About Clinical Trials

Have you ever wondered how new drugs and medical devices are invented?

If not, consider this fact: without people like you and me, there would be no new treatments or cures.

Why?

Because long before a new drug or medical device can be sold, two things must be proven.

That it's safe. And that it's effective.

As it happens, there is only one way to find out.

That's to test it with people who have the medical condition that researchers hope to treat.

This test is called a 'clinical trial'.

We've written this guide so that you can get a better feel for how clinical trials work.

Of course, choosing to join a clinical trial should only be done with a full understanding of what's involved.

That's why you must talk with your physician first.

Together, you can decide if it's the right thing to do.

How clinical trials work

It takes years of research before a drug or medical device can be brought to market.

This time is spent testing how safe and effective it is.

The process starts in a laboratory.

Here, a great deal of work is done to see if the treatment is likely to be safe in humans.

If this testing suggests the proposed treatment may be safe and effective, research may move to the next step.

This involves applying to the FDA for approval to test the drug or medical device in humans.

Once approval has been given, research can move to the next step: clinical trials.

Clinical trials take place in four phases.

The results of each phase must be submitted to the FDA in order for approval to be granted for the next phase to start.

Phase 1

The purpose of a Phase I clinical trial is to see how safe the treatment is.

This involves testing the drug or medical device on humans for the first time.

Normally, the testing involves between 20 and 100 healthy volunteers.

The research team then checks to see how these volunteers' bodies respond.

For example, do they experience any side effects? If so, what are they?

People volunteering for a Phase I trial are usually paid to take part.

These trials normally last for a few months.

About 70% of experimental treatments pass Phase I.

Phase II

The purpose of a Phase II trial is to test how effective the drug or medical device is.

That's why the volunteers usually have the condition that the research team wants to treat.

Lasting between a few months and two years, these trials involve 20 - 300 people.

Normally, these studies are 'randomized'.

That means the people volunteering as patients are split into two groups.

One group receives the potential new treatment. The second group gets a placebo or the current standard treatment.

Meanwhile, these studies are often 'blinded'.

In some trials that means volunteers aren't told if they're receiving the test treatment or the placebo.

In other trials, neither the volunteers nor the researchers are told which patients are receiving the test treatment and which are receiving the placebo.

This blinding ensures the study results aren't influenced by patients' or doctors' knowledge of who has received what.

About 30% of experimental drugs pass Phase I & Phase II.

Phase III

Like Phase II, Phase III tests the drug or device on volunteers who have the condition that the drug or device is seeking to treat.

As with Phase II studies, Phase III studies are normally randomized.

That means one group receives the test treatment and the other group gets a placebo or the standard treatment.

The studies are usually blinded.

A Phase III trial involves many more volunteers than a Phase II.

In fact, there are usually 300 - 3000 (or more) of them.

The idea is to give researchers a better idea of how well the treatment works - and its side effects.

These trials usually last for a few years.

About 70% of drugs that enter a Phase III trial complete this phase of testing.

If the results are good, the research team can now apply to the FDA for approval to start selling the treatment.

Phase IV

Sometimes more trials are done after a drug or medical device has been put on the market.

These Phase IV trials are done for a number of reasons.

For example, a given trial might look at the long-term impact of the treatment on people's quality of life.

Or it might test how effective the new treatment is compared to ones that already exist.

Phase IV trials are also held to see if a treatment has further uses.

For example, does it offer an extra benefit when used with another drug?

These trials can also bring safety problems to light.

This can lead to the treatment being withdrawn from sale. Or having its use restricted.

Who funds clinical trials?

Many organisations fund clinical trials.

These include federal government bodies like the National Institutes of Health, the Department of Defense and the Department of Veteran's Affairs.

Other sources of funding include medical institutions and charitable organisations.

Pharmaceutical, biotechnology and medical device companies also invest heavily.

Why participate in a clinical trial?

There are a number of reasons why people with a medical condition sign up. These include:

• Receiving an experimental new treatment.
• Getting free or reduced cost medical care during the trial.
• Helping researchers find a better treatment - or even a cure - for a given medical condition.

Healthy volunteers usually volunteer because:

• They are typically paid to participate in Phase I trials.
• They wish to help researchers find a better treatment for a given medical condition.
  

Who can volunteer for a clinical trial?

Each trial has guidelines about who can get involved.

These ensure the research team get the most useful results from the trial.

And that potential risks to volunteers are kept to a minimum.

Entry requirements are usually based on:

• Gender
• Age
• The type and stage of disease
• Previous treatment history
• Other medical conditions

Every trial on our website lists these details.

What is informed consent?

To take part in a clinical trial, you must first give your 'informed consent'.

This means you can only take part if you have a full understanding of what volunteering involves.

For example, you must understand:

• The trial's purpose
• The treatment procedures and schedule
• The potential risks and benefits
• Alternatives to participation
• Your rights as a participant

Trial staff discuss each point with anyone thinking about getting involved.

They also provide a document called an 'informed consent form' which talks about each point in detail.

Before being enrolled in a trial, you must first sign this form.

This is to confirm that you fully understand what taking part entails.

However, once you've signed the form, the informed consent process doesn't stop.

In fact, you're encouraged to ask questions and raise concerns throughout the trial.

What's more, the research team will update you on anything that may affect your decision to stay in the trial.

Should you wish, you're free to leave the trial at any time - even after you've signed the informed consent form.

If you leave, you won't lose access to other treatment.

What to ask before volunteering for a clinical trial

Before signing up, it's essential that you have a good understanding of what's involved.

The questions to ask the people running the trial - and your own physician - include:

• What is the purpose of the trial?
• What treatments will be used?
• What are the possible benefits?
• Are there any risks?
• How will patient safety be monitored?
• Where is the trial being conducted?
• How often will I have to attend the trial location?
• How long will the trial last?
• What are the alternative treatments besides the one being tested in the trial?
• Who is sponsoring the trial?
• Do I have to pay for any part of the trial?
• What happens if I am harmed by the trial?
• Can I choose to remain on this treatment, even after termination of the trial?

What are the potential benefits and risks of participating in a clinical trial?

For a volunteer, the potential benefits and risks vary from one trial to the next.

Benefits may include:

• Potentially getting experimental treatments before they're made available to the wider public.
• Getting free or reduced cost medical care during the trial.
• Helping researchers find a better treatment - or even a cure - for a given medical condition.

Risks may include:

• Unpleasant, serious, or even life-threatening side effects
• The experimental treatment may not work.
• More time and general hassle than the standard treatment. For example, there may be trips to the study site, extra treatments or stays in hospital.

For details on the potential benefits and risks of taking part in a clinical trial, ask the team running it.

And discuss the trial with your own physician.

How does the process of applying to participate in a clinical trial work?

The exact process will vary from trial to trial.

Normally, it follows these steps.

Step 1:

If you request details via our website, you'll start by filling out a short online questionnaire.

This will tell you instantly whether you appear to be eligible to get involved.

If you do appear to be eligible, you can then choose to ask for further details from the people running the trial.

Step 2:

A person involved with the trial will phone you to talk about it.

There are two important things they'll want to discuss.

Firstly, whether you're eligible to take part in the trial.

And, secondly, how keen you are to take part.

Step 3:

If it appears that you're eligible and keen to take part, you may be invited to the research site that's running the trial.

There you'll meet with a physician or other healthcare professional to talk about the trial in detail.

This conversation will include topics like:

• The purpose of the trial
• What treatments will be used
• The possible benefits
• The potential risks
• How patient safety will be monitored
• How often volunteers will have to attend the trial location
• How long will the trial last
• Who is sponsoring the trial
• Whether volunteers have to pay for any part of the trial
• What would happen to volunteers harmed by the trial
• Whether volunteers can remain on the treatment, even after termination of the trial

Your medical history may also be looked at to make sure you're eligible for the trial.

You might also get a physical check up.

Finally, you'll be given a document called an informed consent form.

This explains how the trial will work and the possible outcomes for you if you decide to get involved.

You'll need to sign this form to be entered into the trial.

But before you do so, you need to be sure you fully understand everything it contains.

Once you've done this, your participation in the trial can start.

Always remember you have the right to pull out of the trial at any time.

And you don't need to give a reason for doing so.

Nor will it result in you losing access to other treatment.

What happens to the information collected about you in a clinical trial?

The research team can normally see your personal details.

Staff at the other organisations connected with the trial will often see them, too.

These include:

• The sponsor that funds the trial.
• The contract research organisation that coordinates the trial
• The Institutional Review Board that oversees the trial

Before signing up for the trial, you'll be given details of how information about you will be used.

What happens when a clinical trial ends?

At the end of the clinical trial, the research team analyse the data that has been collected.

This will provide information on how safe and effective the treatment is.

Based on the results, they will either decide not to do any more tests - or to move onto the next phase of research.

At the end of a Phase III clinical trial, the researchers decide if the experimental treatment looks hopeful.

If the answer is yes, they may report the results to scientific journals where they can be reviewed by other researchers.

The results may also then be reported to the FDA as part of an application to get approval to start selling the treatment.

If the treatment is approved for sale by the FDA, it may undergo further research in a Phase IV clinical trial.

Where to learn more about clinical trials

Food and Drug Administration: http://www.fda.gov/ForConsumers/byAudience/ForPatientAdvocates/HIVandAIDSActivities/ucm121345.htm#safe

National Institutes of Health: http://www.cc.nih.gov/participate/faqaboutcs.shtml

National Cancer Institute: http://www.cancer.gov/cancertopics/factsheet/Information/clinical-trials