Drug Development Process

The idea for a clinical trial often starts in the lab and requires years of development. As new treatments move through a series of trials called “phases”, researchers learn more information about the treatment, its safety, and its effectiveness. If the drug is eventually approved and made widely available, safety monitoring is continued.

Step 1-Basic Research (average 2-3 years)

Researchers investigate and discover potential new treatments in the laboratory.

Step 2-Preclinical Studies (average 3-5 years)

Researchers investigate the efficacy and safety of the treatment without the use of human subjects.

Step 3-Clinical Trials (average 3-7 years)

Confirmation of efficacy and safety in humans.

  • In Phase I of clinical trials researchers test a treatment in a small group of people, typically healthy adult volunteers, for the first time. The purpose is to learn about safety and side effects.
  • In Phase II researchers study the new medicine or treatment in a larger group of people to further determine safety, effectiveness, and optimal dosage.
  • In Phase III the new treatment is given to a larger group of participants to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments or a placebo, and collect information that will allow the new medicine or treatment to be used safely.

Step 4-New Drug Application & Review (average 1 year)

Filing of an application for approval of the new treatment for the affected community. In the U.S., this is done through the Food and Drug Administration (FDA). Other countries follow a similar development process, though trial results and approvals in other countries may speed up the process in some cases.

Step 5-Broad Usage & Monitoring

In Phase IV, researchers track the safety of the treatment in the general population, seeking more information about the treatment’s benefits and optimal use.

Key Terms Used in Clinical Trials

The main event or outcome that is measured to determine whether the therapy being studied is beneficial.

An outcome is an observable or measurable change that is monitored during treatment for change. A core outcome set (COS) is a group of outcomes measured across multiple trials that reflect measures important to the patient. The ACD has established a Core Outcome Set (COS) for GAMT and CTD trials based on input and consensus from both the GAMT and CTD patient and healthcare/research communities. The COS and more information on the development of the set can be found at creatineinfo.org/cos. It is strongly encouraged that all trials include these important outcomes for comparability between trials and to ensure that the outcomes of most importance to the patient are measured.

When the U.S. Food and Drug Administration (FDA) determines that the benefits of the therapy being tested outweigh the known risks. An FDA approval allows for the intended use of a therapy.

The use of a drug for a purpose other than its approved use, which carries potential unmeasured risks.

The therapy or process that the study focuses on.

The drug being investigated in a clinical study. Investigational drugs are not yet approved.

Inclusion criteria are the key features of the target population that investigators wish to recruit. Exclusion criteria are features of potential participants that could interfere with the success of the study.

Blinding describes the process in which the patient (blind) or both patient and investigator (double blind) do not know if the treatment or a placebo is being given. This prevents bias and allows the collection of scientifically accurate data.

The process by which treatments are assigned to participants by chance rather than by choice, to avoid bias in assigning volunteers to a treatment group.

A document given to participants outlining important information about the study, including its benefits and risks.

 

An administrative body that ensures all clinical studies are ethical and that the rights and welfare of participants are protected, including reviewing the informed consent.

A document that describes the study’s background, objectives, and specific research questions.

The primary individual responsible for the clinical study; also known as the lead researcher.

The entity that funds the study. Sponsors can be pharmaceutical companies, academic medical centers, voluntary groups, and other organizations.

Clinical Trial Frequently Asked Questions

What is a Clinical Trial?

A clinical trial is a research study where human volunteers receive a new treatment — a drug, device, or procedure — to test if the new treatment is safe and effective.

Clinical trials follow a very carefully planned set of procedures described in a document called a protocol. Before patients can be enrolled, the protocol must be reviewed by a panel that includes doctors as well as other members of the community. Any changes must also be reviewed and approved before they can be made.

There may be risks associated with your child’s participation. The new drug, device, or procedure has been tested in the lab and may have been tested in animals. Some side effects may be known but some may be unknown. Each child may react differently, and your child may have to come off usual medication, which carries its own risks. All known risks will be described in the informed consent form provided to you.

Bring any questions to the researcher and your physician. Consider asking about:

The Study

  • What is the purpose of the study?
  • Who will fund it?
  • Who has reviewed and approved it?
  • How long will it last?
  • What are the possible benefits and risks?
  • Will my child be able to take regular medicines during the trial?
  • Will there be costs?
  • What is my insurance likely to cover?
  • Can I withdraw at any time?

Possible benefits and risks

  • What are my possible short-term and long-term benefits?
  • What are my short-term and long-term risks and possible side effects?

Cost issues

  • Will I have to pay for any part of the trial, such as tests or the study medicine?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  •  

Participation and care

  • What kinds of therapies, procedures, and/or tests will I have during the trial?
  • Will I be able to take my regular medicines while taking part in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

There is no guarantee your child will benefit. You may find their condition is better controlled or they experience fewer side effects — but this may not occur. Trials sponsored by pharmaceutical companies often provide free medication and study-related care. Many parents enroll their children because they want to help other children with the same disease or disorder.

Before you decide, you will be told about the study including possible benefits and known risks, what will happen during the trial, how long it will last, and how often you’ll need to visit. Participation is voluntary and you may withdraw at any time without affecting your child’s ongoing medical care.

Yes. Behavioral trials evaluate ways to promote health-improving behavioral changes. Diagnostic trials study tests for diagnosing a disease. Prevention trials look for better ways to prevent a disease. Quality-of-life trials explore ways to improve comfort and quality of life. Screening trials test new ways of detecting diseases. Treatment trials — the focus of this page — test new treatments, drug combinations, or approaches to surgery or radiation.

In single-blind studies, you are not told what you are being given, but the research team knows. In double-blind studies, neither you nor the research team are told — only the pharmacist knows. Randomization means participants are randomly assigned a treatment to avoid bias. If one treatment is found superior during the trial, the study is stopped so all volunteers can receive the better treatment. Participants are eventually informed of which treatment they received.

There is no guarantee a trial will continue. After Phase I and Phase II, researchers decide whether to move on or stop due to safety or ineffectiveness. If a Phase III trial completes and results have medical importance, the treatment may become a new standard of care. Published results are typically available in the National Library of Medicine’s PubMed® database.

A trial may have guidelines restricting participation in an additional trial simultaneously. You may also need to wait through a “washout period” after ending one trial before being eligible for another. This is determined by each study’s design.

Clinical Trials 101 Video

The following video from Children’s Hospital of Philadelphia (CHOP) is a patient and family town hall discussing clinical trials, the vital role patients have played in improving treatments, and the often difficult decision of whether to participate in a trial. The original posting can be found here.

Ultimately YOU decide if a trial is right for you.

Clinical studies are necessary to move treatments forward. Your participation in clinical studies may provide a valuable contribution to new treatment options for both you and future generations affected by Cerebral Creatine Deficiency Syndromes.

The information on this page includes adaptations and excerpts from NIH and CHLA clinical trial resources.