On August 6, 2014 the Government of Canada launched a new pilot project that will seek input from Canadians with rare diseases to inform future orphan drug reviews.
The work derived from this project will be incorporated into how patient input will be gathered once Health Canada implements its Orphan Drug Framework.
Drug manufacturers Hoffmann-La Roche Limited, and Hyperion Therapeutics Inc. are each offering a drug to be reviewed in the pilot project:
- Obinutuzumab – for the treatment of chronic leukemia (CLL)
- Ravicti® (glycerol phenylbutyrate) Oral Liquid – for the treatment of urea cycle disorders.
Health Canada will be working with patient advocacy groups, including the Canadian Organization for Rare Disorders to help engage with Canadians impacted by these rare diseases.
Participants will give feedback on the following areas:
- how the rare disease affects their ability to manage their day-to-day lives;
- what treatments are currently available (if any);
- what therapeutic benefits are most important to them; and
- their risk tolerance for any new treatments.
Participants include patients with CLL or urea cycle disorders, current or former caregivers and health care professionals.
For more information and to inquire about participating, please email Health Canada email@example.com.