Orphan Disease Legislation

There’s good news and bad news in a bill that just passed the Legislature:

A key change is notifying physicians sooner of newborn screening results. In the past, parents had to sign a form before physicians could learn if babies had tested positive for metabolic disorders. Many of the disorders keep the children’s bodies from breaking down proteins and require strict, meatless diets.

"It created a delay that meant the child might be exposed to (regular baby) formula or breast milk," Dr. Schaaf said.

The law requiring signatures resulted in a delay of one or two days, which for children with this rare condition could mean irreversible brain damage. Getting rid of that requirement is a positive step.

Unfortunately, the bill also expands the number of children with this disorder whose special diet is paid for by the state. It would be better if assistance to poor people with health problems went through Medicaid or a general health care voucher program. There are tens of thousands of human genes, and we don’t want a unique law for each harmful mutation. Besides, disorder-specific legislation is unfair to people with other orphan conditions who are less effective at lobbying.