https://orcid.org/0000-0002-6550-5240
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© was consistent with Food and Drug Administration guidelines for patient-reported outcome measure development, and included 1) a targeted literature review; 2) PNH expert clinician input on treatment preferences; 3) review of existing qualitative data on the PNH treatment and disease experience; 4) concept elicitation interviews with 8 PNH patients who received eculizumab and/or ravulizumab; 5) translatability review; and 6) cognitive debriefing with 5 patients. Interview participants were recruited through a United Kingdom PNH patient advocacy group and a Canadian clinical site involved in clinical trial ALXN1210-PNH-302.
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.
Acknowledgments
Copyright for the PNH-PPQ questionnaire is held by Alexion Pharmaceuticals, Inc. A copy of the questionnaire can be obtained from the authors upon request. This study was supported by Alexion Pharmaceuticals, Inc (Boston, MA, USA). The involvement of Alexion Pharmaceuticals, Inc. was limited to funding of the study and reviewing the manuscript for scientific accuracy. Alexion Pharmaceuticals, Inc. had no role in the study design; in collection, analysis, and interpretation of the data; in the writing of the report; or in the decision to submit this article for publication. Editorial and submission support were provided by ApotheCom (Yardley, PA, USA).
Disclosure
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. Donato was an employee and stockholder of Alexion Pharmaceuticals, Inc. at the time of this analysis and her current affiliation is at Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, CT, USA. Richard Wells has received honoraria and consulting fees from Alexion Pharmaceuticals, Inc. Ioannis Tomazos is an employee of, and owns stock in, Alexion Pharmaceuticals, Inc. The authors report no other conflicts of interest in this work.