The FDA recently issued new draft guidance that could accelerate the development of breakthrough medicines — thereby saving millions of patients’ lives.
The draft guidance encourages pharmaceutical companies to embrace “adaptive trial designs,” which can shave years off the traditional drug research and testing process. FDA Commissioner Scott Gottlieb hopes the guidance will make drug development “more efficient and less costly.”
It certainly will. The millions of Americans who are battling cancer, Alzheimer’s and other life-threatening chronic diseases should cheer the move.
Before approving a medicine for sale, the FDA requires drug manufacturers to prove it’s safe and effective by conducting clinical trials, which traditionally take place in three phases. In phase I, researchers give doses of an experimental drug to a few dozen patients to ensure the medicine is safe for human consumption. In phase II, researchers give the medicine to several hundred sick patients to test a drug’s efficacy and side effects.
During phase III, researchers offer the drug to hundreds or thousands of patients and monitor for less common side effects they may have missed in the previous phase.
New Drugs Take Time
Importantly, it can take more than 10 years on average to navigate through clinical trials and the FDA approval process. Unfortunately, cancer patients do not have the time to wait.
Adaptive clinical trials could significantly speed up the approval process. These trials allow researchers to modify their testing midcourse based on data they’ve already collected. By discontinuing dead-end research, scientists can save valuable time and money.
Consider the “Precision Promise” responsive adaptive trial sponsored by the Pancreatic Cancer Action Network. Starting this year, researchers will simultaneously test multiple drugs on pancreatic cancer patients, rather than testing just one new medication at a time, as in traditional clinical trials. Some patients will receive SM-88, a promising, investigational cancer therapeutic that is designed to kill cancer cells without sacrificing quality of life.
SM-88 specifically targets cancer cells’ metabolism, the processes that are necessary for the cancer cell to live, and does not target normal cells. Other therapies expected in this Precision Promise trial include investigational immune-based drugs, investigational therapies for patients with specific genetic mutations, and investigational agents designed to improve drug delivery to the tumor.
As the trial advances, patients will receive the investigational treatments showing the most promise.
A Revolution For Cancer?
This new trial design could revolutionize the treatment of pancreatic cancer, which claims 44,000 American lives each year and has a fatality rate of 95%.
I’ve experienced the horror of this disease firsthand. Two of my brothers, Eddie and Artie, and my business partner, Ervin, died from pancreatic cancer. In one of my last conversations with Eddie, he implored me to help researchers develop a cure. I promised him I would. I’m convinced that responsive adaptive trials are key to finding such a breakthrough.
Finding and securing FDA approval of investigational treatments faster could also save employers and taxpayers billions of dollars. That’s because many new drugs are significantly more effective than previous treatments — and have fewer side effects. As a result, patients miss fewer days of work. Currently, employers lose more than $200 billion a year due to workers’ disease-induced absenteeism.
Improvements like these also help reduce the total costs across the entire health care system — a much-needed reprieve.
Faster drug launches could also help reduce the toll of our most costly chronic diseases, such as Alzheimer’s. There is currently no treatment capable of slowing the disease’s progression. It afflicts 5.7 million Americans, and that total is projected to rise to 14 million by 2050. A treatment that merely delays the onset of Alzheimer’s by five years could save Medicare and Medicaid $218 billion annually by 2050.
Accelerating drug launches by even a few months could make all the difference for Americans battling fatal illnesses. It’s heartening to see the FDA recognize the need for speed, and that will help honor my promise to Eddie and to all patients suffering from these illnesses.
- Thompson is a former Secretary of Health and Human Services (HHS) and Governor of Wisconsin. He is also a board member of Tyme Technologies, which is a clinical-stage biotechnology company developing novel cancer therapeutics intended to be safe and effective across a broad range of tumor types.
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Author: TERRY JONES