Global consensus building and prioritisation of fundamental lupus challenges

The ALPHA project

Susan Manzi, Sandra Raymond, Karin Tse, Yaritza Peña, Annick Anderson, Kathleen Arntsen, Sang Cheol Bae, Ian Bruce, Thomas Dörner, Kenneth Getz, Leslie Hanrahan, Amy Kao, Eric Morand, Brad Rovin, Laura Eve Schanberg, Joan M. Von Feldt, Victoria P. Werth, Karen Costenbader

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Objective Lupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-Term and long-Term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress. Methods A Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic â € nodes' (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus. Results Expert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology. Conclusions ALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

Original languageEnglish
Article numbere000342
Number of pages7
JournalLupus Science and Medicine
Volume6
Issue number1
DOIs
Publication statusPublished - 1 Jul 2019
Externally publishedYes

Keywords

  • barriers to clinical care
  • clinical trial design
  • drug development
  • heterogeneity
  • lupus
  • patient access

Cite this

Manzi, S., Raymond, S., Tse, K., Peña, Y., Anderson, A., Arntsen, K., ... Costenbader, K. (2019). Global consensus building and prioritisation of fundamental lupus challenges: The ALPHA project. Lupus Science and Medicine, 6(1), [e000342]. https://doi.org/10.1136/lupus-2019-000342
Manzi, Susan ; Raymond, Sandra ; Tse, Karin ; Peña, Yaritza ; Anderson, Annick ; Arntsen, Kathleen ; Bae, Sang Cheol ; Bruce, Ian ; Dörner, Thomas ; Getz, Kenneth ; Hanrahan, Leslie ; Kao, Amy ; Morand, Eric ; Rovin, Brad ; Schanberg, Laura Eve ; Von Feldt, Joan M. ; Werth, Victoria P. ; Costenbader, Karen. / Global consensus building and prioritisation of fundamental lupus challenges : The ALPHA project. In: Lupus Science and Medicine. 2019 ; Vol. 6, No. 1.
@article{46f4bb9f5b9a4633b3a570ef3ab5f891,
title = "Global consensus building and prioritisation of fundamental lupus challenges: The ALPHA project",
abstract = "Objective Lupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-Term and long-Term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress. Methods A Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic {\^a} € nodes' (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus. Results Expert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology. Conclusions ALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.",
keywords = "barriers to clinical care, clinical trial design, drug development, heterogeneity, lupus, patient access",
author = "Susan Manzi and Sandra Raymond and Karin Tse and Yaritza Pe{\~n}a and Annick Anderson and Kathleen Arntsen and Bae, {Sang Cheol} and Ian Bruce and Thomas D{\"o}rner and Kenneth Getz and Leslie Hanrahan and Amy Kao and Eric Morand and Brad Rovin and Schanberg, {Laura Eve} and {Von Feldt}, {Joan M.} and Werth, {Victoria P.} and Karen Costenbader",
year = "2019",
month = "7",
day = "1",
doi = "10.1136/lupus-2019-000342",
language = "English",
volume = "6",
journal = "Lupus Science and Medicine",
issn = "2053-8790",
publisher = "BMJ Publishing Group",
number = "1",

}

Manzi, S, Raymond, S, Tse, K, Peña, Y, Anderson, A, Arntsen, K, Bae, SC, Bruce, I, Dörner, T, Getz, K, Hanrahan, L, Kao, A, Morand, E, Rovin, B, Schanberg, LE, Von Feldt, JM, Werth, VP & Costenbader, K 2019, 'Global consensus building and prioritisation of fundamental lupus challenges: The ALPHA project', Lupus Science and Medicine, vol. 6, no. 1, e000342. https://doi.org/10.1136/lupus-2019-000342

Global consensus building and prioritisation of fundamental lupus challenges : The ALPHA project. / Manzi, Susan; Raymond, Sandra; Tse, Karin; Peña, Yaritza; Anderson, Annick; Arntsen, Kathleen; Bae, Sang Cheol; Bruce, Ian; Dörner, Thomas; Getz, Kenneth; Hanrahan, Leslie; Kao, Amy; Morand, Eric; Rovin, Brad; Schanberg, Laura Eve; Von Feldt, Joan M.; Werth, Victoria P.; Costenbader, Karen.

In: Lupus Science and Medicine, Vol. 6, No. 1, e000342, 01.07.2019.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Global consensus building and prioritisation of fundamental lupus challenges

T2 - The ALPHA project

AU - Manzi, Susan

AU - Raymond, Sandra

AU - Tse, Karin

AU - Peña, Yaritza

AU - Anderson, Annick

AU - Arntsen, Kathleen

AU - Bae, Sang Cheol

AU - Bruce, Ian

AU - Dörner, Thomas

AU - Getz, Kenneth

AU - Hanrahan, Leslie

AU - Kao, Amy

AU - Morand, Eric

AU - Rovin, Brad

AU - Schanberg, Laura Eve

AU - Von Feldt, Joan M.

AU - Werth, Victoria P.

AU - Costenbader, Karen

PY - 2019/7/1

Y1 - 2019/7/1

N2 - Objective Lupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-Term and long-Term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress. Methods A Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic â € nodes' (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus. Results Expert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology. Conclusions ALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

AB - Objective Lupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-Term and long-Term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress. Methods A Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic â € nodes' (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus. Results Expert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology. Conclusions ALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

KW - barriers to clinical care

KW - clinical trial design

KW - drug development

KW - heterogeneity

KW - lupus

KW - patient access

UR - http://www.scopus.com/inward/record.url?scp=85070450629&partnerID=8YFLogxK

U2 - 10.1136/lupus-2019-000342

DO - 10.1136/lupus-2019-000342

M3 - Article

VL - 6

JO - Lupus Science and Medicine

JF - Lupus Science and Medicine

SN - 2053-8790

IS - 1

M1 - e000342

ER -