Summary

In fiscal year 1995, the National Institutes of Health (NIH) sponsored about $9 billion in extramural research--research done by groups outside of NIH. About $1.2 billion was spent on Phase III clinical trials, which usually involve hundreds of human participants to evaluate experimental treatments. In the early 1990s, disclosure that falsified data had been used in a large Phase III trial looking at alternative treatments for breast cancer raised concern that the results of this multimillion dollar trial had been compromised. This report discusses NIH's oversight responsibilities and internal controls used to prevent and detect misconduct in Phase III clinical trial research. GAO also reviews NIH's approach to monitoring performance of its institutes that sponsor clinical trials and efforts to implement agencywide policy on misconduct in research.

GAO found that: (1) individual NIH institutes and grantee institutions oversee and monitor NIH-funded Phase III clinical trials; (2) internal controls to guard against fiscal misconduct in extramural research must comply with regulations and policies on federal funds expenditures and be consistently applied; (3) independent auditors review grantees' compliance with these internal control regulations during annual audits; (4) NIH imposes sanctions on offending grantees when scientific misconduct occurs; (5) internal controls to guard against scientific misconduct and ensure participants' safety are generally consistent, but they vary slightly among trials due to differences in the sponsoring institute's management philosophy and past experience, the trial's size, nature, and complexity, and the way the trial is funded; (6) NIH institutes and grantees use clinical monitors and data coordinating centers to ensure data integrity, but these controls are not consistently applied; (7) one institute provides direct funding for certain data verification functions which ensures prompt reporting of data concerns and potential misconduct to NIH, but this is not an agencywide policy for multisite trials; (8) central NIH oversight and monitoring of clinical trials is limited; and (9) NIH has not adopted on an agencywide basis, any of its study committee's recommendations to improve clinical trial oversight because it believes agencywide monitoring policies are inappropriate.