Earlier this month, Colorado governor John Hickenlooper signed the nation’s first “right to try” law. The law allows a patient suffering from a disease, for which no medicine has been approved by the FDA, to try an experimental new medicine before the FDA approves it. The law allows, but does not force, drug-makers to provide their experimental drugs to patients. Other states, such as Louisiana and Missouri, are set to follow.
These patients are in dire straits. They suffer from diseases for which there is no other cure, and have short life expectancies. Most of us cannot imagine being in their position: They are willing to take far greater risks than most would accept, in their search for a cure.
Although the Food and Drug Administration (FDA) has an exemption for “compassionate use”, that exemption requires jumping through too many bureaucratic hoops to be useful. So, scholars at the Goldwater Institute developed the idea of state “right to try” laws that would enable residents to use experimental new drugs without FDA approval.
Joe DiMasi of the Tufts Center for the Study of Drug Development, and colleagues, have reviewed the time it takes for the FDA to review different types of new drugs.
FDA’s Neurology division, which approves drugs for Alzheimer’s disease, multiple sclerosis, Parkinson’s disease, and stroke, takes three times as long to approve drugs as the Oncology division. These differences cannot be explained by differences workload, the type and complexity of the drugs reviewed, or the safety of the drugs approved.
If the FDA could cut the performance gap between the divisions in half, the authors estimate that the cost of developing a new drug would decrease by $46 million — a savings that adds up to approximately $874 million per year.