Quality by Design in Pharmaceuticals: A Review of its Impact on Regulatory Compliance and Product Quality.

The pharmaceutical industry has embraced the quality-by-design (QbD) approach as a promising development, formulation and manufacturing strategy. QbD provides a systematic and science-based framework for designing and producing high-quality products, with a particular focus on identifying, assessing and controlling risks throughout the development process. This review aims to assess the benefits of implementing QbD in pharmaceutical processes, evaluate its impact on regulatory compliance and explore its potential to enhance drug product quality. The primary objective of this review is to evaluate the influence of QbD on pharmaceutical development and manufacturing processes. It also seeks to examine the regulatory requirements associated with the implementation of QbD and highlight the advantages of this approach in terms of product quality and cost-effectiveness. Additionally, the review aims to explore the potential of QbD in improving the safety and efficacy of drug products. The QbD approach holds tremendous potential to revolutionize the pharmaceutical industry by optimizing drug development & manufacturing processes, reducing costs and enhancing product quality and consistency. However, implementing QbD requires a comprehensive understanding of the underlying science, as well as strict adherence to regulatory requirements in drug development and manufacturing. In conclusion, by embracing the QbD approach, the pharmaceutical industry can ensure the production of safe, effective and regulation-compliant products while simultaneously improving process efficiency. This strategic shift toward QbD represents a pivotal step in advancing pharmaceutical research and manufacturing capabilities, ultimately benefiting both the industry and more importantly, patients worldwide.

Relation Between Cardiac T2* Values and Electrocardiographic Parameters in Children With Transfusion-dependent Thalassemia.

BACKGROUND/OBJECTIVES: Cardiac T2* magnetic resonance imaging (MRI) is the gold standard to determine myocardial iron overload. As availability of Cardiac T2* is not uniform across developing nations, our strategy was to identify a more accessible and cost effective tool to assess myocardial iron accumulation. As children with transfusion-dependent thalassemia also experience various electrocardiographic abnormalities, we performed electrocardiography (ECG) as well as Cardiac T2* MRI on all children registered in our thalassemia unit. MATERIALS AND METHODS: Forty-eight transfusion-dependent thalassemia children with transfusion burden ≥12 times/y (6 to 19 y) in the Thalassemia Unit of the Division of Hematology Oncology, Department of Pediatrics were enrolled. Patients were divided into 3 groups based on severity of T2* value, that is group I (T2*<10), group II (T2* 10 to 20), group III (T2*>20). A T2* value >20 was taken as normal. ECG and serum ferritin was also performed on the day of MRI. RESULTS: Among the various ECG parameters, QRS duration, and QTc interval were significantly increased if cardiac iron overload was high with a P-value of 0.036 and 0.000, respectively. Also, high serum ferritin predicted a decline in T2* value with a P-value of 0.001. QT interval and QTc interval significantly correlated inversely with T2* (P=0.042, r=-0.295 and P=0.002, r=-0.446, respectively) but not QRS duration (P=0.05, r=-0.282). Serum ferritin also was found to have a significant inverse correlation with T2* value (P=0.000, r=-0.497). CONCLUSIONS: Abnormalities on ECG, that is prolongation of QRS duration, QT interval, and QTc interval were significantly associated with cardiac iron overload, that is decrease in the value of Cardiac T2* in our study.

Langerhans cell histiocytosis: An enigmatic disease.

BACKGROUND: Langerhans cell histiocytosis (LCH) is a poorly understood disease with heterogeneous clinical presentation ranging from unifocal bony involvement to disseminated disease with life-threatening complications. MATERIALS AND METHODS: The clinical profile, laboratory findings, treatment, and long-term outcome were retrieved from maintained medical records from January 2006 to January 2016 and were retrospectively analyzed. The extent of the disease was classified as per the LCH-III trial of "The Histiocyte Society." The assessment and categorization of treatment response followed LCH III trial definitions. RESULTS: A total of 28 children with LCH were diagnosed. The age ranged between 5 months and 9 years, with a mean of 3½ years. The M: F ratio was 3:1. Single system, unifocal and multifocal bone diseases were seen in nine (32.1%) and two (7.1%) cases, respectively. Disseminated disease without risk organ involvement was seen in six (21.1%), whereas disseminated disease with risk organ involvement was seen in 11 (39.3%) cases. The most common presentation was bony involvement (19 [67.8%]), out of which 16 (88.8%) had skull involvement. During follow-up, 17 (60.7%) were in complete remission though five (17.8%) of them relapsed, but achieved second remission. Two (7.1%) were lost to follow-up. Six (21.4%) had progressive disease of which four expired and two abandoned treatment. Two (10.7%) refused the initiation of treatment. CONCLUSION: A better understanding of the disease, early suspicion, and diagnosis can improve the outcome of patients with LCH.