What is a Botanical Drug?
Botanical drug products, or BDPs, refer to drug products that include or may be derived from plant materials, algae, macroscopic fungi, or combinations thereof and that are intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in humans. BDPs may be available as, but are not limited to, solutions (e.g., tea), powders, tablets, capsules, tinctures, topicals, or injections. BDPs often have unique features, such as complex heterogenous mixtures, uncertainty about the active ingredient(s) and their biological activities, and previous human experience.
Products generally not considered BDPs include:
- Products containing animals or animal parts and/or minerals, except when these are a minor component in a traditional botanical preparation,
- Materials derived from genetically modified botanical species with intention of producing a single molecular entity,
- Products produced by fermentation of yeast, bacteria, plant cells or other microscopic organisms with intention to produce a single molecular entity, and
- Highly purified substances, either derived from naturally occurring sources or chemically modified.
CDER finalized its first BDP guidance in June 2004 and later issued an updated Botanical Drug Development Guidance for Industry in 2016. This updated guidance provides specific recommendations and new sections to better address later-phase development and submission of new drug applications (NDAs). This guidance provides information on appropriate development plans for botanical drugs to be submitted in NDAs and specific recommendations on submitting investigational new drug applications (INDs) in support of future NDA submissions for botanical drugs.
Due to the heterogeneous nature of BDPs and uncertainty surrounding the active constituents and their biological activities, one of the critical issues for BDPs is ensuring therapeutic consistency across the BDP batches. Generally, therapeutic consistency can be supported by the “totality of the evidence” approach. This approach includes:
- Botanical raw material controls (e.g. agricultural practice and collection)
- Quality control by chemical test(s) and manufacturing controls (e.g., analytical tests and process validation)
- Biological assay and clinical data (e.g., reflects drug’s known or intended mechanism of action)
The Botanical Drug Development Guidance for Industry describes considerations and recommendations for the totality of the evidence approach, and instructions for submitting information to support therapeutic consistency for BDPs.
To date, four botanical products have been approved for marketing as prescription drugs under the NDA and biologics license application (BLA) pathways. The three NDAs include drug products containing the active ingredients sinecatechins, crofelemer, and birch triterpenes, respectively. The BLA contains the active ingredient anacaulase-bcdb.
Additionally, botanical active ingredients such as psyllium and witch hazel can be marketed under certain over-the-counter monographs.
For information on the regulation of dietary supplements and the reporting of safety problems for dietary supplements currently on the market, please see the FDA’s webpage on dietary supplements.