Methodological and Policy Issues Related to the Cost-Effectiveness of Newborn Screening in Severe Pediatric Genetic Disorders

Author(s)

Moderator: Peter Neumann, ScD, Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
Panelists: Jamie Sullivan, MPH, EveryLife Foundation For Rare Diseases, Washington, DC, USA; Scott Grosse, PhD, Centers for Disease Control and Prevention, Atlanta, GA, USA; Bryan Amick, PharmD, MS, MBA, Molina Healthcare, Columbia, SC, USA

Presentation Documents

Revising newborn screening guidelines in the United States and other countries is slow and arduous, with the implementation of newborn screening measures often taking 4-5 years. HEOR provides a systematic framework for evaluating proposed revisions and offers the hope of speeding adoption of worthwhile technologies for patients. The objective of this educational symposium is to explore the challenges involved in using HEOR to evaluate potential revisions and make recommendations regarding elements of best practice for the adoption of new diseases in newborn screening panels, with a focus on severe genetic diseases such as for example, metachromatic leukodystrophy (MLD).

The symposium will begin with an overview by a health economist of the potential methodological issues that could be encountered when framing and populating a cost-effectiveness analysis of newborn screening. These will be complemented with perspectives from a clinician, patient advocate and payor. The session will conclude with policy, procedural and methodological recommendations for best practice and future research.

Conference/Value in Health Info

2024-05, ISPOR 2024, Atlanta, GA, USA

Code

216

Topic

Health Policy & Regulatory

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