ODD

Question 1

Orphan disease is

Answer 1

Rare disease

Question 2

Orphan medicinal product?

Answer 2

Drug product specifically designed, developed and tested for patients suffering from a rare disease

Question 3

Commission regulation (EC) 847/2000

Answer 3

laying down the provisions for implementation of the criterial for designation of a medicla product as an orphan medicinal product and definitions of the concepts similar medicinal product and clinical superiority

Question 4

COMP?

Answer 4

Committee for ophan Medicinal products

Question 5

COMP resonsibilities?

Answer 5

evaluation application for prhan designation

Question 6

a disease is defined as rare in EU if

Answer 6

it affects fewer than 5 in 10,000 people across the EU

Question 7

what are further purpose for the estabilshment?

Answer 7

that it is without incentives it is unlikely that the marketing of the MP in the community would generate sufficent retur to justify the necesary investment

Question 8

further establishment

Answer 8

there exists no satisfactory method of diagonis, prevention or treatment of the condition in question or if such method exist that the MP will be of significat benefit to thoes affected bythe condition

Question 9

Prevalence:

Answer 9

Prevalencein epidemiologyis the proportion of a particular population found to be affected by a medical condition

Question 10

Incidence

Answer 10

Incidencein epidemiology is a measure of the probabilityof occurrence of a given medical condition in a population within a specified period of time

Question 11

Incidence proportion(

Answer 11

also known as cumulative incidence) is the number of new cases within a specified time period divided by the size of the population initially at risk.

Question 12

The difference between prevalence and incidence can be summarized thus:

Answer 12

Prevalence answers
“How many people have this disease right now?” or
“How many people have had this disease during this time period?”

Incidence answers
“How many people per year newly acquire this disease?”

Question 13

what is the EU incentives:

Answer 13

Market exclusivity (10 years MA)
Protocol assistance (free scientific advice
EU-Funded research
Contralized procedure
Fee reductions

Question 14

US incentives?

Answer 14

7 years market exclusivity
tay credits total ling half of development costs
fast-track development
waived drug application fees
orphan drug status granted through the FDA
non-economic advantges