当前位置： SCI文献检索 > Therapeutic Innovation & Regulatory Science期刊下所有文献 > Quantitative Extrapolation: An Approach to Validation of Adult Drug Efficacy in Pediatric Subjects.

Quantitative Extrapolation: An Approach to Validation of Adult Drug Efficacy in Pediatric Subjects.

Abstract：

:Confirmation of efficacy in pediatric drug development has traditionally required large, fully powered efficacy studies that have proven to have major feasibility and ethical challenges. Extrapolation of efficacy in the framework provided by the US Food and Drug Administration and European Medicines Agency is an appropriate solution when there is similarity of disease. When there is uncertainty regarding the degree of disease similarity, partial extrapolation may be utilized. The authors propose a more quantitative approach to partial extrapolation (ie, quantitative extrapolation), involving (1) integration of adult pharmacokinetic (PK), pharmacodynamic (PD), and clinical outcome data using pharmacometric models, (2) extrapolation using the adult pharmacometric model to predict PD and efficacy outcomes in pediatric subjects, and (3) validation of pediatric predictions with a streamlined plan of pediatric trials (ie, a quantitative extrapolation plan). A case study is presented for quantitative extrapolation using dipeptidyl peptidase 4 (DPP-4) inhibitors. In this example, the authors demonstrate how adult PK, PD, and HbA1c data can be integrated using a pharmacometric model for DPP-4 inhibitors with pediatric dose selection and efficacy validated with relatively few pediatric subjects.

journal_name

Ther Innov Regul Sci

journal_title

Therapeutic innovation & regulatory science

authors

Leil TA,Zee P,Suryawanshi S,Male C,Portman R

doi

10.1177/2168479013500286

subject

Has Abstract

pub_date

2013-09-01 00:00:00

pages

557-565

issue

5

eissn

2168-4790

issn

2168-4804

journal_volume

47

pub_type

杂志文章

相关文献

Therapeutic Innovation & Regulatory Science文献大全
  • New Benchmarks Characterizing Growth in Protocol Design Complexity.

    abstract：:The Tufts Center for the Study of Drug Development and Medidata Solutions Inc analyzed data from 9737 protocols and 130,601 investigative site contracts associated with these protocols to derive updated benchmarks characterizing protocol complexity. The results of the study indicate that protocol design complexity con...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479017713039

    authors： Getz KA,Campo RA

    更新日期：2018-01-01 00:00:00

  • Over-the-Counter Drugs: Regulatory Analysis of Warning Letters Between Fiscal Years 2015-2019.

    abstract：BACKGROUND:According to 21 Code of Federal Regulation (CFR) Part 211, Over-the-Counter (OTC) drug manufacturers must establish and follow current good manufacturing practices (cGMP) to produce quality products while meeting regulatory standards. The warning letters issued by the U.S. Food and Drug Administration (FDA) ...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-020-00231-2

    authors： Bai HK,Ahearn JD,Bartlett MG

    更新日期：2020-10-23 00:00:00

  • Rheumatoid Arthritis Disease Progression Modeling.

    abstract：:Time progression models provide a significant advantage in developing clinical trials and can also be used to elicit comparisons among therapeutic agents. The authors performed a meta-analysis to construct a time progression model for rheumatoid arthritis (RA), an area of significant interest for pharmaceutical develo...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479013499571

    authors： Reeve R,Pang L,Ferguson B,O'Kelly M,Berry S,Xiao W

    更新日期：2013-11-01 00:00:00

  • Complex Generic Products: Insight of Current Regulatory Frameworks in US, EU and Canada and the Need of Harmonisation.

    abstract：:The regulatory agencies all over the world have defined the pathway and regulations for the approval of simple small-molecule generics. In addition, the agencies are striving to form perspicuous regulatory frameworks for the approval of biosimilars. In this view, there are no defined regulations for the approval of co...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章,评审

    doi：10.1007/s43441-020-00114-6

    authors： Lunawat S,Bhat K

    更新日期：2020-09-01 00:00:00

  • Monitoring and Evaluating the Effect of Regulatory Action: Some Recent Case Studies.

    abstract：:Large electronic health care databases can be used to evaluate the effectiveness of regulatory action. Designing and interpreting such studies can be challenging. We demonstrate using case studies on modafinil, benzodiazepines, dosulepin, and piroxicam that measuring the impact of regulatory action is possible and can...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479015570333

    authors： Thomson A,Meeraus WH,Wong J,Suvarna R

    更新日期：2015-07-01 00:00:00

  • Technology Considerations to Enable the Risk-Based Monitoring Methodology.

    abstract：:TransCelerate BioPharma Inc developed a methodology based on the notion that shifting monitoring processes from an excessive concentration on source data verification to comprehensive risk-driven monitoring will increase efficiencies and enhance patient safety and data integrity while maintaining adherence to good cli...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479014546336

    authors： Barnes S,Katta N,Sanford N,Staigers T,Verish T

    更新日期：2014-09-01 00:00:00

  • Investigating Rates of Food and Drug Administration Approvals and Guidances in Drug Development: A Structural Breakpoint/Cointegration Timeseries Analysis.

    abstract：BACKGROUND:The number of original and supplemental ANDAs, BLAs, NDAs, and Biosimilars FDA drug/biologic approvals (Approvals) has risen dramatically in the recent years, incidentally, so has the number of issued FDA guidances (Guidances). It is hypothesized that if the structures of the two timeseries are similar and/o...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-020-00123-5

    authors： Daizadeh I

    更新日期：2020-09-01 00:00:00

  • Building an Integrated Early Clinical Development Platform to Improve the Path to Proof of Concept.

    abstract：:Probability of success in phase II dominates the drug development cost calculus, with phase I/II as the critical juncture for proof of concept. Failure to address fundamental pharmacologic questions in early development is alarmingly frequent and a strong predictor of failure. Safety, manufacture, formulation, and com...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479014526600

    authors： Cohen O,Sax F

    更新日期：2014-09-01 00:00:00

  • Baseline Assessment of the Evolving 2017 eClinical Landscape.

    abstract：:The volume and diversity of data collected to support each clinical study has increased dramatically in response to the rising scope and complexity of global drug development programs. The Tufts Center for the Study of Drug Development conducted an online survey of 257 unique global companies-77% drug development spon...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479018769292

    authors： Wilkinson M,Young R,Harper B,Machion B,Getz K

    更新日期：2019-01-01 00:00:00

  • A Data-Driven Approach to Risk-Based Source Data Verification.

    abstract：:Source data verification (SDV) is the process of confirming that reliable, accurate information collected from participants during a clinical trial has been reported successfully to the trial's sponsor by investigators conducting the study. Over the past 15 years or so, there has been considerable discussion in the li...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479013496245

    authors： Nielsen E,Hyder D,Deng C

    更新日期：2014-03-01 00:00:00

  • Regulatory Definitions and Good Pharmacovigilance Practices in Social Media: Challenges and Recommendations.

    abstract：:Social media presents new challenges to the biopharmaceutical industry for conducting pharmacovigilance activities. The authors reviewed worldwide regulatory guidance documents related to monitoring of adverse events posted on social media sites and identified gaps in current regulatory definitions for pharmacovigilan...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479015587362

    authors： Naik P,Umrath T,van Stekelenborg J,Ruben R,Abdul-Karim N,Boland R,Ibara M,Ekuta J,Washburn D,Stergiopoulos S

    更新日期：2015-11-01 00:00:00

  • New Drug and Biologics Approvals in 2019: A Systematic Analysis of Patient Experience Data in FDA Drug Approval Packages and Product Labels.

    abstract：BACKGROUND:The FDA Patient-Focused Drug Development Initiative was launched to ensure the incorporation of the patient voice into drug development and evaluation. Since 2017, the FDA must publish a statement outlining patient experience data (PED) considered in the approval of new drugs. This study investigated the pre...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-020-00244-x

    authors： Schultz-Knudsen K,Sabaliauskaite U,Hellsten J,Lassen AB,Morant AV

    更新日期：2020-11-23 00:00:00

  • A Practical Estimation Method for Analyzing Adverse Drug Reactions Using Data Mining.

    abstract：:This study aimed to determine the potentially severe chemical properties of drugs that can cause adverse drug reactions (ADRs) such as erythema multiforme (EM), Stevens-Johnson syndrome (SJS), and toxic epidermal necrolysis (TEN) by using a data mining method. The study data were extracted from the Adverse Event Repor...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/0092861512460759

    authors： Shirakuni Y,Okamoto K,Uejima E,Inui S,Takahara JI,Ohgaru T,Yamasaki H,Tian Y,Kawashita N,Inoue R,Yasunaga T,Takagi T

    更新日期：2013-03-01 00:00:00

  • Risk Evaluation and Mitigation Strategies With Elements to Assure Safe Use: Alignment of the Goals With the Tools to Manage Risk.

    abstract：:The Food and Drug Administration Amendments Act of 2007 gave the FDA the authority to require drug sponsors to submit a risk evaluation and mitigation strategies (REMS) program for those medicines with serious risks such that failure to effectively manage these risks would tip the benefit-risk balance. As of August 8,...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479014527749

    authors： Cronin M,Berger S,Seligman PJ

    更新日期：2014-11-01 00:00:00

  • ADHD & Pharmacotherapy: Past, Present and Future: A Review of the Changing Landscape of Drug Therapy for Attention Deficit Hyperactivity Disorder.

    abstract：:Attention deficit hyperactivity disorder (ADHD) is the most common neurobiological disorder in children, with a prevalence of ~6-7%1,2 that has remained stable for decades2. The social and economic burden associated with patients3, families, and broader systems (healthcare/educational) is substantial, with the annual ...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479015599811

    authors： Connolly JJ,Glessner JT,Elia J,Hakonarson H

    更新日期：2015-09-01 00:00:00

  • Building a Better Approach for the Benefit of Patients: 10 Pillars to Strengthen Regulatory Review Systems Globally.

    abstract：:In the current pharmaceutical regulatory environment, patients continue to benefit from great advances in medical care. Sophisticated regulatory review systems have also evolved to ensure that safe and effective medicines are approved. However, these systems are not optimized in all countries. Gaps in individual regul...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-019-00055-9

    authors： O'Brien J,Lumsden RS,Diehl DH,Macdonald JC

    更新日期：2020-03-01 00:00:00

  • Methods for Employing Information About Uncertainty of Ascertainment of Events in Clinical Trials.

    abstract：BACKGROUND:Uncertain ascertainment of events in clinical trials has been noted for decades. To correct possible bias, Clinical Endpoint Committees (CECs) have been employed as a critical element of trials to ensure consistent and high-quality endpoint evaluation, especially for cardiovascular endpoints. However, the ef...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-020-00206-3

    authors： Chen Y,Lawrence J,Hung HMJ,Stockbridge N

    更新日期：2021-01-01 00:00:00

  • Development and Validation of a Brief Measure of Self-Management Competence: The Self-Management Self-Test (SMST).

    abstract：BACKGROUND:Self-management can be considered a way of dealing with oneself and relates to actions undertaken to create order, discipline, and control. The concept is closely linked to concepts of self-efficacy and self-regulation but can be distinguished from these. The Self-Management Self-Test (SMST) is a 5-item asse...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479019849879

    authors： Wehmeier PM,Fox T,Doerr JM,Schnierer N,Bender M,Nater UM

    更新日期：2019-07-14 00:00:00

  • Improving Drug Development and Patient Access With the Right People, Processes, and Culture: What Needs to Happen Right Now to Bring Better Medicines to the Patients Who Need Them.

    abstract：:In a global environment where health care costs are soaring, R&D efforts are flatlining. Meanwhile, payers are demanding more value for their money. In this environment, the traditional siloed drug development model is not sustainable. Yet innovations such as adaptive trial designs, real-world data collection, precisi...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479018814224

    authors： Grignolo A,Siu A

    更新日期：2019-05-01 00:00:00

  • Implementing the Principle of the 3 Rs Through the Indian Pharmacopoeia.

    abstract：:Quality and safety tests are required for regulatory approval of drugs and pharmaceuticals in the country to guarantee minimum safety standards, and most of these tests include animal usage. In the case of biological medicines, these safety and quality tests have to be performed on a batch-to-batch basis and require a...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479015572371

    authors： Rastogi S,Kalaivani M,Bhatia AK,Prakash J,Singh GN

    更新日期：2015-09-01 00:00:00

  • A Universal Framework for the Benefit-Risk Assessment of Medicines: Is This the Way Forward?

    abstract：:A universal framework for the evaluation of the benefit-risk assessment of medicines during development by pharmaceutical companies and in the regulatory review by regulatory authorities is considered of value, as it would result in the systematic structured approach to support transparency in decision making. Several...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479014547421

    authors： Walker S,McAuslane N,Liberti L,Leong J,Salek S

    更新日期：2015-01-01 00:00:00

  • Considerations and Regulatory Challenges for Innovative Medicines in Expedited Approval Programs: Breakthrough Therapy and Sakigake Designation.

    abstract：BACKGROUND:In the breakthrough therapy designation (BTD) and Sakigake designation programs, rolling submission and close communication between applicants and regulatory authorities enable the timely access of patients to innovative medicines. However, challenges in the quality development, including chemistry, manufact...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-019-00019-z

    authors： Kajiwara E,Shikano M

    更新日期：2020-07-01 00:00:00

  • A Framework for Managing the Minimal Clinically Important Difference in Clinical Trials.

    abstract：:There is a long history of discussions about what is a minimal clinically important difference (MCID) and how this term applies to clinical research. This paper deals with a practical framework for MCID and its applicability to clinical trials. A literature review on the topic confirmed the fundamental role of MCID fo...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479013487541

    authors： Koynova D,Lühmann R,Fischer R

    更新日期：2013-07-01 00:00:00

  • Effective Partnering in Conducting Benefit-Risk Patient Preference Studies: Perspectives From a Patient Advocacy Organization, a Pharmaceutical Company, and Academic Stated-Preference Researchers.

    abstract：BACKGROUND:Formal incorporation of patients' perspectives is becoming increasingly important in medical product development and decision making. This article shares practical advice regarding how patient advocacy organizations, the pharmaceutical industry, and academic experts in stated-preference research can effectiv...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479017746404

    authors： Wolka AM,Fairchild AO,Reed SD,Anglin G,Johnson FR,Siegel M,Noel R

    更新日期：2018-07-01 00:00:00

  • Predicting Regulatory Product Approvals Using a Proposed Quantitative Version of FDA's Benefit-Risk Framework to Calculate Net-Benefit Score and Benefit-Risk Ratio.

    abstract：BACKGROUND:Approval of regulated medical products in the USA is based upon a rigorous review of the benefits and risks as performed by the US Food and Drug Administration (FDA) staff of scientists and is summarized in a descriptive and qualitative format called the FDA's Benefit-Risk Framework (BRF). This present metho...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-020-00197-1

    authors： Sun S,Heske S,Mercadel M,Wimmer J

    更新日期：2021-01-01 00:00:00

  • Global Pediatric Regulations: An Overview.

    abstract：:The purpose of the present review is to summarize the current pediatric regulatory requirements and also the regulatory efforts that need to be taken for the potential benefits of safety and efficacy to the pediatric patients. The importance of pediatric regulations came into existence as adult physiological condition...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章,评审

    doi：10.1007/s43441-019-00087-1

    authors： Gadge PM,Kenjale PP,Pokharkar VB,Gaikwad VL

    更新日期：2020-05-01 00:00:00

  • Outdated Prescription Drug Labeling: How FDA-Approved Prescribing Information Lags Behind Real-World Clinical Practice.

    abstract：BACKGROUND:Prescription drug labeling is an authoritative source of information that guides the safe and effective use of approved medications. In many instances, however, labeling may fail to be updated as new information about drug efficacy emerges in the postmarket setting. When labeling becomes outdated, it loses i...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479018759662

    authors： Shea MB,Stewart M,Van Dyke H,Ostermann L,Allen J,Sigal E

    更新日期：2018-11-01 00:00:00

  • Analyzing Upward Deviation of Actual vs Predicted Drug Sales in Japan for a Reasonable Drug-Pricing Policy.

    abstract：BACKGROUND:Predictions of drug sales play an important role in setting drug prices in Japan, where drug prices are highly regulated. One of 2 primary Japanese drug-pricing methodologies-the cost calculation method- refers extensively to the prediction of drug sales in establishing drug prices. Deviations from predicted...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1177/2168479019860123

    authors： Shibata S,Fukumoto D,Suzuki T,Ozaki K

    更新日期：2019-07-16 00:00:00

  • Exact Confidence Limits on Some New Measures of Concordance and Discordance in Binary Outcomes.

    abstract：:Using a measure of agreement that does not distinguish the "positive" outcome from the "negative" outcome can be sometimes misleading in assessing resemblance. To alleviate this concern, some new indices, including the "positive" and "negative" conditional synchrony measures (CSM) (or the conditional discordant measur...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-019-00074-6

    authors： Lui KJ

    更新日期：2020-03-01 00:00:00

  • Sentiment of Media Coverage and Reputation of the Pharmaceutical Industry.

    abstract：BACKGROUND:Patients and health care professionals receive information about pharmaceutical companies through various sources, including but not limited to print media, social media, and electronic media. The objective of this research was to benchmark the sentiment of electronic newspaper media coverage between 2014 an...

    journal_title：Therapeutic innovation & regulatory science

    pub_type： 杂志文章

    doi：10.1007/s43441-019-00048-8

    authors： Pampulevski V,Giaquinto JR,Rametta M,Toscani M,Barone J,Nadal JC

    更新日期：2020-01-01 00:00:00