chi fda report-embargoed
TRANSCRIPT
-
7/31/2019 CHI FDA Report-Embargoed
1/12
MANAGING PRIORITIES:THERAPEUTIC AREA VARIATION IN
FDA DRUG REGULATION
MAY 2012
Bkgd: FDA Dg Rg Pm ....................................................
Tp A V ......................................................................................
T Imp Rb D ..............................................................................
Appdx .................................................................................................................
1
2
8
10
-
7/31/2019 CHI FDA Report-Embargoed
2/12
chi-caliornia healthcare institute 1
may 2012
p w bjv. T
pvd pd pm
U.S. Fd d Dg Adm
(FDA) w p ppv
dg d bg. T bd dy, Competitiveness and Regulation: Te FDA and
the Future o Americas Biomedical Industry (2011),
dd w B Cg Gp
(BCG). O d bjv p w
d mp d FDA
pm m p .
Ogy, , g py d
Agy d v pd
ppv dg. Idd, FDAd v ggd ppv
gy pd vd mmm
bmd v. B
w d bd by d db
gd xmp w gy dd
m d Agy pm
ggd. A Cg z Pp
Dg U F A (PDUFA), w gg
dp mg p my,
, p ppy FDA: my,
p pp d p g-pmg g Agy.
BACKGROUND: FDA DRUG
REGULATION PERFORMANCE
O ky dg pbd
Competitiveness and Regulation w mkd
m ppv dg, bgg
FY 2007 d g y p PDUFA IV y, w d FY 2008. F
xmp, Fg 1 w dg bmd
FDA 2008, d PDUFA IV, xpd
vg ppv m 18.9 m, 28
p v vg PDUFA III m
14.7 m.
T
FIGURE 1.
-
7/31/2019 CHI FDA Report-Embargoed
3/12
2 chi-caliornia healthcare institute
may 2012
m mp g pd
ppv pp v b py
vw, w d 45 p dg
ppvd Am, b j 5 p
d by Ep Md Agy
(EMA). T FDA ppvd py vw dg
vg 186 dy, w dd vw
m dg m pd vgd 424 dy.1
THERAPE UTIC AREA VARIATIONS
I 2011, vd FDA bg
y mpvg dg ppv
m. I Ob, Agy pbdFY 2011 Innovative Drug Approvals, w
dmd 35 vv dg d
FY2011, dg dv g p C,
1 Wg d N MA, Nw Dg
Appv ICH C: 2002-2011, C
Iv Rgy S (Ap 2012), pp. 1, 6.
I 2009, vg dg ppv m mgy
mpvd m PDUFA III pd (by 1.3
p) 14.5 m. A m m, g,
FDA d m PDUFA qm
mm gy d w x
m py pp d 10 m
dd pp. Lkg w dg
bmd Agy 2008, Fg 2 w
ppv m gg g; 45 p
bm md p m
y b Agy k gy .
Hwv, y bd vw w
dg bm g FY 2011 gg
mpvm ppv m (Fg 3).
R d pbd by C Iv
Rgy S Ud Kgdm
wd md ppv m dg
ppvd U.S. w
Ep d Jp. I pd 2002-2011,
FIGURE 2.
-
7/31/2019 CHI FDA Report-Embargoed
4/12
chi-caliornia healthcare institute 3
may 2012
FIGURE 3.
p, pm, d v d d
p d. Adg p, b
dg w ppvd b g d PDUFA, d mjy
vw y. T Agy mpd
by g xpdd ppv pwy
, my , md ,
wg m, w d. I
m v, y, Fd C
R pbd d g g Agy
pm gy dg, g m
v gy pd v b ppvd
U.S. d Ep.2
2 U.S. Fd d Dg Adm, FY 2011
Innovative Drug Approvals, Nvmb, 2011, pp. 4-8;
Sm A. Rb, J D. A, E V. Sg, Dp
Cm FDA Rvw P, Nw C Dg
R P S Ud S, Health Afairs,
J 2011.
T mp dg. Ad mg
p , gy, d
d p d bg p. B y FDA d gg m d
p. T FDA m;
g dv vw m, dpdg
pd p . A d Fg
4, bw 2000 d 2011, gy d -
v dg, xmp, xpd
vw, d 10-15 m. F
g dv, v ym,
g-, py, . vg vw
m d m 20 30 m.
N g xp d
pm mg p . Sm d
my b y m mpd, w w
bmk w py dd mm
v dg. Dg gg dd
-
7/31/2019 CHI FDA Report-Embargoed
5/12
4 chi-caliornia healthcare institute
may 2012
FIGURE 4.
v ym (CNS), xmp,v d py g ppv m. W
dd k Azm, Pk d
mp mp wdpd g,
mpx bgy md m y
v g dg dvpm. T
gy p m m mpxy.
Dg 15-y pd pg 1996 2010,
vg C Sdy
Dg Dvpm d m
p p ppv m CNS dg w 32m g dg. F CNS
dg mp p k
102.2 m, b 40 p g -
CNS dg. Mw, ppv m CNS
dg vgd 20.3 m, 13 p g
-CNS p. Cmmg
d, pp vg Jp A.DM d, Ag I xpd CNS dg
dvpm d ppv m w g
vg, I w mw pd x
w y w g, ddg, I d
pg -CNS dg w w-d-
m m ky v py vw
g m FDA w CNS dg.3
Idy, gzg FDA m
p d dd p q, wd p b gm
bw g d pb . Y,
m dg, g v g b.
3 C Sdy Dg Dvpm,
Dg CNS D k 35% Lg Dvp
O Dg, M 6, 2012.
-
7/31/2019 CHI FDA Report-Embargoed
6/12
chi-caliornia healthcare institute 5
may 2012
Byd CNS dd, db, by, d
dv d x m, d gwg,
dmg . Rgy pwy
, wv, g w y.
Ad w g pmm dvpg pd
d, w v dg p
b dppd.4
W dy d v mmy
k, mm wdm Agy
, , gd b-k
dd, dmdg m d v g
pd. A S CEO d m m
Pm R d M
Am (PRMA) Cp Vb
xpd, Y g pmy
d bmg mw gd v
pzd dg, w p d
FDA ppd p d-
xg py -vg m.
mk w gg m d
pmy , w d m y d
k-b pdby w w v pd.5
Ay dg bm
dg. O d, w
Fg 5, gy bm v d
gy 2000, py d, p,
xp m f d pdb
4 N V Cp A, V Sg:
T C Ivm U.S. Bmd Iv d Impv F.D.A. Rm, Ob 2011. T vy
dd m g d v mmy:
pdby FDA g dg
vm y-g mp.
5 Ry MBd, S CEO Cg FDA
C Up Rgy Pwy Nw Md, Reuters,
Jy 20, 2012.
vw p. O d, g
vw m d p d
pdb, bm g- d
dv pd dppd dby
v m m pd.
W FDA mgy gd p
gy dg, Agy gv dvp
db d by dg, xmp,
mxd g. U y, dv
m d w md m xvy
dv dg dvpm. Evg
k -dv p w
q g p p d
p g p. Lkg
mp FDA 2008 gd ddg
dv k dg yp 2 db:
Rvw m dbd -mdg
dg.
T m vdgp d
J 2008, 30 m NDA w
d, dd p bm m
FDA qm. (T dg w ppvd EU 2007.)
T mb dmzd p d
p-y NDA d m
2.5 d 4 d, pvy, gd
w pbd.
Oby U.S. d pdm
pp, w b -d d
d b. Id kg p
dg dvpm, wv, Agy
Edg d Mb Dg Advy
Cmm y vd q dv
k m by dg dd
m w p d
dd vd k. T p w d
-
7/31/2019 CHI FDA Report-Embargoed
7/12
6 chi-caliornia healthcare institute
may 2012
qm wd dd y ppv
p d p dd $100
m.6
F my dg, p d FDA
v b wg p d
d mpvy w p py
b gd v m
d b p v g b.
Mw, v p 15 y y by
6 C. Vk, P. Bd, A Ay Imp
FDA Gd Addg Cdv Rk
Dg yp 2 Db C Dvpm,
Contemporary Clinical rials, My 2011, pp. 324-32; Hd
Ldd, H Sd Ndd Oby Dg, FDA
Adv Sy,Nature News Blog, M 30, 2012.
dg md U.S. mk;
w wdw wg p-ppv
dv v. T d Agy
d dd . I 2007,
d d gd by dg, g
-y w mmm 4,500 p:
3,000 dmzd m m, 1,500
pb. A pp, g, dp
by m 30 p 50 p,
vy g m d d w
g g p pw vd dy.
Tkg b k, Sjy K, dg
dvy p, mmd, W
wg d dg k
dpdg b. I y v dg w
5 p wg v w 10 p
FIGURE 5.
-
7/31/2019 CHI FDA Report-Embargoed
8/12
chi-caliornia healthcare institute 7
may 2012
15 p, w mg p m k. S,
b-k m by d db
pd g bd gy, w
FDA pd g-m d (
w dmzd gp) d gdp.7
Byd md, d v d
p dm. Ad p
v bd m dd , gg
p gp, md p d d
f, v mp ppvg
w dg. T m gy
1970 d 1980 w, w d
w AIDS, d-d gz d
w FDA: by gg
d xp; by g
p w md p; d
by ppg dy Cg, w vg
d ppp qd Agy
.
Ogzd dvy b p d
d FDA dj p. T Agyd dd m , d d
p w gzd d
dv w, bm , d
pp. T d d p
pw gzg v
p d p d Agy
dvp ggg m. Umy,
d wy, d AIDS dmy
dmd pw p p,
py pg FDA dp xby dd y d fy by kg
7 Rb Dw, Sy I d Rg Dv
Ev C Dvpm P, Spmb
29, 2011, p://mmy.ppd.m//-p/
y--d-g-dv-v--
dvpm-p
vwp p d
d. I q m,
pd Agy d wd b
v k m p .8
A dp PDUFA 1992, FDA
dd d ppv p. T
py gd dg pp -
g d kd v p.
Lgy p AIDS pdm,
Agy wd p bm d
g dp, d vd
b p. F AIDS dg,
m g dp w by
wd pp HIV v d
p. Ad ppv w d:
p w qd d p-mkg
d vd dg
b. B b v AIDS d
dd. S pgm bg, 81
pd ppvd d d ppv, 32
v gd AIDS, 29 , d 20
dd.9
W ddb FDA wd
mpz dd w w-gzd, v
, b w pp
my mp pb k
g dvy. V, , m
m v dg wd. B
pp w b m m v, m
z v , y pd
by d-d mm. Ty y
8 D Cp, Reputation and Power:
Organizational Image and Pharmaceutical Regulation at the
FDA(2010), pp. 391-402.
9 J Wdk, FDA U F 2012: Hw
Iv Hp P d Jb, Sm b
Cmm Egy d Cmm Sbmm
H, U.S. H Rpv, Ap 18, 2012, p. 7.
-
7/31/2019 CHI FDA Report-Embargoed
9/12
8 chi-caliornia healthcare institute
may 2012
gz v ppv
FDA. Idd, xp pv
AIDS V Advy C
(AVAC). Fdd 1995 d ppd by
dg pb gz, dg$14 m m B d Md G
Fd, AVAC bb FDA m
dvpm pwy AIDS v.10
THE IM PORTANCE OF RE LIABLE DATA
W Cg vy dg PDUFA V
d dd g mpv Agy
pm m,
dy FDA y y.
B, Hvd D Cp p
dy FDA dg g,
pw vy p y d
gv Agy. Owg d
Cg d v dd,
dmv Agy b dwd w
by p, , d v
pp w pdd w py
md md d gd d p d d
py g wd.11
W my b d
g j w m FDA pm
mgm. Hy w ,
b, dg gg d w g
p pb md d ggv p
dvy gp b m m py vw
d d ppv. Exp w
d AIDS gg g d m
10 AIDS V Advy C Rv $14
m m G Fd,Medical News, Ag 21,
2007.
11 Cp, Reputation and Power, p. 750.
dmv dd d, b wd,
mg p xby dpd
x p d dvpg . PDUFA
V pvd Agy
d g dg. B bggdg ppv b gm w pb
d w q mpvm
mgm, d w d mm
. F , p g d
Agy Ob 2011 pp, Driving
Biomedical Innovation gg.12 T m
w wk p, w FDA d
dy vg wk g
dvpm p.
S, m-d pp mgm
w g md g d. W v
d g d wkg v p
w y w BCG d FDA, mg
Agy d d g b
ddg w p, d w
pm m v gd v m. S
w bv g v () gy
gg d yzg b pb d;(b) pdg pm m dg
x PDUFA y d k pm
y d gdy; d () g
gm mg FDA, dy,
d Cg d d w y
pd m pb m
Agy pm. I m
Agy g m 20 p
Am pd, d dpd gy
dy , b
wy d b kg mm
d y.
12 U.S. Fd d Dg Adm, Driving
Biomedical Innovation: Initiatives to Improve Products or
Patients, Ob, 2011.
-
7/31/2019 CHI FDA Report-Embargoed
10/12
chi-caliornia healthcare institute 9
may 2012
W p g dg d
bg, FDA , bm, d mgm
gz. T Agy q dy
p bm d m v
d, d yz ddw dg dd d v.
Ag wy, FDA g d
b w pm. W bv b
d ky b mpvd d mkg
w dg vw p d d
fy Agy.
B dyg b d d
d. I m wy, pbm md
M Lw bk,Moneyball: Te Art o
Winning an Unair Game(2003). I y
Okd A bb m, d
g mg By B, w, w m
m bdg m mp,
dvpd y, vd bd pp
mbg wg m. B
g w vd wdm bb
p py, , mg,
d w b w m pmw dpy wd. F y, bb d
k gd k bg vg,
d vg d bd w
mp m ggg py. Bd
v y pp, B wd g
k -b pg d ggg pg
w m gy d w
d m.
I k Uvy C
S F, J Wdk, d
FDA C Dg Ev d R,
mkd Agy w pb
kg 55,000 pm m; my,
ggd, y dd vw m
pmy wk ppvg w md.
Cy, y w mpd
FDA pm mm ,
FDA-RACK, xpd dqy
my dvd p mm
w w Agy pm d . Pp, , w dd
d d wdm
d mp FDA d (d my
dy pp, w). dy w v
pdd y d
mg g d. W dd
FDA d dy b dvp
vv pp d y
pm.
PDUFA V p x p ,
gg pp bw FDA d
dy. I mp g
m gy d: pp Agy
pm bmd v;
g dd fy;
b mpv p pb y
w mp g b pv-
vm w dg d bg d dv pb . I g
vw, pb d m d
mpv bmd dy
w d m . W mm
vm, x g bkg
g d w v
mz. N w jb pd m.
Cmm Hmbg d FDA
Am Iv Agy, w mg b
dd m p
.13 B p w , d bv
PDUFA V w b mp p
mpg .
13 Mg Hmbg, Am Iv Agy:
T FDA,Wall Street Journal, Ag 1, 2011.
-
7/31/2019 CHI FDA Report-Embargoed
11/12
10 chi-caliornia healthcare institute
may 2012
APPENDIX
Cdg w PDUFA V, Cg
dg dd gv pp
pdg dg ppv.
eFasterAccesstoSpecializedTreatments
(FAST)Act. Idd by Rp. S (R-
FL) d w (D-NY), FAS p w
d ppv pwy dg
w FDA gg
d -g d k
m. Amg g, b wd
b dg p q -k
dg by FDA. I wd
g xby dgg
vvg dd. P FAS
A dd H d v
PDUFA V.
eTransformingtheRegulatory
EnvironmenttoAccelerateAccessto
Treatments(TREAT)Act. Idd
S by S. Ky Hg (D-NC). Sm
FAS, b gy v d ppv d
-k p pd dvpm
p p w -
REPORT AUTHORS
g d. I m
md d, gy dvg
dd , d gd p
-g d.
eAdvancingBreakthrougherapiesforPatientsAct. C-pd by S.
M B (D-CO), O H
(R-U), d Rd B (R-NC),
b wd xb pwy
FDA v pd
g d d
w pmy vd d
dg my dm b
mpvm v xg p
m y g dp.
I dd, g g
FDA dg
f pb. I dd
d d gd p w
k bkg, d ppv,
d/ k dg. Ad
qm Sy HHS
mm dpd y, w
y, v qy, fy, dpdby FDA mp w
g.
PROJECT TEAM
Sm Gd
Partner and Managing Director
T B Cg Gp
Dvd L. G, P.D.
President and CEO
CHI-C H I
dd Gw, CHI
N Bkd, CHI
H Cmb, CHI
-
7/31/2019 CHI FDA Report-Embargoed
12/12
chi-caliornia healthcare institute 11
may 2012
T California Healthcare Institute (CHI), dd 1993,
dpd p gz dvd g
d dvg py wd C
bmd mmy. CHI b mmbp
m 275 dg bmd mp, dm d
d mp vvd ppg
bmd mmy. T m CH I , dvp, d dv
p d pm bmd ,
bgy, pm d md dv v
C. CHI wb www..g. Fw w
@, Fbk, LkdI d Yb.
T Boston Consulting Group (BCG) gb mgm
g m d wd dg dv b
gy. W p w m pv, pb, d
--p g dy g-
v pp, dd m g, dm p. O mzd pp mb
dp g dym mp d mk
w b v gz.
T v b mpv
dvg, bd m pb gz, d g
. Fdd 1963, BCG pv mpy w 75
f 42 .
HEADQUARTERS888 Prospect Street, Suite 220
La Jolla, California 92037
Tel: 858-551-6677 | Fax: 858-551-6688
SACRAMENTO1201 K Street, Suite 1840
Sacramento, California 95814
Tel: 916-233-3497 | Fax: 916-233-3498
WASHINGTON, D .C.1608 Rhode Island Ave., N.W., Room 238
Washington, D.C. 20036
Tel: 202-974-6313 | Fax: 202-974-6330
2012 California Healthcare Institute. All rights reserved.