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

abstract：:Although much information is already available publically from information-sharing initiatives such as ClinicalTrials.gov, information about clinical programs is unstructured, inconsistent, and incomplete. Clinical research within bioscience companies, health care, academia, and governmental agencies could benefit fro...

journal_title：Therapeutic innovation & regulatory science

authors： Vasko L,Sundgren M,Bachmann P,Balinski K,Bleich N,Blom T,Eriksson H,Ferendo R,Forsberg K,King D,Mordiva A,Proeve J,Thomas L,Witch E,Simán M

更新日期：2015-01-01 00:00:00

abstract：BACKGROUND:Pediatric regulations enacted in both Europe and the USA have disrupted the pharmaceutical industry, challenging business and drug development processes, and organizational structures. Over the last decade, with science and innovation evolving, industry has moved from a reactive to a proactive mode, investin...

journal_title：Therapeutic innovation & regulatory science

authors： Severin T,Corriol-Rohou S,Bucci-Rechtweg C,An Haack K,Fuerst-Recktenwald S,Lepola P,Norjavaara E,Dehlinger-Kremer M,EFGCP Children’s Medicines Working Party.,Haertter S,Cheung SYA,IQ Pediatric Working Group.

更新日期：2020-09-01 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 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abstract：BACKGROUND:The US Food and Drug Administration (FDA) put out a call for comments on new draft guidance for industry "Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA."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: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：: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 2010 Congress established a regulatory pathway for the approval of biosimilar products in the United States. FDA has embarked on developing the implementation framework for this pathway which includes creating guidance to assist biosimilar manufacturers in the development of these products. However, to date, the gu...

journal_title：Therapeutic innovation & regulatory science

authors： Davis GC,Bowker GM

更新日期：2014-05-01 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：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：: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：: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：: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：: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：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：: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：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: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：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: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

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：: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 appro...

journal_title：Therapeutic innovation & regulatory science

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

更新日期：2013-09-01 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：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: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: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：:The product pipeline for diseases that disproportionately affect the developing world has considerably expanded over the last decade. Indeed, there are about 134 products for these diseases in the pipeline, including vaccines, drugs, diagnostics, microbicides, and vector control tools, and dozens of these products are...

journal_title：Therapeutic innovation & regulatory science

authors： Saidu Y,De Angelis D,Aiolli S,Stefano G,Georges AM

更新日期：2013-05-01 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：: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