Statistical Considerations on Clinical Efficacy Studies of Biosimilar for PMDA Submission.

abstract：:The American Statistical Association and DIA have created an interdisciplinary working group of drug safety experts from academia, industry and regulatory backgrounds to explore the future direction for safety monitoring. This introduction to the series explains the background and rationale for this special section. ...

journal_title：Therapeutic innovation & regulatory science

authors： Seltzer JH,Li J,Wang W

更新日期：2019-03-29 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：BACKGROUND:Contending with a continuously expanding volume and variety of clinical data poses challenges and opportunities for the industry and clinical data management organizations. METHODS:Tufts CSDD conducted an online survey aimed at further quantifying and understanding the magnitude and impact that expanded dat...

journal_title：Therapeutic innovation & regulatory science

authors： Harper B,Wilkinson M,Indupuri R,Rocchio S,Getz K

更新日期：2020-09-14 00:00:00

abstract：:Much has changed in a relatively short period of time. There is a raging debate over the level of evidence expected to first introduce a treatment to patients based on smaller, more adaptive data sets. Some argue for less data followed by postapproval follow-up, others for more adaptive clinical trial designs and end-...

journal_title：Therapeutic innovation & regulatory science

authors： Pitts PJ,Le Louet H

更新日期：2018-07-01 00:00:00

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

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

更新日期：2019-07-14 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：: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: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：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：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: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：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: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

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

更新日期：2021-01-01 00:00:00

abstract：BACKGROUND:Package leaflets of medicines distributed within the European Union should use the template headings and standard texts created by the Working Group on the Quality Review of Documents (QRD). The following study investigated how the QRD template is implemented in this patient information. METHODS:All English...

journal_title：Therapeutic innovation & regulatory science

authors： Wolf A,Fuchs J,Schweim HG

更新日期：2016-01-01 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: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

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

更新日期：2019-07-16 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: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

authors： Kajiwara E,Shikano M

更新日期：2020-07-01 00:00:00

abstract：BACKGROUND:Obtaining assent from children participating in clinical trials acknowledges autonomy and developmental ability to contribute to the consent process. This critical step in pediatric drug development remains poorly understood, with significant room for improving the clarity, efficiency, and implementation of ...

journal_title：Therapeutic innovation & regulatory science

authors： Lombardi D,Squires L,Sjostedt P,Eichler I,Turner MA,Thompson C

更新日期：2018-01-01 00:00:00

abstract：:Recently, it is becoming increasingly difficult to develop innovative drugs. Thus, the role of regulatory science research in drug development and postmarketing settings has become more important. In this article, the authors discuss the roles of regulatory science research at the Pharmaceuticals and Medical Devices A...

journal_title：Therapeutic innovation & regulatory science

authors： Asahina Y,Tanaka A,Uyama Y,Kuramochi K,Maruyama H

更新日期：2013-01-01 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：: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

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

更新日期：2019-01-01 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: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：:Since 1992 many changes have occurred in the regulations, guidelines, and processes governing the FDA, the biomedical industry, other stakeholders, and their interactions. Of particular importance, the FDA Amendments Act of 2007 made public advisory committee meetings mandatory for new molecular entities and devices r...

journal_title：Therapeutic innovation & regulatory science

authors： McIntyre TD,Pappas M,DiBiasi JJ

更新日期：2013-01-01 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: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

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

更新日期：2020-11-23 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：:When registering food supplements in Russia, restrictions on the labeling and consumer leaflet contents are tightly controlled by the regulatory agencies. Thus, misleading advertising is the most difficult problem with the turnover of these products if information in the commercials does not correspond to the informat...

journal_title：Therapeutic innovation & regulatory science

authors： Lozda R

更新日期：2020-01-01 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