Case Study of Common Drug Review &

pan-Canadian Pharmaceutical Alliance and the first drug for PKU:

restrictive criteria = no access

John Adams

President & CEO Canadian PKU and Allied Disorders Inc. (CanPKU)

November 20, 2015

CORD Expert Patients conference

PKU 101 PKU stands for Phenylketonuria: •  a rare, genetic disorder •  brain-threatening •  intellectual disabilities, seizures & other neurologic problems •  incidence in Canada: 1:12,000 •  every newborn in Canada is tested for PKU since 1960s •  only treatment since 1950s:

- vegan diet without natural protein - synthetic protein (formulas & low-protein foods) - lifetime treatment, only since 2000

CanPKU Role: •  improve lives of people living with PKU and their families •  founded 2008: peer support, knowledge transfer, advocacy

Why do we need a drug for PKU?

PKU diet is very restrictive and hard to manage Francjan van Spronsen, MD, PhD, chair of scientific advisory committee of the European Society for PKU - Presenting Oct. 24, 2015

Government Reimbursement for first drug for PKU

Overview: 2010: Health Canada approves

Drug company applies to CDR Drug company asks for reconsideration on seeing confidential draft report CEDC says Do Not List – special note!

2011: Provinces submit request for advice to clarify special note CEDC does not change recommendation or special note

2012: ON, SK and BC negotiate with drug company; BC in lead 2013: ON & SK list drug with strict criteria: BC drops out 2014-15: SK & ON refuse eight applications 2015: ON for pCPA says drug must go back to CDR

Access to Kuvan™ today

Private •  private insurance •  clinical trials •  compassionate access by drug company

Governments •  Listed in ON & SK: zero patients •  Listed in QC: eight patients •  Health Canada: two patients (aboriginals) •  Alberta: two patients (disabled)

Ontario MD specialists criticized

Govt criteria

pCPA Current Position

1.  No change in access criteria and no province will negotiate individually

2.  Drug company must submit new clinical information to CDR – fourth trip to CEDC since 2010

3.  Only if a positive CDR/CDEC recommendation, then pCPA will negotiate