Protein Replacement

Question 1

What is an orphan disease?

Answer 1

An orphan disease is a disease with a prevalence of less than 200,000 affected individuals in the US and 250,000 in the EU.

Question 2

What is the importance of the Orphan Disease Regulatory Act of 1983?

Answer 2

It provides regulatory, tax and commercial incentives to companies developing drugs for orphan diseases.

Question 3

Which diseases have the most approval as orphan diseases?

Answer 3

Pedriatic indications
Rate types of cancer
Genetic diseases

Question 4

What are monogenic diseases?

Answer 4

Blood factors and enzyme replacement therapies for lysosomal storage diseases are examples of monogenic protein replacement diseases.

Question 5

According to the article, how many clinical trials enter by 2011?
How many finished phase 2?
How many finished phase 3?

Answer 5

N= 144
75%
50%

Question 6

What are the chances of approval for a MPRT according to the new analysis?

Answer 6

85% for LSD

15% for metabolic disorders

Question 7

How is MPRT so successful?

Answer 7

The pathogens sis is very well understood.

Question 8

What is the approval time for MPRT?

Answer 8

8.3 years

Question 9

Why are these results important?

Answer 9

These results show that it is possible to increase the numbers in the probability for success which is required to assure a net reduction of the healthcare cost and justify reimbursement.