As medicine becomes more personalized and therapies more complex, the traditional clinical evaluation model is evolving. Real-World Evidence (RWE) is no longer just a complementary tool — it is now a strategic component in the development of medicines and medical devices. For sponsors, regulators, and healthcare payers, RWE provides the critical perspective: how a treatment truly performs in everyday clinical practice.
RWE refers to clinical evidence derived from the analysis of Real-World Data (RWD) — data collected outside the controlled environment of randomized clinical trials.
Key data sources include:
Electronic Health Records (EHRs)
National or disease registries
Insurance and reimbursement databases
Post-authorization observational studies
Digital devices and remote monitoring technologies
Patient-Reported Outcomes (PROs)
These data reflect real disease behavior, treatment adherence, and population variability, factors that are difficult to fully capture in traditional RCTs.
Randomized clinical trials (RCTs) remain fundamental for demonstrating cause–effect relationships. However, they are designed for control — not for realism.
|
RCT Setting |
Real-World Setting |
|---|
|
Strict inclusion criteria |
Patients with multiple comorbidities |
|
Intensive monitoring |
Variable treatment adherence |
|
Specialized centers |
Heterogeneous healthcare systems |
|
Limited duration |
Long-term treatment use |
This difference creates what the industry calls the “efficacy–effectiveness gap” — the gap between performance in trials and performance in clinical practice.
RWE is the tool that helps close this gap.
RWE enables evaluations that were previously difficult or impossible:
Therapies can be assessed in:
elderly patients
individuals with multiple conditions
populations underrepresented in clinical trials
RWE supports tracking of:
real-world survival
disease progression
impact on quality of life
need for subsequent therapies
In the absence of head-to-head trials, RWE can support:
indirect treatment comparisons
understanding real therapeutic positioning
RWE is essential for modern pharmacovigilance:
identification of rare adverse events
detection of risks in specific subgroups
evaluation of off-label use
long-term safety monitoring
Many important safety signals only become visible after widespread use in real populations.
Regulatory agencies such as EMA and FDA are increasingly integrating RWE into:
indication extensions
accelerated approvals
post-authorization requirements
continuous benefit–risk evaluations
This shifts the regulatory model from a one-time assessment to a dynamic, data-driven process.
For payers, the key question is no longer only:
“Does it work?”
But rather:
“What is the therapy’s real value within the healthcare system?”
RWE supports:
cost-effectiveness analyses
outcome-based reimbursement models
demonstration of clinical value under real conditions
In many cases, RWE makes the difference between approval and true market access.
In this evolving landscape, the role of CROs is expanding significantly. A partner such as Tigermed EMEA supports sponsors through:
design and execution of observational studies
integration and harmonization of multiple data sources
advanced statistical analysis and modeling
evidence generation for regulatory, HTA, and market access needs
lifecycle evidence strategies
RWE is no longer a secondary activity — it is a strategic pillar of clinical development planning.
Real-World Evidence marks the transition from medicine proven under ideal conditions to medicine validated in real life.
For modern therapies, success no longer means only:
“It works in a trial.”
It now means:
“It works for real patients, in real healthcare systems, over the long term.”
In this new paradigm, RWE becomes the essential bridge between research, clinical practice, and healthcare decision-making — and CROs are the architects of this evidence.