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Discrete choice experiments: An overview of experience to date in haemophilia

John Spoors

John Spoors

London School of Hygiene and Tropical Medicine, NHS England and NHS Improvement Medicines Analysis TeamLondon,
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Spoors, John
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Alec Miners

Alec Miners

London School of Hygiene and Tropical MedicineLondon, UK
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Miners, Alec
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John Cairns

John Cairns

London School of Hygiene and Tropical MedicineLondon, UK
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Cairns, John
  
20. Mai 2022
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Artikel-Kategorie: Evidence Review
Online veröffentlicht: 20. Mai 2022
Seitenbereich: 50 - 63
DOI: https://doi.org/10.2478/jhp-2022-0006
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© 2022 John Spoors et al., published by Sciendo
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.
As more therapeutic options become available in haemophilia care, discrete choice experiment may be a useful means of gauging patient preference© Shutterstock
As more therapeutic options become available in haemophilia care, discrete choice experiment may be a useful means of gauging patient preference© Shutterstock

Figure 1

Impact of regulatory developments on capturing patient preferences across the product lifecycle
Impact of regulatory developments on capturing patient preferences across the product lifecycle

Figure 2

Example of a DCE in haemophilia with attributes and levels highlighted [16]
Example of a DCE in haemophilia with attributes and levels highlighted [16]

Figure 3

Search terms for literature search on DCEs in haemophilia care
Search terms for literature search on DCEs in haemophilia care

Figure 4

PRISMA diagram for literature search on DCEs in haemophilia care
PRISMA diagram for literature search on DCEs in haemophilia care

Figure 5

Product attributes mapped over time
Product attributes mapped over time

Figure 6

Comparison of haemophilia gene therapy patient preference studies
Comparison of haemophilia gene therapy patient preference studies

Overview of DCEs within haemophilia

AUTHOR YEAR SURVEY AUDIENCE REGION STUDY TOPIC SAMPLE SIZE (N) MEAN PATIENT AGE (YEARS) SCALE OF QUALITATIVE WORK NO. OF ATTRIBUTES LEVELS PER ATTRIBUTE INDUSTRY FUNDING SURVEY METHODS
Mantovani et al.[27] 2005 Physicians, patients and pharmacists Italy Treatment products in haemophilia Total: 305Physicians: 69Patients: 178Pharmacists: 58 35.9 Focus group (n=N/R) + Pilot (n=15) 6 2–3 Bayer Italia S.p.A. Paper-based survey
Lee et al. [28] 2008 Physicians US Coagulation factor concentrates† Total: 30 N/A Physician engagement (n & format N/R) + testing (N/R) 12 2–4 Novo Nordisk Inc Paper-based survey
Scalone et al.[29] 2009 Physicians, patients and pharmacists Italy Coagulation factor concentrates† Total: 101Physicians: 39Patients: 37Pharmacists: 25 Adults = 41.4Paedatric = 8.2 Focus group (n=N/R) + Pilot (n=35) 8 2–3 Novo Nordisk Denmark N/R
Brown et al.[16] 2011 Patients and caregivers US Treatment products in haemophilia† Total: 53*Patients: 23Caregivers: 30 20.7 Used same as Lee et al. (2008)[28] 12 2–4 Novo Nordisk Inc Paper-based survey
Mohamed et al.[30] 2011 Patients and caregivers US Treatment products in haemophilia A Total: 147Patients: 77Caregivers: 70 Adults = 38.7Juvenile = 12.4 Face-to-face interviews with adult patients and caregivers (n=8) 6 2–3 Baxter Biosciences Web-based survey
Gelhorn et al.[31] 2013 Physicians US and EU Treatment products in haemophilia A† Total: 36Physicians EU: 17Physicians US: 19 N/A Qualitative interviews (n=4) + Pilot (n=5) 5 3 Baxter Biosciences Web-based survey
Chaugule et al.[32] 2015 Patients and caregivers US Willingness to pay for treatment products in haemophilia Total: 79 40.0 Assistance from Haem author + Pilot (n=5) 5 2–5 None iPad at conference
Lock et al. [33] 2016 Patients, caregivers and HCPs Multi-country PK-guided dosing of prophylaxis Total: 224HCPs: 91Patients: 114Caregivers: 19 38.0 Qualitative interviews + Pilot (n=10) 5 2–3 Pfizer Paper-based survey
Fifer et al. [34] 2019 Patients and caregivers Multi-country Treatment products in haemophilia A Total: 54^Patients: 24Caregivers: 30 N/R Qualitative interviews (n=10) 11 2–4 Roche Web-based survey
Su et al. [35] 2020 Patients and caregivers US Treatment products in haemophilia A Total: 209Patients: 113Caregivers: 96 35.5 Qualitative interviews (n=10)** + Pilot (n=6) 6 2–4 Sanofi Genzyme Web-based survey
Park et al. [36] 2021 Patients and caregivers South Korea Treatment products in haemophilia A Total: 505*** 31.5 N/R 5 3 Pfizer Pharmaceuticals Korea Ltd Hospital-based survey
Witkop et al. [37] 2021 Patients US Haemophilia gene therapy Total: 183 38.5 Qualitative interviews (n=7) ** + Pilot (n=14) 6 3–4 uniQure Inc. Web-based survey

Potential future treatments in haemophilia*

PRODUCT NAME THERAPEUTIC FOCUS PRODUCT TYPE PHASE OF CLINICAL DEVELOPMENT
Serpin PC Haemophilia A and Haemophilia B Specific inhibitor of activated protein C (APC) Phase II
Mim8 Haemophilia A Next generation FVIII mimetic antibody Phase III
Etranacogene dezaparvovec Haemophilia B AAV5 gene therapy (in vivo) Phase III
Fidanacogene elaparvovec Haemophilia B AAV8 gene therapy (in vivo) Phase III
Valoctocogene roxaparvovec Haemophilia A AAV5 gene therapy (in vivo) Phase III
Concizumab Haemophilia A and Haemophilia B Monoclonal antibody directed against tissue factor pathway inhibitor (TFPI) Phase III
Fitusiran Haemophilia A and Haemophilia B RNAi therapeutic targeting antithrombin Phase III
Efanesoctocog alfa Haemophilia A Fully recombinant factor VIII therapy independent of von Willebrand factor Phase III
Eptacog beta activated Haemophilia A and Haemophilia B (in patients with inhibitory antibodies to factor VIII or IX) Transgenically produced recombinant human factor VIIa Phase III**
Giroctocogene fitelparvovec Haemophilia A AAV2/6 gene therapy (in vivo) Phase III
Dirloctocogene samoparvovec Haemophilia A AAV-LK03 gene therapy (in vivo) Phase III
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