The value of orphan drugs to treat ultra rare diseases associated with premature death is overrated. They are being developed in extremely small populations and measured with company elected metrics to objectify success since the disease is so rare. Any literature published is subject to considerable bias. The drug is so novel there hasn't been time to show evidence of reduced morbidity and mortality; The FDA acknowledges that certain aspects of drug development that are feasible for common diseases may not be feasible for rare diseases.
Pharma in the orphan drug business can get grants from the FDA then they try to justify the exorbitant prices of their drugs based on the rarity of the condition. Beyond the unreasonable cost of the drug, there is an anticipated increase in medical utilization by potentially prolonging life and ongoing drug monitoring.
Should the FDA change the orphan drug natural history studies from optional to mandatory and hold the investigators responsible for identifying the subpopulations in these rare disease that can benefit as opposed to approving it for all patients with these rare diseases?
Pharma in the orphan drug business can get grants from the FDA then they try to justify the exorbitant prices of their drugs based on the rarity of the condition. Beyond the unreasonable cost of the drug, there is an anticipated increase in medical utilization by potentially prolonging life and ongoing drug monitoring.
Should the FDA change the orphan drug natural history studies from optional to mandatory and hold the investigators responsible for identifying the subpopulations in these rare disease that can benefit as opposed to approving it for all patients with these rare diseases?