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Term of the Day

Natural history study

A natural history study is a preplanned observational study intended to track the course of a disease over time, identifying demographic, genetic, environmental and other variables that correlate with its development and outcomes in the absence of intervention, or under standard of care. Designs may be retrospective (chart review of existing records) or prospective (longitudinal follow-up of a cohort or registry).

Natural history data is particularly important in rare and paediatric diseases, where randomised placebo-controlled trials may be infeasible or unethical. The FDA (guidance on rare disease natural history studies, 2019) and the EMA accept well-designed natural history studies to define endpoints and biomarkers, identify patient subgroups, estimate sample sizes and, in some cases, serve as external or historical control arms for single-arm trials supporting orphan products.

Because they are non-interventional, natural history studies fall outside the CTR and are governed by national law (for example France's MR-003 or MR-004 reference methodologies) and by the GDPR. They typically involve secondary use of medical records, long-term follow-up, genetic data and small populations in which anonymisation is rarely achievable, so pseudonymisation, a DPIA and a robust research legal basis under Art. 9(2)(j) are essential. Registries maintained by patient organisations or academic consortia raise additional questions of joint controllership and data access governance.

P

Pharmacovigilance

Pharmacovigilance is defined by the World Health Organization as the science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other medicine-related problem. In the EU it is governed by Directive 2001/83/EC and Regulation (EC) No 726/2004 as amended by the 2010 pharmacovigilance package, Implementing Regulation (EU) 520/2012, the EMA's Good Pharmacovigilance Practices (GVP) modules, and, for clinical trials, Chapter VII of the Clinical Trials Regulation. In the United States, 21 CFR 314.80 and 312.32 apply.

Marketing authorisation holders must operate a pharmacovigilance system described in a Pharmacovigilance System Master File, appoint a Qualified Person for Pharmacovigilance (QPPV) residing in the EU, collect and report individual case safety reports of suspected adverse reactions to EudraVigilance within 15 days (serious) or 90 days (non-serious), perform signal detection, submit periodic safety update reports, maintain risk management plans and conduct post-authorisation safety studies. In trials, sponsors report SUSARs and annual safety reports. Activities are frequently outsourced to CROs and specialist providers, and safety databases such as Argus or ArisGlobal host the data.

Pharmacovigilance processes health data of patients and personal data of reporters (healthcare professionals, patients, consumers). The legal basis is compliance with a legal obligation (Art. 6(1)(c) GDPR) combined with the public health exception of Art. 9(2)(i), so consent is neither required nor appropriate, the right to erasure does not apply, and records are retained for the life of the product plus at least 10 years. Data minimisation is nevertheless required: reports should contain only medically relevant information, patient identifiers should be limited to initials, age or age group and sex where national law allows, and reporter identity should be protected. Because safety data flows globally to affiliates, licensing partners and regulators, international transfers must be covered by appropriate safeguards (intra-group BCRs or SCCs) or the public-interest derogation, and safety data exchange agreements between partners should include data protection clauses. Pharmacovigilance is a standard chapter of every pharmaceutical company's RoPA and a frequent subject of iliomad gap analyses.