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Fast-Tracking Safe Drugs without Sacrificing Scientific Rigor – Article by Alex Tabarrok

Fast-Tracking Safe Drugs without Sacrificing Scientific Rigor – Article by Alex Tabarrok

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The New Renaissance HatAlex Tabarrok
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Here’s how to do it

In a post earlier this year, I noted that Japan has significantly liberalized its approval process for regenerative medicine. Writing in Forbes, Bart Madden and Nobelist Vernon Smith outline a similar proposal for the United States.

Recently, Japanese legislation has implemented the core Free To Choose Medicine (FTCM) principles of allowing not-yet-approved drugs to be sold after safety and early efficacy has been demonstrated; in addition, observational data gathered for up to seven years from initial launch will be used to determine if formal drug approval is granted. …

FTCM legislation in the U.S. would create a dual track system (see figure below) that preserves the existing FDA clinical trial process while offering patients an alternative. Patients, advised by their doctors, would be able to contract with a drug developer to use not-yet-approved drugs after Phase I safety trials are successfully completed and one or more Phase II trials have demonstrated continued safety and initial efficacy.

The resulting early access could make FTCM drugs available up to seven years before conventional FDA approval, which entails Phase III randomized control trials and a lengthy FDA review before the FDA makes an approval decision. …

bartgraph

The heart of the dual track system is the Tradeoff Evaluation Drug Database (TEDD) which would be available to the public through a government-supervised web portal. TEDD would contain all treatment results of FTCM drugs including patients’ health characteristics and relevant biomarkers, but no personal identification.

This open access database would be a treasure-trove of information to aid drug developers in making better R&D decisions consistent with fast-paced learning and innovation. …

Today’s world of accelerating medical advancements is ushering in an age of personalized medicine in which patients’ unique genetic makeup and biomarkers will increasingly lead to customized therapies in which samples are inherently small. This calls for a fast-learning, adaptable FTCM environment for generating new data.

In sharp contrast, the status quo FDA environment provides a yes/no approval decision based on statistical tests for an average patient, i.e., a one-size-fits-all drug approval process.

I hold the Bartley J. Madden Chair in Economics at the Mercatus Center, so I am biased, but this is an important proposal. Japan is leading the way and similar ideas are being discussed in Great Britain, but as the most important pharmaceutical market in the world, the United States has an outsize influence on world drug development. We need to lower costs and speed new drugs to market.

This post first appeared at Marginal Revolution.

Alex Tabarrok is a professor of economics at George Mason University. He blogs at Marginal Revolution with Tyler Cowen.

Japan Liberalizes Gene Therapy and Regenerative Medicine – Article by Alex Tabarrok

Japan Liberalizes Gene Therapy and Regenerative Medicine – Article by Alex Tabarrok

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The New Renaissance HatAlex Tabarrok
September 17, 2015
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Japan is liberalizing its approval process for regenerative medicine:

Regenerative medicines in Japan can now get conditional marketing approval based on results from mid-stage, or Phase II, human trials that demonstrate safety and probable efficacy.

Once lagging behind the United States and the European Union on approval times, there is now an approximately three-year trajectory for approvals, according to Frost’s Kumar. That compares with seven to 10 years before. …

Around the world, companies have also faced setbacks while pushing such treatments. In the U.S., Geron Corp., which started the first nation-approved trial of human embryonic stem cells, ended the program in 2011, citing research costs and regulatory complexities. …

While scientists globally have worked for years in this field, treatments have been slow to come to market. But there is hope in Japan that without the political red tape, promising therapies will emerge faster and there will be speedier rewards.

Japan is liberalizing because with their aging population treatments for diseases like Alzheimer’s and Parkinson’s disease are in high demand.

Under the new system, a firm with a gene or regenerative therapy (e.g. stem cells) can get conditional approval with a small trial. Conditional approval means that the firm will be able to sell its procedure while continuing to gather data on efficacy for a period of up to seven years. At the end of the seven-year period, the firm must either apply for final marketing approval or withdraw the product.

The system is thus similar to what Bart Madden proposed for pharmaceuticals in Free to Choose Medicine.*

Due to its size and lack of price controls, the US pharmaceutical market is the most lucrative pharmaceutical market in the world.

Unfortunately, this also means that the US FDA has an outsize influence on total world investment. The Japanese market is large enough, however, that a liberalized approval process if combined with a liberalized payment model could increase total world R&D.

Breakthroughs made in Japan will be available for the entire world so we should all applaud this important liberalization.

This post first appeared at Marginal Revolution.

* Editor’s note from the Foundation for Economic Education: There may well by a direct connection here. According to Madden, an early version of his proposal in Free to Choose Medicine was published in a booklet by the Heartland Institute, which was then translated and distributed in Japan by a Japanese free-market think tank.

For more on free markets in medicine, see Bart Madden’s article “The Pathway to Faster Cures” in the autumn print edition of the Freeman and on The Rational Argumentator.

Alex Tabarrok is a professor of economics at George Mason University. He blogs at Marginal Revolution with Tyler Cowen.