“My motor neurons are dying and without treatment I will suffocate under the weight of my own chest,” Bellina said.
But is the bill he advocated before the Senate Homeland Security and Governmental Affairs Committee really needed? Is it a solution in search of a problem?
Data from the Food and Drug Administration (FDA) indicate the answers to those questions are no and yes.
FDA “has authorized more than 99 percent of requests” for what it calls “expanded access” to drugs and other products between 2010-2015, Peter Lurie, an associate commissioner, told the hearing. “Emergency requests are usually granted immediately over the phone and non-emergencies are processed in a median of four days.”
So, if the FDA approves virtually all investigational drug requests for patients so quickly, why is there a need for additional legislation?
The agency’s expanded access program serves relatively few people and more would get the drugs under his legislation, contends committee Chairman Ron Johnson (R-Wis.), the bill’s sponsor.
“About 600,000 people die of cancer each year, and only a small percentage of those patients are allowed into a clinical trial or will successfully apply for expanded access,” he said by email after the hearing. “So when the FDA says 99 percent of requests are approved, it may sound good, but it is a false metric and only represents 1,256 (FDA says 1,268) patients last year. Without the protections of federal Right to Try legislation, tens of thousands of patients never even apply.”
One of those protections would block federal agencies, including the FDA, from using an adverse effect, such as a medical complication from an investigational drug, in the agency’s evaluation of the product for full approval.
Lurie objected to that.
Prohibiting the FDA, which is charged with protecting the public from potentially harmful food and drugs, “from reviewing adverse events that occur in expanded access use would be detrimental and raise significant ethical issues,” Lurie said. “Given that the agency is charged with assessing the safety and effectiveness of medical products, the agency cannot ignore valid scientific information.”
He also rebuffed accusations that the FDA blocks access to the drugs. Before FDA decides if an individual will get one of medicines, the pharmaceuticals company “has the right to approve or disapprove the physician’s request,” Lurie said. “If the company agrees to the physician’s request, the physician can then apply to FDA for permission to proceed. Should they do so, they are highly likely to be allowed to proceed.”
He pointed out that “the company developing the investigational product must be willing to provide it — FDA cannot force a company to manufacture a product or to make a product available.”
Rather than casting FDA as the bottleneck in the investigational drug pipeline, Lurie pointed to the drug industry. “It appears that pharmaceutical companies turn down considerably more applications from physicians than does the agency,” he said, citing one company that wasn’t named that “turned down 98 of 160 applications for a single drug in a six-month period.”
Would Right to Try change that?
Andrew McFadyen doesn’t think so.
When his son, Isaac, was 18 months old, McFadyen learned the child had Mucopolysaccharidosis type four, a progressive disease affecting the skeleton. He was told “every bone, muscle, organ, and tissue in his body would be ravaged by this disease until he eventually succumbed to the condition, probably in his early to late teens.” Isaac is now 12.
But McFadyen, whose Isaac Foundation is based in Ontario and works in Canada and the United States, is no fan of Right to Try.
“The cruel reality with Right to Try legislation is that it will not grant patients the immediate access to treatments they desperately need,” he said. “Although various forms of Right to Try laws have been passed in 31 states, there continues to be no concrete evidence of a patient ever receiving a life-saving or life-sustaining medication under Right to Try legislation when they otherwise wouldn’t have received it under the existing FDA program.”