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.

Topical Cholesterol/Simvastatin Gel for the Treatment of CHILD Syndrome in an Adolescent.

Congenital ichthyoses are a heterogeneous group of genetic skin disorders characterized by defects in the critical barrier function of the skin. These life-long conditions present a significant therapeutic challenge in dermatology. One important example is Congenital Hemidysplasia with Ichthyosiform erythroderma and Limb Defects, or CHILD syndrome. This is a rare congenital ichthyosis caused by mutations in cholesterol biosynthesis. With limited success, the cutaneous features of this condition have historically been managed symptomatically with emollients, topical keratolytics, and topical steroids. However, over the last decade, topical therapy directed at the pathogenesis of this condition has emerged as an effective treatment. Herein, we report a case of successful treatment of the cutaneous features of CHILD syndrome with compounded simvastatin and cholesterol gel and highlight the role of the compounding pharmacist in the care of patients with congenital ichthyosis.

Challenges and opportunities: the role of the district nurse in influencing practice education.

The responsibility of the district nurse (DN), alongside complex case management and leadership, is to ensure Specialist Practitioner Qualification District Nurse (SPQDN) education continues to create practitioners delivering quality evidence-based care. DN leadership and its importance have come to the fore during the COVID-19 crisis, where hospital discharges have increased rapidly to make way for highly complex admissions (HM Government, 2020). This paper examines the importance of the SPQDN qualification, exploring the role of the DN within practice education. Continuation of the vital DN qualification will ensure that the numbers of qualified DNs increase, ultimately protecting community capacity. With a move towards an apprenticeship model to achieve the SPQDN, DNs must engage with and influence curriculum development to confirm courses deliver requirements of the workplace, commissioners and the 2019 NHS Long Term Plan. Expectations of the DN role within practice education have changed, moving away from the practice teacher standards to the new Nursing and Midwifery Council Standards for Student Support and Assessment. This poses new challenges in DN education in practice. The implications of this transition threaten to de-value the quality of the assessment process by removing the high standards of preparation previously demanded; ultimately, this is a risk to the provision of the quality practice education that previously existed.

Recruiting the next generation: applying a values-based approach to recruitment.

The qualified district nurse (DN) role demands high levels of leadership. Attracting the right candidates to apply for the Specialist Practice Qualification District Nursing (SPQDN) education programme is essential to ensure fitness to practice on qualification. Anecdotal evidence suggested that the traditional panel interview discouraged candidates from applying and a need to improve the quality of the overall interview process was identified by the authors. The University of Central Lancashire in partnership with Lancashire Care NHS Foundation Trust adopted the National Values Based Recruitment (VBR) Framework to select candidates to gain entry onto the SPQDN course. This involved using 'selection centres' of varying activities including a multiple mini interview, written exercise, group discussion, and portfolio review with scores attached to each centre. The ultimate aim of utilising VBR was to align personal and profession values to both the nursing profession and the Trust whilst allowing a fairer assessment process. An evaluation of the VBR recruitment process demonstrated 100% pass rate for the course and 100% satisfaction with the interview process reported by all 16 candidates over three academic years. Interviewer feedback showed deeper insight into the candidates' skills and values aligned with the core values and skills required by future District Nurse leaders within the Trust.

Raman Spectroscopy: A Sensitive and Specific Technique for Determining the Accuracy of Compounded Pharmaceutical Formulations.

OBJECTIVES: Raman spectroscopy is a widely used technology to identify chemical unknowns or confirm chemical identity. We have tested Raman spectrometry to identify compounded pharmaceutical formulations. In contrast to the commonly used application mentioned above, compounded pharmaceutical formulations contain a mixture of ingredients, and the Raman spectrometer is being used to correctly identify the composition of the complete pharmaceutical formulation, including the active pharmaceutical ingredient(s). The objective of this pilot study was to document the potential use of Raman spectroscopy as a tool to provide quality control to compounded pharmaceutical formulations. METHODS: "Testing a test" study design was used to prospectively determine whether Raman spectroscopy could verify the accuracy of compounded pharmaceutical formulations. A total of 9 formulations that are commonly compounded at Cook Children's Health Center were selected for testing. Each of the 9 formulations and 2 blank controls were randomly tested for compounding accuracy in replicate. A total of 110 tests were conducted. RESULTS: Raman spectroscopy was found to be a reliable test to determine the accuracy of compounded pharmaceutical formulations with a 100% positive predictive value. CONCLUSIONS: Raman spectroscopy promises to be an excellent tool for compounding pharmacies to provide an objective measure of compounding accuracy to their unique, compounded pharmaceutical formulations.

Development and Validation of an Inexpensive, Simple, and Rapid Technique for Measuring the Accuracy of Extemporaneously Compounded Pharmaceuticals.

Use of ultraviolet detection to quantitate analytes is a basic concept of analytical chemistry. The basis of this application is well defined by Beer-Lambert's law. To this end, the authors applied Beer-Lambert's law as a simple and rapid tool to measure the accuracy of extemporaneously compounded pharmaceuticals. Using two commonly extemporaneously compounded formulations, the authors demonstrated the application of this tool. Advantages and limitations of the ultraviolet-visible technique are discussed. The authors speculate that more advanced spectral techniques for quality control will be adopted in the future. These techniques will be more accurate and will be associated with fewer limitations. However, costs associated with use will be greater.

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.