Table 3.
Comparison of Observed Median Time and Predicted Median Time Estimated using Kaplan–Meier Survival Analysis to Complete Each Phase of the Implementation Process for Statewide Newborn Screening for New Disorders, as of August 2019.
| Mandate/Approval to Start Screening | Fee Increase Implemented | Laboratory Readiness | Follow-Up Readiness | IT Readiness | ||
|---|---|---|---|---|---|---|
| Predictive Median Months from Survival Analysis [95% CI] (number of state NBS programs) |
Pompe | NA + | 24 | 39 | 26 | 16 |
| (27) β | (15) β | (24,68) | (18,36) | (7,37) | ||
| (n = 25) | (n = 27) | (n = 28) | (n = 24) | (n = 19) | ||
| MPS I | 36 * | 49 ** | 39 | 24 | 18 | |
| (18) β | (22,49) | (23,60) | (13,42) | (7,37) | ||
| (n = 25) | (n = 30) | (n = 28) | (n = 24) | (n = 20) | ||
| ALD | NA +,* | 27 | NA + | NA + | 14 | |
| (17) β | (24) β | (31) β | (26) β | (7) β | ||
| (n = 24) | (n = 30) | (n = 30) | (n = 26) | (n = 18) | ||
| Observed Median Months to Complete Phase (Min, Max) (number of state NBS programs) |
Pompe | 18.0 | 16.5 | 23.0 | 21.5 | 12.5 |
| (9,30) | (4,34) | (4,68) | (1,77) | (2,40) | ||
| (n = 9) | (n = 14) | (n = 14) | (n = 14) | (n = 14) | ||
| MPS I | 13.0 * | 19.5 ** | 24.0 | 18.5 | 13.5 | |
| (3,36) | (4,49) | (14,75) | (5,42) | (2,46) | ||
| (n = 11) | (n = 12) | (n = 14) | (n = 14) | (n = 14) | ||
| ALD | 12.0 * | 21.0 | 20.0 | 18.0 | 8.0 | |
| (0,30) | (1,34) | (13,36) | (2,30) | (2,14) | ||
| (n = 9) | (n = 13) | (n = 9) | (n = 9) | (n = 9) | ||
Table excludes newborn screening (NBS) programs that selected “NA” for phase completion variables or did not provide start and/or completion dates. Spinal Muscular Atrophy (SMA) was not included as not enough time had elapsed from its inclusion on the Recommended Uniform Screening Panel (RUSP) to the end of data collection to allow for accurate estimates. Further, education was removed because few states have completed this phase. + The predicted median time could not be calculated because more than 50% of participating programs did not complete the phase. β Upper bound of 95% confidence intervals (CI) could not be calculated because the largest observation was censored. * One NBS program is excluded from analysis because the mandate to screen for MPS I and ALD occurred prior to the start of the phase (i.e., NBS Advisory Committee Approval). ** One NBS program is excluded from Fee Increase Implementation for MPS I as their start date occurred after their completion date.