Pharmacoepidemiology

EMA Qualification of the ECFSPR

In 2018, the European Medicines Agency (EMA) formally qualified the ECFSPR as an appropriate platform for conducting Post-Authorisation Safety Studies (PASS) and Post-Authorisation Efficacy Studies (PAES). This recognition establishes ECFSPR as a trusted source of real-world evidence that directly supports regulatory decision-making related to CF therapies.

The official qualification opinion is available on the EMA website, and it serves as a reference model for other disease registries pursuing EMA qualification.

Role of ECFSPR in Active Pharmacovigilance

The ECFSPR contributes significantly to active pharmacovigilance surveillance in Europe. The analyses represent secondary use of data collected within the ECFSPR and are conducted solely by the ECFSPR team in accordance with the approved study protocol. Individual-level patient data are not shared or disclosed at any stage ensuring compliance with applicable ethical standards and data protection regulations. By analyzing aggregated (pseudonymised) annual data from consenting people with CF across Europe and neighboring countries, the registry enables proactive monitoring of medication safety. This data complements traditional passive surveillance methods and helps fill critical gaps in post-marketing safety reporting, especially important in the context of orphan drugs and rare diseases like CF.

Support for Pharmacoepidemiological Research

ECFSPR data are instrumental in supporting pharmacoepidemiological secondary-use studies. These studies assess the real-world effectiveness and long-term safety of CF therapies, providing valuable insights into treatment patterns, clinical outcomes, and the overall benefit–risk profile of new and existing medicines. By enabling evidence-based evaluations in diverse clinical settings, the registry strengthens the foundation for both regulatory and clinical decision-making.

How to Apply

The primary contact for study requests, is the Director of Pharmacoepidemiological Studies, Prof. Dr. Lutz Naehrlich.

Marketing Authorization Holders (MAHs) seeking collaboration for pharmacoepidemiological studies should send requests to: pmv-coordination@ecfregistry.eu 

Review Process for Multiple Country Study

The formal review period (6-8 weeks depending on the complexity of the study) will commence only after all of the following prerequisites have been fulfilled:

• A fully executed ECFSPR Confidentiality Agreement (CDA), signed by all relevant parties;
• A fully executed ECFSPR Start-Up Agreement (SUA), signed by all relevant parties;
• Submission of a complete, formal, and signed application to the ECFSPR;
• Receipt of payment covering the applicable handling and review fees.

Requests to conduct PASS or PAES are subject to a structured, multi-tiered review process to ensure scientific validity, ethical integrity, and operational feasibility.

1. Initial Scientific and Methodological Review

All study proposals submitted to the ECFSPR are evaluated by the Pharmacoepidemiology Review Committee.

The committee assess the scientific justification, methodological quality, alignment with the objectives of the registry, and the potential impact of the proposed research.

2. Recommendation to the Steering Group

Based on their assessment, the PMV Review Committee issues a formal recommendation to the ECFSPR Steering Group, which comprises national representatives from participating countries. This recommendation advises whether or not participation in the proposed study should proceed.

3. Country-Level Feasibility Assessment

If the recommendation is favorable, the Country Coordinators evaluate the feasibility of implementing the study within their country. Each Country Coordinator must submit a written statement of their decision to ECFSPR within the defined timeframe.

4. Outcome and Resubmission Options

If the study request is approved, a Study Service Agreement will be established between the MAH and the ECFSPR.

If the study request is not approved, the MAH may:

• Submit a revised study request, with a maximum of two resubmissions permitted, provided the modifications are non-structural in nature; or
• Terminate the application process.

In cases where structural revisions to the study design are proposed, the resubmitted proposal shall be considered a new application, subject to a full re-evaluation by the PMV Review Committee.

Single-Country Studies

For studies conducted in a single country, MAHs should contact the respective national authority directly.

Medicine and Real-Life Data: What is Pharmacoepidemiology

Pharmacoepidemiology is the science of studying how medicines are used and how they work in everyday life - not just in clinical trials. It helps make sure that treatments stay safe and effective for everyone who needs them.

Why is this important for people with CF?

After a medicine is approved, doctors and researchers still need to monitor how it works for different people over time. These ongoing studies help to:

• Detect any side effects that might not appear in early trials.
• Understand how the medicine is used in real-world settings.
• Confirm that it continues to help pwCF as expected.
• This kind of research is especially important for rare diseases like CF.

✅ Safer Medicines

✅ Better Understanding of How Medicines Work.

✅More Personalised Care

✅ Improved Treatment Guidelines

The Role of the ECFSPR

ECFSPR collects information of people with CF across Europe. This information helps:

• Track the long-term safety and benefits of CF medicines.
• Improve understanding of treatment patterns and outcomes.
• Support better care, research, and regulatory decisions.

The European Medicines Agency (EMA) officially approved ECFSPR as a reliable source for these important studies.

Examples of studies using ECFSPR Data

• Elexacaftor, Tezacaftor, and Ivacaftor Combination Therapy (ELX/TEZ/IVA) (2018–2024): A study looking at how this combination therapy is used and its effects in real-world settings.
• Lumacaftor/Ivacaftor (2020–2024): A study focused on how the medicine works in children aged 1–5 with CF.

Both studies are funded by the medicine manufacturer, Vertex Pharmaceuticals.