Defining your target product profile: Therapeutics

Emerging Life Sciences Series
MaRS is a member of
Terms and Conditions
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
2
,QWURGXFWLRQ
:KDWLVDWDUJHWSURGXFWSUR¿OH"
$WDUJHWSURGXFWSUR¿OH733LVDNH\VWUDWHJLFGRFXPHQWWKDWSURYLGHVDVXPPDU\
RIWKHIROORZLQJ
•
•
•
•
WKHSURGXFWXQGHUGHYHORSPHQW
WKHSURGXFW¶VGHVLUHGFKDUDFWHULVWLFVDQGIHDWXUHV
WKHVWXGLHVDQGDFWLYLWLHVWKDWPXVWEHFRPSOHWHGWRGHPRQVWUDWHWKHSURGXFW¶V
SHUIRUPDQFHHI¿FDF\DQGVDIHW\
WKHIHDWXUHVRIWKHSURGXFWWKDWSURYLGHDFRPSHWLWLYHDGYDQWDJH
$ZHOOGHVLJQHG733SURYLGHVDVWUXFWXUHWRHQVXUHWKDWDFRPSDQ\HPEDUNVRQD
SURGXFWGHYHORSPHQWSURJUDPWKDWLVHI¿FLHQWDQG\HWGH¿QHVDOLVWLQJRIDOOUHOHYDQW
PHGLFDOWHFKQLFDODQGVFLHQWL¿FLQIRUPDWLRQUHTXLUHGWRUHDFKWKHGHVLUHGFRPPHUFLDO
GHYHORSPHQWRXWFRPH+LVWRULFDOO\WKH86)RRGDQG'UXJ$GPLQLVWUDWLRQ)'$
GHYHORSHGWKHFRQFHSWRID733WRIDFLOLWDWHWKHFRPPXQLFDWLRQVWUDWHJ\UHJDUGLQJ
DSDUWLFXODUGUXJGHYHORSPHQWSURJUDP+RZHYHUPDQ\RIWKHREMHFWLYHVSURYLGH
VRXQGJXLGLQJSULQFLSOHVIRUWKHGHYHORSPHQWRIGLDJQRVWLFSURGXFWVRUPHGLFDO
GHYLFHV
%HQH¿WVRID733
,IXVHGSURSHUO\D733FDQKHOSDGGUHVVLVVXHVHDUO\LQWKHSURGXFWGHYHORSPHQW
SURFHVVDQGSUHYHQWODWHVWDJHGHYHORSPHQWIDLOXUHV$733DOVRSURYLGHVYDULRXV
SDUWLHVDQGVWDNHKROGHUVHJPDQDJHPHQWERDUGPHPEHUVHPSOR\HHVDGYLVRUV
LQYHVWRUVUHJXODWRU\DXWKRULWLHVVWUDWHJLFSDUWQHUVDFOHDUVWDWHPHQWRIWKHGHVLUHG
RXWFRPHRIWKHSURGXFWGHYHORSPHQWSURJUDP7KLVFDQEHXVHGODWHUWRKHOSDVVHVV
HOHPHQWVRIWKHSURFHVVDQGWUDFNSURJUHVV7KH733LVDG\QDPLFVWUDWHJLFGRFXPHQW
WKDWVKRXOGEHUHYLVLWHGGXULQJWKHFRXUVHRIGHYHORSPHQW0XFKRIWKHLQIRUPDWLRQ
GLVFXVVHGLQWKH733VKRXOGEHLQFRUSRUDWHGLQWR\RXUEXVLQHVVSODQ
$733VHUYHVDVD
•
•
•
•
VWUDWHJLFSODQQLQJWRRO
FRPPXQLFDWLRQWRROIRUGLVFXVVLRQVZLWKUHJXODWRU\DXWKRULWLHV
FRPPXQLFDWLRQWRROIRUGLVFXVVLRQVZLWKLQYHVWRUVSDUWQHUVHPSOR\HHVDQG
RWKHUVWDNHKROGHUV
WRROIRUFRPPXQLFDWLQJVXSSRUWLQJDQGWUDFNLQJFKDQJHVGXULQJWKHOLIHF\FOHRI
WKHGHYHORSPHQWSURJUDP
7KH)'$GRFXPHQWZDVVSHFL¿FDOO\GHYHORSHGIRUWKHUDSHXWLFSURGXFWVWRSURYLGH
DIRUPDWIRUGLVFXVVLRQEHWZHHQDVSRQVRUDQGWKH)'$,WZDVGHVLJQHGIRUXVH
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
3
WKURXJKRXWWKHHQWLUHGUXJGHYHORSPHQWSURFHVVLQFOXGLQJSUHLQYHVWLJDWLRQDOQHZ
GUXJDSSOLFDWLRQSUH,1'LQYHVWLJDWLRQDOQHZGUXJDSSOLFDWLRQ,1'SKDVHV,
,,DQG,,,RIGUXJGHYHORSPHQWSRVWPDUNHWLQJSURJUDPVDQGWKHSXUVXLWRIQHZ
LQGLFDWLRQVRURWKHUVXEVWDQWLDOFKDQJHVLQODEHOOLQJ
Read the FDA’s document, ³*XLGDQFHIRU,QGXVWU\DQG
5HYLHZ6WDII7DUJHW3URGXFW3UR¿OH²$6WUDWHJLF'HYHORSPHQW3URFHVV
7RRO” You can view it at http://tinyurl.com/2cky5d6.
7KLVZRUNERRNZLOOKHOS\RXSXWWRJHWKHUDPRGL¿HG733IRUWKHUDSHXWLF
SURGXFWVP7335[WKHJRDOLVWRSURYLGHHDUO\VWDJHFRPSDQLHVDQGQHZ
HQWUHSUHQHXUVZLWKDVWUHDPOLQHGYHUVLRQRIWKH)'$¶V733DVZHOODVH[SHULHQFHLQ
SUHSDULQJVXFKDWHFKQLFDOGRFXPHQW
:KLOHDIXOO733LQFOXGHVXSWRGLIIHUHQWVHFWLRQVWKH0D56P7335[IHDWXUHV
VHYHQNH\VHFWLRQV7KHP7335[]HURHVLQRQWKHIROORZLQJDUHDV
,QGLFDWLRQVDQGXVDJH
'RVDJHDQGDGPLQLVWUDWLRQ
&RQWUDLQGLFDWLRQV
$GYHUVHUHDFWLRQV
&OLQLFDOSKDUPDFRORJ\
1RQFOLQLFDOWR[LFRORJ\
&OLQLFDOVWXGLHV
7KHVHVHYHQVHFWLRQVZHUHVHOHFWHGEDVHGRQWKHLULPSRUWDQFHZKHQGHYHORSLQJ
DEXVLQHVVSODQ6HYHUDOVHFWLRQVSURYLGHRSSRUWXQLWLHVWRGHVFULEHWKHNH\
GLIIHUHQWLDWLQJIHDWXUHVDQGFRPSHWLWLYHSRVLWLRQLQJRI\RXUSURGXFW7KHIRFXV
RQFRPSHWLWLYHSRVLWLRQLQJZLOODVVLVW\RXUFRPSDQ\LQFRPPXQLFDWLQJWKHYDOXH
SURSRVLWLRQDV\RXHPEDUNRQUDLVLQJFDSLWDODQGSUHSDULQJIRUVWUDWHJLFGLVFXVVLRQV
ZLWKSDUWQHUV
+RZWRXVHWKHVHZRUNERRNV
0DNHLWDWHDPH[HUFLVH²EXWPDNHLWTXLFN
:HEHOLHYHWKDWPXFKRIWKHLQIRUPDWLRQ\RXQHHGLVDOUHDG\NQRZQWR\RXU
PDQDJHPHQWWHDPDQGDGYLVRUVVRZHUHFRPPHQGWKDW\RXPDNHWKHFUHDWLRQRI
\RXUP7335[DWHDPHIIRUW5HPHPEHUWLPHLVRIWKHHVVHQFHIRUKLJKWHFKVWDUW
XSVZHHQFRXUDJH\RXWRFRPSOHWHWKHZRUNERRNWHPSODWHWKRURXJKO\\HWHI¿FLHQWO\
7KH¿UVWYHUVLRQRI\RXUP7735[VKRXOGEHQRORQJHUWKDQHLJKWWRSDJHV8VH
EXOOHWVDQGOLVWVWRVSHHG\RXUZRUN
5HFRUGDQGWHVW\RXUDVVXPSWLRQV
$V\RXJRWKURXJKWKHH[HUFLVHVUHFRUGDQGKLJKOLJKWNH\DVVXPSWLRQV,GHQWLI\
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
4
DVVXPSWLRQVWKDWZLOOEHWHVWHGDQGYDOLGDWHGRULQYDOLGDWHGWKURXJKIXUWKHUPDUNHW
UHVHDUFKDVZHOODVDVVXPSWLRQVWKDWZLOOEHWHVWHGWKURXJKSUHFOLQLFDORUFOLQLFDO
VWXGLHV
8VHWKHLFRQVIRUKHOS
7KH0D56ZRUNERRNVDUHVWUXFWXUHGXQGHUWKHDVVXPSWLRQWKDWWKLVLVWKH¿UVWWLPH
\RXWKHUHDGHUKDVXQGHUWDNHQDQH[HUFLVHLQRIWKLVQDWXUH7RKHOSSURYLGHFRQWH[W
IRUVRPHRIWKHLGHDVLQWKHVHZRUNERRNVZHKDYHFODUL¿HGWKHLGHDVE\GH¿QLQJ
NH\WHUPVDQGRIIHULQJUHDOZRUOGH[DPSOHV,QDGGLWLRQZHKDYHSURYLGHGOLQNV
WRDUWLFOHVSURYLGHGE\0D56WKURXJKWKH(QWUHSUHQHXU¶V7RRONLW)RUWKLVUHDVRQ
\RXPD\¿QGLWHDVLHVWWRXVHWKHVHZRUNERRNVRQDFRPSXWHUZLWKDQ,QWHUQHW
FRQQHFWLRQ
/RRNIRUWKHVHLFRQV
GHQRWHVDNH\LQGXVWU\WHUPWKDWZLOOUHFXULQWKHVHZRUNERRNV
LQGLFDWHVDQH[DPSOHGUDZQIURPDUHDOZRUOGEXVLQHVVLQRUGHUWR
LOOXVWUDWHDQLPSRUWDQWLGHD
GHQRWHVDOLQNWRDPRUHLQGHSWKRQOLQHDUWLFOH
DSSHDUVZKHUHYHU\RXDUHDVNHGWRUHFRUGVRPHWKLQJZKLOHFRPSOHWLQJ
WKHH[HUFLVHV
%HIRUH\RXVWDUW
7KHIROORZLQJVL[VWHSVZLOOKHOS\RXJDWKHUWKHGDWD\RXQHHGLQRUGHUWRVWDUWWKHVH
ZRUNERRNH[HUFLVHV
%URDGO\OLVWDOOWKHSRWHQWLDOLQGLFDWLRQVDQGXVDJHVRI\RXUSURSRVHGWKHUDSHXWLF
5DQNWKHLQGLFDWLRQVDQGVHOHFWDQLQLWLDOLQGLFDWLRQ7KHVHOHFWLRQRIWKHLQLWLDO
LQGLFDWLRQLVRQHRIWKHPRVWFULWLFDOVWUDWHJLFGHFLVLRQVIRUDQHDUO\VWDJH
WKHUDSHXWLFGHYHORSPHQWFRPSDQ\&ULWHULDWRFRQVLGHUZKHQVHOHFWLQJWKHLQLWLDO
LQGLFDWLRQLQFOXGH
D
E
F
G
:LOOWKHLQGLFDWLRQYDOLGDWHWKHSURGXFW¶VFOLQLFDOEHQH¿WV"
:LOOWKHLQGLFDWLRQFOHDUO\GHPRQVWUDWHDFRPSHWLWLYHDGYDQWDJH"
'RHVWKHLQGLFDWLRQSURYLGHDQHI¿FLHQWSDWKWRUHJXODWRU\DSSURYDO"
'RHVLWOD\WKHIRXQGDWLRQIRUOHYHUDJLQJH[SDQGHGFOLQLFDOLQGLFDWLRQVLQWKH
IXWXUH"
H 'RHVWKHLQGLFDWLRQWDUJHWDUHDOPDUNHWRSSRUWXQLW\ZLWKUHYHQXHVWKDWZLOO
VXSSRUWWKHFRPSDQ\¶VIXWXUHJURZWK"
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
5
:KLOH\RXPD\QRWEHDEOHWRDQVZHUDOORIWKHVHTXHVWLRQVLQLWLDOO\WKHH[HUFLVH
RIGH¿QLQJD733SURYLGHVDVWUXFWXUHGSODWIRUPWRWHVWWKHLQLWLDOLQGLFDWLRQ
DJDLQVWNH\PHWULFV
2QFH\RXKDYHVHOHFWHGDQLQLWLDOLQGLFDWLRQLGHQWLI\WKHFRPSHWLWLYHSURGXFWV
LQGLFDWHGIRUWKHVDPHFRQGLWLRQV&RQWDFW\RXU0D56DGYLVRUIRUDVVLVWDQFH
0D56KDVDFFHVVWR/LIH6FLHQFH$QDO\WLFV¶0HG75$&.GDWDEDVHZKLFK
FDQUHWULHYHOLVWVRISRWHQWLDOFRPSHWLWRUWKHUDSHXWLFVDWYDULRXVVWDJHVRI
GHYHORSPHQW5HVHDUFK\RXUFRPSHWLWRUVEDVHGRQGLVHDVHLQGLFDWLRQPHFKDQLVP
RIDFWLRQFKHPLFDORUELRORJLFDOFODVVRUDFRPELQDWLRQRIGLVHDVHDQG
PHFKDQLVPRIDFWLRQ5HVXOWVFDQEH¿OWHUHGE\WKHSKDVHRIWKHFOLQLFDOWULDO
$IWHUFROOHFWLQJWKHQDPHVRIFXUUHQWO\DSSURYHGSURGXFWVWKDWWDUJHWWKH
LQGLFDWLRQQDYLJDWHWRWKH'UXJV#)'$VHFWLRQRIWKH)'$ZHEVLWHDW
KWWSZZZDFFHVVGDWDIGDJRYVFULSWVFGHUGUXJVDWIGDLQGH[FIm7KHVLWH
DOORZV\RXWRVHDUFKGUXJVDQGWKHUDSHXWLFVE\GUXJQDPHDFWLYHLQJUHGLHQW
RUDSSOLFDWLRQQXPEHU6HDUFKLQJIRUDVSHFL¿FGUXJZLOODOORZ\RXWRYLHZ
WKHUDSHXWLFHTXLYDOHQWVDSSURYDOKLVWRU\OHWWHUVUHYLHZVDQGUHODWHGGRFXPHQWV
DQGSHUKDSVPRVWLPSRUWDQWO\ODEHOLQIRUPDWLRQ7KHODEHOLQIRUPDWLRQLVWKH
VDPHDVZKDWLVLQFOXGHGZLWKWKHSDFNDJHLQVHUWIRUSUHVFULSWLRQGUXJVDQG
WKHUDSHXWLFV%\FRQYHQWLRQLWSURYLGHVLQIRUPDWLRQFRQFHUQLQJLQGLFDWLRQVDQG
XVDJHGRVDJHDQGDGPLQLVWUDWLRQFRQWUDLQGLFDWLRQVDGYHUVHHYHQWVFOLQLFDO
SKDUPDFRORJ\SUHFOLQLFDOWR[LFRORJ\DQGFOLQLFDOH[SHULHQFH7KHVHVHYHQNH\
DUHDVFRPSULVHWKHP7335[
5HYLHZDQXPEHURIFRPSHWLQJSURGXFWV>VHHDERYH@DQGKLJKOLJKWWKHDUHDV
ZKHUHWKHVHFRPSHWLWLYHSURGXFWVKDYHOLPLWDWLRQVDQGZKHUH\RXUSURGXFWLV
GHVLJQHGWRRIIHULPSURYHGEHQH¿WV
'HWHUPLQHWKHSUHFOLQLFDODQGFOLQLFDOVWXGLHVQHHGHGWRGHPRQVWUDWH\RXU
FRPSHWLWLYHGLIIHUHQWLDWLRQ(YHQLI\RXGRQ¶W\HWKDYHDFRPSOHWHFOLQLFDO
GHYHORSPHQWSODQZHUHFRPPHQG\RXRXWOLQHKRZPDQ\WULDOV\RXZLOOQHHGWKH
DQWLFLSDWHGRXWFRPHIURPWKRVHWULDOVDQGDSURVSHFWLYHGHYHORSPHQWWLPHOLQH
1RWHWKDWWKHDERYHDSSURDFKDSSOLHVWRSRWHQWLDOWKHUDSHXWLFWUHDWPHQWVZKHUH
FRPSHWLWRUSURGXFWVH[LVW1HYHUWKHOHVVFUHDWLQJDQP7335[IRUDQRUSKDQGUXJ
LQGLFDWLRQLVDZRUWKZKLOHH[HUFLVHDVLWZLOOKHOS\RXRUJDQL]H\RXUWKRXJKWVRXWOLQH
WKHVWXGLHVDQGFOLQLFDOWULDOV\RXZLOOKDYHWRXQGHUWDNHDQGGH¿QHPLOHVWRQHV
UHODWHGWRIXQGUDLVLQJ
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
6
:25.%22.
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH
7KHUDSHXWLFV
,QGLFDWLRQVDQGXVDJH
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RI
WKHSURSRVHGSURGXFW7KLVVHFWLRQLVQRWDERXWDWHFKQRORJ\SODWIRUPRUEURDG
GHVFULSWLRQVRIRSSRUWXQLWLHVDFURVVDUDQJHRILQGLFDWLRQVDQGGLVHDVHV,QUHVSRQVH
WRWKHIROORZLQJTXHVWLRQVNHHS\RXUDQVZHUVEULHIDQGIRFXVHG
•
•
•
•
:KDWLVWKHGUXJSURGXFW"
,VLWIRUWUHDWPHQWRUSUHYHQWLRQ"
:KDWGLVHDVHFRQGLWLRQGRHVLWWDUJHW"
:KRDUHWKHWDUJHWSDWLHQWV"
In the corresponding section of the workbook template, write
one or two brief sentences in response to the questions above.
List assumptions and studies that will validate (or invalidate) the
assumptions.
2QWKHIROORZLQJSDJHUHYLHZWKHH[DPSOHRI*HQHQWHFK¶V+HUFHSWLQ®ZLWKUHJDUGWR
LQGLFDWLRQVDQGXVDJH
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
7
([DPSOH,QGLFDWLRQVDQGXVDJH²+HUFHSWLQ®
7KHSKDUPDFHXWLFDOFRPSDQ\*HQHQWHFKKDVGHYHORSHGDKLJKO\VXFFHVVIXO
EUHDVWFDQFHUGUXJFDOOHG+HUFHSWLQ
7KHLQGLFDWLRQDQGXVDJHVHFWLRQIRU+HUFHSWLQUHDGV³+HUFHSWLQLVD+(5QHX
UHFHSWRUDQWDJRQLVWLQGLFDWHGIRUWKHWUHDWPHQWRI+(5RYHUH[SUHVVLQJEUHDVW
FDQFHU´
+HUFHSWLQLVDPRQRFORQDODQWLERG\WDUJHWLQJ+(5DQGKDGEHHQVKRZQLQERWK
in vitroDVVD\VDQGLQDQLPDOVWXGLHVWRLQKLELWSUROLIHUDWLRQRIKXPDQWXPRXUV
WKDWRYHUH[SUHVV+(5$SSURSULDWHO\*HQHQWHFKIRFXVHGWKHFOLQLFDOGHYHORSPHQW
SURJUDPIRU+HUFHSWLQE\WDUJHWLQJWKH+(5RYHUH[SUHVVLQJVXESRSXODWLRQRI
EUHDVWFDQFHUSDWLHQWVZKLFKUHSUHVHQWHGDERXWRIDOOEUHDVWFDQFHUSDWLHQWV
7KHLQLWLDOODEHOOLQJIRU+HUFHSWLQZDVOLPLWHGWRWKHWUHDWPHQWRIZRPHQZLWK
PHWDVWDWLF+(5RYHUH[SUHVVLQJEUHDVWFDQFHU)ROORZLQJ)'$DSSURYDOIRUWKH
QDUURZHULQGLFDWLRQRIPHWDVWDWLFEUHDVWFDQFHU*HQHQWHFKFRPSOHWHGDGGLWLRQDO
VWXGLHVWKDWGHPRQVWUDWHGWKHFOLQLFDOEHQH¿WLQWKHDGMXYDQWVHWWLQJDVZHOO7KH
FRPELQDWLRQRIWKHWZRVWXGLHVVLJQL¿FDQWO\H[SDQGHGWKHPDUNHWIRU+HUFHSWLQ
DQGSURYLGHGWKHEDVLVIRUDEURDGODEHOLQGLFDWLRQIRUDWDUJHWHGSDWLHQW
SRSXODWLRQ
Source: KWWSZZZJHQHFRPJHQHSURGXFWVLQIRUPDWLRQSGIKHUFHSWLQSUHVFULELQJSGI
&RQVLGHUWKHIROORZLQJSRLQWVLIDSSOLFDEOHZLWKUHJDUGWR\RXURZQ
SURGXFW
•
•
•
•
•
'RHV\RXUSURSRVHGLQGLFDWLRQPHHWDQXQPHWPHGLFDOQHHG"
:LOOWKHWDUJHWLQGLFDWLRQUHTXLUHDQ\SUHFOLQLFDOVWXGLHVVXFKDVFDUFLQRJHQLFLW\
WKDWPLJKWVLJQL¿FDQWO\GHOD\WLPHWRPDUNHWHQWU\"
,I\RXDUHSXUVXLQJDQRUSKDQLQGLFDWLRQKDYH\RXFRQVLGHUHGWKHFKDOOHQJHV
DVVRFLDWHGZLWKHQUROOLQJDVXI¿FLHQWQXPEHURISDWLHQWVLQWKHUHTXLUHGFOLQLFDO
GHYHORSPHQWVWXGLHV"'R\RXKDYHSODQVWRPLWLJDWHWKHULVN"
+RZPLJKWWKH¿UVWFOLQLFDOLQGLFDWLRQRIIHUDQRSSRUWXQLW\WRJDLQHDUO\LQLWLDO
DSSURYDORIWKHGUXJ"
:LOO\RXEHDEOHWROHYHUDJHWKH¿UVWVXFFHVVIXODSSURYDODQGH[SDQGWKHPDUNHW
WRDGGLWLRQDOLQGLFDWLRQV"
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
8
5HYLHZWKHH[DPSOHRI$PJHQ¶V9HFWLEL[®ZLWKUHJDUGWRLQGLFDWLRQVDQGXVDJH
([DPSOH,QGLFDWLRQVDQGXVDJH²9HFWLEL[®
7KHSKDUPDFHXWLFDOFRPSDQ\$PJHQKDVGHYHORSHG9HFWLEL[WRWUHDWFRORUHFWDO
FDQFHU
7KHLQGLFDWLRQVDQGXVDJHODEHOIRU9HFLWLEL[UHDGV³9HFWLEL[®SDQLWXPXPDELV
LQGLFDWHGDVPRQRWKHUDS\IRUWKHWUHDWPHQWRISDWLHQWVZLWK(*)5H[SUHVVLQJ
PHWDVWDWLFFRORUHFWDOFDUFLQRPDZLWKQRQPXWDWHGZLOGW\SH.5$6DIWHUIDLOXUH
RIÀXRURS\ULPLGLQHR[DOLSODWLQDQGLULQRWHFDQFRQWDLQLQJFKHPRWKHUDS\
UHJLPHQV´
9HFWLEL[SURYLGHVDQH[DPSOHRIWKHLQFUHDVLQJLPSRUWDQFHRIELRPDUNHUVLQ
WDUJHWLQJGUXJVDQGWKHLPSRUWDQFHRISDWLHQWVHOHFWLRQLQWKHHPHUJLQJHUDRI
SHUVRQDOL]HGPHGLFLQH
,QWKHFDVHRI9HFWLEL[QRWRQO\PXVWWKHFDQFHUEHRYHUH[SUHVVLQJ(*)5WKH
WDUJHWEXWDVHFRQGDU\PDUNHUQRQPXWDWHG>ZLOGW\[email protected]$6PXVWDOVREH
FRQ¿UPHGSULRUWRWUHDWPHQWRIWKHSDWLHQW7KHSKDVH,,,FOLQLFDOVWXGLHVKDG
FOHDUO\GHPRQVWUDWHGWKDW9HFWLEL[KDVQRHIIHFWRQSURJUHVVLRQIUHHVXUYLYDOLQ
SDWLHQWVZLWK.5$6PXWDQWWXPRXUVEXWDSRVLWLYHHIIHFWLQSDWLHQWVZLWKWXPRXUV
EHDULQJWKHQRQPXWDWHG.5$6
0RUHUHFHQWO\$PJHQKDVUHSRUWHGWKDWPXWDWLRQVLQ15$6DQRWKHUPHPEHU
RIWKH5$6JHQHIDPLO\DUHDOVRDVVRFLDWHGZLWKODFNRIUHVSRQVHWR9HFWLEL[
VXJJHVWLQJIXUWKHUUH¿QHPHQWVWRSDWLHQWVHOHFWLRQIRURSWLPL]HGUHVSRQVH
Source: KWWSSLDPJHQFRPXQLWHGBVWDWHVYHFWLEL[YHFWLEL[BSLSGI
&RQVLGHUWKHIROORZLQJSRLQWVLIDSSOLFDEOHZLWKUHJDUGWR\RXURZQ
SURGXFW
•
•
•
+RZZHOOFKDUDFWHUL]HGLVWKHPHFKDQLVPRIDFWLRQRI\RXUWKHUDSHXWLFSURGXFW"
:KDWELRPDUNHUVFRXOGEHXVHGWRVHOHFWWKHPRVWOLNHO\UHVSRQGHUVWRWKHQHZ
WKHUDSHXWLF"
:LOOWKHUHEHDFRPSDQLRQGLDJQRVWLFUHTXLUHGIRUWKHQHZGUXJ"
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
9
'RVDJHDQGDGPLQLVWUDWLRQ
'RVHVFDQEHSUHSDUHGZLWKYDU\LQJDPRXQWVRIDFWLYHLQJUHGLHQWLQYDULRXV
IRUPXODWLRQVDQGFDQEHDGPLQLVWHUHGE\DZLGHYDULHW\RIURXWHVLQFOXGLQJ
QRQLQYDVLYHVWUDWHJLHVVXFKDVRUDOWUDQVGHUPDOWUDQVPXFRVDODQGSXOPRQDU\
LQKDODWLRQRULQYDVLYHVWUDWHJLHVVXFKDVLQWUDYHQRXVVXEFXWDQHRXVRULQWUDPXVFXODU
LQMHFWLRQV
'RVDJHDQGDGPLQLVWUDWLRQFDQRIIHUVLJQL¿FDQWRSSRUWXQLWLHVWRGHYHORSD
FRPSHWLWLYHVWUDWHJ\RUDVHULHVRIOLQHH[WHQVLRQVIRUDQHZWKHUDSHXWLFSURGXFW
0XOWLSOHIRUPXODWLRQVFDQEHEHQH¿FLDOKRZHYHULQVHOHFWLQJWKH¿UVWIRUPXODWLRQ
FKRRVHVRPHWKLQJWKDWZLOOGHPRQVWUDWHHI¿FDF\ZLWKOLPLWHGULVNRIFDXVLQJVLGH
HIIHFWVWKRXJKWKHFKRVHQURXWHRIDGPLQLVWUDWLRQ
,Q\RXUGHYHORSPHQWSURJUDPVHOHFWWKHURXWHRIDGPLQLVWUDWLRQWKDWRIIHUVWKH
EHVWEDODQFHIRUUDSLGPDUNHWHQWU\DQGDFFHSWDEOHPDUNHWDGRSWLRQULVN2SWLRQV
IRUDOWHUQDWLYHURXWHVFDQEHXVHGDVOLQHH[WHQVLRQVKRZHYHUWKHPRVWFRQYHQLHQW
URXWHPLJKWQRWEHUHTXLUHGIRU¿UVWDSSURYDODQG¿UVWPDUNHWHQWU\LI\RXUSURGXFWLV
FOHDUO\VXSHULRULQRWKHUFOLQLFDODUHDV
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RIWKH
SURSRVHGGRVDJHDQGDGPLQLVWUDWLRQ,QUHVSRQVHWRWKHIROORZLQJTXHVWLRQVNHHS
\RXUDQVZHUVEULHIDQGIRFXVHG
•
•
•
•
+RZPXFKGUXJLVUHTXLUHG"
+RZLVLWDGPLQLVWHUHG"
+RZRIWHQLVWKHWUHDWPHQW"
:KDWLVWKHGXUDWLRQRIWUHDWPHQW"
In the corresponding section of the workbook template, write one
or two brief sentences in response to the questions listed above.
List assumptions and studies that will validate (or invalidate) the
assumptions.
2QWKHIROORZLQJSDJHUHYLHZWKHH[DPSOHRI5RFKH¶V%RQLYD®ZLWKUHJDUGWRGRVDJH
DQGDGPLQLVWUDWLRQ
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
10
([DPSOH'RVDJHDQGDGPLQLVWUDWLRQ²%RQLYD®
5RFKHKDVGHYHORSHG%RQLYDDSURGXFWIRUWKHWUHDWPHQWRIRVWHRSRURVLVLQ
SRVWPHQRSDXVDOZRPHQ,WLVDYDLODEOHDVDQRUDOWDEOHWWREHWDNHQRQFHDGD\
DVDQRUDOWDEOHWWREHWDNHQRQFHDZHHNDQGLQDQLQWUDYHQRXVIRUPWREH
DGPLQLVWHUHGRQFHHYHU\WKUHHPRQWKV
:KLOHSDWLHQWVPLJKWSUHIHUGDLO\RUPRQWKO\RUDODGPLQLVWUDWLRQGRVLQJ
LQVWUXFWLRQVIRURUDODGPLQLVWUDWLRQUHTXLUHWKHIROORZLQJ
•
•
•
WKHWDEOHWPXVWEHWDNHQPLQXWHVEHIRUHWKH¿UVWIRRGEHYHUDJHRU
PHGLFDWLRQRIWKHGD\
SDWLHQWPD\RQO\GULQNZDWHUIRUPLQXWHVDIWHUWDNLQJWKHWDEOHW
SDWLHQWVPXVWQRWOLHGRZQIRUDWOHDVWPLQXWHVDIWHUWDNLQJWKHWDEOHW
,QFRQWUDVWWKHTXDUWHUO\LQWUDYHQRXVSURGXFWLVSRVLWLRQHGDVDTXLFNWR
VHFRQGURXWHWRDGPLQLVWHUPHGLFDWLRQWKDWHOLPLQDWHVWKHQHHGIRUSUHDQGSRVW
GRVHIDVWLQJ,WDOVRSURYLGHVDQDWWUDFWLYHDOWHUQDWLYHWRSDWLHQWVZLWKGLI¿FXOW\
VZDOORZLQJRUWKRVHZKRVXIIHUJDVWURLQWHVWLQDOVLGHHIIHFWVZLWKWKHRUDOIRUP
Source: KWWSZZZJHQHFRPJHQHSURGXFWVLQIRUPDWLRQERQLYDSGISLSGI
1
)RUWKHFOLQLFDOLQGLFDWLRQVHOHFWHGIRU\RXUQHZWKHUDSHXWLFSURGXFWUHYLHZWKH
FXUUHQWWKHUDSHXWLFRIIHULQJVDQGWKHLUURXWHVRIDGPLQLVWUDWLRQ'HWHUPLQHLIWKH
DGPLQLVWUDWLRQURXWHIRU\RXUSURGXFWLV
L VLPLODUQRFKDQJHLQFOLQLFDOSUDFWLFH
LL PRUHFRQYHQLHQWRIIHULQJDVLJQL¿FDQWFRPSHWLWLYHDGYDQWDJH
LLL PRUHFRPSOLFDWHGRUPRUHLQYDVLYH
,I\RXUSURGXFWIDOOVLQWRWKHWKLUGFDWHJRU\DERYH\RXZLOOQHHGWRLGHQWLI\RWKHU
EHQH¿WVRIWKHSURGXFWJUHDWHUHI¿FDF\LPSURYHGVDIHW\WKDWZLOOPLWLJDWHWKH
SHUFHLYHGULVNRIPDUNHWDGRSWLRQ
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
11
&RQWUDLQGLFDWLRQV
$FRQWUDLQGLFDWLRQLVDV\PSWRPRUFRQGLWLRQWKDWPDNHVDSDUWLFXODUWUHDWPHQWRU
SURFHGXUHLQDGYLVDEOH7KH)'$¶V733JXLGDQFHGRFXPHQWOLVWVVLWXDWLRQVLQZKLFK
WKHGUXJPLJKWEHFRQWUDLQGLFDWHGWKDWLQFOXGH³LQFUHDVHGULVNRIKDUPEHFDXVHRI
DJHVH[FRQFRPLWDQWWKHUDS\GLVHDVHVWDWH´RWKHU³DGYHUVHUHDFWLRQVZKLFKZRXOG
OLPLWXVH´DQG³NQRZQQRWWKHRUHWLFDOKD]DUGV´
(YHQLQHDUO\VWDJHVRIGHYHORSPHQWLWLVSRVVLEOHWRLGHQWLI\SDWLHQWVZKRPLJKW
EHKDUPHGLIWUHDWHGZLWKWKHGUXJ7KLVLQIRUPDWLRQFDQEHGHWHUPLQHGIURPDQ
XQGHUVWDQGLQJRIWKHPHFKDQLVPRIDFWLRQDQXQGHUVWDQGLQJRIFODVVHIIHFWLIWKH
QHZGUXJEHORQJVWRDQH[LVWLQJFODVVRIGUXJVRUWR[LFLW\VWXGLHVWKDWLGHQWLI\
SRWHQWLDOULVNIDFWRUV
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOODGGUHVVDQGSURYLGHDFRQFLVH
UHVSRQVHWRWKHIROORZLQJTXHVWLRQ:KLFKSDWLHQWVVKRXOGQRWEHWUHDWHGZLWKWKLV
GUXJ"
In the corresponding section of the workbook template, write one or
two brief sentences in response to the question above.
2QWKHIROORZLQJSDJHUHYLHZWKHH[DPSOHVRI/LSLWRU®DQG$FFXWDQH®ZLWKUHJDUGWR
FRQWUDLQGLFDWLRQV
0HGOLQH3OXV)HEUXDU\Contraindications5HWULHYHG-DQXDU\
IURPKWWSZZZQOPQLKJRYPHGOLQHSOXVHQF\DUWLFOHKWm
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
12
([DPSOH&RQWUDLQGLFDWLRQV²/LSLWRU®DQG$FFXWDQH®
•
/LSLWRU3¿]HU¶VEORFNEXVWHUGUXJIRUORZHULQJFKROHVWHUROLVFRQWUDLQGLFDWHG
IRUXVHLQSDWLHQWVZLWKDFWLYHOLYHUGLVHDVHDQGIRUSUHJQDQWDQGODFWDWLQJ
ZRPHQ
•
$FFXWDQHLVRWUHWLQRLQLVDSUHVFULSWLRQPHGLFDWLRQPDUNHWHGE\5RFKHDQG
RWKHUVDQGLQGLFDWHGIRUVHYHUHUHFDOFLWUDQWQRGXODUDFQH,WLVFRQWUDLQGLFDWHG
IRUIHPDOHSDWLHQWVZKRDUHRUPD\EHFRPHSUHJQDQWDVWKHUHLV³DQ
H[WUHPHO\KLJKULVNWKDWVHYHUHELUWKGHIHFWVZLOOUHVXOWLISUHJQDQF\RFFXUV
ZKLOHWDNLQJ$FFXWDQHLQDQ\DPRXQW´
Sources:
KWWSZZZS¿]HUFRP¿OHVSURGXFWVXVSLBOLSLWRUSGI
KWWSWLQ\XUOFRPFE\FI6
&RQVLGHUWKHIROORZLQJSRLQWVLIDSSOLFDEOHZLWKUHJDUGWR\RXURZQ
SURGXFW
•
•
•
•
:KLFKSDWLHQWVPLJKWEHKDUPHGE\\RXUSURGXFW"7KLVLQIRUPDWLRQZLOOLQIRUP
GHFLVLRQVRQZKLFKSDWLHQWVWRH[FOXGHIURPXSFRPLQJFOLQLFDOVWXGLHVDQGZLOO
PLWLJDWHWKHULVNRIJHQHUDWLQJDGYHUVHHYHQWVLQIXWXUHFOLQLFDOVWXGLHV
&DQ\RXLGHQWLI\DQ\FRQWUDLQGLFDWLRQVWKDWPLJKWEHGXHWR\RXUGUXJ¶V
PHFKDQLVPRIDFWLRQRULWVPHWDEROLVP"
$UHWKHUHDQ\EDVHWKHUDSLHVWKDWPD\LQWHUIHUHZLWK\RXUSURSRVHGWKHUDSHXWLF
DQGFRXOGWKHVHEDVHWKHUDSLHVFRQVWLWXWHDFRQWUDLQGLFDWLRQ"
+DYH\RXFKHFNHGWKHSURGXFWPRQRJUDSKVRIVLPLODUSURGXFWVRUSURGXFWFODVVHV
WRLGHQWLI\SRWHQWLDOFRQWUDLQGLFDWLRQV"
$GYHUVHUHDFWLRQV
$QDGYHUVHUHDFWLRQLVDUHVXOWRIGUXJWKHUDS\WKDWLVQHLWKHULQWHQGHGQRUH[SHFWHG
LQQRUPDOWKHUDSHXWLFXVHDQGWKDWFDXVHVVLJQL¿FDQWVRPHWLPHVOLIHWKUHDWHQLQJ
FRQGLWLRQV$GYHUVHUHDFWLRQVDUHFRQVLGHUHGVLGHHIIHFWVDQGFDQEHREVHUYHGLQ
VRPHVXEVHWRIDSRSXODWLRQXQGHUJRLQJWUHDWPHQW7KHREMHFWLYHRIWKHUDSHXWLF
GHYHORSPHQWSURJUDPVLVWRPLQLPL]HWKHLQFLGHQFHRIDGYHUVHUHDFWLRQVZKLOH
PD[LPL]LQJWKHUDSHXWLFEHQH¿W
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RIWKH
SRVVLEOHDGYHUVHUHDFWLRQVDVVRFLDWHGZLWK\RXUSURGXFW,QUHVSRQVHWRWKHIROORZLQJ
TXHVWLRQVNHHS\RXUDQVZHUVEULHIDQGIRFXVHG
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
•
•
•
13
:KDWDGYHUVHHIIHFWVPLJKWEHDQWLFLSDWHGEDVHGRQUHVXOWVRISUHFOLQLFDO
VWXGLHV"
:KDWDGYHUVHHIIHFWVPLJKWEHH[SHFWHGGXHWRGUXJFODVV"
:KDWDGYHUVHUHDFWLRQVKDYHEHHQREVHUYHGLQFOLQLFDOVWXGLHV"
In the corresponding section of the workbook template, write one or
two brief sentences in response to the questions listed above. List
any assumptions and studies that would validate (or invalidate) the
assumptions.
5HYLHZWKHH[DPSOHEHORZRI%D\HU¶V3UHFRVH®ZLWKUHJDUGWRDGYHUVHUHDFWLRQV
([DPSOH$GYHUVHUHDFWLRQV²3UHFRVH®
$GYHUVHHYHQWVGRQRWQHHGWREHOLIHWKUHDWHQLQJEXWPLJKWFRQWULEXWHWRSRRU
FRPSOLDQFHZLWKDGUXJWKHUDS\DQGUHQGHUWKHUDS\LQHIIHFWLYH)RUH[DPSOH
GXULQJFOLQLFDOWULDOVSDWLHQWVWUHDWHGZLWK3UHFRVHDGUXJGHYHORSHGE\%D\HU
+HDOWKFDUH3KDUPDFHXWLFDOVDQGLQGLFDWHGDVDQDGMXQFWWRGLHWDQGH[HUFLVHIRU
SDWLHQWVZLWKW\SHGLDEHWHVH[SHULHQFHGVLJQL¿FDQWJDVWURLQWHVWLQDOV\PSWRPV
LQFOXGLQJDEGRPLQDOSDLQGLDUUKHDDQGÀDWXOHQFH7KHVH
JDVWURLQWHVWLQDOHIIHFWVZHUHDPDQLIHVWDWLRQRIWKHPHFKDQLVPRIDFWLRQRI
3UHFRVH%D\HUSURYLGHVJXLGDQFHRQGLHWDU\VWUDWHJLHVWRPLWLJDWHWKHVLGH
HIIHFWVEXWLIWKHSUHVFULEHGGLHWLVQRWREVHUYHGGXULQJWUHDWPHQWZLWK3UHFRVH
WKHLQWHVWLQDOVLGHHIIHFWVPD\EHLQWHQVL¿HG
Source: KWWSZZZGUXJVFRPSURSUHFRVHKWPO
5HYLHZSRWHQWLDOVLGHHIIHFWVEDVHGRQ\RXUGUXJ¶VPHFKDQLVPRIDFWLRQLWV
PHWDEROLVPDQGDQLPDOWR[LFLW\SUR¿OHDQGIRUWDUJHWHGWKHUDSHXWLFVWKHH[SUHVVLRQ
RIWKHWDUJHWRQVSHFL¿FRUJDQVRUWLVVXHV(YDOXDWHWKHSRWHQWLDOVLGHHIIHFWSUR¿OHRI
\RXUQHZWKHUDSHXWLFGUXJDQGPDNHVXUHWRFRQWLQXRXVO\XSGDWHWKLVVHFWLRQRI\RXU
P7335[DQGODWHU\RXU733
&RQVLGHUWKHIROORZLQJSRLQWVLIDSSOLFDEOHZLWKUHJDUGWR\RXUSURGXFW
•
•
•
:KDWDUHWKHDGYHUVHHYHQWSUR¿OHVOLVWHGLQSURGXFWPRQRJUDSKVRIFRPSHWLWLYH
SURGXFWVDQGSURGXFWVRIWKHVDPHFODVV",GHQWLI\SRWHQWLDORSSRUWXQLWLHVIRU\RXU
SURGXFWWRUHGXFHWKHLQFLGHQFHRIDGYHUVHHYHQWV
:KDWDUHDFFHSWDEOHWKUHVKROGVIRUWKHLQFLGHQFHRIDGYHUVHHYHQWVEDVHGRQ
EHQH¿WULVNUDWLRVDQGWKHSUHFHGHQWVIURPH[LVWLQJWKHUDSHXWLFVWUDWHJLHV"
$UHWKHUHDQ\RSSRUWXQLWLHVWRGHPRQVWUDWHDVXSHULRUVDIHW\SUR¿OHIRU\RXUQHZ
WKHUDSHXWLFSURGXFW"
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
14
&OLQLFDOSKDUPDFRORJ\
&OLQLFDOSKDUPDFRORJ\LQFOXGHVPHFKDQLVPRIDFWLRQSKDUPDFRNLQHWLFLQIRUPDWLRQ
GLVWULEXWLRQDQGSDWKZD\VIRUWUDQVIRUPDWLRQ
&OLQLFDOSKDUPDFRORJ\RIIHUVPXOWLSOHRSSRUWXQLWLHVIRUSURGXFWGLIIHUHQWLDWLRQDQG
FRPSHWLWLYHDGYDQWDJe7KHHOHPHQWVRIFOLQLFDOSKDUPDFRORJ\DUHXVXDOO\WKHVXEMHFW
RIH[WHQVLYHSUHFOLQLFDOUHVHDUFKDQGHDUO\SKDVH,FOLQLFDOGHYHORSPHQW
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RI
WKHFOLQLFDOSKDUPDFRORJ\RI\RXUSURSRVHGSURGXFW,QUHVSRQVHWRWKHIROORZLQJ
TXHVWLRQVNHHS\RXUDQVZHUVEULHIDQGIRFXVHGDQGOLVWWKHUHODWHGVWXGLHV
FRPSOHWHGRUSODQQHG
•
•
•
•
•
:KDWLVWKHPHFKDQLVPRIDFWLRQ"
+RZIDVWGRHVWKHGUXJUHDFKWKHWDUJHWRUJDQV"
+RZLVWKHGUXJGLVWULEXWHGWKURXJKWKHERG\"
+RZORQJGRHVWKHGUXJSHUVLVW"
+RZLVWKHGUXJFOHDUHGIURPWKHERG\"
In the corresponding section of the workbook template, write one or
two brief sentences in response to the questions listed above. List
assumptions and any studies that will need to be completed to support
those assumptions.
$QLPSRUWDQWDVSHFWRIFOLQLFDOGHYHORSPHQWLVWRHQVXUHWKDWFOLQLFDOSKDUPDFRORJ\LV
DOLJQHGZLWKWKHGHVLUHGFOLQLFDORXWFRPH,PSURYHPHQWVLQDQ\DVSHFWRIWKHFOLQLFDO
SKDUPDFRORJ\FDQSURYLGHDFRPSHWLWLYHDGYDQWDJH2QWKHIROORZLQJSDJHUHYLHZ
WKHH[DPSOHVRI=RPLJ®DQG)URYD®
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
15
([DPSOHV&OLQLFDOSKDUPDFRORJ\²=RPLJ®DQG)URYD®
%RWK]ROPLWULSWDQ=RPLJPDUNHWHGE\$VWUD=HQHFDDQGIURYDWULSWDQ)URYD
PDUNHWHGE\(QGR3KDUPDFHXWLFDOVEHORQJWRDFODVVRIGUXJVFDOOHGWULSWDQVWKDW
DUHLQGLFDWHGWRWUHDWPLJUDLQHKHDGDFKHV%RWKGUXJVDUHDYDLODEOHLQRUDOGRVHV
RIPJ8SRQDGPLQLVWUDWLRQIURYDWULSWDQWDNHVDSSUR[LPDWHO\WKUHHKRXUV
WRUHDFKPD[LPXPEORRGSODVPDFRQFHQWUDWLRQZKHUHDV]ROPLWULSWDQUHDFKHV
PD[LPXPEORRGSODVPDFRQFHQWUDWLRQLQKDOIWKDWWLPH$OOHOVHEHLQJHTXDOD
SDWLHQWVXIIHULQJIURPDPLJUDLQHZRXOGOLNHO\SUHIHUDWUHDWPHQWWKDWLVDEVRUEHG
DQGWKXVSRWHQWLDOO\HIIHFWLYHLQDVKRUWHUSHULRGRIWLPH
Source: KWWSZZZGUXJVFRPSURSUHFRVHKWPO
&RQVLGHUWKHIROORZLQJLIDSSOLFDEOHZLWKUHJDUGWR\RXUSURGXFW
•
•
•
•
:KDWDUHWKHOLPLWDWLRQVLIDQ\RIFRPSHWLWLYHWUHDWPHQWPRGDOLWLHV"
,VWKHUHDQHHGWRIRUPXODWHWKHGUXJWRLQFUHDVHWKHRQVHWRIDFWLRQRULQFUHDVH
WKHGXUDWLRQRIDFWLRQWREHQH¿WWKHSDWLHQWRUGHFUHDVHGUXJLQWHUDFWLRQV"
'RHVWKHPHFKDQLVPRIDFWLRQRIIHUPRUHVHOHFWLYHWUHDWPHQWVZLWKEHWWHU
HI¿FDF\DQGSRWHQWLDOO\IHZHUVLGHHIIHFWV"
:KLFKVWXGLHVFKDUDFWHUL]HDQGSRWHQWLDOO\GLIIHUHQWLDWH\RXUQHZWKHUDSHXWLF
SURGXFWIURPH[LVWLQJRUFRPSHWLWLYHSURGXFWVLQGHYHORSPHQW"
1RQFOLQLFDOWR[LFRORJ\
&OLQLFDOWULDOVDORQHFDQQRWDOZD\VDVVHVVWKHSRWHQWLDOORQJWHUPHIIHFWVRID
WKHUDSHXWLFRQDSHUVRQDQGWKXVUHJXODWRU\ERGLHVUHO\RQDQLPDOVWXG\GDWD
WRDVVHVVDWUHDWPHQW¶VORQJWHUPFDUFLQRJHQLFLW\PXWDJHQLFLW\RULWVHIIHFWRQ
UHSURGXFWLRQRUFDUGLRYDVFXODUDQGUHVSLUDWRU\V\VWHPV5HJXODWRU\DJHQFLHVKDYH
GHYHORSHGVSHFL¿FJXLGDQFHGRFXPHQWVUHODWHGWRFRUHWR[LFRORJ\VWXGLHV7KH
VHTXHQFLQJRIWKHVHVWXGLHVPXVWEHFDUHIXOO\SODQQHGWRHQVXUHWKDWWKH\DUH
DSSURSULDWHO\FRPSOHWHGIRUGLIIHUHQWVWDJHVRIFOLQLFDOGHYHORSPHQW$FRUHJURXSRI
VWXGLHVLVUHTXLUHGSULRUWRWKH¿UVWLQKXPDQVWXG\0DNHVXUHWKDW\RXDUHIDPLOLDU
ZLWKWKHUHTXLUHPHQWVIRU\RXUVHOHFWHGLQGLFDWLRQDQGWKDW\RXLGHQWLI\WKHNH\
VWXGLHVUHTXLUHGIRUYDULRXVVWDJHVRIFOLQLFDOGHYHORSPHQW
Read more about toxicology requirements for clinical studies.
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
16
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RI
WKHQRQFOLQLFDOWR[LFRORJ\RI\RXUSURSRVHGSURGXFW,QUHVSRQVHWRWKHIROORZLQJ
TXHVWLRQVNHHS\RXUDQVZHUVEULHIDQGIRFXVHGDQGOLVWWKHUHODWHGVWXGLHV
FRPSOHWHGRUSODQQHG
•
•
•
•
:KDWLVWKHVDIHW\SUR¿OHRIWKHGUXJEDVHGRQFHOOVWLVVXHVDQGRUJDQV\VWHPV
DVVHVVHGWKURXJKin vitroDQGin vitroDQLPDOVWXGLHV"
:KDWLVWKHPD[LPXPWROHUDWHGGRVHLQDQLPDOV"
:KDWZRXOGEHDVDIHVWDUWLQJGRVHLQKXPDQVWXGLHV"
:KDWDUHWKHHIIHFWVRIORQJWHUPH[SRVXUH"
In the corresponding section of the workbook template, write one or
two sentences in response to the questions listed on the preceding
page. List any assumptions and the studies that will validate (or
invalidate) those assumptions.
:KLOHDYDULHW\RIDQLPDOWR[LFRORJ\VWXGLHVDUHOLVWHGDVUHTXLUHPHQWVIRUD
PDUNHWLQJDSSOLFDWLRQQRWDOOVWXGLHVDUHUHTXLUHGWRJDLQDSSURYDOIRUDOOLQGLFDWLRQV
)RUH[DPSOHGUXJVLQWHQGHGIRUDFXWHXVHLQOLIHWKUHDWHQLQJLQGLFDWLRQVPLJKWQRW
UHTXLUHFDUFLQRJHQLFLW\VWXGLHV7KHDQWLFLSDWHGGXUDWLRQRIWUHDWPHQWLQFOLQLFDO
SUDFWLFHZLOODOVRGLFWDWHWKHUHFRPPHQGHGGXUDWLRQRIUHSHDWHGGRVHWR[LFLW\VWXGLHV
WRVXSSRUWYDULRXVVWDJHVRIFOLQLFDOGHYHORSPHQWDQGXOWLPDWHO\WKHPDUNHWLQJRI
WKHSURGXFW
&RQVLGHUWKHIROORZLQJLIDSSOLFDEOHZLWKUHJDUGWR\RXUSURGXFW
•
•
•
•
,I\RXUQHZSURGXFWRIIHUVDQRSSRUWXQLW\IRUDQHQKDQFHGVDIHW\SUR¿OHDVD
UHVXOWRIDQRYHOPHFKDQLVPRIDFWLRQRUDKLJKO\WDUJHWHGWKHUDSHXWLFZKDWLV
WKHQRQFOLQLFDOVWXG\WKDWZRXOGVXSSRUWWKHK\SRWKHVLV":KDWFRPSDUDWRUGUXJ
ZLOO\RXXVHDVDFRQWURODUPLQ\RXUWR[LFRORJ\VWXGLHV"
:KDW*/3*RRG/DERUDWRU\3UDFWLFHQRQFOLQLFDOVWXGLHVDUHUHTXLUHGIRU
GLIIHUHQWVWDJHVRIFOLQLFDOGHYHORSPHQWIRU\RXUWDUJHWLQGLFDWLRQ"
:KDWLVWKHDQWLFLSDWHGGXUDWLRQRIH[SRVXUHWR\RXUQHZGUXJLQWKHWDUJHW
FOLQLFDOLQGLFDWLRQ"'RHV\RXUSODQLQFOXGHWKHDSSURSULDWHQRQFOLQLFDOWR[LFRORJ\
VWXGLHV"
'RWKHQRQFOLQLFDOVWXGLHVDGGUHVVVSHFL¿FTXHVWLRQVDQGFRQFHUQVWKDWPD\EH
UHODWHGWR\RXUGUXJ"5HFRJQL]HWKDWVWDQGDUG*/3WR[LFRORJ\SDQHOVPLJKWQRW
VXI¿FHWRDGGUHVVVSHFL¿FTXHVWLRQVDQGWKDWPRUHVSHFLDOL]HGDQLPDOPRGHOVRU
DQDO\WLFVPLJKWEHUHTXLUHG
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
17
&OLQLFDOVWXGLHV
)RUUHJXODWRU\DSSURYDOFOLQLFDOVWXGLHVPXVWGHPRQVWUDWHDGUXJ¶VHI¿FDF\E\
DFKLHYLQJDVWDWLVWLFDOO\VLJQL¿FDQWLPSURYHPHQWFRPSDUHGWRDSODFHERZKLOH
DOVRDFKLHYLQJDQDFFHSWDEOHVDIHW\SUR¿OH,GHDOO\GHVLJQVWXGLHVVRWKDWWKH\
GHPRQVWUDWHDVWDWLVWLFDOO\VLJQL¿FDQWLPSURYHPHQWRYHUWKHH[LVWLQJVWDQGDUGRI
FDUH7KHVHDFWLYHFRPSDUDWRUVWXGLHVUHTXLUHFDUHIXOSODQQLQJDQGDUHQRWXVXDOO\
XQGHUWDNHQDVLQLWLDOVWXGLHV+RZHYHUDFWLYHFRPSDUDWRUVWXGLHVLIVXFFHVVIXO
SURYLGHFRPSHOOLQJHYLGHQFHDQGDFFHOHUDWHPDUNHWDGRSWLRQRIWKHSURGXFW
7KHGHVLJQRIFOLQLFDOWULDOVWRVXSSRUWWKHPDUNHWLQJVXEPLVVLRQUHTXLUHVYHU\
FDUHIXOSODQQLQJDQGVWUDWHJLFFRQVLGHUDWLRQ7KHVHVWXGLHVRIWHQLQFXUJUHDW
H[SHQVHDQGFDQPDNHRUEUHDNDGHYHORSPHQWSURJUDP,QYHVWPHQWRIWLPHDQG
UHVRXUFHVLQWRWKHGHVLJQSURFHVVLVNH\WRVXFFHVVDQGFOLQLFDODGYLVRU\ERDUGV
FRPSULVHGRIH[SHULHQFHGFOLQLFDOUHVHDUFKHUVFDQSURYLGHVWUDWHJLFDQGWDFWLFDO
JXLGDQFH6XJJHVWLRQVIURPDGYLVRU\ERDUGVVKRXOGEHUHYLHZHGDQGDOLJQHGZLWKWKH
UHTXLUHPHQWVVHWIRUWKE\YDULRXVUHJXODWRU\DJHQFLHV
,QFRPSOHWLQJWKLVVHFWLRQRIWKHP7335[\RXZLOOFUHDWHDFRQFLVHVXPPDU\RIWKH
SURSRVHGVWXGLHVLQUHODWLRQWR\RXUSURSRVHGLQGLFDWLRQDQGXVDJH,QUHVSRQVHWR
WKHIROORZLQJTXHVWLRQVNHHS\RXUDQVZHUVEULHIDQGIRFXVHG
•
•
•
•
:KDWLVWKHFOLQLFDOHYLGHQFHWRVXSSRUWWKHLQGLFDWLRQFODLPV"
+RZVWURQJLVWKHHYLGHQFH"
:KDWLVWKHVDIHW\SUR¿OH"
+RZZLOOWKHHQGSRLQWVLQWKHVWXG\VXSSRUWWKHFRPSHWLWLYHSRVLWLRQLQJDQG
PDUNHWLQJVWUDWHJ\IRUWKHQHZGUXJ"
In the corresponding section of the workbook template, create
bulleted summaries in response to the questions listed above. List
any assumptions and studies that will validate (or invalidate) the
assumptions.
Read about the following:
• the key aspects of developing a regulatory strategy
• the clinical trial application process in Canada, the US and the EU
• the application process for the approval of a new drug in Canada, the
US and the EU
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV
MaRS±(PHUJLQJ/LIH6FLHQFHV6HULHV
18
&RQVLGHUWKHIROORZLQJDQGLPSOHPHQWDVDSSOLFDEOHLQWKHGHVLJQRI\RXU
FOLQLFDOVWXGLHV
•
•
•
•
•
•
•
•
3ODQFDUHIXOO\DVDSRRUGHVLJQRIFOLQLFDOWULDOVFDQVLJQL¿FDQWO\GHOD\WKH
GHYHORSPHQWRIHYHQWKHPRVWSURPLVLQJGUXJV
,QYHVWWLPHDQGHIIRUWWRUHYLHZFOLQLFDOVWXGLHVWKDWKDYHEHHQFRPSOHWHGDQG
UHSRUWHGIRUFRPSHWLWLYHSURGXFWV
5HYLHZWKH6XPPDU\%DVLVRI$SSURYDO6%$IRUFRPSHWLWLYHSURGXFWV7KHVH
6%$UHYLHZVDUHDYDLODEOHWKURXJKWKH)'ADQGSURYLGHDZHDOWKRILQIRUPDWLRQ
RQWKHVWUHQJWKVDQGZHDNQHVVHVRIYDULRXVFOLQLFDOGHYHORSPHQWSURJUDPV
7KHUHDUHPDQ\JXLGDQFHGRFXPHQWVDYDLODEOHRQPDMRUUHJXODWRU\ZHEVLWHV
HJEMA)'$6RPHDUHVSHFL¿FWRWKHUDSHXWLFDUHDVRIGHYHORSPHQW
6HHNJXLGDQFHIURPVWDWLVWLFLDQVWRHQVXUHWKDW\RXUVWXGLHVZLOOLQFOXGHVXI¿FLHQW
QXPEHUVRISDWLHQWVWRGHPRQVWUDWHWKHVHOHFWHGHQGSRLQWV
&RQVLGHUPHHWLQJZLWKUHJXODWRU\DXWKRULWLHVWRGLVFXVVWKHFOLQLFDOGHYHORSPHQW
RI\RXUSURGXFW7KLVLVDQRSWLRQDQ\WLPHSULRUWR\RXU¿UVWLQKXPDQVWXG\DQG
DOVRDWWKHHQGRISKDVH,,
6HOHFWDQGLQFOXGHHQGSRLQWVWKDWQRWRQO\ZLOODFKLHYHUHJXODWRU\DSSURYDOEXW
ZLOOGLIIHUHQWLDWHWKHSURGXFWIURPWKHFRPSHWLWLRQDQGVXSSRUW\RXUPDUNHWLQJ
HIIRUWV
(YDOXDWHWKHYLDELOLW\RI\RXUSDWKWRZDUGFOLQLFDOGHYHORSPHQW)RUH[DPSOHLI
\RXUSURGXFWLVRQO\PLQLPDOO\PRUHHIIHFWLYHWKDQWKHVWDQGDUGRIFDUH\RXZLOO
UHTXLUHWKRXVDQGVRISDWLHQWVRUPXOWLSOHVRIWKRXVDQGVWRGHPRQVWUDWHWKLV
,VWKLVUHDOLVWLF"%HDULQPLQGWKDWGLIIHUHQFHVLQVDIHW\FDQEHYHU\GLI¿FXOWWR
YDOLGDWHVWDWLVWLFDOO\LIWKHLQFLGHQFHRIDGYHUVHHYHQWVZLWKH[LVWLQJGUXJVLVORZ
'H¿QLQJ<RXU7DUJHW3URGXFW3UR¿OH7KHUDSHXWLFV