Common use of Survey Design Clause in Contracts

Survey Design. This cross-sectional survey was administered online using REDCap, a secure online data collection platform.12 Respondents were asked to self-report demographic data and practice characteristics [i.e. gender, race, ethnicity, medical specialty, years in practice, practice type (academic practice/private practice/other), practice location (urban/suburban/rural), involvement in NF research, affiliation with the Children’s Tumor Foundation NF Clinic Network, and whether their practice currently included pediatric and adult NF1, NF2, and schwannomatosis patients.] The research team identified six relevant NF guideline publications3-8 via literature review to include in the survey (as French guidelines from Xxxxxxxxx et al. (2020) were published after this survey was administered). All respondents were asked to identify whether they were previously aware, unaware, or unsure about each publication [which were identified by first author, title, journal, publication year, PubMed ID and primary topic area (i.e. pediatric NF1)]. Respondents were asked about their awareness of all six NF guideline publications, even if their practice did not currently include the specific NF patient population addressed by the publication, under the assumption that clinicians should have broad knowledge of these resources to remain current with the field, to be prepared to coordinate care for new patients, and to help pediatric patients transition to adult care. One author (JTJ) extracted 40 individual guidelines from five guideline publications4-8 for assessment of guideline agreement (as pediatric NF1 guidelines from Xxxxxx et al. (2019) were published after this section of the survey was finalized). Guidelines were largely extracted verbatim, with minor changes in wording to combine nearly identical guidelines from multiple publications or to improve grammar (see Table 1 for full list of guidelines). All guidelines were assessed by two authors (JTJ and VLM) to determine the relevant patient population addressed (i.e. pediatric or adult; NF1, NF2, or schwannomatosis). Respondents were asked to rate their agreement with each individual guideline relevant to their practice population using a 5-point Likert scale (strongly agree to strongly disagree, with neutral as the midpoint). Respondents could also optionally provide free-text comments about any guideline. The study size was determined by the number of eligible respondents who completed at least one guideline agreement rating. Descriptive data on all variables are reported as frequencies and percentages. Only questions assessing study eligibility and determining the subpopulations of NF included in each clinicians’ practice were mandatory; all other questions were optional, and percentages are reported out of the sample size of non-missing responses unless otherwise noted. Due to the risk of acquiescence bias (the tendency for survey respondents to select positive response options)13, we focused our reporting of guideline agreement on the percentage of respondents who selected “strongly agree”, thus actively indicating a strong preference in favor of each guideline. As clinicians’ level of support for a clinical practice guideline is associated with their adherence to guidelines is practice14,15, this type of top-box analysis (used often in patient experience surveys)16-18 may best predict clinicians’ real-world behavior. We calculated the 95% confidence interval for the percentage of respondents who strongly agreed with each guideline using standard formulas for margin of error of a proportion; average margin of error across all guidelines was 12.7% (Supplementary Information). We examined whether clinician demographics or practice characteristics were correlated with level of agreement with NF clinical guidelines. Given the large number of individual guidelines and respondent subgroups of interest (and concomitant risk of Type 1 error from performing multiple comparisons), we assessed clinicians’ overall agreement with all clinical guidelines relevant to their own practice populations. To do this, we calculated the proportion of guidelines to which each clinician ‘strongly agreed’ as a proportion of the total number of guidelines for which they completed ratings. This proportion could range from 0 (does not strongly agree with any guidelines) to 1 (strongly agrees with all guidelines). This proportion was compared across respondent subgroups used t-tests or ANOVA as appropriate. Two NF1 guidelines publications were endorsed by U.S. medical societies – Xxxxxx et al. guidelines for pediatric NF1 published by the American Academy of Pediatrics (AAP) and endorsed by the American College of Medical Genetics and Genomics3 (ACMG) and Xxxxxxx et al. guidelines for adult NF1 published by ACMG4. To better understand guideline dissemination patterns, we assessed whether clinicians in these professions (pediatrics and medical genetics, respectively) were more likely to report being aware of guideline publications than clinicians in other specialties using Xxxxxx’x exact test. A p-value of ≤0.05 was considered statistically significant for all tests. Finally, we used qualitative content analysis to summarize respondent’s free-text comments.19 Eighty-two U.S. based clinicians accessed the survey; twelve did not complete any questions beyond eligibility screening and an additional seven did not complete any guideline agreement questions, resulting in a final analytic sample of 63 people [63/358 (17.6%) of potentially eligible respondents, 63/82 (76.8%) of eligible respondents who accessed the survey]. Clinician demographics, practice characteristics, and type of NF specialization are shown in Table 2. Clinicians represented >8 medical or surgical specialties, with one-third (n=21) of respondents in neurology or neuro-oncology. Clinicians were located across the U.S., representing 26 U.S. states with the greatest number of respondents from New York, California, Florida, Massachusetts, and Minnesota (respectively). Most respondents (n=50, 79.4%) provided clinical care as part of a specialized neurofibromatosis clinic affiliated with the Children’s Tumor Foundation NF Clinic Network20 and the majority were involved in NF research (n=39 for clinical trials and n=47 for non-treatment research studies such as tumor banks, natural history studies or questionnaire-based research). These results are similar to the geographic distribution and proportion of NF Clinic Network clinics who were involved in clinical trials at this time [31/56 (55.4%) of clinics compared to 61.9% of survey respondents, per Children’s Tumor Foundation internal 2019 annual report]. Clinicians self-reported their awareness of publications containing NF clinical care guidelines (Table 3). The percentage of respondents who were aware of each guideline set ranged from 53.2% (for schwannomatosis guidelines within Xxxxx et al. 2017) to 79.4% (for pediatric NF1 guidelines published by Xxxxxx et al. 2007). Among only those respondents who reported currently seeing the relevant patient population, awareness was only marginally increased (by 1.1 to 5.1 percentage points), with the exception of adult NF2 guidelines by Xxxxx et al. 2005, in which awareness increased 11.6 percentage points to 65.7% of respondents. Overall, 26-36% of respondents were unaware of recently published guideline documents (2017-2019). Medical geneticists were significantly more likely to report awareness of adult NF1 guidelines endorsed by the ACMG than clinicians of other specialties (100% of medical geneticists vs. 56.1% of other specialists, p=0.0008). Pediatricians and medical geneticists were also more likely to report awareness of pediatric NF1 guidelines endorsed by the AAP and the ACMG (87.0% of pediatricians and medical geneticists vs. 55.6% of other specialists, p=0.021). Overall, less than half of survey respondents had strong agreement with 16/40 (40%) of NF guidelines. Clinicians’ level of agreement with NF1 guidelines are presented in Figure 1; number of respondents and 95% confidence intervals are available in supplementary table 1. Level of strong agreement with individual guidelines ranged from 17% to 83%. Strong agreement was highest for the preference of MRIs over CT scans to reduce exposure to ionizing radiation exposure (83%); annual blood pressure checks (80%); education about signs and symptoms of malignant peripheral nerve sheath tumors (76%); and annual check-ins on development and school progress for pediatric patients (76%). For 9/26 (34.6%) of NF1 guidelines, less than half of respondents selected ‘strongly agree’. These guidelines addressed breast cancer screening, counseling regarding family planning, vitamin D supplementation, evaluation of hypertension, pregnancy management, assessment of glomus tumors, and use of whole-body MRI. Clinicians’ level of agreement with NF2 and schwannomatosis guidelines are presented in Figure 2; number of respondents and 95% confidence intervals are available in supplementary table 1. Level of strong agreement with individual NF2 guidelines ranged from 36% to 73%. Strong agreement was highest for receiving care at specialized NF clinics (73%) and for receiving care annually at an NF clinic (72%). For 4/11 (36.4%) of NF2 guidelines, less than half or respondents selected ‘strongly agree’. These guidelines addressed the frequency of spinal MRIs in all patients and the timing of brain MRIs for pediatric patients. Agreement was noticeably lower for pediatric imaging recommendations including the provision to start surveillance at age 10 when compared to identical adult guidelines (absolute difference of 15.4 percentage points for brain MRIs and 9.3 percentage points for spinal MRIs). Level of strong agreement with three schwannomatosis guidelines addressing genetic testing for younger patients with potential schwannomatosis and the timing of brain and spine MRIs (including age at baseline scan and frequency of imaging) ranged from 27-38%. The median proportion of guidelines with which each clinician strongly agreed was 0.55 (range, 0-1; 25th-75th percentile, 0.35 to 0.73). There were no statistically significant differences in guideline agreement proportion based on clinicians’ gender, race, specialty, years in practice, practice type, practice location, participation in the NF Clinic Network, involvement in NF treatment trials, or involvement in non-treatment NF research. Medical geneticists were no more likely to than other specialists to strongly agree with adult NF1 guidelines endorsed by the ACMG (agreement index 0.52 vs. 0.53, p=0.89). As guidelines were not systematically extracted from the pediatric NF1 guidelines endorsed by the AAP and ACMG, a parallel test of pediatricians’ agreement with these guidelines was not performed. Twenty optional free-text comments regarding NF guidelines were collected from eleven survey respondents. While there were insufficient comments to robustly explain disagreement with individual guidelines, categories of comments may illuminate potential sources of disagreement with NF guidelines in general. Comments were categorized into five main groups, in descending order of frequency: 1) disagreement about the timing of services recommended by a guideline; 2) disagreement that a guideline should apply to the entire population equally; 3) perception that guidelines were not in the provider’s scope of practice; 4) confusion about the way a guideline was worded; and 5) suggested addition to a guideline.