Rep. Waxman commemorates law that fights rare diseases

Rep. Henry Waxman (D-Calif.) commemorated the 20th anniversary of the passage of the Orphan Drug Act Friday, calling it a “legislation that has proven successful beyond all expectations.”
 
The Orphan Drug Act was signed into law by President Reagan on Jan. 4, 1983, and created financial incentives for products that treated those afflicted with rare diseases.

In the decade preceding the law’s passage, there were only ten drugs developed to treat rare diseases, a situation that Waxman characterized as “a classic case of market failure.”

“By definition, the market for a drug for rare disease was too small to attract drug companies to do the research and go through [the U.S. Food and Drug Administration’s] approval process,” Waxman said in a statement. To kick start that research, “we created incentives: intellectual-property protections, tax benefits and regulatory advantages," he said.
 
Thirty years later, more than 400 treatments have been approved by the FDA, according to the National Organization for Rare Disorders (NORD), a nonprofit founded to advocate for those suffering from rare diseases.
 
Waxman also praised actor Jack Klugman, who utilized his role on the popular NBC drama "Quincy, M.E." to bring attention to the issue. He testified before the subcommittee on Health and the Environment, which Waxman chaired at the time, and Waxman credits him as one of the law’s “first champions.” Klugman died on Dec. 24.