Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative study

Spoors, John; Payne, Katherine; Wright, Stuart; Horsley, Will; Bell, Sadie; Cairns, John

doi:10.2478/jhp-2024-0006

References

Spoors J, McEntee J, Horsley W, Cairns J, Payne K, Wright S. PO178. Enhanced horizon scanning for the haemophilia pharmaceutical pipeline 2022-2026. Haemophilia 2023; 29(S1): 122. doi: 10.1111/hae.14715.
Open DOI Search in Google Scholar Back to article
European Medicines Agency. Advanced therapy medicinal products: Overview [Internet]. 2016. Available from: https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview (accessed July 2020).
Search in Google Scholar Back to article
Spoors J, Cairns J, Miners A. Discrete choice experiments: An overview of experience to date in haemophilia. J Haem Pract 2022; 9(1): 50-63. doi: 10.2478/jhp-2022-0006.
Open DOI Search in Google Scholar Back to article
Spoors J, Miners A, Cairns J, et al. Payer and implementation challenges with advanced therapy medicinal products (ATMPs). BioDrugs 2021; 35(1): 1-5. doi: 10.1007/s40259-020-00457-4.
Open DOI Search in Google Scholar Back to article
Fletcher S, Jenner K, Pembroke L, Holland M, Khair K. The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study. Orphanet J Rare Dis 2022;17(1): 155. doi: 10.1186/s13023-022-02256-2.
Open DOI Search in Google Scholar Back to article
Woollard L, Gorman R, Rosenfelt DJ. Addressing patient education priorities in the era of gene therapy for haemophilia: Towards evidence-informed shared decision-making. Haemophilia 2021; 27(2): e302-e304. doi: 10.1111/hae.14214.
Open DOI Search in Google Scholar Back to article
van Overbeeke E, Hauber B, Michelsen S, et al. Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey. Haemophilia 2021; 27(6): 957-966. doi: 10.1111/hae.14401.
Open DOI Search in Google Scholar Back to article
Limjoco J, Calatroni A, Aristizabal P, Thornburg CD. Gene therapy preferences and informed decision-making: Results from a National Hemophilia Foundation Community Voices in research survey. Haemophilia 2023; 29(1): 51-60. doi: 10.1111/hae.14706.
Open DOI Search in Google Scholar Back to article
Langstaff B. Infected Blood Inquiry [Internet]. 2021. Available from: https://www.infectedbloodinquiry.org.uk/ (accessed June 2023).
Search in Google Scholar Back to article
Terris-Prestholt F, Quaife M, Vickerman P. Parameterising user uptake in economic evaluations: The role of discrete choice experiments. Health Econ 2016; 25: 116-23. doi: 10.1002/hec.3297.
Open DOI Search in Google Scholar Back to article
Horne R, Weinman J. Patients’ beliefs about prescribed medicines and their role in adherence to treatment in chronic physical illness. J Psychosom Res 1999; 47(6):555-67. doi: 10.1016/s0022-3999(99)00057-4.
Open DOI Search in Google Scholar Back to article
Elwyn G, Frosch D, Thomson R, et al. Shared decision making: A model for clinical practice. J Gen Intern Med 2012; 27(10): 1361-1367. doi: 10.1007/s11606-012-2077-6.
Open DOI Search in Google Scholar Back to article
Karazivan P, Dumez V, Flora L, et al. The patient-as-partner approach in health care: A conceptual framework for a necessary transition. Acad Med 2015; 90(4): 437-41. doi: 10.1097/ACM.0000000000000603.
Open DOI Search in Google Scholar Back to article
Hermans C, Noone D, Benson G, et al. Hemophilia treatment in 2021: Choosing the “optimal” treatment using an integrative, patient-oriented approach to shared decision-making between patients and clinicians. Blood Rev 2022; 52: 100890. doi: 10.1016/j.blre.2021.100890.
Open DOI Search in Google Scholar Back to article
Quill TE, Brody H. Physician recommendations and patient autonomy: Finding a balance between physician power and patient choice. Ann Intern Med 1996; 125(9): 763-9. doi: 10.7326/0003-4819-125-9-199611010-00010.
Open DOI Search in Google Scholar Back to article
Fischhoff B, Brewer NT, Downs J. Communicating Risks and Benefits: An Evidence-Based User’s Guide. US Food and Drug Administration, 2011. Available from: https://www.fda.gov/media/81597/download.
Search in Google Scholar Back to article
Nutbeam D. The evolving concept of health literacy. Soc Sci Med 2008; 67(12): 2072-8. doi: 10.1016/j.socscimed.2008.09.050.
Open DOI Search in Google Scholar Back to article
DeWalt DA, Berkman ND, Sheridan S, Lohr KN, Pignone MP. Literacy and health outcomes: A systematic review of the literature. J Gen Intern Med 2004; 19(12): 1228-39. doi: 10.1111/j.1525-1497.2004.40153.x.
Open DOI Search in Google Scholar Back to article
Visscher BB, Steunenberg B, Heijmans M, et al. Evidence on the effectiveness of health literacy interventions in the EU: A systematic review. BMC Public Health 2018;18(1): 1414. doi: 10.1186/s12889-018-6331-7.
Open DOI Search in Google Scholar Back to article
Slovic P, Peters E, Finucane ML, MacGregor DG. Affect, risk, and decision making. Health Psychol 2005; 24(4S): S35-40. doi: 10.1037/0278-6133.24.4.S35.
Open DOI Search in Google Scholar Back to article
Lerner JS, Keltner D. Fear, anger, and risk. J Pers Soc Psychol 2001; 81(1): 146-59. doi: 10.1037//0022-3514.81.1.146.
Open DOI Search in Google Scholar Back to article
Wardman JK. Toward a critical discourse on affect and risk perception. J Risk Res 2006; 9(2): 109-124. doi: 10.1080/13669870500454773.
Open DOI Search in Google Scholar Back to article
Frosch DL, Kaplan RM. Shared decision making in clinical medicine: Past research and future directions. Am J Prev Med 1999; 17(4): 285-94. doi: 10.1016/s0749-3797(99)00097-5.
Open DOI Search in Google Scholar Back to article
Street RL, Gordon HS, Ward MM, Krupat E, Kravitz RL. Patient participation in medical consultations: Why some patients are more involved than others. Med Care 2005; 43(10): 960-9. doi: 10.1097/01.mlr.0000178172.40344.70.
Open DOI Search in Google Scholar Back to article
Sleath B, Tulsky JA, Peck BM, Thorpe J. Provider-patient communication about antidepressants among veterans with mental health conditions. Am J Geriatr Pharmacother 2007; 5(1): 9-17. doi: 10.1016/j.amjopharm.2007.03.002.
Open DOI Search in Google Scholar Back to article
Fried TR, Tinetti ME, Towle V, O’Leary JR, Iannone L. Effects of benefits and harms on older persons’ willingness to take medication for primary cardiovascular prevention. Arch Intern Med 2011;171(10): 923-8. doi: 10.1001/archinternmed.2011.32.
Open DOI Search in Google Scholar Back to article
Lumivero. NVIVO (Version 12) [Internet]. 2018. Available from: https://lumivero.com/products/nvivo/ (accessed July 2023).
Search in Google Scholar Back to article
Braun V, Clarke V. Using thematic analysis in psychology. Qual Res Psychol 2006; 3(2): 77-101. doi: 10.1191/1478088706qp063oa.
Open DOI Search in Google Scholar Back to article
Moxey A, O’Connell D, McGettigan P, Henry D. Describing treatment effects to patients. J Gen Intern Med 2003; 18(11): 948-59. doi: 10.1046/j.1525-1497.2003.20928.x.
Open DOI Search in Google Scholar Back to article
Gigerenzer G. Why does framing influence judgment? J Gen Intern Med 2003; 18(11): 860-961. doi: 10.1046/j.1525-1497.2003.30901.x.
Open DOI Search in Google Scholar Back to article
Edwards A, Elwyn G, Covey J, Matthews E, Pill R. Presenting risk information—a review of the effects of “framing” and other manipulations on patient outcomes. J Health Commun 2001; 6(1): 61-82. doi: 10.1080/10810730150501413.
Open DOI Search in Google Scholar Back to article
Feldman-Stewart D, Brennenstuhl S, McIssac K, et al. A systematic review of information in decision aids. Health Expect 2007; 10(1): 46-61. doi: 10.1111/j.1369-7625.2006.00420.x.
Open DOI Search in Google Scholar Back to article
Ankolekar A, Dekker A, Fijten R, Berlanga A. The benefits and challenges of using patient decision aids to support shared decision making in health care. JCO Clin Cancer Inform 2018; (2): 1-10. doi: 10.1200/CCI.18.00013.
Open DOI Search in Google Scholar Back to article
Trenaman L, Bryan S, Bansback N. The cost-effectiveness of patient decision aids: A systematic review. Healthc (Amst) 2014; 2(4): 251-7. doi: 10.1016/j.hjdsi.2014.09.002.
Open DOI Search in Google Scholar Back to article
Fletcher S, Jenner K, Khair K. Shared decision-making for gene therapy in haemophilia care. J Haem Pract 2023; 10(1): 62-7. doi: 10.2478/jhp-2023-0009.
Open DOI Search in Google Scholar Back to article
Hermans C, Gruel Y, Frenzel L, Krumb E. How to translate and implement the current science of gene therapy into haemophilia care? Ther Adv Hematol 2023; 14: 20406207221145627. doi: 10.1177/20406207221145627.
Open DOI Search in Google Scholar Back to article
Kahneman D, Tversky A. Prospect theory: an analysis of decision under risk. Econometrica 1979; 47(2): 263-292. doi: 10.2307/1914185.
Open DOI Search in Google Scholar Back to article
Barberis NC. Thirty years of prospect theory in economics: A review and assessment. The Journal of Economic Perspectives 2013; 27(1): 173-195. http://www.jstor.org/stable/41825467.
Search in Google Scholar Back to article
De Martino I, D’Apolito R, McLawhorn AS, Fehring KA, Sculco PK, Gasparini G. Social media for patients: benefits and drawbacks. Curr Rev Musculoskelet Med 2017; 10(1): 141-145. doi: 10.1007/s12178-017-9394-7.
Open DOI Search in Google Scholar Back to article
Benetoli A, Chen TF, Aslani P. How patients’ use of social media impacts their interactions with healthcare professionals. Patient Educ Couns 2018; 101(3): 439-444. doi: 10.1016/j.pec.2017.08.015.
Open DOI Search in Google Scholar Back to article
Graham AL, Cobb CO, Cobb NK. The internet, social media, and health decision-making. In: Diefenbach MA, Miller-Halegoua S, Bowen DJ (Eds.). Handbook of Health Decision Science. Springer Science + Business Media; 2016. 335-355.
Search in Google Scholar Back to article
Limjoco J, Thornburg CD. Development of a haemophilia A gene therapy shared decision-making tool for clinicians. Haemophilia 2023; 29(5): 1184-1190. doi: 10.1111/hae.14822.
Open DOI Search in Google Scholar Back to article
Knapp M, Wong G. Economics and mental health: the current scenario. World Psychiatry 2020; 19(1): 3-14. doi: 10.1002/wps.20692.
Open DOI Search in Google Scholar Back to article
Hindmarch T, Hotopf M, Owen GS. Depression and decision-making capacity for treatment or research: A systematic review. BMC Med Ethics 2013; 14(1): 54. doi: 10.1186/1472-6939-14-54.
Open DOI Search in Google Scholar Back to article
Duncan E, Best C, Hagen S. Shared decision making interventions for people with mental health conditions. Cochrane Database Syst Rev 2010; 2010(1): CD007297. doi: 10.1002/14651858.CD007297.pub2.
Open DOI Search in Google Scholar Back to article
Timmermans S. The engaged patient: the relevance of patient-physician communication for twenty-first-century health. J Health Soc Behav 2020; 61(3): 259-273. doi: 10.1177/0022146520943514.
Open DOI Search in Google Scholar Back to article
UKHCDO. UKHCDO Annual Report 2022 & Bleeding Disorder Statistics for the Financial Year 2021/22. A Report from the UKHCDO and NHD. Manchester: UKHCDO; 2022. Available from https://www.ukhcdo.org/wp-content/uploads/2022/12/UKHCDO-Annual-Report-2022-2021-22-Data.pdf.
Search in Google Scholar Back to article
Baillie L. Exchanging focus groups for individual interviews during qualitative data collection: A discussion. Nurse Res 2019. doi: 10.7748/nr.2019.e1633.
Open DOI Search in Google Scholar Back to article
SPS. SPS Horizon Scanning Service [Internet]. Available from: https://www.sps.nhs.uk/articles/sps-horizon-scanning-service/ (accessed January 2022).
Search in Google Scholar Back to article

Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative study

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