Founder of Health Rising and Phoenix Rising
“Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need,” he said. “If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles just like Megan.” Donald Trump - Speech to Congress
One could hardly imagine the bombastic Donald Trump - the man who called veterans with PTSD "weak", who said the National Football League was "soft", who derided Hillary Clinton's and Jeb Bush's lack of energy - thinking anything good about a disease called "chronic fatigue syndrome".
Trump hasn't appointed a new FDA chief but the first name leaked by his team was a doozie. Jim O'Neil, the managing director of Peter Thiel’s Mithril Capital supports a dramatic change in how the FDA approves drugs. O'Neil supports something called "progressive approval" of drugs championed by a former FDA chief Andrew Von Eschenbach.
Citing stats indicating that it takes about 12 years and $1.2 billion to bring a new drug to market in the U.S. Eschenbach asserts that the drug and device approval system employed by the FDA is broken.
Under Eschenbach's system all a company would have to do to get an initial FDA approval would be to do a Phase I trial that proves the drug is safe. If it is, then the drug's effectiveness would be monitored. If it works, insurance companies would pay for it; if it doesn’t they wouldn't. Eschenbach believes that the informational technologies available today make it easy to monitor drug effectiveness in patients.
The main problem, Eschenbach believes, are the large and costly Phase III trials required to prove efficacy. These trials are so costly - they take up about 25% of the cost to bring a drug to market and generally take several years to complete - that they have a name. They’re called the "valley of death" for the many drugs whose progress gets stymied by them.
Pain researcher Linda Watkins blames the "valley of death", for instance, for the overemphasis doctors have on using opioids for pain reduction. Citing dozens of potential pain-killing drugs that got stalled in the “valley of death” she suggested that it was the current drug business model – not the science – that has let consumers down.
“A lot of really innovative stuff reaches ‘Death Valley’—it’s a good idea, it has good rodent data, and it’s on the edge of a clinical trial, but it needs millions of dollars in funding, It’s very difficult to get a pharmaceutical company or venture capitalist firm to invest without data showing that the treatment works in humans, she says. From a business perspective, opioids are a much safer bet."
Eschenbach argues that in the past these trials often worked, but with the precision medicine revolution producing drugs that are increasingly being aimed at smaller and smaller subsets of patients, these large and expensive trials don't make sense. That argument could be made larger if disease groups such as chronic fatigue syndrome which drug manufacturers have been eluctant to fund drug trials for get behind it .
Eschenbach's system sounds something like ME/CFS advocates have proposed for Ampligen for years: get the safety data, then target patients and do post-marketing studies. If the drug fails then pull it - or as Eschenbach proposes - allow insurers to not pay for the drug if it does not succeed.
Under our proposed system, the drug could have come to market after promising early-stage research in targeted patients, with appropriate post-marketing studies required. Payers and patients would be the ultimate judge about the quality of the product, and companies could learn from the experience to develop superior products if needed.
Companies would still be liable for unforeseen side effects, but patients and doctors would be warned -- through the drug’s labeling -- that the product had been approved based on promising but provisional research.
Eschenbach asserts that simply doing away with the Phase III trials would free up drug makers to introduce at least twice as many drugs as they are now. Safety concerns would be largely alleviated by Phase I safety trials. Japan is now using a similar system.
Big pharma, interestingly, is not a fan. With their deep pockets they’re in the position to pick and choose products developed by smaller pharmaceutical companies that lack the cash to go to large phase III trials.
Patients would undoubtedly have to assume more risk under this new system but they would also have access to more drugs. One article cited Dr. Michael West's spinal cord stem cell therapy that was recently approved -20 years after it was invented – to help quadriplegics.
“When you have a drug, you can actually get it approved if it works, instead of waiting for many, many years. We’re going to be cutting regulations at a level that nobody’s ever seen before, and we’re going to have tremendous protection for the people.” He estimated that a jaw-dropping 75% to 80% of regulations are unnecessary.
Dr. Carome of the consumer watchdog group Public Citizen called that idea horrifying.
"Trump's horrifying proposal reflects utter ignorance about the FDA's essential role in protecting public health and once again demonstrates his commitment to placing corporate profits above protecting the safety of the American people,"
Pushing back, the FDA published a report of 22 drugs that did well in smaller trials only to fail in larger ones. FDA proponents reported that the FDA now approves drugs more quickly than Europe does (but that for now 1000 staff appointments are going unfilled because of Trump’s hiring freeze.)
Plus naysayers cite a chaotic future where drug companies pour out legions of drugs most of which end up having little efficacy. Since Medicare and Medicaid are required to pay for FDA approved drugs, the costs of those programs would skyrocket.
Others believe putting companies in charge of assessing drug efficacy after approval would be foolhardy given the dollars involved.
But then there's Ampligen with it’s established safety record but no money for a big phase III trial for decades. Rituximab is working for at least some patients in Norway but there is no push for a Rituximab trial in the U.S. (Dr. Koroshetz made it clear that it would be years before the NIH could even begin thinking about funding ME/CFS treatment trials.)
Dr. Peterson reported that 17 cytokine blocking drugs that might work for some people with ME/CFS are on the market -none of which are available for ME/CFS. Many drugs will never receive Phase III testing for ME/CFS or FM because the numbers don't pan out for drug companies under the current drug testing regime.
Whatever happens with the FDA, Trump hardly looks like his term will be an unalloyed benefit for ME/CFS or FM patients. His plans to increase military spending, repeal and replace the Affordable Care Act, and cut taxes for the wealthy and corporations could impact ME/CFS?FM patients in other ways. NIH spending could be cut and some could lose their heath insurance and/or other benefits. A ballooning deficit under Trump could put pressure on medical funding for years to come.
But his ideas for the FDA? Those could be something. What do you think?