A New Chemical Entity (NCE) refers to a compound identified during the drug discovery process in the pharmaceutical industry. This compound has a novel structure, possibly a new chemical class that has been discovered by the innovator. NCE also implies that such a compound has never been approved for medical use by any regulatory authority worldwide. The discovery of an NCE is a significant milestone in the development of new medications, as it serves as the basis for further research, development, and clinical trials to assess its therapeutic potential. An NCE is defined as a drug that contains no active moiety previously approved by the United States Food and Drug Administration (USFDA) in any other application. Innovator drug manufacturers typically develop an NCE during the early stages of the product cycle. The NCE then undergoes various clinical trials to eventually become a drug product.
In 2015, the FDA issued a draft guidance stating that if an NCE is approved for a drug product under section 505(b), no other manufacturer can apply for a product with the same active moiety for five years from the approval date of the original drug product. However, the guidance also includes an “Umbrella Policy,” allowing products developed simultaneously with the same active moiety to share the five-year exclusivity period. This exclusivity, granted by the FDA, provides the license holders with a competitive advantage in the market. The five-year exclusivity applies to each drug substance present in the product. NCE exclusivity can be obtained by submitting either a 505(b)(1) or 505(b)(2) application.
New Chemical Entities (NCEs) are the driving force behind innovation in drug development, opening new treatment possibilities where current therapies fall short or are unavailable. The market introduction of an NCE can greatly enhance public health by offering new solutions for managing or curing challenging diseases. Moreover, a successful NCE can become a blockbuster drug, generating significant revenue and supporting further research and development efforts. The roles and functions of NCEs are varied, highlighting the broad spectrum of therapeutic areas and disease targets they aim to address. Fundamentally, NCEs serve as the active ingredients in pharmaceutical drugs, engaging with biological targets in the body to produce a therapeutic effect. This interaction can manifest in different ways, such as inhibiting a specific enzyme’s activity, modulating a receptor’s function, or disrupting the replication process of pathogens, among other mechanisms.
The development process for an NCE is rigorous, involving multiple stages to ensure its safety and effectiveness for human use.
- Initially, the compound undergoes extensive in vitro (test tube) and in vivo (animal) studies to evaluate its pharmacological properties, toxicity, and potential side effects. This preclinical testing is essential for identifying any significant risks before moving on to human trials.
- Once preclinical testing is successful, the NCE progresses to the clinical trial phase, where it is tested in humans to assess safety, efficacy, and optimal dosing. Clinical trials are conducted in phases, each designed to answer specific research questions: Phase I trials focus on safety and dosage, Phase II trials assess efficacy and side effects, and Phase III trials confirm the drug’s effectiveness, monitor side effects, compare it to standard treatments, and gather information to ensure its safe use.
- Regulatory authorities play a crucial role in the development of an NCE. Agencies like the S. Food and Drug Administration (FDA)or the European Medicines Agency (EMA) review data from preclinical and clinical trials to evaluate whether the benefits of the NCE outweigh its risks. Upon approval by the regulatory authority, the NCE can be marketed as a new drug, offering patients new treatment options.
In conclusion, NCEs are pivotal to the progression of medical science and the creation of new therapies. Their discovery and development are crucial for meeting unmet medical needs, enhancing patient outcomes, and driving innovation within the pharmaceutical industry. Although the process of bringing an NCE to market is challenging and costly, the potential benefits for public health and the advancement of medical knowledge render this endeavor invaluable.
