WASHINGTON _ Sen. Nancy Kassebaum (R-Kan.),chairwoman of the Senate Labor and Human ResourcesCommittee, has instructed her staff to circulate a draftplan to reform the FDA that is strikingly similar to aproposal already put forth by the biotechnology andpharmaceutical industries.

The Kassebaum plan is the first document developed by achair of a Congressional committee with jurisdiction overFDA reform. Rep. Ron Wyden (D-Ore.) has introducedhis FDA reform plan but Wyden is the ranking Democraton the House Commerce Health Subcommittee and doesnot control the fate of authorizing legislation.

Kassebaum's draft won immediate praise from theBiotechnology Industry Organization (BIO). "It's good tohave something from the Senate Republican leadership,"said Dominique Kahn, BIO's director of federalgovernment relations. Kahn said she anticipates that theHouse Commerce Committee will introduce actuallegislation sometime in September, the same time thatKassebaum's bill is likely to be presented.

"The draft is particularly good news for small biotechfirms which are being pressured by their investors abouttheir progress or lack thereof in FDA's product approvalprocess," Kahn said in an interview with BioWorldToday. "The fact that legislation is close to introductionwill register positively with investors," she said.

The following are excerpts from the staff discussionpaper:

The Efficacy Standard. The FDA's gold standard for newdrug approvals would be modified to permitmanufacturers to submit only one adequate and well-controlled clinical study.

Biotech drugs in the clinical testing phase could beproduced at pilot facilities unless the FDA requires that afull-scale production facility is necessary to ensure safetyand efficacy.

Biotech drugs would be regulated the same as syntheticdrugs "unless the drug is not well characterized or the[FDA] determines that the product or manufacturingprocess continues to require additional special rules toprotect public health and safety."

Under Kassebaum's plan, most biologics would needonly an approved product license application to beginmarketing.

Kassebaum's draft would eliminate the legal requirementthat investigational new drug applications (IND)involving genetic therapy be reviewed by the NationalInstitutes of Health Recombinant DNA AdvisoryCommittee (RAC) in addition to FDA.

FDA Watchdog. Kassebaum's draft would install apermanent FDA oversight committee charged withdeveloping performance standards for the agency'sreview of drugs, medical devices and food. Kassebaumanticipates a tight timetable for the panel's work. Thefirst set of performance standards would be developedwithin six months of the bill's enactment.

The panel would be comprised of "the nation's leadingmedical experts, scientific and health policy authorities,and representatives from the regulated industries andpatient advocacy groups," according to the draft.

Kassebaum anticipates that the performance standardswould become the basis of a required annual report by theFDA to Congress. The report would include an analysisof the reasons for the failure to achieve any of theperformance standards and set forth a plan for bringingthe agency into compliance.

To ensure that FDA Commissioner David Kessler stays inclose touch with the industry he regulates, Kassebaum'sdraft would establish a permanent council "with thefunction of facilitating and improving communicationbetween the agency and the regulated industries andfostering continued technological innovation inpartnership with the regulated industries."

Expedited Approvals. Kassebaum would codify existingregulation into law to "provide access to unapprovedtherapies for serious and life-threatening illnesses andseriously debilitating conditions." The FDA is directed tomake sure the agency's centers and divisions uniformlymake expedited access available to manufacturers whenappropriate.

Drugs and medical devices would be placed on a `fasttrack' review process if "the FDA finds that the risk fromthe product is not greater than the risk from the disease orcondition and there is a reasonable likelihood that theproduct will be effective in a significant number ofpatients."

Manufacturers that have products already approved formarketing abroad would save some money under aprovision in the Kassebaum plan instructing the FDA toaccept approval of the product by the U.K.'s MedicinesControl Agency or the European Medicines EvaluationAgency as evidence that the risk from the product is notgreater than the risk from the condition.

Products on the fast track would have to be approved ordisapproved within 180 days. If a product weredisapproved, the FDA would have to "show cause whythe risk from the product would be greater than the riskfrom the disease or condition and why the product wouldnot be effective in a significant number of patients."

Kassebaum's plan would permit the FDA to limit uses ofthis class of drugs to certain specialists and researchinstitutions.

Revitalizing Investigations. To weed out the number ofunnecessary requests for data, the Kassebaum draft wouldrequire the FDA to respond within 30 days to amanufacturer's request for an investigational drugexemption. "To protect against the excessive use ofclinical holds or requests for addition information to getaround the 30-day requirement, the clinical hold wouldnot be issued by the individual FDA reviewer." Instead, astatement in writing with an explanation for the reasonfor the hold would have to be issued by the director of theoffice to which the review has been assigned.

In addition, the center director (either drugs, devices orbiologics) would have to establish a "mechanism for theperiodic review of clinical holds the ensure that uniformstandards were being applied with regard to thecircumstances under which they were issued," the draftstated.

Clinical holds and requests for additional informationwould trigger inclusion in the annual performance report.

Relying More On Institutional Review Boards (IRB).Phase I and II clinical trials would not need FDAapproval if they were not intended to support a productapproval application. the FDA would be given theauthority to set standards for and certify IRBs for thispurpose. The FDA could issue a clinical hold on suchresearch but regulatory actions would also triggerinclusion in the annual performance report.

To further collaboration with the industry, FDA reviewersare instructed by the Kassebaum draft to meet promptlywith industry officials requesting a meeting about aresearch protocol design. The FDA would be required toprovide in writing at the meeting the agency's review ofthe draft investigation plan, including all deficienciesidentified by FDA reviewers.

Marketing Information. Kassebaum included a morerestrictive provision than the one developed by BIO andthe Pharmaceutical Research Manufacturers of America(PhRMA) to regulate what manufacturers can or cannotsay about off-label uses of drugs. Kassebaum's draft saidthat manufacturers would be "permitted to disseminatemedical tests and compendia articles from peer-reviewedscientific publications; presentations and proceedings ofmedical and scientific meetings; disease managementprograms and protocols; and complete extracts orpublished abstracts of such materials. These materialscould not be prepared by or for the product sponsor andwould have to prominently disclose that the materialscontain information about uses not approved by the FDA.A label or package insert would also have to be attached."

Export Provisions. The draft includes verbatim a bill (S.593) recently passed by the Senate that eases exportrestrictions on drug and device manufacturers. Butmembers of Congress are engaged in a turf battle over theexport bill and its provisions may be changed later in thissession of Congress, Capitol Hill sources have said. n

-- Michele L. Robinson Washington Editor

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