Few medical scenarios are more heartbreaking than a child with a dread rare disease who might be able to be treated if only drugs were available.

“Orphan drugs,” as we recently described, treat extremely rare disorders, generally afflicting 6,000 or fewer patients. Because the consumer market for them is so small, there’s little incentive for manufacturers to conduct the R&D necessary to develop them, or to spend the money marketing them.

Legislative efforts are underway to deliver orphan drugs faster to the people who need them. An article published recently in the journal Pediatrics, “What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis,” reviewed progress for victimized youngsters.

It looked at orphan drug designations and approvals from Jan. 1, 2000, to Dec. 31, 2009, and determined that the Act “led to increased product availability for [rare diseases] overall, with an increasing number of marketing approvals for children this past decade.”

During that time period:

• 1,138 drugs were designated as “orphan,” of which 148 received marketing approval;
• 38 (more than 1 in 4) addressed pediatric diseases;
• the proportion of approvals for pediatric products increased from fewer than 1 in 5 in the first half of the decade to slightly fewer than 1 in 3 in the second half.

This is good news, as is the fact that approval for “orphan” designation and marketing for pediatric diseases with fewer than 20 000 patients grew more than for any other group. Anyone interested in knowing more about the FDA’s Office of Orphan Products Development may link here.

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