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The Orphan Drug Act (as amended)

CONGRESSIONAL FINDINGS FOR THE ORPHAN DR G ACT

The Congress finds that---

(1) there are man diseases and conditions! s"ch as H"ntington#s disease! m oc$on"s! A%S (%o" &ehrig#sdisease)! To"rette s ndrome! and m"sc"$ar d stro'h hich affect s"ch sma$$ n"m ers of indi*id"a$sresiding in the +nited States that the diseases and conditions are considered rare in the +nited States,

( ) ade."ate dr"gs for man of s"ch diseases and conditions ha*e not een de*e$o'ed,

(/) dr"gs for these diseases and conditions are common$ referred to as 0or'han dr"gs0,

( ) eca"se so fe indi*id"a$s are affected an one rare disease or condition! a 'harmace"tica$com'an hich de*e$o's an or'han dr"g ma reasona $ ex'ect the dr"g to generate re$ati*e$ sma$$sa$es in com'arison to the cost of de*e$o'ing the dr"g and conse."ent$ to inc"r a financia$ $oss,

(2) there is reason to e$ie*e that some 'romising or'han dr"gs i$$ not e de*e$o'ed "n$ess changes aremade in the a''$ica $e Federa$ $a s to red"ce the costs of de*e$o'ing s"ch dr"gs and to 'ro*ide financia$incenti*es to de*e$o' s"ch dr"gs, and

(3) it is in the '" $ic interest to 'ro*ide s"ch changes and incenti*es for the de*e$o'ment of or'han dr"gs4

RECO!!ENDATIONS FOR IN"ESTIGATIONS OF DR GS FOR RARE DISEASES OR CONDITIONS

S5C4 2 2 6/37aa84

(a) The s'onsor of a dr"g for a disease or condition hich is rare in the States ma re."est the Secretarto 'ro*ide ritten recommendations for the nonc$inica$ and c$inica$ in*estigations hich m"st econd"cted ith the dr"g efore---

(1) it ma e a''ro*ed for s"ch disease or condition "nder section 272!

( ) if the dr"g is an anti iotic! it ma e certified for s"ch disease or condition "nder section 279! or

(/) if the dr"g is a io$ogica$ 'rod"ct! it ma e $icensed for s"ch disease or condition "nder section /21 of the P" $ic Hea$th Ser*ice Act4

If the Secretar has reason to e$ie*e that a dr"g for hich a re."est is made "nder this section is a dr"gfor a disease or condition hich is rare in the States! the Secretar sha$$ 'ro*ide the 'erson ma:ing there."est ritten recommendations for the nonc$inica$ and c$inica$ in*estigations hich the Secretar

e$ie*es! on the asis of information a*ai$a $e to the Secretar at the time of the re."est "nder thissection! o"$d e necessar for a''ro*a$ of s"ch dr"g for s"ch disease or condition "nder section 272!

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certification of s"ch dr"g for s"ch disease or condition "nder section 279! or $icensing of s"ch dr"g fors"ch disease or condition "nder section /21 of the P" $ic Hea$th Ser*ice Act4

14 The Secretar sha$$ reg"$ation 'rom"$gate 'roced"res for the im'$ementation of s" section(a)4

DESIGNATION OF DR GS FOR RARE DISEASES OR CONDITIONS

S5C4 2 3 6/37 84 (a)(1) The man"fact"rer or the s'onsor of a dr"g ma re."est the Secretar todesignate the dr"g as a dr"g for a rare disease or condition4 A re."est for designation of a dr"g sha$$ emade efore the s" mission of an a''$ication "nder section 272( ) for the dr"g! the s" mission of ana''$ication for certification of the dr"g "nder section 279! or the s" mission of an a''$ication for $icensingof the dr"g "nder section /21 of the P" $ic Hea$th Ser*ice Act4 If the Secretar finds that a dr"g for hicha re."est is s" mitted "nder this s" section is eing or i$$ e in*estigated for a rare disease or conditionand---

(A) if an a''$ication for s"ch dr"g is a''ro*ed "nder section 272!

(;) if a certification for s"ch dr"g is iss"ed "nder section 279! or

(C) if a $icense for s"ch dr"g is iss"ed "nder section /21 of the P" $ic Hea$th Ser*ice Act! the a''ro*a$!certification! or $icense o"$d e for "se for s"ch disease or condition! the Secretar sha$$ designate thedr"g as a dr"g for s"ch disease or condition4 A re."est for a designation of a dr"g "nder this s" sectionsha$$ contain the consent of the a''$icant to notice eing gi*en the Secretar "nder s" section ( )res'ecting the designation of the dr"g4

( ) For '"r'oses of 'aragra'h (1)! the term 0rare disease or condition## means an disease or conditionhich (A) affects $ess than 77!777 'ersons in the +nited States! or (;) affects more than 77!777 in the

+nited States and for hich there is no reasona $e ex'ectation that the cost of de*e$o'ing and ma:inga*ai$a $e in the +nited States a dr"g for s"ch disease or condition i$$ reco*ered from sa$es in the +nitedStates of s"ch dr"g4 Determinations "nder the 'receding sentence ith res'ect to an dr"g sha$$ e madeon the asis of the facts and circ"mstances as of the date the re."est for designation of the dr"g "nderthis s" section is made4

( ) A designation of a dr"g "nder s" section (a) sha$$ e s"

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PROTECTION FOR DR GS FOR RARE DISEASES OR CONDITIONS

S5C4 2 9 6/37cc84 (a) 5xce't as 'ro*ided in s" section ( )! if the Secretar ---

(1) a''ro*es an a''$ication fi$ed '"rs"ant to section 272( )!

( ) iss"es a certification "nder section 279! or

(/) iss"es a $icense "nder section /21 of the P" $ic Hea$th Ser*ice Act for a dr"g designated "ndersection 2 3 for a rare disease or condition! the Secretar ma not a''ro*e another a''$ication "ndersection 272( )! iss"e another certification "nder section 279! or iss"e another $icense "nder section /21of the P" $ic Hea$th Ser*ice Act for s"ch dr"g for s"ch disease or condition for a 'erson ho is not theho$der of s"ch a''ro*ed a''$ication! of s"ch certification! or of s"ch $icense "nti$ the ex'iration se*en

ears from the date of the a''ro*a$ of the a''ro*ed a''$ication! the iss"ance of the certification or theiss"ance of the $icense4 Section 272(c)( ) does not a''$ to the ref"sa$ to a''ro*e an a''$ication "nderthe 'receding sentence4

( ) If an a''$ication fi$ed '"rs"ant to section 272( ) is a''ro*ed for a dr"g designated "nder section 2 3for a rare disease or condition! if a certification is iss"ed "nder section 279 for s"ch a dr"g or if a $icenseis iss"ed "nder section /21 of the P" $ic Hea$th Ser*ice Act for s"ch a dr"g! the Secretar ma ! d"ringthe se*en- ear 'eriod eginning on the date of the a''$ication a''ro*a$! of the iss"ance of thecertification "nder section 279! or of the iss"ance of the $icense! a''ro*e another a''$ication "ndersection 272( )! iss"e another certification "nder section 279! or! iss"e a $icense "nder section /21 of theP" $ic Hea$th Ser*ice Act! for s"ch dr"g for s"ch disease or condition for a 'erson ho is not the ho$der of s"ch a''ro*ed a''$ication! of s"ch certification! or of s"ch $icense if---

(1) the Secretar finds! after 'ro*iding the ho$der notice and o''ort"nit for the s" mission of *ie s! thatin s"ch 'eriod the ho$der of the a''ro*ed a''$ication! of the certification! or of the $icense cannot ass"rethe a*ai$a i$it of s"fficient ."antities of the dr"g to meet the needs of 'ersons ith the disease orcondition for hich the dr"g as designated, or

( ) s"ch ho$der 'ro*ides the Secretar in riting the consent of s"ch ho$der for the a''ro*a$ of othera''$ications! iss"ance of other certifications! or the iss"ance of other $icenses efore the ex'iration ofs"ch se*en- ear 'eriod4

OPEN PROTOCOLS FOR IN"ESTIGATIONS OF DR GS FOR RARE DISEASES OR CONDITIONS

S5C4 2 > 6/37dd84 If a dr"g is designated "nder section 2 3 as a dr"g for a rare disease or condition andif notice of a c$aimed exem'tion "nder section 272(i) or reg"$ations iss"ed there"nder is fi$ed for s"chdr"g! the Secretar sha$$ enco"rage the s'onsor of s"ch dr"g to design 'rotoco$s for c$inica$in*estigations of the dr"g hich ma e cond"cted "nder the exem'tion to 'ermit the addition to thein*estigations of 'ersons ith the disease or condition ho need the dr"g to treat the disease or conditionand ho cannot e satisfactori$ treated a*ai$a $e a$ternati*e dr"gs4

GRANTS AND CONTRACTS FOR DE"ELOP!ENT OF DR GS FOR RARE DISEASES ANDCONDITIONS

S5C4 24 6/37ee8(a) The Secretar ma ma:e grants to and enter into contracts ith '" $ic and 'ri*ateentities and indi*id"a$s to assist in (1) defra ing the costs of ."a$ified c$inica$ testing ex'enses inc"rred inconnection ith the de*e$o'ment of dr"gs for rare diseases and conditions! ( ) defra ing the costs ofde*e$o'ing medica$ de*ices for rare diseases or conditions! and (/) defra ing the costs of de*e$o'ingmedica$ foods for rare diseases or conditions4

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( ) For '"r'oses of s" section (a)?

(1) The term 0."a$ified testing0 means---

(A) h"man c$inica$ testing---

(i) hich is carried o"t "nder an exem'tion for a dr"g for a rare disease or condition "nder section 272(i)of the Federa$ Food! Dr"g! and Cosmetic Act (or reg"$ations iss"ed "nder s"ch section),

(ii) hich occ"rs after the date s"ch dr"g is designated "nder section 2 3 of s"ch Act and efore the dateon hich an a''$ication ith res'ect to s"ch dr"g is s" mitted "nder section 273( ) or 279 of s"ch Act or"nder section /21 of the P" $ic Hea$th Ser*ice Act, and

(;) 'rec$inica$ testing in*o$*ing a dr"g is designated "nder section 2 3 of s"ch Act and efore the date onhich an a''$ication ith res'ect to s"ch dr"g is s" mitted "nder section 272( ) or 279 of s"ch Act or

"nder section /21 of the P" $ic Hea$th Ser*ice Act4

( ) The term 0rare disease or condition0 means

(A) in the case of a dr"g! an disease or conditions hich (A) affects $ess than 77!777 'ersons in the+nited States! or (;) affects more than 77!777 in the +nited States and for hich there is no reasona $eex'ectation that the cost of de*e$o'ing and ma:ing a*ai$a $e in the +nited States a dr"g for s"ch diseaseor condition i$$ e reco*ered from sa$es in the +nited States of s"ch dr"g!

(;) in the case of a medica$ de*ice! an disease or condition that occ"rs so infre."ent$ in the +nitedStates that there is no reasona $e ex'ectation that a medica$ de*ice for s"ch disease or condition i$$ ede*e$o'ed itho"t assistance "nder s" section (a)! and

(C) in the case of a medica$ food! an disease or condition that occ"rs so infre."ent$ in the +nited Statesthat there is no reasona $e ex'ectation that a medica$ food for s"ch disease or condition i$$ ede*e$o'ed itho"t assistance "nder s" section (a)4 Determinations "nder the 'receding sentence ith

res'ect to an dr"g sha$$ e made on the asis of the facts and circ"mstances as of the date the re."estfor designation of the dr"g "nder section 2 3 of the Federa$ Food! Dr"g! and Cosmetic Act is made4

(D) The term 0medica$ food0 means a food hich is form"$ated to e cons"med or administered entera$$"nder the s"'er*ision of a 'h sician and hich is intended for the s'ecific dietar management of adisease or condition for hich distincti*e n"tritiona$ re."irements! ased on recogni@ed scientific'rinci'$es! are esta $ished medica$ e*a$"ation4

(c) For grants and contracts "nder s" section (a) there are a"thori@ed to e a''ro'riated 17!777!777 for fisca$ ear 1B>>! 1 !777!777 for fisca$ ear 1B>B! 1 !777!777 for fisca$ ear 1BB74

(d) ST+D 4---The Secretar of Hea$th and H"man Ser*ices sha$$ cond"ct a st"d to determine hetherthe a''$ication of s" cha'ter ; of cha'ter of the Federa$ Food! Dr"g! and Cosmetic Act (re$ating todr"gs for rare diseases and conditions) and section > of the Interna$ Ee*en"e Code of 1B>3 (re$ating totax credit) to medica$ de*ices or medica$ foods for rare diseases or conditions or to oth is needed toenco"rage the de*e$o'ment of s"ch de*ices and foods4 The Secretar sha$$ re'ort the res"$ts of the st"dto the Committee on 5nerg and Commerce of the Ho"se of Ee'resentati*es and the Committee on%a or and H"man Eeso"rces of the Senate not $ater than one ear after the date of the enactment of this

Act4 For '"r'oses of this section! the term 0rare diseases or conditions0 has the meaning 'rescri ed section 2 of the r'han Dr"g Act ( 1 +4S4C4 /37ee)4

ORPHAN PROD CTS #OARD

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S5C4 9 6 /384 (a) There is esta $ished in the De'artment of Hea$th and H"man Ser*ices a oard for thede*e$o'ment of dr"gs (inc$"ding ;io$ogics) and de*ices (inc$"ding diagnostic 'rod"cts) for rare diseasesor conditions to e :no n as the r'han Prod"cts ;oard4 The ;oard sha$$ e com'rised of the AssistantSecretar for Hea$th of the De'artment of Hea$th and H"man Ser*ices and re'resentati*es! se$ected the Secretar ! of the Food and Dr"g Administration! the =ationa$ Instit"tes Hea$th! the Centers forDisease Contro$ and! an other Federa$ de'artment or agenc hich the Secretar determines hasacti*ities re$ating to dr"gs and de*ices for rare diseases or conditions4 The Assistant Secretar for Hea$thsha$$ chair the ;oard4

( ) The f"nction of the ;oard sha$$ e to 'romote the de*e$o'ment of dr"gs and de*ices for rare diseasesor conditions and the coordination among Federa$! other '" $ic! and 'ri*ate agencies in carr ing o"t theirres'ecti*e f"nctions re$ating to the de*e$o'ment of s"ch artic$es for s"ch diseases or conditions4

(c) In the case of dr"gs for rare diseases or conditions the ;oard sha$$---

(1) e*a$"ate---

(A) the effect of s" cha'ter ; of the Federa$ Food! Dr"g! and Cosmetic Act on the de*e$o'ment of s"chdr"gs! and

(;) the im'$ementation of s"ch s" cha'ter,

( ) e*a$"ate the acti*ities of the =ationa$ Instit"tes of Hea$th and the A$coho$! Dr"g A "se! and Genta$Hea$th Administration for the de*e$o'ment of dr"gs for s"ch diseases or conditions!

(/) ass"re a''ro'riate coordination among the Food and Dr"g Administration! the =ationa$ Instit"tes ofHea$th! the A$coho$! Dr"g A "se! and Genta$ Hea$th Administration! and the Centers for Disease Contro$in the carr ing o"t of their res'ecti*e f"nctions re$ating to the de*e$o'ment of dr"gs for s"ch diseases orconditions to ass"re that the acti*ities of each agenc are com'$ementar !

( ) ass"re a''ro'riate coordination among a$$ interested Federa$ agencies! man"fact"rers! and

organi@ations re'resenting 'atients! in their acti*ities re$ating to s"ch dr"gs!

(2) ith the consent of the s'onsor of a dr"g for a rare disease or condition exem't "nder section 272(i)of the Federa$ Food! Dr"g! and Cosmetic Act or reg"$ations iss"ed "nder s"ch section! inform 'h siciansand the '" $ic res'ecting the a*ai$a i$it of s"ch dr"g for s"ch disease or condition and inform 'h siciansand the '" $ic res'ecting the a*ai$a i$it of dr"gs a''ro*ed "nder section 272(c) of s"ch Act or $icensed"nder section /21 of this Act for rare diseases or conditions!

(3) see: "siness entities and others to "nderta:e the s'onsorshi' of dr"gs for rare diseases orconditions! see: in*estigators to faci$itate the de*e$o'ment of s"ch dr"gs! and see: "siness entities to'artici'ate in the distri "tion of s"ch dr"gs! and

(9) reorgani@e the efforts of '" $ic and 'ri*ate entities and indi*id"a$s in see:ing the de*e$o'ment ofdr"gs for rare diseases or conditions and in de*e$o'ing s"ch dr"gs4

(d) The ;oard sha$$ cons"$t ith interested 'ersons res'ecting the acti*ities of the ;oard "nder thissection and as 'art of s"ch cons"$tation sha$$ 'ro*ide the o''ort"nit for the s" mission of ora$ *ie s4

(e) The ;oard sha$$ s" mit to the Committee on %a or and H"man Eeso"rces of the Senate and theCommittee on 5nerg and Commerce of the Ho"se of Ee'resentati*es an ann"a$ re'ort---

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(1) identif ing the dr"gs hich ha*e een designated "nder section 2 3 of the Federa$ Food! Dr"g! andCosmetic Act for a rare disease or condition!

( ) descri ing the acti*ities of the ;oard! and

(/) containing the res"$ts of the e*a$"ations carried o"t the ;oard4

The Director of the =ationa$ Instit"tes of Hea$th and the Administrator of the A$coho$! Dr"g A "se! andGenta$ Hea$th Administration sha$$ s" mit to the ;oard for inc$"sion in the ann"a$ re'ort a re'ort on therare disease and condition research acti*ities of the Instit"tes of the =ationa$ Instit"tes of Hea$th and the

A$coho$! Dr"g A "se! and Genta$ Hea$th Administration, the Secretar of the Treas"r sha$$ s" mit to the;oard for inc$"sion in the ann"a$ re'ort a re'ort on the "se of the credit against tax 'ro*ided section

H of the Interna$ Ee*en"e Code of 1B2 , and the Secretar of Hea$th and H"man Ser*ices sha$$ s" mitto the ;oard for inc$"sion in the ann"a$ re'ort a re'ort on the 'rogram of assistance "nder section 2 of the

r'han Dr"g Act for the de*e$o'ment of dr"gs for rare diseases and conditions4 5ach ann"a$ re'ort sha$$e s" mitted "ne 1 of each ear for the 'receding ca$endar ear4

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