Switching from Off-label & best supportive care to approved therapies for various autoimmune diseases: Implementation, impact on disease progression and adverse events

Switching from Off-label & best supportive care to approved therapies for various autoimmune diseases: Implementation, impact on disease progression and adverse events - CONTRIBUTE

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00036166

Enrollment

Registered

2025-02-14

Start date

2025-01-24

Completion date

Unknown

Last updated

2025-10-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

G70.0 G36.0 G35.2 M32.1 M31.5 M06.1 M30.1 M34 M32

Interventions

Group 1: Retrospective analysis of secondary data of a statutory health insurance in Germany according to the stated objectives. Group 2: Qualitative primary data collection using semi-structured, gui

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

Inclusion criteria: Positive declaration of consent to participate in the study is available Proficiency in the German language Therapy with one of the following drugs in combination with the respective indications of interest: Belimumab (Benlysta®) - LN and SLE, Anifrolumab (Saphnelo®) - SLE, Tocilizumab (RoActemra®) - RZA, Mepolizumab (Nucala®) - EGPA, Anakinra (Kineret®) - AOSD, Canakinumab (Ilaris®) - AOSD, Nintedanib (Ofev®) - SSc-ILD, Eculizumab (Soliris®) - MG and NMOSD, Efgartigimod alfa (Vyvgart®) - MG, Ravulizumab (Ultomiris®) - MG and NMOSD, Satralizumab (Enspryng®) - NMOSD, Inebilizumab (Uplizna®) - NMOSD, Ocrelizumab (Orevus®) - PPMS, Zilucoplan (Zilbrysk®) - MG

Exclusion criteria

Exclusion criteria: Not proficient in the German language Age under 18 years Missing declaration of consent

Design outcomes

Primary

Measure	Time frame
The overall aim of the project is to investigate the changes in care brought about by the newly approved drugs. The following main objectives are being pursued: Objective 1: Analysis of care implementation, taking into account inhibiting and promoting factors. Objective 2: Analysis of effectiveness and safety in routine care, investigation of possible influencing factors, comparison with the results of clinical studies. Objective 3: Health economic evaluation. Objective 4: Recording of care practice from the perspective of patients and service providers. Objective 5: Development of measures for the transfer of study results. Objective 6: Pharmacological outcomes.	—

Measure

Time frame

The overall aim of the project is to investigate the changes in care brought about by the newly approved drugs. The following main objectives are being pursued: Objective 1: Analysis of care implementation, taking into account inhibiting and promoting factors. Objective 2: Analysis of effectiveness and safety in routine care, investigation of possible influencing factors, comparison with the results of clinical studies. Objective 3: Health economic evaluation. Objective 4: Recording of care practice from the perspective of patients and service providers. Objective 5: Development of measures for the transfer of study results. Objective 6: Pharmacological outcomes.

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Secondary

Measure	Time frame
The following additional questions will be investigated as part of objective 1: What proportion of patients are treated with the respective drugs in the first five years after approval? Which patients (by age, gender, autoimmune disease, severity, comorbidities) receive the newly approved drugs and how can differences be explained? How can the continued use of an off-label drug be explained instead of the use of a newly approved drug for the indication? The following further questions will be investigated as part of objective 2: How do the new approvals improve the progression and disease activity of autoimmune diseases compared to the previous treatment? Is there a correlation between study quality and efficacy in the routine data? What is the safety of the novel active substances? Do the novel drugs in the routine data show an increased risk of side effects reported in EudraCT? The following further questions will be investigated as part of objective 3: What is the impact of the drug approvals on medical costs from the SHI perspective? Are the new drugs cost-effective? The following further questions will be investigated as part of objective 4: What experiences do patients have with their drug therapy and how do they rate it? How do healthcare providers experience their patients' drug therapy and how do they evaluate it? The following additional questions will be investigated as part of objective 5: How do patients, care providers and stakeholders (e.g. health insurance companies, The National Association of Statutory Health Insurance Physicians, patient organizations, professional societies) evaluate the project results, which will be prepared into factsheets, with regard to their relevance, comprehensibility and, if applicable, usefulness in everyday (working) life? How can the project methodology be made publicly available and published? The following further questions will be investigated as part of objective 6: Are there differen	—

Measure

Time frame

The following additional questions will be investigated as part of objective 1: What proportion of patients are treated with the respective drugs in the first five years after approval? Which patients (by age, gender, autoimmune disease, severity, comorbidities) receive the newly approved drugs and how can differences be explained? How can the continued use of an off-label drug be explained instead of the use of a newly approved drug for the indication? The following further questions will be investigated as part of objective 2: How do the new approvals improve the progression and disease activity of autoimmune diseases compared to the previous treatment? Is there a correlation between study quality and efficacy in the routine data? What is the safety of the novel active substances? Do the novel drugs in the routine data show an increased risk of side effects reported in EudraCT? The following further questions will be investigated as part of objective 3: What is the impact of the drug approvals on medical costs from the SHI perspective? Are the new drugs cost-effective? The following further questions will be investigated as part of objective 4: What experiences do patients have with their drug therapy and how do they rate it? How do healthcare providers experience their patients' drug therapy and how do they evaluate it? The following additional questions will be investigated as part of objective 5: How do patients, care providers and stakeholders (e.g. health insurance companies, The National Association of Statutory Health Insurance Physicians, patient organizations, professional societies) evaluate the project results, which will be prepared into factsheets, with regard to their relevance, comprehensibility and, if applicable, usefulness in everyday (working) life? How can the project methodology be made publicly available and published? The following further questions will be investigated as part of objective 6: Are there differen

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Countries

Germany

Contacts

Public ContactSven Schmiedl

Private Universität Witten/Herdecke gGmbH Lehrstuhl für Klinische Pharmakologie

sven.schmiedl@uni-wh.de+49 202 8961854

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026