Expert Patient Advocates & 21st Century Therapies Forum Nov 19 - 20, 2015

1. 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

3. 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

4. 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

5. 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

7. 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)

8. Ontario MD
specialists
criticized
Govt criteria

9. 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