ARLINGTON, Va. The National Task Force on AIDS DrugDevelopment (NTFADD) recommended on Tuesday that regulatoryreview of research protocols involving human gene transfer bestreamlined. The recommendations could affect approval proceduresfor all gene therapy clinical trial protocols done in the U.S.The proposal to streamline gene therapy review was the most concreteof 10 recommendations and action items adopted unanimously byNFTADD members on Tuesday, the final day of the task force'ssecond meeting. Other recommendations included a proposal that theNational Institutes of Health (NIH) create and implement an electronicdata base of abstracts in the HIV/AIDS field and that it expandprograms to make available suitable vectors and related technology forgene therapy. Action items included plans to convene workshops thatwill study AIDS animal models and drug screening efforts.In addition to its formal recommendations, the NFTADD voted torecommend that the current hiring freeze at the NIH be lifted.Although Washington is awash in task forces and recommendations,NFTADD appears uniquely capable of swallowing its own medicine.Established in November 1993, the NFTADD was given a herculeantask: to identify and attempt to remove all barriers to the nation's AIDSdrug development effort.The NFTADD reports directly to Clinton cabinet member and Healthand Human Services Secretary Donna Shalala. By charter, the taskforce is comprised of individuals who can commit at the table toimplement the task force's recommendations.FDA commissioner David Kessler, NIH director Harold Varmus, NIHOffice of AIDS Research chief William Paul and, last but not least,HHS Assistant Secretary Philip Lee all sit attentively around the tablewhen this task force deliberates. Indeed, these people are so directlyinvolved in the matters at hand, they must often recuse themselvesfrom voting (as Varmus, Kessler and Lee did on the proposal to lift theNIH hiring freeze).Gregg Gonsalves, a member of the New York-based Treatment ActionGroup (TAG), an AIDS activist organization that is represented on theNTFADD, told BioWorld that many activists are skeptical of taskforces. But he said that NFTADD is unique because of its high-levelmembership.'You have to put this in a historical context. How many commissionsand task forces have we had on AIDS?' he said. 'Things don't getdone by task forces or commissions, they get done by people with lineauthority. The people on this task force have that authority.'Gonsalves said that the Clinton administration's response to theNFTADD's recommendation to lift the NIH hiring freeze will serve asa litmus test for its commitment to biomedical and AIDS research.Streamlined Gene Therapy ReviewThe NFTADD has already passed one test of its clout with flyingcolors. It devised its recommended overhaul of gene therapy regulatoryprocedures in record time, about three months.The NFTADD recommendations represent a joint effort of the FDAand the NIH. At the first NTFADD meeting last April, both agencieswere charged with the task of determining whether current genetherapy review processes are an obstacle to AIDS drug development.The recommendations they produced will apply to all drugs, not justAIDS drugs.Currently, investigational new drug applications (IND) for genetherapies undergo the same FDA review required of all drugs.However, companies wishing to conduct clinical trials using genetherapies must also make a separate submission to and obtain clearancefrom the NIH's Recombinant DNA Advisory Committee (RAC).Industry representatives have complained that RAC submissionrequirements, which differ from the FDA's, are cumbersome. SinceRAC meets only three to four times per year, a missed deadline or arequest for more data can mean months of delays for companieswishing to launch clinical trials.The NFTADD recommendations attempt to create a 'one-stopshopping' environment for companies seeking to test gene therapies.Gene transfer protocols would be submitted directly to the FDA in theFDA's IND format (no additional RAC submission using a RACformat would be required).FDA review of the IND would proceed while the NIH Office ofRecombinant DNA Activities (ORDA) would simultaneously rule onwhether or not RAC review of the protocol was even necessary. ORDAreviewers would consider whether or not the gene therapy proposedwas well-established or represented an unknown or novel approach thatwarranted RAC and public review.According to ORDA director Nelson Wivel, the NFTADDrecommendations will expand RAC's role such that the group advisesboth the NIH's Varmus and the FDA's Kessler (currently its charter isto advise only Varmus). At the same time, RAC's role will be restrictedto making suggestions.For example, companies will now deal primarily with the FDAregarding their protocols. The RAC will no longer have authority torequest additional data directly from companies to support theirsubmissions. Instead, the RAC can advise Varmus and Kessler torequest the data. Ultimately, FDA reviewers will decide if theadditional data are necessary.Wivel said his office is already coordinating with FDA offices in orderto implement the new procedures. Gene therapy protocol submissionsfor RAC's next meeting - scheduled for Sept. 12-13 - were due onMonday, so the current crop of gene therapy protocols will be reviewedusing old procedures. At its next meeting, RAC will be presented withthe streamlining proposal as a courtesy.Dr. Varmus and Dr. Kessler made it clear that they want the processstreamlined for one-stop shopping and that's the bottom line. It's notlikely that RAC will disagree, Wivel told BioWorld. Therecommendation is not contingent on RAC approval. RAC has shapedthe field but the ultimate power has always resided with the FDA.Nevertheless, the changes won't be finalized or go into effect until afterthe September meeting. Kessler has already promised that his agencywill pay close attention to RAC recommendations, saying that the FDAwill have to answer for each and every RAC suggestion it ignores.We've potentially made a breakthrough and provided a more coherentgovernment interface for biotech companies, said Daniel Hoth,NTFADD member and senior vice president of Foster City, Calif.-based Cell Genesys Inc. These changes, if implemented, could allowgene therapy research and development to achieve parity withtraditional drug development.The biotechnology industry lobbied for and won two spots on the 18-member NFTADD. One went to Hoth and the other to Genentech Inc.chief executive officer Kirk Raab. However, Raab hasn't shown up foreither of the task force's first two meetings.At Tuesday's meeting, Lee expressed frustration at the number of taskforce members who were absent (two) and one NFTADD membersuggested that non-attending members be replaced. The matter will bediscussed at the task forces October meeting.A Genentech spokesman said that Raab had previous conflictingcommitments at the time he was appointed to NFTADD, but that heplans to attend the October meeting. Raab sent a Genentechrepresentative in his stead to the first two meetings, but only fullmembers can participate in NFTADD deliberations. n
-- Lisa Piercey Washington Editor
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