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2. Evaluation of NIH Pediatric Inclusion Policy: Data from the NICHD Dr. Duane Alexander, Director Eunice Kennedy Shriver National Institute of Child Health and Human Development

3. Introduction

4. History of the NIH Pediatric Inclusion Policy • Historically, women, children, minorities were often excluded from research -- concerns about exploitation • Renewed efforts to include minorities and women in research followed HIV/AIDS epidemic • 1993: NIH Revitalization Act mandated inclusion of women and minorities in research • 1994: NIH published policy on inclusion of women and minorities • 1994-1995: NIH asked to develop a policy on inclusion of children • 1996: NIH held workshop on inclusion of children • 1998: NIH policy on inclusion of children implemented

5. The NIH Pediatric Inclusion Policy Policy Objective: Increase the participation of children in research so that adequate data will be developed to support treatment for disorders and conditions that affect adults and may also affect children Potentially Pediatric and Adult Pediatric only Adult only

6. The NIH Policy on Inclusion of Children in Research • Policy states that children must be included in all human subjects research – • Unless there are scientific and ethical reasons not to include them • Policy covers all NIH conducted or supported research, including research otherwise “exempt” from HHS policy for protection of human research subjects • “Children” are defined as individuals under the age of 21

7. Policy Implementation • Applicant responsibilities: • Describe plan to appropriately include children in research • If not including children under 21, applicant must justify exclusion, using one of the 7 specific reasons outlined in the policy • Study section responsibilities: • Evaluate proposed plan and scientific rationale for inclusion or exclusion of children • Assign appropriate codes to application • (e.g. includes children, plan acceptable)

8. Policy Implementation • Scientific review administrator responsibilities: • Include appropriate expertise for review of applications involving children • Describe rationale for inclusion/exclusion in summary statement • Institute/center responsibilities: • For applications coded unacceptable, resolve compliance issues before funding • Fund only applications that fully comply with the policy on inclusion of children

9. Evaluation Questions How often are children included? How often are only certain groups of children included? How often do PIs plan to analyze data by age? When children are not included, why not? How did the study sections implement the NIH policy?

10. Scope and Methodology • The data you will see today encompass: • NICHD type 1 and 2 grants funded in FY 2007 • With human subjects • Where the topic is not inherently exclusively pediatric • Plus a separate analysis of NICHD 2007 grants that were rated “unacceptable” for inclusion of children, whether they were funded or not

11. Scope and Methodology • 397 grants reviewed in their entirety • Information recorded on ages included, ages analyzed, and reasons for inclusion or exclusion • Standardized rules developed to cover specific situations • “reproductive age” (i.e. teen mom rule) • categories (e.g. “school age”, “grade 2”) • secondary data analysis (e.g. NLSY) • Rigorous, detailed procedures for calibration and quality control

12. Key Limitation • We have only what is available in the application and summary statement therefore • We can only assess the PI’s intent and plan to include children—not whether the PI actually did include children

13. Rates of Inclusion of Children

14. Inclusion of Children for NICHD Funded Grants, 2007

15. Variables Associated with Inclusion of Children • Grant mechanism – R01s most likely to include kids, SBIRs least likely • Branch/subject matter – some programs more likely to include children than others • PI degree – MDs more likely to include kids than PhDs or others • Priority score – grants that included children received slightly better priority scores

16. Analysis of Data by Age

17. PI Plans for Analysis by Age, for Grants Including Children < 21

18. Reasons for Exclusion of Children

19. Reasons Under Policy for Exclusion of < 21 • Condition being studied is not relevant for children • Laws or regulations barring inclusion of children • The issue was already studied in children—including them would be redundant • A separate study is warranted or preferable because of adult/child differences • Insufficient data available in adults to judge the risk to children • Study is collecting additional data on pre-enrolled adult participants • Other reasons acceptable to the review committee and IC Director

20. Most Common Reasons Given for Exclusion of < 21 • Condition being studied is not relevant for children (51 %) • Laws or regulations barring inclusion of children (0 %) • The issue was already studied in children—including them would be redundant (0 %) • A separate study is warranted or preferable because of adult/child differences (35 %) • Insufficient data available in adults to judge the risk to children (2 %) • Study is collecting additional data on pre-enrolled adult participants (2 %) • Other reasons acceptable to the review committee and IC Director (10 % —including grants with no justification)

21. Justifications for Exclusion of Children Under 18 • Justification required for exclusion of under 21, but not for under 18 • Of studies excluding children under 18, 45 % provided justification • Reasons for exclusion of children under 18 were similar to reasons for exclusion of children under 21 • But there were a few (n=6 of 87) cases where cost or convenience was cited

22. Implementation of Policy by Study Sections

23. Variation in Inclusion of Children by Study Section • Most study sections reviewed only a small number of NICHD grants, so no conclusions could be made about study section behavior • Study section differences were consistent with subject matter CSR study sectionincluding rehab topics NICHD broad-basedstudy section

24. Grants Rated “Unacceptable” on Inclusion of Children • Rate of “unacceptables” ranged from 1 to 3 percent across NIH ICs • 1% (n=24 of 2720) of NICHD FY 2007 applications were rated “unacceptable” • 3 of these were resolved and later funded • Reasons for “unacceptable” ratings • No justification provided for exclusion of children (n=11, 46%) • Ages of subjects not given (n=8, 33%) • Concerns about human subjects protection (n=2, 8%) • Other (n=3, 13%)

25. Variables Associated with Rating of “Unacceptable” • PI degree – PhDs over-represented in group with “unacceptable” rating (19 of 24, or 79.2 %) • Grant mechanism – SBIR/STTR grants more likely to have “unacceptable” rating (6 of 24, or 25 %) • Study section – Proportionally more “unacceptable” ratings came from the PN study section (5 of 24, or 20.8 %)

26. Implications for the Future

27. When Does Adulthood Begin?

28. Implications of NICHD Data • Children were included in NICHD grants funded in 2007 • 87 % include under 21, 13% include over 21 only • 65 % include under 18, 35 % include over 18 only • Of the grants that restricted subjects to 18 and over: • Only a few (6%) directly cited reasons of convenience for choosing 18 and over; • Yet over half (55%) volunteered no reason for choosing 18 and over • Applicants, reviewers do not always consistently define adulthood as 21+

29. Implications of NICHD Data • Further education about policy may be useful • SBIR/STTR applicants may be a useful target group for education about policy • Inclusion of children is not sufficient to ensure that PIs will produce age-specific data • Reviewers need to discuss whether including only subjects 18 and older meets the intent of the NIH policy

30. Thank You!