February 29th, 2016

Rare Disease Day 2016

Derick Mitchell

I #rarediseaseday

“It’s rare when you have everything going perfectly

all at the same time”

Sigourney Weaver

What we know….

• Irish health system is unique + requires an Irish solution

• 2014: 9 ODs assessed, majority eventually approved

• High budget impact = decisions take longer

• No funding process in place

What we don’t know…..

• What the final decisions are based on?

• How many ODs are currently available in Ireland?

• What reimbursement of ODs actually costs?

• How ODs are being prescribed/monitored?

IPPOSI views

• Separate assessment system with different criteria for assessing OD in Ireland

• Criteria should consider • where patient perspective can be incorporated

• the cost to the state of not treating someone

• Transparency and Communication

Health Technology Assessment

Active Partnership with HIQA + NCPE

• HIQA have patient involvement on the advisory board of individual HTAs

• Annual HTA Training for patients through IPPOSI

• Acknowledgement that data provided by patient organisations is invaluable to NCPE assessments

• Irish patient groups exploring measuring patient outcomes with the NCPE

Irish National Platform

• John Dowling, Men Against Cancer

• Caitriona Dunne, Fighting Blindness

• Joan Jordan, MS Ireland

• Katie Murphy, CF Ireland

• Rachel Lynch, FibroIreland

• Damien Peelo, COPD Support Ireland

• Julie Power, Vasculitis Ireland Awareness

• Sharon Thompson, Rare Dis. + Palliative care

8 Irish Trainees

The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n° 115334, resources of which are composed of financial contribution from the European Union's Seventh Framework Programme (FP7/2007-2013) and EFPIA companies.

I #rarediseaseday

“May you live in interesting times”