Primary Sjögren's Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren's Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI)

Simon J Bowman¹, Raphaele Seror², Raphael Porcher³, Suzanne Arends⁴, Liseth de Wolff⁴, Gwenny Verstappen⁴, Valerie Devauchelle-Pensec⁵, Sandrine Jousse-Joulin⁵, Chiara Baldini⁶, Michele Bombardieri⁷, Elena Pontarini⁷, Wolfgang Hueber⁸, Jessica Marvel⁹, Pushpendra Goswami⁸, Divi Cornec⁵, Benjamin A Fisher¹⁰, Saba Nayar¹¹, Francesca Barone¹², Wan-Fai Ng¹³, Jacques-Eric Gottenberg¹⁴, Hendrika Bootsma⁴, Thomas Dörner¹⁵, Maggy Pincemin¹⁶, Coralie Bouillot¹⁷, Katherine M Hammitt¹⁸, Marie Wahren-Herlenius¹⁹, Joel van Roon²⁰, Gaetane Nocturne², Laurence Laigle²¹, Philippe Moingeon²², Antonia Christodoulou²³, Antoine G Sreih²⁴, Andre van Maurik²⁵, Wen-Hung Chen²⁶, Nicolas Wisniacki²⁵, Alena Piatrova²⁷, Roland Jonsson²⁸, Peter Gergely⁸, Xavier Mariette²⁹

Affiliations

¹ Department of Rheumatology, University Hospitals Birmingham, Birmingham, UK; School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK; Milton Keynes University Hospital, Milton Keynes, UK. Electronic address: simon.bowman@uhb.nhs.uk.
² Paris-Saclay University, INSERM UMR1018: CESP, Le Kremlin-Bicetre, France; Rheumatology, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris (AP-HP), Le Kremlin-Bicêtre, France.
³ Clinical Epidemiology Center, Assistance Publique-Hôpitaux de Paris, Hôtel-Dieu, Paris, France; Université Paris Cité, Université Sorbonne Paris Nord, INSERM, INRAE, Centre for Research in Epidemiology and StatisticS (CRESS), Paris, France.
⁴ Department of Rheumatology and Clinical Immunology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
⁵ Department of Rheumatology, Brest University CHU de Brest, INSERM UMR 1227, LBAI, Brest, France.
⁶ Rheumatology Unit, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
⁷ Experimental Medicine and Rheumatology, William Harvey Research Institute, Queen Mary University of London, London, UK.
⁸ Novartis Pharma AG, Basel, Switzerland.
⁹ Novartis Services Inc, East Hanover, NJ, USA.
¹⁰ Department of Rheumatology, University Hospitals Birmingham, Birmingham, UK; School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK.
¹¹ National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK; Rheumatology Research Group, School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK.
¹² Rheumatology Research Group, Institute of Inflammation and Ageing, University of Birmingham, Birmingham, UK.
¹³ University College Cork, Cork, Republic of Ireland; University of Newcastle, Newcastle, UK.
¹⁴ Rheumatology, University Hospital of Strasbourg, Strasbourg, France; Université de Strasbourg, IBMC, CNRS, UPR3572, Strasbourg, France.
¹⁵ Rheumatology and Clinical Immunology, Charité University Medicine Berlin, Berlin, Germany.
¹⁶ Association Française du Gougerot Sjögren, Ostwald, France.
¹⁷ Sjögren Europe, Gland, Switzerland.
¹⁸ Sjögren's Foundation, Reston, VA, USA.
¹⁹ Division of Rheumatology, Department of Medicine, Karolinska Institutet, Stockholm, Sweden.
²⁰ Immunology, Rheumatology and Clinical Immunology, Center of Translational Immunology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
²¹ Servier, Research and Development, Saclay, France.
²² Servier, Research and Development, Saclay, France; University Paris-Saclay, Saclay, Paris, France.
²³ Bristol-Myers Squibb, Lawrenceville, NJ, USA.
²⁴ Cullinan Therapeutics, Cambridge, MA, USA.
²⁵ GlaxoSmithKline, Stevenage, UK.
²⁶ Independent Researcher, MD, USA.
²⁷ Paris-Saclay University, INSERM UMR1018: CESP, Le Kremlin-Bicetre, France; Hôpitaux Universitaires Paris-Sud - Hôpital Bicêtre, Le Kremlin-Bicêtre, France.
²⁸ Broegelmann Research Laboratory, Department of Clinical Science, University of Bergen, Bergen, Norway.
²⁹ Rheumatology, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris (AP-HP), Le Kremlin-Bicêtre, France; Université Paris-Saclay, INSERM UMR1184, CEA, Centre de recherche en Immunologie des infections virales et des maladies auto-immunes, 94270, Le Kremlin Bicêtre, France.

PMID: 40527714
DOI: 10.1016/j.ard.2025.05.004

Free article

Review

Primary Sjögren's Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren's Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI)

Simon J Bowman et al. Ann Rheum Dis. 2025 Jul.

Free article

. 2025 Jul;84(7):1068-1089.

doi: 10.1016/j.ard.2025.05.004. Epub 2025 Jun 17.

Authors

Affiliations

¹ Department of Rheumatology, University Hospitals Birmingham, Birmingham, UK; School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK; Milton Keynes University Hospital, Milton Keynes, UK. Electronic address: simon.bowman@uhb.nhs.uk.
² Paris-Saclay University, INSERM UMR1018: CESP, Le Kremlin-Bicetre, France; Rheumatology, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris (AP-HP), Le Kremlin-Bicêtre, France.
³ Clinical Epidemiology Center, Assistance Publique-Hôpitaux de Paris, Hôtel-Dieu, Paris, France; Université Paris Cité, Université Sorbonne Paris Nord, INSERM, INRAE, Centre for Research in Epidemiology and StatisticS (CRESS), Paris, France.
⁴ Department of Rheumatology and Clinical Immunology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
⁵ Department of Rheumatology, Brest University CHU de Brest, INSERM UMR 1227, LBAI, Brest, France.
⁶ Rheumatology Unit, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
⁷ Experimental Medicine and Rheumatology, William Harvey Research Institute, Queen Mary University of London, London, UK.
⁸ Novartis Pharma AG, Basel, Switzerland.
⁹ Novartis Services Inc, East Hanover, NJ, USA.
¹⁰ Department of Rheumatology, University Hospitals Birmingham, Birmingham, UK; School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK.
¹¹ National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK; Rheumatology Research Group, School of Infection, Inflammation & Immunology, University of Birmingham, Birmingham, UK.
¹² Rheumatology Research Group, Institute of Inflammation and Ageing, University of Birmingham, Birmingham, UK.
¹³ University College Cork, Cork, Republic of Ireland; University of Newcastle, Newcastle, UK.
¹⁴ Rheumatology, University Hospital of Strasbourg, Strasbourg, France; Université de Strasbourg, IBMC, CNRS, UPR3572, Strasbourg, France.
¹⁵ Rheumatology and Clinical Immunology, Charité University Medicine Berlin, Berlin, Germany.
¹⁶ Association Française du Gougerot Sjögren, Ostwald, France.
¹⁷ Sjögren Europe, Gland, Switzerland.
¹⁸ Sjögren's Foundation, Reston, VA, USA.
¹⁹ Division of Rheumatology, Department of Medicine, Karolinska Institutet, Stockholm, Sweden.
²⁰ Immunology, Rheumatology and Clinical Immunology, Center of Translational Immunology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
²¹ Servier, Research and Development, Saclay, France.
²² Servier, Research and Development, Saclay, France; University Paris-Saclay, Saclay, Paris, France.
²³ Bristol-Myers Squibb, Lawrenceville, NJ, USA.
²⁴ Cullinan Therapeutics, Cambridge, MA, USA.
²⁵ GlaxoSmithKline, Stevenage, UK.
²⁶ Independent Researcher, MD, USA.
²⁷ Paris-Saclay University, INSERM UMR1018: CESP, Le Kremlin-Bicetre, France; Hôpitaux Universitaires Paris-Sud - Hôpital Bicêtre, Le Kremlin-Bicêtre, France.
²⁸ Broegelmann Research Laboratory, Department of Clinical Science, University of Bergen, Bergen, Norway.
²⁹ Rheumatology, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris (AP-HP), Le Kremlin-Bicêtre, France; Université Paris-Saclay, INSERM UMR1184, CEA, Centre de recherche en Immunologie des infections virales et des maladies auto-immunes, 94270, Le Kremlin Bicêtre, France.

PMID: 40527714
DOI: 10.1016/j.ard.2025.05.004

Abstract

Primary Sjögren's disease (pSjD) is an autoimmune rheumatic disease involving exocrine glands and associated with high symptom burden (dryness, fatigue, pain), systemic features and salivary gland dysfunction. B-cell hyperactivity is common, with an increased risk of mucosa-associated lymphoid tissue lymphoma. This review describes the unmet need, scientific validity of outcome measures, optimisation of clinical trial design, therapeutic advances and how clinical improvement relates to health-related quality of life, additional quality-adjusted life years and economic benefit in pSjD. It derives from the EU-funded Necessity IHI Academic-Industry collaborative Consortium project while also drawing on work by the European Alliance of Associations for Rheumatology Sjögren's task force and others. The NECESSITY Consortium, formed within the framework of the Innovative Health Initiative (IHI), comprises 20 academic partners, 1 patient group partner and 4 industry partners (NECESSITY; https://necessity-h2020.eu). Patient leaders have been closely involved, with expert advice obtained from the European Medicines Agency and the United States Food and Drug Administration during the development phase of a new outcome measure, the Sjögren's Tool for Assessing Response composite response criteria. This tool is now undergoing validation through the NECESSITY IHI clinical trial and industry-sponsored trials.

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Conflict of interest statement

Competing interests SJB reports consultancy for Abbvie, Aera, Amgen, Argenx, Aurinia, Bain, BMS, EcoR1, Galapagos, Iqvia, J&J, Kiniksa, Novartis, Scitaris. RS reports consulting fees/honoraria from GSK, Bristol Myer Squib, Boehringer, Amgen, Novartis, Janssen Pfizer and Roche; support for meeting attendance from Amgen and GSK. SA is consultant for Argenx, BMS, Galapagos and Novartis. GV is consultant for Argenx, Aurinia. VD-P reports a role in advisory boards for Abbvie, Chugai, Novartis and financial support from Abbvie, Lilly, Novartis. Chiara Baldini reports consulting fees/honoraria from Amgen, GSK, Johnson & Johnson, Sanofi, Novartis, Aurinia. MB reports unrestricted grants from Janssen Pharmaceuticals, GSK and BMS and is an advisory board member and/or has received speaker fees from Novartis, Amgen, Ono Pharmaceutical, GSK, UCB, sGTX. EP is in consultancy for GSK. WH is an employee and shareholder of Novartis Pharma AG. JM is an employee of Novartis Services Inc and stockholder of Novartis Corporation. PG is an employee and stockholder of Novartis Pharma AG. DC reports research grants from Novartis, GSK, Servier, BMS, Roche-Chugai, CSL Behring, Astra-Zeneca. BAF reports consultancy: Novartis, BMS, Servier, Galapagos, Roche, UCB, Sanofi, Janssen, AZ, Otsuka, Amgen, Kiniksa. BAF reports funding from Janssen, Servier, Galapagos, Celgene. FB is an employee and stockholder of Candel Therapeutics.W-FN reports consultancy for Amgen, Bristol Myers Squibb, Novartis, Johnsons & Johnsons Innovations, Sanofi, Argenx, IQVIA, Quotients, Resolve Therapeutics, EQT & Veloxis. J-E G is involved with Abbvie, BMS, Chugai, Galapagos, Gilead, Lilly, Janssen, Novartis, Pfizer, Roche, Sanofi. HB reports grants from BMS, AstraZeneca, Novartis. HB reports consultancy in BMS, AstraZeneca, Novartis, Galapagos, Argenx, UCB. TD is involved with Novartis, J&J, Sanofi. MW-H reports unrestricted grants from Merck KGaA and Janssen Pharmaceuticals. GN reports honoraria from Novartis, Boehringer, AbbVie, and Galapagos SASU and travel fees from Amgen, AbbVie, Novartis and UCB. LL is a former employee of Servier, R and D Institute, Gif-Sur-Yvette, France. PM is a former employee of Servier, R and D Institute, Gif-Sur-Yvette, France. AC is an employee and stockholder of Bristol-Myers Squibb SA. AGS is an employee of Cullinan Therapeutics and is a former employee and current stockholder of Bristol-Myers Squibb. AvM is an employee and stockholder of GSK. W-HC is a former employee and current stockholder of GSK and Astrazeneca. NW is an employee and stockholder of GSK. Peter Gergely is an employee and stockholder of Novartis Pharma AG. XM is in consultancy and has received honorarium from BMS, GSK, Janssen, Novartis, Otsuka, Pfizer. All other authors declare they have no competing interests.

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Primary Sjögren's Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren's Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI)

Affiliations

Primary Sjögren's Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren's Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI)

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Abstract

Conflict of interest statement

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