Sunday, December 07, 2014

Japan Accelerates Regenerative Medicine with Regulatory Reforms
Why the United States Must Follow Their Lead

Sign the Petition Supporting Regenerative Medicine

Kobe Bryant, an elite professional athlete from the National Basketball Associationís Los Angeles Lakers, has gone to Germany multiple times to be treated with a portion of his own blood for his creaky, arthritic knees. The reason why he went to Germany is simple. He wanted to have the best chance to play at the highest level in the NBA and he had exhausted all his options in the United States. He went there to be treated with a component of his own blood that targets the inflammation associated with knee osteoarthritis. The treatment he received is not approved here in the United States.

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. Letís 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 patientís 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 Japanís 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 Mooreís 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.

Perhaps, then Kobe will be treated in California instead of Germany or Japan.

Allan Mishra, MD