Drug Development

Therapeutic Pipeline*

* Other therapeutics may be under development.

Discovery to Therapy

Please click below to watch a video from Novartis that explains the Discovery of Therapy.

Video Credit: Novartis

 

Clinical Trial Phases

The idea for a clinical trial often starts in a laboratory. After researchers test new treatments or procedures in the lab and in animals, the most promising treatments are moved into clinical trials to find out how well they work in humans. Clinical trials follow a very strict plan known as a protocol. The protocol must address risks and benefits to trial participants as well as answer specific research questions. Sections of the protocol include:

  • The goal of the study

  • Who can be in the trial

  • How participants are protected from risks

  • What information do the researchers want

  • Details about tests, procedures, and treatments

  • How long the trial will last

Study sponsors send the protocol to an Institutional Review Board (IRB) for approval. The IRB consists of doctors, researchers, patient advocates, data analysts, etc. and their job is to make sure the proposed trial passes legal and ethical standards. No trial takes place without approval from an IRB.

Once approved, the principal investigator and research team recruit trial participants and begin their study. Most clinical trials belong in a certain “phase” that dictates how many people can be in the trial and the purpose of the trial. While you may join a phase I trial, it’s more likely that you will join during phase II or phase III.

 
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Phase I

An experimental treatment is given to a small number of participants (20-80). Research staff want to identify the drug/device/treatment’s most common and serious side effects. This phase focuses on safety and how the team can adjust the treatment to make it better for the participant.

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Phase II

Information learned in Phase I is applied to the treatment in Phase II. The focus then turns to the specific research question, disease, or condition being tested (100-300 people).

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Phase III

The experimental treatment, drug, or device gets compared to the standard treatment or product. The investigation explores variables like age, dosage amount, race, health status, etc., to identify areas of success or failure and confirm its effectiveness (1,000-3,000 people).

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Phase IV

The drug or treatment must receive approval from the FDA. Once approved, the drug becomes available to the public and researchers monitor its safety in the general population. Data about the benefits, best use, or issues related to the treatment are collected and analyzed by the research team.

 

After a clinical trial ends, the researchers carefully review information collected during the study. The results drive the decision to continue or stop the clinical trial. For example, after a phase I or phase II study, the researchers may choose to stop the process because the new treatment was unsafe or did not help the patient. On the other hand, they may move on to phase III because results from the first two rounds were so promising.

Once a new drug/device/treatment is proven safe and effective in a clinical trial, it may become a new standard of medical practice.

 

Measurements in Clinical Trials

All clinical trials choose an "endpoint" for their study. An endpoint is the primary outcome being measured in the clinical trial; it’s how they identify success. General examples of an endpoint include relief of symptoms, improvement in quality of life, and survival.

Researchers classify endpoints as either clinical or surrogate.

Clinical endpoints measure how much a patient benefited from the experimental drug or treatment. Did pain levels decrease? Did movement increase? Did the tumor get smaller?

Surrogate endpoints are a laboratory measure or physical sign used as a substitute when a clinical endpoint might not be possible or practical. Example – blood pressure and cholesterol levels are measured during a trial about stroke prevention. Those endpoints predict how likely the person is to have a stroke in the future. The clinical endpoint, did the person have a stroke, may not happen for 10 years.

The Angelman Biomarkers and Outcome Measures Alliance (A-BOM) is an alliance formed by the Angelman Syndrome Foundation and the Foundation for Angleman Syndrome Therapeutics, together with researchers and pharmaceutical corporations to help move new treatments to the clinical trial phase. There are many medications and treatments for Angelman syndrome that are coming close to being ready for clinical trials. There is a need for rigorous ways to measure if these treatments improve the quality of life for patients and families. Alliance members work together to share in data, research, trial design, and stories to help people with Angelman Syndrome.