| 0:00:01 | i married uncle and i'm the vice president of communications foreign word |
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| 0:00:06 | a national organisation for real just workers |
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| 0:00:09 | my article is a thirty year retrospective on or |
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| 0:00:13 | he or from drug act and the emerging role of rare disease patient advocacy groups |
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| 0:00:20 | here twenty thirteen with an important year in the rare disease community because mar thirtieth |
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| 0:00:27 | anniversary of the establishment of ignored |
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| 0:00:30 | to represent whole |
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| 0:00:31 | where disease patients and patient advocacy groups |
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| 0:00:35 | and also the thirtieth anniversary of the orphaned right |
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| 0:00:40 | which was the law passed by the us congress in nineteen eighty three that created |
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| 0:00:46 | financial incentives to encourage |
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| 0:00:48 | pharmaceutical companies to develop treatments for people with rare diseases |
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| 0:00:54 | the article takes a look at the really very interesting story of how patient litres |
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| 0:01:00 | back in the late nineteen seventies and early eighties came together and realise that they |
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| 0:01:07 | had shared interests even though they represented people with very different diseases |
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| 0:01:12 | these patient advocates many of whom or patients or parents or spouses of patience |
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| 0:01:19 | learned how to work with |
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| 0:01:21 | government officials at n i h and f d a who were also concerned about |
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| 0:01:27 | the problem in learned eventually how to do outreach to the us congress and the |
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| 0:01:32 | cayman testifying before an important congressional subcommittee and ultimately they also learned how to work |
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| 0:01:40 | with the media |
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| 0:01:41 | in this case specifically jack like manner who was the star of a popular tv |
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| 0:01:47 | show at that time called quincy |
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| 0:01:50 | so it's just a really interesting story of how the orphan drug act came to |
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| 0:01:54 | be |
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| 0:01:55 | and the important role played by patient advocacy groups |
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| 0:02:00 | and looking at how the groups have grown and develop and become more sophisticated about |
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| 0:02:07 | research in the years since then and |
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| 0:02:11 | taken on it really a very important role in trying to make meaningful contributions to |
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| 0:02:19 | the development of treatments and in general to improving the lives of people with rare |
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| 0:02:23 | disease |
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