The CEA Registry Blog

Jan 24

by CEA Registry Team 1/24/2017 3:44 PM  RssIcon

 By Paige Lin, Ph.D.

Would you test your newborn for a rare but serious genetic disorder for which no direct treatment is available?
 
In a paper published this week in Pediatric Neurology, my colleagues and I demonstrate that most people would prefer testing their newborn for Spinal Muscular Atrophy (SMA) and express a reasonably high willingness to pay for the test, even in the absence of direct treatment [1].
 
SMA, the most fatal genetic neuromuscular disease among children, affects approximately 1 in 10,000 infants [2]. While early diagnosis can provide a window of opportunity for early intervention, current mandatory newborn screening programs in the United States do not require SMA testing primarily due to lacking disease-modifying therapy, until very recently [3].
 
In order to gauge the public’s preferences for SMA testing for newborns, we surveyed 982 US adults and asked how much, if any, they would pay out-of-pocket for the test using a bidding game approach. Each respondent was presented with two scenarios: the standard of care (no available treatment) and one of three hypothetical substitute scenarios that were randomly assigned:
 
  1. Improved functioning: New treatment improves functioning, but does not affect survival
  2. Improved survival: New treatment may improve survival, but it does not improve functioning
  3. Improved functioning and survival: New treatment would improve both functioning and survival
 
From this survey, we found that most people (79%) would prefer to test their newborn for SMA even without a treatment available.  A greater proportion (85% to 86%) would prefer the test if a treatment improved either functioning or survival, respectively. The vast majority (87%) would opt to pay for the test if an available treatment improved both functioning and survival. Respondents also demonstrated an equitably high willingness-to-pay value for the test (average $253 to $297).
 
Despite preferences for the test information, adding SMA to national newborn screening programs remains controversial because currently there is insufficient evidence on long-term patient outcomes as a result of early identification and early treatment for SMA at a population level. Our results provide insight into how the general public views the value of SMA testing, and how potential treatment benefits may affect their testing decisions.  Our findings add a new dimension to the discussions parents, physicians, and policymakers should have on whether SMA screening should be legislatively mandated.    
 
Tufts Medical Center received research funding support for this study from Biogen, which markets the first FDA-approved SMA treatment SPINRAZA™ (nusinersen).
 
[1] Lin PJ, Yeh WS, Neumann PJ. Willingness to Pay for a Newborn Screening Test for Spinal Muscular Atrophy. Pediatr Neurol. 2017 Jan;66:69-75. doi: 10.1016/j.pediatrneurol.2016.09.008. PubMed PMID: 27769729.
[2] Dolgin E. Call in the backup. Nat Med. 2012; 18:1602-1606.
[3] U.S. Food & Drug Administration. FDA approves first drug for spinal muscular atrophy [Press release]. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm

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