What is a clinical trial?

A clinical trial is a medical research study to determine the safety and effectiveness of a drug or treatment and discover any side effects. Clinical trials use humans; pre-clinical trials use animals.

The drug or treatment under study may be:
• New and never been tested on people before
• An existing one being used in a new way

In some clinical trials, a new drug or treatment may be compared with the best-known standard therapy to see if it is more effective or causes fewer side effects.

Information from clinical trials:
• Helps regulatory authorities decide whether to approve a drug for use in their countries
• Guides health professionals in decisions about prescribing drugs or other treatments to patients
• Assists consumers with the proper use of over-the-counter and prescription medications

Types of Clinical Trials

A new drug or other medical treatment is tested in humans only after it has shown promise as an effective, safe treatment in laboratory and animal studies. Testing in humans involves several phases of study in clinical trials.

Phase I
Phase I studies are undertaken mainly to assess a drug’s safety.

Goals:
• Observe how a drug is absorbed, metabolized and excreted by the body
• Identify the most common side effects of the drug
• Investigate whether the side effects increase as dosage levels increase
• Determine the maximum safe dosage

Testing population: Small group of healthy volunteers (20-100)

Duration: 6 months to 1 year


Phase II
Once a drug is shown to be safe, it can be tested for effectiveness in treating the intended illness or condition.

Goal:
• Determine efficacy

Testing population: Up to several hundred people who suffer from the targeted condition

Duration: Up to two years

Phase II studies are often
• Controlled
o One group of patients is given the drug under investigation
o A second group, the control group, is given either the standard drug for the condition or a placebo

• Blind
o Neither the patients nor the researchers know which group is receiving the drug under study

Results from the two groups are compared to determine efficacy of the drug under study.

Phase III
Goal:
• More thorough understanding of the drug’s effectiveness and side effects

Testing population: Thousands of patients

Duration: Several years

Late Phase III and Phase IV
These are also known as post-marketing studies.

Goals:
• Compare a new drug with drugs already on the market
• Study long-term effectiveness
• Determine cost-effectiveness relative to other therapies

Why are clinical trials important?

Finding new treatments

Clinical trials help everyone who has, or may someday have, a particular illness or condition by serving as a testing ground for potentially helpful treatments.

If a medication shows promise and is approved by the FDA for widespread use, then everyone in the country who is affected by that illness or condition stands to benefit.
 
Providing access to pre-market treatments

Clinical trials give trial participants an opportunity to receive pre-market treatments that could improve their condition or help restore their health.

There is no guarantee that the treatment being tested will help them — indeed, in blind studies, there is no guarantee that they will even be receiving the treatment under study — but if a new treatment does prove effective, trial participants will be among the first to benefit.

What is informed consent?

Your patients must be given all the facts about a study before consenting to participate, including:
• Details about treatment and tests
• Possible benefits and risks

Process

At the beginning of a trial, the patient signs an informed consent form, which presents key facts about the study. The informed consent process continues throughout the study.

For example, the patient may be informed of new findings about the treatment under study that could affect the decision to participate. At that point, the patient may need to sign a new consent form.

Giving informed consent does not obligate the patient to complete a study; patients retain the right to leave at any time.

How are your patients protected in medical research?

All clinical research studies are reviewed by an Institutional Review Board (IRB) or Ethics Committee, made up of experts and lay persons.

An IRB/Ethics Committee consists of at least five people who meet to review research plans for proposed studies.

This committee
• Has the authority to review, approve or modify these protocols for research
• May review and approve the informed consent form that research volunteers are asked to sign
• May review the research as it progresses.

The review ensures that:
• Patient risk is minimized
• Informed consent is obtained
• Risks are reasonable in proportion to potential benefits
• There are provisions to protect patient privacy
• Additional safeguards are in place to protect the rights of particularly vulnerable populations, such as women, children, prisoners, those with mental disabilities, or those who are economically or educationally disadvantaged.

Regulatory agency policy includes other patient protections, particularly in the financial and legal arenas.