An introduction to real-world evidence (RWE) in multiple sclerosis (MS)
Collection of high-quality real-world data (RWD) in MS
Source | Explanation | Advantages | Limitations | Examples in MS |
---|---|---|---|---|
RCTs extensions | • Supplement trial data | • Extend RCT into real world | • Short study duration • Limited patient population • No information on rare events | • ENDORSE: EQ-5D and SF-36 in patients treated with DMF [10] |
Registries | • Population-based collection of information | • Long-term data natural history and disease management • Regional comparisons | • Non-randomized design • Incomparable patient groupings • Discontinuous visit schedule • Varying practice patterns • Inter-regional extrapolation not always appropriate | • Lyons MS database: disability progression [11] |
Prospective observational studies | • Pre-defined outcome measures in clinical practice | • Robust dataset powered to answer specific questions | • Hawthorne effect (patients behave differently because they know they are being observed) | |
PASSs | • Voluntary or imposed by regulatory authorities for approval | • Ongoing monitoring of the benefit–risk profile | • No obligation for regulatory submission of protocols and study reports for voluntary PASSs | • PANGAEA: German voluntary PASS [20] |
Administrative data | • Data required for reimbursement | • Quick, low-cost analyses • Large patient populations provide information on rare events | • Privacy concerns limit access to data • Incomplete or inaccurate data • Costs and charges are not differentiated | |
Health surveys | • Descriptive data | • Provide broadly generalizable data | • Not product-specific • Subjective • Relies on participant recollection | • NARCOMS survey: symptoms, comorbidities and health-related quality of life [18] |
EMRs | • Real-time data collection | • Low cost • Detailed information over long periods | • High-end statistical analysis tools required | • EMRs: diagnosis, disease progression, symptoms and treatment [12] |
Performing and identifying high-quality RWE studies in MS
How has RWE helped in understanding the disease course and patient management in MS?
Comparative effectiveness of DMTs in clinical practice
Safety and tolerability of a DMT in clinical practice
Impact of a DMT on resource use and medical costs
Impact of a DMT on pharmacoeconomic outcomes
Impact of a DMT on patient-reported outcomes (PROs)
Evolving RWD collection in MS
Registry | Patients, n |
---|---|
Europe
| |
Croatia (SDMSH) | 2477 |
Denmark (DMSC) | 12,500 |
France (EDMUS) | ~40,000 |
Germany (DMSG) | ~30,000 |
Germany (MSDS3D Users) | >5000 |
Greece (GMSS) | 3500 |
Italy (iMed/web) | ~20,000 |
Norway (Nasjonal kompetansetjeneste for multippel sklerose – MS) | 5100 |
Russia | 21,500 |
Serbia | 3500 |
Spain (Catalonia; EpidEMcat) | >5000 |
Sweden (SMSreg) | 12,900 |
Switzerland (SMSR) | 270 |
United Kingdom (MS Register) | 8300 |
North America
| |
Canada (London Ontario) | 1099 |
Canada (British Columbia) | 2837 |
USA (New York; NYSMSC) | 9600a |
USA (NARCOMS) | 19,297 |
Global
| |
MSBase (31 countries) | 39,030 |