scholarly journals EL2 CHANGES IN JAPANESE ACADEMIC CLINICAL TRIALS AND FRAMEWORKS FOR PLANNING CLINICAL TRIALS

2019 ◽  
Vol 1 (Supplement_2) ◽  
pp. ii4-ii4
Author(s):  
Kenichi Nakamura
Keyword(s):  
Clinical Trials ◽  
Public Funding ◽  
Drug Application ◽  
Scientific Validity ◽  
The Social ◽  
Social Scientific ◽  
The Cost ◽  
Scientific Value ◽  
New Drug Application ◽  
Research Budget

Abstract After the enforcement of the Japanese Clinical Trials Act, the number of investigator-initiated registration-directed trials (IIRDT, Chiken) is increasing while the number of non-registration academic trials is decreasing. Pharmaceutical companies tend to make an investment in IIRDT because the data derived from IIRDT can be utilized for new drug application for PMDA, which means the goals and return are clear for industries. On the other hand, the reason of the decrease of non-registration academic trials is the burden of cost and procedures specified in the Clinical Trials Act. In order to start academic trials, certain amount of research budget is indispensable due to the cost for certified review board and clinical trial insurance. Also, even minor changes of site information in jRCT should be submitted to certified review board and the hospital directors of all participating sites, which is one of the most serious burden for investigators. Confirmation of COI declaration in participating sites is another burden for investigators/sites. Under these circumstances, the number of non-registration academic trials will be decreasing for the time being. In the Clinical Trials Act era, investigators must prepare some budget to start clinical trials. In order to obtain public funding, social/scientific value and scientific validity are substantially important. To express the social value sufficiently, the purpose of the trial should focus not on the researcher’s interest but on the contribution for patients. In terms of scientific validity, the framework of PICO is useful; PICO means Patient, Intervention, Control and Outcome. Utilization of this framework and the consistency of these four factors are essential to make the trial design sound.

scholarly journals REVIEW ON SPONSOR / APPLICANT MEETINGS WITH FDA: HIGHLIGHTS OF NEW GUIDANCE OVER EXISTING

2018 ◽  
Vol 4 (2) ◽  
pp. 19-26
Author(s):  
Charmy Kothari ◽  
Kavina Shah
Keyword(s):  
Clinical Trials ◽  
Management Practices ◽  
Drug Application ◽  
Development Program ◽  
The United States ◽  
United States Department ◽  
Biological Products ◽  
New Drug ◽  
License Application ◽  
New Drug Application

The United States Department of Health and Human Services has a federal agency called the Food and Drug Administration (FDA or USFDA). A pre-planned assembling of two or more people who have been together for the purpose of getting a common goal via verbal interaction is called a formal meeting. During development stage of any drug or biological products pharmaceutical companies face trouble for both scientific and regulatory point of view, here role of formal meetings comes. Formal meetings between sponsor or applicant and FDA are usually related to development and review of drug and biological products. Center for Drug evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) regulates the formal meetings. These meetings are applicable to Pre – Investigational New Drug Application, Pre – Biologics License Application, New Drug Application for drugs and biological products and not applicable to Abbreviated New Drug Applications (ANDA), application of medical devices and submission of biosimilar biological products. Meetings between FDA and sponsor or applicant are for resolution of dispute, clinical holds discussion, Assessment of protocols of clinical trial, during clinical trials, in between clinical trials – at the phase 1 ending or at the phase 2 ending, to discuss development program. The FDA has classified these formal meetings in different types based on the nature of the request, the information in the meeting request and each meeting type is handled through different procedures. The principles of Good Meeting Management Practices (GMMPs) must be maintained. There are specific requirements and procedures to request, prepare, schedule, conduct and document formal meetings. As the guidance documents for meetings are revised by FDA, Change in procedure and requirements takes place. Any pharmaceutical company need to be in line with new guidance requirements to avoid rejection. Formal meetings between sponsor or applicant and FDA save time, cost and will increase the probability of product approval.

scholarly journals A Review on Multifunctional Excipients with Regulatory Considerations

2021 ◽  
pp. 189-201
Author(s):  
Arti Swami ◽  
Prajakta Chavan ◽  
Shivani Chakankar ◽  
Amol Tagalpallewar
Keyword(s):  
High Speed ◽  
Drug Application ◽  
Review Article ◽  
Drug Formulation ◽  
Active Ingredients ◽  
Time Saving ◽  
The Cost ◽  
New Drug Application ◽  
Development Methods ◽  
Very High

The practice of multifunctional excipients is gaining more and more attention as it simplifies the process of drug formulation by substituting the necessity of using mixture of many excipients. The multifunctional excipients are the class of excipients which includes pre-–processed and co-processed excipients and it provides added functionalities to the formulation. Functionality of an excipient is a useful property which helps in manufacturing and improves quality as well as applicability of the material. Researchers have identified that single component excipients may not always give the required results during development and manufacturing of definite API, hence they are concentrating to develop multifunctional excipients which will have improved quality that will fulfil the requirements of the formulation experts in terms of cost. The cost of new excipient development is very high as it demands toxicity studies, hence the industry is now focusing on co-processing of approved materials. The demand for directly compressible co-processed excipients has also increased due to the availability of high-speed tableting machines, time saving in Abbreviated New Drug Application (ANDA), simplified validation and stability of active ingredients. The intention of this review article is to highlight applicability and increasing attention focusing the benefits of co-processed excipients. Their advantages over conventional blend of excipients include development methods, testing and also highlighting their regulatory consideration.

scholarly journals Age disparity between a cancer population and participants in clinical trials submitted as a new drug application of anticancer drugs in Japan

Cancer ◽  
2007 ◽  
Vol 109 (12) ◽  
pp. 2541-2546 ◽  
Author(s):  
Akiko Hori ◽  
Taro Shibata ◽  
Masahiro Kami ◽  
Eiji Kusumi ◽  
Hiroto Narimatsu ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Anticancer Drugs ◽  
Drug Application ◽  
New Drug ◽  
New Drug Application ◽  
Age Disparity

scholarly journals Non-static framework for understanding adaptive designs: an ethical justification in paediatric trials

2021 ◽  
pp. medethics-2021-107263
Author(s):  
Michael OS Afolabi ◽  
Lauren E Kelly
Keyword(s):  
Clinical Trials ◽  
Cost Effective ◽  
Adaptive Designs ◽  
Research Subjects ◽  
Public And Private ◽  
Age Related ◽  
Public And Private Stakeholders ◽  
Social Scientific ◽  
Trial Efficiency ◽  
Scientific Value

Many drugs used in paediatric medicine are off-label. There is a rising call for the use of adaptive clinical trial designs (ADs) in responding to the need for safe and effective drugs given their potential to offer efficiency and cost-effective benefits compared with traditional clinical trials. ADs have a strong appeal in paediatric clinical trials given the small number of available participants, limited understanding of age-related variability and the desire to limit exposure to futile or unsafe interventions. Although the ethical value of adaptive trials has increasingly come under scrutiny, there is a paucity of literature on the ethical dilemmas that may be associated with paediatric adaptive designs (PADs). This paper highlights some of these ethical concerns around safety, scientific/social value and caregiver/guardian comprehension of the trial design. Against this background, the paper develops a non-static conceptual lens for understanding PADs. It shows that ADs are epistemically open and reduce some of the knowledge-associated uncertainties inherent in clinical trials as well as fast-track the time to draw conclusions about the value of evaluated drugs/treatments. On this note, the authors argue that PADs are ethically justifiable given they (1) have multiple layers of safety, exposing enrolled children to lesser potential risks, (2) create social/scientific value generally and for paediatric populations in particular, (3) specifically foster the flourishing of paediatric populations and (4) can significantly improve paediatric trial efficiency when properly designed and implemented. However, because PADs are relatively new and their regulatory, ethical and logistical characteristics are yet to be clarified in some jurisdictions, the cooperation of various public and private stakeholders is required to ensure that the interests of children, their caregivers and parents/guardians are best served while exposing paediatric research subjects to the most minimal of risks when they are enrolled in paediatric trials that use ADs.

scholarly journals CONVALESCENT PLASMA THERAPY FOR COVID-19 PATIENTS: REGULATORY GUIDANCE ON COLLECTION, TESTING, PROCESSING, STORAGE, DISTRIBUTION, AND CLINICAL TRIALS

2021 ◽  
pp. 6-14
Author(s):  
AMIT PORWAL ◽  
KAMLA PATHAK ◽  
DEVENDER PATHAK ◽  
RAMAKANT YADAV
Keyword(s):  
Clinical Trials ◽  
Systematic Approach ◽  
Drug Application ◽  
Plasma Therapy ◽  
Regulatory Guidance ◽  
Life Threatening ◽  
New Drug Application ◽  
Investigational New Drug Application ◽  
Storage Distribution ◽  
Experimental Stage

Convalescent plasma can be transfused to patients suffering from the same infection or for preparing immunoglobulin concentrates. Plasma obtained from recovered patients can be a valuable alternative during the COVID-19 pandemic for supporting its treatment within a randomized or case-control clinical trials or observational studies of plasma transfusion and for preparing plasma-derived biological products. WHO Blood Regulators Network highlighted that a systematic approach for collecting convalescent plasma from patients recovered from COVID-19 could provide a useful intervention. Structured clinical trials can be used to assess safety and effectiveness of convalescent plasma. The convalescent plasma therapy is still in the experimental stage and is currently not included in the interim clinical guidelines of WHO. However, an emergency investigational new drug application (eIND) process has been induced to ensure the availability of COVID-19 convalescent plasma to the patients with severe or life-threatening COVID-19 conditions. USFDA is regularly amending its guidance as new results, and best practices are emerging. The write-up provides an overview of convalescent plasma, from a regulatory considerations viewpoint, systematic workflow protocol, and a cross-section of clinical trials underway.

Stroke Prevention in Non-Valvular Atrial Fibrillation

1997 ◽  
Vol 17 (03) ◽  
pp. 166-169
Author(s):  
Judith O’Brien ◽  
Wendy Klittich ◽  
J. Jaime Caro
Keyword(s):  
Atrial Fibrillation ◽  
Clinical Trials ◽  
Care Home ◽  
Relevant Literature ◽  
Clinical Trial Data ◽  
Outcome Data ◽  
Sensitivity Analyses ◽  
Bleeding Events ◽  
Discharge Data ◽  
The Cost

SummaryDespite evidence from 6 major clinical trials that warfarin effectively prevents strokes in atrial fibrillation, clinicians and health care managers may remain reluctant to support anticoagulant prophylaxis because of its perceived costs. Yet, doing nothing also has a price. To assess this, we carried out a pharmacoe-conomic analysis of warfarin use in atrial fibrillation. The course of the disease, including the occurrence of cerebral and systemic emboli, intracranial and other major bleeding events, was modeled and a meta-analysis of the clinical trials and other relevant literature was carried out to estimate the required probabilities with and without warfarin use. The cost of managing each event, including acute and subsequent care, home care equipment and MD costs, was derived by estimating the cost per resource unit, the proportion consuming each resource and the volume of use. Unit costs and volumes of use were determined from established US government databases, all charges were adjusted using cost-to-charge ratios, and a 3% discount rate was applied to costs incurred beyond the first year. The proportions of patients consuming each resource were estimated by fitting a joint distribution to the clinical trial data, stroke outcome data from a recent Swedish study and aggregate ICD-9 specific, Massachusetts discharge data. If nothing is done, 3.2% more patients will suffer serious emboli annually and the expected annual cost of managing a patient will increase by DM 2,544 (1996 German Marks), from DM 4,366 to DM 6,910. Extensive multiway sensitivity analyses revealed that the higher price of doing nothing persists except for very extreme combinations of inputs unsupported by literature or clinical standards. The price of doing nothing is thus so high, both in health and economic terms, that cost-consciousness as well as clinical considerations mandate warfarin prophylaxis in atrial fibrillation.

Narrating Society: Enacting ‘Immigrant’ Characters through Negotiating, Naturalization, and Forgetting

2020 ◽  
Vol 1 (1) ◽  
Author(s):  
Sanne Boersma
Keyword(s):  
Scientific Work ◽  
Immigrant Integration ◽  
European Countries ◽  
Special Issue ◽  
Global Perspectives ◽  
Ethnographic Fieldwork ◽  
Scientific Institutions ◽  
The Social ◽  
Social Scientific ◽  
Media And Communication

This article scrutinizes how ‘immigrant’ characters of perpetual arrival are enacted in the social scientific work of immigrant integration monitoring. Immigrant integration research produces narratives in which characters—classified in highly specific, contingent ways as ‘immigrants’—are portrayed as arriving and never as having arrived. On the basis of ethnographic fieldwork at social scientific institutions and networks in four Western European countries, this article analyzes three practices that enact the characters of arrival narratives: negotiating, naturalizing, and forgetting. First, it shows how negotiating constitutes objects of research while at the same time a process of hybridization is observed among negotiating scientific and governmental actors. Second, a naturalization process is analyzed in which slippery categories become fixed and self-evident. Third, the practice of forgetting involves the fading away of contingent and historical circumstances of the research and specifically a dispensation of ‘native’ or ‘autochthonous’ populations. Consequently, the article states how some people are considered rightful occupants of ‘society’ and others are enacted to travel an infinite road toward an occupied societal space. Moreover, it shows how enactments of arriving ‘immigrant’ characters have performative effects in racially differentiating national populations and hence in narrating society. This article is part of the Global Perspectives, Media and Communication special issue on “Media, Migration, and Nationalism,” guest-edited by Koen Leurs and Tomohisa Hirata.

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