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Original research
Global consensus building and prioritisation of fundamental lupus challenges: the ALPHA project
  1. Susan Manzi1,
  2. Sandra Raymond2,
  3. Karin Tse2,
  4. Yaritza Peña3,
  5. Annick Anderson3,
  6. Kathleen Arntsen4,
  7. Sang-Cheol Bae5,
  8. Ian Bruce6,
  9. Thomas Dörner7,
  10. Kenneth Getz3,
  11. Leslie Hanrahan2,
  12. Amy Kao8,
  13. Eric Morand9,
  14. Brad Rovin10,
  15. Laura Eve Schanberg11,
  16. Joan M Von Feldt12,
  17. Victoria P Werth13 and
  18. Karen Costenbader14
  1. 1Medicine, Allegheny Health Network, Pittsburgh, Pennsylvania, USA
  2. 2Lupus Foundation of America Inc, Washington, District of Columbia, USA
  3. 3Tufts Center for the Study of Drug Development, Boston, Massachusetts, USA
  4. 4Lupus and Allied Diseases Association, Inc, Verona, New York, USA
  5. 5Rheumatology, Hanyang University Seoul Hospital, Seongdong-gu, Seoul, Korea (the Republic of)
  6. 6Musculoskeletal and Dermatological Sciences, The University of Manchster, Manchester, England, UK
  7. 7Rheumatology and Clinical Immunology, Charite University Hospitals Berlin, Berlin, Germany
  8. 8EMD Serono Research and Development Institute, Billerica, Massachusetts, USA
  9. 9Rheumatology, Monash Medical Centre, Melbourne, Victoria, Australia
  10. 10Internal Medicine/Nephrology, Ohio State University Wexner Medical Center, Columbus, Ohio, USA
  11. 11Pediatrics, Duke University, Durham, North Carolina, USA
  12. 12GlaxoSmithKline USA, Philadelphia, Pennsylvania, USA
  13. 13Dermatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA
  14. 14Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA
  1. Correspondence to Sandra Raymond; raymond{at}lupus.org

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 ‘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.

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

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

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Footnotes

  • Contributors All authors contributed to the discussions and data interpretation contained in this paper and provided input on the manuscript and approved its final version. AA and YP conducted expert interviews, survey programming and data analysis. KT and YP contributed to manuscript writing and finalising. KG and LH oversaw and approved all research efforts.

  • Funding The ALPHA Project was launched in partnership with founding partner EMD Serono Research & Development, Inc. (a business of Merck KGaA, Darmstadt, Germany) and through additional support by GlaxoSmithKline.

  • Competing interests IB is an NIHR Senior Investigator and is funded by Arthritis Research UK, the National Institute for Health Research Manchester Biomedical Research Unit and the NIHR/Wellcome Trust Manchester Clinical Research Facility. IB has received grants and/or honoraria and/or consultancy fees from Genzyme Sanofi, GSK, UCB, AstraZeneca, Medimmune, Eli Lilly, Merck Serono and ILTOO. KC receives research support to study lupus from the NIH (R01 AR057327 and K24 AR066109), Merck and AstraZeneca. KC has received consulting honoraria < $5000 from AstraZeneca. TD has received grants for study conduct and/or honoraria and /or consultancy fees from Janssen, GSK, Roche/Genentech, Eli Lilly, UCB, Merck Serono, AbbVie and Boston Pharmaceuticals. SM has received grants and/or honoraria and/or consultancy fees from Exagen Diagnostics, GSK, UCB and AstraZeneca. EM has received grants for study conduct and/or honoraria and/or consultancy fees from AbbVie, Sanofi, GSK, UCB, AstraZeneca, Eli Lilly, Merck Serono and Janssen. BR has received grants and/or honoraria and/or consultancy fees from Aurinia, EMD Serono, Janssen and MedImmune. BR is also a Principal Investigator on research projects for EMD Serono Research and Development Institute Inc. (Collaborated by Merck KGaA); Human Genome Sciences Inc, a GSK Company; NIH and RILITE Foundation. LES receives research support from CARRA and PCORI. LES is also on Data and Safety Monitoring Boards for UCB and Sanofi. VPW has received grants and/or honoraria from Celgene, Janssen, Biogen, Gilead, Viela, Medimmune, Pfizer, Resolve, AstraZeneca, Kyowa Hakko Kirin, Eli Lilly.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data availability statement Data are available upon reasonable request.

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