Objective 1: To develop and assess sensitive clinical endpoints, for use in future clinical trials, able to evaluate response to drug treatments in patients with primary Sjögren’s Syndrome with high disease burden and/or systemic involvement:
0%
Objective 1: To develop and assess sensitive clinical endpoints, for use in future clinical trials, able to evaluate response to drug treatments in patients with primary Sjögren’s Syndrome with high disease burden and/or systemic involvement:
We will re-analyse data obtained from all available primary Sjögren’s Syndrome randomized controlled trials, with the goal to identify measures of outcome/s clinically pertinent and that differentiate patients treated with the drug or the placebo.
Thanks to this re-analysis, we will propose a new composite responder index in primary Sjögren’s Syndrome: the Sjögren’s syndrome Tool for Assessing Response (STAR), which will be integrated with ESSDAI (EULAR Sjögren’s syndrome Disease Activity Index) and ESSPRI (EULAR Sjögren’s syndrome Patient Reported Index) as endpoints of future studies.
We will also develop and validate innovative tools to assess the multiple facets of the disease.
Objective 2: To identify and evaluate discriminative biomarkers for stratification of primary Sjögren’s Syndrome patients predictive of organ involvement and disease progression and thus available for inclusion in clinical trials:
We will explore the performance of already discovered biomarkers as markers of stratification and as predictors of response.
We will apply latest generation “omics” to biological samples obtained in existing clinical trial cohorts to identify “smart biomarkers” for disease stratification and prediction.
Objective 3: To set-up and perform a clinical trial to validate the newly defined clinical endpoints and the identified biomarkers:
We will set up an original design for an innovative “multi-arm multi-stage platform trial” able to include all the different types of patients with primary Sjögren’s Syndrome in different arms, with different types of drugs and with different methodology.
In addition, we aim to reach consensus with the key stakeholders – health authorities, payers and patients´ groups – regarding the use of previous and new endpoints for regulatory approvals and reimbursement, respectively, in the management of the primary Sjögren’s Syndrome:
We will incorporate through fruitful discussions the views and expectations of key stakeholders – Health Technology Assessment (HTAs), the European Medicines Agency (EMA), payers and organizations representing patients with primary Sjögren’s Syndrome (The French Association of Sjögren’s Syndrome, Association Française du Gougerot Sjögren (AFGS), on behalf of the International Sjögren’s network (ISN))
Abbreviations: ESSDAI = EULAR Sjögren’s syndrome Disease Activity Index; ESSPRI = EULAR Sjögren’s syndrome Patient Reported Index; EULAR = European League Against Rheumatism; STAR = the Sjögren’s syndrome Tool for Assessing Response.
