Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©)
Karen Kaiser,1 Susan E Yount,1 Christa E Martens,1 Kimberly A Webster,1 Sara Shaunfield,1 Amy Sparling,1 John Devin Peipert,1 David Cella,1 Scott T Rottinghaus,2 Bonnie MK Donato,2 Richard Wells,3 Ioannis Tomazos2 1Department of Medical Social Sciences, Northwestern University Feinberg School of Med...
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Dove Medical Press
2020-04-01
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| Series: | Patient Preference and Adherence |
| Subjects: | |
| Online Access: | https://www.dovepress.com/assessing-preferences-for-rare-disease-treatment-qualitative-developme-peer-reviewed-article-PPA |
| _version_ | 1811222791394951168 |
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| author | Kaiser K Yount SE Martens CE Webster KA Shaunfield S Sparling A Peipert JD Cella D Rottinghaus ST Donato BMK Wells R Tomazos I |
| author_facet | Kaiser K Yount SE Martens CE Webster KA Shaunfield S Sparling A Peipert JD Cella D Rottinghaus ST Donato BMK Wells R Tomazos I |
| author_sort | Kaiser K |
| collection | DOAJ |
| description | Karen Kaiser,1 Susan E Yount,1 Christa E Martens,1 Kimberly A Webster,1 Sara Shaunfield,1 Amy Sparling,1 John Devin Peipert,1 David Cella,1 Scott T Rottinghaus,2 Bonnie MK Donato,2 Richard Wells,3 Ioannis Tomazos2 1Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 2Alexion Pharmaceuticals Inc, Boston, MA, USA; 3Sunnybrook Health Sciences Centre, Toronto, ON, CanadaCorrespondence: Karen KaiserDepartment of Medical Social Sciences, Northwestern University Feinberg School of Medicine, 625 N. Michigan Ave. Suite 2700, Chicago, IL 60611, USATel +1 312-503-3521Fax +1 312-503-4800Email k-kaiser@northwestern.eduPurpose: 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.Keywords: paroxysmal nocturnal hemoglobinuria, questionnaire development, ravulizumab, eculizumab, treatment experience |
| first_indexed | 2024-04-12T08:21:27Z |
| format | Article |
| id | doaj.art-e1504a16e4924b06822c30f1c2bf100b |
| institution | Directory Open Access Journal |
| issn | 1177-889X |
| language | English |
| last_indexed | 2024-04-12T08:21:27Z |
| publishDate | 2020-04-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Patient Preference and Adherence |
| spelling | doaj.art-e1504a16e4924b06822c30f1c2bf100b2022-12-22T03:40:34ZengDove Medical PressPatient Preference and Adherence1177-889X2020-04-01Volume 1470571552948Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©)Kaiser KYount SEMartens CEWebster KAShaunfield SSparling APeipert JDCella DRottinghaus STDonato BMKWells RTomazos IKaren Kaiser,1 Susan E Yount,1 Christa E Martens,1 Kimberly A Webster,1 Sara Shaunfield,1 Amy Sparling,1 John Devin Peipert,1 David Cella,1 Scott T Rottinghaus,2 Bonnie MK Donato,2 Richard Wells,3 Ioannis Tomazos2 1Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 2Alexion Pharmaceuticals Inc, Boston, MA, USA; 3Sunnybrook Health Sciences Centre, Toronto, ON, CanadaCorrespondence: Karen KaiserDepartment of Medical Social Sciences, Northwestern University Feinberg School of Medicine, 625 N. Michigan Ave. Suite 2700, Chicago, IL 60611, USATel +1 312-503-3521Fax +1 312-503-4800Email k-kaiser@northwestern.eduPurpose: 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.Keywords: paroxysmal nocturnal hemoglobinuria, questionnaire development, ravulizumab, eculizumab, treatment experiencehttps://www.dovepress.com/assessing-preferences-for-rare-disease-treatment-qualitative-developme-peer-reviewed-article-PPAparoxysmal nocturnal hemoglobinuriaquestionnaire developmentravulizumabeculizumabtreatment experience |
| spellingShingle | 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 Preference and Adherence paroxysmal nocturnal hemoglobinuria questionnaire development ravulizumab eculizumab treatment experience |
| title | Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©) |
| title_full | Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©) |
| title_fullStr | Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©) |
| title_full_unstemmed | Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©) |
| title_short | Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©) |
| title_sort | assessing preferences for rare disease treatment qualitative development of the paroxysmal nocturnal hemoglobinuria patient preference questionnaire pnh ppq copy |
| topic | paroxysmal nocturnal hemoglobinuria questionnaire development ravulizumab eculizumab treatment experience |
| url | https://www.dovepress.com/assessing-preferences-for-rare-disease-treatment-qualitative-developme-peer-reviewed-article-PPA |
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