Great Strides 2006
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Q+A: Following the Drug Development Pipeline from Laboratory to Pharmacy

What are the different phases of development a potential CF drug must go through to reach the hands of a patient?

Discovery Phase — In a series of test tube experiments called assays, chemical compounds are tested to determine if any is a viable candidate for further development.

Preclinical Phase — Potential drugs identified in the discovery phase are tested and evaluated in tissue samples from people with CF and in animal models (primarily mice and rats). Testing at this phase shows whether a potential drug has toxic side effects and how safe it is at different doses.

Phase 1 Clinical Trials — The purpose of this phase is to assess the safety of the drug and identify any side effects caused by it. These studies also determine safe doses and administration methods (e.g., oral, intravenous, aerosol). Usually, a small number of healthy volunteers are the first to participate in Phase 1 trials.

Phase 2 Clinical Trials — Phase 2 begins only after Phase 1 data suggest that the drug is safe for patients and may be effective against its primary target (e.g., mucus reduction, infection). In this phase, the drug is tested for its beneficial effects or efficacy on a moderate sized group of patients with CF, as well as for continued safety monitoring. The optimal dose is usually determined at this phase.

Phase 3 Clinical Trials — The Phase 3 trial is an important turning point. These studies measure how well a drug works in a large number of people and “fine tunes” dosage amounts, safety and effectiveness. Phase 3 trials involve many CF care centers and hundreds of patients. Once these studies are successfully completed, final approval by the Food and Drug Administration (FDA) can be sought.

How long does the entire drug development process take?

The process typically takes 10-15 years. With significant CF Foundation support, TOBI ®, the first aerosolized antibiotic to treat lung infections in those with CF, took approximately 10 years to develop.


The CF Foundation has played an integral role in the development of four key therapies used for CF: Pulmozyme ®, TOBI ®, azithromycin and, most recently, hypertonic saline.

Why does it take so long?

One simple reason is safety. The number one priority of researchers and scientists in the drug development process is to ensure that new drugs and treatments are safe for and effective in people. Protecting the patient in this process means adhering to a strict, step-by-step process, which involves numerous scientists, researchers and patients. Before a drug can be considered by the FDA for approval, a minimum of three clinical trials must be conducted, each of which may take a year or longer and often involve a lengthy enrollment process. The drug research process is not only time-consuming, but costly. Literally hundreds, and sometimes thousands, of chemical compounds must be made and tested to find one that can achieve the desired result.

How many drugs that enter clinical trials achieve FDA approval on average?

Only about one in five drugs that enter clinical trials will ultimately enter the marketplace.

What is the CF Foundation doing to increase the number of drugs in development?

Through its strong fund-raising efforts, the CF Foundation is dedicated to supporting innovative research that will lead to new treatments and a cure for CF. The CF Foundation has successfully diversified its portfolio of research investments, keeping the pipeline well stocked with potential compounds at every stage of development from the earliest test tube studies through Phase 3 clinical trials. Currently, there are more than 25 potential therapies in the CF drug discovery and development pipeline.

Why is it important to participate in a clinical trial?

Clinical trials are the safest and fastest way to evaluate potentially effective treatments and new means for treating disease. New medications cannot enter the marketplace without going through this rigorous testing to ensure that they are safe and effective. People with CF are critical partners in this process. Without the participation of CF patients in clinical trials, new medications would not be available today, nor will today’s most promising scientific discoveries result in new drugs tomorrow.