Academic Year 2020/2021

  • Moduli: Roberto Rimondini Giorgini (Modulo 1) Emanuel Raschi (Modulo 2)
  • Teaching Mode: Traditional lectures (Modulo 1) Traditional lectures (Modulo 2)
  • Campus: Bologna
  • Corso: Second cycle degree programme (LM) in Medical Biotechnology (cod. 9081)

Learning outcomes

Identify key parameters for risk/benefit assessment of drug therapies. Understand sources of variability in clinical drug responses (including pharmacogenomics). Describe the basic methodology of pre-clinical and clinical drug trials, their regulatory and ethical implications, and procedures for marketing authorization, with special reference to biotechnological drugs. Present therapeutic options (conventional and biotech drugs) in the context of a given disease, highlighting innovative aspects. Critically evaluate research articles presenting pre-clinical and clinical data, through consultation of specific databases and documentation provided online by regulatory agencies.

Course contents

INTRODUCTION TO PHARMACOLOGY

  • The drug definition. Evaluation of the efficacy and safety of a drug; use of surrogate endpoints and clinical endpoints; adverse drug reactions; elements for a critical assessment of the risk / benefit ratio of a drug.
  • Concept of innovative drug. Biotech medicines: classification, pharmacokinetic characteristics, pharmacodynamic and toxicological.
  • Classification and sources of information related to drugs.

    PHARMACOGENOMICS

  • The variability factors in drug response in individual and in a population.
  • Definition of pharmacogenetics and pharmacogenomics. Examples of applications of pharmacogenetic tests. Genetic models for the development of new drugs.

    PRECLINICAL DRUG TESTING

  • Definition of in vitro experimentation, ex vivo, in vivo.
  • Definition, use, development and ethics of animal models for preclinical testing
  • Concept of 3R and outline of the three-dimensional cell culture models for the replacement of the animal studies

THE CLINICAL EVIDENCE OF DRUGS

  • The phases of clinical development; the concept of randomized controlled: the hypothesis, the primary objective of defining and secondary objectives, control of variability factors, randomization and blind, placebo; the good clinical practice; limitations of clinical trials of medicines; ethical aspects of the clinical trial; the role of ethics committees.
  • The concept of real world evidence and the role of pharmacovigilance
  • Principles of risk-benefit assessment: bias, number needed to treat/harm, the role of systematic review and meta-analysis

    PHARMACEUTICAL LEGISLATION

  • The authorization for the clinical trial: molecules of the new institution (the concept of first-in-class drug) and not to the new institution (drug repurposing).
  • The marketing authorization; centralized procedure and mutual recognition (decentralized): EU directives, with particular reference to biotechnology and new drug medications; European Agency's role and the Italian Drug Agency.
  • Orphan drugs.

    PHARMACOECOMIC PRINCIPLES

  • The consumption of drugs: ATC classification and concept of Defined Daily Dose (DDD).
  • The cost/benefit ratio in drug therapies; types of economic evaluation (cost-minimization analysis, cost-benefit, cost-effectiveness, cost-utility); managed entry agreements (e.g., payment by results).

Readings/Bibliography

It is not necessary to purchase specific texts. Updated teaching materials will be provided in class.

Teaching methods

Plenary lessons and interactive seminars.

Assessment methods

The assessment test will be an oral presentation (15 min, PowerPoint  presentation) on a drug chosen by the candidate. During the presentation,student should be able to demonstrate that she/he has acquired the ability to critically analyze the chosen topic in at least four of the following areas:

- Use of experimental models (n vivo, ex vivo and in vitro) for the study of drugs

- Regulatory aspects relating to the preclinical and clinical testing of medicines (with particular reference to biotechnological medicines and advanced therapy medicines) and to the procedures necessary to obtain marketing authorization;

- Evaluation of the risk / benefit ratio of drug therapies by identifying the correct sources of documentation;

- Evaluation of the cost / benefit ratio of drug therapies, identifying adequate sources of information;

- Identification of the variability factors implicated in the clinical response to drugs (including pharmacogenomics);

- Identification of the innovative aspects of a medicine within a given therapeutic indication;

- Identification of ethical issues related to preclinical and clinical trial projects for medicines.

The presentation will receive a score for each of the following aspects:

  • clarity and style of the presentation: up to 10 points
  • completeness of coverage of the four selected items (taking into account the time limit indicated above): up to 10 points
  • critical assessment by the student of the selected sources of information: up to 10 points.

The final mark will be determined by the sum of the three scores indicated above.


The mark obtained in this test contributes to the formation of the final mark of the Innovative Medical Therapies exam in proportion to the credits assigned to the Pharmacology module.

Teaching tools

Teaching material (access restricted to UNIBO student) can be downloaded through IOL.

Office hours

See the website of Roberto Rimondini Giorgini

See the website of Emanuel Raschi