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Apr
12

When is FAST not fast enough for rare diseases

Sitting here in SOHO I cannot help thinking how fast nearly a year of blogging has gone. Some articles have had a life of their own but its really hard to predict how something will end up and whether people will read it.

I think there is a great analogy here with government legislation. When people fight to get their words into a document they sure do not want to see them deleted. Although my interest in rare diseases has never really stretched to interacting with government, (probably because I do not have patience or political ability to navigate DC) as a bystander watching the whole  FAST language getting into PDUFA makes for an interesting spectator sport.

It makes me wonder why PhRMA could not use its weight to help out the rare disease organizations, patient advocates and all fighting to get their voices heard and more importantly treatments discovered and approved. I think PhRMA and by extension Pharmas should be helping get the right language approved so that drugs get approved faster for rare diseases. You do not have to be a genius to figure out how this would help them too.

And for that matter what about the AAPS, the ACS – these all have big lobbying groups and their members, many of whom work on rare diseases, would surely love to see their dues put to good use. This is a call to arms. All of you know more about biomarkers, surrogate endpoints etc than a few politicians.

FAST is not fast enough.

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4 pings

  1. greta beekhuis says:

    Thank you for this, Sean! Those of us on the front lines of rare disease research appreciate the call to arms. Looking forward to getting you involved with our Consortium and spreading the word about the app once it is *approved*!

  2. sean says:

    Hi Greta,
    ODDT app just released in app store tonight..separate blog on this and more to come once I get down to earth..let’s set up that lunch again!

  3. JIll Wood says:

    Didn’t see this until now. Thanks for sharing, much appreciated and next time your in town I’m taking you out on the town.

  1. PDUFA passes » Collaborative Chemistry says:

    […] So I blogged about this before-  based on press reports the PDUFA bill has passed ! This could have big implications for getting drugs for rare diseases. Personally this legislation is long overdue and it will be interesting to see how the FDA responds and also the impact it might have on drugs approved over the next few years. Something had to be done because of the dire approval rate of new drugs – however this legislation may not impact the pharma productivity side, unless they undergo some radical rethink or disruption of how drug discovery & development occurs. Share this:EmailPrintStumbleUponRedditFacebookDigg […]

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