A Guide to Phase I Clinical Trials

Introduction

Most of you who are already involved or familiar with clinical research are probably aware that there are different phases in conducting a clinical study: Phase I, Phase II, Phase III and Phase IV. Another thing you might be familiar with is that Phase I studies can be particularly hard to get, especially for new research sites.

How different are Phase I studies in practice?

To answer this question and many more, we’ve decided to clarify some common misconceptions regarding Phase I trials. We will help you understand what it means to conduct a Phase I trial and how profitable these trials will be for your site.

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What is a Phase I Clinical Trial?

After the drug has been designed, discovered, and then been through animal testing (if applicable), the Phase I of the trial involving human subject begins.

Phase I entails testing of the drug/treatment on a small number of healthy volunteers (participants), in

order to determine its proper dosage, safety, and eventual side effects.

Usually, Phase I trials will require anywhere from 20 to 100 healthy volunteers who will be given the IP (investigational product) in order to determine the maximum dosage that can be received without showing any side effects. This dosage (the maximum one that doesn’t cause any intolerable effects) will then be called ‘’maximum tolerated dose’’ or MTD.

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What is a Phase I Clinical Trial?

Apart from determining a safe dosage, Phase I clinical trials are also designed to see how the IP is going to be absorbed, metabolized, and excreted by human subjects/participants. Around 70% of IPs should pass the Phase I and move on to Phase II. However, only 10 to 15% of the IPs that passed Phase I actually manage to make it all the way to the market.

Even though most Phase I trials require exceptionally healthy volunteers, there are some studies that might use terminally ill patients affected with cancer or HIV. Such patients are used as volunteers in cases when the investigational drug has high chances of making otherwise healthy individuals ill.

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Phases

100% 70% … ...Market

10 to 15%

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Phase I Studies and the FDA

In order to be able to begin a Phase I trial, the sponsor first has to submit an IND (investigational new drug) application to the FDA, containing the details of all previously gathered data from animal studies and cellular models.

After the FDA approves, Phase I can be started.

According to the FDA:

“Under current regulations, any use in the United States of a drug product not previously authorized for marketing in the United States first requires submission of an IND to the FDA. Current regulations at 21 CFR 312.22 and 312.23 contain the general principles underlying the IND submission and the general requirements for an IND's content and format. “

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A lot of staff (can dose more patients)

Well-equipped

Enrolls more patients

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Common misconception: Phase I studies are hard to get

Most people involved in the clinical research business have the opinion that Phase I studies are extremely hard to get. However, we’ll see that this is not always true.

The main reason why new and/or smaller sites might have a problem acquiring a Phase I study is because these studies are usually performed by bigger clinical research facilities in this industry.

Advantages of Big Research Centers

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How can smaller research sites acquire Phase I trials?

The number one strategy that clinical research sites can use in order to get more Phase I trials is to look into the more specific population groups of prospective patients.

For example, some good ideas for such targeted population can be renal impairment patients, DDI studies (drug-drug interaction), diabetes studies, etc.

These are the types of studies that you, as a smaller and/or newer research facility, should place a bid for. The more specific, narrower, and smaller your target population of patients is, the easier it can be to get a Phase I study for which you’re able to find those patients.

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How profitable are Phase I studies?

Even though they might be harder to acquire, Phase I trials are the most

profitable type of research.

The clinical research business is lucrative and profitable no matter what type of studies a site is performing, but Phase I trials are the most beneficial ones. As a matter of fact, many physicians/PIs have actually closed their medical practices once they started conducting Phase I trials because they saw that they can make much more profits with this.

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Which therapeutic areas to choose for a Phase I trial?

When it comes to Phase I clinical trials, the best way to choose a therapeutic area is based on your PI’s specialty.

In this way, your site will have better chances of getting the study because you’ll be seen as a site that can enroll patients for that area from your PI’s private practice. For example, if your doctor/PI is specialized in GI, then you should try to find GI studies. However, if you have a general medicine doctor as your PI, then you’ll be able to apply to many more studies. With a general medicine PI, you will be able to bid on diabetes studies, cardiovascular studies, endocrine studies, etc.

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PI specialty

Study Types9

Which therapeutic areas to choose for a Phase I trial?

When it comes to applying, Phase I studies are the same as any others. You have to find the study and then place your bid.

One of the best ways to find a Phase I study is to go on www.clinicaltrials.gov and try to find a study that’s struggling or not doing well. In such cases, the study sponsor will usually be desperate to enroll patients. If you place a bid on a study like this, you have much greater chances of acquiring it.

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Find a struggling study, bid for it, and make it better!

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How to enroll patients for Phase I trials?

Once you’ve acquired a Phase I study, you will be expected to enroll the necessary number of patients. For this purpose, you will be given a certain recruitment budget.

There are many recruitment strategies that can be used, but as a general rule, you should first use the ones that cost the least money. Such recruitment methods are social media, ads, word of mouth, and finally - through your PI’s office. If your recruitment budget is small, try to stay away from expensive recruitment methods such as TV commercials. All things considered, the best way to recruit patients that fit your study is through your own PI’s database of patients.

Word of mouth, social media

Ads, PI office

TV commercials

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CONCLUSION

As a final note, we can assure you that Phase I clinical trials are not so hard to get with the right strategy. Furthermore, it’s a great idea to try and get a Phase I study since they are more profitable than studies from Phases two to four. In order to increase your chances of a Phase I study, look into some more narrow and specific patient populations, and try to find a struggling study. Then, jump in on this opportunity and show the sponsor that you’re able to save and successfully conduct their Phase I trial.

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