Program

Discover detailed program for 6 modules!

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