Click here to open the filter panel
F
Fairness and transparency
F
Federated learning
F
Filing system
F
First-in-class drugs
F
Food and Drug Administration (FDA)
F
Fundamental rights impact assessment (FRIA)
G
General Data Protection Regulation (GDPR)
G
General-purpose AI model (GPAI)
G
Genetically modified organism (GMO)
G
Genetic data
G
Global public health security
G
Good clinical practice (GCP)
H
Health data warehouse (HDW)
H
High-risk AI system
H
HIPAA (Health Insurance Portability and Accountability Act)
H
Human oversight (AI Act)
H
Human subjects protection review board
I
Incapacitated subject
I
Information Commissioner's Office (ICO)
I
Informed consent
Click a letter to view all its definitions
Thank you! Your submission has been received!
Oops! Something went wrong while submitting the form.

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.

D

Data Governance Act (DGA)

The Data Governance Act (DGA), Regulation (EU) 2022/868, is an EU regulation applicable since 24 September 2023 that establishes mechanisms to increase trust in voluntary data sharing and to facilitate the reuse of certain data held by the public sector. It is one of the pillars of the European strategy for data, alongside the Data Act and sector-specific data spaces such as the European Health Data Space.

The DGA has three main components. First, it sets conditions for the reuse of public-sector data protected by third-party rights (commercial confidentiality, statistical confidentiality, intellectual property and personal data), requiring public bodies to use secure processing environments, anonymisation or pseudonymisation, and to establish single information points; this complements the Open Data Directive for data that cannot simply be published. Second, it creates a notification and supervision regime for data intermediation services, neutral platforms that connect data holders and data users without exploiting the data themselves. Third, it introduces "data altruism", allowing individuals and companies to make data available voluntarily for objectives of general interest such as health research, through recognised data altruism organisations and a common European consent form.

For life sciences, the DGA matters where researchers or companies seek access to protected hospital, registry or public health datasets, and where patient organisations or platforms wish to pool data donated by individuals. It provides governance structures but not a legal basis: processing of personal data under DGA mechanisms remains fully subject to the GDPR, including Art. 9 conditions for health data, DPIAs and, where data users are outside the EEA, transfer rules.