Department of Health and Human Services

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Bringing Your Voice to Drug and Device Approval and Safety

Frequently Asked Questions

We have tried to answer your questions about drugs and devices, the regulatory and approval process for medical products, and ways we can help make our products safer.

If you cannot find an answer to your question, submit one at Ask the FDA.

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General

What are orphan drugs?

Orphan drugs include two categories of drugs:

  • those that treat, diagnose, or prevent rare diseases and disorders that affect less than 200,000 people in the U.S.; or
  •  those that affect more than 200,000 people but are not expected to recover the costs of developing and marketing the drug. The FDA Office of Orphan Products Development works to identify and evaluate medical products that show promise or rare diseases or conditions.

Where do I find information on want is going on to find treatments for rare diseases?

The FDA Office of Orphan Products Development (OOPD) provides incentives for sponsors to develop products for rare diseases. The office works with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.

Where do I find information on approved drugs for my specific disease or condition?

FDA does not organize drug information according to disease or condition. However, our Drugs@FDA site allows you to search for information about drugs by name. To find out more about treatments available for your specific condition, you may wish to visit MedlinePlus, NIH’s health information web site.

Why does the drug/device development process take so long?

The path a drug travels from a lab to your medicine cabinet is long to ensure their safety. Most drugs and devices go through preclinical (animal) research and clinical (human) research before they can be reviewed by FDA. Read more at Learn How Drugs and Devices Get Approved to better understand why it takes so long.

Investigational Drugs

Why Do Patients Seek Access to Investigational Drugs?

Patients may decide to seek access to investigational drugs for different reasons. Some patients with serious or life-threatening illnesses seek treatment with investigational drugs if FDA-approved therapies are not working or if their side effects are too severe. Others may have heard about promising early study results for a specific investigational drug, and they might want to learn more.

Visit Basics About Clinical Trials.

How Can I Gain Access to Investigational Drugs?

If you’ve considered available FDA-approved treatment options and still want access to an investigational drug, you have two options:

How Is FDA Speeding Up Access to New Therapies?

FDA has begun several programs to shorten the drug approval process and provide patients with access to important new therapies.

Clinical Trials

What are clinical trials?

Clinical trials are research studies in which people volunteer to help find answers to specific health questions. When carefully conducted, they are the safest and fastest way to find new treatments and ways to improve health.

Clinical trials are conducted according to a plan, called a protocol, which describes:

  • the types of patients who may enter the study
  • the schedules of tests and procedures
  • the drugs involved
  • the dosages, or amount of the drug
  • the length of the study
  • what the researchers hope to learn from the study.

Volunteers who participate in the study must agree to the rules and terms outlined in the protocol. Similarly, researchers, doctors, and other health professionals who manage the clinical trials must follow strict rules set by the FDA. These rules make sure that those who agree to participate are treated as safely as possible.

Learn more about the basics of clinical trial participation, read first hand experiences from actual clinical trial volunteers, and see explanations from researchers at the NIH Clinical Research Trials and You Web site.

Why are clinical trials done?

Clinical trials are conducted for many reasons:

  • to determine whether a new drug or device is safe and effective for people to use.
  • to study different ways to use standard treatments or current, approved treatments so that they will be more effective, easier to use, or decrease certain side effects.
  • to learn how to safely use a treatment in a population for which the treatment was not previously tested, such as children.

Who should consider clinical trials and why?

Some people participate in clinical trials because none of the standard (approved) treatment options have worked, or they are unable to tolerate certain side effects. Clinical trials provide another option when standard therapy has failed. Others participate in trials because they want to contribute to the advancement of medical knowledge.

All clinical trials have guidelines, called eligibility criteria, about who can participate. The criteria are based on such factors as age, sex, type and stage of disease, previous treatment history, and other medical conditions. This helps to reduce the variation within the study and to ensure that the researchers will be able to answer the questions they plan to study. Therefore, not everyone who applies for a clinical trial will be accepted.

It is important to test drugs and medical products in the people they are meant to help. It is also important to conduct research in a variety of people, because different people may respond differently to treatments.  FDA seeks to ensure that people of different ages, races, ethnic groups, and genders are included in clinical trials. Learn more about FDA’s efforts to increase diversity in clinical trials.

Where are clinical trials conducted?

Clinical trials can be sponsored by organizations (such as a pharmaceutical company), Federal offices and agencies (such as the National Institutes of Health or the U.S. Department of Veterans Affairs), or individuals (such as doctors or health care providers). The sponsor determines the location(s) of the trials, which are usually conducted at universities, medical centers, clinics, hospitals, and other Federally or industry-funded research sites.

Are clinical trials safe?

FDA works to protect participants in clinical trials and to ensure that people have reliable information before deciding whether to join a clinical trial. The Federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control the risks to participants, some may be unavoidable because we are still learning more about the medical treatments in the study.

The government requires researchers to give prospective participants complete and accurate information about what will happen during the trial. Before joining a particular study, you will be given an informed consent document that describes your rights as a participant, as well as details about the study, including potential risks. Signing it indicates that you understand that the trial is research and that you may leave at any time. The informed consent is part of the process that makes sure you understand the known risks associated with the study.

What should I think about before joining a clinical trial?

Before joining a clinical trial, it is important to learn as much as possible. Discuss your questions and concerns with members of the health care team conducting the trial. Also, discuss the trial with your health care provider to determine whether or not the trial is a good option based on your current treatment. Be sure you understand:

  • what happens during the trial
  • the type of health care you will receive
  • any related costs once you are enrolled in the trial
  • the benefits and risks associated with participating. 

What is FDA’s role in approving new drugs and medical treatments?

FDA makes sure medical treatments are safe and effective for people to use. We do not develop new therapies or conduct clinical trials. Rather, we oversee the people who do. FDA staff meet with researchers and perform inspections of clinical trial study sites to protect the rights of patients and to verify the quality and integrity of the data.

Learn more about the Drug Development Process.

Where can I find clinical trials?

One good way to find out if there are any clinical trials that might help you is to ask your doctor. Other sources of information include:

  • FDA Clinical Trials Search. Search a database of Federally and privately supported studies available through clinicaltrials.gov. Learn about each trial’s purpose, who can participate, locations, and who to contact for more information.
  • Clinicaltrials.gov. Conduct more advanced searches
  • National Cancer Institute or call 1–800–4–CANCER (1–800–422–6237). Learn about clinical trials for people with cancer.
  • AIDS Clinical Trials and Information Services (ACTIS) or call 1–800–TRIALS–A (1–800–874–2572). Locate clinical trials for people with HIV.
  • AIDSinfo. Search a database of HIV/AIDS trials, sponsored by the National Institutes of Health’s National Library of Medicine.

What is a placebo and how is it related to clinical trials?

A placebo is a pill, liquid, or powder that has no treatment value. It is often called a sugar pill. In clinical trials, experimental drugs are often compared with placebos to evaluate the treatment’s effectiveness.

Is there a chance I might get a placebo?

In clinical trials that include placebos, quite often neither patients nor their doctors know who is receiving the placebo and how is being treated with the experimental drug. Many cancer clinical trials, as well as trials for other serious and life-threatening conditions, do not include placebo control groups. In these cases, all participants receive the experimental drug. Ask the trial coordinator whether there is a chance you may get a placebo rather than the experimental drug. Then, talk with your doctor about what is best for you.

How do I find out what Phase a drug is in as part of the clinical trial?

Talk to the clinical trial coordinator to find out which phase the clinical trial is in. Learn more about the different clinical trial phases and whether they are right for you.

What happens to drugs that don't make it out of clinical trials?

Most drugs that undergo preclinical (animal) research never even make it to human testing and review by the FDA. The drug developers go back to begin the development process using what they learned during with their preclinical research. Learn more about drug development.

What are clinical trials?

Clinical trials are research studies in which people volunteer to help find answers to specific health questions. When carefully conducted, they are the safest and fastest way to find new treatments and ways to improve health.

Clinical trials are conducted according to a plan, called a protocol, which describes:

  • the types of patients who may enter the study
  • the schedules of tests and procedures
  • the drugs involved
  • the dosages, or amount of the drug
  • the length of the study
  • what the researchers hope to learn from the study.

Volunteers who participate in the study must agree to the rules and terms outlined in the protocol. Similarly, researchers, doctors, and other health professionals who manage the clinical trials must follow strict rules set by the FDA. These rules make sure that those who agree to participate are treated as safely as possible.

Learn more about the basics of clinical trial participation, read first hand experiences from actual clinical trial volunteers, and see explanations from researchers at the NIH Clinical Research Trials and You Web site.

Expanded Access

How Can I Get Expanded Access to an Investigational Drug?

The process must begin with your healthcare provider, who should follow these steps:

  • Your healthcare provider must contact the company that manufactures the drug to make sure it is willing to provide the drug.
  • Your healthcare provider must submit an Investigational New Drug (IND) [link to section below] application to the appropriate FDA review division.
  • In an emergency situation, the request to use the drug may be made via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the FDA official over the telephone. With emergency INDs, shipment of and treatment with the drug may begin prior to FDA’s receipt of the written IND submission that is to follow the initial request.
  • In a nonemergency situation, the IND must be received by FDA before shipment of and treatment with the drug may begin. These nonemergency requests are known as individual patient INDs.

What Is the IND Application?

The IND application is required to gain access to an investigational drug outside a clinical trial. The application should include the following information.

  1. Statement that this is a request for an individual patient IND for treatment use (specifying whether it is an emergency IND or individual patient IND), which should be included at the top of the correspondence and on the mailing cover.
  2. Brief clinical history of the patient including:
    • Diagnosis
    • Disease status
    • Prior therapy
    • Response to prior therapy
    • Rationale for requesting the proposed treatment, including a list of available therapeutic options that would ordinarily be tried before the investigational drug, or an explanation of why use of the investigational drug is preferable to the use of available therapeutic options
    • Reference for a published protocol or journal article, if appropriate
  3. Proposed Treatment Plan including:
    • Dose (how much and how often)
    • Route of administration (by mouth, injection, etc.)
    • Planned duration (how long the product is to be taken)
    • Monitoring procedures
    • Modifications (e.g., dose reduction or treatment delay) for toxicity
  4. Chemistry, Manufacturing, and Controls Information and Pharmacology and Toxicology Information, including a description of the manufacturing facility. This can be done by providing a letter of authorization (LOA) that refers to this information if it has been previously submitted to FDA (for example, to an existing IND or new drug application). The treating physician should contact the sponsor of the previously submitted information for the authorization and letter. The LOA should include identifying information, such as the sponsor’s application (e.g., IND) number.
  5. Informed Consent Statement noting that informed consent and approval by an appropriate institutional review board (IRB) will be obtained prior to beginning treatment. In the case of an emergency, treatment may begin without prior IRB approval, provided the IRB is notified of the emergency treatment within 5 working days of treatment.
  6. Investigator Qualification Statement that specifies the training, experience, and licensure of the treating physician. The first two pages of a curriculum vitae typically contain this information and are usually sufficient.
  7. FDA Form 1571 completed with the treating physician listed as the sponsor. Download Form 1571 and view the instructions.

Can Any Individual Patient Qualify for Expanded Access to Any Drug?

No, to gain access to an investigational drug outside of a clinical trial:

  • You must have a serious or immediately life-threatening disease or condition and no treatment options available to you.
  • The drug maker and your healthcare provider must make special arrangements to obtain the drug for you through FDA approval. These safeguards are in place to avoid exposing patients to unnecessary risks.

If I Meet These Criteria, Will I Qualify for Expanded Access to a Drug?

Not necessarily. Even if you meet these criteria, there may still be obstacles.

  • Your healthcare provider may not be able to seek expanded access for you because of your medical history or the risks associated with taking an investigational drug. Your healthcare provider must determine that the probable risk from the drug is not greater than the probable risk from the disease.
  • Your healthcare provider may not be willing to manage the use of an investigational drug.
  • The company that makes the drug is not required to offer it outside their clinical trials, and they may not be willing or able to do so.
  • The company may not have enough of the drug available for all patients requesting expanded access. Some companies establish a lottery system to determine which patients will have treatment access. Others make the decision on a case-by-case basis.

The FDA released a draft guidance for Industry "Expanded Access to Investigational Drugs For Treatment Use - Qs & As" in May 2013

Are Costs Associated With Expanded Access?

Investigational drugs are expensive to make. It is important to know that:

  • Some companies provide the drug for free to patients.
  • Some companies charge patients costs based upon how much the drug costs to make.
  • Most insurance companies will not pay for access to an investigational drug.
  • There also may be additional costs for administration and monitoring of the investigational drug by your healthcare provider.

The FDA released a draft guidance "Charging for Investigational Drugs Under an IND - Qs & A's" on May 2013

Am I Protected From Risks?

When a drug is investigational, Federal law requires that its use be reviewed by an institutional review board (IRB). The IRB works to protect the people receiving the drug and to ensure that the risks are reasonable given the potential benefit. However, significant unknown risks may exist. The IRB will require and review an informed consent document to make sure patients are aware of potential risks and are willing to accept the level of possible risk associated with the drug.

How Can I Find Out Whether I Can Access a Particular Investigational Drug Outside of a Clinical Trial?

Some drug companies have established expanded access programs. To find them:

  • Use our clinical trials search tool or visit ClinicalTrials.gov to view a list of expanded access studies.
  • Search for specific expanded access programs through an online search engine.
  • Call a drug company directly to ask about their policies.
  • Contact patient advocacy groups to see if they have information on expanded access options.
  • Talk with your healthcare provider about your options.
  • Contact us, the FDA Office of Health and Constituent Affairs, for information and assistance at OSHI@fda.hhs.gov.

Physicians can visit Physician Request for an Individual Patient Investigational New Drug Application (IND) for Compassionate or Emergency Use.

Information about expanded access to unapproved medical devices explains emergency use, single patient or small group access, treatment use, and continued access after a controlled clinical trial under an Investigational Device Exemption has been completed.

What Are my Responsibilities if I Choose To Seek Access to an Unapproved Drug Outside of a Clinical Trial?

You can do several things:

  • Talk with your healthcare provider to see whether use of an investigational drug for your treatment is right for you. Be sure to consider how much is known about the investigational drug, the severity of your condition, and the likelihood that the therapy will be effective. You and your healthcare provider should consider the kind of illness you have, the stage of disease, other conditions you may be experiencing, and organ function (e.g., liver and kidney function), among other factors.
  • Be sure your healthcare provider is willing to oversee therapy with a drug s/he is not familiar with, and to work with the company and FDA to obtain the drug, monitor you, and file necessary paperwork.
  • Consider the cost of the investigational drugs and the medical services associated with its use that are not covered by third-party payers such as insurance or Medicare.
  • Carefully read the informed consent presented to you and make sure that you understand the risks associated with the drug before signing. Federal law requires that this form also be signed and reviewed by an IRB before access to any investigational drug can be obtained. Your healthcare provider may or may not have access to an IRB, which is usually associated with a hospital or research institution, but there are independent IRBs as well. Depending on the IRB, a cost may be associated with the review, which may be your responsibility.

Patient Representatives

Who Can Be a Patient Representative?

Patient Representatives usually have a history of the disease being discussed; they are the “voice” for patients’ concerns. In some cases, a caregiver or a member of a patient advocacy organization might be selected.

Patient Representatives can take part in one or both of the following ways:

  1. during private discussions between FDA and a company that is creating a new product,
  2. as a participant in an FDA Advisory Committee meeting reviewing a medical product or public health policy.

Learn about the Patient Representative Program.

Do Patient Representatives Need a Scientific Background?

No, a background as a scientist or researcher is not necessary. However, general knowledge of the disease and the ability to consider complex information is helpful for understanding and discussing the scientific data. Most importantly, experience as a patient helps Patient Representatives bring a unique and important perspective to FDA.

What Does an FDA Advisory Committee Do?

Advisory Committees provide expert advice to help FDA make good decisions about new drugs, vaccines, medical devices, and other public health issues. Committee members typically vote on questions FDA poses to the committee. The meetings are usually held in the Washington, DC, area in an open session with an audience, including members of the public and the media. Learn more about FDA Advisory Committees.

Do Patient Representatives Vote When Serving on an Advisory Committee?

Patient Representatives who serve on Advisory Committees that review drug and biologic therapies are usually voting members. Patient Representatives who serve on Advisory Committees that review medical device therapies are non-voting members. Other than voting on the questions FDA poses to the committee, non-voting members have the same rights as other committee members.

How Does a Patient Representative Take Part in an FDA Advisory Committee meeting?

Patient Representatives are notified about an upcoming Advisory Committee meeting and given plenty of time to make arrangements to attend. The meetings are held on 1 to 2 days in the Washington, DC, area. The Patient Representative participates in person. FDA pays a salary for the time spent in meetings and covers travel expenses, such as lodging and meals, if the meeting is more than 50 miles from the Patient Representative’s home.

Who Are the Members of an FDA Advisory Committee?

Committees have experts in the appropriate scientific fields (e.g., medicine, engineering, statistics, or other sciences), as well as consumer, industry, and Patient Representatives.

How Does a Patient Representative Take Part in an FDA Drug Development Meeting?

Generally, a Patient Representative will participate via telephone in meetings with FDA and the company that is creating a new product. FDA pays a salary for the time spent in meetings.

How Does a Patient Representative Prepare for an Advisory Committee or Drug Development Meeting?

It is important for the Patient Representative to have background information on the medical product being reviewed. About 2 to 3 weeks before the meeting, the Patient Representative will receive a meeting package that contains all information necessary to get ready for the meeting. After the Patient Representative reads the information, FDA staff can answer any questions by telephone.