Opinion: The ‘cruel joke’ of compassionate use and right to try: Pharma companies don’t have to comply
From my days in medical school, I vaguely remember learning about lysosomal storage disorders. They occupied at most part of a lecture or two in my second-year pathophysiology course. I memorized a few details about these rare diseases in preparation for my board exam, and then never gave them another thought. These diseases were treated by pediatric specialists and wouldn’t be part of my life as a cardiologist.
That changed a few weeks ago when my 28-month-old daughter, Radha, was diagnosed with a lysosomal storage disorder. Now I know far more about these diseases than I did in medical school. I’ve also learned a frustrating fact that no medical school teaches its students: While the FDA has a compassionate use program to allow people access to experimental drugs, it can’t compel a company to provide those drugs. The newly signed “right-to-try” law doesn’t either.
Radha’s birth went perfectly. She was a healthy baby and met all of her developmental milestones — until it came to walking. My wife, Sonal, a pediatric gastroenterologist, recognized this and we had Radha
