scholarly journals Data silos are undermining drug development and failing rare disease patients

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Nathan Denton ◽  
Monique Molloy ◽  
Samantha Charleston ◽  
Craig Lipset ◽  
Jonathan Hirsch ◽  
...  
Keyword(s):  
Natural History ◽  
Drug Development ◽  
Research And Development ◽  
Rare Disease ◽  
National Institutes Of Health ◽  
Future Research ◽  
Development Activity ◽  
Redundant Data ◽  
Vulnerable Patients ◽  
Disease Specific

AbstractData silos are proliferating while research and development activity explode following genetic and immunological advances for many clinically described disorders with previously unknown etiologies. The latter event has inspired optimism in the patient, clinical, and research communities that disease-specific treatments are on the way. However, we fear the tendency of various stakeholders to balkanize databases in proprietary formats, driven by current economic and academic incentives, will inevitably fragment the expanding knowledge base and undermine current and future research efforts to develop much-needed treatments. The proliferation of proprietary databases, compounded by a paucity of meaningful outcome measures and/or good natural history data, slows our ability to generate scalable solutions to benefit chronically underserved patient populations in ways that would translate to more common diseases. The current research and development landscape sets too many projects up for unnecessary failure, particularly in the rare disease sphere, and does a grave disservice to highly vulnerable patients. This system also encourages the collection of redundant data in uncoordinated parallel studies and registries to ultimately delay or deny potential treatments for ostensibly tractable diseases; it also promotes the waste of precious time, energy, and resources. Groups at the National Institutes of Health and Food and Drug Administration have started programs to address these issues. However, we and many others feel there should be significantly more discussion of how to coordinate and scale registry efforts. Such discourse aims to reduce needless complexity and duplication of efforts, as well as promote a pre-competitive knowledge ecosystem for rare disease drug development that cultivates and accelerates innovation.

scholarly journals Bayesian modeling and simulation to inform rare disease drug development early decision-making: application to Duchenne muscular dystrophy

2021 ◽  
Author(s):  
Janelle L. Lennie ◽  
John T. Mondick ◽  
Marc R. Gastonguay
Keyword(s):  
Decision Making ◽  
Natural History ◽  
Drug Development ◽  
Rare Disease ◽  
Bayesian Model ◽  
Short Term ◽  
Decision Accuracy ◽  
Model Based ◽  
Term Trial ◽  
Frequentist Method

AbstractRare disease clinical trials are constrained to small sample sizes and may lack placebo-control, leading to challenges in drug development. This paper proposes a Bayesian model-based framework for early go/no-go decision making in rare disease drug development, using Duchenne muscular dystrophy (DMD) as an example. Early go/no-go decisions were based on projections of long-term functional outcomes from a Bayesian model-based analysis of short-term trial data informed by prior knowledge based on 6MWT natural history literature data in DMD patients. Frequentist hypothesis tests were also applied as a reference analysis method. A number of combinations of hypothetical trial designs, drug effects and cohort comparison methods were assessed.The proposed Bayesian model-based framework was superior to the frequentist method for making go/no-go decisions across all trial designs and cohort comparison methods in DMD. The average decision accuracy rates across all trial designs for the Bayesian and frequentist analysis methods were 45.8 and 8.98%, respectively. A decision accuracy rate of at least 50% was achieved for 42 and 7% of the trial designs under the Bayesian and frequentist analysis methods, respectively. The frequentist method was limited to the short-term trial data only, while the Bayesian methods were informed with both the short-term data and prior information. The specific results of the DMD case study were limited due to incomplete specification of individual-specific covariates in the natural history literature data and should be reevaluated using a full natural history dataset. These limitations aside, the framework presented provides a proof of concept for the utility of Bayesian model-based methods for decision making in rare disease trials.

Technology Transfer in Pharmaceutical Manufacturing - A Review

2018 ◽  
Vol 9 (3) ◽  
Author(s):  
Amit B Patil ◽  
Bharath Kumar B ◽  
Ajay P Karnalli
Keyword(s):  
Technology Transfer ◽  
Drug Development ◽  
Research And Development ◽  
Product Quality ◽  
Pharmaceutical Product ◽  
Pharmaceutical Manufacturing ◽  
Shop Floor ◽  
R And D ◽  
Production Shop ◽  
Execution Procedures

Technology Transfer (TT) is vital action from drug development in Research and Development (R and D) Department to commercial manufacturing till the product discontinuation. This review is an attempt to give an insight about the transfer of pharmaceutical product from R and D to production including necessary documents required to review the supporting documents and execution procedures in production shop floor. TT is considered effective, if there is a documented evidence that the process and its parameters, repeatedly results in desired product quality which was established upon during TT between the transferee and transferor. For the execution of TT process, expertise from different department such as Engineering, R and D, QA, process analyst and production are teamed. the transmission comprises of arrangements procured in these flows of improvement to achieve the quality as planned throughout manufacture.

A Case Study: Analysis of Patents of Coronaviruses and Covid-19 for Technology Assesment and Future Research

2020 ◽  
Vol 26 ◽  
Author(s):  
Pankaj Musyuni ◽  
Geeta Aggarwal ◽  
Manju Nagpal ◽  
Ramesh K. Goyal
Keyword(s):  
Disease Management ◽  
Research And Development ◽  
Technological Development ◽  
Research Work ◽  
Large Family ◽  
Future Research ◽  
Biological Drugs ◽  
Ongoing Research ◽  
Patent Literature ◽  
Patent Documents

Background: Protecting intellectual property rights are important and particularly pertinent for inventions which are an outcome of rigorous research and development. While the grant of patents is subject to establishing novelty and inventive step, it further indicates the technological development and helpful for researchers working in the same technical domain. The aim of the present research work is to map the existing work through analysis of patent literature, in the field of Coronaviruses (CoV), particularly COVID-19 (2019-nCoV). CoV is a large family of viruses known to cause illness in human and animals, particularly known for causing respiratory infections as evidenced in earlier times such as in MERS i.e. Middle East Respiratory Syndrome; SRS i.e. Severe Acute Respiratory Syndrome. A recently identified novel-coronavirus has known as COVID-19 which has currently caused pandemic situation across the globe. Objective: To expand analysis of patents related to CoV and 2019-nCoV. Evaluation has been conducted by patenting trends of particular strains of identified CoV diseases by present legal status, main concerned countries via earliest priority years and its assignee types and inventors of identified relevant patents. We analyzed the global patent documents to check the scope of claims along with focuses and trends of the published patent documents for the entire CoV family including 2019- nCoV through the present landscape. Methods: To extract the results, Derwent Innovation database is used by a combination of different key-strings. Approximately 3800 patents were obtained and further scrutinized and analyzed. The present write-up also discusses the recent progress of patent applications in a period of the year 2010 to 2020 (present) along with the recent developments in India for the treatment options for CoV and 2019-nCoV. Results: Present analysis showed that key areas of the inventions have been focused on vaccines and diagnostic kits apart from the composition for treatment of CoV. We also observed that no specific vaccine treatments is available for treatment of 2019-nCov, however, developing novel chemical or biological drugs and kits for early diagnosis, prevention and disease management is the primarily governing topic among the patented inventions. The present study also indicates potential research opportunities for the future, particularly to combat 2019-nCoV. Conclusion: The present paper analyzes the existing patents in the field of Coronaviruses and 2019-nCoV and suggests a way forward for the effective contribution in this upcoming research area. From the trend analysis, it was observed an increase in filing of the overall trend of patent families for a period of 2010 to the current year. This multifaceted analysis of identified patent literature provides an understanding of the focuses on present ongoing research and grey area in terms of the trends of technological innovations in disease management in patients with CoV and 2019-nCoV. Further, the findings and outcome of the present study offer insights for the proposed research and innovation opportunities and provide actionable information in order to facilitate policymakers, academia, research driven institutes and also investors to make better decisions regarding programmed steps for research and development for the diagnosis, treatment and taking preventive measures for CoV and 2019-nCoV. The present article also emphasizes on the need for future development and the role of academia and collaboration with industry for speedy research with a rationale.

scholarly journals Identification of Technology Diffusion by Citation and Main Paths Analysis: The Possibility of Measuring Open Innovation

2021 ◽  
Vol 7 (1) ◽  
pp. 104
Author(s):  
Wei-Hao Su ◽  
Kai-Ying Chen ◽  
Louis Y. Y. Lu ◽  
Ya-Chi Huang
Keyword(s):  
Research And Development ◽  
Technology Development ◽  
Surgical Navigation ◽  
Future Research ◽  
Integrated Analysis ◽  
Reference Network ◽  
Research Topics ◽  
Academic Literature ◽  
Development Trajectory ◽  
Historic Development

This study collected literature on augmented reality (AR) from academic and patent databases to plot the historic development trajectory of AR and forecast its future research and development trends. A total of 3193 and 13,629 papers were collected from academic and patent databases, respectively. First, a network was established using references from the academic literature; main path analysis was conducted on this reference network to plot the overall development trajectory. Subsequent cluster and word cloud analyses revealed the following five major groups of AR research topics: AR surgical navigation applications, AR education applications, AR applications in manufacturing, AR applications in architecture, and AR applications in visual tracking. Subsequently, the relationships between the overall development trajectory and the five AR research topics were compared. Next, the title and abstract of AR-related academic and patent papers were subjected to text mining to identify keywords with a high frequency of occurrence. The results can provide a reference for industry, government, and academia when planning future development strategies for the AR field. This research adopted an integrated analysis procedure to plot the trajectory of AR technology development and applications successfully and effectively, predict future patent research and development directions and produce technological forecasts.

scholarly journals Endocrine Disrupting Chemicals and Thyroid Cancer: An Overview

Toxics ◽  
2021 ◽  
Vol 9 (1) ◽  
pp. 14
Author(s):  
Mathilda Alsen ◽  
Catherine Sinclair ◽  
Peter Cooke ◽  
Kimia Ziadkhanpour ◽  
Eric Genden ◽  
...  
Keyword(s):  
Thyroid Cancer ◽  
Flame Retardants ◽  
Endocrine Disrupting Chemicals ◽  
National Institutes Of Health ◽  
Future Research ◽  
National Library ◽  
Comprehensive Overview ◽  
Carcinogenic Effect ◽  
Increased Risk ◽  
Endocrine Disrupting

Endocrine disruptive chemicals (EDC) are known to alter thyroid function and have been associated with increased risk of certain cancers. The present study aims to provide a comprehensive overview of available studies on the association between EDC exposure and thyroid cancer. Relevant studies were identified via a literature search in the National Library of Medicine and National Institutes of Health PubMed as well as a review of reference lists of all retrieved articles and of previously published relevant reviews. Overall, the current literature suggests that exposure to certain congeners of flame retardants, polychlorinated biphenyls (PCBs), and phthalates as well as certain pesticides may potentially be associated with an increased risk of thyroid cancer. However, future research is urgently needed to evaluate the different EDCs and their potential carcinogenic effect on the thyroid gland in humans as most EDCs have been studied sporadically and results are not consistent.

A systematic review of the evidence supporting post-operative medication use in congenital heart disease

2021 ◽  
pp. 1-27
Author(s):  
Elizabeth J. Thompson ◽  
Henry P. Foote ◽  
Caitlin E. King ◽  
Sabarish Srinivasan ◽  
Elizabeth C. Ciociola ◽  
...  
Keyword(s):  
Cardiopulmonary Bypass ◽  
Adverse Events ◽  
Drug Development ◽  
Drug Disposition ◽  
Controlled Vocabulary ◽  
Coagulation System ◽  
Future Research ◽  
Pulmonary Vasodilators ◽  
Post Operative Period ◽  
Operative Care

Abstract Background: Targeted drug development efforts in patients with CHD are needed to standardise care, improve outcomes, and limit adverse events in the post-operative period. To identify major gaps in knowledge that can be addressed by drug development efforts and provide a rationale for current clinical practice, this review evaluates the evidence behind the most common medication classes used in the post-operative care of children with CHD undergoing cardiac surgery with cardiopulmonary bypass. Methods: We systematically searched PubMed and EMBASE from 2000 to 2019 using a controlled vocabulary and keywords related to diuretics, vasoactives, sedatives, analgesics, pulmonary vasodilators, coagulation system medications, antiarrhythmics, steroids, and other endocrine drugs. We included studies of drugs given post-operatively to children with CHD undergoing repair or palliation with cardiopulmonary bypass. Results: We identified a total of 127 studies with 51,573 total children across medication classes. Most studies were retrospective cohorts at single centres. There is significant age- and disease-related variability in drug disposition, efficacy, and safety. Conclusion: In this study, we discovered major gaps in knowledge for each medication class and identified areas for future research. Advances in data collection through electronic health records, novel trial methods, and collaboration can aid drug development efforts in standardising care, improving outcomes, and limiting adverse events in the post-operative period.

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

Transnational Corporations as the Driving Forces Behind the World's Research and Development Activity in Terms of R&D Expenditure

2019 ◽  
pp. 73-97
Author(s):  
Kamil M. Kraj
Keyword(s):  
Comparative Analysis ◽  
Research And Development ◽  
Driving Forces ◽  
Dominant Role ◽  
Science And Technology ◽  
Transnational Corporations ◽  
Financial Data ◽  
International Level ◽  
Development Activity

As discussed in the literaturę, more and more transnational corporations (TNCs) were attaching importance to research and development (R&D) activity from the 1970s through the 2000s. This growing involvement of TNCs in R&D resulted in their dominant role in global R&D expenditure. Indeed, a comparative analysis of financial data collected for the group of the 102 largest corporate R&D spenders worldwide in 2007 showed that this group of TNCs accounted for a significant share of the worlds R&D expenditure not only in 2007 alone but also in the period of 2000-2011. Moreover, a similarity between their home countries and the countries being top R&D spenders was found; however, most of these corporations were conducting their R&D at international level. Furthermore, the analysed TNCs operated mostly in technology-intensive industries, for which the foun- dations were provided by a multidisciplinary science and technology basis.

Degradation Mode of PBAT Mulching Film and Control Methods During Its Degradation Induction Period

2021 ◽  
Vol 18 ◽  
Author(s):  
Runmeng Qiao ◽  
Xin Wang ◽  
Guangjiong Qin ◽  
Jialei Liu ◽  
Aocheng Cao ◽  
...  
Keyword(s):  
Research And Development ◽  
Biodegradable Polymers ◽  
Large Scale ◽  
Degradation Process ◽  
Future Research ◽  
Plastic Film ◽  
Plastic Mulching ◽  
Controllable Degradation ◽  
Mulching Film ◽  
Agricultural Film

: The plastic film plays an important role in China's agricultural production. However, the large-scale use of plastic film has also produced a very serious problem of agricultural film pollution. Biodegradable polymers have attracted much attention because of the environmental pollution caused by traditional plastic mulching film. The most typical one is poly (butylene adipate co butylene terephthalate, PBAT). Poly (Butylene Adipate-co-Terephthalate) (PBAT) is a kind of aliphatic–aromatic polyesters with excellent biodegradability and mechanical processing properties. Therefore, it has been rapidly developed and widely used in the industry. However, there are clear requirements for the degradation period of agricultural film. At present, the degradable materials available on the market are difficult to meet the requirements of all crops for their degradation period. In this paper, the basic properties，degradation process and ways to delay the degradation of PBAT are reviewed to improve the degradation period of plastic film prepared by using this kind of material. Among them, the degradation process includes photodegradation, biodegradation and hydrolysis. The ways to delay the degradation include adding chain extender, light stabilizer, anti-hydrolysis agent and antibacterial agent. These can provide a theoretical basis for the research and development of biodegradable film with controllable degradation cycle. The future research and development of biodegradable polymers mainly focus on controllable degradation rate, stable degradation cycle, new materials and reducing research and development costs.

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