Sen. Cruz: The RESULT Act is a Commonsense Reform Congress Can Pass to Champion Medical Innovation
Calls on Congress to pass RESULT Act following subcommittee hearing on rare diseases
October 4, 2018
WASHINGTON, D.C. – U.S. Sen. Ted Cruz (R-Texas) commended Sen. Rand Paul (R-Ky.) for holding a subcommittee hearing on Wednesday titled “Rare Diseases: Expediting Treatments for Patients,” and called on Congress to swiftly pass the RESULT Act.
“The current regulatory system is blocking Americans’ access to critical drugs, medical devices, and treatments that are already saving and improving lives in other trusted countries,” Sen. Cruz said. “As these witnesses testified yesterday, there are reforms Congress can pass to increase access to medical innovations, mitigate critical drug shortages, and lower the cost of medical devices and drugs. I urge my colleagues to pass the RESULT Act, reform the FDA, and unleash a supply-side medical revolution for patients in the United States.”
The bill, which Sen. Cruz introduced in December of 2015 and October of 2017, would amend the Food, Drug and Cosmetic Act to allow for reciprocal approval of drugs, devices, and other medical therapies, increasing access to potentially life-saving innovations. Specifically, the RESULT Act would require the Food and Drug Administration (FDA) to quickly review drug, device, and biologic applications from sponsors who have products approved and sold in trustworthy countries.
During the hearing, witnesses provided testimony on the importance of reducing regulatory hurdles that prevent Americans from benefiting from medical innovations, including those available in other countries.
Mark Dant, chair of EveryLife Foundation for Rare Diseases, noted that the regulatory burdens often leave medical breakthrough and innovation in the “valley of death,” and not with the patients.
“There is great science already available that goes to what is referred to often as the ‘valley of death,’” Mr. Dant said. “Because it takes so long to move great science through the pipeline through the FDA and then to treatment -- it costs too much. So companies cannot invest in science to move it along and so the therapies don’t come forward.”
Another witness, Mallory Factor, founder and CEO of IntraBio, discussed his experience with trying to obtain FDA approval for a drug and the need for reform at the FDA, saying they need to focus on “risk-benefit and timeliness.”
“Hundreds of millions of doses have been used of this drug since 1957,” Mr. Factor said. “Hundreds of millions. Tens of millions of people have been on this drug. Its safety profile has been shown to be safer than aspirin and yet we can’t even begin to put it into people here yet in a trial. I can go into great detail in the interactions with the FDA. The FDA’s first and foremost concern is risk. What they need to be concerned with is risk-benefit and timeliness. And they do not understand the risk-benefit analysis. I’m not sure what we can do. But the real problem is try to reach somebody at the FDA. You can’t. It is impossible to sit down other than in a formal setting with months -- it took us eight months to get a meeting and I won’t tell you how many children passed away from these diseases during those eight months -- just to begin to have a discussion with them. And you have to submit, we submitted a 150 page briefing book, which I’m not sure anybody really read thoroughly.”