Trump’s FDA Chief May Implement Progressive Approval for Drugs

By: John_Mauldin

BY PATRICK COX : President-elect Trump's cabinet appointments are generating a great deal of attention and controversy. After eight years of Democrat rule, it would be foolish to expect otherwise.

One appointment, though, will have a profound impact on your health… and the health of your loved ones.

The “progressive approval” process for drugs

The first potential FDA chief appointee leaked by the Trump transition team is Jim O’Neill. Managing director of Peter Thiel’s Mithril Capital, O’Neill has publicly supported proposals to do away with the FDA’s requirement for phase 2 and 3 trials. Instead, he favors “progressive approval” of drugs and other medical technologies.

O’Neill would not be the first FDA head to favor progressive drug approval. Andrew von Eschenbach developed this system when he served as FDA chief. But during his tenure, Eschenbach was not able to put such a far-reaching reform into practice in the US.

The Japanese, though, saw the value of Eschenbach’s plan. They now use it for regenerative medicine. Japan legalizes therapies following proof of safety. Once a drug is in use, companies can move to phase 4. This means they monitor their patients and disclose efficacy data regularly.

If O’Neill gets the nod, he could implement progressive approval for drugs. But he would face fierce opposition from those who profit from the current system.

The current drug approval system is flawed

Big pharma benefits a great deal from the status quo. Since many small biotechs can’t fund most phase 2 or 3 trials, they give up significant ownership of their biotechnologies to these giants in exchange for financial help.

Many other organizations are also paid billions each year for services related to phase 2 and 3 clinical trials. They too will argue that doing away with those trials would endanger patients.

Since therapies would have to be proven safe before reaching the public, they will charge that patients will be at risk from using treatments that don’t work.

This, of course, is true. But it’s also true that patients are put at risk because they are denied access to unapproved but effective drugs.

Also, we pay much more for available drugs because companies must recoup costs imposed by the regulatory system. How big are those costs? Estimates of the average cost of one new medicine range from $1 to $4 billion.

Another side effect of the current system is that many drugs never leave the lab. These are drugs that could effectively treat serious illness but aren’t worth the cost of approval. This is because the patient population is too small to recoup approval costs.

The FDA has made efforts to help to help biotechs develop orphan drugs, but these steps have not been enough to make much of an impact.

Genomics and the need for a new regulatory process

Genomics has changed the way we treat illness. As the cost of genome sequencing falls, the ability to design drugs that work in small populations with specific genetic characteristics is rapidly increasing.

Eric Topol is the Director of the Scripps Translational Science Institute. He suggests that randomized clinical trials for this kind of indication are not just impractical, they’re unethical. InSilico Medicine’s Alex Zhavoronkov is one of the leading researchers in genomics-based personalized medicine. He believes we will be able to design and manufacture drugs for a single genome.

But how do you do a blinded controlled trial for a drug that may work on just one person?

This is not just an issue for the future. Many life-saving breakthroughs that could save millions of lives and reduce medical costs are stalled right now. Two of the most important areas of research are geroprotectors and induced tissue regeneration (ITR).

Geroprotectors are compounds that repair the systems that accelerate aging and age-related disease. Major universities and research organizations have found at least half a dozen molecules that could slow aging and extend health spans… even in older people.

Why haven’t you seen these compounds? Because phase 2 and 3 trials would take a decade or more… while increasing their prices by orders of magnitude.

Induced Tissue Regeneration (ITR) has even greater potential. ITR is an emerging technology that could reactivate the embryonic healing powers hidden deep in your DNA. It could restore lost limbs, broken spinal cords, or any other body part to youthful function. Eyes, hearts, joints, skin, and livers could be taken back in time using relatively simple compounds.

If it weren’t for the fact that Dr. Michael West is spearheading this new field, I would dismiss it as a pipe dream. West’s spinal cord stem cell therapy recently saved complete quadriplegics from a life of total dependency. Almost no one thought that he could do this. In fact, I got emails saying that the trial would fail, and this was after the first patient got back to ability to lift weights, text, and care for himself.

The important thing to note about this therapy is how long it took to get approved. West invented it over 20 years ago. It took that long to drag this miraculous biotechnology through the regulatory maze.

The big picture for ITR

Over the past 20 years, the pace of West's research and scientific progress has accelerated along with breakthroughs in computers and genomics. Now, he is on the verge of activating in adults the same genetic power evident in developing embryos. 

For years, I’ve gleaned what I could about this project by following West’s public statements and patent applications. Finally, he’s begun to tell the whole revolutionary story. Following the success of spinal cord technology, he laid out the big ITR picture in a keynote address to the prestigious World Stem Cell Summit.

ITR may be the biggest breakthrough in the history of medicine. The ability to make old and damaged organs and tissues young again will change countless lives. For millions of older people, though, it will come too late unless the regulatory bureaucracy is transformed to accelerate life-saving therapies.

Maybe Jim O’Neill will be the person to make it happen.

In the interest of transparency, I should note that I own stock in Michael West’s company BioTime (*see disclosure below).

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(*Disclosure: The editors or principals of Mauldin Economics have a position in this security. They have no plans to sell their position at this time. There is an ethics policy in place that specifies subscribers must receive advance notice should the editors or principals intend to sell.)

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