69888 - Pharmacology

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

• Definition of drugs and medicinal products, with a focus on advanced therapy medicinal products (ATMPs). Evaluation of the efficacy and safety of a drug; adverse drug reactions
• Concept of General Pharmacology: Pharmacokinetics and pharmacodynamics
• Concept of innovative drug. Biotech medicines: classification, pharmacokinetic characteristics, pharmacodynamic and toxicological.

PHARMACOGENOMICS

• The variability factors in drug response in individual and in a population.
• Definition of pharmacogenetics and pharmacogenomics. Examples of applications of pharmacogenetic tests.

PRECLINICAL DRUG TESTING

• Basic statistical analysis in preclinical study: mean, median, distribution (parametrical and non-parametrical), t-test, one- and two-ANOVA, MANOVA, non-parametrical analysis
• Definition of in vitro experimentation, ex vivo, in vivo.
• Definition, use, development and ethics of animal models for preclinical testing. Genetic models for the development of new drugs.
• Concept of 3R and outline of the three-dimensional cell culture models for the replacement of the animal studies

THE CLINICAL EVIDENCE OF DRUGS

• Classification and sources of information related to drugs.
• The phases of clinical development; the concept of randomized controlled trial: scientific aspects; the use of surrogate and clinical endpoints, ethical aspects of placebo;
• The concept of real-world evidence and the role of pharmacovigilance
• Principles of benefit-risk and cost-benefit assessment: bias, number needed to treat/harm, types of pharmacoeconomic studies; managed entry agreements. The role of systematic review and meta-analysis.
• Critical analysis of the innovation of biotechnological products.

REGULATORY ASPECTS

• The authorization for a clinical trial: molecules of the new institution (the concept of first-in-class drug) and not to the new institution (drug repurposing). The role of ethics committees.
• The marketing authorization; centralized procedure and mutual recognition (decentralized): EU directives, with particular reference to biotechnological products; Roles of the European Medicines Agency (EMA) and the Italian Drug Agency (AIFA).
• Orphan drugs.

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.

Gradation of the final grade

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