Table 4

Primary and secondary unmet scientific needs within systemic lupus erythematosus with regard to translational science, clinical science and therapeutic trials, and clinical care∗

Primary unmet needsSecondary unmet needs
Translational/basic science Better understanding of the role of T and B lymphocytes (and subsets), the epigenetic modification of various cell types (in connexion with environmental factors) and metabolic perturbations in the pathophysiology of disease
Identification of factors that lead to tissue specificity of SLE disease manifestations
Further development of longitudinal, clinically well-characterised cohorts (immunologically, genetically and metabolically) with appropriate imaging, tissue and fluid samples; improved data-sharing among investigators
The development of diagnostic tools to identify preclinical disease states
Identification of contribution of genetic variants and soluble mediators to risk of disease
Better understanding of the natural history of disease flares
Clinical science and therapeutic trials Further refinement of clinical response measures/index
Critical appraisal and potential revision of the current instruments to assess disease activity and treatment response
Standardisation of a definition of disease remission
Clinical trials that incorporate IFN signature and emphasise responder analyses
Large pragmatic trials of existing and emerging therapies. These trials should focus also on prevention of disease in those identified to be at risk
Small proof of mechanism trials for emerging therapies
Improved identification and targeting of the innate immune response
Improved identification and use of biomarkers within clinical practice and trials
Broaden membership of groups designing trials
Clinical care Better characterise patient concerns (vs provider concerns)
Optimisation of steroid-sparing approaches to treatment including the development of toxicity scoring systems, the development of sustained release or organ-targeted steroid preparations, and consideration of different ‘phases’ of steroid use
Improved understanding of targeting specific therapies to specific disease clinical manifestations
Identification of socioeconomic factors that contribute to long-standing disease
Establish patient support groups and guides/advocates to improve adherence to medical regimen
Better understand cognitive dysfunction associated with disease and the development of a usable instrument to quantify in clinical practice
  • *Sequence of needs within the table is random, and is not meant to imply order of importance.

  • IFN, inteferon; SLE, systemic lupus erythematosus.