WASHINGTON _ One regulatory avenue the FDA uses to speeddrug approvals may hold promise as a model for Congress as it looksfor workable solutions to streamline FDA regulations withoutcompromising public health standards.

Called the Subpart E regulations, these rules were launched in 1988by the FDA to move drugs for life-threatening illnesses through theclinical development and regulatory review phases quickly. Productswere cleared for marketing with considerable efficiency both by themanufacturer and the FDA, which was able to exercise considerableflexibility in applying traditional regulatory standards.

The Biotechnology Industry Organization (BIO) considers theSubpart E regulations to be so promising that it included languageincorporating the principles behind the regulations in its proposedFDA reform bill, said Stephan Lawton, a partner with Hogan &Hartson law firm in Washington.

The regulations work so well that "consideration should be given tobroadening [their application] to other categories of diseases anddrugs," stated a new study of FDA early access and fast trackapproval initiatives.

Study author Sheila Shulman, of Tufts Center for the Study of DrugDevelopment in Boston told BioWorld that one of the key reasonsthe Subpart E regulations work so well is that they allow for a "closercollaboration between industry and the FDA to facilitate approval ofa drug after Phase II clinical trials. The regulations make it possiblefor company officials and FDA staffers to actually sit down togetherand design a broad-based, multi-center Phase II clinical trial togenerate sufficient data to permit approval at the end of Phase II,"Shulman said.

A recent study of the Subpart E regulations and other FDA fast-trackapprovals, written by Shulman and Jeffery Brown, also of Tufts,found that of all FDA accelerated approval programs, the Subpart Eprotocol perhaps worked the best.

Under Subpart E regulations, "average total development time of 7.5years was achieved largely through the compression of the clinicalphase and to a lesser extent by an abbreviated FDA review phase,"according to the study.

The study's authors urged manufacturers to consider using theSubpart E accelerated approval process.

"These results suggest that the Subpart E pathway should be amongthe options explored in the initial stages of product development.This may be of particular relevance for orphan drugs since the natureof most orphan indications makes these drugs likely candidates forSubpart E approval."

Of the 28 drugs that were approved by the FDA's Center for DrugEvaluation and Research in the six-year period since the Subpart Eprogram began, 61 percent involved drugs for the treatment ofcancer, AIDS and other HIV-related conditions. Some 77 percentwere rated by the FDA as having a high therapeutic value. More thanone-third _ 36 percent _ were distributed prior to approval under atreatment investigational new drug protocol; 50 percent receivedorphan drug designation.

Biotech drugs approved as part of the process include two GenzymeCorp. products for Type I Gaucher's disease: Alglucerase andCerezyme.The Tufts researchers found that drugs on the Subpart Eapproval fast track fared very well. "On average, the total regulatoryphase for Subpart E drugs was 3.3 years less than that for the non-Subpart E comparison group. Although a difference was observed inboth the clinical and the FDA review phases, the greater disparitywas evident in the clinical phase which was 2.7 years shorter for theSubpart E than for the non-Subpart E approvals." n

-- Michele L. Robinson Washington Editor

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

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