Setting the Story Straight on Rare Diseases

With 7,000 rare diseases in the world, it may look like research isn't being done.

PhRMA StaffMarch 4, 2011

column in The Boston Globe today tells the exciting story about a local company that is reporting some very positive results from its development program for a medicine to treat cystic fibrosis (CF), a rare disease that affects about 30,000 Americans. Unlike current therapies for CF, which treat the symptoms of the ailment, this drug is being developed to potentially attack the disease itself.

It's gratifying to see a newspaper like the Globe pay attention to this important issue.

Unfortunately, the author, Steven Syre, was wrong when he suggested that biopharmaceutical companies don't engage in research for rare diseases.

Admittedly, he is correct that the cost and the odds of rare disease research can pose challenges.

But, as we've discussed in this forum, there are still 460 medicines being developed to treat rare diseases. Mr. Syre should also know that, according to our forthcoming state impact reports, 837 active clinical trials of these medicines are currently being conducted throughout the state of Massachusetts.

With 7,000 rare diseases around the world, it may look like the research isn't being done. But many of those diseases affect so few patients, our current scientific understanding limits the progress that has been made so far.

That doesn't mean that researchers across America don't have the drive to battle rare diseases. They do, and they are.

In fact, there are 30 medicines in development to treat CF (two of which are being developed by the local company in Mr. Syre's column); two of these have been granted Fast-Track status by the FDA.

As the week that began with Rare Disease Day draws to a close, it's great to see that dialogue about the challenges associated with rare diseases continues. We just hope that as it continues, the whole side of the story is told.

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