Drug Approval and Labeling

Aprobado por la Junta Editorial de Cancer.Net, 07/2018

A primary 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 made up of chemical ingredients and biological products that are made in or contain living organisms. Biological products may also be called "biologics" and include vaccines and products that come from blood, genes, proteins, and tissues.

In January 2017, the FDA created the Oncology Center of Excellence (OCE) as the first center to focus on a group of diseases rather than specific types of drugs or treatments. The OCE works closely with experts from the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research to review research on new drugs and biologics. This research helps the FDA decide whether a drug or biologic should be approved for use.

Improving the process

Drug development and approval is often lengthy. For many doctors and patients, the development and approval of new cancer treatments is not fast enough. The FDA is working to make the process more efficient in several ways:

  • Meeting with drug sponsors early in the drug development process. This helps to design clinical trials in the best possible way and to improve the review of the data from these studies.

  • Working with drug regulatory agencies in Europe, Japan, Canada, and Australia to streamline the approval of drugs in the United States that are approved in these countries. The International Conference on Harmonization (ICH) is an effort among the drug agencies of Europe, Japan, and the United States. It was created to make sure that the details required for drug approval are similar between countries.

  • Using the Orphan Drug Act to provide incentives for sponsors to develop new drugs that treat rare diseases. This also 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.

FDA programs for drug development and approval

The FDA has several programs to help new drugs reach people sooner:

  • Fast track. This process helps speed up the review of drugs that treat an unmet medical need for serious or life-threatening diseases, such as cancer. An unmet need exists when there is no available therapy or when a disease is not treated by an existing therapy. Based on this, drug sponsors may request fast track designation for a new drug. Through this process, the drug sponsor and the FDA can communicate before the submission of 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).

  • Breakthrough therapy. This designation is available for drugs that treat serious conditions. The application for a breakthrough designation is based on early research that shows that the treatment is a major improvement when compared with the currently available treatments. The designation helps a company to meet with the FDA earlier in the testing process to speed up the development and review. The FDA may suggest that a company apply for breakthrough therapy designation after reviewing the early data.

  • Accelerated approval. This program is designed for drugs that treat serious, life-threatening conditions and fill an unmet medical need. It provides a way for drug makers to request an approval based on study results that are a sign of how well the drug may help people live longer.

    For instance, such a results could be tumor shrinkage or a change in lab test results. After approval, the sponsor must conduct a clinical trial to show that these early results are linked with improved survival. If these studies do not confirm the benefits, the approval for that indication will be withdrawn.

  • Priority review. This process is for drugs that 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. If the FDA grants priority review, it sets a goal of taking action on the application within 6 months instead of within 10 months, which it takes for standard review. But the sponsor can only apply for priority review after submitting an NDA. Therefore, it does not speed the drug development and testing process. Instead, it lessens the time between when the application is submitted and the FDA approves the drug.

Learn more about the fast track, breakthrough therapy, accelerated approval, and priority review programs from the FDA.

Access to drugs in development

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 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 greater than the risk of possible side effects. The FDA recognizes that people who have tried all of the existing treatments may be willing to accept a higher level of risk. It has created programs to provide people with cancer and other serious illnesses with access to investigational drugs.

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. Some people may still be able to receive the investigational drug outside of a clinical trial with an expanded access/compassionate exemption (see below).

Federal laws permit drug makers to charge a person for the actual cost of an investigational drug they receive. Patients and their doctor 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. Learn more about this regulation.

  • Expanded access/compassionate exemption. People may be able to take the drug in an expanded access program through the drug maker. If they do not offer a program for a certain drug, the person or doctor may submit an individual patient request. In this instance, the 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/compassionate exemption from the FDA. You can also visit the Expanded Access Navigator which provides additional information, a list of expanded access policies and criteria, and downloadable forms.

  • Individual Patient INDs. 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 able to join any clinical trials studying the drug and there are no other treatments. There must be enough data to show that the drug may be effective and has no unreasonable risks.

  • Right-to-Try. In May 2018, the United States Congress passed legislation commonly known as “right-to-try.” The law aims to speed up patient access to investigational treatments that have not been approved by the FDA.

    Unlike the expanded access program, right-to-try does not require review by the FDA of an application to use an investigational drug. 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 right-to-try does not require drug companies to make investigational drugs or treatments available to people.

Off-label use

When the FDA approves a new drug, it only approves the drug to treat a specific condition 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. 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 agent received FDA approval. After approval, researchers may find that it is an effective treatment for other types 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 NCI.

Approval by the FDA for new labeling information

To gain FDA approval of new uses or indications for an already-approved drug, the manufacturer must submit a supplemental marketing application to the FDA. 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, the data required to submit another request may not be as extensive as the one for initial approval.

Patient involvement

One of the best ways for people to get involved in the drug development process is to enroll in a clinical trial. Not only does it help researchers better understand new treatment options, it also provides the person with closely monitored care. The FDA has a program to include patients in the review and approval process, called the Patient Representative Program.

This program 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.

Related Resources

Drug Discovery and Development

Drug Information Resources

PRE-ACT: Preparatory Education About Clinical Trials

PRE-ACT, Preparatory Education About Clinical Trials

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