Click here to open the filter panel
#
510(k) premarket notification
A
Accountability
A
Accuracy
A
Adequacy decision
A
Ad hoc services
A
Adverse event
A
AI literacy (Art. 4 EU AI Act)
A
AI Officer
A
Analysis Data Model (ADaM)
A
Anonymisation
A
Appropriate safeguards
A
Article 29 Working Party (WP29)
A
Artificial intelligence (AI)
A
Authorised representative (EU AI Act)
A
Automated individual decision-making
A
Auxiliary medicinal product
B
Best-in-class drugs
B
Binding corporate rules (BCR)
B
Biobank
B
Biometric data
B
Biotechnology
B
Broad consent
B
Business associate agreement (BAA)
C
Central imaging
C
Central laboratory
C
Chemistry, Manufacturing and Controls (CMC)
C
Clinical outcome assessment (COA)
C
Clinical research
C
Clinical study
C
Clinical trial
C
Clinical trial agreement (CTA)
C
Clinical trial phases
C
Clinical Trials Information System (CTIS)
C
Clinical trial site
C
Clinical Trials Regulation (CTR) 536/2014
C
Clinician-reported outcome (ClinRO)
C
CNIL (Commission nationale de l'informatique et des libertés)
C
Codes of conduct
C
Consent
C
Contract research organisation (CRO)
C
Controller
C
Cookies
C
Cross-border processing
D
Data Act
D
Data concerning health
D
Data Governance Act (DGA)
D
Data management plan (DMP)
D
Data minimisation
D
Data monitoring committee (DMC)
D
Data processing agreement (DPA)
D
Data protection authority (DPA)
D
Data protection by default
D
Data protection by design
D
Data Protection Impact Assessment (DPIA)
D
Data Protection Officer (DPO)
D
Data Protection Representative (DPR)
D
Data quality
D
Data retention
D
Data subject
D
Data subject access request (DSAR)
D
Decentralised clinical trial (DCT)
D
De-identification (HIPAA)
D
Differential privacy
E
Early termination of a clinical trial
E
Electronic case report form (eCRF)
E
Electronic clinical outcome assessment (eCOA)
E
Electronic data capture (EDC)
E
Electronic health record (EHR)
E
Electronic informed consent (eConsent)
E
Encryption
E
End of a clinical trial
E
Enterprise
E
Ethics committee
E
EU Artificial Intelligence Act (AI Act)
E
European Data Protection Board (EDPB)
E
European Data Protection Supervisor (EDPS)
E
European Health Data Space (EHDS)
E
European Medicines Agency (EMA)
E
EU-US Data Privacy Framework (DPF)
E
Explicit consent
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.

C

Clinical trial phases

Clinical trial phases are the successive stages through which a new medicine is tested in humans, each with distinct objectives, participant numbers and risk profiles.

  • Phase 0 (exploratory) involves micro-dosing in a handful of volunteers to assess pharmacokinetics before formal development.
  • Phase I tests safety, tolerability and pharmacokinetics in 20 to 100 healthy volunteers or patients, often in dedicated units.
  • Phase II evaluates efficacy and dose in 100 to 300 patients with the target condition.
  • Phase III confirms efficacy and monitors adverse reactions in 300 to 3,000 or more patients, usually in randomised, controlled, multinational trials, and supports the marketing authorisation application.
  • Phase IV (post-authorisation) studies long-term safety and effectiveness in routine use, often through observational designs and real-world data.

The data protection profile changes with each phase. Phase I units hold detailed identifying data on healthy volunteers, including payment records and screening databases shared across units to prevent over-volunteering. Phase III trials involve dozens of countries, multiple CROs and vendors, and complex international transfers. Phase IV and post-marketing studies rely on secondary use of records and registries, where the legal basis and national research rules need particular attention.