaus, S. T., Donato, B. M. K., Wells, R., & Tomazos, I. (2020). Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ. Patient preference and adherence, 14705-715. https://doi.org/10.2147/PPA.S233830
Kaiser, Karen, et al. "Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ." Patient preference and adherence vol. 14 (2020): 705-715. doi: https://doi.org/10.2147/PPA.S233830
Kaiser K, Yount SE, Martens CE, Webster KA, Shaunfield S, Sparling A, Peipert JD, Cella D, Rottinghaus ST, Donato BMK, Wells R, Tomazos I. Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ. Patient Prefer Adherence. 2020 Apr 05;14:705-715. doi: 10.2147/PPA.S233830. eCollection 2020. PMID: 32308375; PMCID: PMC7147619.
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Patient Prefer Adherence. 2020 Apr 05;14:705-715. doi: 10.2147/PPA.S233830. eCollection 2020.

Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ.

Patient preference and adherence

Karen Kaiser, Susan E Yount, Christa E Martens, Kimberly A Webster, Sara Shaunfield, Amy Sparling, John Devin Peipert, David Cella, Scott T Rottinghaus, Bonnie M K Donato, Richard Wells, Ioannis Tomazos

Affiliations

  1. Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
  2. Alexion Pharmaceuticals Inc, Boston, MA, USA.
  3. Sunnybrook Health Sciences Centre, Toronto, ON, Canada.

PMID: 32308375 PMCID: PMC7147619 DOI: 10.2147/PPA.S233830

Abstract

PURPOSE: To develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH).

PATIENTS AND METHODS: The development of the PNH-PPQ

RESULTS: Six themes were identified as most relevant to the PNH treatment experience from the concept elicitation interviews: disease symptoms (n=8/8); treatment frequency (n=7/8); quality of life impact of treatment/disease (n=7/8); treatment burden (n=7/8); treatment efficacy (n=5/8); and treatment side effects (n=5/8). An initial list of 88 preference questions was reduced to 11 highly relevant and non-redundant questions reflecting the 6 themes. Cognitive interview participants unanimously agreed that the PNH-PPQ instructions were clear; response options were understandable, easy to use, and provided enough choices; and the questions captured the factors that inform treatment preferences.

DISCUSSION: When new drugs have similar efficacy to existing medications, documenting patient preferences is important for confirming patient benefit from the new medication. Understanding what matters most to patients is essential for delivering patient-centered care and may play a particularly significant role in treatment decision making. The availability of such a tool may be especially important as new orphan drugs are developed and patients with rare diseases have more than one treatment option to consider.

CONCLUSION: 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.

© 2020 Kaiser et al.

Keywords: eculizumab; paroxysmal nocturnal hemoglobinuria; questionnaire development; ravulizumab; treatment experience

Conflict of interest statement

David Cella is a consultant to, and has received grant support from, Alexion Pharmaceuticals, Inc. Scott T. Rottinghaus is an employee of, and owns stock in, Alexion Pharmaceuticals Inc. Bonnie M. K.

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