An Assessment of Concerns Regarding New Regulatory Guidance for Combination Products: A Review of the Submissions Made to the FDA Regarding Their Proposed Draft New Guidance on Human Factors Studies for a Combination Product in an Abbreviated New Drug App

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：: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：: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：: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：: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: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：: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：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：: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: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：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: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：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：: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：:In general, similar to FDA and other health authorities, the PMDA requires clinical efficacy study(ies) to evaluate equivalence between a reference biological product and a Biosimilar product for new drug applications. Even if an identical clinical efficacy study is included in both of PMDA and FDA submissions, the co...

journal_title：Therapeutic innovation & regulatory science

authors： Asami Y,Pan J,Oh M,Sato A

更新日期：2020-09-01 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：: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

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

更新日期：2015-09-01 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: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: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: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：: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: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：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:(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：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: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: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：: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