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Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs: Update, July 2010 to June 2014
Conclusions: The findings further support that FDA has demonstrated extraordinarily reasonable flexibility in its review of certain applications for orphan drugs and reinforce the need for FDA and drug companies to better understand and discuss the various types of flexibility. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Sasinowski, F. J., Panico, E. B., Valentine, J. E. Tags: Regulatory Science Source Type: research

iCAN: Providing a Voice for Children and Families in Pediatric Research
This article describes the foundational building blocks of iCAN and provides tools to investigators and practitioners in an effort to increase the instances of children and families being invited to share their unique point of view in medicine. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Thompson, H., Frederico, N., Smith, S. R., Chowdhury, M., Dicks, P., Preston, J., Thompson, C. Tags: Special Section on Pediatrics Source Type: research

Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients
Many drugs approved by the US Food and Drug Administration (FDA) for use in adults lack adequate data on safety and efficacy in pediatric patients, a potential source of unintended harm to pediatric patients. Through a series of laws, regulations, and guidance documents, the US Congress and FDA have created a program both to encourage and mandate clinical studies in pediatric patients to develop evidence-based dosing, safety, and efficacy information. A "Pediatric Study Plan" (PSP) is required for every new drug. FDA provides incentives for the voluntary conduct of clinical trials in pediatric patients, including opportuni...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Tabor, E. Tags: Special Section on Pediatrics Source Type: research

Regulatory and Funding Strategies to Develop a Safety Study of an Auditory Brainstem Implant in Young Children Who Are Deaf
Conclusions: Promising innovative treatments face a number of obstacles along the pathway to full commercialization. A strategy that included early conversations with the FDA and the device manufacturer, and successfully obtaining external funding, resulted in an approved IDE protocol. Early results indicated that the risks, though not minimal, can be successfully mitigated. These young children appear to benefit audiologically from the ABI. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Fisher, L. M., Eisenberg, L. S., Krieger, M., Wilkinson, E. P., Shannon, R. V., the Los Angeles Pediatric ABI Team Tags: Special Section on Pediatrics Source Type: research

Best Practice Recommendations Regarding the Assessment of Palatability and Swallowability in the Development of Oral Dosage Forms for Pediatric Patients
The palatability of oral drugs influences patient adherence to prescribed regimens, especially for children. Various factors influence palatability, including smell, taste, texture, and dose volume. Evaluation of these factors plays an important role in pediatric drug development and is a target for regulatory scrutiny. The Global Alliance for Pediatric Therapeutics, a public–private consortium under the guidance of the Institute for Pediatric Innovation, convened the Alliance Palatability Working Group to discuss the issues related to the assessment of palatability in the development of pediatric oral dosage forms. ...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Thompson, C., Lombardi, D., Sjostedt, P., Squires, L. Tags: Special Section on Pediatrics Source Type: research

Advancing Pediatric Psychiatry Research: Linking Neurobiological Processes to Novel Treatment and Diagnosis Through the Research Domain Criteria (RDoC) Project
This article reviews the literature related to progress in child psychiatry, development of RDoC, and the role of genetic research in the advancement of other areas of medicine. Limited diagnostic and treatment advances have occurred in child psychiatry over recent decades, particularly in comparison to other medical fields. The rapidly evolving fields of molecular genetics, neurobiology, and behavioral science offer substantial promise for refining diagnosis and treatment modalities. Utilizing the advancement of care for other pediatric conditions such as cystic fibrosis and pediatric cancer as guides, this article outlin...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Drury, S., Cuthbert, B. Tags: Special Section on Pediatrics Source Type: research

Attention-Deficit Hyperactivity Disorder and Pharmacotherapy--Past, Present, and Future: A Review of the Changing Landscape of Drug Therapy
Attention-deficit hyperactivity disorder (ADHD) is the most common neurobiological disorder in children. Efficacy of pharmacotherapy in treating ADHD symptoms has generally been considerable with at least three-fourths of individuals benefiting from pharmacotherapy, typically in the form of stimulants. In this review, we begin by briefly reviewing the history of pharmacotherapy in relation to ADHD, before focusing (primarily) on the state of the field on themes such as biophysiology, pharmacokinetics, and pharmacogenomics. We conclude with a summary of emerging clinical and research studies, particularly the potential role...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Connolly, J. J., Glessner, J. T., Kao, C., Elia, J., Hakonarson, H. Tags: Special Section on Pediatrics Source Type: research

Applying Regulatory Science to Develop Safe and Effective Medicines for Neonates: Report of the US Food and Drug Administration First Annual Neonatal Scientific Workshop, October 28-29, 2014
This report will review potential approaches to enhancing neonatal drug development. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Offringa, M., Davis, J. M., Turner, M. A., Ward, R., Bax, R., Maldonado, S., Sinha, V., McCune, S. K., Zajicek, A., Benjamin, D. K., Bucci-Rechtweg, C., Nelson, R. M. Tags: Special Section on Pediatrics Source Type: research

Enhancing Quality and Efficiency in Clinical Development Through a Clinical QMS Conceptual Framework: Concept Paper Vision and Outline
A quality management system (QMS) is an integrated framework through which organizations can systematically plan and achieve their quality objectives. While the International Conference on Harmonisation (ICH) Q10 provides clear guidance for QMS in the pharmaceutical manufacturing sector, there is no harmonized regulatory guidance describing a framework that provides an enterprise-wide view of achievement of clinical quality objectives, that is capable of being customized to fit an organization’s unique circumstances, and that accommodates the variability inherent in clinical development. In the absence of such guidan...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Meeker-O'Connell, A., Borda, M. M., Little, J. A., Sam, L. M. Tags: Sponsored Special Section by TransCelerate BioPharma Source Type: research

New Horizons in Pediatric Drug Development
(Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2015 Category: Drugs & Pharmacology Authors: Spielberg, S. P., Portman, R. Tags: Editorial Source Type: research

Choosing Appropriate Estimands in Clinical Trials (Leuchs et al): Letter to the Editor
(Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - June 29, 2015 Category: Drugs & Pharmacology Authors: Garrett, A., on behalf of the PSI/EFSPI Working Group on Estimands Quintiles, Reading, UK Tags: Letters to the Editor Source Type: research

Risk Minimization Activities and Measures of Effectiveness: A New Approach
(Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - June 29, 2015 Category: Drugs & Pharmacology Authors: Valentino, R. Tags: Letters to the Editor Source Type: research

Sample Size and the Precision of the Confidence Interval in Meta-analyses
Sample sizes affect the precision of the confidence interval for the common effect size in a meta-analysis, which includes a number of independent studies of varying sizes. This paper provides a simplified method to estimate the precision of the confidence interval for the common effect size by using the number of independent studies and the average sample size of the independent studies. The simplified method proves to be very accurate for the retrospective meta-analyses. Two examples are then given to show how an investigator can use the number of independent studies and the average sample size of the individual studies ...
Source: Therapeutic Innovation and Regulatory Science - June 29, 2015 Category: Drugs & Pharmacology Authors: Liu, X. S. Tags: Biostatistics Source Type: research

Choosing Appropriate Estimands in Clinical Trials
This study presents a process chart including all aspects to consider during planning. After deciding on the primary estimand, the trial design should be specified, followed by the primary analysis. Both should appropriately address the chosen estimand. Finally, sensitivity analyses should be taken into account. Provided are suggestions for all the planning steps involved, especially on choosing between efficacy and effectiveness, and relevant examples from clinical practice to illustrate them. It is recommended that one bear in mind the process chart during planning of any clinical trial and give reasonable justification ...
Source: Therapeutic Innovation and Regulatory Science - June 29, 2015 Category: Drugs & Pharmacology Authors: Leuchs, A.-K., Zinserling, J., Brandt, A., Wirtz, D., Benda, N. Tags: Biostatistics Source Type: research

Comparison of Approved Dosages for Pediatric and Adult Populations Based on US Labeling Information
Conclusion: The approved dosages for pediatrics based on BW were higher than those of adults, but the ones based on BSA were almost the same as those of adults. The closer the age groups, the stronger the correlation of relative dosages between the groups. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - June 29, 2015 Category: Drugs & Pharmacology Authors: Hanada, K., Kaneko, M., Narukawa, M. Tags: Special Populations Source Type: research