Global consensus building and prioritisation of fundamental lupus challenges: the ALPHA project

Abstract

ObjectiveLupus 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.MethodsA 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.ResultsExpert 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.ConclusionsALPHA 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.

“There’s so much to be done”: a qualitative study to elucidate research priorities in childhood-onset systemic lupus erythematosus

“…While there have been advances in understanding disease mechanisms and treatment of cSLE in the last decade, significant needs remain, underscoring the necessity of a focused research agenda for allocation of available resources. Expanding on the findings from the ALPHA study5 6 and CARRA-LFA survey regarding cSLE research priorities,7 we elucidated approaches to address cSLE research priorities, exploring both barriers and opportunities to advancing cSLE research. Multidisciplinary experts in our study identified research priorities represented by five domains: (1) expanding disease knowledge; (2) investigator collaboration; (3) partnering with patients and families; (4) improving care to optimise research; and (5) overcoming investigator barriers.…”

“…Although paediatric rheumatologists were included in both the initial interview and survey components, only 6% of survey respondents primarily practised paediatric rheumatology. Responses of phase I of the ALPHA survey indicated that the lack of attention to paediatric issues is a barrier to SLE drug development and clinical care 5 ; however, specific aspects of these issues were not detailed. Subsequently, phase II of the ALPHA study proposed actionable solutions to the identified research barriers from phase I of the study.…”

Health-Related Quality of Life Improvements in Systemic Lupus Erythematosus Derived from a Digital Therapeutic Plus Tele-Health Coaching Intervention: Randomized Controlled Pilot Trial

“…As discussed, disease heterogeneity is perhaps the most significant obstacle to progress. Other barriers to advancement are lack of diagnostic, predictive, prognostic, and drug-response biomarkers; ineffective management of SLE due to social determinants of care in predominantly lower socioeconomic status areas; and lack of treatment adherence [ 24 ]. Nontraditional solutions to these challenges should be explored and digital therapeutics offer one such novel approach.…”

“…Widely variable initial presentation, disease course, organ involvement, and response to treatment complicate diagnosis, management, and clinical research efforts. An international team of experts have identified SLE heterogeneity as “the primary barrier hindering advancement” [ 24 ]. Given this heterogeneity, it is reasonable to hypothesize that numerous dietary, environmental, and lifestyle SLE triggers exist and differ from patient to patient.…”

The ALPHA Project: Establishing consensus and prioritisation of global community recommendations to address major challenges in lupus diagnosis, care, treatment and research

“…Phase I, during which these barriers were identified, was completed in 2019. 9 As described in this article, the goals of phase II were to confirm the barriers, identify and reach consensus on actionable solutions to each barrier, and determine preliminary steps to implement these solutions. Figure 1 summarises the phases of the ALPHA Project.…”