H e a lth h T e ch h n n o o l lo o g g y y A A - - PowerPoint PPT Presentation

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H e a lth h T e ch h n n o o l lo o g g y y A A - - PowerPoint PPT Presentation

H e a lth h T e ch h n n o o l lo o g g y y A A sse ssm e n n t t f fo o r r R a re D ise a se : L L e e sso o n n s s L L e e a rn n e e d d f fro o m m T h h a a ila n n d d


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SLIDE 1

H e a lth h T e ch n

  • l

h n

  • lo

g y

  • g

y A A sse ssm e n t n t f fo r

  • r R

a re D ise a se : L e L e sso n s

  • n

s L e L e a rn e n e d f d fro m

  • m

T h a h a ila n d n d

P a tta ra L e e la h a v a ro n g , P h D P ro g ra m L e a d e r o f H e a lth In te rv e n tio n a n d T e c h n

  • lo

g y A s s e s s m e n t P ro g ra m (H IT A P )

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SLIDE 2

2

R a r a re d e d ise a s e a se e te r e rm in

  • lo

g y a n a n d p re v e va l a le n e n ce e th re s e sh

  • ld

: R e v e vie w e w l lite r e ra t a tu re

R ic h te r T , e t a

  • l. R

a red is e a s e te rm in

  • lo

g y a n d d e fin itio n s — a s y s te m a tic g lo b a l re v ie w : re p

  • rt o

f th eIS P O R ra re d is e a s e s p e c ia l in te re s t g ro u p . V a lu e H e a lth . 2 1 5 ;1 8 (6 ):9 6 – 1 4

Average 40 cases/100,000 people

T h a h a ila n d : n d : 1 5 1 5

(T

  • ta

l a l # < # < 1 , 1 ,0 ca ca se se s) s)

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SLIDE 3

U sin g H n g H T A fo r

  • r c

co v

  • ve

ra g e g e d e d e cisio n

  • n

T

  • n
  • n

T h a h a ila n d n d

2 . P P ri rio ri rity se ttin g

  • f

n g

  • f

to p i

  • p

ics

3 . H T H T A

4 . M M a kin g n g co ve r e ra g a g e e d e d e cisio n s

  • n

s

3

S ta k a ke h e h

  • ld

e r e r e n e n g a g a g e m e m e n e n t

1 . T T

  • p

i

  • p

ic se l e le c e ctio n

W

  • r
  • rkin

g n g G ro u p s

  • u

p s o f

  • f M

e d i d ica l e e xp e p e rt W

  • r
  • rkin

g n g G ro u p s

  • u

p s o f

  • f T
  • p

i

  • p

ic se le ctio n

  • n

N

  • N
  • n
  • m

e d e d ica l a l b b e n e n e f e fit p a c a cka g a g e

  • N

e w T e ch n

  • l

h n

  • lo

g y

  • g

y a a n d n d in t n te rve n t n tio n s

  • n

s

  • E

ffe c e ctive e C

  • ve

r e ra g a g e f e fo r e x e xistin g p a c a cka g a g e s T e c e ch n ica l a l H H e a l e a lth E co n

  • m
  • n
  • m

ics W W

  • r
  • rkin

g n g G ro u p

  • u

p M e d e d ica l a l b b e n e n e f e fit p a c a cka g a g e

  • N

e w e w m m e d e d icin e s e s S u b c u b co m

  • m

m itte e

  • f
  • f

N a tio n a

  • n

a l L i L ist o f

  • f

E sse n tia ia l M l M e d ic icin in e S u b c u b co m

  • m

m itte e

  • f
  • f

U n ive r e rsa l a l C C

  • ve

r e ra g a g e e S ch e m e m e s e s B B e n e n e f e fit P a c a cka g a g e

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SLIDE 4

4

S t S te p 1 1 S ta ke h

  • l

h

  • ld

e d e r E n g a n g a g e g e m e n t n t f fo r

  • r T
  • p

i

  • p

ic s se le ctio n

  • n
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SLIDE 5

C rite r e ria a fo r d d e v e ve l e lo p m e n e n t n

  • n

n

  • n
  • m

e d e d ica l a lre i e im b u rse m e m e n e n t p p a c a cka g a g e

5

T

  • p

i

  • p

ic S e le ctio n

  • n

1 . 1 .B u r u rd e d e n

  • f

n

  • f d

i d ise a se 2 . 2 .S e v e ve r e rity o f d d ise a s e a se e  3 . 3 .E ffica c a cy a n a n d /o r e f e ffe c e ctive n e n e s e ss 4 . 4 .D iffe r e re n e n ce a m e a m

  • n

g h e a l e a lth b b e n e n e f e fit sch e m e m e s e s 5 . 5 .C a ta stro p h i

  • p

h ic i illn e n e ss a a n d n d h

  • u

s h

  • u

se h

  • l

h

  • ld

d e co n

  • m
  • n
  • m

ic i im p a p a ct  6 . 6 .S

  • c
  • cia

l a a n d n d E q u i q u ity c co n s

  • n

sid e d e ra tio n

  • n

T

  • p

i

  • p

ic S e le ctio n

  • n

S t S te p 2

1 . 1 .B u r u rd e d e n

  • f

n

  • f d

i d ise a se 2 . 2 .S e v e ve r e rity o f d d ise a s e a se e  3 . 3 .S a f a fe t e ty a n a n d E E ffica c a cy  4 . 4 .C lin lin ic ica l p l p ra ctic ice g g u id id e lin lin e 

  • 5. B

u d g e u d g e t I Im p a p a ct  6 .O th e h e r s su p p

  • r

u p p

  • rtin

g n g e vid e d e n c n ce e e .g . g . co co

  • d

e d e p e p e n d e n d e n c n ce t te ch n

  • l

h n

  • lo

g y

  • g

y a a n d n d fe a sib ib ilit ility C rite r e ria a fo r d d e v e ve l e lo p m e n e n t m e d e d ica l a lre i e im b u rse m e m e n e n t p p a c a cka g a g e

S t S te p 2

 R a r a re d e d ise a s e a se * e * to p i

  • p

ics ( (m e d i d ica l a n d n

  • n

n d n

  • n
  • m

e d e d ica l a l) w e r e re n e n

  • m

in a t a te d e d fro m m m e d ica l e xp e p e rt g r g ro u p

  • u

p

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SLIDE 6

Clinical: Efficacy, Safety, Effectiveness Economic: Efficiency, Costs, Cost- effectiveness Patient- related: Social impact, Ethics, Acceptability Organisationa l: Feasibility, Utilisation, Assessibility

6

HTA

Source: Draborg et al. International comparison of the definition and the practical application of health technology assessment Int J Technol Assess Health Care 2005 Winter; 21(1):89-95.

S t S te p 3 3 H T H T A

 R a r a re d e d ise a s e a se e to p i

  • p

ics ( (m e d i d ica l a n d n d n

  • n

n

  • n
  • m

e d e d ica l a l) w w e r e re e se l e le c e cte d e d f fo r H T A

1. Medical: Imiglucerase for Gaucher disease type I 2. Non-medical: Neonatal Screening for Inborn Errors of Metabolism Using Tandem Mass Spectrometry

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SLIDE 7

C rite r e ria a fo r d d e v e ve l e lo p m e n e n t n

  • n

n

  • n
  • m

e d e d ica l a lre i e im b u rse m e m e n e n t p p a c a cka g a g e

7

1 . 1 .C

  • C
  • st-e

f e ffe c e ctive n e n e s e ss 2 . 2 .C lin lin ic ica l p l p ra ctic ice g g u id id e lin lin e 3 . 3 .F e a s e a sib ility a n a n d p p re p e p a r a re d e d n e s e ss o f h e a l e a lth s se r e rvice s e s 4 . 4 .A ffo r

  • rd

a d a b i b ility o f

  • f p

u b l p u b lic h e h e a lth i h in s n su r u ra n c n ce 5 . 5 .S

  • cia

l a l a n a n d e t e th ica l a l i issu e s e s 6 . 6 .O th e h e r co n s

  • n

sid e d e ra tio n e

  • n

e .g . m m e d e d ica l a l r re i e im b u rse m e m e n e n t p a c a cka g a g e

M a kin g d e n g d e cisio n

  • n

1 . 1 .C

  • C
  • st-e

f e ffe c e ctive n e n e s e ss a n a n d p rice e n e g e g

  • tia

t a tio n 2 . 2 .C lin lin ic ica l p l p ra ctic ice g g u id id e lin lin e 3 . 3 .F e a s e a sib ility a n a n d p p re p e p a r a re d e d n e s e ss o f h e a l e a lth s se r e rvice s e s 4 . 4 .A ffo r

  • rd

a d a b i b ility o f

  • f p

u b l p u b lic h e h e a lth i h in s n su r u ra n c n ce 5 . 5 .S

  • cia

l a l a n a n d e t e th ica l a l i issu e s e s 6 . 6 .O th e h e r co n s

  • n

sid e d e ra tio n e

  • n

e .g . n

  • n

n

  • n
  • m

e d e d ica l a l re i e im b u rse m e m e n e n t p p a c a cka g a g e

M a kin g d e n g d e cisio n

  • n

C rite r e ria a fo r d d e v e ve l e lo p m e n e n t m e d e d ica l a lre i e im b u rse m e m e n e n t p p a c a cka g a g e

S t S te p 4 S t S te p 4

 B

  • n

e

  • n

e m m a rro w

  • w

t tra n s n sp l p la n t n ta tio n

  • n

: cu ra t a tive e tre a t e a tm e n e n t fo r G a u a u ch e r e r T T yp e I e I  Im ig lu ce r e ra s a seb e b e fo r

  • re

u n d e u n d e rg

  • i
  • in

g B

  • n

e

  • n

e m a rro w

  • w

t tra n s n sp l p la n t n ta tio n

  • n
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SLIDE 8

C

  • ve

r e ra g a g e d e d e c e cisio n f fo r r ra r a re d e d ise a s e a se

Imig iglu lucerase for G Gaucher er t type e 1

It was s not

  • t cos
  • st-ef

effec ective e due ue to very hi high h cost t tr treatm tment t (ICER 197, 197,000 000 USD/ QALY , Y , thr hresho hold 5, 5,000 000 USD/ QALY LY). The drug is is lif life-sa saving, and it is s need eeded ed for

  • r bone

e mar arrow tran ansplan ant (curat ative e treat eatmen ent cover ered ed by UHC). Equity co conce cern : if if this is medic icin ine is is not inc nclud uded in n the he list but ut the he bone ne marrow tran ansplan ant is in the e ben enef efit pac ackag age e under er

  • UCS. This mean

eans only the e rich can an ac acces ess the e treat eatmen ent as as they ey ar are e ab able e to pay ay for im imig iglu lucerase (around und 120, 120,00 00 USD/ year ear) Inc nclus usion n in n the he NLEM after price neg egotiat ation

Sources: Teerawattananon Y et al. The use of economic evaluation for guiding the pharmaceutical reimbursement list in Thailand. Zeitschrift für Evidenz, Fortbildung und Qualität im Gesundheitswesen, 2014,108.7:397-404.

Budget 411,000 USD Budget 2 million USD *Only 2 patients could undergo bone marrow transplantation

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SLIDE 9

In p ro c e s s : T

  • p

ic n

  • m

in a te d

N

  • N
  • .

T e ch n

  • l

h n

  • lo

g y

  • g

y In d i n d ica tio n

  • n

E stim a te d d n u m n u m b e b e r o f

  • f p

a p a tie n t n t 1 A g a l a lsid a s a seb e t e ta F a F a b ryd ise a e s e a e s

4

2 A lg l g lu c u co s

  • sid

a d a sea l a lfa P

  • m
  • m

p e p ed ise a s e a se

6

3 Id u r d u rsu l u lfa se M u c u co p

  • l
  • p
  • lysa

cch a h a rid

  • s

d

  • sis t

typ e p e II (H u n t u n te r syn d r n d ro m

  • m

e )

6

4 L a L a ro n i

  • n

id a d a se M u c u co p

  • l
  • p
  • lysa

cch a h a rid

  • s

d

  • sis t

typ e p e I H u r u rle r (M S M S P

  • IH

) H ) & H u H u rle r-S ch e ie(M S M S P

  • IH

S H S )

6

5 N e

  • e
  • n

a t a ta l a l S S cre e n e e n in g U sin g n g T a n d e n d e m M M a ss S p e c e ctro m e t e try 3 3 in d i n d ica tio n s

  • n

s o f

  • f In

b

  • r

n b

  • rn

E n E rro r

  • rs o

f

  • f

M e t e ta b a b

  • lism

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SLIDE 10

C h a l a lle n e n g e s e s o f H H T A fo r r ra r a re e d ise a s e a se

  • E

stim a te

  • f
  • f t

to t

  • ta

l n u m n u m b e b e r o f

  • f r

ra re d i d ise a se p a p a tie n t n ts a n d b u d g e n d b u d g e t im p a p a ct t th a h a t sh

  • u

l h

  • u

ld d b e b e ca l a lcu la t a te d e d fro m a l a ll re l e le v e va n a n t t te c e ch n

  • lo

g ie s e s (co co

  • d

e d e p e p e n d e n d e n t te ch n

  • l

h n

  • lo

g y

  • g

y)

  • C

lin i n ica l a n d n d

  • t
  • th

e h e r e vid e d e n c n ce n e n e e d e d e d d fo r

  • r H

T A e .g . g . e ffica cy, co s

  • st, h

e h e a lth h q u a q u a lity o f

  • f life
  • U

n c n ce rta in t n ty o f

  • f re

su l u lt

  • F

e a s e a sib ility a n a n d p re p e p a r a re d e d n e s e ss o f h h e a l e a lth s se r e rvice s e s e . e .g . m e d e d ica l a l s sp e c e cia l a list, r re g e g istry syst syste m , p a ym ym e n t m e ch ch a n ism sm

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