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Is NICE Going To Play Nice When Reviewing The High Cost of Orphan Drugs For Rare Diseases ?

December 20, 2012

       Under the UK’s Health and Social Care Act of 2012, the National Institutes for Health and Clinical Excellence (NICE) is going to start assessing high-cost drugs for rare conditions with the start of the UK fiscal year in April 2013.


NICE July 20, 2012 News

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  1. Hopefully this will make it less likely we see a repeat of the Kalydeco process with it taking nearly 5 months from regulatory approval to the NHS (England) agreement to fund. Scotland, Wales and N.Ireland still waiting. The ethics of the UK clinical trial are called into question too as kids who showed big improvements in the trial had the drug withdrawn post-trial and have seen their health decline again. So horrendous for parents to watch. Let’s hope the new arrangements streamline the process and enable the NHS to take a more consistent, integrated view. Pharma needs better visibility on this too so that investment is justified and more of the treatable is actually treated. It can’t be sustainable for the NHS to fully fund so many “mass market” elective treatments, things which only have a marginal effect or things whose effect is unproven (e.g. Tamiflu) while at the same time denying poorly children new treatments that definitely work and have a transformative effect.

    • Thank you for visiting the Orphan Druganaut Blog. I agree with you that we hope that the new process with NICE works & is effective starting in April 2013. Things hopefully will go smoother. Thanks for your comments on Kalydeco & the situation of it taking almost 5 months from regulatory approval to the NHS (England) agreement to fund. Unfortunately, as you said, Scotland, Wales & N. Ireland are still waiting. Have a great Holiday & Happy New Year.

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