How do you price a drug for a rare disease/ orphan status drug? The question that comes up time and time again. Truthfully, I don’t have the answer. Biogen feels that Spinraza is fairly priced at $750,000 for it’s first year of treatment. Maybe it is –maybe it’s not.
It begs the questions of how the industry, payors and the population as a whole, place value on health and quality of life. Moreover, if pharma companies are not given the band width to price high a drug for rare disease will they continue to pursue this avenue? Some rare diseases may have less than 5000 or even 100 patients in the world—does the public feel that a pharma company should dedicate 5-8 years of R&D and millions of dollars and not recoup this?
Do we allow disease modifiers to be priced higher than those that just manage symptoms or provide progression free survival? How do we price quality of life? Do we assign the same value to a person in their mid 30’s as we do to someone is their 70’s?
I take the case of a lady aged 62 that I know. She has been on a HER2+ agent for over a decade. A Stage IV HER2+ breast cancer patient with brain and bone mets at diagnosis, she was given less than a year to live. Her drug therapy is keeping her alive. Her diagnosis 10 years back shortened her much loved teaching profession. She now volunteers for free at a local public school twice a week assisting elementary students with special learning needs. Is their value to that? The money in Canada comes from the same pocket. Is she saving our government money by them not having hire a teacher to manage children with special needs at this particular school?
How do payors decide what is acceptable? For instance, a cure for Hepatitis C drug is priced less than what a liver transplant would cost. Is that the model that we should be using? Do payors factor in a lifetime of medication, appointments etc... Is all this moot during an election year?
I wish I had the answers…