PKU AI logo What is PKU 1960s Political

PKU (AI) logo?

What is PKU?

1960’s- Political and Clinical vision!

2009! • Less brain damage • Better functioning • Ability to eat more ‘normal’ foods

NCPE 2009 2017

So Kuvan didn’t demonstrate ‘cost-effectiveness’ • This does mean it is not cost-effective • The makers of Kuvan were unable to satisfy the NCPE’s ‘requirements’

Measuring ‘Benefits’ in Rare Diseases • • They are rare! Generally not well understood Expertise is limited What to measure? What to compare to? Over how long? Incentives?

Where is Kuvan Available today?

Next stages in process • Makers of Kuvan try to negotiate price with NCPE/ HSE • They await appointment from HSE’s ‘Corporate Pharmaceutical Unit’ • HSE drugs group to meet in Nov • • No transparency, no accountability We don’t know what decision criteria they use We don’t know who sits on the drugs group We don’t know what inputs they receive and from whom

Embed PKU video

PKU An Irish Mother’s Perspective Bernadette Sheehan Gilroy

Living with PKU

My inspiration Mrs Mary Jones

What dietary therapy doesn’t achieve alone! Unrealistic diet Demands on family life Complications with management Side effects of dietary therapy

What does it look like?

What do we want? • Best practice - what others have! KUVAN • Kuvan responders in Irish Study!

PKU Patient and Advocate Kevin Alexander

PKU Patient Charlie Box

What are the experts saying in Europe and worldwide? “Kuvan at present being the only pharmaceutical treatment for pku not only helps to further improve the intellectual and social outcomes but also improves patients quality of life” Professor Francjan J. van Spronsen Head of Division Metabolic Disease in Children Chair Of Scientific Advisory Committee European Society of PKU

What are the experts saying in Europe and worldwide? “We have found that responders to sapropterin are more likely to maintain blood phe within treatment range resulting in improved neurocognitive function, patients that are treated with diet alone are rarely able to maintain metabolic control” Professor Laurie Berstein MS RD FADA FAND Professor of Pedeatrics, Director of IMD Nutrition Inherited Metabolic Diseases Clinic Children’s Hospital, University of Colorado

“Give us the future, we have had enough of your past” Michael Collins