A main role of the U.S. Food and Drug Administration (FDA) is to make sure that all prescription and over-the-counter drugs in the United States are safe and effective. This includes drugs that are used in cancer care.
Drug development and approval is often lengthy. There are efforts underway at the FDA to make the process more efficient. In January 2017, the FDA created the Oncology Center of Excellence (OCE). This is the first center at the FDA to focus on a group of diseases rather than specific types of drug treatments by building review teams across various FDA centers.
The OCE works closely with experts from FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) to review research on new drugs and biologics for cancer. This research helps the FDA decide whether a drug or biologic should be approved for use. Biologics are drugs that are made up of chemical ingredients and biological products that are made in or contain living organisms. Biologics include vaccines and products that come from blood, genes, proteins, and tissues.
How does the FDA encourage drug development?
For many doctors and patients, the development and approval of new cancer treatments is not fast enough. Efforts at the FDA to make the process more efficient include:
Meeting with drug sponsors early in the drug development process. This helps researchers design clinical trials in the best possible way. It also helps to improve the review of the data from these studies.
Working with drug regulatory agencies in Europe, Japan, Canada, Australia, and others to streamline the approval of drugs in the United States and other countries. The International Conference of Harmonization (ICH) is an international effort among drug regulatory agencies and the drug development industry. It was created to make sure that the details required for drug approval are similar between countries.
Using the Orphan Drug Act. The Orphan Drug Act is a U.S. law that provides incentives for sponsors to develop new drugs that treat rare diseases. This includes many types of cancer. "Orphan drugs" are therapies that treat rare diseases or conditions that affect fewer than 200,000 people in the country. The FDA Office for Orphan Drug Development funds grants for these orphan drugs and works with patients and researchers alike to develop them.
How is the FDA trying to speed up cancer drug approval?
The FDA has several programs to make the drug approval process more efficient, with more details provided below on each:
What is fast track drug approval?
"Fast track" helps the FDA speed up the review of drugs for serious or life-threatening diseases that have no treatment. This can include cancer. Drug makers may request fast track designation. They work with the FDA before submitting a New Drug Application (NDA) to avoid issues that could delay FDA review and approval. Most drugs that are suitable for fast track designation may also apply for priority review (see below). Learn more on FDA's website about fast track drug approval.
What is breakthrough therapy designation?
Breakthrough therapy designation can also speed the approval of cancer drugs. In early research, a drug maker must show that a drug significantly improves outcomes compared to other available treatments. Sometimes the FDA suggests this designation based on early data. Breakthrough therapy designation helps a company meet with the FDA earlier in the testing process to speed up the development and review. Learn more on the FDA's website about breakthrough therapy designation.
What is accelerated approval?
Accelerated approval is another program to speed drug approval for diseases like cancer. It lets drug makers request an approval based on positive, interim study results. For example, a drug might shrink tumors in early research.
After FDA's approval, the drug maker must continue clinical trials to show that these early results are linked with improved survival. If these studies do not confirm the earlier-detected benefits, the approval for that indication will be withdrawn. Learn more on the FDA's website about accelerated approval.
What is priority review?
Priority review lessens the time allowed for the FDA to review a drug application. The drug must offer major advances in treatment, safety of a therapy, or advantages for new groups of people who the drug had not previously been studied in.
With priority review, the FDA sets a goal of taking action on the application within 6 months instead of within the average 10 months it takes for standard review. A drug maker can only apply for priority review after submitting an NDA. Learn more on the FDA's website about priority review.
Are investigational cancer drugs available?
Drugs that are not yet approved by the FDA and are undergoing research are called investigational drugs. These drugs are often only given to people who choose to and are eligible to participate in a clinical trial.
The FDA may approve a drug after researchers complete the clinical trials and analyze the data. It is approved if the benefit of taking the drug is shown to be greater than the risk of possible side effects.
Due to a growing demand in the 1970s and 1980s, patients who had tried all of the existing treatments and were willing to accept a higher level of risk urged the FDA for access to experimental treatments. In response, the FDA has created programs to provide people with cancer and other serious illnesses with access to investigational drugs through the Expanded Access or "Compassionate Use" program and individual patient requests.
The best way to gain access to investigational drugs is to join a clinical trial. Clinical trials have certain rules called “eligibility criteria” that help structure the research and keep patients safe. Learn more about eligibility criteria in a separate article on this same website.
However, federal laws permit drug makers to charge for investigational drugs as part of a clinical trial. Patients and their doctors should talk with the drug maker about potential costs before getting the drug. Most insurance companies will not reimburse the doctor or patient for these charges, but often times the drug makers cover these costs for participants in the clinical trial. Learn more on the FDA's website about these federal laws.
Programs for investigational cancer drugs
Outside of clinical trials, the programs that offer access to investigational drugs include:
Expanded access or compassionate use
Individual patient investigational new drug (IND) requests
Right to Try
Expanded access or compassionate use allows access to investigational drugs through drug makers. If no expanded access program currently exists for a particular investigational drug, you or your doctor may submit an individual patient request for the drug. In this instance, your doctor works with the drug company to try to get the drug. The doctor then submits a request to the FDA, which reviews requests on a case-by-case basis. Learn more about expanded access on the FDA's website.
You can also visit the Expanded Access Navigator on a separate website that provides additional information, a list of expanded access policies and criteria, and downloadable forms.
The FDA's Individual Patient Investigational New Drug (IND) application allows doctors to request access to a new drug for a person. In this instance, the person is not eligible to join any clinical trials studying the drug and there are no other treatments available. There must be enough data to show that the drug may be effective and has no unreasonable risks.
Additionally, in May 2018, the U.S. Congress passed legislation commonly known as “Right to Try.” The law aims to speed up patient access to investigational treatments.
Unlike the expanded access program, Right to Try does not require review or approval by the FDA of a patient's application to use an investigational drug under this particular pathway. The law also does not provide important patient protections, such as guidance for doctors on appropriate dosing and managing side effects. Insurance companies are not required to cover the cost of the drugs, and this law does not require drug companies to make investigational drugs or treatments available. The FDA’s role in the Right to Try pathway is limited to drug makers having to submit annual reports to the FDA, including any serious adverse events, when they participate in a right to try request. Learn more on the FDA's website about Right to Try.
What is off-label use?
When the FDA approves a new drug, it only approves the drug to treat 1 or more specific conditions when given in the manner described on the drug's label.
Drug makers may still do more research on other uses for the drug, such as treating another type of cancer. A doctor may prescribe an FDA-approved drug to treat a condition not listed on the label or use it in a different way from what is listed on the label, but supported by a credible body of published scientific data. This is called an off-label use of the drug.
Off-label drug use in cancer treatment is common for many reasons. First, the FDA often approves drugs for treating only a certain type or stage of cancer. The label only reflects past research when the drug received FDA approval. After approval, researchers may find that it is an effective treatment for other types or stages of cancer. Second, many cancer treatments involve a combination of multiple drugs. In such instances, 1 or more of the drugs may be used in an off-label way. Multiple drug therapies are also constantly changing as doctors study new combinations to improve patient care.
Medicare and other types of health insurance plans provide coverage for certain off-label uses if data on the effectiveness is available. Learn more about off-label drug use from the National Cancer Institute and from the FDA.
New uses for existing drugs
A drug maker must submit an application to the FDA for approval of new uses or indications for an already-approved drug. This is called a Supplemental New Drug Application (sNDA). A sNDA establishes the safety and effectiveness of the product for the new use. In some cases, due to existing data, the research data required to submit another request may not be as extensive as the one for initial approval.
How can I get involved?
One of the best ways for people to get involved in the drug development process is to enroll in a clinical trial. Cancer clinical trials help researchers better understand new drugs and provide patients with additional treatment options.
The FDA has a program to include patients in the review and approval process, called the FDA's Patient Representative Program. It allows a person with cancer to participate in FDA’s public advisory committee meetings as a representative. A representative provides insight on issues, problems, and questions important to patients and family members.
There are other ways to get involved in cancer research. Ask at your local cancer center, talk with your doctor, or reach out to a patient advocacy organization to learn more.
Drug Discovery and Development
How Patient Advocates Help Cancer Research: An Expert Q&A
What to Know About Getting Access to New Cancer Advances Presented at Scientific Meetings
Why I Work to Bridge the Gap Between People With Cancer and Researchers
Don't Be Fooled: How to Protect Yourself From Cancer Treatment Fraud
U.S. Food and Drug Agency: FDA Basics
FDA's Orphan Drug Act
FDA's Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review
FDA's Learn About Expanded Access and Other Treatment Options