A Seattle Times investigation

Pharma's Windfall

About the project

This year, the 30th anniversary of the Orphan Drug Act, The Seattle Times set off to examine the act’s impact on patients.

Over the years, many health and business reports cited the success of the Orphan Drug Act, saying it had spurred hundreds of new drugs for rare disease. This year, the 30th anniversary of the act, The Seattle Times set off to examine the act’s impact on patients.

Our first hurdle was to determine how many orphan drugs had been approved by the government.

We turned first to a database of drug applications and approved drugs maintained by the U.S. Food and Drug Administration. The database incorporates a variety of information about the drugs, such as compound name, dates of approval and related manufacturer details.

The government’s terminology can be confusing.

Pharmaceutical companies that pursue a rare disease must file a “new drug application” with the FDA. Federal officials then decide whether to “designate” a drug with orphan status, which means it is approved only for use in clinical studies. This also kicks in an array of tax breaks, grants and regulatory shortcuts. If the trials are successful, only then can the drug receive “market approval” and be sold to the public.

The FDA has issued 449 market approvals since the Orphan Drug Act was adopted in 1983. But some drugs are approved for more than one disease variation. For instance, we found that the cancer drug Gleevec is approved for nine disease categories. Consequently, as of Oct. 18, the FDA has authorized the sale of 363 different orphan drugs.

Medical literature and research also conflicted on the number of rare diseases. We analyzed a database from the National Institutes of Health to determine that there were 6,432 rare diseases as of last month.

To determine the number and circumstances of illegal marketing allegations against pharmaceutical companies, we created a custom database to track FDA administrative violations and warning letters.

Our reporting extended beyond databases. To determine drug revenue and pricing, we analyzed financial reports from the U.S. Securities and Exchange Commission or relied on private market research, primarily from IMS Health, a research firm that contracts with the federal government and the pharmaceutical industry, among others.

We reviewed dozens of research reports and government studies about orphan drugs, including thousands of pages of federal and state court records.

Michael J. Berens and Ken Armstrong

Credits

Reporters: Michael J. Berens and Ken Armstrong; photographers: John Lok, Steve Ringman; video producers: Genevieve Alvarez, Danny Gawlowski; editor: James Neff; desk editor: Laura Gordon; photo editor: Fred Nelson; print designer: Bob Warcup; producer, web designer: Katrina Barlow; graphics: Kelly Shea; database: Justin Mayo; developer: Benjamin Turner; researcher: Gene Balk

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