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Implementation of Structured Benefit-Risk Assessments in Marketing Authorization Applications: Lessons Learned
Conclusions: Sponsors can apply these approaches to incorporate benefit-risk assessments into their marketing authorization applications. Further shared learnings and benchmarking among the pharmaceutical industry will be necessary to further advance the science and practice of benefit-risk assessment. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Wang, J., Wolka, A., Bullok, K., Anglin, G., Radawski, C., Noel, R. Tags: Regulatory Science Source Type: research

Adaptive Design Practice at the Center for Devices and Radiological Health (CDRH), January 2007 to May 2013
Adaptive designs have generated great interest in the clinical trial community as a result of their versatility and efficiency. Recently, the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration (FDA) surveyed all adaptive design applications submitted between 2007 and May 2013 for regulatory review. In this paper, we discuss the overall results and findings that emerged from an in-depth examination of the submissions. We summarize the current status of adaptive designs used in medical device studies. We also identify some of the lessons learned and common pitfalls that we encountered in...
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Yang, X., Thompson, L., Chu, J., Liu, S., Lu, H., Zhou, J., Gomatam, S., Tang, R., Zhao, Y., Ge, Y., Gray, G. W. Tags: Regulatory Science Source Type: research

Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period
Conclusions: The expanded access program is viewed as a success from FDA’s perspective based on the large number of applications processed and allowed to proceed each year. However, the actual number of patients and their health care providers that desire drug treatments available under expanded access is not known. It is exceedingly rare for a serious adverse event under expanded access to affect the development program for that drug. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Jarow, J. P., Lemery, S., Bugin, K., Khozin, S., Moscicki, R. Tags: Regulatory Science Source Type: research

An FDA Analysis of Formal Dispute Resolution in the Center for Drug Evaluation and Research: 2003 Through 2014
Scientific and/or medical disputes will inevitably arise with regard to the US Food and Drug Administration’s (FDA’s) decision making related to drug development, new drug review, generic drug review, and postmarketing oversight. As these disputes can involve complex judgments and issues that are scientifically and commercially important, it is critical that FDA have procedures for effective and efficient resolution. FDA regulations allow a sponsor to obtain a review of an FDA decision by submitting a request for formal dispute resolution (an appeal). FDA’s Center for Drug Evaluation and Research (CDER) r...
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Sharma, K., Harrington, A., Worrell, S., Bertha, A. Tags: Regulatory Science Source Type: research

Optimizing the Use of Electronic Data Sources in Clinical Trials: The Landscape, Part 1
Conclusions: Based on survey results, companies generally have taken steps to leverage current eSource technologies and prepare for optimal utilization of electronic data sources. The TransCelerate eSource Initiative will continue to evaluate the technology, regulatory, standards, and health care landscape to support the goal of improving global clinical science and global clinical trial execution. Forthcoming publications will focus on technology landscape, future vision, and demonstration projects. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Kellar, E., Bornstein, S. M., Caban, A., Celingant, C., Crouthamel, M., Johnson, C., McIntire, P. A., Milstead, K. R., Patterson, J. K., Wilson, B. Tags: Sponsored Special Section by TransCelerate BioPharma Source Type: research

Glory Enough for All
(Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - October 23, 2016 Category: Drugs & Pharmacology Authors: Spielberg, S. P. Tags: Editorial Source Type: research

Product Development Under FDAs Animal Rule: Understanding FDAs Expectations and Potential Implications for Traditional Development Programs
This article reviews how different products met the requirements for licensure under the Animal Rule, based on information publicly available on FDA’s website. The primary aim of this manuscript is to offer an understanding of FDA’s interpretation of relevant regulations and guidances in the context of this licensure pathway. Some of the methods used for Animal Rule approvals may also have potential application in more traditional development programs. Thus, this article may also offer insight into methods for accelerating product development in general. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Allio, T. Tags: Regulatory Science Source Type: research

Responsibilities of Data Monitoring Committees: Consensus Recommendations
Conclusions: The group recommended practices for each phase of the DMC process from inception through execution of a clinical trial, with appropriate considerations for confidentiality. The group’s practical guidance should assist in comprehensive oversight of appropriate clinical trials and should help DMC members execute their obligations with greater assurance. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Bierer, B. E., Li, R., Seltzer, J., Sleeper, L. A., Frank, E., Knirsch, C., Aldinger, C. E., Levine, R. J., Massaro, J., Shah, A., Barnes, M., Snapinn, S., Wittes, J. Tags: Clinical Trials Source Type: research

A Comparative Review of Waivers Granted in Pediatric Drug Development by FDA and EMA From 2007-2013
Conclusion: Despite the different legal frameworks, criteria, and processes of determination, the waiver decisions of the 2 agencies were similar in the majority of cases. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Egger, G. F., Wharton, G. T., Malli, S., Temeck, J., Murphy, M. D., Tomasi, P. Tags: Special Populations Source Type: research

Nonclinical Safety Considerations for the Development of Pediatric-First Drugs: An Industry View
This paper provides considerations on approaches to the development of medicines initially developed for pediatric use (ie, "pediatric-first" or "pediatric-only" drugs). The most common development approach for these types of medicines involves a first-in-human (FIH) clinical trial with healthy adult volunteers to assess safety and tolerability. This approach generally requires nonclinical repeat-dose studies in adult animals; safety pharmacology and in vivo genetic toxicology studies in adult animals are also performed for small-molecule drugs. Additional studies in juvenile animals may be required prior to clinical trial...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Schmitt, G., Ridings, J., De Schaepdrijver, L., van Doesum-Wolters, F. L. C., Cappon, G. D., Hartmann, A. Tags: Special Populations Source Type: research

Pragmatic Multicriteria Decision Analysis (MCDA) Combined With Advanced Pharmacoepidemiology for Benefit-Risk Assessments of Medicines Adapted to the Real-Life Constraints of Regulators: Development and Case Study
Conclusions: The approach allows quantification and visualization of benefit-risk over time and across comparators. Combination of pragmatic MCDA designed to integrate criteria beyond benefit-risk and advanced statistics supports application of MCDA to further accountable benefit-risk assessments for real-life decision making. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Goetghebeur, M. M., Wagner, M., Nikodem, M., Zyla, A., Micaleff, A., Amzal, B. Tags: Statistics Source Type: research

The Impact of Adaptive Design on Portfolio Optimization
Conclusions: It is critical to minimize missed opportunities to initiate new promising trials, and to increase sample size only in regions that promise meaningful improvements in power. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Antonijevic, Z. Tags: Statistics Source Type: research

Attitudes Toward Clinical Trials Among Physicians in China With Different Levels of Experience
Conclusions: Our results revealed that physicians with different positional titles perceived different benefits and difficulties in conducting clinical trials. Junior physicians need more administrative assistance in clinical trials and related training. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Zheng, H., Wang, L., Wu, H., Wang, M., Sun, H. Tags: Global Perspectives Source Type: research

Regulation of New Drug Approval in Taiwan
This article summarizes the efforts TFDA has been making in the domain of medicinal product management, highlighting policies and strategies for the future. (Source: Therapeutic Innovation and Regulatory Science)
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Yang, Y.-T., Huang, H.-W., Chen, Y.-T., Chiang, Y.-M., Tzou, M.-C. Tags: Global Perspectives Source Type: research

The Early Engagement Model in Product Development: Linking "Proof of Concept" to "Proof of Medical Value"
In a rapidly changing health care environment, it is more important than ever that pharmaceutical manufacturers improve the quality and efficiency of their research and development efforts in order to help ensure the right drug gets to the right patient at the right time. The evolving role of the Medical Affairs, Health Economics & Outcomes Research (HEOR) and other functions engaged in evidence generation within the pharmaceutical industry is leading to earlier involvement in the clinical development process so that the proof of concept for new therapies can be more strongly linked to the proof of medical value. In th...
Source: Therapeutic Innovation and Regulatory Science - August 25, 2016 Category: Drugs & Pharmacology Authors: Iqbal, S. U., Salimi, T., Dunlop, J., Paramore, L. C. Tags: Product Development and Innovation Source Type: research