A Japanese cardiac surgeon believes he has come up with an effective way to use induced pluripotent stem cells to treat heart disease. His results have proved so successful in lab animals that the procedure has now been fast-tracked by the Japanese government. Three human patients will be the first to undergo the revolutionary procedure in the near future.
Japan’s fast-tracking system for medical research is one of several things that have combined to make the country an emerging leader in stem cell development. Their fast-track system gets new procedures to market faster, thus encouraging the private sector to take more risks in R&D. So here’s the million-dollar question: do we need something similar here?
Research into various stem cell therapies in the United States is a very slow and tightly regulated process. It could take more than a decade to get a new procedure to market due to all the studies and bureaucratic red tape associated with FDA approval. Perhaps we could improve stem cell treatments by streamlining our own approval process.
How the Japanese System Works
Japan does have a standard medical approval system that can be as cumbersome as our own. Their fast-track system was developed to move things in the field of regenerative medicine along a bit more quickly. Under this system, introduced in 2014, research organizations and private sector companies can obtain preliminary approval for a regenerative medicine procedure as long as such procedures are proven safe.
Mind you, they do not have to prove effective. This is a big distinction, according to the Advanced Regenerative Medicine Institute. Under standard approval procedures both in Japan and the U.S., researchers have to prove efficacy before approval is given. Patients cannot be treated outside of a clinical study setting without such approval.
The Japanese fast-track system bypasses the need for long, cumbersome clinical studies as long as a procedure is safe. What this essentially means is that a research organization or private provider can begin offering safe treatments right away, using those treatments as informal clinical studies. But there is a catch: researchers must compile data over a set period of time to prove the efficacy of what they’re doing. That data will be reviewed at some point by government officials who will then determine whether to make approval permanent.
Arguments against the System
Critics of the fast-track system cite a number of arguments against it. First is the idea that patients are being used as guinea pigs in the absence of formal clinical studies. But shouldn’t patients have the right to choose whether they want to receive fast-tracked treatments or not? They can hardly be guinea pigs if they are voluntarily seeking to be treated.
The second argument is that fast-tracked treatments offer little more than hope in exchange for thousands of dollars in treatment fees. Critics say that charging people so much for a procedure that is not guaranteed to work is not right. But don’t we already do that?
How many patients in the U.S. are offered FDA-approved treatments even though efficacy cannot be guaranteed? The only difference is that our patients have no idea of the true cost because their insurance companies are paying for it.
The fast-track system could be beneficial here for the same reasons it seems to be working in Japan. Fast-tracking eliminates much of the burden that goes along with obtaining FDA approval. Furthermore, fast-tracking allows for real world treatments that can provide the clinical data that will eventually determine efficacy. It seems like a great way to do things.