Phases of Clinical Development

Clinical Trials for New Treatments

New treatments undergo a rigorous development and review process prior to being available to those living with lupus and as treatment options for providers. The process begins with laboratory research, progresses to preclinical research and then, if previous stages are successful, moves to clinical development and regulatory review (by the Food and Drug Administration (FDA) in the United States). Once approved by the FDA, the treatment will be available to prescribe, and Post-Market Safety Monitoring, monitors all treatments and devices safety. 

The clinical development process involves phases of clinical trials, which can lead to new treatments that can improve the lives of those impacted by diseases like lupus. Clinical trials can also provide an avenue for patients to access treatments while in development. 

There are 4 phases to clinical trial research:

Phase 1

Phase 1 studies involve a small group of participants (20 to 100 healthy participants or people with the disease/condition) to examine the safety, pharmacokinetics (what the body does to the treatment) and pharmacodynamics (how the treatment may be acting on the body) of the new treatment. This is the first time an experimental therapy is given to humans, and generally this phase can last several months. Participants are monitored closely, sometimes even continuously at a clinical trial center. If the treatment is well tolerated and has favorable characteristics during Phase 1 study, the evaluation will proceed to Phase 2.

According to, an estimated 70% of treatments/interventions move to Phase 2.

Phase 2

Phase 2 studies can involve up to several hundred participants living with the condition for which the treatment is intended, to examine efficacy, and confirm dose and side effects of a treatment over the course of several months to up to two years. It is in this phase that researchers focus on determining the most effective dose and the most appropriate method of delivery (e.g., tablets, extended-release capsules, infusions, injections) and patients are monitored carefully with several visits and more stringency for criteria to participate than during Phase 3. If there are favorable results from the Phase 2 study, a treatment or device continues to Phase 3.

According to, an estimated 33% of treatments/interventions move to Phase 3. This transition has been challenging for lupus treatments.

Phase 3

Phase 3 studies examine efficacy and monitoring of adverse reactions among hundreds to thousands of participants, studying effects in different populations, often among individuals with a broader presentation of a disease. This phase can last between one and four years. Much of the information obtained and analyzed during the Phase 3 trial is included in the final product label if/when the experimental treatment is approved by the FDA. At the end of the study period, the sponsor of the study will submit a New Drug Application (an NDA) to FDA for review. This review typically takes months, and then the FDA will render a decision regarding approval. FDA will approve a treatment if the outcome of Phase 3 study is positive from an efficacy standpoint, there are no safety concerns, and there is confidence that the benefit-to-risk profile is favorable.

According to, an estimated 25-30% of treatments/interventions move to Phase 4.

Phase 4

Phase 4 studies occur after a treatment has been approved and generally monitor a treatment’s safety and efficacy in a larger, more diverse population for a longer period of time. They are also known as “post-approval” or “post-marketing” studies. These Phase 4 trials allow researchers to collect additional information about the longer-term risks, benefits, and optimal use of the drug. This information is important since even the most well-designed Phase 3 studies may not address considerations that can become apparent once the treatment is widely used.

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