Clinical Trials

The Journey to Develop a New Medication

Developing and bringing a single drug or device to the market is a long and expensive process. Each drug must go through substantial research, and it must be approved by the U.S. Food and Drug Administration (FDA) before it is considered safe and effective for patients. It can take more than a decade and millions of dollars to discover and develop a product that is approved for use by the public.

Pre-discovery

A thorough understanding of a disease is the first step in developing a new medication or treatment. Scientists analyze what might have caused the cancer, how it can be treated and ways to alleviate its symptoms. In the United States, this research is a group effort with contributions from government, industry and academic institutions helping to filter thousands of potential drugs or therapeutic agents down to only hundreds that show the most promise.

Drug discovery

After an overall understanding of the disease is established, research focuses on biologic “targets” of the disease, such as a specific protein, molecule or gene. Researchers conduct studies to determine whether that target can be influenced by a medication. They also look for molecules that can influence this target, which are potential medications also known as compounds.

Pre-clinical testing

In the pre-clinical testing phase, the compounds identified during drug discovery are tested (in the laboratory and in animals) to determine if the new drug is safe and if it will work as intended. This phase can take several years to complete. Once the compounds make it to the next level (and only a small percentage do), the pharmaceutical research company must submit an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA). The IND must include specific details, including results from the animal studies as well as the drug’s ingredients, manufacturing and processing information and protocols (outline of how the study will be conducted) for all proposed clinical trials.

Clinical trials

When the new drug is approved for testing in humans, clinical trials begin. The safety and integrity of these trials are extremely important factors in this part of the process. These federally regulated studies are heavily monitored to ensure that participants aren’t subjected to unreasonable harm and that all volunteers are fully aware of the risks associated with each trial. Before information about a drug can be submitted to the FDA for review, the safety and efficacy of the drug must be evaluated in all the primary phases of clinical testing, which can typically take six to seven years.

Most cancer treatment clinical trials are part of the development of a new drug that has already been shown to destroy cancer cells in animals or in the laboratory. Some clinical trials involve a drug that has already been approved by the FDA to treat a different type of cancer, and testing is needed to approve the drug for use in patients with other cancer types.

Clinical trials are completed in phases, with each study helping to accumulate knowledge to be applied during the next phase of the trial, somewhat like building blocks. The trials are classified as phase I, II or III. Phase IV is used to study the long-term effects associated with certain treatments (see page 2 for more information on the specific phases).

 

Explaining orphan drugs

Orphan drugs are pharmaceutical medications used to treat rare diseases or disorders (those affecting fewer than 200,000 people in the United States). Because most pharmaceutical companies find it more lucrative to dedicate dollars and effort to drugs for the masses, developing and marketing orphan drugs is extremely expensive. To ensure that treatments for rare conditions aren’t ignored, the U.S. Food & Drug Administration (FDA) established the Office of Orphan Products Development (OOPD).

OOPD’s mission is to advance the evaluation and development of products that demonstrate promise for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions. Assigning “orphan drug status” to these products has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.

OOPD encourages the development of orphan drugs by providing incentives to drug sponsors, including exemptions from normal prescription drug user fees, tax credits for clinical testing and a grant program. In September 2015, the FDA awarded 18 new research grants totaling more than $19 million to boost the development of orphan drugs, which will affect the lives of nearly 30 million Americans.

 
 

Review and approval

If the drug is shown to be safe and effective in all three phases of clinical trials, the pharmaceutical company may submit a New Drug Application (NDA) to the FDA. This application must include results from the pre-clinical studies, data from the clinical trials, drug ingredients and drug manufacturing details, and processing and packaging information.

For the drug to be approved, the FDA reviewers must agree that it is safe and effective for its proposed use, that the benefits outweigh the risks, that the labeling is accurate and that the methods of manufacturing adequately preserve the identity, purity, quality and strength of the drug.

Manufacturing

Once a new drug has received FDA approval, it can be manufactured in an FDA-inspected manufacturing facility. Because the drug must be consistently and efficiently produced for as long as it’s needed, the manufacturing companies are regulated by the FDA and are held to the highest standards to ensure safety and high-quality production of human pharmaceutical agents.

Ongoing research

Even after a drug is available for prescription, the FDA requires continued monitoring and safety reporting of any drug as long as it is on the market. Any adverse events (such as side effects) not reported initially must be reported. Continued research may also focus on additional uses for the drug, improved dosage forms and delivery systems, or its potential use in combination treatments.

Additional Resources

 

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