This article describes the product development lifecycle of a new therapeutic, such as a new drug or biologic. For details on the product development lifecycle of a new medical device, please see Product development lifecycle: Medical device design and development.
The development of a new therapeutic product (i.e., a new drug or biologic) is a long, complex and expensive process which typically takes 10 to 12 years (and sometimes more) from product identification to commercialization.1 This lifecycle usually involves the following stages:
This article focuses on the development stage (#2) outlined above.
Development of a new therapeutic product normally begins with non-clinical testing followed by different phases of human clinical trials in support of the licensing application. Chemistry, Manufacturing and Controls (CMC) activities are conducted concurrently to support these studies. 1
Non-clinical testing (laboratory experimentation and animal investigation) assesses the potential therapeutic effects of a drug substance and demonstrates the reasonable safety of a substance before it can move to human studies. It may also include long-term studies (e.g., reproductive and carcinogenicity studies) that are conducted after the clinical trial is initiated. Non-clinical studies must be conducted following Good Laboratory Practices (GLP). This phase of testing may include in vitro and in vivo studies to research metabolism (pharmacodynamics [PD] and pharmacokinetics [PK]), safety, toxicity, dosage and efficacy. When designing these studies, ensure you review all related regulatory materials, such as guidance documents from your regulatory agency and safety topics from the International Conference on Harmonization [ICH].
The objective of clinical trials is to evaluate the safety and efficacy of a product in humans. A clinical program involves four phases and must comply with regional requirements as well as Good Clinical Practices (GCP). Phases I to III are conducted to collect safety and efficacy information in support of the licensing application. Phase IV is conducted post-marketing (i.e., once the product reaches the market).
Phase I starts with the initial administration of an investigational product into humans (healthy volunteers, or in patients if for the use of cytotoxic drugs). These studies usually have non-therapeutic objectives. The study design can be open and baseline-controlled, or may use randomization and blinding to improve the validity of observations in the study. Phase I clinical trials may include:
Phase II explores the therapeutic efficacy in patients, with the designs including concurrent controls and comparisons with the baseline status. The patient population is normally selected with narrow criteria. A major objective is to determine the dose(s) and regimen to support
Phase III trials. Other objectives may include potential study end point evaluations, therapeutic regimens and target populations (e.g., mild versus severe disease) via exploratory analyses, examining data subsets and using multiple end points in clinical trials.