August 1, 2019
By Naomi Lopez Bauman
The United States leads the world in drug development, but we’re in danger of falling behind, to the detriment of countless patients. However, new legislative efforts in the Senate could be an important step in helping more of our patients get the treatment they need.
Back in the 1980s and 1990s, the U.S. lagged behind Europe in drug development. Since then, and thanks in large part to the efforts of patient advocates, lawmakers, and agency personnel working toward reform from the inside, the Food and Drug Administration made great strides to modernize and speed up the nation’s drug approval process. Roughly two-thirds of innovative treatments are now launched first in the U.S.
In recent years, however, Japan, which is second in the world in treatment development, has been taking steps to both cut red tape and roll out the red carpet for the world’s leading pharmaceutical and biotech innovators. Japan’s Pharmaceuticals and Medical Devices Act and the Act on the Safety of Regenerative Medicine allow for the marketing approval of some regenerative treatments after basic safety has been established. The results have been strong international research and development investment, a more robust pipeline of drugs in development, and, more recently, an increasing number of new treatments launched first in Japan.
Japan now has the world’s fastest approval process for some regenerative medicine treatments, which replace or restore the patient’s tissue or organs to treat disease. One of the most promising types of regenerative medicine is stem cell treatments: A person’s own cells might be extracted, manipulated, and then administered back into the patient intravenously to reverse or cure disease.
Last year, a stem cell treatment that aims to cure patients diagnosed with mild to moderate Alzheimer’s became commercially available in Japan while it continues clinical evaluation. The treatment is undergoing clinical trial in the U.S. at three locations and will likely not be available for several more years. Of the 5.8 million Americans living with Alzheimer’s today, just 21 are enrolled in the Phase 1/2 clinical trial — leaving almost all of this country’s Alzheimer’s patients, their loved ones, and their doctors to rely on outdated treatments.
A treatment that is available and improving lives in an advanced country should be available to Americans, as well. Fortunately, some of our lawmakers recognize this.
Earlier this month, legislation was introduced in the Senate that would allow for reciprocal approval of drug treatments approved in other advanced nations. Sen. Ted Cruz, R-Texas, reintroduced his Reciprocity Ensures Streamlined Use of Lifesaving Treatments Act (S. 2161), or the RESULT Act, which would require the FDA to quickly review drug, device, and biologic applications from sponsors who have products commercially available in select developed countries with a stringent treatment approval process. Another similar proposal introduced this past March by Sen. Mike Braun, R-Ind., The Accelerated Drug Approval for Prescription Therapies (ADAPT) Act (S. 658) would create an expedited drug approval process at FDA for drugs that are commercially available in select developed countries.
Both approaches retain the important role the FDA has in establishing treatment safety and efficacy. But they do so in manner that recognizes that innovations don’t happen exclusively in the U.S.
Since 2014, dozens of drugs, biologics, and biosimilars obtained European Medicines Agency approval and are awaiting FDA approval. Patients shouldn’t be forced to wait unnecessarily for years for the FDA to evaluate a treatment that has already undergone clinical evaluation in another country that shares a rigorous approval process.
Despite many billions invested to find a cure for Alzheimer’s and roughly 400 drug treatments that have failed clinical trials, a cure remains elusive. When we know there is a promising treatment for this disease that is already available to patients elsewhere, shouldn’t we remove barriers and obstacles that force patients to wait for years for additional, sometimes duplicative, evaluation?
These important legislative proposals recognize that when promising treatments aren’t available to U.S. patients who are facing devastating illnesses, there should be a pathway for harnessing the important scientific work that has already been undertaken elsewhere on behalf of patients.
Naomi Lopez Bauman is the Director of Healthcare Policy at the Goldwater Institute.