Box 2.

The example of Congenital Central Hypoventilation Syndrome. Should it receive priority for treatment over SMA type 1?

Congenital Central Hypoventilation Syndrome (CCHS) is a rare genetic condition in which there is abnormal regulation of breathing during sleep (Trang et al. 2005). Affected infants develop abnormal slowing and pauses in their breathing during sleep. If untreated, the severe form of this condition leads to death within the first month or two of life. However, with treatment, children may survive for years (Trang et al. 2005). During the day, while awake, these children have completely normal function. They do not require any assistance with their breathing. They are able to run, walk, communicate, attend school. Overnight, however, they must be attached via a tracheostomy to a ventilator to maintain regular breathing and to stay alive. Should CCHS be treated differently from SMA type 1?  It is plausible that the costs of treatment and the probability and duration of survival could be similar.a However, on many accounts of quality of life, the quality of life of a child with CCHS is much higher than a child with SMA

^aOlder children with CCHS may be able to be managed with non-invasive ventilation, and thus no longer require tracheostomy (Ramanantsoa and Gallego 2013). It is possible therefore that there are in reality differences in prognosis between CCHS and SMA type 1. For the sake of argument though, let us assume that these factors are identical—or at least similar enough that on grounds of cost/duration/probability the two conditions should be treated similarly