List date(s) this work was published or aired.
1-Aug-11
Provide a brief synopsis of the story or stories, including any significant findings.
With an estimated 7,000 rare diseases affecting more than 30 million Americans and some 250 million people worldwide, rare diseases as a group are not, despite their name, rare. Yet treatments for them are. Today, the paucity of treatments for rare diseases may soon be remedied. This article examines the influx of interest and money from big pharmaceutical companies, the government and small biotechs to find treatments for rare diseases. With recent advances demonstrating that developing drugs for rare diseases can be a profitable and sustainable endeavor, business is booming.
Explain types of documents, data or Internet resources used. Were FOI or public records act requests required? How did this affect the work?
General scientific literature search. Also some plumbing of legislation on the Orphan Drug Act.
Explain types of human sources used.
This story involved speaking with life sciences investors, CEOs of biotech companies, government officials, and heads of rare disease research at several large pharmaceutical companies–covering a large swatch of groups pursuing rare disease drug development.
Results:
Published last August.
Follow-up (if any). Have you run a correction or clarification on the report or has anyone come forward to challenge its accuracy? If so, please explain.
No.
Advice to other journalists planning a similar story or project.
Big pharma is not big about talking on the phone with journalists. All I can say is, persevere! Also, while it’s nice to write about the science or medical implications of things like rare diseases or emerging pathogens, journalists should never forget that it’s often all about the money. Tracking where pharmaceutical companies were spending their money is what led to this story, as several companies have put significant funds into rare disease research in recent years.