7 januariIntroduction to Clinical Trials

Question 1

What are ophan diseases?

Answer 1

Very little people having a disease, how do you get companies interested in developing a cure for this disease?

Question 2

Efficacy =

Answer 2

does it do anything? (e.g. does it reduce the headache, stops the growth of tumors)

Question 3

Effectiveness =

Answer 3

how well does it work? it may do something, but not very well (e.g. the growth of tumor is halted (=efficacy), but no improvement of disease state (=no effectiveness), headache diminished to 45% is not real effective).

Question 4

Efficiency =

Answer 4

does it work soon, well (e.g. headache reduces headache after an hour, not real efficient)

Question 5

Name and describe the 3 types of trials, including the latest type used mostly in the EU. Give example of each one.

Answer 5

Intervention trials: subject undergoes procedures other than usual practices

Observational trials: following usual practice, anonymously (e.g. when a product is already on the market, not homologous to phase 4 CT)

Low interventional trial: (new type following new European law), amount of regulation can be reduced, similar to phase 4, the product already has market authorization, labelled use, minimal risks to the subjects. They fall within non-industry trials (e.g. in academia, physicians that want to compare treatments, to verify work of industry). e.g. different method of distribution

Question 6

Describe how you would acquire MAA (when is your CT successful?)

Answer 6

compare with best proven product ‘golden standard’ and out-compete it. The golden standard is not per se the most used or, the same in every country because (1) cost effectiveness (2) reimbursement is not the same in each country, each drug.

Question 7

Which parties are involved in MAA for CTs?

(1, 2, 3, 4a/b, 5a/b/c/d/e) and name their responsibilities.

Answer 7

1. Sponsor
2. investigator
3. Institutional review Board (USA)/ Independent Ethics Committee (IRB/IEC (EU)
4. Regulatory Authorities/ Health Authorities
   4a. EMA / FDA
   4b. CA for each country
5. Vendors
   5a. CRO
   5b. SMO
   5c. Central Laboratory
   5d. IVRS Vendor
   5e. Central ECG reading

Question 8

Describe the three phases of CTs

Answer 8

Trial prep:

• development/collection of essential documents
• site collection
• obtaining IEC/CA approval
• site initiation
• report writing

Trial execution

• site management activities
• Routines monitoring visitis
• update essential docs
• report writing
• query resolution
• close-out visits

DATABASE LOCK

Trial, analysis & reporting

• adminstrations
• filing
• updating databases
• clinical study report

Question 9

Name the sponsor’s tasks (R&D and non-R&D)

Answer 9

Sponsor is any company that is responsible for setting up, initiating and financing a trial.

R&D:
 Monitoring/ Site management
 Data management; sets up clinical trials (responsible for collection of information)  Statistics
 Medical writing
 Drug safety/ Pharmacovigilance
 Quality assurance; assuring that the performance is accurately working
 Regulatory affairs.

Non- R&D
 Sales & Marketing
 Medical Department (head: medical director:
 Supporting sales
 Supporting R&D
 Financial department

Question 10

explain the differences between confirmatory and exploratory trial

Answer 10

exploratory trial = the outcome could lead to a different question, e.g. new product could be effective in oncology, trials on multiple tumors.
confirmatory trial = confirming the hypothesis

Question 11

Why would companies get involved in developing treatments for orphan diseases?

Answer 11

1) societal pressure

2) subsidized by governmental instances