Japan Accelerates Regenerative Medicine with Regulatory Reforms
Why the United States Must Follow Their Lead
Will he go to Japan next time to get the most advanced therapy? That may happen because in November 2014 Japan enacted legislation creating a new pathway that accelerates the approval process for regenerative
medicine products. We here in the United States need to
figure out how to best accelerate our own regenerative medicine efforts or continue
to watch our elite athletes and other patients seek treatment beyond our
shores. Let’s explore why Japan acted
boldly in favor of regenerative medicine.
A Nobel Prize was awarded to Japanese researcher and former
orthopedic surgeon, Dr. Shinya Yamanaka in 2012 for his pioneering work on how
to reprogram mature skin cells into pluripotent stem cells. His potentially life saving work and the
national pride associated with it helped Japan become a worldwide leader in
stem cell research. Importantly, Japan
has also prospectively pursued and passed legislation that will dramatically stimulate
regenerative medicine. The country took
a leap into a bright future last week with the implementation of regulatory
reforms directed at accelerating the development of cell-based treatments.
The new law established a separate pathway for regenerative
medicine products apart from traditional drugs and medical devices. The
regulations also created a system that fosters faster commercialization of
novel biologic products and can lead to time limited approvals for
up to seven years. Post market data
analysis will continue to confirm the safety and efficacy of the regenerative
medicine products with the potential to revoke approvals.
The action by the Japanese government creates the proper equilibrium
between the need for accelerated approvals for cell based therapies that have
established efficacy while ensuring product safety. The typical phase one, phase two and phase
three trials for new drugs are not a proper pathway for cell-based therapies
that have highly complex mechanisms of action that may or may not be identified
even with elite preclinical models. Importantly,
many cell-based products come directly from a patient’s own body and therefore
have a dramatically higher safety profile.
Americans are screaming for new treatments for a variety of
difficult problems such as arthritis, heart disease and diabetes. Regenerative medicine and especially cell-based
treatments have a chance to help treat and potentially cure these
problems. We need to follow the Japanese
lead and create similar pathways in the United States. Last year during a visit to Japan and its PMDA
(the equivalent of the FDA), I was staggered by how engaged they were about
regenerative medicine and receptive they were to understanding how to bring
safe and effective biologic products to their populace.
The United States can learn a great deal from Japan’s enlightened
approach to regenerative medicine.
Safety of course should be paramount.
The idea of a conditional, time-limited approval with reimbursement
strikes a perfect chord as we seek better treatments for our patients at more
affordable prices. Competition will
arise to obtain approvals based on phase two trials that confirm safety and
show a solid degree of efficacy.
Importantly, more competition will lead to better options and faster
innovation similar to the computer industry over the last several decades.
We need to also emulate our Silicon Valley technology
colleagues and call for a Medical Moore’s Law.
Identify a specific clinical problem, create a novel solution and within five
years double the efficacy and decrease the price by fifty percent. Data analysis via powerful graphical algorithms should assist with new discoveries and also help with better management of many diseases.
We must seek dramatically higher horizons instead of being satisfied with iterative improvements. The Japanese government should be congratulated for passing regulatory reform and implementing transformative solutions. Sooner, not later, we need to find ways to execute our own regulatory reforms here in the United States to help our patients and to remain competitive on the world stage in regenerative medicine.
We must seek dramatically higher horizons instead of being satisfied with iterative improvements. The Japanese government should be congratulated for passing regulatory reform and implementing transformative solutions. Sooner, not later, we need to find ways to execute our own regulatory reforms here in the United States to help our patients and to remain competitive on the world stage in regenerative medicine.
Perhaps, then Kobe will be treated in California instead of
Germany or Japan.
TotalTendon
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References
Accessed 11/30/14
Accessed 11/30/14
