Summary: In response to a congressional request, GAO reviewed the clinical testing of anticancer drugs and the regulation of that testing by the Food and Drug Administration (FDA) to determine: (1) how well, during its review of new anticancer drug applications, FDA discharges its responsibility to protect human test subjects; (2) the manner in which drug sponsors and institutional review boards carry out their responsibilities; and (3) whether there is therapeutic intent during testing of anticancer drugs.
GAO found that FDA and the National Cancer Institute (NCI) have made some improvements to better ensure that patients involved in clinical testing of anticancer drugs are protected. These improvements include: (1) more monitoring of investigators who perform the clinical studies for the sponsors; (2) improved reporting of adverse drug reactions (ADR); and (3) increased controls over investigational new drug (IND) supplies. However, GAO believes that additional actions are needed. Delays by FDA reviewers in completing written reports on deficiencies resulted in FDA sending deficiency letters to sponsors 2 to 5 months after an application was received. GAO found instances where FDA was notified that its recommendations were not implemented because of a lack of a reporting system. Also, sponsors do not always submit amendments to FDA for review and, when they are submitted, FDA frequently does not review them or does not review them in a timely manner. GAO found 12 protocols involving five drugs that had not been submitted to FDA for review. FDA regulations are not specific regarding: (1) the definition of ADR; and (2) the time frames during which these reactions should be reported to FDA. In addition, it is difficult to determine in many instances whether a change in a patient's condition was caused by IND or by some other factor. Finally, GAO found that NCI does not require monitoring visits, but plans to expand the number of investigators visited.
