What are the Stages of Clinical Development?

It can take 10 to 20 years to bring a new medicine to patients. Explore the timeline from discovery to distribution, and learn why clinical research is a critical part of the process.

It takes many years to approve a new medication. Clinical development begins in the laboratory before a new medication can be tested in people. Clinical trials are conducted in people to determine the best dose and to assess safety and efficacy. Health authorities such as the US Food & Drug Administration (FDA) and European Medicines Agency (EMA) review all available data to determine if the medication should be approved for use. After a new medication is approved, additional clinical trials and safety assessments may continue per local country requirements. It is common for the process to take 10 to 20 years.

Discovery

New medications are identified from research laboratories based on data from research conducted around the world

Optimization

Chemical and in vitro biological systems, which are tests conducted without animal or human participants, are designed to optimize the intended therapeutic effect

In Vivo Testing

The medications are then tested with in vivo and animal disease models, which are tests conducted in live animals, to assess biological activity

Toxicology Studies

Before testing in human participants can begin, extensive toxicology and drug metabolism studies need to be carried out in many in vitro and in vivo systems according to guidance provided by health authorities or regulatory agencies

Types of clinical trials

Interventional Clinical Trials

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Participants are assigned to receive an intervention according to a detailed research plan. The intervention may be a medication, medical device, or procedure. The goal of the clinical trial is to determine if the intervention is safe and effective. In order to determine if the intervention is safe and effective, the intervention may be compared to a similar product or a placebo (no active ingredient). Throughout the clinical trial, the study doctor collects safety information and other health outcomes for each participant. Study results are prepared after the end of the study to assess the safety information and to evaluate the health outcome measures.

Observational Studies

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Participants may receive an intervention or procedure according to a detailed research plan but they are not assigned to a particular group or intervention by the study doctor.

Expanded Access Studies

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Participants who are not eligible for clinical trials may be able to join an expanded access program, also called compassionate use programs (CUPs), to obtain access to investigational products. Please contact your healthcare provider if you are interested in joining an expanded access/compassionate use program. 

Phases of Clinical Trials

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Phase 1

Phase 1 clinical trials are small-scale clinical trials with very few participants to examine the way the intervention (e.g., medication) interacts with the human body. The purpose of the study is to collect information about the safety of the interventions assessed. These trials are typically conducted in healthy participants without a specific medical condition.

Phase 2

Phase 2 clinical trials are conducted with up to several hundred participants. They are designed to collect additional information about whether the intervention is safe and effective. The results of these types of clinical trials may be used to design future trials or to develop additional research methods.

Phase 3

Phase 3 clinical trials are larger trials typically conducted with up to several thousand participants and are designed to establish if the intervention is safe and effective in preparation for product approval. The results show whether or not the intervention is beneficial to a particular group of participants.

After successful phase 3 studies, all data from all phases including discovery (early testing) and non-clinical studies conducted in animals are submitted to health authorities (e.g., New Drug Application submitted to US FDA) for review for marketing authorization

If the medication is approved, there will be detailed instructions (e.g., the label and prescribing information) created for patients based on the available data

Throughout the process, extensive safety monitoring will be conducted. Prescribing information may be modified during this period based on new data.

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Three doctors comparing notes at the respiratory ward in a hospital.

Phase 4

Phase 4 clinical trials are large-scale trials typically conducted with up to several thousand participants. They are designed to collect additional information after the product has been approved by the relevant health authority. The additional information collected may include efficacy and safety information or information about the product’s risks, benefits, or optimal use.

Why participate in a clinical trial?

You may want to participate in a clinical trial to help collect information about whether a new medication or medical device not yet on the market is safe and effective. You may get more frequent health check-ups while participating in the study. You may get information about support groups or other treatment options for your medical condition.

Clinical Trials Summary Sources

NPS-ALL-NP-01270 MAY 2024