Best of the Week
Most Popular
1.Are UK Savings Interest Rates Finally Starting to Rise? Best Cash ISA 2017 - Nadeem_Walayat
2.Inflation Tsunami - Supermarkets, Retail Sector Crisis 2017, EU Suicide and Burning Stocks - Nadeem_Walayat
3.Big Moves in the World Stock Markets - Big Bases - Rambus_Chartology
4.The Next Financial Implosion Is Not Going To Be About The Banks! - Gordon_T_Long
5.Why EU BrExit Single Market Access Hard line is European Union Committing Suicide - Nadeem_Walayat
6.Trump Ramps Up US Military Debt Spending In Preparations for China War - Nadeem_Walayat
7.Watch What Happens When Silver Price Hits $26...  - MoneyMetals
8.Stock Market Fake Risk, Fake Return? Market Crash? - 2nd Mar 17 - Axel_Merk
9.Global Inflation Surges, Central Banks Losing Control and Triggered the Wage Price Spiral? - Nadeem_Walayat
10.Why Gold Will Boom In 2017 - James Burgess
Last 7 days
Here’s Why Interstate Health Insurance Won’t Fix Obamacare / Trumpcare - 23rd Mar 17
China’s Biggest Limitations Determine the Future of East Asia - 23rd Mar 17
This is About So Much More Than Trump and Brexit - 23rd Mar 17
Trump Stock Market Rally Over? 20% Bear Drop By Mid Summer? - 22nd Mar 17
Trump Added $3 Trillion in Wealth to Stock Market Participants - 22nd Mar 17
What's Next for the US Dollar, Gold and Stocks? - 22nd Mar 17
MSM Bond Market Full Nonsense Mode as ‘Trump Trades’ Unwind on Schedule - 22nd Mar 17
Peak Gold – Biggest Gold Story Not Being Reported - 22nd Mar 17
Return of Sovereign France, Europe’s Changing Landscape - 22nd Mar 17
Trump Stocks Bull Market Rolling Over? You Were Warned! - 22nd Mar 17
Stock Market Charts That Scream “This Is It” - Here’s What to Do - 22nd Mar 17
Raising the Minimum Wage Is a Jobs Killing Move - 22nd Mar 17
Potential Bottoming Patterns in Gold and Silver Precious Metals Stocks Complex... - 22nd Mar 17
UK Stagflation, Soaring Inflation CPI 2.3%, RPI 3.2%, Real 4.4% - 21st Mar 17
The Demise of the Gold and Silver Bull Run is Greatly Exaggerated - 21st Mar 17
USD Decline Continues, Pull SPX Down as well? - 21st Mar 17
Trump Watershed Budget - 21st Mar 17
How do Client Acquisition Offers Affect Businesses? - 21st Mar 17
Physical Metals Demand Plus Manipulation Suits Will Break Paper Market - 20th Mar 17
Stock Market Uncertainty Following Interest Rate Increase - Will Uptrend Continue? - 20th Mar 17
Precious Metals : Who’s in Charge ? - 20th Mar 17
Stock Market Correction Continues - 20th Mar 17
Why The Status Quo Is Under Increasing Attack By 'Populist People Power' - 20th Mar 17
Why the SNP WILL Destroy Scotland, Exit UK Single Market for EU - IndyRef2 - 19th Mar 17
Crypto Craziness: Bitcoin Plunges on Fork Concerns, Steem Skyrockets and Dash Surges Above $100 - 19th Mar 17
What ‘Ice-Nine’ Means for Your Money - 19th Mar 17
Stock Market 4 Year Cycle - 18th Mar 17
The Only Article You Need to Read to Understand the Trump Phenomenon - 17th Mar 17
Janet Yellen Just Popped the Stock Market Bubble - 17th Mar 17
Financial Crisis, Steve Eisman: Smart, Lucky, Abrasive & Now One Of Them - 17th Mar 17
Gold Cup – Horse Racing’s Greatest Show, Gambling and ‘Going for Gold’ - 17th Mar 17
Trader Education Week - Free Event to Help You Learn to Spot Trading Opportunities - 17th Mar 17
$1.4 Trillion of SPX Notionals Due to Expire - 17th Mar 17
Preserving Order Amid Change in NAFTA, U.S. Sovereignty v. WTO - 17th Mar 17
3 Maps That Explain Why Syria Raqqa Battle Will Drag On - 17th Mar 17
Crude Oil Price Outlook 2017 - Video - 16th Mar 17
Dutch and French Electons - Winners are Losers and Left is Right - 16th Mar 17
The Straddle Trade Stock Market Brief - 16th Mar 17
Gold Up 1.8%, Silver Up 2.6% After Dovish Fed Signals Slow Interest Rate Rises - 16th Mar 17
Stocks Get Close To Record High Again As Fed Hikes Interest Rates - 16th Mar 17
Scotland Second Independence Referendum War - SNP Determined to Destroy the UK - 16th Mar 17
Here’s How Pharma Is Using AI Deep Learning To Cure Aging - 16th Mar 17
Stock Market Chaos in the Chicken Coop - 15th Mar 17
Gold and Silver Price Manipulation: The Biggest Financial Crime In History - 15th Mar 17
“Ryancare” Dead on Arrival: Can We Please Now Try Single Payer? - 15th Mar 17
Fanaticism, Stock Market Crash 2017 or Continuation of Bull Market - 15th Mar 17
Stock Market Most Overvalued On Record — Worse Than 1929? - 15th Mar 17
Desperate Saudi Arabia Turns to Asia for Investment - 15th Mar 17
Startups Will Define the Future of US Employment - 15th Mar 17
Fed Rate Hikes, Fiscal vs. Monetary Policy and Why Again the Case for Gold? - 15th Mar 17
SNP Declare Scotland to Commit Economic Suicide Early 2019, 2nd Independence Referendum - 14th Mar 17

Market Oracle FREE Newsletter

Elliott Wave Trading

Trump’s FDA Chief May Implement Progressive Approval for Drugs

Companies / Healthcare Sector Dec 20, 2016 - 07:35 AM GMT

By: John_Mauldin

Companies

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).

Stay in the Loop on Life-Extending Research with Patrick Cox's Tech Digest

This weekly newsletter by biotech expert Patrick Cox highlights research that is much more advanced than most people know, and the profit potential for investors is vast. Read about the latest breakthroughs—from new, non-invasive cancer treatments to age-reversing nutraceuticals and vaccines that kill any virus–as well as the innovative companies that work on them. Get Tech Digest free in your inbox every Monday.

(*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.)

John Mauldin Archive

© 2005-2016 http://www.MarketOracle.co.uk - The Market Oracle is a FREE Daily Financial Markets Analysis & Forecasting online publication.


Post Comment

Only logged in users are allowed to post comments. Register/ Log in

Catching a Falling Financial Knife