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Clinical development

Drug

From molecule to medicine: a 10 to 15 year journey, from candidate screening through to post-marketing surveillance. Clinical research is its core — four phases, each answering a distinct question: tolerability, dose, efficacy, then real-world use.

Medicinal products

Any substance presented as having curative or preventive properties, or intended to restore, correct or modify physiological functions: chemical molecules, biological medicines (antibodies, insulins, recombinant proteins), vaccines, generics and biosimilars, blood-derived medicines, radiopharmaceuticals. They follow phases I to IV through to marketing authorisation (MA).

Advanced therapy medicinal products

ATMPs — gene therapy, cell therapy, tissue engineering, or their combinations with a device — are biological medicines derived from genes, cells or tissues, often developed for rare diseases or refractory cancers. They follow the same path in a compressed version: few eligible patients, phases I/II often combined, mandatory European MA and patient follow-up that can last 15 years.

GMO medicinal products

A medicine is a genetically modified organism (GMO) when it contains or consists of an organism whose genetic material has been altered — as is the case for most gene therapies (viral vectors, CAR-T) and some live recombinant vaccines. This status does not change the trial phases, but adds a dedicated assessment of the risk to health and the environment (containment, release), with specific authorisations and requirements of their own for investigator sites.

DRUG · VACCINE · BIOLOGIC — 10 TO 15 YEARS OVERALL Preclinical Phase I Phase II Phase III MA Phase IV screening of thousandsof molecules · in vitro, in vivo safety and tolerabilitydozens of healthy volunteers efficacy and optimal dosea few hundred patients large-scale confirmationthousands of patients, multicentre marketingauthorisation · EMA / ANSM pharmacovigilance, real worldafter launch ATMP — COMPRESSED PHASES Preclinical Combined phase I / II Phase III MA · EMA Long-term follow-up + GMO dossier /human tissue safety and first efficacy signalsin a single trial · few eligible patients limited numbersvs standard of care mandatory centralisedCAT committee cell-to-patient traceabilityup to 15 years
Top lane: the conventional drug, vaccine or biologic. Bottom lane: advanced therapies, compressed phases and long-term follow-up.

Regulatory frameworks

  • WorldwideDeclaration of Helsinki · ICH E6(R3) good clinical practice, applicable in Europe since July 2025 · phases I-IV and CTD dossier harmonised by ICH · public trial registration (WHO principle) · in the United States: IND application to the FDA, ethics review by IRBs
  • European UnionRegulation (EU) No 536/2014, applicable since January 2022 · single submission through the CTIS portal, coordinated assessment between Member States · centralised marketing authorisation (European Commission on EMA opinion) or national · ATMPs: Regulation (EC) No 1394/2007, marketing authorisation necessarily centralised with an opinion from the Committee for Advanced Therapies (CAT) · GMOs: Directives 2001/18/EC (deliberate release) and 2009/41/EC (contained use) — environmental risk assessment alongside the clinical trial dossier
  • FranceLoi Jardé — RIPH 1 · double green light: ANSM authorisation + favourable opinion from an ethics committee (CPP), through CTIS · written consent mandatory · GMOs: contained-use or deliberate-release application to the ministry in charge of research, on the opinion of ANSES (successor to the Haut Conseil des biotechnologies since 2022) — GMO approval of investigator sites · ATMPs: human-tissue authorisations depending on the product
  • BelgiumLaw of 7 May 2017 on clinical trials of medicinal products for human use (implementing Regulation 536/2014) · joint assessment by the FAMHP and an accredited ethics committee, the FAMHP being the national contact point · written consent
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