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Base Case

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