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Natural history study
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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.

Q

Qualified Person for Pharmacovigilance (QPPV)

The Qualified Person for Pharmacovigilance (QPPV) is the individual, appropriately qualified and residing and operating in the EU or EEA, whom every marketing authorisation holder must have permanently and continuously at its disposal to be responsible for the establishment and maintenance of its pharmacovigilance system (Art. 104(3) Directive 2001/83/EC; Art. 8(3)(ia); GVP Module I). The QPPV's name and contact details are notified to the EMA through the Article 57 database and recorded in the Pharmacovigilance System Master File, and a back-up procedure must ensure continuity when the QPPV is absent.

The QPPV has oversight of the entire safety system: the collection, evaluation and reporting of adverse reactions, signal detection, risk management plans, periodic safety update reports, responses to authority requests and inspection readiness. The role may be held by an employee or an external consultant, and one QPPV may serve several authorisation holders, but personal accountability cannot be delegated. Since Brexit the UK requires a separate UK QPPV residing in the UK (or the EU under transitional arrangements), and Switzerland has its own responsible person requirements under Swissmedic rules. Sponsors of clinical trials are not required to appoint a QPPV, but many designate a safety lead with equivalent responsibilities for SUSAR reporting.

From a data protection perspective, the QPPV is the operational owner of some of the most sensitive processing a pharmaceutical company performs: individual case safety reports containing health data of patients and personal data of reporters, processed under legal obligation and public health (Art. 6(1)(c) and 9(2)(i) GDPR). The QPPV should work closely with the Data Protection Officer on the pharmacovigilance chapter of the RoPA, on contracts with safety database vendors and CROs, on transfer mechanisms for global safety data exchange, and on minimisation of identifiers in case narratives. The QPPV is also a named individual whose own contact details are published, and therefore a data subject in the company's records.