• Planning your PIP submission
• Which in-house departments should be involved?
• Interaction with CRO/ writer
• Interaction with PDCO
• Key points for successful PIP outsourcing
Planning your Paediatric Investigation Plan (PIP) Submission in Europejbarag
During this presentation, Dr. Susan Bhatti, an experienced regulatory affairs professional, shared best practices and experiences learned from submitting PIPs. This included a brief review of the pediatric regulation requirements, insight for interacting with PDCO, and an overview of the PIP submission including procedures, timelines, structure and compliance.
Walter King is a seasoned senior R&D executive with over 30 years of experience in medical device and diagnostic product development. He has a proven track record of successfully directing projects from research through product launch on time and on budget. Key accomplishments include overseeing development of personalized diagnostic tests and medical devices that were paramount in the sale of two companies. He possesses strong leadership skills and experience managing multi-disciplinary teams across major companies.
This document summarizes the global regulatory procedures for promotional review of materials produced by ExL Pharma's global marketing team. It discusses what materials are in scope for review, the membership and standards of the Global Promotional Review Committee, and the responsibilities of global reviewers to ensure materials are consistent with core data sheets and scientifically accurate. It also reviews standards from the IFPMA, EFPIA, and ABPI codes and the process for escalation, training, self-regulation, and resources on promotional codes and regulations.
This document provides a summary of Maria Bloodgood's professional experience and qualifications. She has over 20 years of experience in project management roles in the pharmaceutical industry, focusing on clinical development and medical affairs. Her most recent role is as a Global Project Manager at Eurofins Central Laboratory, where she is responsible for managing projects and ensuring they are completed on time, on budget, and meeting quality standards. She has extensive experience managing clinical studies and programs across multiple therapeutic areas.
SMi Group's Prefilled Syringes East Coast 2018 conferenceDale Butler
This document summarizes a two-day conference on pre-filled syringes taking place in Boston on April 10-11, 2018. The conference will include two pre-conference workshops on human factors for connected drug delivery systems and design considerations for drug delivery outside the clinic. The main conference will focus on challenges and solutions for PFS manufacturing and processes, integrating quality by design principles, and trends toward increasing biologics and their implications for PFS. It will feature speakers from major pharmaceutical companies and include panel discussions on challenges of biologics delivery and PFS development.
ExL Pharma Clinical Trials Phase I and Phase IIa Conference Brochure: Phase 1...bryonmain
There is a pill or treatment for almost everything, or at least, that is how it seems. However, the amount of effort that goes into a pill or treatment before it is launched is extensive, expensive and often inefficient.
Efficiency and innovation go hand-in-hand with R&D and the development of clinical trials, however, FDA regulations and clinical trial standardization end up stifling these two key factors. This leads to drawn out processes that cost companies hundreds of millions of dollars before the drugs hit the market. Efforts have been made to increase efficiency in phase I/IIA with some companies changing their clinical trial manifestos to suit the available patient population at clinical sites, but more emphasis should be placed on creating more efficient processes for first in human studies by optimizing pharmacokinetics/pharmacodynamics, dosage selection, technological advancements to improve efficacy and structured patient mapping to increase successful trial and patient recruitment opportunities.
This program will give delegates the opportunity to share proven strategies between companies to help increase efficiency in this space and streamline processes to cut down costs. This event will bring together large and small companies and experts in this space to share best practices to decrease the financial drain theses phases have on the overall clinical trial budget. Life science corporations need the most up-to-date tools and practices to increase success by streamlining processes, sharing successful biomarker strategies, anticipating dosing quantities, and optimizing healthy or specialty patient recruitment and retention. Current strategies include patient mapping before organizing and setting up a clinical space, tailoring early phase clinical trials to patient populations, purchasing biological samples from collection companies, and trying to accelerate the process by submitting for breakthrough therapy designation.
Top Reasons To Attend
Identify Compound Development Strategies to Optimize Success in Clinical Trials
Learn Best Practices for Early Decision-Making Through Analysis of Biomarker Utility in Drug Development
Utilize Analytical Technology to Evaluate Multiple Configurations of a Small Molecule to Increase the Feasibility of Drug in Clinical Trials
Implement Adaptive Design in Proof of Concept Studies to Increase Efficiency, Decrease Time and Decrease Overall Cost
Explore the Seamless Development of Phase I to Phase II in Clinical Trials
NINE Case Studies and a Panel Session on Early Phase Clinical Trial Strategies
• Planning your PIP submission
• Which in-house departments should be involved?
• Interaction with CRO/ writer
• Interaction with PDCO
• Key points for successful PIP outsourcing
Planning your Paediatric Investigation Plan (PIP) Submission in Europejbarag
During this presentation, Dr. Susan Bhatti, an experienced regulatory affairs professional, shared best practices and experiences learned from submitting PIPs. This included a brief review of the pediatric regulation requirements, insight for interacting with PDCO, and an overview of the PIP submission including procedures, timelines, structure and compliance.
Walter King is a seasoned senior R&D executive with over 30 years of experience in medical device and diagnostic product development. He has a proven track record of successfully directing projects from research through product launch on time and on budget. Key accomplishments include overseeing development of personalized diagnostic tests and medical devices that were paramount in the sale of two companies. He possesses strong leadership skills and experience managing multi-disciplinary teams across major companies.
This document summarizes the global regulatory procedures for promotional review of materials produced by ExL Pharma's global marketing team. It discusses what materials are in scope for review, the membership and standards of the Global Promotional Review Committee, and the responsibilities of global reviewers to ensure materials are consistent with core data sheets and scientifically accurate. It also reviews standards from the IFPMA, EFPIA, and ABPI codes and the process for escalation, training, self-regulation, and resources on promotional codes and regulations.
This document provides a summary of Maria Bloodgood's professional experience and qualifications. She has over 20 years of experience in project management roles in the pharmaceutical industry, focusing on clinical development and medical affairs. Her most recent role is as a Global Project Manager at Eurofins Central Laboratory, where she is responsible for managing projects and ensuring they are completed on time, on budget, and meeting quality standards. She has extensive experience managing clinical studies and programs across multiple therapeutic areas.
SMi Group's Prefilled Syringes East Coast 2018 conferenceDale Butler
This document summarizes a two-day conference on pre-filled syringes taking place in Boston on April 10-11, 2018. The conference will include two pre-conference workshops on human factors for connected drug delivery systems and design considerations for drug delivery outside the clinic. The main conference will focus on challenges and solutions for PFS manufacturing and processes, integrating quality by design principles, and trends toward increasing biologics and their implications for PFS. It will feature speakers from major pharmaceutical companies and include panel discussions on challenges of biologics delivery and PFS development.
ExL Pharma Clinical Trials Phase I and Phase IIa Conference Brochure: Phase 1...bryonmain
There is a pill or treatment for almost everything, or at least, that is how it seems. However, the amount of effort that goes into a pill or treatment before it is launched is extensive, expensive and often inefficient.
Efficiency and innovation go hand-in-hand with R&D and the development of clinical trials, however, FDA regulations and clinical trial standardization end up stifling these two key factors. This leads to drawn out processes that cost companies hundreds of millions of dollars before the drugs hit the market. Efforts have been made to increase efficiency in phase I/IIA with some companies changing their clinical trial manifestos to suit the available patient population at clinical sites, but more emphasis should be placed on creating more efficient processes for first in human studies by optimizing pharmacokinetics/pharmacodynamics, dosage selection, technological advancements to improve efficacy and structured patient mapping to increase successful trial and patient recruitment opportunities.
This program will give delegates the opportunity to share proven strategies between companies to help increase efficiency in this space and streamline processes to cut down costs. This event will bring together large and small companies and experts in this space to share best practices to decrease the financial drain theses phases have on the overall clinical trial budget. Life science corporations need the most up-to-date tools and practices to increase success by streamlining processes, sharing successful biomarker strategies, anticipating dosing quantities, and optimizing healthy or specialty patient recruitment and retention. Current strategies include patient mapping before organizing and setting up a clinical space, tailoring early phase clinical trials to patient populations, purchasing biological samples from collection companies, and trying to accelerate the process by submitting for breakthrough therapy designation.
Top Reasons To Attend
Identify Compound Development Strategies to Optimize Success in Clinical Trials
Learn Best Practices for Early Decision-Making Through Analysis of Biomarker Utility in Drug Development
Utilize Analytical Technology to Evaluate Multiple Configurations of a Small Molecule to Increase the Feasibility of Drug in Clinical Trials
Implement Adaptive Design in Proof of Concept Studies to Increase Efficiency, Decrease Time and Decrease Overall Cost
Explore the Seamless Development of Phase I to Phase II in Clinical Trials
NINE Case Studies and a Panel Session on Early Phase Clinical Trial Strategies
Este documento proporciona una dieta equilibrada dividida en 6 comidas principales que incluyen frutas, vegetales, granos integrales, proteínas magras y agua. También advierte sobre los riesgos de falta de actividad física como presión arterial alta, daño a las articulaciones y mayor riesgo de enfermedades cardíacas, diabetes y cáncer.
es el concepto que engloba todas las creaciones realizadas por el ser humano para expresar una visión sensible acerca del mundo, ya sea real o imaginario. Mediante recursos plásticos, lingüísticos o sonoros, el arte permite expresar ideas, emociones, percepciones y sensaciones.
Lee todo en: Definición de arte - Qué es, Significado y Concepto http://definicion.de/arte/#ixzz3qU8d9i9f
Este documento describe las características principales de las hojas de estilo en cascada (CSS). Explica que CSS permite separar la presentación del contenido y que pequeños cambios en la hoja de estilo pueden modificar el aspecto de todas las páginas. También cubre conceptos como la herencia de estilos, el orden de prioridad de las reglas CSS, y los diferentes métodos para incluir hojas de estilo como incrustación, vinculación e importación.
Razafimandimby Olivier, PGRM, Ministry of Energy and Mines, Madagascar, Actual Situation of Small-scale and Artisanal Mining in Madagascar: Gold and stones
Domestic tropical timber markets: informal, illegal and unsustainable?IIED
A presentation by Paolo Cerutti of CIFOR.
The presentation was made at a conference on "The biggest 'private sector': what place for the informal economy in green and inclusive growth?" on 25 February 2016.
The event was hosted by IIED and the Green Economy Coalition, WIEGO, the Center for International Forestry Research (CIFOR) and the OECD's Sahel and West Africa Club.
More details: http://bit.ly/1T8MGqJ
O documento discute o estresse no trabalho e a síndrome de burnout. Aponta que 70% dos trabalhadores brasileiros sofrem com estresse excessivo e 30% desenvolvem burnout, causando gastos de quase 4% do PIB. Descreve os sintomas e estágios de burnout, incluindo esgotamento físico e mental. Discute também as causas de estresse em professores, como falta de recursos e apoio, e estratégias para reduzir seu estresse no trabalho.
This white paper focuses on overcoming the challenges of participating in a pediatric trial. One of the biggest issues is that it is difficult to enroll participants in pediatric trials. Read these 5 strategies to help make it easier to enroll trial participants and complete successful trials.
Biosimilars: Regulatory and Clinical ConsiderationsCovance
Other considerations for clinical studies are that the regulations are written with some flexibility, and although clinical studies have been required thus far, they are not mandated by the regulations. Perception from sponsors is that innovators make process changes all the time that impact structure and no clinical study is done because of good analytical characterization so why are biosimilars different? With solid analytical and functional data, we should continue to challenge regulators on the need for clinical studies.
Outsourcing bioavailability (BA) and bioequivalence (BE) studies to contract research organizations (CROs) is common practice to reduce costs and improve efficiency. When selecting a CRO, companies should thoroughly assess the CRO's clinical trial, bioanalytical, pharmacokinetic, and timeline capabilities. Additionally, companies should qualify proposed clinical sites and bioanalytical laboratories and ensure the CRO can provide final reports and data to regulatory agencies like the FDA as required. Proper CRO selection involves due diligence, competitive bidding, and clearly defining deliverables and report requirements.
Anthony Presentation DIA Florida Ctd Nov2007AKTaylor
Creating a business process that is accurate, predictable, and capable of
meeting the changing needs for disclosing information about clinical trials
in multiple study registries and results databases is difficult. Companies
need to leverage their current clinical trial process, determine if the disclosure
steps will be centralized or decentralized, determine how much to
leverage technology, and decide whether, and how, to use outside
resources. During this session, we will hear how a large pharma, a small
biopharmaceutical company, and a device company developed their clinical
trial disclosure business process.
Significance of BA/BE studies in drug research and evaluation of different as...inemet
PharmaCon2007 Congress, Dubrovnik, Croatia "New Technologies and Trends in Pharmacy, Pharmaceutical Industry and Education" http://www.pharmacon2007.com
Abstract is available at http://www.pharmaconnectme.com
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. Aptorum's current drug pipeline includes indications in orphan diseases, infectious diseases, and metabolic diseases. The Company launched two new clinical trials in 2021. Aptorum’s Smart-ACT™ platform is designed to bring an average of three drug candidates for orphan diseases to clinical trials every 12-18 months. The Company is now preparing to launch a dietary supplement for women undergoing menopause and experiencing related symptoms, including osteoporosis. Targeting a global woman’s health supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to generate near-term revenue with significant long-term growth potential.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2020 the company plans to bring two candidates
to clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of
three drug candidates for orphan diseases to clinical trials every 12-18 months. The
company is now in the process of launching a dietary supplement for women undergoing
menopause and experiencing related symptoms, including osteoporosis. Targeting a
global woman’s health supplement market that is expected to reach $17 billion in 2025,
Aptorum is expected to generate significant revenue in the next 12 months with this
product.
The 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that their drug be designated as a "Breakthrough Therapy". This designation is primarily based on the early clinical finding of substantial efficacy in s serious medical need indication. A Breakthrough Therapy Designation provides fast track program advantages alongside a frequent FDA guidance on an efficient drug development program. The FDA also makes an organizational commitment to involve experienced reviewers and senior management in such guidance. This presentation provides an overview of Breakthrough Therapy Designation and discusses why CMC aspects can lag-behind clinical development and how this may be addressed.
The time has come to seriously work on leveraging End-of-Phase II for setting regulatory specifications.
This document discusses the regulatory requirements for clinical development of biosimilars in India. It provides an overview of the applicable regulations, guidelines, and authorities overseeing clinical trials. The key principles for developing biosimilars are to demonstrate comparability to the reference biologic through a stepwise characterization and clinical development program involving pharmacokinetic, pharmacodynamic, and confirmatory safety and efficacy studies. Post-marketing requirements include pharmacovigilance plans and potential post-marketing studies to further evaluate safety and immunogenicity.
During this presentation, Dr. Charlene Sanders and Angi Robinson reviewed topics including the evaluation of study design considerations such as duration of treatment, required assessments, use of placebo, and inclusion of specific age groups; selection of appropriate sites for pediatric trials and the unique needs of these sites; identification of pediatric recruitment/retention hurdles and site specific strategies to overcome these as well as a reflection on ethical concerns related to pediatric research.
Intelgenx Investor Presentation - July 6 2016ItelGenx
This document is an investor presentation for Innovative Drug Delivery Solutions that discusses the company's forward-looking statements and oral thin film drug delivery technology. It summarizes the market opportunities for oral thin films in treating conditions like migraines, erectile dysfunction, and opioid dependence. The presentation outlines IntelGenx's product pipeline including marketed products like Forfivo XL and products in development stages. It also discusses the company's new manufacturing facility, management team additions, and financial results that demonstrate its transformation into a growth-oriented company well positioned in oral thin film drug delivery.
Este documento proporciona una dieta equilibrada dividida en 6 comidas principales que incluyen frutas, vegetales, granos integrales, proteínas magras y agua. También advierte sobre los riesgos de falta de actividad física como presión arterial alta, daño a las articulaciones y mayor riesgo de enfermedades cardíacas, diabetes y cáncer.
es el concepto que engloba todas las creaciones realizadas por el ser humano para expresar una visión sensible acerca del mundo, ya sea real o imaginario. Mediante recursos plásticos, lingüísticos o sonoros, el arte permite expresar ideas, emociones, percepciones y sensaciones.
Lee todo en: Definición de arte - Qué es, Significado y Concepto http://definicion.de/arte/#ixzz3qU8d9i9f
Este documento describe las características principales de las hojas de estilo en cascada (CSS). Explica que CSS permite separar la presentación del contenido y que pequeños cambios en la hoja de estilo pueden modificar el aspecto de todas las páginas. También cubre conceptos como la herencia de estilos, el orden de prioridad de las reglas CSS, y los diferentes métodos para incluir hojas de estilo como incrustación, vinculación e importación.
Razafimandimby Olivier, PGRM, Ministry of Energy and Mines, Madagascar, Actual Situation of Small-scale and Artisanal Mining in Madagascar: Gold and stones
Domestic tropical timber markets: informal, illegal and unsustainable?IIED
A presentation by Paolo Cerutti of CIFOR.
The presentation was made at a conference on "The biggest 'private sector': what place for the informal economy in green and inclusive growth?" on 25 February 2016.
The event was hosted by IIED and the Green Economy Coalition, WIEGO, the Center for International Forestry Research (CIFOR) and the OECD's Sahel and West Africa Club.
More details: http://bit.ly/1T8MGqJ
O documento discute o estresse no trabalho e a síndrome de burnout. Aponta que 70% dos trabalhadores brasileiros sofrem com estresse excessivo e 30% desenvolvem burnout, causando gastos de quase 4% do PIB. Descreve os sintomas e estágios de burnout, incluindo esgotamento físico e mental. Discute também as causas de estresse em professores, como falta de recursos e apoio, e estratégias para reduzir seu estresse no trabalho.
This white paper focuses on overcoming the challenges of participating in a pediatric trial. One of the biggest issues is that it is difficult to enroll participants in pediatric trials. Read these 5 strategies to help make it easier to enroll trial participants and complete successful trials.
Biosimilars: Regulatory and Clinical ConsiderationsCovance
Other considerations for clinical studies are that the regulations are written with some flexibility, and although clinical studies have been required thus far, they are not mandated by the regulations. Perception from sponsors is that innovators make process changes all the time that impact structure and no clinical study is done because of good analytical characterization so why are biosimilars different? With solid analytical and functional data, we should continue to challenge regulators on the need for clinical studies.
Outsourcing bioavailability (BA) and bioequivalence (BE) studies to contract research organizations (CROs) is common practice to reduce costs and improve efficiency. When selecting a CRO, companies should thoroughly assess the CRO's clinical trial, bioanalytical, pharmacokinetic, and timeline capabilities. Additionally, companies should qualify proposed clinical sites and bioanalytical laboratories and ensure the CRO can provide final reports and data to regulatory agencies like the FDA as required. Proper CRO selection involves due diligence, competitive bidding, and clearly defining deliverables and report requirements.
Anthony Presentation DIA Florida Ctd Nov2007AKTaylor
Creating a business process that is accurate, predictable, and capable of
meeting the changing needs for disclosing information about clinical trials
in multiple study registries and results databases is difficult. Companies
need to leverage their current clinical trial process, determine if the disclosure
steps will be centralized or decentralized, determine how much to
leverage technology, and decide whether, and how, to use outside
resources. During this session, we will hear how a large pharma, a small
biopharmaceutical company, and a device company developed their clinical
trial disclosure business process.
Significance of BA/BE studies in drug research and evaluation of different as...inemet
PharmaCon2007 Congress, Dubrovnik, Croatia "New Technologies and Trends in Pharmacy, Pharmaceutical Industry and Education" http://www.pharmacon2007.com
Abstract is available at http://www.pharmaconnectme.com
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. Aptorum's current drug pipeline includes indications in orphan diseases, infectious diseases, and metabolic diseases. The Company launched two new clinical trials in 2021. Aptorum’s Smart-ACT™ platform is designed to bring an average of three drug candidates for orphan diseases to clinical trials every 12-18 months. The Company is now preparing to launch a dietary supplement for women undergoing menopause and experiencing related symptoms, including osteoporosis. Targeting a global woman’s health supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to generate near-term revenue with significant long-term growth potential.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2020 the company plans to bring two candidates
to clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of
three drug candidates for orphan diseases to clinical trials every 12-18 months. The
company is now in the process of launching a dietary supplement for women undergoing
menopause and experiencing related symptoms, including osteoporosis. Targeting a
global woman’s health supplement market that is expected to reach $17 billion in 2025,
Aptorum is expected to generate significant revenue in the next 12 months with this
product.
The 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that their drug be designated as a "Breakthrough Therapy". This designation is primarily based on the early clinical finding of substantial efficacy in s serious medical need indication. A Breakthrough Therapy Designation provides fast track program advantages alongside a frequent FDA guidance on an efficient drug development program. The FDA also makes an organizational commitment to involve experienced reviewers and senior management in such guidance. This presentation provides an overview of Breakthrough Therapy Designation and discusses why CMC aspects can lag-behind clinical development and how this may be addressed.
The time has come to seriously work on leveraging End-of-Phase II for setting regulatory specifications.
This document discusses the regulatory requirements for clinical development of biosimilars in India. It provides an overview of the applicable regulations, guidelines, and authorities overseeing clinical trials. The key principles for developing biosimilars are to demonstrate comparability to the reference biologic through a stepwise characterization and clinical development program involving pharmacokinetic, pharmacodynamic, and confirmatory safety and efficacy studies. Post-marketing requirements include pharmacovigilance plans and potential post-marketing studies to further evaluate safety and immunogenicity.
During this presentation, Dr. Charlene Sanders and Angi Robinson reviewed topics including the evaluation of study design considerations such as duration of treatment, required assessments, use of placebo, and inclusion of specific age groups; selection of appropriate sites for pediatric trials and the unique needs of these sites; identification of pediatric recruitment/retention hurdles and site specific strategies to overcome these as well as a reflection on ethical concerns related to pediatric research.
Intelgenx Investor Presentation - July 6 2016ItelGenx
This document is an investor presentation for Innovative Drug Delivery Solutions that discusses the company's forward-looking statements and oral thin film drug delivery technology. It summarizes the market opportunities for oral thin films in treating conditions like migraines, erectile dysfunction, and opioid dependence. The presentation outlines IntelGenx's product pipeline including marketed products like Forfivo XL and products in development stages. It also discusses the company's new manufacturing facility, management team additions, and financial results that demonstrate its transformation into a growth-oriented company well positioned in oral thin film drug delivery.
Presentation about Biosimilars on Save Your Skin Foundation webinar, January 19, 2018. This is presentation #1 of 3 in the webinar: 1. Health Canada 2. CADTH 3. Louise Binder, Save Your Skin Fdn.
When designing pediatric research, one size does not fit all. Simply adapting the adult clinical study protocol into its child-sized version will not be enough to gain IRB approval.
Research involving minors must prioritize the rights, safety, and welfare of its young participants, and the FDA has issued substantial regulations to ensure pediatric research is conducted safely and ethically.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. Aptorum's current drug pipeline includes indications in orphan diseases, infectious diseases, and metabolic diseases. The Company launched two new clinical trials in 2021. Aptorum’s Smart-ACT™ platform is designed to bring an average of three drug candidates for orphan diseases to clinical trials every 12-18 months. The Company is now preparing to launch a dietary supplement for women undergoing menopause and experiencing related symptoms, including osteoporosis. Targeting a global woman’s health supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to generate near-term revenue with significant long-term growth potential.
This document summarizes requirements for pediatric drug studies under the Pediatric Research Equity Act (PREA). It outlines criteria for when deferrals or waivers of pediatric studies are appropriate. Deferrals are allowed if drug approval in adults is needed first or additional safety data is required. Waivers are granted if studies are impossible, unsafe or the drug provides no meaningful benefit over existing therapies. The document advises discussing PREA requirements early in development and including plans for deferrals or waivers in pediatric study plans. It offers to help review products and prepare justifications for requested waivers or deferrals of pediatric studies.
Genetic Technologies is a diversified molecular diagnostics company. A global leader in genomics-based tests in health, wellness and serious disease through its geneType and EasyDNA brands. GENE offers cancer predictive testing and assessment tools to help physicians to improve health outcomes for people around the world. The Company has a proprietary risk stratification platform that has been developed over the past decade and integrates clinical and genetic risk to deliver actionable outcomes to physicians and individuals. Leading the world in risk prediction in oncology, cardiovascular and metabolic diseases, Genetic Technologies continues to develop risk assessment products.
Similar to Global Paediatric Studies–A CRO perspective (20)
Feasibility Solutions to Clinical Trial Nightmaresjbarag
Slow patient recruitment and poor retention cause recurrent nightmares and perpetual problems often resulting in missing recruitment milestones. The cost of these delays represents hundreds of thousands of dollars for drug and device developers. By recognizing this issue, early detailed feasibility can provide planning and contingency solutions that are focused on reducing the impact of delayed recruitment. Furthermore understanding what motivates investigators and patients to actively participate in clinical studies and how patient recruitment strategies and materials can support all stakeholders to complete studies on time are critical aspects of clinical study delivery planning.
During this presentation, an experienced Premier Research feasibility and patient recruitment specialist, reviewed feasibility approaches to address protocol evaluation as well as addressed influences on country selection, site distribution and patient recruitment strategies to provide for more effective clinical trial planning and conduct.
For more information, go to http://www.premier-research.com.
Successful Pediatric Studies: Key Study Design and Site Selection Considerationsjbarag
The industry recognizes the importance of ensuring the safety and well‐being of children involved in research studies. Medical and regulatory bodies have worked to provide a framework to support appropriately designed studies through regulations and guidance documents in this vulnerable population. However, it is crucial to understand the nuances associated with pediatric trials, for the site, patient and family, in order to manage them to successful completion.
During the 2012 ACRP Annual Meeting, Dr. Charlene Sanders and Angi Robinson from Premier Research reviewed topics including the evaluation of study design considerations such as duration of treatment, required assessments, use of placebo, and inclusion of specific age groups; selection of appropriate sites for pediatric trials and the unique needs of these sites; identification of pediatric recruitment/retention hurdles and site specific strategies to overcome these as well as a reflection on ethical concerns related to pediatric research.
For more information, go to http://www.premier-research.com/pediatrics.
Over the past decade, CDISC data standards have become the FDA preferred method for the data submission. In fact, the FDA is considering a proposed rule change that would mandate the submission of data in CDISC Study Data Tabulation Model (SDTM) and Analysis Data Model (ADaM) formats for all new marketing applications. However, the implementation of this standard has proved to be intimidating to many with only a very small percentage of drug companies actually developing and submitting data in this format.
During the webinar, Thomas Kalfas, an experienced data management professional and CDISC subject matter expert, shared his knowledge and strategies for implementing CDSIC. Topics included a brief review of CDISC, implementation challenges, and insight into the best timing for implementation.
Creating Effective Pediatric Assent Forms: Overcoming Common Obstaclesjbarag
This document discusses creating effective pediatric assent forms by overcoming common obstacles. It identifies five main obstacles: 1) treating assent as an afterthought, 2) lack of direction from sponsors/IRBs, 3) failure to account for developmental ages and reading levels, 4) difficulty creating readable forms, and 5) not planning the assent process logistics. It provides tools to write forms at appropriate reading levels, ensure all elements of assent are addressed, and plan who will obtain assent and where. The goal is to engage children in a developmentally-appropriate way and respect their participation in research decisions.
This document summarizes a webinar on streamlining data management for clinical trials. The webinar covered the need for streamlined approaches given rising drug development costs. It discussed areas for improving efficiencies, including using standards, a parallel approach, identifying key reviewers, and tailoring processes based on trial type (e.g. a "Premier Express" approach for small phase 1 trials). An example case study showed how streamlining tasks and performing work in parallel reduced timelines for developing case report forms, annotated case report forms, databases, and edit checks for a small phase 1 trial from 9 weeks to 5 weeks.
This presentation, led by Ryan Michaud, explored how to best employ IV/IWRS platforms for collecting and managing Patient Reported Outcomes (PRO) data, clinical supply management including drug accountability, visit tracking and randomization.
During this presentation, Ron Kershner, Ph.D. discussed the responsibilities of DMCs from the perspective of protecting patient safety and providing critical, independent oversight to key study objectives. Drawing on past clinical trials to illustrate key points, Ron addressed DMC operational considerations, such as meeting frequency and content, control of information, data cleaning issues and scope/format of data tabulations.
Guidelines for Effective and Appropriate Pediatric Assent and Parental Permis...jbarag
During this presentation, Angi Robinson and Elizabeth Jay reviewed the regulatory requirements for parental permission and pediatric assent; provided practical tips for compliant and age-appropriate form development including which elements to incorporate, the number of required signatures, and how to check for reading comprehension level; and offered recommendations for documentation of the consenting/assenting process.
Developing a Feasible Pediatric Plan for PREA/PMDSIA Compliancejbarag
This presentation will address issues surrounding the development of a pediatric plan for PREA/PMDSIA compliance including: when to develop a pediatric plan, what age groups should be included, is a pediatric formulation necessary, timing of the pediatric studies, what information should be submitted to FDA, and when a waiver or deferral is appropriate.
Centralized Resourcing Model for Clinical Trialsjbarag
A centralized resourcing model within clinical research organizations can provide efficiencies in resource management. It involves having a central point of contact to manage the resource assignment, deployment, and utilization across programs. Key ingredients for success include regular communication, standardized processes for resource requests and tracking, and reliable tools for resource planning and metrics. This model allows resources to be strategically allocated, utilization to be maximized through short-term assignments, and proactive planning to improve cost and time efficiencies.
Medical Writing Essential: Reviewing Statisitical Analysis Plansjbarag
Regulatory medical writers are tasked with generating high-quality clinical study reports (CSRs) promptly. To this end, statistical analysis plan (SAP) reviews are essential as they allow medical writers to verify that the SAP contains the information required for the CSR per regulatory guidance. This session will explain how to conduct SAP reviews and how to assess whether data presentations in addition to those proposed are needed for the CSR.
Ind Applications: A Case Study of Document Development from the Medical Writi...jbarag
This session is a presentation of a case study on management of a complex document development program for Investigational New Drug (IND) submissions. The scope of writing 19 documents for 3 IND submissions in 6 months dictated expert project management by a medical writing (MW) team. Through meticulous organization, strategic planning and communication, MW effectively managed the process and delivered the required documents well before the IND submission deadlines.
Meeting Enrollment Goals in a Competitive Environmentjbarag
Challenges in patient recruitment continue to be the number one cause in clinical trial delays. Through involvement in this workshop, participants will become familiar with how to develop, implement, manage and track site enrollment plans. This will include understanding the core elements that constitute an enrollment plan as well as understanding how the development of strategic tools and tactics can aid sites in the successful implementation, monitoring and tracking of results. Both project management and site perspectives on enrollment and recruitment plans will be discussed.
This document discusses managing high performance project teams. It emphasizes that conducting clinical research requires contributions from all team members. It outlines some fundamentals and challenges of project management including forming a cohesive team, maintaining motivation, and communication. It provides basics for managing teams such as establishing roles and responsibilities, communication plans, and holding regular meetings. It also discusses important leadership skills like being a good listener, connecting with others, and insulating the team from issues. Proactive communication techniques are also covered like being mindful of tone, email management, and making requests.
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdfRahul Sen
Time-lapse embryo monitoring is an advanced imaging technique used in IVF to continuously observe embryo development. It captures high-resolution images at regular intervals, allowing embryologists to select the most viable embryos for transfer based on detailed growth patterns. This technology enhances embryo selection, potentially increasing pregnancy success rates.
Travel Clinic Cardiff: Health Advice for International TravelersNX Healthcare
Travel Clinic Cardiff offers comprehensive travel health services, including vaccinations, travel advice, and preventive care for international travelers. Our expert team ensures you are well-prepared and protected for your journey, providing personalized consultations tailored to your destination. Conveniently located in Cardiff, we help you travel with confidence and peace of mind. Visit us: www.nxhealthcare.co.uk
- Video recording of this lecture in English language: https://youtu.be/Pt1nA32sdHQ
- Video recording of this lecture in Arabic language: https://youtu.be/uFdc9F0rlP0
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
Travel vaccination in Manchester offers comprehensive immunization services for individuals planning international trips. Expert healthcare providers administer vaccines tailored to your destination, ensuring you stay protected against various diseases. Conveniently located clinics and flexible appointment options make it easy to get the necessary shots before your journey. Stay healthy and travel with confidence by getting vaccinated in Manchester. Visit us: www.nxhealthcare.co.uk
The skin is the largest organ and its health plays a vital role among the other sense organs. The skin concerns like acne breakout, psoriasis, or anything similar along the lines, finding a qualified and experienced dermatologist becomes paramount.
Discover the benefits of homeopathic medicine for irregular periods with our guide on 5 common remedies. Learn how these natural treatments can help regulate menstrual cycles and improve overall menstrual health.
Visit Us: https://drdeepikashomeopathy.com/service/irregular-periods-treatment/
The biomechanics of running involves the study of the mechanical principles underlying running movements. It includes the analysis of the running gait cycle, which consists of the stance phase (foot contact to push-off) and the swing phase (foot lift-off to next contact). Key aspects include kinematics (joint angles and movements, stride length and frequency) and kinetics (forces involved in running, including ground reaction and muscle forces). Understanding these factors helps in improving running performance, optimizing technique, and preventing injuries.
Breast cancer: Post menopausal endocrine therapyDr. Sumit KUMAR
Breast cancer in postmenopausal women with hormone receptor-positive (HR+) status is a common and complex condition that necessitates a multifaceted approach to management. HR+ breast cancer means that the cancer cells grow in response to hormones such as estrogen and progesterone. This subtype is prevalent among postmenopausal women and typically exhibits a more indolent course compared to other forms of breast cancer, which allows for a variety of treatment options.
Diagnosis and Staging
The diagnosis of HR+ breast cancer begins with clinical evaluation, imaging, and biopsy. Imaging modalities such as mammography, ultrasound, and MRI help in assessing the extent of the disease. Histopathological examination and immunohistochemical staining of the biopsy sample confirm the diagnosis and hormone receptor status by identifying the presence of estrogen receptors (ER) and progesterone receptors (PR) on the tumor cells.
Staging involves determining the size of the tumor (T), the involvement of regional lymph nodes (N), and the presence of distant metastasis (M). The American Joint Committee on Cancer (AJCC) staging system is commonly used. Accurate staging is critical as it guides treatment decisions.
Treatment Options
Endocrine Therapy
Endocrine therapy is the cornerstone of treatment for HR+ breast cancer in postmenopausal women. The primary goal is to reduce the levels of estrogen or block its effects on cancer cells. Commonly used agents include:
Selective Estrogen Receptor Modulators (SERMs): Tamoxifen is a SERM that binds to estrogen receptors, blocking estrogen from stimulating breast cancer cells. It is effective but may have side effects such as increased risk of endometrial cancer and thromboembolic events.
Aromatase Inhibitors (AIs): These drugs, including anastrozole, letrozole, and exemestane, lower estrogen levels by inhibiting the aromatase enzyme, which converts androgens to estrogen in peripheral tissues. AIs are generally preferred in postmenopausal women due to their efficacy and safety profile compared to tamoxifen.
Selective Estrogen Receptor Downregulators (SERDs): Fulvestrant is a SERD that degrades estrogen receptors and is used in cases where resistance to other endocrine therapies develops.
Combination Therapies
Combining endocrine therapy with other treatments enhances efficacy. Examples include:
Endocrine Therapy with CDK4/6 Inhibitors: Palbociclib, ribociclib, and abemaciclib are CDK4/6 inhibitors that, when combined with endocrine therapy, significantly improve progression-free survival in advanced HR+ breast cancer.
Endocrine Therapy with mTOR Inhibitors: Everolimus, an mTOR inhibitor, can be added to endocrine therapy for patients who have developed resistance to aromatase inhibitors.
Chemotherapy
Chemotherapy is generally reserved for patients with high-risk features, such as large tumor size, high-grade histology, or extensive lymph node involvement. Regimens often include anthracyclines and taxanes.
Osvaldo Bernardo Muchanga-GASTROINTESTINAL INFECTIONS AND GASTRITIS-2024.pdfOsvaldo Bernardo Muchanga
GASTROINTESTINAL INFECTIONS AND GASTRITIS
Osvaldo Bernardo Muchanga
Gastrointestinal Infections
GASTROINTESTINAL INFECTIONS result from the ingestion of pathogens that cause infections at the level of this tract, generally being transmitted by food, water and hands contaminated by microorganisms such as E. coli, Salmonella, Shigella, Vibrio cholerae, Campylobacter, Staphylococcus, Rotavirus among others that are generally contained in feces, thus configuring a FECAL-ORAL type of transmission.
Among the factors that lead to the occurrence of gastrointestinal infections are the hygienic and sanitary deficiencies that characterize our markets and other places where raw or cooked food is sold, poor environmental sanitation in communities, deficiencies in water treatment (or in the process of its plumbing), risky hygienic-sanitary habits (not washing hands after major and/or minor needs), among others.
These are generally consequences (signs and symptoms) resulting from gastrointestinal infections: diarrhea, vomiting, fever and malaise, among others.
The treatment consists of replacing lost liquids and electrolytes (drinking drinking water and other recommended liquids, including consumption of juicy fruits such as papayas, apples, pears, among others that contain water in their composition).
To prevent this, it is necessary to promote health education, improve the hygienic-sanitary conditions of markets and communities in general as a way of promoting, preserving and prolonging PUBLIC HEALTH.
Gastritis and Gastric Health
Gastric Health is one of the most relevant concerns in human health, with gastrointestinal infections being among the main illnesses that affect humans.
Among gastric problems, we have GASTRITIS AND GASTRIC ULCERS as the main public health problems. Gastritis and gastric ulcers normally result from inflammation and corrosion of the walls of the stomach (gastric mucosa) and are generally associated (caused) by the bacterium Helicobacter pylor, which, according to the literature, this bacterium settles on these walls (of the stomach) and starts to release urease that ends up altering the normal pH of the stomach (acid), which leads to inflammation and corrosion of the mucous membranes and consequent gastritis or ulcers, respectively.
In addition to bacterial infections, gastritis and gastric ulcers are associated with several factors, with emphasis on prolonged fasting, chemical substances including drugs, alcohol, foods with strong seasonings including chilli, which ends up causing inflammation of the stomach walls and/or corrosion. of the same, resulting in the appearance of wounds and consequent gastritis or ulcers, respectively.
Among patients with gastritis and/or ulcers, one of the dilemmas is associated with the foods to consume in order to minimize the sensation of pain and discomfort.
PGx Analysis in VarSeq: A User’s PerspectiveGolden Helix
Since our release of the PGx capabilities in VarSeq, we’ve had a few months to gather some insights from various use cases. Some users approach PGx workflows by means of array genotyping or what seems to be a growing trend of adding the star allele calling to the existing NGS pipeline for whole genome data. Luckily, both approaches are supported with the VarSeq software platform. The genotyping method being used will also dictate what the scope of the tertiary analysis will be. For example, are your PGx reports a standalone pipeline or would your lab’s goal be to handle a dual-purpose workflow and report on PGx + Diagnostic findings.
The purpose of this webcast is to:
Discuss and demonstrate the approaches with array and NGS genotyping methods for star allele calling to prep for downstream analysis.
Following genotyping, explore alternative tertiary workflow concepts in VarSeq to handle PGx reporting.
Moreover, we will include insights users will need to consider when validating their PGx workflow for all possible star alleles and options you have for automating your PGx analysis for large number of samples. Please join us for a session dedicated to the application of star allele genotyping and subsequent PGx workflows in our VarSeq software.
2. Overview
▪ PREA and PIP – similar, but different
▪ Keeping your paediatric commitments feasible
▪ Choosing your CRO partner
▪ Ensuring compliance with PIP and PREA
▪ Key points for successful paediatric studies
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
3. PREA and PIP – similar, but different
Pediatric Research Equity Act (PREA) Paediatric Investigation Plan (PIP)
Required for all marketing
Applicable to all NDAs and BLAs with authorisation applications for
New active ingredient New substances
New indication New indications for patent
protected authorised products
New dosage form
New dosing regimen
New route of administration
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
4. PREA and PIP – similar, but different
Products developed for the US market may fall
under PREA requirements although a PIP is not
required for a marketing authorisation in the EU
Products developed for the EU market may
require paediatric studies if they are to be
marketed in USA
Requirements for paediatric studies need to
be assessed early in development and planned
into the development strategy
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
5. PREA and PIP waivers
PREA waiver conditions PIP waiver conditions
Paediatric studies are impossible or highly Product is ineffective or unsafe in part or
impractical (e.g. number of patients too all of the paediatric population
small or geographically dispersed)
Strong evidence that drug or biological Disease or condition only occurs in
product will be ineffective or unsafe in adults
paediatric age groups
No meaningful therapeutic benefit over Product does not represent a significant
existing therapies and is not likely to be therapeutic benefit over existing
used in a substantial number of paediatric treatments for paediatric patients
patients
Partial waiver if reasonable attempts to
produce a paediatric formulation
necessary for a particular age group have
failed
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
6. PREA and PIP waivers
A waivers may be granted for paediatric studies
by the FDA...
but the conditions for granting a waiver may not
be fulfilled in EU so that studies are required to
obtain a marketing authorisation
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
7. PREA and PIP deferrals
PREA grounds for deferral PIP grounds for deferral
Drug or biological product is ready for If it is appropriate to perform studies in
approval for use in adults before adults prior to initiating studies in
paediatric studies are complete children
Paediatric studies should be delayed until In order prevent a delay in the
additional safety or effectiveness data availability of new medicinal products to
have been collected the adult population
There is another appropriate reason for
deferral
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
8. PREA and PIP deferrals
Deferrals until after approval in adults may be
granted for paediatric studies by the FDA...
but if the PDCO identifies an unmet need in the
paediatric population, studies may have to be
conducted earlier to obtain marketing
authorisation in Europe
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
9. PREA and PIP – similar, but different
▪ PREA and PIP requirements are not identical
▪ Differences in definitions of “new” products, as well as
conditions for waivers and deferrals can affect both the
requirements for studies and their timelines
▪ A comparison of the US and EU paediatric
requirements should be conducted early during
development if the product is intended for both
markets
▪ Strategic paediatric trial planning is essential!
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
10. Keeping your paediatric commitments
feasible
Promises, promises...
“We have some commitments to perform paediatric
studies, but we would like to go back to the FDA and try to
renegotiate”
“We must not promise what we ought not, lest we be called
on to perform what we cannot” Abraham Lincoln
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
11. Keeping your paediatric commitments
feasible
▪ Make sure the data on the prevalence and incidence of the
paediatric condition in the literature is reliable
▪ Check the feasibility of finding paediatric patients who will
fit the protocol(s) and use the results of these feasibility
studies to justify the paediatric development plan
▪ Consider whether the studies are ethically viable (e.g. blood
sampling, placebo groups, pharmacokinetic studies,
contraceptive requirements)
▪ Remember you are not alone...there will be (more and
more) competitive studies
▪ Try to avoid agreeing to do something until you know you
can!
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
12. Keeping your paediatric commitments
feasible
Case study:
A large pharmaceutical company made the following
commitment to FDA:
▪ Perform a paediatric study in a very rare indication in 31
patients
▪ Single treatment study
▪ Based on calculations of disease incidence over 24 months,
only 1 patient expected to be recruited per country
▪ Study therefore required initiation of one site with one
patient in 31 different countries
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
13. Keeping your paediatric commitments
feasible
Implications of the commitment were:
▪ Difficult logistics
▪ High costs due to regulatory submission fees for numerous
countries, multiple site initiation costs, far ranging project
management, drug importation hurdles, etc.
▪ No guarantee of finding the patients within the 24 month
timeframe
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
14. Choosing your CRO partner
Checklist for your choice of CRO
Experience of conducting paediatric studies in both USA
and Europe
Experience in the relevant indication
Understanding of the paediatric legislation as well as
the health authority and ethical requirements in both
USA and EU
Understanding of the work and views of the FDA
paediatric division and the PDCO
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
15. Choosing your CRO partner
Experience confirms:
“The difference between theory and practice tends to be
very small in theory, but in practice it is very large indeed”
Anon
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
16. Ensuring compliance with PIP and PREA
A paediatric development plan must be submitted
along with the NDA or BLA at the latest, and it is
reviewed together with the rest of the application
by the review division responsible for the relevant
therapeutic area.
A PIP is reviewed and approved by the PDCO well
in advance of any application for marketing
authorisation for the product. An approved and
compliant PIP is required for validation of the
application.
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
17. Ensuring compliance with PIP and PREA
Modifications to the paediatric development plan
can (and should) be discussed with the FDA during
the approval process in order to avoid unworkable
commitments.
Due to the ‘theoretical’ nature of the PIP originally
approved, modifications will generally be required
prior to MA submission to adapt to the ‘real life’
situation and these should be planned into the
development strategy and timelines.
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
18. Ensuring compliance with PIP and PREA
Adapting to reality...
Sponsors should submit their proposed changes to a Written
Request or Pediatric Proposal communications, along with
scientific and clinical-based evidence to support their suggested
modifications, to the FDA as amendments to the IND or NDA.
Amendments to paediatric research plans may be made by
Sponsors until all aspects of the WR or the PP have been
scrutinized and/or analyzed for feasibility and scientific merit; or
until the FDA’s threshold for issuing waivers/partial waivers have
been met.
All changes and agreements with FDA must be made in sufficient
time to meet the timetable outlined in the agreements.
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
20. Ensuring compliance with PIP and PREA
Adapting to reality...
▪ Modifications to a PIP are possible if there are difficulties
with the PIP implementation i.e. the original plan is either
unworkable or is no longer appropriate
▪ Modifications should preferably be prospective
▪ Multiple modifications are possible
▪ New waivers/deferrals can also be requested
▪ New opinion supersedes original
▪ All changes have to be justified
FOCUS. EXPERTISE. QUALITY. Susan Bhatti
21. Key points for successful paediatric studies
Plan your PREA and PIP strategy early in development
Be aware of the differences between US and Europe in
requirements and timelines for paediatric plans
Conduct adequate feasibility on proposed studies to
ensure these are practicable
Chose a CRO partner experienced in paediatric trials
Monitor amendments to protocols and check for impact
on key binding elements of the PDCO opinion
Justify all modifications and submit in a timely manner!
FOCUS. EXPERTISE. QUALITY. Susan Bhatti