Drug development and
preclinical research

• Long before a new drug is tested in a clinical trial, the manufacturer makes strategic decisions on the conditions and drug types to focus on. Hundreds of compounds are screened to identify a handful of promising ones, which are then thoroughly characterized in in-vitro and in-vivo studies to ensure basic safety.

Clinical study design

• Clinical trials are an essential step to get marketing approval for a drug. Each trial phase is carefully designed to answer questions about the safety and efficacy of the candidate compound in humans. The exact specifications of a trial are documented in the study protocol which has to be followed during trial execution to ensure both credible and reliable data is collected.

Site selection, preparation, investigator and staff training

• A clinical study is conducted in one or more sites in which participants will be seen by study investigators. Sites need to be experienced in treating the condition, have good access to eligible participants and have the resources to support a clinical study.

Patient recruitment, enrollment, and informed consent

• Individual study sites identify candidate participants from their patient pool, through local outreach activities and/or awareness campaigns with patient advocacy groups. Prior to inclusion in the study, each candidate has to be evaluated against the study's eligibility criteria, and give their informed consent.

Data collection, monitoring and quality assurance

• Investigators collect data about the treatment as defined in the study protocol and monitor participants for eventual adverse events. The data is collected centrally. Where a data monitoring committee is in place, interim analyses of the data might be performed to ensure it is still scientifically and ethically justified to continue the study.

Regulatory submissions

• Both to proceed to the next phase of clinical studies and to obtain marketing authorization, extensive documentation has to be submitted to the regulator (e.g. FDA in the US, EMA in the EU, etc.). The documentation must include results from all previous studies along with additional supporting information.

Post-market surveillance

• Once the drug is on the market, a Phase IV study monitors the safety and efficacy of the drug in a clinical setting to improve knowledge about its safety profile, characterize its effects in population sub-groups which have not previously been studied (e.g. children, immunocompromised patients, etc.) and potentially discover additional effects.