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Executive Summary

Drug sponsors may request approval from FDA to charge for a drug being tested in a parallel track program, according to a policy statement published in the April 15 Federal Register. The final policy, which expands the availability of investigational drugs to people with AIDS and HIV-related diseases, differs little overall from the proposed policy announced in May 1990. The question of cost recovery was left open in the proposed statement. FDA received several comments about the costs to drug manufacturers participating in the parallel track program. "The concerns raised included the costs of increased production of the drug for parallel track use without the guarantee of approval, as well as insurance and other potential liability costs," the policy statement notes. "Questions were raised about eligibility for cost recovery under parallel track protocols." Noting that IND sponsors are "ordinarily not permitted to charge for investigational drugs," FDA said they may request approval for charging "based upon an explanation of why charging is necessary to undertake or continue the study." Sponsors of parallel track studies may now make a similar request, FDA states. The agency "recognizes the importance of the reimbursement issues concerning experimental therapies and reaffirms its commitment to help facilitate consideration of these issues," the document states. However, "even if such approval is obtained, under no circumstances may a sponsor commercialize a product by charging more than needed for cost recovery," FDA cautioned. Applying for permission to charge may create a double-edged sword for drug sponsors. Once a sponsor charges a certain price for a drug in a parallel track study, post-approved price increases may pose public relations problems. Some sponsors may simply chose not to avail themselves of the cost-recovery option: Bristol-Myers Squibb, for example, did not charge for its AIDS drug Videx (ddI) when it was distributed under a Treatment IND and open safety protocols. Warner-Lambert, on the other hand, plans to charge patients $100 per month after the first six weeks of its Treatment IND for its Alzheimer's drug Cognex (tacrine). The issue of cost recovery may become an important factor when parallel track is expanded beyond AIDS drugs. FDA Commissioner Kessler stated April 9 that the parallel track policy will be broadened to include drugs for other serious illnesses once FDA has experience with the program and is assured that parallel track will not interfere with ongoing controlled clinical trials. The April 15 announcement also alludes to future expansion of parallel track. FDA "invites patient groups, physicians and sponsors interested in developing a similar process for other life-threatening diseases to work with PHS on issues concerning expanding the parallel track mechanism for other life-threatening diseases." The Public Health Service said it received 1,210 comments on the proposed policy, of which 200 were unique and 1,010 were form letters. As previously outlined, proposals for parallel track will be submitted to FDA as amendments to existing IND applications. Unless the sponsor objects, FDA will refer the proposal to the National Institute of Allergy and Infectious Diseases' AIDS Research Advisory Committee (ARAC). After ARAC's review, the director of NIAID will forward the committee's recommendation to the FDA commissioner. Under the expanded access program, people with AIDS/HIV for whom standard therapy is ineffective or inappropriate and who are ineligible to enter ongoing controlled clinical trials may receive investigational drugs in parallel with controlled trials through studies without concurrent control groups. FDA has revised its proposed policy to allow "flexibility in determining what constitutes standard treatment for the particular condition and patient population identified in the proposed parallel track study, in order to take into account unique circumstances." The policy statement no longer includes the parenthetical phrase defining standard therapy as "a drug approved for marketing or available under a Treatment IND for the same clinical condition for which the investigational drug is being studied." As originally proposed, a national human subjects panel is to be established to provide protection for patients involved in parallel track studies. The panel would generally replace local Institutional Review Board (IRB) review. Until a permanent panel is established, NIAID's AIDS Program Advisory Committee will carry out the duties of the panel. FDA had initially proposed that ARAC serve in this temporary role, but then concurred with comments that argued ARAC's role in evaluating therapies for parallel track conflicted with the role of an IRB. FDA said it received a number of comments arguing that any person should be able to petition ARAC to consider parallel track protocols for specific drugs. While noting that information proprietary to a drug manufacturer is necessary to evaluate the appropriateness of a drug for parallel track, FDA said NIH "can be requested to take on the obligation of developing a drug lacking private sector sponsorship, and in that role also assume any responsibilities for implementing a parallel track program." FDA explained in the introduction to the document that "there may be extraordinary circumstances in which a non-sponsor has sufficient information about the drug and its potential usefulness for the intended patient population and condition to be treated, and about the controlled clinical trials to permit meaningful review of a parallel track proposal. In such circumstances, the non-sponsor could request NIAID to refer the matter to the ARAC for review and recommendation." However, "because PHS expects that such circumstances would be rare, the policy statement has not been amended to refer specifically to such requests by non-sponsors," the document says. The document lists eight criteria for consideration of a drug's availability through parallel track. As initially proposed, patient enrollment into Phase II controlled clinical trials must be initiated prior to or simultaneously with expanded availability of drugs under a parallel track protocol. If there is evidence that the parallel track study is interfering with enrollment or completion of the controlled studies, the parallel track protocol may be terminated. In a same-day Federal Register notice, FDA issued a final rule setting forth the grounds on which the agency may place a parallel track study on clinical hold. The final rule appears identical to the agency's May 21, 1990 proposed rule. The final rule sets out seven reasons for putting a parallel track study on clinical hold, including "reasonable evidence that the noncurrently controlled study is impeding enrollment in, or interfering with, an adequate and well-controlled study of the same or another investigational drug." Other reasons for instituting a clinical hold include: insufficient quantities of the drug for both the parallel track study and the controlled studies; studies show that the drug is not effective; another drug has a better potential benefit/risk balance; the drug is approved for the same indication and patient population; the sponsor is not pursuing approval with due diligence; or the commissioner decides that continuing the parallel track study "would not be in the public interest." In some cases, such as when there are insufficient supplies of the drug, the drug appears ineffective or the drug is approved, "FDA ordinarily intends that clinical holds...would apply only to additional enrollment in nonconcurrently controlled trials, rather than eliminating continued access to individuals already receiving the investigational drug," the final rule says. Parallel track studies can also be placed on clinical hold for reasons contained in FDA's existing IND regulations, the final reg notes, "exposure of subjects to unreasonable and significant risks."

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