The high cost of source data verification (SDV), particularly in large trials, has made it a target of scrutiny over the last decade. In addition, the positive impact (ie, cost-benefit ratio of SDV) on overall data quality is often questioned. As a result, regulators and industry groups have started looking at alternative SDV approaches. This article evaluates the FDA-supported risk-based approach to SDV and provides a proposal on how to modify the SDV process without undermining the validity and integrity of the trial data. It summarizes alternative approaches to 100% SDV and evaluates the advantages and disadvantages of risk-based SDV (rSDV). The regulatory, data quality, and cost implications of each approach are considered. The economics of rSDV are discussed and the cost implications of rSDV are presented based on the results of exploratory analyses for four hypothetical trials in cardiology and oncology.

The Infant Health and Development Program was an eight-site randomized controlled trial testing the efficacy of early intervention to enhance the cognitive, behavioral, and health status of low birth weight, premature infants. The 377 intervention families received for the first 3 years of life: (1) pediatric follow-up, (2) home visits, (3) parent support groups, and (4) a systematic educational program provided in specialized child development centers. The control group (n = 608) received the same pediatric follow-up and referral services only. This paper describes the delivery of the intervention and its outcomes. A Family Participation Index that was the sum of participation frequencies in each of the program modalities unique to the intervention revealed that program implementation was not different across the eight sites. Index scores did not vary systematically with mother's ethnicity, age, or education or with child's birth weight, gender, or neonatal health status; but they were positively related to children's IQ scores at age 3. Only 1.9% of children of families in the highest tercile of participation scored in the mentally retarded range (IQ ≤70), whereas 3.5% and 13% of children in the middle and lowest participation terciles, respectively, scored in the retarded range. Similar findings were obtained for borderline intellectual functioning. These findings are consistent with previous research linking intensity of intervention services with degree of positive cognitive outcomes for high-risk infants. The determinants of variations in individual family participation remain unknown.

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It is recommended that SDV, rather than just focusing on key primary efficacy and safety outcomes, focus on data clarification queries as highly discrepant (and the riskiest) data.