KIRSTEN HARKINS 2015 CADTH SYMPOSIUM APRIL 13, 2015 SASKATOON, SK Daring to be Rare: Striving to Meet the Needs of Patients with Rare Diseases

March 2001: Nicklas diagnosed with MPS I

August 2003: Nicklas’s first infusion

A journey into patient advocacy “We are both burdened and blessed by the great responsibility of free will – the power of choice. Our future is determined, in large part, by the choices we make now. We cannot always control our circumstances, but we can and do choose our own response to whatever arises. Reclaiming the power of choice, we find the courage to live fully in the world.” - Dan Millman, The Laws of Spirit: Tales of Transformation

The early days of advocacy 2004: CDR recommend against funding Aldurazyme There was no avenue for patient input in the process 2005: Rare disease patients demanded to be heard

Realizing collaboration is the key Working together essential for success Canadian MPS Society; International MPS Network CORD; Rare Disease Foundation (RDF) Working with provincial health ministries when federal government failed to create rare disease programs (eg: Elaprase for MPS II was pilot drug for Ontario’s Drugs for Rare Diseases program) Building bridges and learning how to work with all stakeholders in a passionate, but educated, sophisticated and reasonable fashion

Patient involvement: Patient Centred Care 2012: Health Minister’s announcement of a new rare disease framework, which would involve patients as partners in rare disease care CIHR funded grants to figure out how to integrate rare disease treatments into the Canadian healthcare system CDR initiated patient input process – huge step forward! (But questions remain as to the weighting and the capacity of small organizations to create strong submissions on lean budgets) PRISM: patient advocates on advisory panel

Meaningful involvement Integrating patient input to the process is important, but not easy! Opportunities for inclusion:  Research grant funding  Registries & data collection  Clinical trial development (ie: meaningful endpoints – MPS IVA pain example; trial design; protocol advice; increased inclusion)  Risk assessment; assessing benefits vs harms  Guideline development  Treatment value: take patients’ and their families’ experiences seriously! Rare diseases affect families & societies, as do treatments  TRANSPARENCY is crucial as we deal with uncertainty; life-cycle approach & post-market analysis necessary

Remember why we’re here! “Aerodynamically, the dragonfly shouldn’t be able to fly, but the dragonfly doesn’t care, so it just goes on flying anyway.” These kids are fearless. What are we afraid of?