10 - drug discovery 2 Flashcards

1
Q

What LDL level would a patient with a PCKS9 gene mutation have?

A

<130 mg/dL

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2
Q

What is the normal MOA of LDL-C regulation?

A
  • PCSK9 is secreted
  • PCKS9 binds to a LDL-R
  • LDL particle binds to a PCSK9 bound LDL-R
  • LDL is brought into the cell for lysosomal degradation
  • LDL-R recycling is blocked
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3
Q

What is the MOA of LDL-C regulation when a human monoclonal antibody against PCSK9 is introduced?

A
  • The antibody binds to the PCSK9, which stops PCSK9 from binding to LDL-R
  • LDL-R concentration is increased
  • Without a bound LDL, the LDL-R is recycled
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4
Q

Are PCSK9i considered a blockbuster drug at this moment?

A

No

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5
Q

What is ICER?

A

Incremental cost effectiveness ratio

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6
Q

What is QALY?

A

Quality adjusted life year

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7
Q

How is ICER calculated?

A

ICER = (Cost2-cost1)/(QALY2-QALY1)

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8
Q

How many estimated US adults might be eligible for PCSK9i if the cost is low enough?

A

Nearly 10 million

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