00351 - Pharmacology

Learning outcomes

At the end of the course the student: - knows the main parameters for the definition of the risk / benefit ratio of drug therapies; - He learned concepts useful for understanding the variability of factors involved in clinical drug response (including pharmacogenomics); - He learned basic notions on the methodology of clinical trials of drugs (with special reference to biotechnology drugs); - Know the main rules governing clinical trials in the European Union (with particular reference to advanced therapy medicinal products), as well as the procedures required to obtain the marketing authorization for a medicinal product for human use. - As part of a clinical trial project of medicines (with particular reference to biotechnology medicinal products), - is able to be able to recognize the relevant regulatory and ethical implications, as well as the elements of innovation of a draft of a medicinal product; of knowing how to identify the elements to come to a critical assessment of the risk / benefit ratio and the cost / benefit of drug therapies. Provide, at least in broad terms, a risk / benefit assessment of a drug therapy. - In the context of a given disease, you know how to present the different therapeutic options (conventional and biotech drugs), highlighting the innovative elements. - It 'can make updates on legislation concerning advanced therapy medicinal products, even using the documentation made available on the websites of regulatory agencies and through consultation and the study of literature sources and specific databases.

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 TRIALS OF DRUGS

• The phases of clinical development; controlled clinical trial: 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.

  PHARMACEUTICAL LEGISLATION

• The authorization for the clinical trial: molecules of the new institution and not to the new institution.
• 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 consumption of drugs by therapeutic class; the containment policies of public pharmaceutical expenditure.
• The cost / benefit ratio in drug therapies; types of economic evaluation (cost-minimization analysis, cost-benefit, cost-effectiveness, cost-utility).

Readings/Bibliography

No books are needed. Teaching material will be distribuited during the course.

Teaching methods

Plenary lecture

Assessment methods

The final examination consists of an oral dissertation on pharmacological topic, choosen in advance, in which the student has to proves that he has acquired the ability to critically analyze the topic selected in at least four of the following areas:
- Use and ethics of animal models to study in vivo, ex vivo and in vitro to study the mechanism of action of new drugs
- Regulatory aspects related to preclinical and clinical trials of medicines (with particular reference to biotechnology medicinal products and advanced therapy medicinal products) and the procedures required to obtain the marketing authorization;
- Evaluation of the risk / benefit ratio of drug therapies by identifying the correct documentation sources;
- Assessment of the cost / benefit ratio of drug therapies, identifying appropriate sources of information;
- Identification of factors involved in clinical variability in drug response (including pharmacogenomics);
- Identification of the innovative aspects of a medicinal product in the context of a given therapeutic indication;

- Identification of the ethical issues relating to preclinical and clinical trials of medicinal products projects.
The grade obtained in this test contributes to the formation of the final grade examination of Innovative Medical Therapies in proportion to the credits assigned to the Pharmacology module.

Teaching tools

Students could download teaching material from AMS CAMPUS web site.

Office hours

See the website of Roberto Rimondini Giorgini

See the website of Emanuel Raschi