From Lab to Patient Care

Posted: Aug. 1, 2004

By law, the Federal Drug Administration (FDA) must review all test results for new treatments to ensure that products are safe and effective for specific uses. "Safe" does not mean that the treatment is free of possible adverse side effects; rather, it means that the potential benefits have been determined to outweigh any risks.

Clinical trials
The testing process begins long before the first person takes the treatment, with preliminary research and animal testing.

If a treatment proves promising in the lab, the drug company or sponsor must apply for FDA approval to test it in clinical trials with people. The application is called an Investigational New Drug (IND) application.

Once the IND is allowed to proceed, clinical trials can begin. If the treatment makes it through the clinical trials process – that is, if the studies show the treatment is safe and effective – the sponsor may submit to the FDA for another application. For drugs, this is a New Drug Application (NDA); for biologics, it's a Biologics License Application (BLA). The application must include the following:

• The exact chemical makeup of the drug or biologic
• Results of animal studies
• Results of clinical trials
• How the drug or biologic is made, processed, and packaged
• Quality control standards

Once the FDA receives the NDA or BLA from the sponsor, the formal New Drug Application Review Process or Biologics/Product License Application Review Process begins.

Speed versus safety in the approval process
During the approval process, the FDA classifies as "priority" those treatments that offer significant medical advances over existing therapies.

But even in non-priority cases, the FDA's goal is that no more than 10 months will pass between when a complete application is submitted and the FDA has finished its review, either approving the drug or biologic, or providing the sponsor with a complete list of the issues that need to be addressed.

The process is not always smooth, however. Sometimes the FDA requests (or the FDA's advisory panel recommends) additional research or data. Some new approvals have taken as little as 42 days from the time the last part of the BLA/NDA is received; other more difficult applications have spent years in the approval process.

Click here to learn more about Advanced Healthcare’s clinical trials.

Expert advice
The FDA relies on a system of independent advisory committees, made up of professionals from outside the agency, for expert advice and guidance in making sound decisions about drug approval.

Each committee meets as needed to weigh available evidence and assess the safety, effectiveness, and appropriate use of products considered for approval. In addition, these committees provide advice about general criteria for evaluation and scientific issues not related to specific products. The Oncologic Drugs Advisory Committee (ODAC) meets regularly to provide expert advice on cancer-related treatments and preventive agents.

Each committee is composed of representatives from the fields of research science and medical practice. At least one member on every advisory committee must represent the consumer perspective.

Final approval
As the FDA looks at all the data submitted and the results of its own review, it applies two benchmark questions to each application:

• Do the results of well-controlled studies provide substantial evidence of the treatment's effectiveness?
• Do the results show the product is safe under the conditions of use in the proposed labeling? In this context, "safe" means that potential benefits have been determined to outweigh any risks.

Continued vigilance
The FDA's responsibility for new medical treatments does not stop with final approval. The Office of Compliance tracks drugs after approval to make sure that drug makers comply with current standards and regulations. The Office of Drug Marketing, Advertising, and Communication monitors new drug advertising to make sure it is truthful and complete.

And through a system called MedWatch, the FDA gets feedback from health professionals and consumers on how the new treatments are working, any adverse reactions, and potential problems in labeling and dosage.

Source: Federal Drug Administration

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