What is an investigational drug?

Have you ever thought about joining a clinical trial that is trying to find out if an investigational drug works in treating your disease or medical condition? Or maybe your healthcare provider has talked to you about treating you with an investigational drug through expanded access.

An investigational drug can also be called an experimental drug and is being studied to see if your disease or medical condition improves while taking it. Scientists are trying to prove in clinical trials:

• If the drug is safe and effective.

• How the drug might be used in that disease.

• How much of the drug is needed.

• Information about the potential benefits and risks of taking the drug.

When to consider using an investigational drug

Not every person’s disease or medical condition responds the same way to approved drugs. Your healthcare provider might have talked to you about using an investigational drug if you have:

• Experienced side effects that are too severe to continue taking

• Limited treatment options available.

• Heard about promising early study results for a specific investigational drug.

• No approved drugs available to treat your disease or medical condition.

Questions you may want to consider

If your healthcare provider thinks that using an investigational drug is an option for you, then consider asking questions like those listed below before deciding whether it is right for you.

• What is this drug being studied for?

• Is there a clinical trial site near you?

• How much is already known about the investigational drug?

  • What basis is there for thinking that the drug will work better for your medical condition than using an approved treatment?

  • What scientific evidence is available to support the use of this investigational drug?

  • What are the potential risks of using this investigational drug?

• Are there other drugs that are already approved to treat your disease or condition?

  • Have you already tried them?

  • Why or why not?

Many of these questions may be answered for you when you read the informed consent document. Before you can be given an investigational drug either through a clinical trial or through expanded access, your healthcare provider must give you additional information about the potential risks and potential benefits of the drug.

As promising as an investigational drug may sound. It is still being tested in clinical trials to determine if it can be used to treat a disease or medical condition. And not everyone who wants to enroll in a clinical trial will be able to participate. Clinical trials have strict inclusion and exclusion criteria about who can participate. The criteria can be based on such factors as:

• Having a certain type or stage of disease.

• Having received (or not received) a certain kind of therapy in the past.

• Being in a certain age group.

• Your medical history.

• Your current health status.

Criteria like those listed above can help researchers understand the medical differences among people in the trial and help them answer the questions they are studying about the drug.

Finally, remember that approved drugs have completed extensive testing in clinical trials and there is scientific proof that they are safe and effective for treating the particular disease or medical condition that has been studied. It is important to talk with your healthcare provider on a regular basis about the medicines you are taking and talking about the side effects you may be experiencing.

According to a survey by the Centers for Disease Control and Prevention (CDC) conducted in 2008, at that time almost 50% of Americans had used at least one prescription drug in the past month. A higher percentage of Americans, however, had used over-the-counter medications and dietary and herbal supplements at least once in that time frame. Pharmacology provides the studies to determine the safety and effectiveness of these medications.

Studies of Side Effects and Effectiveness in Different Populations

While the safety of medications is important for everyone, certain groups are at particular risk of adverse reactions. Researchers are studying the reasons behind drug reactions and looking for ways to personalize treatment according to people’s genetic make-up. Among the groups at greater risk for medication problems are the following:

Seniors

Seniors often take multiple medications that may interact with each other and lessen the effects of the drugs or, worse, cause serious harm. Pharmacology and pharmacogenetics investigate how these drugs interact with each other in the body to help health care providers choose the right medication and the right dosage for patients. For more information on medication use and seniors, visit the NIH National Institute on Aging's Senior Health page on medication safety.

Pregnant Women

According to the CDC, one-half of pregnant women take up to four medications daily. Many medications may be given to pregnant women to manage their health conditions or to help the fetus develop. However, very few medications and supplements have been tested on pregnant women to determine the effectiveness and safety of these agents. A lack of testing can be harmful, however, as certain medications, for example, medications to treat acne and some dietary and herbal supplements can cause preterm labor or severe problems in fetal development or long-term development of the infant.

Infants and Children

According to the U.S. Food and Drug Administration (FDA), most drugs prescribed for children have not been tested on that age group. Until the early 2000s, only about 20% of drugs approved by the FDA were labeled for use in children. Health care providers often had to prescribe medication for children without the benefit of research and government regulations to back up their dosages and prescriptions. Because of this, children were often given a smaller dose of a medication for adults. Since 2002, as a result of the Best Pharmaceuticals for Children Act, the NICHD has been identifying drugs and supporting studies that contribute to pediatric labeling and improved medication safety and effectiveness in children.

Certain Races and Ethnicities

Particular drugs can have different effects in different groups of people. It is important that health care providers consider their patients’ racial and ethnic backgrounds when prescribing drugs. Here are a few examples of different effects of drugs based on race/ethnicity:

• A cholesterol-lowering drug called rosuvastatin (Crestor®) has much more powerful effects in Asians than in other groups. One-half the standard dose seems to effectively lower cholesterol levels in Asians, and full doses can increase their risk of heart damage.
• Whites are more likely than Asians to have abnormally low levels of an enzyme called cytochrome p450 2D6, which is important for metabolizing different drugs. Having low levels of the enzyme can put people at risk for accidental overdoses of medications.
• African Americans tend to respond poorly to several classes of blood pressure medicines, including beta-blockers, ACE inhibitors, and angiotensin II agonists.

Research to Improve Drug Safety Testing

Research is also under way to find better and faster ways to test drugs for safety and effectiveness. For example, NIH scientists are working with researchers in the Department of Defense and the FDA to develop “tissue chips,” miniature systems made with living human cells and tissues. In the future, the tissue chips might provide a way to reduce animal testing, make sure that the results of drug tests reflect the drugs’ actions in human beings, and get new drugs approved more quickly.

Participating in Drug Safety Testing

By taking part in a clinical trial, you might be able to assist with learning more about a new drug and whether it is better than those that already exist.

A clinical trial is a biomedical or behavioral research study of human subjects that is designed to answer specific questions about biomedical or behavioral interventions (such as drugs; treatments; devices; or new ways of using known drugs, treatments, or devices). Learn more about clinical trials, including definitions of clinical trials, outcomes of clinical trials, and how to get more information.

Each clinical study has its own criteria for who can participate. These are called eligibility criteria or inclusion criteria. These might be based on age, sex, health status or type of disease, medical history, and other factors. Some studies are open only to participants with certain health conditions, and others recruit healthy volunteers. Doctors and other health care providers run the clinical trials according to strict rules set by the FDA and other organizations to make sure that the trials are done safely and ethically.

You can find out about clinical trials that you might be eligible for by speaking with your health care provider or by checking online at www.clinicaltrials.gov.

The U.S. Food and Drug Administration (FDA) is the federal agency responsible for labeling medications and supplements. However, the approval process is different for prescription and for over-the-counter medications.

Prescription Drugs

The FDA must regulate and approve new prescription drugs before they can be sold to the public. In 2011, the last year for which information is available, the FDA approved 35 new drugs for use by the general public. Although this number may seem small, the approval process for drugs is extensive.

The manufacturer or sponsor follows a series of important steps, through animal studies and clinical trials in humans, to ensure that the medication is safe, does what it claims to do, and will provide a health benefit. Testing is performed first in laboratories and on animals to determine that the drug is safe and try to understand how the drug might work in humans.

"Clinical trial" is the scientific term for a test or study of a drug or medical device in people. These tests are done to see if the product is safe and effective for people to use.

Sometimes, multiple trials are completed to measure the effectiveness of a particular medication. Participants in the studies are often followed over a period of time. For comparison purposes, some are given a placebo, an inactive or "dummy" medication, while others are given the actual drug. The NICHD and other institutes of the NIH help conduct and support the research but are not involved in the approval or labeling process.

The clinical trial phase can take years to complete. However, once research has shown that the drug is safe and useful, the FDA typically reviews and either approves or denies an application for a new drug within 6 months.

Over-the-Counter Drugs, Dietary and Herbal Supplements

Over-the-counter drugs and dietary and herbal supplements are not formally approved through the FDA. The active ingredients and labeling of medications and supplements in certain classes (like antacids, for example) are reviewed by the FDA to ensure that acceptable ingredients are used and that they are safe. Drug companies must conform to the requirements in the medication or supplement class, but they do not need formal FDA approval to make and sell over-the-counter medications and supplements.

The label will indicate whether a drug or supplement has been FDA approved. Talk to your health care provider about any medications you are currently taking or are considering taking to ensure that they are safe.

Generic Drugs

A generic drug is a medicine that is contains the same active pharmaceutical ingredient as a name-brand drug but is not sold under a brand name. FDA-approved generic drugs meet the same strict standards for safety, efficacy, and quality as name-brand drugs.

Consumers usually can buy generic drugs at a much lower price than brand-name drugs. Generic versions are not available for every medicine, and in some cases may not be able to be substituted for a brand name product.

When a pharmaceutical company develops a new drug, it is protected under a patent, usually for 20 years. During this time, no other company is allowed to make or sell it. Once the patent expires, that company or another company can make and test another version of the drug. After testing and FDA approval, the company can sell the drug as a generic.

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when there are no comparable or satisfactory therapies available.

Investigational medical products are investigational drugs, biologics or medical devices that are being tested to see whether they are safe and effective. This means that these products have not yet been approved or cleared by the FDA and the FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause serious side effects that were not expected.

When seeking expanded access to an investigational medical product, it is critical that you and your licensed physician (typically your doctor, but not all doctors or healthcare providers are licensed physicians) consider all possible risks.

Whenever possible, an investigational medical product should be used as part of a clinical trial. However, there are times when it is not possible for a patient to be a part of a clinical trial (e.g., that there are no ongoing trials, a patient may not have access to a clinical trial or may not be eligible for the clinical trials, distance to get to a trial prevents access). In those circumstances, expanded access may be the only way for a patient to receive an investigational medical product.

FDA is committed to increasing awareness about the expanded access process and the procedures for getting access to investigational medical products. It is important to note that, even if you meet the criteria under the law and FDA regulations, the licensed physician, the Institutional Review Board (IRB), and the company all need to agree that expanded access is appropriate for you in order for you to receive the investigational medical product. In addition, there may be costs not covered by third-party payers such as private insurance or Medicare.

The Right to Try Act is one way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain investigational treatment options. FDA’s role in implementing the Right to Try Act is limited to receiving and posting certain information submitted to the agency. 

For patients with serious or immediately life-threatening diseases or conditions, the FDA remains committed to enhancing access to promising investigational medicines for those unable to access investigational medical products through clinical trials. This is the mission of our expanded access program. The agency is dedicated to these purposes, and it has been for more than three decades.

The Right to Try Act permits/allows eligible patients to have access to eligible investigational drugs.

A patient who is eligible for Right to Try is a patient who has:

• Been diagnosed with a life-threatening disease or condition
• Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
• Provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician

An eligible investigational drug is an investigational drug:

• For which a Phase 1 clinical trial has been completed
• That has not been approved or licensed by the FDA for any use
• For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA
• Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA

If you are interested in Right to Try, you should discuss this pathway with your doctor. Companies who develop and make drugs and biologics can provide information about whether their product is considered an eligible investigational drug and whether they are able to provide the product under the Right to Try Act. Ultimately, companies developing drugs for life-threatening diseases or conditions are responsible for determining whether to make their products available to patients who qualify for access under the law.

Right to Try FAQs

1. If I’m a patient or a physician and I have a question about the Right to Try Act, who do I ask?

A: FDA recommends that patients first consult with their physician and that physicians consult with the sponsor of the investigational drug or biological product.  The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act. 

2. If I identify a drug that I would like to use under the Right to Try Act, how do I know if it is an “eligible investigational drug”?

A: FDA recommends that physicians consult with the sponsor of the investigational drug or biological product. The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act. 

3. Does FDA review or approve the Right to Try Act requests?

A: FDA does not review or approve requests for Right to Try Act use. FDA’s role is limited to receipt and posting of certain information submitted under the Right to Try Act. 

4. What does FDA do with the annual summaries submitted under the Right to Try Act?

A: FDA will receive annual summaries from manufacturers or sponsors on use of an eligible investigational drug under the Right to Try Act. FDA will post a consolidated annual summary report of Right to Try Act use. 

5. Does an IRB review and/or approve Right to Try Act requests?

A: Individual Right to Try Act requests do not require IRB review or approval; however, eligible investigational drugs under the Right to Try Act must meet certain criteria.

6. Is written informed consent required for the Right to Try Act?

A: Yes, a physician is responsible for getting written informed consent from the eligible patient or their legally authorized representative. 

7. Does the Right to Try Act obligate a sponsor to provide an eligible investigational drug to an eligible patient?

A: No, the Right to Try Act does not require a sponsor provide an eligible investigational drug to an eligible patient.

8. If the IND for a drug is on clinical hold, is the drug an eligible investigational drug for use under the Right to Try Act?

A: No, a drug under IND clinical hold is not an eligible investigational drug and a sponsor cannot provide the drug for use under the Right to Try Act.

9. I am a sponsor or manufacturer who has provided an eligible investigational drug under the Right to Try Act.  What are my annual reporting responsibilities?

A: The FDA has published a final rule that specifies the deadline and content for submission of an annual report of eligible investigational drugs supplied under the Right to Try Act. Mandatory reporting includes:

1. The name of the eligible investigational drug
2. The IND (Investigational New Drug) number
3. The number of doses supplied
4. The number of patients treated
5. The disease or condition that the drug is intended to treat; and
6. Information on serious adverse events

10. I am a sponsor or manufacturer who has provided an eligible investigational drug under the Right to Try Act. How should I submit my annual report?

A: A Right to Try annual reporting form is available.  Submit annual reports to RTTAnnualReports@fda.hhs.gov by March 31 annually as detailed in the final rule. 

An experimental drug is one that has been tested in the lab and with animals and approved for testing in people by the U.S. Food and Drug Administration (FDA). But, such a drug can’t yet be advertised, sold, or prescribed. Experimental drugs may also be called “investigational drugs.”

Ways to Access an Experimental Drug

Experimental drugs may be available through clinical trials, expanded access, or right to try.

Clinical Trials

The most common and preferred way to access an experimental drug is through a clinical trial. Clinical trials are research studies that involve people. Trials are carefully designed to answer scientific questions about the new drug. People who take part are fully informed and carefully monitored. The data that are collected during a clinical trial help researchers learn how the drug behaves in people, if it is safe and effective, or how it compares to standard treatments.

See Clinical Trials Information for Patients and Caregivers to learn more about clinical trials and how to find one for which you may be eligible.

Expanded Access (Compassionate Use)

Expanded access, which is also called compassionate use, is the use of an experimental drug outside of clinical trials to treat people with serious or life-threatening diseases. It is regulated by the FDA. If you meet certain criteria, you might be able to obtain an experimental drug through expanded access. To qualify, you must:

• have an illness that is life-threatening
• have no standard treatments available
• not be eligible for a clinical trial

How to Obtain a Drug through Expanded Access

The only way to obtain a drug through expanded access is with the help of your doctor. He or she will need to:

• Ask the company that is developing and testing the experimental drug if it is willing to provide it for you. The company may not be willing to provide the drug if it believes that doing so is against its interests or if there isn’t enough drug available.
• Apply to the FDA, if the drug company agrees to supply the drug. Your doctor should refer to Expanded Access: Information for Physicians on the FDA site for complete information.

How Your Safety Is Protected if You Obtain a Drug through Expanded Access

Several measures help protect your safety if you are treated with a drug through expanded access:

• An Institutional Review Board or its representative must approve the use.
• The FDA must approve the use.
• You will go through an informed consent process to ensure that you understand the potential risks and benefits of taking the experimental drug.

Right to Try

Right to try, like expanded access, allows the use of an experimental drug outside of clinical trials to treat people with serious or life-threatening diseases. But unlike expanded access, it is not regulated by the FDA.

If you meet certain criteria, you might be able to obtain an experimental drug through right to try. These criteria include:

• You have an illness that is life-threatening.
• There are no standard treatments available for you.
• You are not eligible for a clinical trial of the drug.

The drug that you are seeking must meet certain criteria, as well. These criteria include:

• It has already been studied in a phase 1 clinical trial.
• It has not been approved by the FDA for any use.
• The company that owns it has applied for FDA approval or is running a clinical trial to further study the drug before seeking approval.

How to Obtain a Drug through Right to Try

The only way to obtain a drug through right to try is with the help of your doctor. He or she will need to consult with the company that is developing and testing the experimental drug to find out:

• if the drug is eligible for use through right to try
• if the company is willing to provide it, which it is not obligated to do

How Your Safety Is Protected if You Obtain a Drug through Right to Try

Before you are treated with a drug through right to try, you will go through an informed consent process to ensure that you understand the potential risks and benefits of taking the experimental drug.

Deciding on Treatment with an Experimental Drug

Your doctor is in the best position to help you understand all your treatment options.

As you think about your options, keep in mind that there may be unknown serious side effects with experimental drugs. Also, cost may be an issue. Federal law requires most insurance plans to cover routine patient care costs related to taking part in a clinical trial. But your insurance may not cover the cost of an experimental treatment received outside of a trial.

Because some medications can harm a developing fetus, it is important to tell your health care provider about all of the medications you are taking, including herbal and dietary supplements. Even ibuprofen or aspirin can cause problems in pregnancy, particularly during the last 3 months.

Many women take medications to treat health problems that first appeared during pregnancy, like diabetes, asthma, heartburn, or morning sickness. Other women take medications to treat conditions they had before they became pregnant. Often, your health care provider will encourage you to continue taking your medication. However, in some cases, a safer alternative may be available.

All medications can have adverse, or unpleasant, effects. These can include side effects of the drug, interactions with other medications or food, and allergic reactions. You can ask your pharmacist or health care provider about the side effects of any medication you may be taking. In addition, known side effects are listed on the drug label or the package insert.

To help avoid an adverse effect to medication:

• Read the medicine label carefully. Learn about over-the-counter drug labeling and see an example label.
• Take the medication as prescribed by your health care provider or as the drug label instructs. Do not take a higher dose than the label says, and continue taking the medication as long as directed.
• Take the medication exactly as the label says. For example, some labels say to take the drug with food or plenty of water.
• Tell your health care provider about any over-the-counter or prescription medicine you may be taking, including herbal or dietary supplements.