WASHINGTON _ The Biotechnology Industry Organization (BIO)unveiled its legislative and regulatory agenda for near-term,"achievable" FDA reform on Monday, including a proposal totransfer much of the burden of reviewing Phase I clinical trials toInstitutional Review Boards (IRBs).

Other ideas for change include eliminating the requirement forseparate approval of the manufacturing process (the EstablishmentLicensing Application, or ELA) for certain biological products;utilizing qualified non-governmental experts to perform toxicologyand environmental reviews, validation assays and lot release tests;prohibiting the FDA from requiring prior approval of sales andmarketing literature; and modifying laws so that companies canexport drugs and biological products not approved for sale in theU.S.

"We are shooting for a wholly achievable agenda," BIO presidentCarl Feldbaum told BioWorld. "In this first cut, we tended to putmarginal and more controversial proposals to the side." More radicalproposals could include plans for a fundamental restructuring of theagency.

Although framed as a legislative proposal, goals outlined in BIO's"concept paper" on FDA reform may be achievable "by reorientingFDA policy or through Agency rulemaking," according to the report.Indeed, some experts argue that a "cultural" or "attitudinal"revolution at the agency could be more important than any merestatutory change.

According to Feldbaum, BIO discussed its short-term plans forchange with top FDA officials last week, including Kathryn Zoon,director of the Center for Biologics Evaluation and Research(CBER). The group also met with Chris Jennings, a President Clintonaide who has been tapped to lead the Administration's FDA reformefforts.

In addition, copies of BIO's proposal are being sent to keylawmakers in the House and Senate, including Reps. Newt Gingrich(R-Ga.), Thomas Bliley (R-Va.), John Dingell (D-Mich.) and HenryWaxman (D-Calif.) and Sens. Nancy Kassebaum (R-Kan.), KayBailey Hutchison (R-Texas), Orrin Hatch (R-Utah), Judd Gregg (R-N.H.) and Daniel Coats (R-Ind.).

"As ideas began to gel, we stepped out and tested them with variouscritical constituencies," explained Feldbaum. "So far, people seem toregard this as a responsible, moderate proposal that addresses clearand present needs."

He added that BIO will forward copies of its 15-page proposal to theplethora of Washington-based think tanks working on FDA reform,including the Progress and Freedom Foundation, the CompetitiveEnterprise Institute, the American Enterprise Institute, the CatoInstitute, Citizens for a Sound Economy and the Washington LegalFoundation.

BIO's concept paper is organized around problems and solutions. Forexample, to solve the problem of outdated and inconsistent lawsgoverning the manufacture of biologics, BIO suggests consolidatingcurrent biologics regulation under the Public Health Service (PHS)Act with those of the Federal Food, Drug and Cosmetic Act. Such amove could eliminate inconsistencies between the way traditionalchemical and biotechnology drugs are regulated and could removethe PHS Act's stipulation that, for all biologics, an ELA must be filedsimultaneously with a Product License Application (PLA).

Changes Proposed In Clincal Trial Procedures

Another problem BIO identifies is "the costly and time-consumingdrug approval process." Statutory solutions proposed include:

* Congressional committee report language to encourage the FDA "tothe extent feasible and while maintaining high standards of efficacyand safety" to work with sponsors so that one pivotal clinical trial canserve as the basis for approval of "breakthrough drugs."

* Discontinuance of FDA review of Phase I clinical trials that aresponsored by individual researchers or academic institutions (BIOclaims that most of these studies do not result in "commercialtherapies" and gobble up about 60 percent of the valuable time ofFDA reviewers). Instead, the FDA or the National Institutes of Healthcould certify IRBs at academic medical centers or elsewhere toreview such Phase I proposals. Commercial sponsors of Phase Istudies could opt for either IRB or FDA review of their early-stageinvestigational new drug (IND) study plans. All sponsors of Phase IINDs would still be required to notify FDA when a clinical trial isinitiated and to report adverse events during the trial.

* A change in regulations giving the FDA 30 days to respond to newdata regarding clinical holds or other IND amendments orsupplements. If FDA does not respond, a trial may proceed.

* A narrowing and focusing of the FDA research activities to ensurethat they are linked to the drug review process, such as developmentof surrogate endpoints for various diseases. A Scientific ReviewBoard, appointed by the Secretary of Health and Human Services,would oversee all research activities of the agency.

Many Groups Target FDA Reform

John Villforth, president of the non-profit, educational Food andDrug Law Institute based in Washington, said he has seen countlessproposals for FDA reform in recent months. "I feel reform is less acase of the mechanics of any particular fix for the FDA and more acase of a cultural change," he told BioWorld. "None of theseproposals will work unless there's a cultural change at the agencyand, conversely, if you had a profound cultural change, you probablywouldn't need any of these other changes."

Villforth also noted that there is a wide disparity between the averageAmerican's view of the FDA and its current commissioner, DavidKessler, and that of the average Washington insider. "The averageAmerican thinks the FDA does an outstanding job and that Kessler isa hero," said Villforth. "The average lawyer inside the beltway thinksthe present system is ruining American competitiveness."

This disparity in perception could hinder the efforts of would-beFDA reformers, according to Villforth. Abbey Meyers, president ofthe National Organization for Rare Disorders, agrees. "The FDA isviewed by people as the chief consumer protection agency," she toldBioWorld. "If industry and others really pursue big changes, therewill be an uproar. This is one area where you'll find that people wantbig government. They don't want to be popping unsafe or ineffectivepills."

Feldbaum was careful to portray BIO's first round of proposals forchange in unalarming terms on Monday. "Our proposals totallysupport the FDA's authority to assure public safety," he said. "Wehave designed our proposals to allow the FDA to focus its resourceson getting safe, effective therapies and cures to the people who needthem more quickly than the outdated existing system allows." n

-- Lisa Piercey Washington Editor

(c) 1997 American Health Consultants. All rights reserved.