AIDS TREATMENT NEWS Issue #235, November 17, 1995
John S. James

The AIDS community should be aware of this bill now because important negotiations may happen before it is publicly introduced. And the position of our community does matter -- especially since the support of Senator Edward Kennedy, the senior Senate Democrat on health policy, will likely be critical for this bill to succeed. The Kassebaum bill could establish the leadership of a moderate, consensus approach to FDA reform over what might be much more radical proposals from the House. This bill deserves careful evaluation on its merits (keeping in mind that the draft which exists now will change greatly during the legislative process).

Any FDA legislation is likely to be controversial in the AIDS community, because many activists, including this writer, wanted to see FDA and drug-development reform through administrative action, instead of through legislation. Administrative reform can be much faster; also, there is reason to fear what Congress might do once it opens up this issue. But a package of administrative FDA reforms just announced by Vice President Al Gore and FDA Commissioner David Kessler is disappointing. According to the November 10 and October 30 BIOCENTURY (a weekly faxed newsletter published by BioCentury Publications Inc. in San Carlos, California), "the reforms center on elimination of requirements for establishment licenses and lot release approvals for well characterized biologics." In other words, this highly touted administrative reform concerns technical changes which will save money for business, but not touch the structural issues associated with the astonishing lack of significant improvement in new treatment for patients, despite the great resources devoted to medical research.

There has been no major FDA legislation since 1962. Since that time, the FDA's role has become increasingly important. And the FDA is unusual among government agencies in that there is generally no appeal from even a lower-level individual's decision, and little effective institutional oversight. AIDS does get high-level attention -- and in effect has an informal appeals system which often works well. But away from the limelight of a high-profile issue, when companies feel that they have been treated wrongly, perhaps by an inexperienced employee who did not understand the matter or did not have the resources to address it properly, they have to swallow hard and keep quiet.

What's In the Kassebaum Bill?

The 76-page draft bill is hard to evaluate and summarize, but below are some provisions we noticed. Major bills change greatly during the legislative process; the following should be accurate as of November 14, but later will become obsolete. The points below are in the order in which they appear in the draft of the bill.

* Mission. The FDA's mission would now explicitly include "facilitating the rapid and efficient development and availability of products subject to its regulations," as well as protecting the public from unsafe or ineffective products.

* Performance standards and review. The FDA commissioner would be required to publish and use quantifiable performance standards, to measure how well the FDA is doing in processing new drug applications, and in other matters, including responding to citizen petitions.

* Information. The Secretary (of the Department of Health and Human Services, the FDA's boss in the government) would be required to set up an information system to track all applications, petitions, notifications, and other requests submitted to the FDA -- and permit access by the applicant. In addition, all "policy statements of general applicability" (guidelines, points-to-consider, protocols, recommendations, etc.) must be formally published and compiled. These measures would greatly help the interested public in understanding what actually happens at the FDA.

[Comment: The three proposals above are important in view of the fact that today nobody really knows why the drug development process works as poorly as it does. In this situation, the first step toward improvement is to clarify and open the process, so that problems and solutions can come into view.]

* Appeals. The Secretary "shall by regulation establish a system for the appeal," within the FDA, of any decision by an FDA employee (except for administrative or judicial proceedings). Any person could request an evaluation by an appropriate (advisory) scientific review group, and that group would decide which appeals it would hear. Other appeals systems could also be used. Within 60 days of conclusions and recommendations from the appeals process, the appropriate FDA official will make a final determination, and explain in writing the reason for any differences from the appeals recommendation.

* Expedited access for seriously ill patients. The bill outlines a system which looks to us much like the expanded access systems today. It may help to unify the various different programs for access to experimental treatments, and apply them more equally to all major diseases. It allows companies to charge "cost recovery" for experimental drugs or devices, as they can today under one program, the "treatment IND." [But since the costs to be recovered under the current draft of the bill can include not only manufacturing and handling, but also "research" and "development," it is questionable whether this "cost recovery" language places any real limit on what companies can charge. Clearly this issue needs attention.] At present, large companies do not charge because they do not want to set prices early, or reveal information about their costs. Any reform should be scrutinized to make sure it will not make the existing situation worse.

* Compromise on new drug research. Apparently Kassebaum's office considered proposals to allow institutional review boards to approve human research in some cases, without going through the FDA -- but then backed off from that approach, and proposed a compromise instead. We are unclear how the compromise differs from current practice.

* Outside review. The bill would allow (not require) the Secretary to contract for outside expert review on part or all of any application for approval of a drug. The FDA, however, would still make the final decision -- within 60 days after the outside review is complete.

* Information exchange. Under rather strict controls, pharmaceutical companies would be able to tell health professionals about "off label" uses of approved drugs (and treatment uses for experimental drugs, when those uses have been approved by the Secretary). The only information the companies could distribute would be complete peer-reviewed articles or textbook chapters published by an organization independent of the pharmaceutical industry, or text approved by an independent continuing medical education accrediting agency, or information relating "to a use that is recognized under Federal law for purposes of third-party coverage or reimbursement," or information that is "an accurate and truthful summary" of material from the three kinds of sources listed above.

* Restrictions in evaluating effectiveness. Companies trying to prove the effectiveness of a new drug could not be required to prove relative effectiveness, or cost effectiveness, unless those were claimed. And a "diagnostic device" would not have to prove effectiveness in clinical outcome, unless that were claimed. [For example, a test for HIV RNA could be approved based on proof that it accurately measured what it said it did -- without requiring a difficult and time-consuming clinical trial to accumulate statistical proof that patients whose doctors use viral load tests ultimately do better than patients whose doctors do not.]

* Export reform. This bill takes seriously the responsibility to regulate the export of unapproved drugs to countries which do not have their own regulatory system.

* New drug approval standard. The bill would add the following language to current law: "Substantial evidence shall consist of at least data from one well-controlled clinical investigation (which may be waived by the Secretary) and confirmatory evidence (obtained either before or after such investigation)." Currently, the FDA almost always requires at least two well-controlled clinical trials to approve a drug; but it does have the power to accept only a single trial in rare circumstances. The new language, which clearly allows but does not require the FDA to accept less than two trials, seems more a clarification rather than a change to current practice.

* Drugs and biological products. The bill continues the existing movement in the direction of unifying the regulation of "drugs" and "biologics." In the past, these were distinct because biological products often could not be well characterized as to what they contained. As technology advances, the need for this distinction diminishes.

* Pilot plants. A pharmaceutical company would not necessarily be required to set up a full-scale manufacturing facility to produce the drug supplies needed for clinical trials to prove safety and effectiveness.

* Other sections include changes in the regulation of biological products, medical devices, and food additives.

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