A Novel Collaborative Approach to Building Better Clinical Trials: New Insights From a Patient Engagement Workshop to Propel Patient-Centricity Forward.

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

authors： Lunawat S,Bhat K

更新日期：2020-09-01 00:00:00

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：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：:Lack of adherence to study protocol and missing data are often unavoidable in clinical trials, and both increase the need to differentiate between the ideal treatment effect if the medication is taken as directed and the treatment effect in presence of the actual adherence pattern. In this regard, estimands have becom...

journal_title：Therapeutic innovation & regulatory science

authors： Leuchs AK,Zinserling J,Brandt A,Wirtz D,Benda N

更新日期：2015-07-01 00:00:00

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

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

更新日期：2021-01-01 00:00:00

abstract：BACKGROUND:Although a large number of clinical trials have been conducted, the types of clinical trials that are scientifically influential, frequently utilized by society, and contribute to the progress of evidence-based medicine (EBM) have not been studied. Thus, we aimed to investigate the relationship between the c...

journal_title：Therapeutic innovation & regulatory science

authors： Noguchi Y,Kaneko M,Narukawa M

更新日期：2020-11-01 00:00:00

abstract：:A growing number of organizations-including pharmaceutical and biotechnology companies, foundations and associations-are routinely implementing patient advisory boards (PAB) given their high reported value for minimal relative investment. Organizations are typically implementing PABs to solicit patient voices and pers...

journal_title：Therapeutic innovation & regulatory science

authors： Anderson A,Getz KA

更新日期：2018-07-01 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：: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：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

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

更新日期：2018-11-01 00:00:00

abstract：BACKGROUND:This study compared the timing, regulatory marketing authorization decisions, and the final labeling for products submitted to Swissmedic to those submitted to European Medicines Agency (EMA) and the US Food & Drug Administration (FDA). METHODS:The Centre for Innovation in Regulatory Science (CIRS) conducte...

journal_title：Therapeutic innovation & regulatory science

authors： Dörr P,Wadworth A,Wang T,McAuslane N,Liberti L

更新日期：2016-11-01 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：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

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

abstract：BACKGROUND:(1) To assess the differing viewpoints of actual and potential adult clinical trial participants in 5 age cohorts, (2) identify misperceptions and opportunities to reduce unnecessary burdens on volunteers, and (3) provide age-specific feedback to study sponsors on their current engagement efforts. METHODS:B...

journal_title：Therapeutic innovation & regulatory science

authors： Anderson A,Borfitz D,Getz K

更新日期：2020-01-01 00:00:00

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

authors： Lui KJ

更新日期：2020-03-01 00:00:00

abstract：BACKGROUND:The pace of innovation, the creative engine of the pharmaceutical industry, has been variably described as stagnant, stable, or accelerating depending on the metric used for assessment or the quality of evidence. If the predominant perception holds that the speed of innovation is sluggish, pressure for chang...

journal_title：Therapeutic innovation & regulatory science

authors： Robberson M,Breder CD

更新日期：2020-08-31 00:00:00

abstract：BACKGROUND:Timely access to new medicines may be addressed through strengthening of registration efficiencies and timelines by establishing and refining value-added registration processes, resources, and systems. The aims of this study were to evaluate the timelines of the milestones of the South African review process...

journal_title：Therapeutic innovation & regulatory science

authors： Keyter A,Salek S,Gouws J,Banoo S,Walker S

更新日期：2020-07-01 00:00:00

abstract：BACKGROUND:Medical information (MI) departments across pharmaceutical companies respond to medical inquiries about a company's products to healthcare providers, payors, patients, and caregivers. Responses provided are constructed to be relevant, accurate, scientifically balanced and delivered in a timely manner. Benchm...

journal_title：Therapeutic innovation & regulatory science

authors： Byun J,Jong JA,Gangat A,Flowers T,Gazo A,Shah I

更新日期：2020-11-01 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：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：BACKGROUND:Several regulatory guidelines recommend that assessments of endpoints supporting drug approval should be verifiable by applicants and the regulatory agencies to minimize the potential for bias. This becomes especially critical when assessments are not based on measurable data but are derived from the interpr...

journal_title：Therapeutic innovation & regulatory science

authors： Krumholz-Bahner S,Garibbo M,Getz KA,Widler BE

更新日期：2015-11-01 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：:Data quality is critical for clinical trials to obtain robust conclusions about drug safety and efficacy evaluation. Effective data quality evaluation has been one of the major obstacles to new drug approvals in China, which hinders innovation in drug discovery and development ultimately. To improve the data quality s...

journal_title：Therapeutic innovation & regulatory science

authors： Wang J,Wang G,Li M,Han J,Zeng X,Pan J,Yang J

更新日期：2019-03-01 00:00:00

abstract：BACKGROUND:The influence of patient advocates on FDA regulatory decision making has increased. Despite enhanced engagement with FDA, there remain challenges to achieving the regulatory goals of patients within FDA's regulatory framework. Gaps exist between patient advocates' knowledge of the agency's processes and FDA'...

journal_title：Therapeutic innovation & regulatory science

authors： Kuehn CM

更新日期：2018-09-01 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：: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

authors： Cronin M,Berger S,Seligman PJ

更新日期：2014-11-01 00:00:00

abstract：:Adaptive designs are increasingly used in clinical trials. The Drug Information Association's Adaptive Design Scientific Working Group (ADSWG) works to foster collaboration among regulatory agencies, academia, and pharmaceutical and biotech companies to further the science of adaptive clinical development. The ADSWG S...

journal_title：Therapeutic innovation & regulatory science

authors： Morgan CC,Huyck S,Jenkins M,Chen L,Bedding A,Coffey CS,Gaydos B,Wathen JK

更新日期：2014-07-01 00:00:00

abstract：BACKGROUND:Increasing generic drug price competition by facilitating abbreviated new drug applications (ANDA) submission may help patients have access to affordable care. This study examined factors associated with first ANDA submission for the brand drug to be copied [the "reference listed drug" (RLD)]. METHODS:This ...

journal_title：Therapeutic innovation & regulatory science

authors： Wittayanukorn S,Rosenberg M,Schick A,Hu M,Wang Z,Babiskin A,Lionberger R,Zhao L

更新日期：2020-11-01 00:00:00

abstract：:After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Althoug...

journal_title：Therapeutic innovation & regulatory science

authors： Schulthess D,Baird LG,Trusheim M,Unger TF,Lumpkin M,Hoos A,Garner S,Gavin P,Goldman M,Seigneuret N,Chlebus M,Van Baelen K,Bergstrom R,Hirsch G

更新日期：2016-05-01 00:00:00

abstract：:Biopharmaceutical medicines are complex molecules obtained from a living organism (plant or animal cells) and may contain components of a living organism using biotechnology. Biosimilars are closely similar to already approved biopharmaceutical products that could form a new generation of medicines that are available ...

journal_title：Therapeutic innovation & regulatory science

authors： Sharma A,Khante S,Mahadik KR,Gaikwad VL

更新日期：2020-09-01 00:00:00