The twenty-first century might well see medical innovations that could rid humanity of crippling and deadly illnesses, extending the healthy lifetimes of all by decades. But while the United States has cutting-edge research facilities and experts, its government-approval process for those innovations is as sclerotic and deadly as any bodily ailment. Japan, on the other hand, has adopted the Free To Choose Medicine model for its tissue-based therapies. If Americans are not to find themselves forced to travel to Japan for cures for their illnesses, the U.S. should adopt this model as well.
The High Human Toll
In recent decades, the time and costs required to obtain new medications, treatments, and therapies have increased significantly. A Tufts survey found that in the U.S. it takes on average over a decade and at a cost of $2.6 billion to bring new products from lab to market. A good portion of that time and expense is produced by the certification requirements of the U.S. Food and Drug Administration (FDA.)
After researchers develop what they think is a promising medication, it is tested on a small group of volunteers for basic safety during FDA’s Phase I test stage. Often in this first phase, it is apparent which products are the most promising. But ahead are usually years of Phase II and III trials on test groups and control groups of patients to determine general efficacy, the best dosages and the like. During the standard randomized clinical tests required in the latter phases, usually half the test participants receive the medication under review while the other half receives placebos.
This is where the real human toll comes in. During those years, sick Americans who are not in the very small test groups are denied access to safe, promising products. Many suffer and die during those years. When these products are eventually approved by the FDA, it is too late for many. The FDA and its over 15,000 employees do not suffer adverse consequences for the invisible graveyard their policies help to fill.
Japan faced similar problems and others as well. It has the oldest average population in the world and the longest life expectancy. Finding innovative treatments for the elderly was imperative. Many of the new and most promising regenerative medicine products did not fit into its existing regulatory regime.
Fortunately, Japanese policymakers had at hand the right idea at the right time: Free To Choose Medicine (FTCM), an innovative approach developed by Bartley Madden that would create an alternative pathway to traditional government drug certification, granting patients access to safe and promising treatments earlier and allowing physicians and patients to make sounder judgements about the efficacy of treatments. A Japanese translation of an early version of Madden’s FTCM book was promoted by activists in that country beginning in 2007.
In late 2014, Japan passed two new laws. The Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act (PMDA) adopted the FTCM regime for regenerative medicine products that could be reimbursed under that country’s health insurance system. The Act on the Safety of Regenerative Medicine (ASRM) set new rules for cell and tissue-based treatments that are provided by private clinics not reimbursed by the Japanese health insurance system. (The ASRM was fine-tuned in 2017.)
Under PMDA, essentially the FTCM model, treatments can be made available to patients after Phase I safety approval. Conditional and time-limited approval is available for up to seven years. A product or treatment, however, must still ensure product effectiveness to the satisfaction of Japanese regulators.
Access to Data
Of special note, PMDA not only accelerates access to products and therapies but also shifts the determination of efficacy from traditional premarket clinical trials to a post-market mechanism. The Japanese PMDA reforms were meant to facilitate the collection of and access to data based on market use. A central benefit of the FTCM system is its Tradeoff Evaluation Drug Database (TEDD). TEDD provides a mechanism for collecting real-world data as patients try different medications or therapies. Japanese regulators are now authorized to establish a similar system, composed of patient data. As patients use conditionally approved therapies outside the standard clinical testing track, the results can be placed in that registry, where names and other private information are shielded from researchers’ view.
Bring patient choice to America
In the United States, it was entrepreneurs pursuing their own innovative visions in a market relatively free of government regulation who created the computing, information, and communications revolution. American entrepreneurs, researchers, medical scientists and every one of us who are subject to the illnesses and aging that are the lot of all humans long to see a similar revolution in medicine. That is now possible. The cost of sequencing a human genome has dropped from $100 million in 2001 to around $1,000 today. Genetic engineering and bio-hacking are the cutting edge of this revolution. But only with the duel drivers of liberty for producers and individual choice for patients will the revolution occur.
Free To Choose medicine is the reform that can usher in a future of longer, healthier lives for all.
[Originally Posted at FEE.org]