Is Real World Evidence (RWE) a reality for the pharmaceutical industry?

26 de agosto de 2025
Michele Rocha

Real-world evidence (RWE) refers to the use of data collected outside of traditional clinical trials—such as electronic health records, medical charts, pharmacovigilance databases, health insurance providers, and even pharmacies—to generate evidence about the use, benefits, or risks of health products.

This concept has gained global traction, especially in contexts where randomized controlled trials (RCTs) have limitations, such as in rare diseases, personalized therapies, or off-label use scenarios.

But to what extent is this approach already accepted by agencies like Anvisa? What are the main challenges to applying it nationwide? And how can pharmacists and pharmacies position themselves within this new paradigm?

Índice

How does RWE differ from traditional clinical trials?

Randomized controlled trials are considered the gold standard of evidence-based medicine. However, they involve rigorous inclusion and exclusion criteria, controlled samples, and ideal study conditions. This does not always reflect the reality of clinical practice.

RWE, on the other hand, offers a complementary perspective, showing how healthcare products work in real-world situations, with diverse patients and in varying contexts—broadening the understanding of safety, effectiveness, adherence, and economic impact.

Abroad, the FDA and EMA have already accepted the use of RWE to expand therapeutic indications, approve medications for rare diseases, or even authorize the replacement of generics and biosimilars. In Brazil, although in its initial stages, Anvisa has already been... recognizing the relevance of RWEparticularly in pilot programs focused on rare diseases, compassionate use, and discussions about incorporating technology into healthcare.

In what contexts is Anvisa considering the use of RWE?

According to the Anvisa Guide on RWE (2023), RWE can be considered as supplementary evidence in the following situations:

Requests for orphan drug registry (for rare diseases);
Expansion of therapeutic indication;
Post-marketing follow-up;
Support for package insert modification, especially in cases with a low number of patients.

The Brazilian agency is aligned with international movements for the responsible use of RWE, such as in the US (FDA), Europe (EMA) and Canada (Health Canada).

Is it possible to use clinical pharmacy data as Real World Data?

Yes. Data from pharmacies, when collected systematically and following defined protocols, can be considered Real World Data (RWD), which is the basis for generating RWE.

However, for this data to have regulatory value, They must be planned in advance....with clear data collection criteria, ethical consent when applicable, and assured record quality. This includes, for example, documented pharmaceutical interventions, monitored clinical outcomes, and the use of standardized record-keeping systems.

What are the main regulatory challenges in the use of RWE in Brazil?

Despite its potential, there are still significant obstacles to consolidating RWE as a regulatory tool:

Data quality: Lack of standardization, incomplete or inconsistent data can compromise the robustness of the conclusions.
Bias and confusion: In observational studies, it is difficult to isolate confounding variables as in an RCT.
Reproducibility and transparency: It is essential to ensure that studies with RWE follow documented protocols and can be audited.
Technical training: Both regulators and researchers need to be prepared to critically evaluate and apply this evidence.
Ethics and data protection: especially when the data comes from medical records, pharmacies, or health insurance providers.

What criteria does Anvisa (Brazilian Health Regulatory Agency) expect to accept studies with RWE (Respiratory Stress Evaluation)?

Based on the technical note and the slides presented, it is essential to observe the following criteria:

The study protocol should be prospective or with a well-defined plan in advance.even if it uses a retrospective database.
The study should present Clear objectives, explicit hypotheses, and an appropriate statistical plan. and robust justifications for the use of the chosen database.
The data source should be accessible for auditing and validationand adhere to the principles of good clinical and pharmacovigilance practices.

What is the future of RWE in pharmaceutical regulation and practice?

Technological advancements, the growth of clinical databases, and the promotion of value-based healthcare models indicate that RWE (Research, Writing, Evidence) is becoming increasingly relevant. In the pharmaceutical context, in addition to influencing regulatory decisions, RWE can also be used to:

Expand the clinical role of the pharmacist in generating evidence;
Contribute data for pricing or refund negotiations;
Validate the results of pharmacotherapeutic monitoring programs.

However, the effective integration of RWE into the regulatory system will depend on collaboration between companies, healthcare professionals and regulatory bodies, focusing on building trust, robust methodologies, and concrete benefits for patients.

Can the Sentinel Program generate RWE data?

Yes. The Sentinel Program, from Anvisa, acts as a national network of health institutions that monitor adverse events and failures in health technologies, and it is one of the largest sources of real-world data available in the country.

Although the original focus of the Sentinel is more aligned with pharmacovigilance and post-market surveillance, their databases — if properly structured and analyzed — can become valuable sources for RWE studies. The records kept by these institutions (hospitals, clinics, laboratories) provide information on everyday use that can support safety and effectiveness assessments.

The integration between networks like Sentinel and RWE initiatives can accelerate the Brazilian regulatory maturityincluding enabling the creation of interoperable databases and multicenter studies based on local data.

Can pharmacists and pharmacies generate RWE?

Yes — and that's a field of Great opportunity and professional responsibility..

Community pharmacies and pharmaceutical clinics are in a privileged position to collect clinical data on actual drug useincluding adherence to treatment, adverse reactions, patient preferences, and even minor clinical outcomes.

With well-defined protocols, this data can feed into the system. structured bases, which in turn become material for RWE studies, especially in the case of Simplified notification medicines, herbal medicines, food supplements and cosmetics with functional claims. — classes that do not undergo prior evaluation by Anvisa.

For the pharmacist, knowing the criteria of data validation, traceability and integrity It is essential for operating safely and contributing to regulatory science. And for pharmacies, participating in clinical data collection projects can add institutional value and strengthen the pharmacy's image as a professional. point of care in health and open up new possibilities for partnerships with the pharmaceutical industry or public health programs.

Conclusion

RWE is already an irreversible trend on the international stage and is beginning to gain traction in Brazilian regulatory discussions. However, for its use to expand safely, ethically, and technically, a concerted effort will be necessary. of the industry, to invest in quality education; from regulatory authorities, to create clearer regulatory frameworks; and of health professionals...such as pharmacists, who may be on the front lines of collecting and interpreting real data.

By staying up-to-date on methodological requirements, regulatory frameworks, and local challenges, Brazil can take its place at the forefront of global regulatory science—and transform real-world data into safer, more effective, and patient-centered decisions.

How can pharmacists prepare for this new scenario?

Working with RWE requires technical knowledge, mastery of regulatory fundamentals, and an understanding of the methodological requirements involved. If you are a pharmacist or work in pharmacies and want to be at the forefront, Empower yourself!

You can delve deeper into this and other fundamental topics for modern pharmaceutical practice with the courses from [Company Name]. LegalPharma Academy, including customized training for pharmacies. And, to make your day-to-day life easier, the LegalPharma app It brings together all relevant health and regulatory standards organized by subject, with practical summaries and constant updates — including specific materials on real-world studies.

The regulatory future will be built by those who master the data, interpret the legislation, and know how to apply it in practice. Are you and your pharmacy prepared? Contact LegalPharma today to create a customized training program for your pharmacy, institution, or team!

Reference: Anvisa (2023). Guidelines for Submitting Real-World Evidence (RWE) in Drug Regulatory Applications. Version 1