Biotech Industry Liaison Committee Review

Research overview:

In order for a drug or therapy to receive regulatory approval and be available on the market, each must go through a series of steps or phases to evaluate whether it is safe and effective.6 The different phases are:

  1. Pre-clinical: Important safety, toxicity, and preliminary efficacy testing conducted using tissue and animal models. This phase helps researchers characterize the drug and is required before a drug can be studied in humans.
  2. Phase I: Once a drug completes all of the pre-clinical testing, the drug is given to humans for the first time in a phase I trial. This typically involves a small group of normal, healthy adult volunteers to assess the safety of the drug.
  3. Phase II: If the safety assessment in phase I is acceptable, the drug can then be studied in the target population (in this case achondroplastic children). Safety continues to be assessed, while also beginning to assess efficacy.
  4. Phase III: Now that the drug has demonstrated to be relatively safe and effective for further testing, a phase III trial is completed to compare the safety and effectiveness of the new treatment against the current standard treatment. In cases where there is no standard treatment like with achondroplasia, a placebo (an inactive substance like distilled water) can be used as a reference to compare the safety and efficacy of the study drug.
  5. Regulatory approval: If successful in phase III, a regulatory agency, like the Food and Drug Administration (FDA), reviews all of the data collected from the different research phases to evaluate the risks and efficacy to determine if the drug should be approved for general use by the public.
    1. Accelerated approval:  The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.  A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign, or another measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. Drug companies are still required to conduct studies to confirm the anticipated clinical benefit, which will determine whether the drug receives traditional approval or is removed from the market.
  6. Phase IV: If approved, research on the drug is not stopped. The drug continues to be studied to understand the long-term safety and efficacy outcomes.