August 1, 2019
By Naomi Lopez
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patients, their loved ones, and their doctors to rely on outdated treatments.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 is the Director of Healthcare Policy at the Goldwater Institute.
This
article originally appeared in The
Washington Examiner on July 31, 2019.
