Prescription Drugs:

FDA Guidance and Regulations Related to Data on Elderly Persons in Clinical Drug Trials

GAO-07-47R: Published: Sep 28, 2007. Publicly Released: Oct 29, 2007.

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Elderly persons use drugs at a higher rate than younger persons, in part because elderly persons experience higher rates of certain diseases--such as cancer, Parkinson's disease, and heart disorders. Elderly persons--those age 65 and older--are also more likely than younger adults to experience complications when taking some prescription drugs. For example, because of their decreased liver and kidney functions, elderly persons often lack the ability to eliminate drugs from their bodies as efficiently as younger adults, making elderly persons more likely to experience side effects associated with drugs. As a result, the Food and Drug Administration (FDA) has noted that it is important that drugs be studied for use by elderly persons during the clinical drug trials--that is, those drug studies conducted by drug sponsors before they submit an application to have a drug approved for marketing. FDA is responsible for oversight of clinical drug trials and deciding whether to approve new drugs for marketing in the United States. This responsibility includes determining if drugs are safe and effective for the people expected to use them, including elderly persons. To implement its responsibilities, FDA issues guidance and regulations for drug sponsors on conducting clinical drug trials and submitting new drug applications (NDA) to FDA to seek drug approval based in part on the results of those clinical drug trials. NDAs include data on both the safety and effectiveness of the drug being studied. FDA guidance describes the process FDA medical officers (typically doctors) are to follow in reviewing those NDAs as part of the NDA review process and recommending whether to approve a new drug for marketing in the United States. As part of the process by which drugs are considered for approval, medical officers also meet in teams with supervisors and other experts, including biochemists and statisticians, to discuss the merits of the NDA. Concerns have been raised about the inclusion of elderly persons in clinical drug trials.7 You asked us to examine FDA's activities related to data reported about elderly persons in the study of new drugs. In this report, we examine FDA's guidance and regulations related to (1) drug sponsors' reporting of data to FDA to describe the effects of a proposed drug on elderly persons and (2) FDA medical officers' review of safety and effectiveness data that drug sponsors provided for elderly persons. Assessing whether a drug is safe and effective for use by elderly persons requires that a sufficient number of elderly persons be included in clinical drug trials. As the number of elderly participants in clinical drug trials increases, the ability of drug sponsors to detect responses unique to elderly persons for a given safety or effectiveness outcome also increases. Failing to include sufficient numbers of elderly persons in clinical drug trials may make it less likely that safety concerns and effectiveness outcomes unique to elderly persons will be detected during the clinical drug trials. FDA's guidance recommends that drug sponsors not exclude elderly persons from clinical drug trials on the basis of their age alone because such exclusions might prevent drug sponsors from collecting sufficient information about the effects of drugs in elderly persons.20 Instead, the guidance suggests that, when necessary, exclusion criteria should focus on more relevant issues, such as either the presence of an illness that could make participation in the clinical drug trial dangerous or the person's inability to provide informed consent.

FDA guidance recommends that drug sponsors include elderly persons in clinical drug trials, and FDA regulations require that the drug sponsors report clinical drug trial data by age. The agency guidance recommends that drug sponsors avoid excluding persons from clinical drug trials on the basis of advanced age and offers various suggestions on how drug sponsors should report the age of clinical drug trial participants to help FDA determine the number of elderly participants. To identify differences in the safety and effectiveness of a drug associated with age, FDA regulations require that drug sponsors report clinical drug trial data by age. For the 36 NDAs we reviewed, drug sponsors generally included elderly persons and reported safety and effectiveness data for elderly persons in clinical drug trials. FDA officials are developing guidance that would combine information currently available in multiple guidance documents on the format drug sponsors should use for reporting safety data in an NDA, including data about age. FDA guidance recommends that medical officers determine whether the proposed drugs are safe and effective for the populations expected to use them, but the guidance does not mention elderly persons specifically. FDA officials told us that the agency expects that medical officers will assess the safety and effectiveness of a drug for elderly persons when they review data in an NDA; however, these expectations are not conveyed in agency guidance. We found that about two-thirds of the medical officer reviews we examined included a discussion by the medical officer of the safety and effectiveness of the drug for elderly persons. However, agency guidance does not direct medical officers to report whether sufficient numbers of elderly persons participated in NDA clinical drug trials to assess the safety and effectiveness of a drug for elderly persons. We found that about one-quarter of the medical officer clinical review summaries that we reviewed documented the medical officer's review of the sufficiency of representation of elderly persons. FDA officials told us that the agency's reviewers understand that the request in agency guidance for an analysis of a drug's effects by age includes an analysis of the drug's effects upon elderly persons. They also said that age differences are almost always discussed during team meetings. FDA officials added that approval recommendations are not made independently by one medical officer, but rather result from discussions among medical officers and others on a review team.

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