The FDA denies approval to cures for diseases because of decades-old research standards that don’t make sense for today’s science.
America has always been a pioneering place. It has spurred innovation and offered hope and opportunity to millions of people from around the world. Many of our greatest innovators have been doctors and researchers who have risked everything to save lives. Yet now scientists and patients are too frequently being denied the opportunity to advance medical cures.
We continue to lose far too many of our loved ones to the “invisible graveyard,” as economist Alex Tabarrok has described: lives that could have been saved but for a bureaucratic barrier that rejects medical cures and innovation. In 2015, 700,000 Americans are expected to die of Alzheimer’s; nearly 600,000 lives will be taken by cancer. Moreover, millions of Americans suffer from countless chronic diseases that significantly impair their daily well-being. For instance, 25.8 million are afflicted by diabetes, and 30 million are afflicted by rare diseases, 95 percent of which have no known treatment.
These numbers are more than mere statistics: Each is a member of our family, a friend, a neighbor. Just one cure could rescue millions among us from suffering. While we will never know how many lives could have been saved by research, we can search for cures to save those who otherwise would fall prey to those same diseases.
We need to tear down the barriers blocking a new era of medical innovation, and the primary inhibitor is the government itself. Right now, the Food and Drug Administration stifles new treatments, operates with decades-old methodology, and discourages scientific development.
The FDA model is risk-averse, by its very nature obstructing promising innovations.
The FDA model is risk-averse, by its very nature obstructing promising innovations. It largely assumes that the biology of patients is the same, rather than recognizing that individuals’ genetic makeup varies widely. As a result, the only drugs the agency tends to approve are those that help a broad spectrum of patients and harm close to no one. That method may work to fight diseases that affect us all in a similar way, such as smallpox or cholera, but it does not work for diseases such as Alzheimer’s and cancer, which are highly tailored to each individual’s genetic makeup. In medicine, a one-size-fits-all approach ignores the diversity of the human person and limits the discovery of innovative cures to a small segment of those afflicted with disease.
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We now have the tools to reveal individuals’ biological differences at the most basic level. Biomarkers, or signs at the molecular level, enable the efficacy of a drug to be tested without waiting for the clinical effects of the treatment to surface. In other words, we can anticipate and prevent a disease without having to wait for symptoms to show up, often years later, when it’s too late. Yet the FDA continues to operate on a backwards model that largely rejects forward-looking discovery.
It’s past time to unleash a supply-side medical revolution, so that instead of simply caring for people with debilitating diseases, we cure them.
It’s past time to unleash a supply-side medical revolution, so that instead of simply caring for people with debilitating diseases, we cure them. For this reason, I am introducing legislation to reform the FDA and champion innovation. Right now, a number of drug companies and investors are fleeing the U.S. market. In reporting recent sharp drops in biotech stocks, the Wall Street Journal noted that economists cite as one of the key reasons the fear of a Hillary Clinton– or Bernie Sanders–imposed diktat of price controls. And as scientists and investors abandon the U.S., the FDA continues to prevent life-saving drugs from coming into the country. Take, for example, the multiple-sclerosis drug Lemtrada, which has been approved and used in Europe, Canada, Mexico, and Australia. Research has demonstrated that patients who are prescribed Lemtrada are only half as likely to relapse as those prescribed a leading drug in the U.S., but the FDA has refused to approve Lemtrada because the trials did not fit within the agency’s placebo testing requirements. Patients are getting sicker, and the FDA’s outmoded scheme is keeping them that way.
#share#The bill I am introducing takes the first step to reverse this trend. It provides for reciprocal drug approval, so that cures and medical devices that are already approved in other countries can more expeditiously come to the U.S. It requires the FDA to respond to the treatment sponsor within 30 days of its application. The legislation also ensures that the FDA is more accountable to Congress, and ultimately the American people, so that the FDA’s risk-averse approach does not stand in the way of medical advances. Beyond reforming reciprocity, we need to modernize the FDA’s approach, expand the Accelerating Medicines Partnership (AMP), and embrace a culture of innovation, as foundations like XPrize are doing. If we welcome new medical treatments, we will set in motion a new era of medicine that seeks not simply to care for those afflicted with disease, but to free them from it. Cures not only save lives, but also time and resources; with a cure, we eliminate the enormous costs of chronic care.
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If we want to keep good doctors and researchers here, and if we want the best scientists to bring their best solutions, we have to start by welcoming new discoveries, rather than cutting them off. An eminent example to follow is that set by the legendary cancer researcher Dr. Emil J. Freireich, now 88. As Malcolm Gladwell recounts in David and Goliath, Dr. Freireich survived a harrowing childhood in Chicago and went on to earn a medical degree. He spent his lifetime achieving what others supposed was impossible. As a young doctor, Dr. Freireich was stationed at the leukemia ward of the National Cancer Institute, where 90 percent of children bled to death within their first six weeks at the hospital. Determined to find a cure, he discovered the need for platelet transfusions, and perfected the method of giving them. The clinical director told Freireich he was crazy and threatened to fire him. But soon, the treatment began to work, the bleeding stopped, and his pioneering perseverance saved — and continues to save — thousands of children who are living their dreams. As Dr. Freireich now warns: “There’s always resistance to innovation.” But, like the great doctor, we must persist in our search for cures.
It’s time to open wide the frontier for innovation. We have an opportunity to unharness solutions that could be the difference between a lifetime of pain and suffering, even death, and a life full of promise and strength. America has always chosen the latter, and I am confident we will again.
— Ted Cruz is the junior U.S. senator from Texas.