This includes all clinical data and significant adverse event reports, as well as informed consent and other documents for patients, so the DSMB can provide timely, ongoing evaluation to ensure patient safety. If the CF Foundation-supported DSMB determines that patient safety is at risk, it will recommend to the trial sponsor and to the FDA that the trial be halted. The FDA should be able to share the reports publicly if there is a danger to patients, with protections for patient confidentiality and proprietary information. Several years ago, the CF Foundation established a DSMB to oversee all clinical trials performed through our Therapeutics Development Network. Experience with CF enables DSMB members to best ensure the safety of CF patients because they can determine if any adverse event is due to the trial itself, or to the patient s underlying disease.
Members maintain independence from the clinical investigators and the trial sponsors.
The DSMB also reviews informed consent documents, reports from investigators of any significant adverse events in the trials, balances risks and benefits of the trial for patients, and evaluates other information to enhance patient safety. The makeup of the DSMB will be adjusted to assure that it includes the capability, appropriate expertise, and resources to review all aspects of gene therapy research for safe and responsible clinical trials. With the ongoing political debate in Washington, D.C. on how to best ensure safety of patients in gene therapy trials, the CF Foundation strongly encourages the additional use of Data Safety Monitoring Boards, rather than increased government oversight. We believe gene therapy will be an important future component in the care of CF patients. Therefore, gene therapy trials must proceed in a responsible and timely fashion.