In this unique and detailed case study you will follow Dr G and his international project team as they develop a drug and monoclonal antibody.
With an international project team starting from an idea, target product profile, development is followed in detail from idea through to the market with reference to all key steps, documents, guidances and cost and time plans. Typical development problems arise and are solved. Theoretical concepts are linked to the Base Text and use is made of the Spreadsheets and Templates in the e-Tools , all of which are provided with the case.
Beginners or specialists in one area may in parallel follow the Base Course which is linked optionally to the Case for those licensing this feature.
This is a long and eventful ride, but by the time you finish it you will understand the practice behind the theory of bringing a new medicine from the laboratory to the market.
Activities covered are further described below.
Global Medicines Development in Practice
Goal: To develop deep understanding of all aspects of medicines development through multiple hands-on simulations of key activities in small molecule and biologic development. Participants should have completed the Introduction to Global Medicines Development Course or be able to demonstrate broad basic knowledge of all development areas through previous participation in development courses and/or practical work experience
All case work is submitted in writing as signed pdf files. After assessment each file is entered into the participant’s Binder as a permanent record and future reference source.
Introduction to the simulated cases, Junglex and Junglinamab, and the virtual project team
Creation of target product profiles; use of label-based spreadsheets to build up the desired profile and identify associated development needs
Creation of medicines development plans, including graded risk scenarios, Go-No-Go Decisions and Budgets
Interpretation of nonclinical pharmacokinetic assessments including ADME
Interpretation of validation reports for bioanalytical assays, and assays for biomarkers
Reviewing genotoxicity data
Reviewing safety pharmacology including HERG channel data
Reviewing CYP profiling data
Reviewing general repeat dose toxicity data
Assessment of safety margins
Reviewing typical reprotoxicity data
Reviewing typical carcinogenicity data
Planning the timing of toxicity studies in relation to clinical development
Planning of non-clinical safety testing for biologics and advanced therapies
Applying PK-PD modelling approaches at the preclinical development stage
Review of Medicines Development Plan
Managing changes in manufacturing process during development, including dealing with the issue of new impurities
Identifying and managing Issues around isomers, crystalline forms and other variants in drug substance during development
Reviewing the typical specifications for drug substance for small molecules and biologics
Reviewing and planning stability data requirements for clinical studies
Management of expiry dates for clinical formulations
Reviewing process validation reports and understanding their central importance in GMP
Handling blinding of clinical supplies for double-blind studies
Planning the quantity and timing of supplies for clinical studies
Planning an IVRS system
Planning bioequivalence studies, requesting biowaivers
Review of Medicines Development Plan (2)
Preparation of briefing books and questions for regulatory authorities.
Compiling an Investigator`s brochure
Compiling IND submission documents
Compiling CTA/IMPD submission documents in EU
Understanding compilation of clinical trial applications in Japan and China
Filing for Orphan designation
Motivating teams
Review of Medicines Development Plan (3)
Estimating sample sizes for simple clinical studies
Conducting simple statistical analyses for clinical results
Compiling Ethics Committee and IRB submissions
Reviewing informed consent forms
Understand the special aspects of informed consent in vulnerable populations
Handling issues in a typical site contracts
Selecting a CRO
Making a Gantt chart to cover all aspects of study start up
Drafting a clinical study protocol
Assessing study budgets
Reviewing Case Report Forms
Reviewing steps in database setup
Submitting a protocol amendment
Reviewing a statistical analysis plan
Planning investigator meetings
Planning training sessions and rater qualification
Conducting a monitoring visit including pre and end of study visit
Handling protocol deviations and violations
Recording compliance
Locking a database
Planning an interim analysis
Setting up and running a Data Safety Monitoring Board
Conducting an external audit of a clinical site
Evaluating adverse events during clinical trials including clinical laboratory and ECG findings
Managing reporting of Serious Adverse Events during clinical studies
Reporting SUSARs during a clinical study
Preparing a Development Safety Update Report
Preparing a Periodic Risk Benefit Assessment
Handling communication in international teams
Review of Medicines Development Plan
Calculating starting doses for small molecules and monoclonal antibodies
Designing a first in man study
Describing basic pharmacokinetic results
Incorporating pharmacogenetics in early drug development
Incorporating pharmacogenomics in development
Making typical study designs for the influence of food, age, gender, renal and hepatic dysfunction on pharmacokinetics
Identifying need for and designing typical drug-drug interaction studies
Designing of a thorough QTc study
Incorporating PK/PD modelling in early clinical development
Review of Medicines Development Plan (4)
Planning parallel activities in Phase II
Designing proof of concept studies with a) biomarkers and b) clinical endpoints
Understanding the goals of Phase IIB and the issues around combination of this Phase with Phase III
Designing a dose finding study, including use of PK-PD modelling
Making an outline for a study with adaptive design, and describing related issues
Understand through examples the concept of population pharmacokinetics and modelling
Making a table of contents for the trial master file.
Handling slow recruitment in clinical studies
Review of Medicines Development Plan
Designing Phase III programs including planning of parallel nonclinical activities
Choosing and incorporating comparator drugs in Phase III
Incorporating population pharmacokinetic modelling in late stage development
Planning how to reduce variability when scaling up to many clinical sites in Phase III: planning training and other investigator meetings
Describing the typical analysis of safety data as presented in study reports
Describing the typical analysis of efficacy data for different types of data
Drafting a Clinical Study Report
Designing programs for drug/device combinations, incorporating key medical device regulations
Describing the typical pharmacoeconomic analyses
Describing the key elements and terminology of Evidence Based Medicine, Meta-analyses and Health Technology Assessment
Review of Medicines Development Plan
Compilation of a submission for approval using the Common Technical Dossier:
Module 1
Module 2
Module 3
Modules 4 and 5
Describing the procedures and timelines for centralised, mutual recognition and descentralised procedure in EU
Describing the timelines and procedures for NDA review in the US
Describing the regulatory aspects of pediatric study planning in EU and US
Compilation of a Risk Management Plan in EU and Risk Evaluation and Mitigation Strategy in US
Describing the submission process in Japan including the concept of bridging studies
Describing the submission process in China
Compiling the submission of a generic drug/ANDA submission
Describing the submission of biosimilar products
Making typical label changes/Type I and 2 variations
Preparing for an advisory committee/scientific advisory group hearing during the submission process
Review of Medicines Development Plan
Reviewing issues around advertising and promotion
Preparing a high quality publication of your results
Describing red flags in reviewing publications
Reviewing examples of the different types of Phase IV studies
Describing pharmacovigilance and signal detection techniques for drugs on the market
Outlining the contents of a Periodic Safety Update Report
Putting together a typical Life Cycle Management Plan
Identifying upside potential – new indications
Identifying upside potential – new formulations
Constructing a sales forecast
Making a Net Present Value estimation
Conduction of due diligence on a potential in-licensing candidate, with customisation of checklists which can also be used for in-house project risk assessment
Cost assessment of new project using Gray´s Medicines Development e-Tools