An Estimate of the Benefit-Cost Impact of the FDA Guidance on Data Monitoring Committees.

BACKGROUND

There is little information available about the impact that FDA guidances have on the clinical trial enterprise.

OBJECTIVE

To estimate the impact of the FDA's Guidance for Industry, "The Establishment and Operation of Clinical Trial Data Monitoring Committees for Clinical Trial Sponsors."

METHODS

An economic model was developed to measure the costs and potential savings associated with the change in data monitoring committee (DMC) usage since issuance of the DMC guidance for industry-sponsored clinical trials. To establish the change in use following the issuance of the DMC guideline, a literature search was conducted among high-impact medical journals during publication year 2010 and compared to a similar analysis conducted during publication year 2000. Costs were obtained for DMCs and applicable clinical trials. The results were applied to an analysis of ClinicalTrials.gov completed trials during 2007 to 2013.

RESULTS

Review of 4200 manuscripts from publication year 2010 was compared to a similar literature search of publication year 2000. The mention of DMCs in industry-sponsored randomized controlled trials from high-impact journals increased from 24% to 47% (risk ratio = 1.9, P < .0001). This increased rate of DMCs is associated with an increase of 1045 DMCs for industry-sponsored phase 2 and 3 interventional trials that were commenced and completed from 2007 to 2013 and were listed in ClinicalTrials.gov . The increased cost due to these additional DMCs was approximately US$231 million, and the savings associated with early termination of clinical trials due to these DMCs was approximately US$428 million.

CONCLUSION

The DMC guidance has had a net positive economic impact on the clinical trial enterprise. However, noneconomic factors need to be evaluated. ClinicalTrials.gov could be further leveraged to explore further noneconomic benefits and costs of DMCs.