Screening for spinal muscular atrophy (SMA)-Scoping of Work

Scoping work for a decision analytic model and cost effectiveness evaluation of newborn screening for SMA. provision of a rapid review of the literature and for a plan to prepare for a future modelling study with clinical and cost effectiveness outcomes. It is not a screening pilot or an evaluation of one. The purpose of this work is to prepare for a modelling study by:

• Objective 1: Conducting a review of available decision analytic modelling studies and cost effectiveness evaluations which address new-born screening for SMA in the era of novel treatments • Objective 2: Developing an evidence map of published studies and evaluations of PCR based screening and treatment in presymptomatic SMA • Objective 3: Proposing a plan for a modelling study with clinical and cost effectiveness outcomes

Contract term: 12 months with the option to extend for a period or periods of up to further 18 months