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Living With Cancer

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Process

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New cancer treatments are developed through a sequence of experiments, first in laboratories and animals (preclinical testing) and then in patients (clinical trials). If the clinical trial results show that the treatment helps patients, the treatment becomes part of standard treatment.

This development of new cancer treatments is called the drug development process.

The relationship between clinical trials and the drug development process is complex. All development of new drugs is done in clinical trials; however, not all clinical trials are done to test new drugs. The clinical trials section has information specifically about clinical trials.

An easy-to-read resource about the drug development process is:

  • "From Test Tube to Patient: Improving Health through Human Drugs" (pdf file)

Drug Development Glossary

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The FDA review of the drug company's application for the new drug uses many technical terms. Some of the most common terms follow:

Clinical benefit. New drugs must show a benefit for patients that is objective and observable.

Endpoint. The end result of a clinical trial, chosen to show the clinical benefit of the treatment being studied. Typically, the gold standard of endpoints for cancer trials is survival — does the treatment increase survival?

Surrogate endpoint. A measurement that may not be a direct measurement of how a patient feels functions or survives, but nevertheless is considered likely to predict benefit. An example is tumor shrinkage. If a Phase II orPhase III trial is conducted using surrogate endpoints (eg, if a trial shows that a drug results in tumor shrinkage), further Phase IV trials must be conducted to confirm that the surrogate endpoint (eg, tumor shrinkage) actually results in a clinical benefit such as increased survival.

Not all drugs that achieve a surrogate endpoint actually result in increased survival, which is why confirmation is critically important.

Investigational New Drug Application, or IND. An application that a drug sponsor must submit to FDA before beginning tests (clinical trials) of a new drug on humans. The IND contains the plan for the study and is supposed to give a complete picture of the drug, including its structural formula, animal test results, and manufacturing information.

New Drug Application, or NDA. An application requesting FDA approval to market a new drug for people. The application must contain, among other things, data from specific technical viewpoints for FDA review — including chemistry, pharmacology, medical, biopharmaceutics and statistics.

Treatment IND. A mechanism that allows promising investigational drugs to be used in "expanded access" protocols — relatively unrestricted studies in which the intent is both to learn more about the drugs, especially their safety, and to provide treatment for people with immediately life-threatening or otherwise serious diseases for which there is no real alternative. But these expanded access protocols also require researchers to formally investigate the drugs in well-controlled studies and to supply some evidence that the drugs are likely to be helpful. The drugs cannot expose patients to unreasonable risk.

There are four requirements that must be met before a treatment IND can be issued:

  1. the drug is intended to treat a serious or immediately life-threatening disease
  2. there is no satisfactory alternative treatment available
  3. the drug is already under investigation, or trials have been completed; and
  4. the trial sponsor is actively pursuing marketing approval.

Standard review. Standard reviews are given to new drug applications that do not represent an advance in the treatment for a disease. A standard review must be completed within 10 months of the application.

Priority review. A priority review is granted for a new drug that, if approved, would be a significant improvement over current treatment options for a disease - in other words the new drug would fill an unmet medical need. While a standard review must be completed in 10 months, a priority review must be completed in 6 months.

Once a drug receives a priority review designation the drug is often approved in much less than 6 months. Gleevec, a leukemia drug, was approved in 8 weeks in June 2001, and Eloxatin, a colorectal cancer drug, was approved in 7 weeks in August 2002.

Accelerated approval. To be considered for accelerated approval a drug must treat a serious or life threatening illness and provide a meaningful therapeutic benefit to patients over existing treatments. Accelerated approval uses surrogate endpoints. However, even though a new drug may be approved with an accelerated approval, the drug company must go on to prove in a Phase IV clinical trial that the drug has a meaningful clinical benefit such as prolonging patients' lives or their quality of life. Generally, any accelerated approval drug would get a priority review.

Fast track. A drug may receive a fast track designation if it is intended to treat a serious or life threatening condition and it demonstrates the potential to address an unmet medical need. Fast tracking actually has little effect on the patient's access to the drug. It means that the drug company is engaged in continuous discussions with the FDA about the drug's development plan and the company may also submit its new drug application to the FDA in sections that can be reviewed while the company prepares other sections of the application for submission to the FDA. This is also known as a "rolling submission."