A well-designed observational study is harder to design than an interventional trial. In an interventional study, you control the exposure. In an observational study, you are describing what happened in routine practice, and the design choices you make determine whether your findings are interpretable, defensible, and usable.

Every major design decision – the study population definition, the comparator, the index date, the follow-up window, the primary endpoint, the confounding adjustment strategy – is a methodological commitment that will be scrutinised by regulators, HTA bodies, peer reviewers, and competitor statisticians. If the design is not grounded in data reality from the start, the findings will not survive that scrutiny.

APICES has been designing and executing observational studies for real-world evidence and health economics since the Kappa Santé team was founded in 2003. The combined organisation brings over two decades of experience designing studies built around available data sources – not around what would be ideal if the data existed.

How APICES Approaches Observational Study Design

Observational study design at APICES begins with the research question and the intended use of the evidence. A study designed to support a PASS under EU pharmacovigilance requirements has different design constraints than a study designed to support AMNOG or HAS evaluation. A drug utilisation study serving a label expansion application has different requirements than a comparative effectiveness study for a payer formulary submission.

We work with sponsors to define the primary research questions, the population of interest, the exposures or interventions to be studied, the comparators, and the endpoints – in that sequence. Design decisions are then evaluated against the available data sources to ensure they can actually be answered. This is the critical step: testing design feasibility before the study begins, not discovering data limitations during analysis.

Methodological frameworks applied at APICES include the ENCePP Code of Conduct, ISPE Good Pharmacoepidemiology Practices (GPP), and EMA PASS guidance. Where studies will be submitted as part of an MAA or post-approval commitment, GRACE and STROBE reporting standards are applied.

Study Types Covered

• Retrospective cohort studies using national claims, EHR, or registry data
• Prospective non-interventional studies (NIS) with primary data collection at clinical sites
• Post-Authorization Safety Studies (PASS) for EMA risk management plan obligations
• Drug utilisation studies (DUS) for regulatory and access purposes
• Patient registries, including long-term safety registries for ATMPs and biologics
• Real-world performance studies for medical devices under MDR Annex XIV
• Comparative effectiveness studies for HTA submissions (AMNOG, HAS, NICE, AIFA)

Alignment with Data Sourcing and Analytics

Observational study design at APICES is always developed in close alignment with data sourcing and governance. The team that assesses data source suitability is the same team that designs the study. Design assumptions – variable availability, coding completeness, population size estimates, follow-up data density – are tested against real data access before the protocol is finalised.

This prevents the most common failure mode in observational research: a scientifically rigorous design that cannot be implemented because the data source does not support the required variables, population, or follow-up structure.

What You Can Expect

• A protocol aligned with ENCePP, ISPE GPP, and applicable EMA/FDA guidance
• Design feasibility assessment against actual data sources before protocol finalisation
• Study registration on the EU PAS Register where required
• Statistical analysis plan developed in parallel with the study protocol
• Design appropriate for the specific regulatory or access use case
• Integrated delivery from design through data sourcing, analytics, and reporting

Frequently Asked Questions

What types of observational studies does APICES design and run?

Retrospective cohort and case-control studies, prospective non-interventional studies, registries, drug-utilisation studies, post-authorization safety studies (PASS), and real-world performance studies for medical devices.

Are studies aligned with ENCePP and PASS guidance?

Yes. Designs are aligned with ENCePP Code of Conduct, ISPE GPP, and EMA PASS guidance where applicable. Study protocols are EU PAS Register-ready.

Can you source real-world data for me?

Yes. We work with European claims, EHR, hospital, registry, and prescription data sources, and we coordinate licensing, governance, and ethics. Data-source recommendations are part of the feasibility memo.

Do you handle HEOR alongside RWE?

Yes. Cost-of-illness, budget-impact, cost-effectiveness, and indirect treatment comparison are part of our HEOR practice, with senior health economists embedded in study teams.

How is APICES different now that Kappa Santé is integrated?

Kappa Santé brought 25+ years of European real-world data and HEOR experience, particularly in France. The combined team designs, sources, executes, and reports without sub-contracting the data work.