Content

1- Setting the scene : the context of drug development 

• Overview and drivers of pharmaceutical innovation 
• Specificities of the pharmaceutical market 
• Changes in the healthcare environment 

2- Integrated product development : from discovery phase to market authorization

• Overview of the process of drug development 
• How to achieve marketing approval and commercial excellence in a changing health care environment ?
• Evolution from blockbuster to personalized medicine 
• The rising importance of orphan drugs 
• Patenting : basic criteria, issues in a changing health care environment and open-innovation 

3- Drug screening : from animals to recombinant proteins and translational research 

• Testing in animals : current use, ethical and regulatory aspects, limits ans alternative approachs 
• High Troughput Screening : setting-up of new functional assays, robustness, troughput and quality of assays
• Systems biology and translational research

4- Big data and personnalized medicine : How AI can potentially reinvent the drug design and development ?

• Concept of personalized medicine 
• Patient selection 
• AI for Clinical Trial design 
• Big data, Robots and AI in Medecine and Healthcare
• Impact on drug development and marketing
• AI in Medecine 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- Principal steps in drug development 

2- Specificities of the current changing heath care environment 

3- Principal steps and innovations in drug screening 

4- Principles of translational research and personnalized medecine 

5- Would-be contribution of Artificial Intelligence in drug design and development  

Content

1- Pre-clinical strategy and Study design 

• GLP and Pre-clinical testing requirements 
• Stages of development and registration  
• Study protocol design  
• Pre-clinical strategy : time and costs analysis 
• Special population requirements and Regulatory Compliance 

2- Pre-clinical safety  : Principles of pharmacology and toxicology 

•  Non-clinical testing for chemical and biological compounds, including pharmacology (ADME)
• How to choose the dose for first in human administration ? 
• Toxicology testing : general toxicology, genotoxicity, carcinogenicity
• Genetic and genomic factors in drug development and drug response 
• Novel approaches in investigative toxicology 
• In silico toxicology methods : applications 

3- Drug formulation 

• Main apsects of drug formulation 
• Concept of drug bioavailability 
• Non-conventional dosage forms : oral sustained release dosage forms, transdermal forms, injectable biodegradables microspheres, etc 
• Nanotechnologies for drug delivery : potential and challenges 
• 3D printing (drugs and medical devices) : what is possible to do now ? 

4- Manufacturing  

• Pharmaceutical technical development : formulations, primary packaging, delivery system 
• Quality Management System, Quality by design and Process Analytical Technologies 
• GMPs and Required structures of departments/systems in development and manufacturing 
• EMA and FDA agencies and inspection-operating environment for product development and manufacturing (concept of inspection readiness)  

 5- Moving to First-in-Human studies 

• Ethics in the Pharmaceutical Industry 
• Requirements, ethics and regulations of First-in-Human studies 
• Accelerating the move to clinical trials (possibilities and opportunities of computer assisted modelling on the way of proof of concept) 
• Strategic importance and practical organization of Phase I studies (responsibilities, planning and activities) 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- Principles of pre-clinical strategy 

2- Requirements, ethics and regulation of pre-clinical and First-in-Human studies 

3- Basic concepts of pre-clinical pharmacology and toxicology 

4- Main aspects of drug formulation and innovations in this area 

5- Pharmaceutical technical development 

6- Requirements and regulations in drug manufacturing 

7- Planning and practical organization of Phase I studies 

Content

1- Early studies in patients 

• Dose-finding studies 
• Proof of mechanism studies 
• Bioequivalence testing 
• Clinical Pharmacokinetics and Pharmacodynamics
• Confirmatory clinical development plan 

2- Clinical trials Designs 

• Different types of clinical trials : study designs 
• Randomisation modes 
• Statistical aspects in the planning of clinical trials (choice of the enpoints, hypothesis, Alpha and Beta risks, sample size calculation) 
• Innovative study designs 

3- Planning and managing clinical trials 

• Regulatory aspects troughout the product development cycle (GCP requirements) 
• Set-up of clinical trial 
• Monitoring safety in clinical trials and Drug Development 
• Development Risk Management Plans – The challenges 
• Risk-based and remote monitoring in clinical trials 
• Patient centric approach : Direct-to-patients trials and logistics
• Outsourcing of clinical trials and contrat management 
• Audits in clinical trials : Key feature of audits and inspections, keys to preparing for and effectively managing inspection, roles and expectations of key personnel, differences between inspections from various authorities 

4- Collecting clinical trials data 

• Clinical trial data collection and data management 
• Patient adherence and persistence in trials (uptake of mobile health, e-tools, big data analysis and personnalized medicine) 

5- Blockchain in Clinical trials 

• Hope or reality ? 
• Cases for blockchain application in clinical trials 
• Barriers and challenges yet to be resolved 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- Main aspects of early studies in patients and their impact on the drug development plan 

  2- Design of various types of clinical studies ans statistical aspects of these designs 

  3- Key issues involved in a conduct of a clinical study in terms of Good Clinical Practice (GCP) 

  4- Main safety aspects in clinical trials and drug development 

  5- Principles and tools of data collection 

  6- Key steps and methods in data management 

  7- Applications of Blockchain in clinical trials 

Content

1- Clinical trial protocol and Investigator drug brochure (IDB)  

• Clinical trial protocol : a key document 
• Reading and understanding a clinicial trial protocol 
• Role of the Ivestigator Drug Brochure (IDB) 
• Regulatory codes and guidance for authoring the IDB 

2- Biostatistics : Clinical data Evaluation 

• Statistical methods used in clinical research 
• Common errors in statistics and how avoid them 
• Critical reading of a scientific publication on biostatistics 
• Understanding the crisis around the p-value : Statistical methods for the 21st century 

3- Safety data Evaluation  

• Methods for the evaluation of Safety Data in clinical trials
• Background and implementation of Development Safety Update Reports (DSUR) 
• Data Safety Monitoring boards 

4- Data Interpretation and Scientific writing 

• Different aspects of the Clinical study report : data and conclusions on paper 
• Preparation of review documents for regulatory submissions 
• Writing a manuscript for publication 
• Submission to a peer-review journal, dealing with the reviewer’s comments 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- Development of a clinical trial protocol and the role of the Investigator Drug Brochure (IDB) 

2- Evaluation and interpretation of clinical trial results in a scientific publication 

3- Main statistical methods used in clinical data analysis and understand the current challenges aroud the p-value 

4- Main aspects of the Clinical Study Report and the documents for regulatory submission 

Content

1- Drug Registration : European procedures and International environment 

• Overview of the regulatory environment and medicines registration in the EU 
• Comparison with the US FDA requirements and Japanese authorities 
• Comparison with requirements in international and emerging-markets 
• The Common Technical Document (CTD) format 
• Special issues : orphan products 
• Understanding defects in the application and improving the chances of approval 
• Collaborate with the FDA to facilitate innovation 

2- Pharmacovigilance & Medical information

• Back to basics in Pharmacovigilance 
• EU regulations and Guidelines in Pharmacovigilance 
• Pharmacovigilance in daily practice 
• Overview of Patient Support ProgramS (PSPS)
• Recent trends in Pharmacovigilance 
• Medical information : communication with health practitioners 
• Summary of products characteristics and user package leaflet 

3- Pharmacoepidemiology 

• Principles of pharmacoepidemiology 
• Study methods and data ressources 
• Interpretation of pharmacoepidemiological data 
• Role of the pharmacoepidemiology in the lifecycle of a medicine 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- General principles of medicinesregulation (pre- and post- approval) 

2- Principles of pharmacovigilance and medical information 

3- Role of Pharmacoepidemiology in the lifecycle of a medecine 

Content

1- Principles and practical applications of health economics 

• Health Economic Evaluation of medicinal products : concept of study quality and the methods used to assess quality 
• Health technology assessment 
• Interpretation of the cost-effectiveness plane 
• Impact of the economic evaluation in the field of market access 
• Pricing and reimbursement of medicinal products (overview, issues and challenges in EU) 
• Patient reported outcomes – Real world data and Real world evidence 

2- Market access  

• Ethical and legal aspects 
• Macroeconomic aspects 
• Medical affairs, regulatory environment and market access in emerging markets 
• Pharmaceutical marketing : lifecycle of a medicinal product, trademark, business model 

3- Innovation and improvement of health care 

• Which new products are really innovative ? 
• Health data bases for innovation and improving health care 

Learning outcomes

At the end of this module, the student should be able to outline the : 

1- Principles and practical applications of health economics and Health Technology Assessment within the healthcare marketplace 

2- Ethical and legal aspects of market introduction of a drug 

3- Specificities of a really innovative product 

Biologics, Vaccines, ATMPs, Medical advices : how do they differ from conventional drugs ?

1. Specificities of the clinical development of Biologics
2. Medical advices : Overview from manufacturer to patient
3. Advanced Therapy Medicinal Products (ATMPs)
4. Vaccines development challenges : from preclinical to clinical studies