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The path to a new medicine is long and demanding, typically requiring 10 to 12 years and an investment of approximately 600 &ndash 700 million Euros. The entire process requires an immense amount of effort with a great deal of risk. According to the Pharmaceutical Research and Manufacturers of America, for every 5,000 potential medicines tested, on average, only five are tested in patients, and only one of the five is approved for patient use.

The Medicines Discovery and Development process begins by determining the diseases for which to find a treatment and ends with the filing and launching of the New Medicine.
Technology and computer-aided techniques are used to identify and understand disease targets leads are generated and synthesised with the aim to ing the Best Candidate.
Before testing in humans, a range of preclinical tasks are undertaken to study all aspects of the candidate, evaluate pharmacokinetic and safety as well as to characterize the Active Product Ingredient (API) and design a Drug Delivery System (DDS).
Key is the First Time in Humans (Phase I). Early clinical trials and ongoing non-clinical studies continue to test and refine the drug and to prove the hypothesis about how it will act in people.
Studies proceed from healthy volunteers to patients (Phase II) suffering from the disease.
Phase III studies are conducted to fully establish efficacy while continuing to evaluate safety these are large studies that typically involve thousands of patients in centres worldwide and may require years of effort.
Industrialization meanwhile is taking place to make available the New Medicine for the launch and market.
During the course the different stages of the Medicines Discovery and Development both pre-clinical and clinical process &ndash from early research to launching a new product in markets will be presented. (see the below course outline).
For each stage critical aspects will be discussed in depth with cases studies to exemplify how specific issues has been tackled and solved and enabling technologies applied

Creating medicines

Programma

 


 

Course outline

1. Determining which Disease to Investigate - The R&D process begins by determining the diseases for which we wish to find a treatment (disease ion). Then, in order to arrive at a new medicine, we must identify potential drug targets in the body (target ion).
&bull Disease ion Disease Areas Family ion
2. Identifying Genes Associated with Disease - Technology and computer-aided techniques are used to identify and understand disease targets.
&bull Genetic Technologies Gene Sequencing SNP Mapping Pharmacogenetics

3. Finding Potential Leads - Once a target has been identified, chemists synthesise compounds which interact with the target. This is called lead generation.

There are three approaches to lead generation:

1. Modify the structure of a known drug
2. Randomly screen an already existing store of compounds against a target of interest
3. Synthesise novel compounds based on knowledge of which chemical structures will most likely interact with the target of interest.
&bull High Throughput Lead Optimisation

4. ing the Best Candidate - The lead candidate ed for further development must possess the characteristics required for clinical and commercial success.

&bull ion Criteria - The multi-disciplinary teams that make candidate ions must answer numerous questions, such as:

Which compounds are likely to be most effective?
a.What are the projected dose range and easiest method of delivery?
b.Which compounds are easy to manufacture on a large scale?
c.What return on investment will be generated: Can the compound meet
or surpass the market standards?

&bull Tests Modifying the Lead Scaled-up Synthesis
5. Studying All Aspects of the Candidate - Before testing a new medicine in humans, a range of preclinical tasks is undertaken.
&bull Preclinical Tasks Further Studies Chemistry Pharmaceutical Development

6. Special topics - Biopharmaceutics of Oral Drug Delivery - Key drivers and challenges. (GI tract physiology, physical-chemical properties of the API, delivery systems and role of in vitro dissolution tools) In silico and in-vitro/in-vivo prediction. Understanding cause of attrition


7. Testing for the First Time in Humans - Early clinical trials and ongoing non-clinical studies continue to test and refine the drug and to prove the hypothesis about how it will act in people.
&bull Phase I Trials Proof of Concept

8. Progressing to Final Testing - Studies proceed from healthy volunteers to patients suffering from the disease.
&bull Phase II Trials

9. Conducting Final Testing - Phase III studies are large studies that typically involve thousands of patients in centres worldwide and may require years of effort. It is these studies that fully establish efficacy while continuing to evaluate safety.
&bull Larger-Scale Testing Phase III trials Preparing for Registration Making the Product Preparing the Product

10. Filing and Launching the New Medicine - A dossier is submitted to the regulatory authorities, asking for approval to market the new drug. Once approval is granted, the drug is launched.
&bull File Preparing for Market Launch

11. Managing a Medicine's Lifecycle - After a new medicine is launched, safety monitoring continues, and further management of the product's lifecycle looks toward finding other potential uses for the drug.
&bull Post-Marketing Surveillance Uses and Value

 

 

Svolgimento

Il corso si terrà, dal 7 al 29 novembre 2014, presso l'Aula 1-17 della Sede dello IUSS (Palazzo del Broletto, Piazza della Vittoria n. 15).

Bibliografia

Le indicazioni bibliografiche saranno comunicate a inizio corso.
 

Esame

Esame - Aula 1-17:

Sabato 13 dicembre, ore 9 &ndash 12
 

Semestre: Semestre I

Anno accademico: 2014-2015