Content

1- Setting the scene: drug development in context

• Overview and drivers of pharmaceutical innovation 
• Particularities of the pharmaceutical market 
• Evolutions in the healthcare environment 

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

• Overview of the drug development process
• Obtaining marketing approval and delivering commercial excellence in an evolving healthcare environment
• The shift from blockbusters to personalized medicine 
• The rising importance of orphan drugs
• Patenting: basic criteria, challenges in an evolving health care environment, and open-innovation 

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

• Animal testing: current use, ethical and regulatory aspects, limitations and alternative approaches 
• High Throughput Screening: setting-up new functional assays, robustness, throughput and assay quality
• Systems biology and translational research

4- Big data and personalized medicine: can AI reinvent drug design and development?

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

Learning outcomes

At the end of this module, students should be able to describe: 

1- The main steps in drug development 

2- Particularities of the evolving healthcare environment today

3- The Main steps and innovation in drug screening 

4- Principles of translational research and personalized medicine 

5- How Artificial Intelligence can play a role 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 cost analysis 
• Special population requirements and regulatory compliance 

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

• Non-clinical testing of chemical and biological compounds, including pharmacology (ADME)
• The right 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 aspects of drug formulation 
• Concept of drug bioavailability 
• Non-conventional dosage forms : oral sustained release dosage forms, transdermal forms, injectable biodegradable microspheres, etc.
• Nanotechnologies for drug delivery: possibilities and challenges

4- Manufacturing 

• Technical pharmaceutical development: formulations, primary packaging, delivery systems
• Quality Management System, Quality by Design, and Process Analytical Technologies 
• GMPs and the department/system structures necessary for development and manufacturing 
• EMA and FDA agencies and inspections, 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 for first-in-human studies
• Accelerating the move to clinical trials (possibilities and opportunities with computer assisted modelling on the way to proof of concept)
• Strategic importance and practical organization of Phase I studies (responsibilities, planning and activities)

Learning outcomes

At the end of this module, students should be able to describe: 

1- The principles of pre-clinical strategy 

2- Requirements, ethics and regulations for pre-clinical and first-in-human studies 

3- Basic concepts of pre-clinical pharmacology and toxicology 

4- The main aspects of drug formulation and innovation in this field

5- Technical pharmaceutical 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 trial design

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

3- Planning and managing clinical trials 

• Regulatory aspects throughout product development cycle (GCP requirements) 
• Setting up a clinical trial 
• Monitoring safety in clinical trials and drug development 
• Developing Risk Management Plans – challenges 
• Risk-based and remote monitoring in clinical trials 
• Patient-centric approach: direct-to-patient trials and logistics
• Outsourcing clinical trials and managing contracts
• Audits in clinical trials: key features of audits and inspections, the keys to preparing for and effectively managing inspections, roles and expectations of key personnel, differences between inspections by different authorities

4- Gathering data from clinical trials

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

5- Blockchain in Clinical trials 

• Science-fiction or science-fact? 
• The case for blockchain applications in clinical trials 
• Barriers and challenges yet to be overcome

Learning outcomes

At the end of this module, students should be able to describe: 

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

2- The design of various types of clinical studies, along with the statistical aspects of these designs 

3- Key issues involved in conducting a clinical study in line with Good Clinical Practice (GCP) 

4- The main safety issues in clinical trials and drug development 

5- Data collection principles and tools

6- Key steps and methods in data management 

7- Blockchain applications in clinical trials 

Content

1- Clinical trial protocols and Investigator Drug Brochure (IDB)  

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

2- Biostatistics: evaluating clinical data

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

3- Evaluating safety data

• 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 clinical study reports: data and conclusions in writing
• Preparing review documents for regulatory submissions
• Writing a paper for publication
• Submitting to peer-reviewed journal, handling the reviewer’s comments

Learning outcomes

At the end of this module, students should be able to describe: 

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

2- How to evaluate and interpret clinical trial results in scientific papers

3- The main statistical methods used in clinical data analysis, and the current challenges around the p-value 

4- The main aspects of clinical study reports, and documents for regulatory submission 

Content

1- Drug Registration: European procedures and the international environment

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

2- Pharmacovigilance & medical information

• Back to basics in pharmacovigilance 
• EU regulations and guidelines in pharmacovigilance 
• Everyday pharmacovigilance
• Overview of Patient Support Programs (PSPS)
• Recent trends in Pharmacovigilance
• Medical information: communicating with healthcare practitioners 
• Summary of product characteristics and user package leaflet 

3- Pharmacoepidemiology 

• Principles of pharmacoepidemiology 
• Study methods and data resources 
• Interpreting pharmacoepidemiological data 
• Role of pharmacoepidemiology in a medicinal product’s lifecycle

Learning outcomes

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

1- General principles of medicine regulation (pre- and post- approval) 

2- Principles of pharmacovigilance and medical information

3- The role of pharmacoepidemiology in a medicinal product’s lifecycle

Content

1- Principles and practical applications of health economics 

• Health economics assessment of medicinal products: study quality concept and methods used to assess quality 
• Health technology assessment 
• Interpreting cost-effectiveness
• How the economic assessment affects market access 
• Pricing and social security coverage 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 truly innovative? 
• Health data as the basis for innovation and improving health care 

Learning outcomes

At the end of this module, students should be able to describe:

1- The principles in, and practical applications of, health economics and Health Technology Assessment within the healthcare marketplace 

2- The ethical and legal aspects of bringing a drug to market 

3- What makes a truly innovative product?

Biologics, vaccines, ATMPs, medical advice: how do they differ from conventional drugs?

1. Particularities in the clinical development of Biologics
2. Medical advice: an overview from manufacturer to patient
3. Advanced Therapy Medicinal Products (ATMPs)
4. Challenges in Vaccine development: from preclinical to clinical studies