The Orphan Drug Act and NORD at their 40th anniversary: Dramatic achievements and ongoing innovation
In 1982, Rep. Waxman convened a hearing and invited several pharmaceutical companies to attend. They declined the invitation. Ms. Meyers testified at the hearing, which was covered by the Los Angeles Times, giving the issue notoriety.
The issue was also addressed in the popular television series Quincy, M.E. Episodes depicted challenges facing a patient with Tourette syndrome and a patient with myoclonus. Series star Jack Klugman testified before Congress about the need for legislation regarding the development of drugs targeting the treatment of rare diseases.4
Rep. Waxman became the bill’s primary House sponsor. The ODA was approved by the House of Representatives on Dec. 14, 1982.
The combined efforts of the advocacy coalition ultimately culminated in the original ODA being signed into law by President Ronald Reagan on Jan. 4, 1983. The ODA incentivized pharmaceutical companies to allot more resources toward research on and development and distribution of therapeutics for people with a rare disease who had, until that point, been “orphaned,” so to speak, by the medical and scientific community.5
What is it to be a ‘rare disorder’ or an ‘orphan drug’?
The ODA defines a rare disorder as either a condition that affects fewer than 200,000 people or a condition that affects more than 200,000 people but for which there is no reasonable expectation that a pharmaceutical company will recoup the cost of developing a drug by selling it.5
In an interview Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders, commended Ms. Meyers on her tireless work: “She went from being a mom to a powerful advocate who got thousands of people in D.C. moving Congress to a point where they felt passing the law was necessary. In fact, she was there when the bill was signed into law.”
The FDA can grant orphan drug approval for several categories of products. These include new molecular entities, already-approved drugs for which an additional indication has been designated, and new formulations of existing or established drugs.5
Orphan-drug designation includes tax credits for the developer-manufacturer, a waiver of the usual approval fees that the company must pay to the FDA, and 7 years’ market exclusivity, during which time the FDA cannot award approval of similar treatments for the same indication.
“This [designation] ensures that the company that developed the drug can retain the opportunity for profit,” explained Mr. Saltonstall. “Companies thought: ‘We can afford to do this now. And if we market it the right way and have exclusivity, we can recover some of our profits.’”
Before the ODA, 30 drugs had been approved by the FDA for orphan conditions. By the end of 2022, more than 880 drugs were FDA approved to treat hundreds of rare disorders.
“This just shows the motivation and how the process works,” Mr. Saltonstall said. In fact, almost 50% of novel drugs approved in 2022 by the FDA were an orphan product or had an orphan indication.
In addition to the impact on drug development in the United States, enactment of the ODA had a broader ripple effect, inspiring similar changes internationally in Europe, Australia, Singapore, Japan, and other regions.5
