Demographic information

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1 Thank you for taking the time to complete this survey about comparative effectiveness research in neonatology. It should take minutes to complete. Please be assured that your responses are anonymous. Everyone accesses this survey with the same web address; your home or work computer internet address are not traced or identified. If you are called away from the survey and are unable to complete it, you may access it again and retake it. Only completed surveys will be analyzed in this study. Your voluntary participation implies your permission to analyze these anonymous responses for the purpose of research with possibility of sharing our findings with the clinical and academic communities via poster, presentation, or publication. This survey has been approved by the IRB of NorthShore University HealthSystem's Evanston Hospital.

2 Demographic information 1. Which best describes your specialty? (Choose one.) neonatologist general pediatrician other pediatric subspecialist other

3 2. How do you spend your time at work? majority research, some clinical care equal time research and clinical care majority clinical care, some research 3. Which best describes your years of experience? fellow attending for <5 years attending for 5 9 years attending for 10 to 20 years attending for over 20 years

4 4. How has your research been funded in the last 5 years? (Please mark all that apply.) my own institution private endowment/grant professional organization grant federal funding source 5. Are you a member of an Institutional Review Board? Yes No 6. Have you conducted any comparative effectiveness research (comparing standard care therapies)? Yes No 7. Regarding the OHRP s Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care (DG), which best applies to you? I ve never heard of it until now I know a little about it I ve read it

5 8. If you ve read it, please mark all that apply regarding providing feedback to the OHRP: Not applicable I haven't read it I did not submit a comment I submitted a written comment individually or with others I attended the Institutes of Medicine meeting on the Draft Guidance

6 Please consider this proposed study to answer the remaining survey question... Disclaimer: A January 2015 search of clinical trials.gov for trials concerning did not reveal studies similar to the one presented here; any likeness to an actual study in process is purely coincidental. Treatment of hyperbilirubinemia of the preterm infant Study question: For premature infants (28 to 34 weeks gestation), what are the optimal thresholds for treating hyperbilirubinemia? Background: Evidence based guidelines for treating hyperbilirubinemia in babies born <35 weeks gestation do not exist. Practices are variable among NICUs and among individual neonatologists. In the U.S., consensus based guidance has been published in the literature (Maisels et al., Journal of Perinatology 2012), though it does not meet the American Academy of Pediatrics' criteria for evidence based guidelines. The United Kingdom s National Institute for Health and Care Excellence (NICE) has published consensus based guidelines for preemies down to 23 weeks based on extrapolations from a weight and gestation based formula. Per the authors of both guidelines, thresholds for treatment are similar to clinical practice. It is unclear at what level of serum bilirubin a baby should receive phototherapy or exchange transfusion to avoid kernicterus. While premature babies are at higher risk for developing kernicterus, there is also evidence that aggressive phototherapy may be harmful to this population. A large prospective randomized controlled trial in the U.S. (Tyson, et al.) found that aggressive phototherapy may increase mortality in the sickest ELBW infants while decreasing impairment and profound impairment for other preemies on follow up at months corrected gestational age. Maisels, et al. suggest long term follow up comparisons of large populations of babies treated by their guidance vs. those treated by the more aggressive NICE guidelines to help choose the best approach. This study aims to determine which approach is safest for premature infants treatment at higher bilirubin thresholds (Maisels) or lower bilirubin thresholds (NICE) with phototherapy and exchange transfusion. Eligibility criteria: Babies between 28 0/7 weeks to 34 6/7 weeks of corrected gestational age will be included. Because the Maisels guidance leaves treatment of earlier gestational ages open to far greater variability (including consideration of prophylactic phototherapy for babies < 26 weeks), those younger than 28 weeks will be excluded. Babies 35 0/7 corrected gestational weeks and older are excluded because evidence based AAP

7 guidelines are available. Study design: Upon enrollment, we will randomize babies at multiple centers to receive phototherapy and exchange transfusions based either on guidance published by Maisels, et al. (Group A) or that of NICE (Group B). Serum bilirubin will be sampled at least once in the first 24 hours and again between hours, with additional levels drawn at the clinician's discretion. The NICE guidelines provide chronological age based graphs for each week of corrected gestation. The guidance by Maisels et al. gives a table of bilirubin ranges for each week of corrected gestation with instructions on choosing lower or higher ends of the range based on several clinical factors. Strictly for purposes of comparison for you as a study reviewer, a table has been constructed for bilirubin levels at phototherapy thresholds below. Clinicians caring for study patients will be following the original published guidance. Outcomes: 1.) kernicterus (based on clinical findings or autopsy if available); 2.) mortality; 3.) level of impairment at follow up around 20 months corrected gestational age; 4.) developmental indices upon entering kindergarten Informed consent: Formal informed consent will be obtained in the first day of life. We believe that there is genuine uncertainty about which of these widely used treatment approaches is best for these babies.

8 9. What guides your treatment of hyperbilirubinemia for NICU patients 28 to 34 weeks gestation? Guidance by Maisels, et al. (Journal of Perinatology, 2012) NICE guidelines Formula based on weight, age, or both (e.g., 5 x weight in kg) I do not use one particular rule or guideline Not applicable I do not provide clinical care in the NICU 10. Does enough equipoise exist to justify randomizing infants as proposed? Yes No

9 11. (Optional) Why do you think equipoise does not exist to accept this as an ethical study? IRBs have to consider what level of risk studies carry. Which statement about "minimal risk" best applies to this study? 6 No comment I'm not familiar with the "minimal risk" concept. This study is not minimal risk because risks of participating in the study are greater than those ordinarily encountered in daily life or during the performance of routine physical or psychological examinations or tests. This study is not minimal risk because risks of participating in the study are greater than risks of standard neonatal intensive care management of hyperbilirubinemia. This study is minimal risk because risks of participating in the study are similar to those ordinarily encountered in daily life or during the performance of routine physical or psychological examinations or tests. This study is minimal risk because risks of participating in the study are similar to risks of standard neonatal intensive care management of hyperbilirubinemia.

10 Please read the following excerpt from OHRP s draft guidance to answer the... "For comparative effectiveness or standard of care research, OHRP s general position is that the reasonably foreseeable risks of research include already identified risks of the standards of care being evaluated as a purpose of the research when the risks being evaluated are different from the risks subjects would be exposed to outside of the study." 13. Based on this OHRP excerpt, which risks must be disclosed in this study s informed consent document?(please mark all that apply.) Risk of phototherapy rash Risk of dehydration Risk of death Risk of impairment or profound impairment Risk of school age developmental problems Risk of kernicterus Risk of exchange transfusion and its associated risks: necrotizing enterocolitis, electrolyte abnormalities, thrombocytopenia, infection None of these 14. How certain are you that you interpreted the OHRP statement correctly as it relates to determining which foreseeable risks must be disclosed for this study? Very certain whether I Very uncertain whether I Neutral interpreted correctly interpreted correctly

11 Please read the following excerpt from 46.11(a)(2) of the Common Rule to an... the IRB should consider only those risks and benefits that may result from the research (as distinguished from risks and benefits of therapies subjects would receive even if not participating in the research). 15. Based on this Common Rule excerpt, which risks must be disclosed in this study s informed consent document?(please mark all that apply.) Risk of phototherapy rash Risk of dehydration Risk of death Risk of impairment or profound impairment Risk of school age developmental problems Risk of kernicterus Risk of exchange transfusion and its associated risks: necrotizing enterocolitis, electrolyte abnormalities, thrombocytopenia, infection None of these 16. How certain are you that you interpreted the Common Rule statement correctly as it relates to determining which foreseeable risks must be disclosed for this study? Very certain whether I Very uncertain whether I Neutral interpreted correctly interpreted correctly

12 17. Do you usually obtain consent for starting phototherapy? Yes No Not applicable I do not provide clinical care in the NICU 18. Do you usually obtain consent for exchange transfusion for hyperbilirubinemia? Yes No Not applicable I do not provide clinical care in the NICU 19. For a premature baby receiving phototherapy, which risks do you review (formally or informally) with parents? Risk of phototherapy rash Risk of dehydration Risk of death Risk of impairment or profound impairment Risk of school age developmental problems Risk of kernicterus Risk of exchange transfusion and its associated risks: necrotizing enterocolitis, electrolyte abnormalities, thrombocytopenia, infection None of these Not applicable I do not provide clinical care in the NICU 20. Please indicate your agreement with this statement: Randomizing a baby to one of the study groups puts baby at an overall higher risk of harm than if baby is not enrolled in the study. Strongly agree Neutral Strongly disagree 21. If your child were eligible for this study, would you give consent for enrollment? Yes No

13 22. Please indicate which group carries: Group A (Maisels: phototherapy at Group B (NICE: phototherapy at lower higher bilirubin thresholds) bilirubin thresholds) Unknown Higher risk of phototherapy rash Higher risk of dehydration Higher risk of death Higher risk of impairment or profound impairment Higher risk of school age developmental problems Higher risk of kernicterus Higher risk of exchange transfusion 23. Please indicate which group carries: Group A (Maisels: phototherapy at higher bilirubin thresholds) Patient in your unit not in the study Unknown Higher risk of phototherapy rash Higher risk of dehydration Higher risk of death Higher risk of impairment or profound impairment Higher risk of school age developmental problems Higher risk of kernicterus Higher risk of exchange transfusion 24. Please indicate which group carries: Group B (NICE: phototherapy at lower bilirubin thresholds) Patient in your unit not in the study Unknown Higher risk of phototherapy rash Higher risk of dehydration Higher risk of death Higher risk of impairment or profound impairment Higher risk of school age developmental problems Higher risk of kernicterus Higher risk of exchange transfusion

14 25. (Optional) Please share any additional comments about comparative effectiveness research in neonatology, research ethics and regulations, or this study here. 5 6

15 Thank you so much for sharing your views with us! Your time is greatly appr... Your response has not been traced. Could you please Dalia Feltman at to let us know you completed the survey? That will ensure you will not be bothered by future reminder s.