The document discusses the stages of clinical drug development, including preclinical testing, Phase I-III trials, and regulatory approval. Preclinical testing assesses safety in animals before human trials. Phase I trials primarily evaluate safety in small human groups. Phase II trials further assess safety and preliminary efficacy. Phase III trials are large-scale, placebo-controlled trials to prove efficacy and long-term safety for regulatory approval. The goal is to advance safely from preclinical to clinical testing and approval.
The document summarizes the process of bringing a new drug to market. It involves pre-clinical research for 4.5 years, clinical trials for 5 years, and seeking FDA approval for 2.5 years, for a total of 12 years. The estimated total cost is $800 million, including $335 million for pre-clinical research and $465 million for clinical trials and FDA approval. The process involves research and development teams, management, doctors, pharmacists, and clinical trial subjects working towards milestones of IRB proposal and approval, completing the three phases of clinical trials, and ultimately obtaining FDA approval to release the new drug to the market.
Introduction to Regulatory Affairs - Pauwels Consulting AcademyPauwels Consulting
On Tuesday, June 14, our colleagues Fiorenzo Savoretti, Senior Regulatory and Quality Consultant at Pfizer and Nick Deschacht, Senior RA Consultant at GSK, gave an interesting “Introduction to Regulatory Affairs”.
Fiorenzo and Nick talked about RA and their projects, each from their unique angle. They delivered their presentations for ## attendees at our Brussels office at the Lambroekstraat 5a in Diegem.
Fundamental concept of regulatory affairs in pharmaceutical & biotechnologyHitendra Singh
RA is a comparatively new profession which developed from the desire of governments to protect public health by controlling the Quality, safety and efficacy of products in areas including pharmaceuticals, Biotechnology, veterinary medicines, medical devices, pesticides, agrochemicals, cosmetics and complementary medicines.
Goals of Regulatory Affairs Professionals:-
Protection of human health
Ensuring safety, efficacy and quality of drugs
Ensuring appropriateness and accuracy of product information
Phase 1 clinical trials are the first studies conducted in humans of a new drug or treatment. They aim to determine the drug's safety and tolerability, identify the maximum tolerated dose, and understand the drug's pharmacokinetics. Phase 1 trials typically involve small groups of healthy volunteers or patients and start with low doses that are gradually increased. The results of phase 1 trials provide information needed to design subsequent phase 2 and 3 trials to further evaluate efficacy.
The presentation aims at a students focussed perspective of Abbreviated New Drug Application filing with premier regulatory body like USFDA, the eCTD is followed worldwide for drug submission aimed for gaining particular market approvals.When submitted with FDA it is evaluated by CDER. eCTD is further a mandatory submission for ANDAs with FDA and for NDAs with EU and Japan.
CFTCC
2015 Learning about the IND/IDE Process and Reimbursements for New Drugs and Devices
Erin O'Reilly, PhD, RAC
Assoc. Director, Regulatory Affairs
Translational Medicine Institute
Introduces the basics of filing an Investigational New Drug (IND) Application with the FDA
Central Drugs Standard Control Organization (CDSCO)KapilKumar198
The Central Drugs Standard Control Organization (CDSCO) is India's national regulatory body for pharmaceuticals and medical devices. It functions under the Directorate General of Health Services and regulates drug approval, clinical trials, manufacturing standards, and import of drugs. CDSCO has six zonal offices, four sub-zonal offices, and seven drug testing laboratories. It is headed by the Drug Controller General of India, who is responsible for approving licenses for certain drug categories and setting quality standards for drug manufacturing and distribution in India.
To compare filing process of NDA of different countries of India, US and Euro...Aakashdeep Raval
To compare filing process of NDA of different countries of India, US and Europe.
B) Preparation of global list documents of registration of IND and NDA as per USFDA and Europe.
The document summarizes the process of bringing a new drug to market. It involves pre-clinical research for 4.5 years, clinical trials for 5 years, and seeking FDA approval for 2.5 years, for a total of 12 years. The estimated total cost is $800 million, including $335 million for pre-clinical research and $465 million for clinical trials and FDA approval. The process involves research and development teams, management, doctors, pharmacists, and clinical trial subjects working towards milestones of IRB proposal and approval, completing the three phases of clinical trials, and ultimately obtaining FDA approval to release the new drug to the market.
Introduction to Regulatory Affairs - Pauwels Consulting AcademyPauwels Consulting
On Tuesday, June 14, our colleagues Fiorenzo Savoretti, Senior Regulatory and Quality Consultant at Pfizer and Nick Deschacht, Senior RA Consultant at GSK, gave an interesting “Introduction to Regulatory Affairs”.
Fiorenzo and Nick talked about RA and their projects, each from their unique angle. They delivered their presentations for ## attendees at our Brussels office at the Lambroekstraat 5a in Diegem.
Fundamental concept of regulatory affairs in pharmaceutical & biotechnologyHitendra Singh
RA is a comparatively new profession which developed from the desire of governments to protect public health by controlling the Quality, safety and efficacy of products in areas including pharmaceuticals, Biotechnology, veterinary medicines, medical devices, pesticides, agrochemicals, cosmetics and complementary medicines.
Goals of Regulatory Affairs Professionals:-
Protection of human health
Ensuring safety, efficacy and quality of drugs
Ensuring appropriateness and accuracy of product information
Phase 1 clinical trials are the first studies conducted in humans of a new drug or treatment. They aim to determine the drug's safety and tolerability, identify the maximum tolerated dose, and understand the drug's pharmacokinetics. Phase 1 trials typically involve small groups of healthy volunteers or patients and start with low doses that are gradually increased. The results of phase 1 trials provide information needed to design subsequent phase 2 and 3 trials to further evaluate efficacy.
The presentation aims at a students focussed perspective of Abbreviated New Drug Application filing with premier regulatory body like USFDA, the eCTD is followed worldwide for drug submission aimed for gaining particular market approvals.When submitted with FDA it is evaluated by CDER. eCTD is further a mandatory submission for ANDAs with FDA and for NDAs with EU and Japan.
CFTCC
2015 Learning about the IND/IDE Process and Reimbursements for New Drugs and Devices
Erin O'Reilly, PhD, RAC
Assoc. Director, Regulatory Affairs
Translational Medicine Institute
Introduces the basics of filing an Investigational New Drug (IND) Application with the FDA
Central Drugs Standard Control Organization (CDSCO)KapilKumar198
The Central Drugs Standard Control Organization (CDSCO) is India's national regulatory body for pharmaceuticals and medical devices. It functions under the Directorate General of Health Services and regulates drug approval, clinical trials, manufacturing standards, and import of drugs. CDSCO has six zonal offices, four sub-zonal offices, and seven drug testing laboratories. It is headed by the Drug Controller General of India, who is responsible for approving licenses for certain drug categories and setting quality standards for drug manufacturing and distribution in India.
To compare filing process of NDA of different countries of India, US and Euro...Aakashdeep Raval
To compare filing process of NDA of different countries of India, US and Europe.
B) Preparation of global list documents of registration of IND and NDA as per USFDA and Europe.
International Journal of Drug Regulatory Affairs; 2014, 2(1), 1- 11
Abstract:
Developing a new drug requires great amount of research work in chemistry, manufacturing, controls, preclinical science and clinical trials. Drug reviewers in regulatory agencies around the world bear the responsibility of evaluating whether the research data support the safety, effectiveness and quality control of a new drug product to serve the public health. Every country has its own regulatory authority, which is responsible to enforce the rules and regulations and issue the guidelines to regulate the marketing of the drugs. This article focuses on drug approval process in different countries like USA, Europe and India.
The document discusses the FDA drug approval process and lists some drugs that have been withdrawn by the FDA. It describes the multi-phase approval process including pre-clinical testing in animals, followed by three phases of clinical trials in humans. It can take over a decade for a drug to be approved. Several drugs are mentioned that were withdrawn from the market due to serious safety issues discovered after approval, such as thalidomide causing birth defects, phenformin causing fatal lactic acidosis, and rofecoxib increasing heart attack and stroke risk.
Centralize & decentralize regulatory framework of brazil and south africaHarishankar Sahu
ANVISA is Brazil's centralized regulatory body that oversees pharmaceuticals and health products. It regulates drugs, medical devices, cosmetics and other products. In contrast, regulatory frameworks in African countries are more decentralized and vary between countries. The African Medicines Registration Harmonisation initiative aims to strengthen regulatory capacity and harmonize requirements across African nations to facilitate regional access to medicines.
Regulatory affairs professionals play a crucial role in ensuring pharmaceutical products meet regulatory standards for safety, efficacy and quality. They provide strategic guidance to various departments on regulatory requirements and work to obtain approvals from regulatory authorities. Key responsibilities include filing product registrations, tracking legal changes, and facilitating clinical trials and product approvals. Regulatory affairs has become increasingly important given historical issues that prompted stronger drug regulations to protect public health.
USFDA Approval Process For Drug Products & Biological Product i.e NDA Vs. BLA
Comparison of NDA and BLA application process in USA. IND, NDA, ANDA & BLA dossier submission procedure.
The document discusses regulations around natural health products in Canada. The Natural Health Products Regulations came into force on January 1, 2004 and apply to all natural health products. A review of the regulations was launched within the first three to five years of implementation to address any challenges. Natural health products must be licensed and manufacturers must hold site licenses to ensure safety standards are met.
Orphan Drugs – the Challenges and Benefits of Navigating FDA’s Regime Governi...Michael Swit
Webinar sponsored by The Weinberg Group on Orphan Drugs, covering these topics:
The Basics of the Orphan Drug Act
Benefits of Orphan Drug status
Exclusivity
Protocol assistance, tax credits, and research grants
When is an indication is “rare”?
Orphan Drug Designation Requests – ensuring yours
robust and persuasive
Approval criteria for orphan products – how they
compare to non-orphan products
Challenges in the Orphan Drug Process
This is really a dry topic so I think these slide are helpful to you to learn well, this is generally covered in III year of Pharm.D under Pharmaceutical Jurisprudence.
So make use of this notes and prepare well.
Regulatory affairs professionals act as the interface between the pharmaceutical industry and drug regulatory authorities. Their main goals are to protect human health, ensure drug safety and quality, and ensure accurate product information. Key roles include liaising with regulatory agencies, preparing regulatory submissions, and advising on regulatory requirements and guidelines. An investigational new drug (IND) application is filed with regulatory agencies to legally test an experimental drug in humans after preclinical testing. A new drug application (NDA) is filed to obtain approval to market a new drug, while an abbreviated new drug application (ANDA) is filed for generic drug approval based on demonstrating bioequivalence to an existing drug. Drug master files and active substance master files provide confidential manufacturing
The document discusses different types of drug applications required for drug approval and marketing: Investigational New Drug Application (INDA), New Drug Application (NDA), and Abbreviated New Drug Application (ANDA). The INDA is required to begin clinical trials, the NDA contains all data from preclinical and clinical trials for new drug approval, and the ANDA allows generic versions of approved drugs to be marketed without repeating trials.
Presentation at the Biosimilars and Follow-On Biologics 2014 Americas Conference, sponsored by Paradigm Global Events, February 12, 2014. Presentation focused on:
•Interchangeability
•State Substitution Laws
•Naming
•Risk Evaluation & Mitigation Strategies (REMS) and Their Impact on Biosimilars
•Where FDA Stands on Biosimilars
The document provides an overview of the Waxman-Hatch Act of 1984, which established the modern generic drug approval pathway in the United States. It discusses the reasons for its creation, key provisions such as bioequivalence standards and patent certification requirements, and subsequent amendments. The Act sought to balance increased availability of low-cost generic drugs with incentives for continued pharmaceutical innovation.
COMMON JOB INTERVIEW QUESTIONS WITH ANSWERS ASKED IN DRUG REGULATORY AFFAIRS ...Pristyn Research Solutions
THESE ARE SOME COMMON JOB INTERVIEW QUESTIONS WITH ANSWERS ASKED IN DRUG REGULATORY AFFAIRS INTERVIEW
FOR ENROLLMENT CALL US ON - 9028839789
https://pristynresearch.com/
MAIL ID - pristynresearch@gmail.com
FACEBOOK- https://www.facebook.com/pristynsolutions
INSTAGRAM- https://www.instagram.com/pristyn_res...
TWITTER- https://twitter.com/Pristynresearch
SLIDESHARE- https://www.slideshare.net/azherkhan5916
LINKEDIN- https://www.linkedin.com/in/pristyn-research-191072119/
ADDRESS-
1) Parmar Trade Centre, A-wing,105/106, Sadhu Vaswani Chowk, Pune, 411001. Email: info@pristynresearch.com Phone: 09028839789
2)T-21/4 ,Opposite To Expert Global, Garware Stadium Road , Software Technology Park of India(STPI), MIDC, Aurangabad-431001. Email: info@pristynresearch.com Call us: 09607709586
The document outlines the phases of clinical trials:
- Phase 0 involves microdosing to determine pharmacokinetics and pharmacodynamics.
- Phase 1 studies a drug's safety on 20-100 healthy volunteers and finds the optimal dose.
- Phase 2 trials on 100-300 people study a drug's biological effects and continues safety monitoring. It has two types: 2a determines dosing and 2b is pivotal, blinded, and multicenter.
- Phase 3 are large randomized controlled trials on 300-3000 people comparing a drug to standard treatment. It has two types: 3a tests different indications and 3b continues trials pending regulatory approval.
- Phase 4 occurs after approval to detect rare adverse effects
The document discusses orphan drugs and regulations around them in various markets. It provides an overview of orphan drug policies in the US, EU, Australia, and Canada. The US Orphan Drug Act of 1983 was the first legislation to promote orphan drug development. It offers 7 years of market exclusivity. The EU and Canada have since established their own orphan drug frameworks that similarly aim to incentivize development of treatments for rare diseases through exclusivity periods, fee waivers, and assistance programs. However, orphan drugs regulations still face challenges around definitions of rare diseases, clinical data requirements, pricing and reimbursement.
Federal Regulatory Issues Us Food And Drug Administration Medical Device Amen...Jacobe2008
Authors:
Harvard-MIT Division of Health Sciences and Technology HST.535: Principles and Practice of Tissue Engineering Instructors: Myron Spector
Massachusetts Institute of Technology
Harvard Medical School Brigham and Women's Hospital VA Boston Healthcare
Drug design involves modifying molecules to complement biological targets through computer modeling. The drug development process includes research, clinical trials, and addressing financial costs that can take 10-15 years and $897 million to $1.9 billion per drug. Key steps involve identifying drug targets, developing assays to test effectiveness, finding lead compounds through ligand- or structure-based design, and refining activity through pre-clinical and clinical trials for FDA approval. After approval, drugs are formulated, produced, marketed, and can be produced generically once patents expire.
The document provides an overview of the drug discovery process. It discusses the various stages involved including target selection, lead discovery, medicinal chemistry, in vitro studies, in vivo studies, and clinical trials.
Target selection involves identifying biological targets implicated in disease through methods like genomics, proteomics, and bioinformatics. Lead discovery focuses on identifying small molecule modulators through synthesis, combinatorial chemistry, assay development, and high-throughput screening. Medicinal chemistry optimizes leads through approaches such as library development, SAR studies, in silico screening, and chemical synthesis. In vitro and in vivo studies evaluate drug candidates prior to clinical trials in humans.
International Journal of Drug Regulatory Affairs; 2014, 2(1), 1- 11
Abstract:
Developing a new drug requires great amount of research work in chemistry, manufacturing, controls, preclinical science and clinical trials. Drug reviewers in regulatory agencies around the world bear the responsibility of evaluating whether the research data support the safety, effectiveness and quality control of a new drug product to serve the public health. Every country has its own regulatory authority, which is responsible to enforce the rules and regulations and issue the guidelines to regulate the marketing of the drugs. This article focuses on drug approval process in different countries like USA, Europe and India.
The document discusses the FDA drug approval process and lists some drugs that have been withdrawn by the FDA. It describes the multi-phase approval process including pre-clinical testing in animals, followed by three phases of clinical trials in humans. It can take over a decade for a drug to be approved. Several drugs are mentioned that were withdrawn from the market due to serious safety issues discovered after approval, such as thalidomide causing birth defects, phenformin causing fatal lactic acidosis, and rofecoxib increasing heart attack and stroke risk.
Centralize & decentralize regulatory framework of brazil and south africaHarishankar Sahu
ANVISA is Brazil's centralized regulatory body that oversees pharmaceuticals and health products. It regulates drugs, medical devices, cosmetics and other products. In contrast, regulatory frameworks in African countries are more decentralized and vary between countries. The African Medicines Registration Harmonisation initiative aims to strengthen regulatory capacity and harmonize requirements across African nations to facilitate regional access to medicines.
Regulatory affairs professionals play a crucial role in ensuring pharmaceutical products meet regulatory standards for safety, efficacy and quality. They provide strategic guidance to various departments on regulatory requirements and work to obtain approvals from regulatory authorities. Key responsibilities include filing product registrations, tracking legal changes, and facilitating clinical trials and product approvals. Regulatory affairs has become increasingly important given historical issues that prompted stronger drug regulations to protect public health.
USFDA Approval Process For Drug Products & Biological Product i.e NDA Vs. BLA
Comparison of NDA and BLA application process in USA. IND, NDA, ANDA & BLA dossier submission procedure.
The document discusses regulations around natural health products in Canada. The Natural Health Products Regulations came into force on January 1, 2004 and apply to all natural health products. A review of the regulations was launched within the first three to five years of implementation to address any challenges. Natural health products must be licensed and manufacturers must hold site licenses to ensure safety standards are met.
Orphan Drugs – the Challenges and Benefits of Navigating FDA’s Regime Governi...Michael Swit
Webinar sponsored by The Weinberg Group on Orphan Drugs, covering these topics:
The Basics of the Orphan Drug Act
Benefits of Orphan Drug status
Exclusivity
Protocol assistance, tax credits, and research grants
When is an indication is “rare”?
Orphan Drug Designation Requests – ensuring yours
robust and persuasive
Approval criteria for orphan products – how they
compare to non-orphan products
Challenges in the Orphan Drug Process
This is really a dry topic so I think these slide are helpful to you to learn well, this is generally covered in III year of Pharm.D under Pharmaceutical Jurisprudence.
So make use of this notes and prepare well.
Regulatory affairs professionals act as the interface between the pharmaceutical industry and drug regulatory authorities. Their main goals are to protect human health, ensure drug safety and quality, and ensure accurate product information. Key roles include liaising with regulatory agencies, preparing regulatory submissions, and advising on regulatory requirements and guidelines. An investigational new drug (IND) application is filed with regulatory agencies to legally test an experimental drug in humans after preclinical testing. A new drug application (NDA) is filed to obtain approval to market a new drug, while an abbreviated new drug application (ANDA) is filed for generic drug approval based on demonstrating bioequivalence to an existing drug. Drug master files and active substance master files provide confidential manufacturing
The document discusses different types of drug applications required for drug approval and marketing: Investigational New Drug Application (INDA), New Drug Application (NDA), and Abbreviated New Drug Application (ANDA). The INDA is required to begin clinical trials, the NDA contains all data from preclinical and clinical trials for new drug approval, and the ANDA allows generic versions of approved drugs to be marketed without repeating trials.
Presentation at the Biosimilars and Follow-On Biologics 2014 Americas Conference, sponsored by Paradigm Global Events, February 12, 2014. Presentation focused on:
•Interchangeability
•State Substitution Laws
•Naming
•Risk Evaluation & Mitigation Strategies (REMS) and Their Impact on Biosimilars
•Where FDA Stands on Biosimilars
The document provides an overview of the Waxman-Hatch Act of 1984, which established the modern generic drug approval pathway in the United States. It discusses the reasons for its creation, key provisions such as bioequivalence standards and patent certification requirements, and subsequent amendments. The Act sought to balance increased availability of low-cost generic drugs with incentives for continued pharmaceutical innovation.
COMMON JOB INTERVIEW QUESTIONS WITH ANSWERS ASKED IN DRUG REGULATORY AFFAIRS ...Pristyn Research Solutions
THESE ARE SOME COMMON JOB INTERVIEW QUESTIONS WITH ANSWERS ASKED IN DRUG REGULATORY AFFAIRS INTERVIEW
FOR ENROLLMENT CALL US ON - 9028839789
https://pristynresearch.com/
MAIL ID - pristynresearch@gmail.com
FACEBOOK- https://www.facebook.com/pristynsolutions
INSTAGRAM- https://www.instagram.com/pristyn_res...
TWITTER- https://twitter.com/Pristynresearch
SLIDESHARE- https://www.slideshare.net/azherkhan5916
LINKEDIN- https://www.linkedin.com/in/pristyn-research-191072119/
ADDRESS-
1) Parmar Trade Centre, A-wing,105/106, Sadhu Vaswani Chowk, Pune, 411001. Email: info@pristynresearch.com Phone: 09028839789
2)T-21/4 ,Opposite To Expert Global, Garware Stadium Road , Software Technology Park of India(STPI), MIDC, Aurangabad-431001. Email: info@pristynresearch.com Call us: 09607709586
The document outlines the phases of clinical trials:
- Phase 0 involves microdosing to determine pharmacokinetics and pharmacodynamics.
- Phase 1 studies a drug's safety on 20-100 healthy volunteers and finds the optimal dose.
- Phase 2 trials on 100-300 people study a drug's biological effects and continues safety monitoring. It has two types: 2a determines dosing and 2b is pivotal, blinded, and multicenter.
- Phase 3 are large randomized controlled trials on 300-3000 people comparing a drug to standard treatment. It has two types: 3a tests different indications and 3b continues trials pending regulatory approval.
- Phase 4 occurs after approval to detect rare adverse effects
The document discusses orphan drugs and regulations around them in various markets. It provides an overview of orphan drug policies in the US, EU, Australia, and Canada. The US Orphan Drug Act of 1983 was the first legislation to promote orphan drug development. It offers 7 years of market exclusivity. The EU and Canada have since established their own orphan drug frameworks that similarly aim to incentivize development of treatments for rare diseases through exclusivity periods, fee waivers, and assistance programs. However, orphan drugs regulations still face challenges around definitions of rare diseases, clinical data requirements, pricing and reimbursement.
Federal Regulatory Issues Us Food And Drug Administration Medical Device Amen...Jacobe2008
Authors:
Harvard-MIT Division of Health Sciences and Technology HST.535: Principles and Practice of Tissue Engineering Instructors: Myron Spector
Massachusetts Institute of Technology
Harvard Medical School Brigham and Women's Hospital VA Boston Healthcare
Drug design involves modifying molecules to complement biological targets through computer modeling. The drug development process includes research, clinical trials, and addressing financial costs that can take 10-15 years and $897 million to $1.9 billion per drug. Key steps involve identifying drug targets, developing assays to test effectiveness, finding lead compounds through ligand- or structure-based design, and refining activity through pre-clinical and clinical trials for FDA approval. After approval, drugs are formulated, produced, marketed, and can be produced generically once patents expire.
The document provides an overview of the drug discovery process. It discusses the various stages involved including target selection, lead discovery, medicinal chemistry, in vitro studies, in vivo studies, and clinical trials.
Target selection involves identifying biological targets implicated in disease through methods like genomics, proteomics, and bioinformatics. Lead discovery focuses on identifying small molecule modulators through synthesis, combinatorial chemistry, assay development, and high-throughput screening. Medicinal chemistry optimizes leads through approaches such as library development, SAR studies, in silico screening, and chemical synthesis. In vitro and in vivo studies evaluate drug candidates prior to clinical trials in humans.
Computer aided drug design uses computational methods to facilitate the design and discovery of new therapeutic solutions. There are two main types of drug design - ligand-based which relies on knowledge of molecules that bind to the target, and structure-based which relies on the 3D structure of the target. The main steps in structure-based design are target selection, binding site identification, molecular docking to predict how ligands bind to the target, and scoring to evaluate interactions. Computational tools are used for databases, molecular modeling, docking, screening, and predicting absorption and toxicity properties. These tools help speed up the drug design process and make it more efficient.
Computer-aided drug design (CADD) uses computer modeling to help design and discover new drug molecules. Molecular docking is an important technique in CADD that predicts how drug molecules bind to their protein targets. Docking involves representing the drug and protein, searching for favorable orientations, and scoring the interactions. It is used in virtual screening to identify potential drug leads. Quantitative structure-activity relationships (QSAR) relate chemical structure to biological activity through statistical models. 3D-QSAR extends this by considering molecular 3D properties and is useful for drug design. CADD techniques like docking and QSAR help optimize drug candidates and reduce costs in the drug development process.
The document summarizes the process of drug discovery and development. It involves several long steps: understanding the disease, finding a biological target, discovering a lead compound through screening or nature, conducting preclinical testing on animals, and then clinical trials in three phases with humans to test safety and efficacy before the FDA decides whether to approve the drug. The entire process from discovery to approval takes an average of 10-15 years and costs $1-2 billion. Drugs also have different categories depending on how they are regulated and prescribed.
2016-11-28 Mentlife seminar: Pharmaceutical Drug Development; An Overall Pers...MentLife
This seminar provided an understanding of modern pharmaceutical drug development – the different phases of drug development and insight into different jobs.
Computer aided drug design (CADD) uses computational methods to simulate drug-receptor interactions. CADD is heavily dependent on bioinformatics tools like homology modeling, similarity searches, and physicochemical modeling. These "gears" can be aided by greasing them through easier collaboration between researchers using tools of Web 2.0 like Google Docs, mind maps, and slide sharing to integrate CADD gears. Gaming consoles like the PS3 are also being explored as affordable clusters for CADD applications.
The document discusses computer aided drug design (CADD). It describes CADD as using computational methods to aid in drug discovery and design. Some key points include:
- CADD uses tools like bioinformatics, cheminformatics, and computational chemistry to discover, study, and enhance drug molecules.
- Target-based and ligand-based approaches are two main computational methods used in CADD. Target-based approaches use structural information about biological targets while ligand-based approaches analyze characteristics of known active ligands.
- Other stages of drug design discussed include lead identification, lead optimization, docking simulations to model drug-target binding, and pre-clinical trials to evaluate drug properties before human testing.
This document discusses computational aided drug design. It begins by defining drug and the drug design process. It describes that the selected drug molecule should be an organic small molecule that is complementary in shape and oppositely charged to the target biomolecule. It then discusses ligand based and structure based drug design approaches. Various techniques used in drug design are also summarized such as x-ray crystallography, NMR, homology modeling, and computer aided drug design. Benefits of computational aided drug design include streamlining drug discovery, eliminating compounds with undesirable properties, and identifying and optimizing new drugs in a time and cost effective manner.
Drug discovery process style 5 powerpoint presentation templatesSlideTeam.net
The document describes the key stages in the drug discovery process, including cellular and genetic target identification, compound synthesis and isolation, high-throughput screening, lead optimization, preclinical testing in animal models and in vitro/in vivo studies, and clinical trials in humans. The flow diagram shows the iterative process moving from early research to identify biological targets through compound development and testing, culminating in clinical evaluation and potential approval of new therapeutics.
The document discusses herbal and drug design technology. It covers the process of drug discovery including target identification, lead identification, lead optimization, preclinical and clinical testing. It also discusses sources of lead compounds including natural products, observed drug side effects, and in silico computer-assisted drug design. Advances in molecular biology, genomics, high throughput screening and computer-aided drug design have revolutionized the field. Herbal drug design involves a multidisciplinary approach combining botanical, phytochemical and biological techniques to discover drugs from plants.
1) The document discusses the basics of drug design including defining the disease process, identifying targets for drug design like enzymes, receptors and nucleic acids, and the different approaches of ligand-based drug design and structure-based drug design.
2) It also covers important techniques in drug design like computer-aided drug design using computational methods, quantitative structure-activity relationships (QSAR), and the uses of computer graphics in molecular modeling and dynamics simulations.
3) Important experimental techniques discussed are x-ray crystallography and NMR spectroscopy that provide structural information for target biomolecules essential for structure-based drug design.
This document provides an overview of the history and methods of drug discovery, including traditional and computer-aided approaches. It discusses the traditional drug discovery life cycle from hit identification through random screening and the use of natural products and synthetic chemicals. It then introduces computer-aided drug design (CADD) and describes how it can be used throughout the drug discovery process, including structure-based design, ligand-based design, and de novo design to speed up screening and enable more rational drug design. It also lists some advantages of CADD over traditional methods and examples of drugs successfully developed using these approaches.
The document discusses the process of drug discovery, including target selection, lead discovery, medicinal chemistry, in vitro and in vivo studies, and clinical trials. Target selection involves identifying cellular or genetic targets involved in disease through techniques like genomics, proteomics, and bioinformatics. Lead discovery focuses on identifying small molecule modulators of protein function through methods like synthesis, combinatorial chemistry, assay development, and high-throughput screening. Medicinal chemistry then works to optimize these leads. [/SUMMARY]
The document discusses the complex and unpredictable nature of the FDA drug approval process. While the steps of drug development may seem formulaic, including discovery, preclinical testing, and clinical trials, success is not guaranteed as programs face many risks and intangible factors. Understanding these challenges is important for mitigating risks and strategizing development approaches. The FDA approval process aims to ensure new drugs are safe and effective for patients.
Naila Kanwal's document summarizes the new drug development and approval process. It describes the preclinical research phase involving animal and lab testing to determine safety and effectiveness. It then explains the clinical trial phases involving human subjects to further evaluate these factors. The document outlines the steps of submitting an Investigational New Drug application to the FDA for review and potential approval or requests for additional information before studies can begin. The overall process is designed to demonstrate a new drug is safe and effective for its intended use before being approved and marketed to the public.
Speaker: Anne Tomalin, BA, BSc, RAC (US, CAN & EU), President of CanReg Inc.
Topics Addressed
* Planning ahead to add credibility and value with your partners, investors and outside experts
* What is the landscape of various filings for a drug, a medical device or a diagnostic device?
* Developing a multi-disciplinary project team to manage your regulatory strategy:
o Different strategies for different products
o How to formulate a sound path to drug development decision-making
* What is needed for approval of a medical device
* The preclinical studies required for an IND filing
* The multiple components of the actual IND submission
* Management and communication between the teams assembling the IND
o When to outsource and bring in consultants
* Interacting with the regulatory authority
Download an audio file of this presentation at:
http://www.marsdd.com/bioent/jan15
Applications of bio-pharmaceutics in new drug deliveryAkshata shettar
Biopharmaceutics plays an integral role in new drug development from discovery through post-approval stages. The development process takes 10-15 years and costs $800 million to $1 billion, involving testing 5000-10000 molecules to find 1 approved drug. Biopharmaceutics evaluates drug properties like absorption, distribution, metabolism, and excretion during discovery and preclinical testing in animals. If successful, drugs then undergo three phases of clinical trials in humans to test for safety, efficacy, and dosage before potential approval and post-marketing surveillance. Biopharmaceutics aims to develop drug formulations and delivery systems that allow for optimal dosing intervals based on a drug's pharmacokinetic profile.
Typically, researchers discover new drugs through: New insights into a disease process that allow researchers to design a product to stop or reverse the effects of the disease. Many tests of molecular compounds to find possible beneficial effects against any of a large number of diseases.
This document provides an overview of the Investigational New Drug (IND) application process. It discusses how an IND is required to begin clinical trials on new drugs and allows pharmaceuticals to be transported between states for research purposes. The document outlines the various stages of pre-clinical and clinical testing, including pre-clinical studies in animals to establish safety, and the three phases of human clinical trials. It provides details on the key components of an IND application, including chemistry and manufacturing information, clinical protocols, and safety data from non-clinical studies. The overall goal of an IND is to obtain permission from the FDA to begin human clinical trials by demonstrating the new drug and trial design will not place subjects at unreasonable risk.
This document provides an overview of the Investigational New Drug (IND) application process. It discusses how an IND is required to begin clinical trials on new drugs and allows pharmaceuticals to be transported between states for research purposes. The document outlines the various stages of pre-clinical and clinical testing, including pre-clinical studies in animals to establish safety, and the three phases of human clinical trials. It provides details on the key components of an IND application, including chemistry and manufacturing information, clinical protocols, and safety data from non-clinical studies. The overall goal of an IND is to obtain permission from the FDA to begin human clinical trials by demonstrating the new drug and trial design will not place subjects at unreasonable risk.
Principles of Drug Discovery and DevelopmentMANIKANDAN V
This document provides an overview of the drug development and approval process, including:
1) Drug discovery typically involves new disease insights, testing molecular compounds, existing treatments with unexpected effects, or new technologies. Thousands of candidates are narrowed down based on early testing.
2) Once a compound looks promising, development involves experiments to understand absorption, benefits, dosage, delivery method, side effects, and effectiveness compared to other drugs.
3) The FDA approval process includes multiple phases of clinical trials involving healthy volunteers and patients to evaluate safety, efficacy, and side effect management strategies before approval or accelerated approval in some cases.
The document outlines the various stages involved in the new drug discovery and development process, including target identification, validation and screening, lead identification and optimization, preclinical and clinical testing through four phases, regulatory approval, and post-marketing surveillance. It notes that it takes on average 12-15 years and $900 million to $2 billion to develop a new drug, with only one in 5,000-10,000 compounds ultimately being approved due to the high failure rate of drug candidates.
Clinical trials and new drug developmentRahul Bhati
- The drug development process involves several phases of clinical trials and regulatory approval before a new drug can be approved and marketed. Key phases include pre-clinical testing in animals, followed by Phase I-III clinical trials in humans to test safety, efficacy, and proper dosing.
- After successful Phase III trials, companies submit a New Drug Application to regulators like the FDA with all clinical trial data. If approved, the drug is monitored in Phase IV post-marketing trials and surveillance.
- The overall process takes an average of 8-12 years and costs $500-600 million, with only about 20% of candidate drugs ultimately being approved due to rigorous testing requirements.
The new drug approval process involves several phases of laboratory and clinical testing that can take over a decade and cost hundreds of millions of dollars. Only about 1 in 1000 compounds that enter pre-clinical testing are approved for human testing. After pre-clinical animal testing, companies submit an Investigational New Drug (IND) application to the FDA to request permission for human clinical trials. If approved, the drug then enters three phases of clinical trials involving several hundred to thousands of human subjects to evaluate safety, efficacy, and proper dosing. If phase 3 trials are successful, companies submit a New Drug Application (NDA) to the FDA for review and potential approval allowing marketing and post-market safety surveillance.
This document discusses the process of new drug evaluation, which involves 3 phases - drug discovery, preclinical testing, and clinical trials. In the drug discovery phase, candidate molecules are selected. Preclinical testing involves animal studies to evaluate safety. Clinical trials with human subjects are then conducted in 3 phases to evaluate efficacy, safety, and adverse effects. The clinical trials process is highly regulated to ensure safety and data integrity. If successful, a New Drug Application is filed with regulatory authorities for approval to market the new drug.
This document discusses the phases of clinical trials for drug development. It explains that clinical trials have four phases - preclinical, phase I, phase II, and phase III - to test safety and efficacy in humans. Phase I trials use small groups of healthy volunteers, phase II uses patient groups to evaluate efficacy and side effects, and phase III uses thousands of patients to confirm effectiveness and monitor risks. After approval, phase IV trials monitor long-term safety and side effects. The document outlines the goals and procedures of each phase of clinical trials.
Phase III clinical trials involve 300 to 3,000 volunteers and last 1 to 4 years. They are designed to further assess a drug's safety, efficacy, and effectiveness. Only about 25-30% of drugs proceed from Phase II to Phase III. Phase III trials provide most of the safety data needed for regulatory approval and marketing. If results are satisfactory, trial findings are compiled into a regulatory submission for review by health authorities. Approximately 50% of drug candidates either fail Phase III trials or are rejected by regulatory agencies.
1. The document discusses regulatory requirements for drug approval, including non-clinical and clinical studies that must be conducted and submitted to regulatory agencies like the FDA.
2. It describes the various teams involved in drug development, including discovery, preclinical, clinical, manufacturing, and marketing teams. The responsibilities and roles of each team are provided.
3. The approval process is outlined, including requirements for an Investigational New Drug (IND) application to the FDA. The IND must provide data from animal and other preclinical studies. It allows clinical trials to proceed if approved by the FDA within 30 days.
The document provides an overview of the drug discovery and development process. It discusses the various stages including drug discovery, preclinical drug development, investigational new drug application, clinical trials in three phases, FDA review and approval process, new drug application, and post-approval monitoring. The key stages involve identifying a target, developing lead compounds, optimizing drug candidates, conducting preclinical studies in animals, clinical trials in humans to test safety and efficacy, regulatory review and approval, and post-market surveillance. The goal is to develop new drugs and therapies to treat diseases and medical conditions.
This presentation discusses the various phases of clinical trials for drug development. It explains that clinical trials are conducted in phases (1-4) to evaluate a drug's safety, efficacy, optimal dosage and monitor long-term effects. Phase 1 trials test safety on a small group. Phase 2 assesses effectiveness on patients with the disease. Phase 3 studies the overall benefit-risk ratio on a large patient group. Phase 4 occurs after marketing approval to collect additional safety data from a larger population over a longer period. Regulatory approval from bodies like DCGI and FDA is required by submitting investigational documents and new drug applications between phases.
The document discusses the Investigational New Drug (IND) application process with the FDA. An IND application allows a company to ship an experimental drug across state lines and begin clinical trials. It must include preclinical data to show the drug is safe for initial human use as well as protocols for proposed studies. The FDA reviews the IND for 30 days before clinical trials may begin to ensure subject safety. The overall goal of an IND is to facilitate testing of new drugs while protecting clinical trial participants.
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1. The Drug Development Process
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The Drug Development Process – Clinical Development
The Progressive Stages of Clinical Development
• Following the “Drug Discovery” process, a “Drug Candidate”
is nominated for clinical development
• A single “Lead Compound” is chosen to move forward.
• There may also be additional “back-up” compounds
nominated, but treated with a (much) lower priority.
3.
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The Drug Development Process – Clinical Development
Stages of Clinical Development
1.) Preclinical
2.) Phase I
3.) Phase II (Phase IIa & Phase IIb)
4.) Phase III
5.)Registration
6.) Commercial Launch
7.) Phase IV
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The Drug Development Process – Preclinical Development
Preclinical Development
• Before testing a new medicine in humans, researchers conduct
extensive “preclinical testing” of the molecule.
• They perform various experiments with “in vitro” (i.e. in a petri dish,
vial, or beaker in the laboratory) or in animal models (usually simpler
species such as rodents).
• The activity of the drug candidate is carefully examined under many
different conditions prior to moving it into human testing in medical
clinics.
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Preclinical Development (continued)
• Under FDA requirements, a company must first submit data showing
that the drug is reasonably safe for use in initial, small-scale clinical
studies.
• Genotoxicity screening is performed, as well as investigations on
drug absorption and metabolism, the toxicity of the drug's metabolites,
and the speed with which the drug and its metabolites are excreted
from the body.
The Drug Development Process – Preclinical Development
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Preclinical Development (continued)
At the preclinical stage, the FDA will generally ask, at a minimum,
that the developers:
(1) develop a pharmacological profile of the drug;
(2) determine the acute toxicity of the drug in at least
two species of animals.
(3) conduct short-term toxicity studies ranging from 2 weeks to
3 months, depending on the proposed duration of use of the
substance in the proposed clinical studies.
The Drug Development Process – Preclinical Development
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Preclinical Development (continued)
Sponsor/FDA Meetings (Pre-IND)
• Prior to clinical studies, the sponsor needs evidence that the compoun
is biologically active, and both the sponsor and the FDA need data
showing that the drug is reasonably safe for initial administration to
humans.
The Drug Development Process – Preclinical Development
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Investigational New Drug Application
• The investigational new drug (IND) application is the result of a
successful preclinical development program.
• The IND is also the vehicle through which a company advances to the
next stage of drug development known as clinical trials (human trials).
• The IND is not an application for marketing approval.
• It is a request for an exemption from the Federal statute that prohibit
an unapproved drug from being shipped in interstate commerce.
The Drug Development Process – Preclinical Development
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Investigational New Drug Application(continued)
• Animal Pharmacology and Toxicology Studies
Preclinical data to permit an assessment as to whether the product
is reasonably safe for initial testing in humans.
• Manufacturing Information
Information pertaining to the composition, manufacture, stability,
and controls used for manufacturing the drug substance and the
drug product. This information is assessed as to ensure the company
can adequately produce and supply consistent batches of the drug.
The Drug Development Process – Preclinical Development
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Investigational New Drug Application (continued)
• Clinical Protocols and Investigator Information
Detailed protocols for proposed clinical studies to assess whether
the initial-phase trials will expose subjects to unnecessary risks.
Also, information on the qualifications of clinical investigators—
professionals (generally physicians) who oversee the administration
of the experimental compound--to assess whether they are qualified to
fulfill their clinical trial duties.
The Drug Development Process – Preclinical Development
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Phase 1 Considerations
•CMC safety issues as they relate to the quality aspects of product
•What is the risk for human subjects? Are there any signals from
preclinical studies?
•The product class and the individual product affect, to some extent,
the type and extent of information needed to assess safety.
–Often novel and complex products require additional
information to address unknowns and added
complexity (e.g., transgenic, xenotransplantation)
The Drug Development Process – Preclinical Development
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The Drug Development Process – Phase I Clinical Development
Phase I Clinical Development
• Phase I clinical trials are designed to examine the safety of a
drug candidate.
• Further clinical trials and development cannot happen unless Phase I
trials demonstrate the drug candidate to be reasonably safe when
administered to humans.
• Some side effects may be acceptable in relation to the severity of
the targeted disease (i.e. chemotherapy).
• Participants in Phase I are closely monitored for the smallest
indication of harm caused by the medicine.
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Phase I Clinical Development (continued)
• Safety is the key issue. Thus these trials are purposely conducted
with a small scope (limited number of participants).
• An attempt is made to establish the dose range tolerated by
volunteers for single and for multiple doses. .
•Phase I trials are sometimes conducted with severely ill patients
(e.g. in the field of cancer).
The Drug Development Process – Phase I Clinical Development
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Phase I Clinical Development (continued)
• In Phase 1 studies, CDER can impose a clinical hold (i.e., prohibit
the study from proceeding or stop a trial that has started) for reasons
of safety, or because of a sponsor's failure to accurately disclose the
risk of study to investigators.
FDA Role in Phase I
CDER = Center for Drug Evaluation & Research
The Drug Development Process – Phase I Clinical Development
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Phase I Clinical Development
Phase I Clinical Development is important
Why?
Because that is when the dosage form is beginning to
be determined.
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The Drug Development Process – Clinical Development
Phase IIa Clinical Development
•Pilot clinical trials (“Pilot” means focused on obtaining information
and working out trial logistics) evaluate efficacy (and safety) in
selected populations of patients with the disease or condition to be
treated, diagnosed, or prevented.
•Objectives may focus on dose-response, type of patient, frequency
of dosing, or numerous other characteristics of safety and efficacy.
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The Drug Development Process – Clinical Development
Phase IIb Clinical Development
Well-controlled trials to evaluate efficacy (and safety) in patients
with the disease or condition to be treated, diagnosed, or prevented.
These clinical trials usually represent the most rigorous demonstration
of a medicine’s efficacy. Sometimes referred to as pivotal trials.
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The Drug Development Process – Clinical Development
Phase II Clinical Development
• Phase II clinical trials are designed to further assess safety and
evaluate efficacy in humans over the short term.
• Phase II clinical trials help set up parameters (e.g. dosage) of
the longer-term Phase III trials.
• Phase II trials are typically placebo controlled and double
blinded.
• Neither the patient nor the medical personnel know whether
the patient is receiving the drug or the placebo.
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The Drug Development Process – Clinical Development
Phase II Clinical Development (continued)
• These trials are larger in scope than Phase I and take more time.
• In an effort to save time and money, some companies try to
minimize the size and scope of Phase II trials.
• Some companies rely on information from preclinical and Phase I
trials to design comprehensive Phase II trials that can clearly
demonstrate the efficacy (or lack thereof) of a drug before a
Phase III trial.
• This approach minimizes the risk of unpleasant surprises
following the far more expensive Phase III trials.
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The Drug Development Process – Clinical Development
Phase III Clinical Development
•Phase III trials are designed to prove the efficacy and confirm
the safety of the drug.
• They are usually double-blinded and placebo-controlled and can
involve hundreds or even thousands of patients over many months
or even years.
• Though the numbers of patients involved can be great, the risks
are relatively minimal because of the earlier testing to establish safety
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The Drug Development Process – Clinical Development
Phase IIIa Clinical Development
•Trials conducted after efficacy of the medicine is demonstrated, but
prior to regulatory submission of a New Drug Application (NDA)
or other dossier.
•These clinical trials are conducted in patient populations for which
the medicine is eventually intended.
•Phase IIIa clinical trials generate additional data on both safety and
efficacy in relatively large numbers of patients in both controlled
and uncontrolled trials.
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The Drug Development Process – Clinical Development
Phase IIIa Clinical Development
•Clinical trials are also conducted in special groups of patients
(e.g. renal failure patients), or under special conditions dictated
by the nature of the medicine and disease.
•These trials often provide much of the information needed for the
package insert and labeling of the medicine.
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The Drug Development Process – Clinical Development
Phase IIIb Clinical Development
•Clinical trials conducted after regulatory submission of an NDA or
other dossier, but prior to the medicine’s approval and launch.
•These trials may supplement earlier trials, complete earlier trials, or
may be directed toward new types of trials (e.g. quality of life,
marketing) or Phase IV evaluations.
•This is the period between submission and approval of a regulatory
dossier for marketing authorization.
•Comparisons to standard therapy (termed comparator studies) can
be included in either Phase IIIa or Phase IIIb.
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The Drug Development Process – Clinical Development
Phase II & III Clinical Development
FDA Involvement
• In both Phase 2 and 3, CDER can impose a clinical hold if a study
is unsafe (as in Phase 1), or if the protocol is clearly deficient in
design in meeting its stated objectives.
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The Drug Development Process – Clinical Development
Phase IV/Registration
• Once all phases of clinical testing have been completed, if the data
warrants it, the company applies to the Food and Drug Administration
for regulatory approval to market the medicine in the United States.
• If the FDA agrees that the data proves the safety and efficacy of the
drug, approval may come in several months or years, with those
medicines with the most urgent medical need typically approved
more quickly.
• The company should work closely with regulatory agencies at all
stages of clinical development to help ensure it can provide needed
data to obtain approval as quickly as possible.
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The Drug Development Process – Clinical Development
New Drug Application
• Since 1938, every new drug has been the subject of an approved NDA
before U.S. commercialization.
• The data gathered during the animal studies and human clinical trials
of an Investigational New Drug (IND) become part of the NDA.
• The components of any NDA are, in part, a function of the nature
of the subject drug and the information available to the applicant at
the time of submission.
• The NDA must provide all relevant data and information
that has been collected during the product's research and development
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The Drug Development Process – Clinical Development
New Drug Application (continued)
• Although the quantity of information and data submitted in NDAs
can vary significantly, the components of NDAs are more uniform.
• NDAs can consist of as many as 15 different sections:
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The Drug Development Process – Clinical Development
New Drug Application (continued)
•Index;
•Summary;
•Chemistry, Manufacturing, and Control;
•Samples, Methods Validation Package, and Labeling;
•Nonclinical Pharmacology and Toxicology;
•Human Pharmacokinetics and Bioavailability;
•Microbiology (for anti-microbial drugs only);
•Clinical Data;
•Safety Update Report (typically submitted 120 days after the NDA's
submission);
•Statistical;
•Case Report Tabulations;
•Case Report Forms;
•Patent Information;
•Patent Certification; and
•Other Information.
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NDA Actions
• Once an approval, approvable, or non-approvable recommendation is
reached by the reviewers and their supervisors, the decision must be
evaluated and agreed to by the director of the applicable drug review
division or office.
• The division director generally serves as the final FDA ruling.
• Once the division director (or office director, as appropriate) signs an
approval action letter, the product can be legally marketed starting that
day in the United States.
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The Drug Development Process – Clinical Development
Phase IV
•Studies or trials conducted after a medicine is marketed to provide
additional details about the medicine’s efficacy or safety profile.
•Different formulations, dosages, durations of treatment, medicine
interactions, and other medicine comparisons (termed comparator
studies) may be evaluated.
•New age groups, races, and other types of patients can be studied.
•Detection and definition of previously unknown or inadequately
quantified adverse reactions and related risk factors are an important
aspect of many Phase IV studies.
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The Drug Development Process – Clinical Development
Phase IV
•If a marketed medicine is to be evaluated for another (i.e. new)
indication, then those clinical trials are considered Phase II clinical
studies.
•The term post-marketing surveillance is frequently used to describe
those clinical studies in Phase IV (i.e. the period following marketing)
that are primarily observational or non-experimental in nature, to
distinguish them from well controlled Phase IV clinical trials or
marketing studies.
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Accelerated Development/Review
Other Types of Approvals/Reviews
• Accelerated development/review is a highly specialized
mechanism for speeding the development of drugs that promise
significant benefit over existing therapy for serious or
life-threatening illnesses for which no therapy exists.
• This process incorporates several novel elements aimed at making
sure that rapid development and review is balanced by safeguards
to protect both the patients and the integrity of the regulatory process
• The fundamental element of this process is that the manufacturers
must continue testing after approval to demonstrate that the drug
indeed provides therapeutic benefit to the patient. If not, the FDA
can withdraw the product from the market more easily than usual.
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Other Types of Approvals/Reviews
Treatment IND
• Treatment Investigational New Drugs are used to make promising new
drugs available to desperately ill patients as early in the drug
development process as possible.
• FDA will permit an investigational drug to be used under a treatment
IND if there is preliminary evidence of drug efficacy and the drug is
intended to treat a serious or life-threatening disease, or if there is no
comparable alternative drug or therapy available to treat that stage of
the disease in the intended patient population.
• In addition, these patients are not eligible to be in the definitive clinical
trials, which must be well underway, if not almost finished.
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Other Types of Approvals/Reviews
Treatment IND (continued)
• An immediately life-threatening disease means a stage of a disease
in which there is a reasonable likelihood that death will occur
within a matter of months or in which premature death is likely
without early treatment.
• Treatment INDs are made available to patients before general
marketing begins, typically during Phase 3 studies.
• Treatment INDs also allow FDA to obtain additional data on the
drug's safety and effectiveness.
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Other Types of Approvals/Reviews
Parallel Track
• The "parallel track" policy was developed by the U.S. Public Health
Service in response to AIDS.
• Under this policy, patients with AIDS whose condition prevents them
from participating in controlled clinical trials can receive
investigational drugs shown in preliminary studies to be promising.
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•An orphan drug is any drug developed under the Orphan Drug Act of
January 1983 ("ODA"), a federal law concerning rare diseases
("orphan diseases"), defined as diseases affecting fewer than 200,000
people in the United States or low prevalence (prevalence of less than
5 per 10,000 in the community.
•This has been adopted as a subclause of the Food and Drug
Administration (FDA) regulations. The granting of orphan drug
status is designed to encourage the development of drugs which are
necessary but would be prohibitively expensive/un-profitable to
develop under normal circumstances.
Orphan Drug
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Because medical research and development of drugs to treat such
diseases is financially disadvantageous, companies that do so are
rewarded with tax reductions and marketing exclusivity (a "monopoly")
on that drug for an extended time (seven years post-approval).
The concept behind the ODA is that the longer period of exclusivity
will encourage more companies to invest money in research.
Orphan Drug
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Under the act many drugs have been developed, including drugs to
treat glioma, multiple myeloma, cystic fibrosis, phenylketonuria and
snake venom.
In the US, from January 1983 to June 2004, a total of 1,129 different
orphan drug designations have been granted by the Office of Orphan
Products Development (OOPD) and 249 orphan drugs have received
marketing authorization in the US.
In contrast, the decade prior to 1983 saw fewer than ten such
products come to market.
Orphan Drug