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

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

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

更新日期：2015-09-01 00:00:00

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

authors： Grignolo A,Siu A

更新日期：2019-05-01 00:00:00

abstract：BACKGROUND:The coherence between the relationship of QTc and drug plasma concentration (this relationship is measured through the slope) and ICH E14 findings based on hundreds of QT study reports was studied. RESULTS:Based on ICH E14 analysis, our findings indicate that if the slope was not positive, in most cases (86...

journal_title：Therapeutic innovation & regulatory science

authors： Zhang J,Chen H,Tsong Y,Stockbridge N

更新日期：2015-05-01 00:00:00

abstract：BACKGROUND:Several articles showed that statistical efficiency of multi-arm randomized clinical trials (RCTs) is much better than conventional two-arm RCTs. Multi-arm RCTs attract interest mainly when the experimental treatment regimen is not optimized or several pipelines under development exist. Breast cancer is a po...

journal_title：Therapeutic innovation & regulatory science

authors： Nomura S,Miyauchi Y,Ajisawa Y,Isogaya K,Sozu T

更新日期：2020-09-01 00:00:00

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

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

abstract：BACKGROUND:A new incentive program, "Premium Rewards for the Promotion of Innovative Drug Discovery and Resolution of Off-Label Use, etc," was introduced in Japan in 2010 and subsequently applied in 2010, 2012, and 2014. In our previous study, we reported statistical evidence regarding the attributes of drugs for which...

journal_title：Therapeutic innovation & regulatory science

authors： Shibata S,Uemura R,Suzuki T

更新日期：2016-01-01 00:00:00

abstract：:The QT interval is correlated with heart rate; therefore, the QT interval is usually corrected by heart rate when drug-induced QT effect is studied. Currently, there are many correction methods that use either fixed or data-driven approaches. The effectiveness of correction methods depends on many factors and varies f...

journal_title：Therapeutic innovation & regulatory science

authors： Dang Q,Zhang J

更新日期：2013-03-01 00:00:00

abstract：:Efficient use of limited pharmaceutical product development resources requires integrating multiple attributes, such as efficacy, safety, pharmacology, and so on, to decide at any stage whether the development of a product should proceed aggressively or slowly or be terminated. The decision process proceeds most effec...

journal_title：Therapeutic innovation & regulatory science

authors： Gould AL,Krishna R,Khan A,Saltzman J

更新日期：2015-03-01 00:00:00

abstract：:The U.S. and Japan have preapproval access systems for patients who have exhausted approved treatment options. We compare the systems of both countries and discuss some ethical concerns surrounding non-trial access to investigational drugs. Notable points of comparison of the two countries' systems include cost of non...

journal_title：Therapeutic innovation & regulatory science

authors： Nakada H,Folkers KM,Takashima K

更新日期：2020-10-23 00:00:00

abstract：BACKGROUND:Access and use of historical control data was identified as a top stakeholder concern across organizations according to results of a survey of needs and challenges related to nonclinical data conducted by the FDA/PhUSE Nonclinical Working Group in 2011. There is a perception there may be additional ways to c...

journal_title：Therapeutic innovation & regulatory science

authors： Mihalcik LM,Bausman M,Brown P,Everds N,Feldmann J,Henck J,Potenta D,Sims J,Zandee J

更新日期：2016-03-01 00:00:00

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

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

更新日期：2015-01-01 00:00:00

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

authors： Cohen O,Sax F

更新日期：2014-09-01 00:00:00

abstract：:The authors consider a statistically valid method that has little concern caused by down weighting in stage 2 or slight loss in efficiency and possibly improves power performance for a trial design that allows for adaptation of statistical information. In addition to the need for a priori sound data-based planning tha...

journal_title：Therapeutic innovation & regulatory science

authors： Wang SJ,Hung HMJ

更新日期：2014-01-01 00:00:00

abstract：OBJECTIVE:To identify, evaluate, and characterize the variety, quality, and intent of the health economics and outcomes research studies being conducted in SAARC (South Asian Association for Regional Cooperation) nations. METHODS:Studies published in English language between 1990 and 2015 were retrieved from Medline d...

journal_title：Therapeutic innovation & regulatory science

authors： Mehta M,Nerurkar R

更新日期：2018-05-01 00:00:00

abstract：BACKGROUND:A data monitoring committee (DMC) has special responsibilities for protecting the safety of clinical trial participants. Few guidance documents are available that address the operations and mechanics of establishing, serving on, or reporting to a DMC. This article provides a practical guide to sponsors, inst...

journal_title：Therapeutic innovation & regulatory science

authors： Bierer BE,Li R,Seltzer J,Sleeper LA,Frank E,Knirsch C,Aldinger CE,Levine RJ,Massaro J,Shah A,Barnes M,Snapinn S,Wittes J

更新日期：2016-09-01 00:00:00

abstract：BACKGROUND:The human papillomavirus (HPV) vaccine coverage is very low in Japan since the government suspended the active encouragement of the vaccination. We aimed to conduct a benefit-risk assessment of HPV vaccination and explore different consequent scenarios to identify potential improvements to the current Japane...

journal_title：Therapeutic innovation & regulatory science

authors： Matsumoto T,Matsumaru N,Scuffham P,Neels P,Tsukamoto K

更新日期：2021-01-01 00:00:00

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

authors： Koynova D,Lühmann R,Fischer R

更新日期：2013-07-01 00:00:00

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

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

更新日期：2015-07-01 00:00:00

abstract：BACKGROUND:The US Food and Drug Administration (FDA) put out a call for comments on new draft guidance for industry "Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA." This call for comments elicited 7 submissions from various organization...

journal_title：Therapeutic innovation & regulatory science

authors： Lance PT,Greenaway RV,Edwards B

更新日期：2019-03-01 00:00:00

abstract：BACKGROUND:Communicating the clinical impact of immunogenicity in labeling is important for safe and effective use of certain prescription products. Current U.S. Food and Drug Administration (FDA) guidance does not provide comprehensive recommendations on the communication of clinical impact of immunogenicity in labeli...

journal_title：Therapeutic innovation & regulatory science

authors： Guinn D,Madabushi R,Wang YM,Brodsky E,Zineh I,Maxfield K

更新日期：2020-11-01 00:00:00

abstract：BACKGROUND:Substance abuse research can raise ethical concerns about the comprehension and decision-making capacities of participants with drug dependence. In this study, the competence and willingness to consent to research participation were examined among patients with heroin dependence. METHODS:Twenty patients wit...

journal_title：Therapeutic innovation & regulatory science

authors： Zhao L,Shi H,Ying B,Li Q

更新日期：2020-09-01 00:00:00

abstract：:The DIA Study Endpoints Community Working Group on Mobile Sensor Technology (MST) series addresses considerations that may be useful for selecting MST for use in a clinical trial. This article describes considerations regarding the selection of MST for clinical trials including expectations around technology specifica...

journal_title：Therapeutic innovation & regulatory science

authors： Russell C,McCarthy M,Cappelleri JC,Wong S

更新日期：2021-01-01 00:00:00

abstract：:Risk-based monitoring (RBM) has disrupted the clinical trial industry, challenging conventional monitoring norms, business processes, and organizational structures. Endorsed by regulators and leading industry forums, and further driven by escalating drug development costs and enabling technology shifts making data ava...

journal_title：Therapeutic innovation & regulatory science

authors： Limaye N,Jaguste V

更新日期：2019-03-01 00:00:00

abstract：:Expanded access is a regulatory mechanism by which an investigational drug can be made available outside of a clinical trial to treat patients with serious or life-threatening conditions for which there are no satisfactory treatment options. An expanded access program (EAP) is the formal plan under which preapproval a...

journal_title：Therapeutic innovation & regulatory science

authors： Klopfenstein M,Van Campen LE,Garnett T

更新日期：2015-05-01 00:00:00

abstract：BACKGROUND:(1) A growing number of pharmaceutical and biotechnology organizations are engaging patients, their support networks, and clinical trial site staff at various touchpoints along the clinical research development spectrum to solicit feedback on how to reduce the burden of clinical trial participation and admin...

journal_title：Therapeutic innovation & regulatory science

authors： Gregg A,Getz N,Benger J,Anderson A

更新日期：2019-05-22 00:00:00

abstract：:In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is v...

journal_title：Therapeutic innovation & regulatory science

authors： Vaggelas A,Seimetz D

更新日期：2019-05-01 00:00:00

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

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

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

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

更新日期：2013-11-01 00:00:00

abstract：BACKGROUND:With the pharmaceutical industry's increased attention on enhancing patient experiences during their participation in clinical trials, the use of mobile nurse (MN) services to support the conduct of clinical trial assessments in the home (or in alternative locations other than in investigational sites) has b...

journal_title：Therapeutic innovation & regulatory science

authors： Stergiopoulos S,Eustace C,Stem K,Getz KA

更新日期：2016-07-01 00:00:00

abstract：BACKGROUND:The number of diabetes patients is steadily increasing worldwide. Consequently, the social burden of diabetes is huge, requiring urgent countermeasures. We performed an intensive survey of antidiabetic drugs approved in Japan, the United States, and the European Union. METHODS:Information about approved ant...

journal_title：Therapeutic innovation & regulatory science

authors： Tsukamoto K,Cnop M,Mori D,Kume S,Anazawa T,Doi M,Chikazawa K,Matsumaru N

更新日期：2019-07-01 00:00:00