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

References

• Loschi M, Porcher R, Barraco F, et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. Am J Hematol. 2016;91(4):366–370. doi:10.1002/ajh.24278
   PubMed Web of Science ®Google Scholar
• Brodsky RA. Narrative review: paroxysmal nocturnal hemoglobinuria: the physiology of complement-related hemolytic anemia. Ann Intern Med. 2008;148(8):587–595. doi:10.7326/0003-4819-148-8-200804150-00003
   PubMed Web of Science ®Google Scholar
• Parker CJ. Historical aspects of paroxysmal nocturnal haemoglobinuria: ‘defining the disease’. Br J Haematol. 2002;117(1):3–22.
   PubMed Web of Science ®Google Scholar
• Socie G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet. 1996;348(9027):573–577. doi:10.1016/S0140-6736(95)12360-1
   PubMed Web of Science ®Google Scholar
• Nishimura J, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore). 2004;83(3):193–207. doi:10.1097/01.md.0000126763.68170.46
   PubMed Web of Science ®Google Scholar
• Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333(19):1253–1258. doi:10.1056/NEJM199511093331904
   PubMed Web of Science ®Google Scholar
• Moyo VM, Mukhina GL, Garrett ES, Brodsky RA. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. Br J Haematol. 2004;126(1):133–138. doi:10.1111/bjh.2004.126.issue-1
   PubMed Web of Science ®Google Scholar
• Dmytrijuk A, Robie-Suh K, Cohen MH, Rieves D, Weiss K, Pazdur R. FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist. 2008;13(9):993–1000. doi:10.1634/theoncologist.2008-0086
   PubMed Web of Science ®Google Scholar
• Luzzatto L. Recent advances in the pathogenesis and treatment of paroxysmal nocturnal hemoglobinuria. F1000Res. 2016;5. doi:10.12688/f1000research.7288.1
   Google Scholar
• Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786–6792. doi:10.1182/blood-2011-02-333997
   PubMed Web of Science ®Google Scholar
• Thomas TC, Rollins SA, Rother RP, et al. Inhibition of complement activity by humanized anti-C5 antibody and single-chain Fv. Mol Immunol. 1996;33(17–18):1389–1401. doi:10.1016/S0161-5890(96)00078-8
   PubMed Web of Science ®Google Scholar
• Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233–1243. doi:10.1056/NEJMoa061648
   PubMed Web of Science ®Google Scholar
• Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804–2811. doi:10.1182/blood-2014-02-522128
   PubMed Web of Science ®Google Scholar
• FDA approves new treatment for adult patients with rare, life-threatening blood disease. Available from: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629022.htm?rel=0”?rel=0”. December 21, 2018. Accessed September 23, 2019.
   Google Scholar
• Ravulizumab [Prescribing Information]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2018.
   Google Scholar
• Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530–539. doi:10.1182/blood-2018-09-876136
   PubMed Web of Science ®Google Scholar
• Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540–549. doi:10.1182/blood-2018-09-876805
   PubMed Web of Science ®Google Scholar
• RAND Corporation. Delphi method. Available from: http://www.rand.org/topics/delphi-method.html. Accessed September 23, 2019.
   Google Scholar
• Cella D, Rosenbloom SK, Beaumont JL, et al. Development and validation of 11 symptom indexes to evaluate response to chemotherapy for advanced cancer. J Natl Compr Canc Netw. 2011;9(3):268–278. doi:10.6004/jnccn.2011.0026
   Web of Science ®Google Scholar
• Kaiser K, Beaumont JL, Webster K, et al. Development and validation of the functional assessment of cancer therapy–antiangiogenesis subscale. Cancer Med. 2015;4(5):690–698. doi:10.1002/cam4.385
   Web of Science ®Google Scholar
• Kaiser K, Cheng WY, Jensen S, et al. Development of a shared decision-making tool to assist patients and clinicians with decisions on oral anticoagulant treatment for atrial fibrillation. Curr Med Res Opin. 2015;31(12):2261–2272. doi:10.1185/03007995.2015.1096767
   PubMed Web of Science ®Google Scholar
• Magasi S, Mallick R, Kaiser K, et al. Importance and relevance of pulmonary symptoms among patients receiving second-and third-line treatment for advanced non–small-cell lung cancer: support for the content validity of the 4-item pulmonary symptom index. Clin Lung Cancer. 2013;14(3):245–253. doi:10.1016/j.cllc.2012.07.001
   Web of Science ®Google Scholar
• Glaser B, Strauss AL. The Discovery of Grounded Theory. Chicago: Aldine; 1967.
   Google Scholar
• Correia H. Translatability & cultural harmonization review. In: PROMIS^® Instrument Development and Validation Scientific Standards. Vol. 2; 2013.
   Google Scholar
• Willis GB. Cognitive Interviewing: A Tool for Improving Questionnaire Design. Thousand Oaks, CA: Sage; 2005.
   Google Scholar
• Atkinson MJ, Sinha A, Hass SL, et al. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004;2(1):12. doi:10.1186/1477-7525-2-12
   PubMedGoogle Scholar
• Flood EM, Beusterien KM, Green H, et al. Psychometric evaluation of the Osteoporosis Patient Treatment Satisfaction Questionnaire (OPSAT-Q), a novel measure to assess satisfaction with bisphosphonate treatment in postmenopausal women. Health Qual Life Outcomes. 2006;4:42. doi:10.1186/1477-7525-4-42
   PubMed Web of Science ®Google Scholar
• Gold DT, Horne R, Coon CD, et al. Development, reliability, and validity of a new preference and satisfaction questionnaire. Value Health. 2011;14(8):1109–1116. doi:10.1016/j.jval.2011.06.010
   Web of Science ®Google Scholar
• Horne R, Weinman J, Hankins M. The beliefs about medicines questionnaire: the development and evaluation of a new method for assessing the cognitive representation of medication. Psychol Health. 1999;14(1):1–24. doi:10.1080/08870449908407311
   Web of Science ®Google Scholar
• Iwamoto J, Okano H, Furuya T, et al. Patient preference for monthly bisphosphonate versus weekly bisphosphonate in a cluster-randomized, open-label, crossover trial: Minodroate Alendronate/Risedronate Trial in Osteoporosis (MARTO). J Bone Miner Metab. 2016;34(2):201–208. doi:10.1007/s00774-015-0653-7
   Web of Science ®Google Scholar
• Kimman ML, Rotteveel AH, Wijsenbeek M, et al. Development and pretesting of a questionnaire to assess patient experiences and satisfaction with medications (PESaM questionnaire). Patient. 2017;10(5):629–642. doi:10.1007/s40271-017-0234-z
   Google Scholar
• Meltzer EO, Hadley J, Blaiss M, et al. Development of questionnaires to measure patient preferences for intranasal corticosteroids in patients with allergic rhinitis. Otolaryngol Head Neck Surg. 2005;132(2):197–207. doi:10.1016/j.otohns.2004.10.010
   PubMed Web of Science ®Google Scholar
• Milic M, Foster A, Rihawi K, Anthoney A, Twelves C. ‘Tablet burden’ in patients with metastatic breast cancer. Eur J Cancer. 2016;55:1–6. doi:10.1016/j.ejca.2015.11.015
   Web of Science ®Google Scholar
• Peipert JD, Beaumont JL, Bode R, Cella D, Garcia SF, Hahn EA. Development and validation of the functional assessment of chronic illness therapy treatment satisfaction (FACIT TS) measures. Qual Life Res. 2014;23(3):815–824. doi:10.1007/s11136-013-0520-8
   PubMed Web of Science ®Google Scholar
• Ruiz MA, Pardo A, Rejas J, Soto J, Villasante F, Aranguren JL. Development and validation of the “Treatment Satisfaction with Medicines Questionnaire” (SATMED-Q)©. Value Health. 2008;11(5):913–926. doi:10.1111/j.1524-4733.2008.00323.x
   PubMed Web of Science ®Google Scholar
• Shikiar R, Rentz AM. Satisfaction with medication: an overview of conceptual, methodologic, and regulatory issues. Value Health. 2004;7(2):204–215. doi:10.1111/j.1524-4733.2004.72252.x
   PubMed Web of Science ®Google Scholar
• Ahmed F, Burt J, Roland M. Measuring patient experience: concepts and methods. Patient. 2014;7(3):235–241. doi:10.1007/s40271-014-0060-5
   PubMedGoogle Scholar
• Chewning B, Bylund CL, Shah B, Arora NK, Gueguen JA, Makoul G. Patient preferences for shared decisions: a systematic review. Patient Educ Couns. 2012;86(1):9–18. doi:10.1016/j.pec.2011.02.004
   PubMed Web of Science ®Google Scholar
• Chewning B, Sleath B. Medication decision-making and management: a client-centered model. Soc Sci Med. 1996;42(3):389–398. doi:10.1016/0277-9536(95)00156-5
   PubMed Web of Science ®Google Scholar
• Street RL, Elwyn G, Epstein RM. Patient preferences and healthcare outcomes: an ecological perspective. Expert Rev Pharmacoecon Outcomes Res. 2012;12(2):167–180. doi:10.1586/erp.12.3
   PubMed Web of Science ®Google Scholar
• Institute of Medicine. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington, DC: National Academy Press; 2001.
   Google Scholar
• Lindhiem O, Bennett CB, Trentacosta CJ, McLear C. Client preferences affect treatment satisfaction, completion, and clinical outcome: a meta-analysis. Clin Psychol Rev. 2014;34(6):506–517. doi:10.1016/j.cpr.2014.06.002
   PubMed Web of Science ®Google Scholar
• Sharma R, Keyzner A, Liu J, Bradley T, Allen SL. Successful pregnancy outcome in paroxysmal nocturnal hemoglobinuria (PNH) following escalated eculizumab dosing to control breakthrough hemolysis. Leuk Res Rep. 2015;4(1):36–38. doi:10.1016/j.lrr.2015.05.001
   PubMedGoogle Scholar
• Strasser S, Aharony L, Greenberger D. The patient satisfaction process: moving toward a comprehensive model. Med Care Rev. 1993;50(2):219–248. doi:10.1177/107755879305000205
   PubMedGoogle Scholar
• Chern B, Joseph D, Joshua D, et al. Bisphosphonate infusions: patient preference, safety and clinic use. Support Care Cancer. 2004;12(6):463–466. doi:10.1007/s00520-004-0628-z
   Web of Science ®Google Scholar
• Hadji P, Ziller V, Gamerdinger D, et al. Quality of life and health status with zoledronic acid and generic alendronate–a secondary analysis of the Rapid Onset and Sustained Efficacy (ROSE) study in postmenopausal women with low bone mass. Osteoporos Int. 2012;23(7):2043–2051. doi:10.1007/s00198-011-1834-4
   Web of Science ®Google Scholar
• Ijiri S. Further characterization between oral bisphosphonate dosing interval and patient preference–patient preference for daily, weekly and monthly bisphosphonate, based on results of 3052 outpatients questionnaires. Nihon Rinsho. 2013;71(12):2223–2231.
   Google Scholar
• Reginster JY, Rabenda V, Neuprez A. Adherence, patient preference and dosing frequency: understanding the relationship. Bone. 2006;38(4 Suppl 1):S2–S6. doi:10.1016/j.bone.2006.01.150
   PubMed Web of Science ®Google Scholar
• Ryzner KL, Burkiewicz JS, Griffin BL, Komperda KE. Survey of bisphosphonate regimen preferences in an urban community health center. Consult Pharm. 2010;25(10):671–675. doi:10.4140/TCP.n.2010.671
   Google Scholar
• Willeke P, Becker H, Wassenberg S, Pavenstadt H, Jacobi AM. Patient/rheumatologist evaluation of infusion treatment for rheumatoid arthritis. Z Rheumatol. 2011;70(3):232–234, 236–238. doi:10.1007/s00393-011-0752-3
   Web of Science ®Google Scholar
• Richter A, Anton SF, Koch P, Dennett SL. The impact of reducing dose frequency on health outcomes. Clin Ther. 2003;25(8):2307–2335; discussion 2306. doi:10.1016/S0149-2918(03)80222-9
   Web of Science ®Google Scholar
• Updated Study Analyzes Use and Cost of Orphan Drugs [press release]. Available from: https://rarediseases.org/updated-study-analyzes-use-and-cost-of-orphan-drugs/. October 18, 2018. Accessed September 23, 2019.
   Google Scholar
• US Department of Health and Human Services Food and Drug Administration. Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. Available from: https://www.fda.gov/media/77832/download. December 2009. Accessed September 23, 2019.
   Google Scholar
• Peipert JD, Kulesekararaj A, Fernandez FAG, et al. Patient preferences for the treatment of paroxysmal nocturnal hemoglobinuria: interim results of a patient survey of ravulizumab (ALXN1210) and eculizumab. Abstract presented at Academy of Managed Care & Specialty Pharmacy Annual Meeting; San Diego, CA. J Manag Care Specialty Pharm. 2019;25(3–a):S37.
   Google Scholar