fda is not the problem: why undermining the drug approval process is not the answer to high drug...

8/19/2019 FDA Is Not the Problem: Why Undermining the Drug Approval Process Is Not the Answer to High Drug Prices

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1 Center for American Progress | The FDA Is Not the Problem

The FDA Is Not the ProblemWhy Undermining the Drug Approval ProcessIs Not the Answer to High Drug Prices

By Maura Calsyn and Thomas Huelskoetter March 9, 2016

Given he soaring cos s o prescrip ion drugs, i is no surprising ha more han 70 per-cen o Americans hink ha drug prices are “unreasonable” and ha“ drug companies

pu pro s be ore people.”1

Te pharmaceu ical indus ry and i s allies have respondedin par by rying o shif blame o he ederal Food and Drug Adminis ra ion, or FDA,claiming ha he reason drug prices remain high is because he FDA approval processis oo burdensome and slow, keeping compe ing drugs rom he marke and “s iing American innova ion.”2

Tis argumen is no jus misleading, i is dangerous. Te drug approval process needso s rike a care ul balance be ween speed and diligence pa ien s need sa e, effec ive

drugs, and i akes ime and clinical rials in order o de ermine whe her a drug mee shose s andards. oday, he FDA approves he overwhelming majori y o new drugs, and

does so a a quicker pace han any o her na ion. Fur hermore, ederal law already allowshe FDA o shor en bo h he clinical rial and approval processes or a large number odrugs in order o speed hem o marke . 3 Ye even or drugs deemed more innova ive orurgen ly needed, rade-offs persis ; drugs approved under hese expedi ed programs canla er be ound o have dangerous side effec s or be ar less effec ive han rs hough .

Despi e hese risks and he FDA’s exis ing au hori y o expedi e review or new rea men sin order o mee pa ien need, he pharmaceu ical indus ry con inues o push or policies

ha are designed o has en drug approvals.4 For example, he 21s Cen ury Cures Acincludes a number o provisions aimed a expanding he ypes o da a ha he FDA mayrely on when de ermining whe her a drug is sa e and effec ive.5 Since hese addi ional

ypes o da a are generally less rigorous, his would lead o more drugs en ering he marke wi h ar less in orma ion abou heir risks and evidence abou heir po en ial bene s.

Even i here were no concerns ha hese re orms increase risks o pa ien s, here is litlereason o believe ha pushing new branded drugs o marke would guaran ee meaning-

ul price reduc ions in he absence o generic compe i ion. Newly approved drugs enjoypa en and marke ing exclusivi y ha limi compe i ion, and manu ac urers con inue ose prices based on wha hey hink he marke will bear, no he drug’s value o pa ien s.


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Tis issue brie rs will describe how drugs are developed and approved in he Uni edS a es. I hen will explore he reasons why undermining he FDA’s au hori y and speed-ing up he drug developmen and approval process will harm pa ien s and do no hing olower drug prices.

Drug development and approval processes

oday, he FDA’s approval process is, on average, as er han ha o any o her majorna ion.6 Te FDA is as er han bo h he European Union and Japan no only in heoverall average approval ime or new drugs bu also in he average ime or every majorca egory o drug.7 Similarly, s udies comparing he FDA wi h Canadian drug regula orshave ound ha he FDA consis en ly approves drugs more quickly.8 In 2015, 64 perceno innova ive new drugs were approved by he FDA be ore hey received approval in anyo her coun ry in he world.9

In response, he pharmaceu ical indus ry and i s allies poin ou ha he ac ual regula oryreview ime is jus one par o he drug developmen and approval process. Some cri icsargue ha he pre-FDA process o drug developmen and es ing including he variouss ages o clinical rials akes oo long and cos s oo much, hus s iing innova ion.10

I is rue ha bringing a new prescrip ion drug o marke is a ime-consuming, expen-sive, and risky process or drug manu ac urers. Bu he FDA approval requiremen s exis

o ensure ha new drugs are sa e and o prove ha hey ac ually work. Te curren U.S.drug approval process has evolved as policymakers have weighed compe ing concernsincluding he signican nancial inves men ha drug manu ac urers make hroughou

he clinical rial and approval processes, he need or drugs o be sa e and effec ive, andhe need o speed a drug o marke in cases where here are gaps in rea men s.

Standard drug development and the FDA approval process

For all drugs, he pharmaceu ical indus ry generally uses he ndings rom basicresearch, which s udies he mechanisms o diseases, as a s ar ing poin or i s appliedresearch and developmen effor s. Basic research is unded in large par by he ederalgovernmen and conduc ed by researchers a he Na ional Ins i u es o Heal h and inacademic labora ories.11

Te rs s ep o indus ry-sponsored research is labora ory and animal es ing o evalua e ihe inves iga ional use o he new produc in people is reasonably sa e.12 Te FDA reviewshese ndings, and i i agrees ha he resul s show ha he produc is reasonably sa e or

people, he company may move orward wi h clinical rials, which es he drug in pa ien s.


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Tese clinical rials occur in several phases, wi h each phase es ing he drug in increas-ingly larger groups o pa ien s in order o ga her in orma ion abou he produc ’s sa e yand effec iveness, as well as side effec s, dosing, and in erac ions wi h o her drugs, ood,and drinks.13 Phase 1 s udies, which ypically consis o 20 o 80 heal hy volun eers, s udy

he drug’s oxici y and sa e y, as well as how people process he drug. I he drug is nounaccep ably oxic, Phase 2 s udies ollow. Tese s udies s ar o consider he drug’s effec-

iveness by collec ing preliminary da a on how he drug works in pa ien s wi h specicdiseases or condi ions, while con inuing o s udy he drug’s sa e y and side effec s. Tenumber o people enrolled in a Phase 2 s udy can range rom a ew dozen o abou 300.

I he Phase 2 s udies show evidence o effec iveness, he drug manu ac urer and heFDA will ry o agree o he design o he Phase 3 s udy. Te FDA moni ors clinical rials

hroughou heir en ire dura ion, bu he pre-Phase 3 period is one o he mos commonmee ing poin s hroughou he clinical rial process. Phase 3 s udies collec addi ionalin orma ion abou sa e y and effec iveness and can s udy he drug’s effec in differenpopula ions, a differen dosages, and when combined wi h o her drugs. Tese s udies

ypically range in size rom several hundred o abou 3,000 people.

When he company believes ha hese s udies offer enough evidence o show hahe produc is sa e and effec ive, i will seek he FDA’s approval by submiting a New

Drug Applica ion, or NDA, ha includes all preclinical and clinical rial da a, as wellas in orma ion abou how he drug works in he body and how i is manu ac ured.14 Iis also common or manu ac urers and he FDA o mee a his poin , righ be ore anNDA is submited.

Afer he NDA submission, he FDA has 60 days o decide whe her he applica ion is

comple e and ready or review. Te Prescrip ion Drug User Fee Ac , or PDUFA se sreview imelines or he FDA, and he FDA’s goal is o review and ac on a leas 90 per-cen o NDAs or drugs under he s andard review process no la er han 10 mon hs afer

he applica ions are received.15

Te FDA approves a drug or marke ing and sale i he da a show ha he drug is sa eand effec ive in i s proposed use, and i i s bene s ou weigh he known risks.16 Federallaw requires “subs an ial evidence” ha he drug is sa e and effec ive.17 Subs an ialevidence means, in par , “adequa e and well-con rolled inves iga ions, including clinical

rials.”18 As FDA explains, he “NDA is supposed o ell he drug’s whole s ory, including wha happened during he clinical es s, wha he ingredien s o he drug are, he resul so he animal s udies, how he drug behaves in he body, and how i is manu ac ured,processed and packaged.”19 As a condi ion o approval, he FDA may require he manu-

ac urer o conduc addi ional pos marke s udies o con inue o ga her da a abou hedrug’s sa e y and effec iveness.


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Expedited programs

Tere are a number o FDA programs ha policymakers have designed o speed hedevelopmen and approval o new drugs ha “address unme medical need in he rea -men o a serious or li e- hrea ening condi ion.”20 Te our principal programs are as

rack designa ion, break hrough herapy designa ion, accelera ed approval, and priori y

review.21 Each program expedi es drug approvals or serious condi ions, bu each hasdifferen quali ying cri eria and approval requiremen s. (see able 1)

According o he FDA, hese pa hways “help ensure ha herapies or serious condi ionsare approved and available o pa ien s as soon as i can be concluded ha he herapies’ bene s jus i y heir risks.”22 Tese pa hways are o par icular impor ance when here are

ew or no rea men op ions, and hey are par icularly help ul “in setings in whichhe disease course is long and an ex ended period o ime would be required o measurehe in ended clinical bene s o a drug.”23 Be ween 2000 and 2013, 32 percen o new

molecular produc s, including biologics, which are drugs made rom living cells, were

approved under he accelera ed approval and as rack pa hways.24

Al hough he FDA’s s andard review ime is abou 12 mon hs, hese programs speed uphe review process.25 More impor an ly, however, he drug developmen process can be

shor ened in hree o hese our programs, because he FDA can make i s de ermina-ion o he drug’s sa e y and effec iveness based on limi ed clinical da a. Shor ening he

clinical rial period speeds cri ical new drugs o marke , bu i comes wi h a risk, becausehere is limi ed da a o prove heir sa e y and effec iveness.

Under he as rack designa ion, or example, he FDA may approve a drug based on da a

rom a single Phase 2 s udy.26

And he accelera ed approval pa hway allows approval basedon surroga e endpoin s ha are reasonably likely o predic pa ien ou comes.27 Surroga eendpoin s are markers such as labora ory resul s or radiology images, while clinical end-poin s measure he reduc ion in symp oms or mor ali y.28 For cancer rea men s, surroga eendpoin s used o approve drugs include a shrinking umor or lower biomarker levels,ins ead o he clinical endpoin s o longer survival or improved quali y o li e.

Surroga e endpoin s can be measured sooner, allowing pa ien s access o new rea -men s much as er, bu hey are no always accura e indica ors o how well a rea menmay work, especially in a larger real world popula ion.29 As one s udy no ed, “drugs haare approved afer a shor ened premarke period or based on … [limi ed da a] may la er be ound o have grea er risks or less cer ain bene s han ini ially believed o be hecase.”30 For his reason, drugs approved using limi ed da a are hen subjec o pos ap-proval es ing o ul ima ely conrm ha hey are in ac sa e and effec ive.31


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TABLE 1

The FDA’s expedited programs

Program name Qualifying criteria Features

Fast track Treats a serious medical condition and has thepotential to address unmet medical need

Acts to expedite development and review, withapproval possible after a single Phase 2 study;rolling review

Priority reviewIncludes drugs that would provide a signicant

improvement in safety or effectiveness

Has shortened FDA review time of four months;

can be combined with other expedited programs

Accelerated approvalTreats a serious condition that generally providesa meaningful advantage over available therapies

Can approve on the basis of a surrogate or interme-diate endpoint that is reasonably likely to predict aclinical benet

Breakthrough therapyTreats a serous condition; preliminary clincialevidence indicates that the drug may be asubstantial improvement over existing therapies

Has all fast track features; intensive guidance onefficient drug development

Sources: Food and Drug Administration, Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics (U.S. Department of Health and Human Services,2014), available at http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf; Trinia Cain and Stephanie Shapley, “ExpeditedPrograms for Serious Conditions Drugs and Biologics (Draft Guidance)” (Silver Spring, MD: U.S. Food and Drug Administration, 2013), available at http://www.fda.gov/downloads/ Drugs/UCM363903.pdf; Aaron S. Kesselheim and others, “Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study,” British MedicalJournal 351 (2015).

Drug approval rate

Under he curren regula ory s ruc ure, he FDA approves almos every new drug appli-ca ion i receives. In 2015 and 2014, he FDA approved 89 percen and 100 percen ,respec ively, o novel drug applica ions, which are dened as more innova ive NDAs

ha involve new molecular en i ies.32 While no unpreceden ed, his is a higher approvalra e han he FDA generally has produced in he pas ; in 2007 and 2008, or example,

he FDA approved 51 percen and 71 percen o novel drug applica ions, respec ively.33

O her sources conrm he FDA’s numbers. Forbes commissioned an analysis o FDAapprovals rom BioMed racker and concluded ha “ he FDA is basically approvingevery hing.”34 In o her words, no only are he FDA’s s andards no overly harsh, hey arecurren ly unusually lenien .

Undermining the FDA approval processwill benefit the drug industry, not patients

Given he speed and approval ra e o he FDA, here is no need o ur her expedi e hedrug approval process. Tis approach would lead o addi ional drugs en ering he marke wi h litle evidence o suppor heir sa e y and effec iveness, which can harm pa ien s. In

ac , growing numbers o exper s including a ormer FDA commissioner have warnedha weakening he FDA’s approval s andards by changing he ypes o evidence ha he

FDA reviews when approving drugs would pu pa ien s a risk.35


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Never heless, many lawmakers suppor he 21s Cen ury Cures Ac and o her similarproposals.36 As no ed previously, he 21s Cen ury Cures Ac includes a number o rou- bling policies ha would give FDA he au hori y o nd a drug sa e and effec ive on he basis o less scien ically rigorous evidence.37

For example, he legisla ion lays he groundwork or FDA o use broader ca egories o

evidence rela ed o “clinical experience,” which includes “observa ional s udies, regis-ries, and herapeu ic use” ins ead o randomized con rolled rials or approving new

uses or exis ing drugs or o sa is y pos -approval s udy requiremen s.38 Bu as exper scau ion, “al hough such da a can provide impor an in orma ion abou drug u iliza-

ion and sa e y once a medica ion is in use, here is considerable evidence ha heseapproaches are no as rigorous or valid as randomized rials in assessing efficacy.”39 Ano her sec ion o he bill au horizes he FDA o approve new an ibio ics and an i un-gal medicines in ended o rea serious or li e- hrea ening in ec ions in cer ain pa ien s based on limi ed da a, including preclinical rials.40 Moreover, he legisla ion crea esa nancial incen ive or hospi als o use hese largely un es ed drugs: For each pa ien

rea ed wi h hese newly approved drugs, he hospi al receives an add-on paymen .41

Despi e he signican limi a ions o surroga e endpoin s, he legisla ion also encour-ages he FDA o expand i s use o hem o assess a drug’s sa e y and effec iveness. TeFDA, however, already has he au hori y o use hese da a when i is mos appropri-a e: when he drug is in ended o rea a serious or li e- hrea ening condi ion.42I alsogives indus ry exper s an increased role in de ermining when i is appropria e o use

hese surroga e endpoin s.43

Ul ima ely, however, here is no evidence ha changing hese s andards would speed

he approval o game-changing, ruly innova ive new produc s, nor is here any evidenceha less rigorous review would lower drug prices.

Approving drugs with less evidence would put patients at risk

Te curren expedi ed programs already place a premium on speed o approval. Whendrugs are approved using an expedi ed pa hway, i comes wi h a rade-off: less in orma-

ion and da a abou how he drug ac s in a pa ien ’s body.

In si ua ions where here are ew exis ing rea men s or where a new rea men can sig-nican ly improve pa ien ou comes, speed is a priori y. Bu i he drugs being approvedare less urgen ly needed or offer litle o no improvemen over exis ing rea men s, heneed o priori ize speed over sa e y diminishes. In such cases, he bene s are muchless likely o ou weigh he risks o pa ien sa e y ha are inheren in permiting drugs oen er he marke afer shor ened clinical rials or based on limi ed da a.44


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Evidence suppor s his concern. For example, researchers have ound ha since 1992, when policymakers adop ed he priori y review and accelera ed approval programs,

he number o approved prescrip ion drugs ha received black-box warnings whichis he mos serious sa e y warning ha he FDA can impose on a drug or ha were wi hdrawn rom he marke or sa e y-rela ed reasons has increased 25 percen .45 Teresearchers sugges ha one reason or his increase is ha growing numbers o new

drugs are en ering he marke wi h more limi ed da a abou heir sa e y and efficacy.46

No ably, he Governmen Accoun abili y Office has already raised concerns ha heFDA lacks sufficien da a o horoughly moni or he sa e y o new drugs afer hey have been approved especially or drugs ha are approved under an expedi ed pa hway, where pos approval oversigh is crucial due o he abbrevia ed clinical rial process.47

Tis worry will become even more signican in he coming years, because an increas-ing number o drugs are being channeled in o expedi ed programs. In 2014, more han60 percen o approved drugs were approved by he FDA based on reduced da a and

evidence requiremen s.48

Te FDA now approves more han wo- hirds o drugs basedon da a rom s udies las ing six mon hs or less.49Moreover, one- hird o new drugs areapproved on he basis o a single rial, and he median size or all such rials is jus more

han 750 pa ien s.50 Te FDA is also approving an increasing number o supplemen alnew drug applica ions or addi ional clinical uses or pa ien popula ions includingchildren using surroga e endpoin s and limi ed da a.51 Some o hese addi ional usesmigh be ruly innova ive or he newly approved uses or pa ien groups and allow drugcompanies o marke hese drugs o popula ions in need o new rea men op ions; as

he s udy’s au hors no e, however, hese ndings once again demons ra e “ he impor-ance o pos -approval surveillance o drugs’ supplemen al indica ions, par icularly

hose ha expand he eligible pa ien popula ion.”52

Current FDA requirements do not stifle innovation

Under oday’s expedi ed programs, ruly rans orma ive drugs can reach pa ien squickly. Gleevec, which has been described as a “miracle drug” ha can urn deadlycancers in o chronic condi ions, is an excellen example o how well his process can work.53 Gleevec was rs approved in 2001 or he rea men o chronic myelogenousleukemia a rare blood cancer afer only Phase 2 s udies and jus 2.5 mon hs o FDAreview.54 Since i s ini ial approval, he FDA has approved addi ional uses o he drug o

rea several differen gas roin es inal umors, and pos -marke ing s udies have con-rmed i s effec iveness.55

Gleevec’s approval shows how he FDA can ac quickly o approve ruly rans orma-ive produc s. Bu no all drugs ha go hrough expedi ed programs are similar game-

changers. A s udy by researchers a Harvard Medical School and Brigham and Women’sHospi al ound ha rom 1987 o 2013, he number o drugs ha he FDA approved


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under expedi ed programs increased 2.6 percen each year. Ye drugs ha were no “rsin class” drove his rend – meaning he drugs ollowed unc ionally similar produc s.56 Te s udy au hors concluded ha al hough “some drugs associa ed wi h an expedi edprogram may indeed provide no iceable clinical advances, his rend is being driven bydrugs ha are no rs in class and hus po en ially less innova ive.”57

Skewing he process oward grea er speed is simply no necessary.58 And i is illogicalo hink ha such changes will necessarily lead o u ure break hroughs like Gleevec.

On he con rary, Gleevec shows ha he curren process works o expedi e innova ivedrugs; effor s o allow more drugs on he marke wi h even lower evidence o sa e y andeffec iveness will pu pa ien s a risk unnecessarily.

Speeding drugs to market will not necessarily lower prices

Proponen s o loosening he FDA’s requiremen s ofen argue ha speeding he drug

approval process will help reduce prices. Ye here is litle indica ion ha pas policy changeso he FDA have had his effec ; a he same ime as he FDA has approved an increasing

number o drugs hrough expedi ed pa hways, drug prices have con inued o rise.59

Par o he ension in his argumen is ha ruly break hrough produc s ha represensignican advances or address urgen medical needs he precise drugs ha should beapproved hrough expedi ed pa hways based on more limi ed in orma ion generally by deni ion have no real compe i ion or price cons rain s afer en ering he marke .Once again, Gleevec is a good example o he disconnec be ween expedi ed approvalsand price. When he drug was rs approved, i s manu ac urer, Novar is, charged abou

$4,500 in 2014 dollars or one mon h o rea men . In 2014, ha price had gone up onearly $8,500 per mon h.60

I is rue ha speeding compe i or drugs o marke can help reduce prices in somecases. Branded compe i ion can give payers addi ional leverage o nego ia e sizablediscoun s or limi price increases by ying drug coverage o price. For example, insur-ers can encourage demand or a specic drug by lowering i s cos -sharing in exchange

or price discoun s rom he manu ac urer.61 Bu each si ua ion differs based on hesimilari y o he compe ing drugs, he pricing s ra egies o he drug companies, ando her available rea men op ions.62

A recen example is ha o high-priced new cures or Hepa i is C, when Viekira Pak ol-lowed Sovaldi o marke only a year la er. Te pharmacy bene manager Express Scrip s was able o nego ia e a large discoun by making a deal wi h Viekira Pak’s manu ac urer, AbbVie, o s op covering Sovaldi and exclusively cover Viekira Pak once he second drughi he marke .63 However, he enormous amoun o media aten ion his received dem-ons ra es he rela ive rari y o such high-prole arrangemen s. And al hough Express


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Scrip s managed o receive a signican discoun , Viekira Pak’s lis price s ill closelyracked ha o Sovaldi demons ra ing ha branded compe i ion does no necessarily

correc or an excessive original price, since he original price ofen se s he price s ar ingpoin or u ure compe i ors.64

No ably, however, Sovaldi and i s compe i ors received expedi ed approval rom he

FDA, which demons ra ed ha he agency can already ac quickly o bo h expedi e break hroughs and help increase compe i ion.65 Te FDA is no he barrier in suchsi ua ions; how quickly branded compe i ion can appear depends primarily on whe hercomparable drugs are already in he developmen pipeline. Fur hermore, he FDAla er required AbbVie o add in orma ion o i s label abou serious liver injury risk wi h Viekira Pak* demons ra ing once again he rade-offs be ween speed and pa iensa e y, even or innova ive cures.66

Ye , while in eres ing, he Sovaldi example is no represen a ive o all drugs. Hepa i is C was an unusual si ua ion in which an ex remely high-priced break hrough drug was ol-

lowed rela ively quickly by mul iple compe i ors ha had been close behind he originalin he developmen process. Fur hermore, ou rage over he high price o he originaldrug, Sovaldi, opened oppor uni ies or compe i ors o s rike exclusive deals wi h majorpayers who were growing despera e.67

For every example o a new drug spurring price compe i ion, here is ano her o pharma-ceu ical companies aking advan age o shifing marke dynamics in order o price heircompe i or drugs higher han he original drugs.68 S udies have ound ha in he absenceo generic compe i ion, increases in branded compe i ion ofen only resul in marginalprice reduc ions or as he ollowing examples illus ra e, even price increases.69

For ins ance, al hough 11 major drug al erna ives o rea mul iple sclerosis have en eredhe marke over he pas wo decades, all o hem are priced in roughly he same high-cos

range.70 Tese manu ac urers have no atemp ed o undercu each o her’s prices in ordero gain marke share. Ra her, a s udy looking a nine o hese drugs ound ha each ime

a new drug al erna ive has en ered he marke wi h a higher price, he manu ac urers ohe older drugs have raised heir prices o ma ch he new drug’s price.71 Four o he oldes

drugs were originally priced 230 percen o 380 percen lower han hey are now.72

Tis s unning “shadow pricing,” in which drug compe i ors mirror each o her’s priceincreases, also exis s or o her drugs. 73 Te prices or biologic rheuma oid ar hri is drugshave all risen signican ly as a group over he pas ew years.74 For example, he manu-

ac urers o wo rheuma oid ar hri is drugs, Enbrel and Humira, bo h raised heir pricesin parallel by similar double-digi percen ages in 2014 and 2013.75 Similarly, he insulinmarke has seen compe i ors raising heir prices in andem, ofen wi hin days o eacho her; as a resul , mul iple brands o insulin saw price increases o 160 percen o 400percen be ween 2007 and 2014.76


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In si ua ions like hese, monopoly pro ec ion and he manu ac urers’ knowledge hahey ace no ex ernal limi s on price appear o scramble he normal marke calcula ions,

wi h manu ac urers raising prices o maximize pro s as much as possible be ore heylose heir marke ing exclusivi y and ace generic compe i ion. Essen ially, manu ac urersare aware ha demand or heir drugs is rela ively inelas ic: I hey all s ick oge her onhigh prices, pa ien s who need hese drugs will have nowhere else o urn.

Tis is no normal marke behavior. And i should make clear ha here is no normal,unc ioning marke or prescrip ion drugs. As a resul , increasing branded compe i ion

alone will no guaran ee price reduc ions.

Tere is no evidence ha shor ening he clinical rial or drug approval process willchange hese pharmaceu ical indus ry behaviors. And while prices con inue o rise,pa ien s will be pu a risk by he approval o drugs based on limi ed evidence.

The generic backlogCritics of the FDA commonly point to the agency’s backlog of generic

drug applications as their primary example of how the agency allegedly

holds back competition. Yet while the backlog is a problem for generic

drugs, no such backlog exists for brand name drugs. Often, the FDA’s de-

tractors conate these two separate issues, which overstates the impact

of the backlog on drug approval and price ination. Although generic

drug price spikes harm patients and must be addressed, they have not

been widespread enough to be a central contributor to recent increases

in national drug spending; a recent analysis by the U.S. Department ofHealth and Human Services concluded that “they exert no sizable inu-

ence on overall drug spending.” 77

Furthermore, evidence suggests that the backlog is not even the primary

factor in generic drug price ination. Overall, 10 percent of all drugs have

expired market exclusivity but lack generic drug applications submitted to

the FDA, compared with the 2 percent that have submitted applications and

are awaiting FDA approval.78 In other words, for more than 80 percent of off-

patent drugs that lack generic competition, no generic manufacturer has

submitted an application to the FDA. This suggests that lack of competition

in the generic drug market—which leads to monopolies and price hikes—is

primarily due to market dynamics that are unrelated to the FDA, such as

mergers of generic manufacturers, drug shortages resulting from supply

chain issues, and efforts to limit distribution through specialty pharmacies. 79

That being said, the FDA’s backlog of new generic drug applications is

a signicant problem that has existed for several years. 80 In 2012, the

Generic Drug User Fee Amendments, or GDUFA, authorized the FDA

to collect fees from generic drug manufacturers, in part to nance an

acceleration of the generic approval process. 81 Yet despite the GDUFA

the backlog continues today, numbering roughly 4,300 at the end of

2015.82 Although most of the applications from the original backlog

have been cleared by now, the volume of new applications over the past

two years has been much higher than anticipated. The estimates used

when developing GDUFA—and providing funding for the FDA’s OfficeGeneric Drugs—were for 750 new generic drug applications annually.

In practice, however, the actual volume of applications since 2012 has

dwarfed these estimates, leaving the Office of Generic Drugs under-

funded and understaffed to process them. The FDA received more than

1,400 new generic drug applications in 2014 and received around 1,000

in both 2012 and 2013. 84

The FDA does use some criteria to prioritize applications in order to help

manage the backlog, including whether an application is a rst generic or

whether a drug shortage exists. 85 However, the FDA is not currently prio

tizing based on the prices of existing drugs, or on whether those prices ar

increasing faster than ination. 86 Prioritizing on these factors could help

maximize the impact of limited resources and reduce the potential impact

of the backlog on generic drug prices. 87


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Conclusion

oday, here is enough exibili y in he FDA approval process o bring li esaving drugso marke in a imely process. And i policymakers are willing o increase unding or he

FDA o review drug applica ions more quickly, more drugs could en er he marke aferappropria e veting. Cuting corners by pushing he FDA o approve more drugs on he

basis o more limi ed and less rigorous da a, however, will simply pu more pa ien s arisk and do no hing o lower drug prices.

Maura Calsyn is he Direc or of Heal h Policy a he Cen er for American Progress. TomasHuelskoeter is he Research Associa e for Heal h Policy a he Cen er.

* Correction, March 11, 2016: Tis issue brief incorrec ly s a ed he ype of label changerequired by he FDA. o clarify, he FDA issued a safe y announcemen and required hemanufac urer of Viekira Pak o add informa ion o i s label warning ha he drug can causeserious liver injury mos ly in pa ien s wi h underlying advanced liver disease.


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Endnotes

1 Bianca DiJulio, Jamie Firth, and Mollyann Brodie, “KaiserHealth Tracking Poll: August 2015,” The Henry J. Kai serFamily Foundation, August 20, 2015, available at http://kff.org/health-costs/poll-nding/kaiser-health-tracking-poll-august-2015/.

2 For one example of such a statement, see Rep. JasonChaffetz’s (R-UT) quote in Bronwyn Mixter, “At Drug PriceHearing, FDA’s Generic Work Critici zed,” Bloomberg BNAHealth Care Blog, February 5, 2016, available at http://www.bna.com/drug-price-hearing-b57982067047/: “I believe theFDA has failed to meet its statutory responsibilities . . . Ifsomebody increases the price of a prescription drug, that’sgoing to invite more competition, but if that competitioncan’t get approval from the FDA, there will be no competi-tion”; See also Congressman Fred Upton, “Upton Suppor tsRegulatory Reform to Keep Medical Device IndustryCompetitive, Protect Michigan Jobs,” Press release, July 20,2011, available at http://upton.house.gov/news/documen-tsingle.aspx?DocumentID=252907: “The overly burdensomeregulations that are stiing American innovation and jobcreation must end.”.

3 U.S. Food and Drug Administration, Guidance for Industry:Expedited Programs for Serious Conditions—Drugs andBiologics (U.S. Department of Health and Human Services,2014), available at http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/

ucm358301.pdf.

4 21st Century Cures Act , H.R. 6, 114th Cong., 1st sess. (May 19,2015).

5 Ibid.

6 Jerry Avorn and Aaron Kesselheim, “The 21st Century CuresAct—Will It Take Us Back in Time?”The New England Journalof Medicine 372 (26) (2015): 2473-2475; Magdalena Bujarand Neil McAuslane, “New Drug Approvals in ICH Countries2004-2013” (London: Centre for Innovation in RegulatoryScience, 2014), available at http://cirsci.org/publications/CIRS_R&D_Brieng_54_%20ICH_approval_times_2004-2013_22apr2014.pdf.

7 Bujar and McAuslane, “New Drug Approvals in ICH Countries2004-2013.”

8 Nicholas S. Downing and others, “Regulatory Review of

Novel Therapeutics—Comparison of Three RegulatoryAgencies,” The New England Journal of Medicine 366 (24)(2012): 2284-2293.

9 U.S. Food and Drug Administration, Novel Drugs Summary2015, 5th ed. (Washington: U.S. Department of Health andHuman Services, 2015).

10 Sen. Richard Burr and Sen. Tom Coburn, “Caution kills,”TheWashington Times , February 15, 2011, available at http://www.washingtontimes.com/news/2011/feb/15/caution-kills/.

11 National Science Board, “A Companion to Science andEngineering Indicators 2008,” available at http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141 (last accessed March 2016).

12 U.S. Food and Drug Administration, “Investigational NewDrug (IND) Application,” available at http://www.fda.gov/drugs/developmentapprovalprocess/howdrugsaredevel-opedandapproved/approvalapplications/investigation-alnewdrugindapplication/default.htm (last accessed July2015).

13 U.S. Food and Drug Administration, “The FDA’s Drug ReviewProcess: Ensuring Drugs Are Safe and Effective,” availableat http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htm (last accessed March 2016).

14 U.S. Food and Drug Administration, “New Drug Application(NDA),” available at http://www.fda.gov/Drugs/Develop-mentApprovalProcess/HowDrugsareDevelopedandAp-proved/ApprovalApplications/NewDrugApplicationNDA/ (last accessed February 2015).

15 U.S. Food and Drug Administration,PDUFA ReauthorizationPerformance Goals and Procedures Fiscal Years 2013 Through2017 (U.S. Department of Health and Human Services,2012), available at http://www.fda.gov/downloads/ForIn-dustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.

16 The manufacturer must also demonstrate (1) that the drug’sproposed labeling (package insert) is appropriate, and whatit should contain, and (2) that the methods used in manu-facturing the drug and the controls used to maintain thedrug’s quality are adequate to preserve the drug’s identity,strength, quality, and purity. For more information, see:U.S. Food and Drug Administration, “New Drug Application(NDA).”

17 Federal Food, Drug, and Cosmetic Act , Public Law 75-717,75th Cong., 3d. sess., (June 25, 1938), Title 21, section 505(c)and (d).

18 Ibid., section 355(d); John Farley, “The Substantial EvidenceStandard, Endpoints, and O ther Regulatory Considerations,”(U.S. Food and Drug Administration, 2014), available athttp://www.fda.gov/downloads/Drugs/NewsEvents/UCM412711.pdf .

19 U.S. Food and Drug Administration, “New Drug Application(NDA).”

20 U.S. Food and Drug Administration, Guidance for Industry:Expedited Programs for Serious Conditions—Drugs andBiologics.

21 The goal for priority review is six months, although it gener-ally takes eight months when the ling period is included.Priority review also shortens the clinical trial period. First,FDA may approve drugs intended to treat life-threateningor severely debilitating conditions under “fast track” ap-proval based on early evidence of efficacy alone. For moreinformation, see U.S. Food and Drug Administration, “Fast Track,” available at http://www.fda.gov/ForPatients/Approv-als/Fast/ucm405399.htm (last accessed July 2015). Second,under the accelerated approval pathway, FDA approvedrugs intended to treat serious or life -threatening condi-tions if the drug is “reasonably likely” to provide meaningfultherapeutic benet not provided by existing treatments. Formore information, see U.S. Food and Drug Administration,“Accelerated Approval,” available at http://www.fda.gov/ForPatients/Approvals/Fast/ucm405447.htm (last accessed

July 2015). Between 2000 and 2013, 32 percent of newmolecular products, including biologics, were approvedunder these two pathways. For more information, see AaronS. Kesselheim and o thers, “Existing FDA Pathways HavePotential to Ensure Early Access to, and Appropriate Use of,Specialty Drugs,”Health Affairs 33 (10) (2014): 1770-1778;U.S. Food and Drug Administration, “Breakthrough Therapy,”available at http://www.fda.gov/ForPatients/Approvals/Fast/ucm405397.htm (last accessed July 2015). Third, com-panies may apply for a “breakthrough” therapy designationfor drugs that show early signs of clinical promise. In 2013,FDA received about 100 applications for this designation. For more information, see Aaron S. Kesselheim, and o thers,“Existing FDA Pathways Have Potential to Ensure Early Ac-cess to, and Appropriate Use of, Specialty Drugs.”

22 U.S. Food and Drug Administration, Guidance for Industry:Expedited Programs for Serious Conditions—Drugs andBiologics.

23 Ibid.

24 Kesselheim and others, “Existing FDA Pathways HavePotential to Ensure Early Access to, and Appropriate Use of,Specialty Drugs.”

25 The standard review goal for the FDA is 10 months. Whilethe agency generally meets this goal, the clock begins aftera 60-day ling period, so the total time is about 12 monthsfrom application. See U.S. Food and Drug Administration,“The FDA’s Drug Review Process: Ensuring Drugs Are Safeand Effective,” available at http://www.fda.gov/Drugs/Re-sourcesForYou/Consumers/ucm143534.htm (last accessedSeptember 2015).

http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/http://www.bna.com/drug-price-hearing-b57982067047/http://www.bna.com/drug-price-hearing-b57982067047/http://upton.house.gov/news/documentsingle.aspx?DocumentID=252907http://upton.house.gov/news/documentsingle.aspx?DocumentID=252907http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htmhttp://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htmhttp://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/downloads/Drugs/NewsEvents/UCM412711.pdfhttp://www.fda.gov/downloads/Drugs/NewsEvents/UCM412711.pdfhttp://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143534.htmhttp://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143534.htmhttp://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143534.htmhttp://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143534.htmhttp://www.fda.gov/downloads/Drugs/NewsEvents/UCM412711.pdfhttp://www.fda.gov/downloads/Drugs/NewsEvents/UCM412711.pdfhttp://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htmhttp://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htmhttp://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.nsf.gov/statistics/nsb0803/start.htm?CFID=18933716&CFTOKEN=37176006&jsessionid=f0306881bd4a0bb7ffd3b5e2e63c535b7141http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://www.washingtontimes.com/news/2011/feb/15/caution-kills/http://upton.house.gov/news/documentsingle.aspx?DocumentID=252907http://upton.house.gov/news/documentsingle.aspx?DocumentID=252907http://www.bna.com/drug-price-hearing-b57982067047/http://www.bna.com/drug-price-hearing-b57982067047/http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/http://kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-august-2015/


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26 Aaron S. Kesselheim and others, “Trends in utilization of FDAexpedited drug development and approval programs, 1987-2014: cohort study,” The BMJ 351 (4633) (2015): 1-7.

27 U.S. Food and Drug Administration,Guidance for Industry:Expedited Programs for Serious Conditions—Drugs andBiologics.

28 Kesselheim and others, “Trends in utilization of FDAexpedited drug development and approval programs, 1987-2014: cohort study.”

29 National Cancer Institute, “NCI Dictionary of Cancer Terms,”

available at http://www.cancer.gov/publications/diction-aries/cancer-terms?cdrid=729831 (last accessed March2016); Kesselheim and others, “Existing FDA PathwaysHave Potential to Ensure Early Access to, and AppropriateUse of, Specialty Drugs”; Carolyn Y. Johnson, “Politicianswant to speed up drug approvals. That cou ld backre,” TheWashington Post , November 24, 2015, available at https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backre/ .


31 Ibid.

32 U.S. Food and Drug Administration, “Novel Drugs Summary2015” (U.S. Department of Health and Human Services,2016), available at http://www.fda.gov/Drugs/Developmen-

tApprovalProcess/DrugInnovation/ucm474696.htm. 33 Ibid.

34 Matthew Herper, “The FDA Is Basically Approving Every-thing. Here’s The Data to Prove It,” Forbes , August 20, 2015,available at http://www.forbes.com/sites/matthewher-per/2015/08/20/the-fda-is-basically-approving-everything-heres-the-data-to-prove-it.

35 Gregg Gonsalves, Mark Harrington, and David A. Kessler,“Don’t Weaken the F.D.A.’s Drug Approval Process,”The NewYork Times, June 11, 2015, available at http://www.nytimes.com/2015/06/11/opinion/dont-weaken-the-fdas-drug-approval-process.html.

36 21st Century Cures Act ; Sheila Kaplan, “With comprehensivelegislation stalled, Senate panel clears 7 smaller biomedbills,” STAT News, February 9, 2016, available at http://www.statnews.com/2016/02/09/senate-biomedical-bills/.

37 Gonsalves, Harrington, and Kessler, “Don’t Weaken theF.D.A.’s Drug Approval Process.”

38 21st Century Cures Act , sec. 2061; In addition, the 21stCentury Cures Act would lower the level of evidence thatis required for the FDA to approve medical devices as safeand effective, despite the fact that devices are alreadygenerally approved based upon weaker evidence thannew drugs. For more information, see Rita F. Redberg andSanket S. Dhruva, “The F.D.A.’s Medical Device Problem,”The New York Times, July 17, 2015, available at http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0 .

39 Avorn and Kesselheim, “The 21st Century Cures Act—Will It Take Us Back in Time?”

40 21st Century Cures Act , sec. 2121.

41 Ibid., sec. 2123.

42 21st Century Cures Act ; , Julia Belluz, “This new bill would add$9 billion for medical research. Here are 5 reasons critics areterried,” Vox, July 14, 2015, availab le at http://www.vox.com/2015/7/14/8961923/21st-century-cures-act.

43 21st Century Cures Act , sec. 2021 and 2022.


45 Cassie Frank and others, “Era of Faster FDA Drug ApprovalHas Also Seen Increased Black-Box Warnings And MarketWithdrawals,” Health Affairs 33 (8) (2014):1453-1459; LaraMaggs and Aaron Kesselheim, “The Role Of Black BoxWarnings In Safe Prescribing Practices,” Health Affairs Blog,August 20, 2014, available at http://healthaffairs.org/blog/2014/08/20/the-role-of-black-box-warnings-in-safe-prescribing-practices/.

46 Ibid.

47 Government Accountability Office, “FDA Expedites ManyApplications, But Data for Postapproval Oversight Need

Improvement, GAO-16-192, Report to the Ranking MemberSubcommittee on Labor, Health, and Human Services,Education and Related Agencies, Committee on Appropria-tions, House of Representatives, December 2015, availableat http://www.gao.gov/assets/680/674183.pdf.

48 Rita F. Redberg, “Faster Drug Approvals Are Not AlwaysBetter and Can Be Worse,” Journal of the American Medical Association, 175 (8) (2015): 1398.


50 Ibid.

51 Bo Wang and Aaron S. Kesselheim, “Characteristics ofefficacy evidence supporting approval of supplementalindications for prescription drugs in United States, 2005-14:systematic review,” The BMJ 351 (4679) (2015): 1-8.

52 Ibid.53 Leslie A. Pray, “Gleevec: the Breakthrough in Cancer Treat-

ment,” Nature Education 1 (1) (2008): 37.

54 Kesselheim, Wang, Franklin, and Darrow, “Trends in utiliza-tion of FDA expedited drug development and approvalprograms, 1987-2014: cohort study”; Pray, “Gleevec: theBreakthrough in Cancer Treatment.”

55 Ibid.

56 Kesselheim and others, “Trends in utilization of FDAexpedited drug development and approval p rograms, 1987-2014: cohort study.”

57 Ibid.


59 Brady Dennis, “Prescription drug prices jumped more than10 percent in 2015, analysis nds,” The Washington Post, ” January 11, 2016, available at https://www.washingtonpost.com/news/to-your-health/wp/2016/01/11/prescription-drug-prices-jumped-more-than-10-percent-in-2015/;Sean P. Keehan and o thers, “National Health ExpenditureProjections, 2014–24: Spending Growth Faster Than Recent Trends,”Health Affairs 34 (8) (2015): 1407-1417.

60 Peter B. Bach, “Why Drugs Cost So Much,”The New YorkTimes, January 14, 2015, available at http://www.nytimes.com/2015/01/15/opinion/why-drugs-cost-so-much.html.

61 Rahul Guha, Andrew M. Lacy, and Sally Woodhouse,“Analyzing Competition in the Pharmaceutical Industry,”(Section of Antitrust Law of the American Bar Associa-tion, 2008), available at https://www.cornerstone.com/GetAttachment/5f41372f-11ac-4f45-9f27-45b1dad16444/Analyzing-Competition-in-the-Pharmaceutical-Indust.pdf.

62 Adam J. Fein, “The Power of Formulary Non-Exclusion:Express Scripts Adds PCSK9 drugs,” Drug Channels, October8, 2015, available at http://www.drugchannels.net/2015/10/the-power-of-formulary-non-exclusion.html.

63 Caroline Humer, “Express Scripts drops Gilead hep C drugsfor cheaper AbbVie rival,” Reuters, December 22, 2014, avail-able at http://www.reuters.com/article/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222.

64 Ibid.

https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm474696.htmhttp://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm474696.htmhttp://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.gao.gov/assets/680/674183.pdfhttp://www.drugchannels.net/2015/10/the-power-of-formulary-non-exclusion.htmlhttp://www.drugchannels.net/2015/10/the-power-of-formulary-non-exclusion.htmlhttp://www.reuters.com/article/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222http://www.reuters.com/article/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222http://www.reuters.com/article/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222http://www.reuters.com/article/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222http://www.drugchannels.net/2015/10/the-power-of-formulary-non-exclusion.htmlhttp://www.drugchannels.net/2015/10/the-power-of-formulary-non-exclusion.htmlhttp://www.gao.gov/assets/680/674183.pdfhttp://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=0http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm474696.htmhttp://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm474696.htmhttps://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/https://www.washingtonpost.com/news/wonk/wp/2015/11/24/politicians-want-to-speed-up-drug-approvals-that-could-backfire/


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65 U.S. Food and Drug Administration, “FDA approves Sovaldifor chronic hepatitis C,” Press release, December 6, 2013,available at http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm377888.htm; U.S. Food and DrugAdministration, “FDA approves Viekira Pak to treat hepatitisC,” Press release, December 19, 2014, available at http://www.fda.gov/NewsEvents/Newsroom/PressAnnounce-ments/ucm427530.htm.

66 Peter Loftus, “FDA Warns of Liver Damage from AbbVie’sHepatitis C Treatments,” The Wall Street Journal , October22, 2015, available at http://www.wsj.com/articles/fda-warns-of-liver-damage-from-abbvies-hepatitis-c-treatmen-

ts-1445544456.

67 Stephanie M. Lee, “Cost of Gilead’s hepatitis C pill, Sovaldi,spurs revolt,” The San Francisco Chronicle, April 13, 2014,available at http://www.sfgate.com/health/article/Cost-of-Gilead-s-hepatitis-C-pill-Sovaldi-spurs-5398315.php .

68 Peter Bach, “Cancer: Unpronounceable Drugs, Incom-prehensible Prices,” Forbes , August 13, 2014, available athttp://www.forbes.com/sites/matthewherper/2014/08/13/cancer-unpronounceable-drugs-incomprehensible-prices/#40398a653f36 .

69 Aidan Hollis, “Me-too drugs: is there a problem?” (Geneva,Switzerland: World Health Organization, 2004), availableat http://www.who.int/intellectualproperty/topics/ip/Me-tooDrugs_Hollis1.pdf.

70 John Tozzi, “How Much Would You Pay for an Old Drug?If You Have MS, a Fortune,” Bloomberg Business, April 24,

2015, available at http://www.bloomberg.com/news/articles/2015-04-24/health-the-price-of-multiple-sclerosis-drugs-only-goes-up .

71 Daniel M. Hartung, Dennis N. Bourdette, Sharia M. Ahmed,and Ruth H. Whitham, “The cost of multiple sclerosis drugsin the US and the pharmaceutical industry,” Neurology 84(21) (2015): 2185-2192, available at http://www.neurology.org/content/84/21/2185.full.

72 Richard Harris, “Multiple Sclerosis Patients StressedOut By Soaring Drug Costs,” NPR, May 25, 2015,available at http://www.npr.org/sections/health-shots/2015/05/25/408021704/multiple-sclerosis-patients-stressed-out-by-soaring-drug-costs.

73 John Tozzi, “How Much Would You Pay for an Old Drug? IfYou Have MS, a Fortune.”

74 Optum Labs, “Rheumatoid Arthritis Cost Drivers,” November14, 2014, available at https://www.optum.com/thought-leadership/rheumatoid-arthritis-cost-drivers.html.

75 The Express Scripts Lab, “The 2014 Drug Trend Report”

(2015), available at http://lab.express-scripts.com/lab/drug-trend-report; The Express Scripts Lab, “The 2013 Drug TrendReport” (2014), available at http://lab.express-scripts.com/lab/drug-trend-report/previous-reports .

76 Robert Langreth, “Hot Drugs Show Sharp Price Hikes inShadow Market,” Bloomberg Business, May 6, 2015, availableat http://www.bloomberg.com/news/articles/2015-05-06/diabetes-drugs-compete-with-prices-that-rise-in-lockstep; Robert Langreth, “Big Pharma’s Favorite Prescription: HigherPrices,” Bloomberg Business, May 8, 2014, availab le athttp://www.bloomberg.com/bw/articles/2014-05-08/why-prescription-drug-prices-keep-rising-higher.

77 Office of the Assistant Secretary for Planning and Evalua-tion, “Understanding Recent Trends in Generic Drug Prices”(U.S. Department of Health and Human Services, 2016),available at https://aspe.hhs.gov/sites/default/les/

pdf/175071/GenericsDrugpaperr.pdf.

78 Ibid.; Janet Woodcock, Testimony before the House Over-sight and Government Reform Committee, “Developmentsin the Prescription Drug Market: Oversight,” February 4,2016, available at https://oversight.house.gov/hearing/developments-in-the-prescription-drug-market-oversight/ .

79 Office of the Assistant Secretary for Planning and Evalua-tion, “Understanding Recent Trends in Generic Drug Prices.”

80 Bob Pollock, “The Backlog at OGD—a Historical Look,” Lach-mann Consultants, July 16, 2015, available at http://www.lachmanconsultants.com/2015/07/the-backlog-at-ogd-a-historical-look/ .

81 Janet Woodcock, “Implementation of the Generic DrugUser Fee Amendments of 2012 (GDUFA),” Testimony beforethe House Oversight and Government Reform Committee,February 4, 2016, available at https://oversight.house.gov/

wp-content/uploads/2016/02/Woodcock-FDA-Statement-1-26-Prescription-Drugs.pdf.

82 Sheila Kaplan, “One reason for high drug prices: a hugebacklog of unapproved generic drugs,” STAT News,December 29, 2015, available at http://www.statnews.com/2015/12/29/generic-drugs-backlog/ .

83 U.S. Food and Drug Administration, “Generic Drug User FeeAct Program Performance Goals and Procedures,” availableat http://www.fda.gov/downloads/ForIndustry/UserFees/GenericDrugUserFees/UCM282505.pdf (last accessed March2016).

84 Woodcock, “Implementation of the Generic Drug User FeeAmendments of 2012 (GDUFA).”

85 Ibid.

86 Jeremy A. Greene, Gerard Anderson, and Joshua M. Sharfs-tein, “Role of the FDA in Affordability of Off-Patent Pharma-ceuticals,” Journal of the American Medical Association 315 (5)(2015):461-462.

87 Ibid.; Aaron Kesselheim, “Why Are Some Generic DrugsSkyrocketing in Price?” Testimony before the Senate Health,Education, Labor, and Pensions Committee, November 20,2014, available at http://www.help.senate.gov/imo/media/doc/Kesselheim.pdf .
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