How Compounding Pharmacies Fill Critical Gaps in Pediatric Drug Development Processes: Suggested Regulatory Changes to Meet Future Challenges.

Drugs administered to children in the United States fall into two broad categories: (1) those that have followed the US Food and Drug Administration (US-FDA) pediatric drug approval process and are marketed as finished dosage forms with pediatric labeling; and (2) all others, many of which are used "off-label". The use of most drug products in pediatrics is still off label, often requiring special preparation, packaging, and, in some cases, compounding into preparations. The latter category includes compounded preparations that incorporate either a US-FDA approved finished dosage form (e.g., a sterile solution, sterile powder, nonsterile capsules, oral solution, crushed tablets, etc.), or rely on bulk active pharmaceutical ingredients (APIs). Compounded preparations are prepared for individual patients in 503A pharmacies, or on a larger scale and not just for specific patients, in licensed 503B establishments. Critical gaps in the current drug approval process for finished dosage forms have created a proverbial "Gordian knot" that needs to be untangled thoughtfully to facilitate increased production and approval of vitally needed medications for pediatric patients. This opinion will describe current regulatory processes pertaining to pediatrics-only drug approval in the United States. Additionally, discussed are steps required for a product to acquire pediatric labeling. Gaps in regulatory approval pathways for both manufactured and compounded pediatric drugs will be identified, especially those that complicate and slow development and availability to patients. Finally, suggestions for regulatory modifications that may enhance pediatric product development strategies for both manufacturers and compounders are suggested.

Compounded Nonsterile Preparations and FDA-Approved Commercially Available Liquid Products for Children: A North American Update.

The purpose of this work was to evaluate the suitability of recent US Food and Drug Administration (US-FDA)-approved and marketed oral liquid, powder, or granule products for children in North America, to identify the next group of Active Pharmaceutical Ingredients (APIs) that have high potential for development as commercially available FDA-approved finished liquid dosage forms, and to propose lists of compounded nonsterile preparations (CNSPs) that should be developed as commercially available FDA-approved finished liquid dosage forms, as well as those that pharmacists should continue to compound extemporaneously. Through this identification and categorization process, the pharmaceutical industry, government, and professionals are encouraged to continue to work together to improve the likelihood that patients will receive high-quality standardized extemporaneously compounded CNSPs and US-FDA-approved products.

Quality-control standard operating procedures-an essential tool for developing quality preparations.

Maintaining quality in compounded preparations must be a top priority of top management, and it must involve all pharmacy employees. Pharmacy compounding requires the development and maintenance of standard operating procedures to ensure quality and minimize compounding errors. Standard operating procedures are detailed instructions initiated by the pharmacist-in-charge or the quality control officer for the purpose of assuring the reliable, consistent performance of routine tasks involved in formulation development, purchasing, compounding, testing, maintenance, materials handling, quality assurance, and dispensing. Maintaining written standard operation procedures is an important step in assuring that a phamacy has developed a quality compounding practice that meets and/or exceeds United States Pharmacopeial standards.