The FDA’s myriad acronyms—and the differences between them—can be perplexing. BioSpace looks at the unique advantages of certain regulatory classifications for drug developers.
Pictured: FDA Headquarters/iStock, Grandbrothers
The FDA’s name isn’t the only acronym in the organization. Rife with three-letter abbreviations, the agency’s departments and classifications can feel a bit like an alphabet soup. While this can be a bit overwhelming, understanding these designations is essential in setting appropriate timeline expectations for budgeting and stakeholders.
Understanding how the agency will classify a new therapy is a useful piece of the puzzle for any potential regulatory submission. Comparing an asset to previously approved products that fall under the same classification could help inform the regulatory path for a New Drug Application (NDA).
Additionally, certain classifications will determine how far out the Prescription Drug User Fee Act (PDUFA) date will be set from the time of the NDA’s receipt. With biotech bankruptcies skyrocketing in 2023 and capital raise challenges, time is of the essence in biopharma, where the average drug takes 10.5 years to go from Phase I testing to regulatory approval, according to the Biotechnology Innovation Organization.
Every time the FDA receives an NDA, a tentative classification code is assigned prior to the target PDUFA date and then reassessed at the time of the drug’s approval. The classification codes are based on the relationship of the new therapy to products already approved by the FDA. Here are five that are particularly relevant for drug developers.
NDA Classification Codes
Note: Drugs may have more than one classification, such as Type 1,4 if one of the components is an NME. |
Every Rectangle Is Not a Square
The difference between two particular FDA classifications—new molecular entity (NME) and new chemical entity (NCE)—can be particularly perplexing. An NME is a drug whose active ingredient contains no active moiety that has been previously approved by the FDA, while an NCE is a drug that does not contain an active moiety previously approved by the FDA.
With the near word-for-word similarities between these two classifications, they can appear to be duplicative. But rather than being identical, the two fall under the old adage—every square is a rectangle, but not every rectangle is a square.
These terms are actually distinct and used for different purposes, Charles Kohler, press officer for the Center for Drug Evaluation and Research (CDER), told BioSpace in an email. NME is a categorization used to help CDER manage its workload. The term is not defined in regulations or statutes. It is additionally used to develop the agency’s annual novel drug approval list.
In contrast, NCE status is used only to determine a drug’s eligibility for exclusivity. This classification is particularly important to drug developers, as New Drug Product Exclusivity provides the holder of an approved NDA for a product containing a new chemical entity a five-year period of protection from market competition. CDER reviews all relevant applications for exclusivity determination, Kohler said.
Because a drug tagged as an NME is unique from previously approved medicines, the timeline for an NDA with this classification will be slightly longer than a non-NME application, Kohler said. The PDUFA goal date for an NME is 12 months after receipt, while a non-NME application can expect a decision within 10 months of NDA receipt.
NME status may also be useful for drug developers hoping to snag an expedited program designation for an asset aimed at a serious or life-threatening condition. In 2023, 65% of CDER’s 55 novel drug approvals used one or more expedited programs such as Fast Track or Priority Review, Kohler noted.
For example, Eli Lilly’s lymphoma treatment Jaypirca was classified as an NME and received Priority Review, which sets the PDUFA goal date within six months. Jaypirca also received Orphan Drug status, which does not speed approval but gives the developing company exclusive rights for seven years once the drug is approved. Kohler clarified that for a potential drug to qualify, the application must meet the criteria for each program, regardless of NME classification.
Therapeutic Purpose and Potential
NDAs are additionally classified according to therapeutic potential. Drug products with a high therapeutic rating are prioritized and reviewed more quickly than drugs with a lower rating.
Each new drug is also assigned to a therapeutic category by its intended effect and broken down further into a pharmacologic class. Knowing the pharmacologic class of a drug is not only helpful for the FDA but also vital for healthcare professionals. This classification identifies what a prescribing doctor or dispensing pharmacist should expect from a drug and how it relates to other therapeutic options. Additionally, it helps reduce the risk of duplicative therapy and negative drug interactions.
In 2022, the FDA received 130 NDAs, of which 118 were ultimately approved. Understanding the unique advantages of each regulatory classification can help a company get its application out of the slush pile and in front of FDA reviewers sooner.
Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.
