Nigel Rawson is President of Eastlake Research Group
Donna Lawrence is Director, HTA and Health Economics, with PDCI Market Access Inc.
You’ve been diagnosed with a rare disorder. It’s probably taken several referrals to many different medical specialists, countless tests and some misdiagnoses for you to get here because it frequently takes years to reach a correct diagnosis. Now what? Is there a medicine available to reduce your suffering or extend your life?
The odds are against you because fewer than one tenth of the estimated 7,000 rare disorders have an effective treatment. However, more and more drugs for these disorders are becoming available. You’re lucky – your physician tells you that a safe and effective drug has been approved by Health Canada. But, accessing it will likely mean travelling the Beatles’ long and winding road, surmounting obstacles along the way.