Continuing Medical Innovation for Rare Diseases

Continuing Medical Innovation for Rare Diseases

02.28.13 | By Stephanie Fischer

Rare Disease Day 2013As one of the 25 million rare disease patients in the United States, I was pleased to see President Obama stress the need for continued medical innovation when he acknowledged international Rare Disease Day:

"Occasions like Rare Disease Day remind us that we must continue to harness our creativity and ingenuity to remain at the forefront of medical research and innovation. With each new discovery we make today, we not only move closer to building a healthier tomorrow for every American, but we also pave the way for breakthroughs that will save and enrich lives around the world."

With treatments for only about 5% of the nearly 7,000 rare diseases, there is universal agreement on the need to do better. And we are. As I noted in an earlier post, Care about Rare, a recent report on the biopharmaceutical pipeline found there are nearly 1,795 potential therapies in the pipeline for orphan diseases. What do we need to do to encourage the innovation necessary to reach more breakthroughs?

In a speech today in London, PhRMA President & CEO John Castellani talked about the dedication of researchers and the huge investment in research & development by America's biopharmaceutical companies. Since 2000, PhRMA member companies have invested over $500 billion in the search for new treatments and cures, including an estimated $49.5 billion in 2011 alone.

John also talked about the need for collaboration and for industry, government, academia, patients and other stakeholders to forge the long-term regulatory, scientific and economic policies needed to generate investment and build a sustainable innovation ecosystem.

Only with that support can we recognize the promise of the biopharmaceutical pipeline and move new therapies from the lab into the hands of patients.

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