N E W S

NATURE BIOTECHNOLOGY VOLUME 22 NUMBER 5 MAY 2004 495

Officials of the US Food and DrugAdministration (FDA; Rockville, MD, USA)on March 16 launched an effort to acceler-ate products into and through the drug reg-ulatory system. Although biotechnologyindustry representatives are praising theagency’s ‘critical path’ initiative, questionsare being raised about precisely what theagency is planning to do, and how soon andhow well this new wish list can be imple-mented in the wake of Commissioner MarkMcClellan’s departure and amid the currentpolitical and budgetary turmoil.

“Today, as never before, we face a tremen-dous potential for new medicines to pre-vent and cure diseases, but fewer newproducts are actually reaching the FDA,”said former commissioner McClellan whenthe agency released its report (see Fig. 1).The chief reason behind this disappointingtrend, according to the agency report“Innovation or Stagnation?—Challengeand Opportunity on the Critical Path toNew Medical Products,” is that new scienceis not being “adequately harnessed to guidethe technology development process in thesame way that it is accelerating the discov-ery process.”

Thus, agency officials are promising towork with scientists in industry, at otherfederal agencies such as the NationalInstitutes of Health (NIH; Bethesda, MD)and at universities “to make the critical path[to product approval and marketing] muchfaster, predictable, and less costly.” The nextsteps in this initiative include a series ofworkshops and meetings—for example, asNature Biotechnology went to press, officialsplanned to devote the April Science Boardmeeting with stakeholders to this topic.

According to the FDA, this effort includes“a new focus on modernizing the tools thatapplied biomedical researchers and productdevelopers use to assess the safety and effec-tiveness of potential new products, and themanufacturing tools necessary for high-quality mass production of cutting-edgetherapies.” Carl Feldbaum, president ofBiotechnology Industry Organization(BIO; Washington, DC), hopes this new“product development toolkit” will “elimi-nate inefficiencies and…lower costs.” Hestresses the importance of this because “theenormous costs and difficulties associatedwith moving research from the lab bench tothe patient cannot be overstated.”

Both McClellan and current acting com-missioner Lester Crawford say that imple-

menting the critical path initiative willdepend in part on the FDA developing andfunding new research programs, while alsoexpanding agency capacity for its more cus-tomary regulatory activities. The need forthis augmented capacity anticipates a surgein candidate products—mainly from indus-try, but with some of them expectedbecause of a buildup resulting from theNIH annual budget’s doubling during thepast five years to about $28 billion.

NIH Director Elias Zerhouni’s 2003Roadmap Initiative emphasizes ‘transla-tional’ research among several goal-ori-ented pledges to increase the efficiency ofefforts to bring therapeutic products intoclinical use (Nat. Biotechnol. 21, 1253–1254,2003). The FDA report applauds suchadvances and concludes that the agencyshould undertake a third type of researchbeyond the familiar “basic and transla-tional.” This other kind of research entailstargeting “the process of creating safe andeffective products from new scientific dis-coveries.”

Exactly what FDA officials have in mindremains uncertain, according to SaraRadcliffe, BIO’s director of science and reg-ulatory affairs. Agency scientists in somecases might need “to get in the lab and kickstart the process and set the framework—

for example, as to appropriate animal stud-ies needed to evaluate vaccines becausecompanies need to know what tests will berequired,” she says. But beyond such efforts,she adds, scientists working in the privatesector should be choosing animal modelsand studying how best to use them, whilethe agency continues to focus on its princi-pal mission of being a regulator. “I don’tthink it appropriate for FDA to aim atbecoming a research organization.”

Top NIH officials appear to be on boardwith the new FDA plan, but their approvalis no surprise, because both agencies oper-ate within the Department of Health andHuman Services (Washington, DC, USA)under secretary Tommy Thompson, who isinsistent on such agreement within thedepartment. However, in view of currentconstraints on the federal budget, it isunlikely that the FDA will have newresources for new research programs,meaning this initiative will depend on redi-rected funds.

Meanwhile, agency officials have“extended an invitation to stakeholders tohelp them make this more concrete,” saysRadcliffe, noting that ample “leadership isin place” through acting commissionerCrawford, Cross Center InitiativesTaskforce director Janet Woodcock andother centers’ directors to pursue this ini-tiative. “BIO is taking this very seriously,”she adds, noting that BIO will be consultingwith member companies to assemble spe-cific examples of what might be done toimprove current practices. “Industry andFDA have to come closer together,” she says.

Jeffrey L Fox, Washington

FDA plans to improve and accelerate product reviewsFigure 1 10-yeartrends in major drugand biological productsubmissions to the USFDA. NME, newmolecular entity; BLA,biologics licenseapplication. Regulatoryagencies worldwidehave observed similartrends. Source: USFDA.

199310

20

30

40

50

60

70

80

1994 1995 1996 1997 1998 1999 2000 2001 2002 2003

Su

bm

issi

on

s to

FD

A

Year

Total NMEs received by FDA

Original BLAs received by FDA

It is unlikely that the FDA willhave new resources for newresearch programs

©20

04 N

atur

e P

ublis

hing

Gro

up

http

://w

ww

.nat

ure.

com

/nat

ureb

iote

chno

logy