Conversations: Medicines in Development for Rare Disease

Issues of Importance to Rare Disease Community to be Focus of Tweetchat on October 17th

10.08.13 | By Stephanie Fischer

The world’s biopharmaceutical research companies continually strive to develop new medicines that prevent diseases, improve patient health, and help save lives.  There are currently more than 5,000 medicines in development globally.

While we are making progress against many diseases - the U.S. Food and Drug Administration (FDA) has approved more than 400 new medicines since 2000 – there is much work to be done, particularly for patients with one of the nearly 7000 rare diseases that have been identified.

While each rare disease affects a relatively small patient population (fewer than 200,000 patients in the U.S.), collectively they affect 1 in 10 Americans – including me.

Yesterday, PhRMA released a new report on the 452 medicines and vaccines in development for rare diseases.  While not all of these will be approved for patient use, I am optimistic that biopharmaceutical companies are actively pursuing therapies to treat rare disease, even if the patient populations are smaller and the science is (sometimes) more complex than for well-studied diseases that affect many more people.

I am excited to host a Tweetchat on rare diseases on Thursday, October 17th, at 4pm ET.  Ahead of that, we posed the following question to patient advocates and other thought leaders:

On 10/17, PhRMA will host a Twitter chat session around the subject of medicines in development for rare diseases. What are the shared priorities and interests of the diverse rare disease community that should be discussed in the Tweetchat?

I hope you will take some time to read the report, check out the related infographic, and contribute your thoughts in the comments section of this week’s Conversations question.  Alone we are rare, together we are strong!


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