by Patrick Cox
June 5, 2018
A lot of people were surprised how quickly Right to Try (RTT) became federal law.
In theory, RTT will help patients with terminal diseases get life-saving drugs. In practice, it will have marginal if any benefit for most people who need drugs that haven’t gone through the FDA’s entire clinical trial process. However, what its passage says about the future is extremely important.
To understand why, we need to understand the two sides of the story.
Many of the most important institutions in our healthcare system, from Big Pharma to the FDA, opposed RTT. These institutions are incentivized to put caution and safety above all because they suffer directly when approved drugs cause unexpected harm. From their perspective, RTT is reckless.
On the other side of the argument is a large majority of Americans who favor a shift of medical responsibility from the federal government to doctors and patients. They want more control of their own medical decisions and tend to focus on the harm that comes from being denied potential cures. They clearly won.
A POLITICO-Harvard poll found that two-thirds of Democrats endorsed RTT, with slightly higher support from independents and Republicans. In a period of strident partisan polarization, this is a remarkable consensus. RTT has strong bipartisan backing from wide swaths of society, ranging from academic researchers to advocates of alternative medicine.
Overwhelming public support led, in less than four years, to the enactment of separate RTT laws in 40 states. But state laws couldn’t force the FDA to streamline existing processes for patients seeking access to experimental therapies. So, in 2017, the Senate responded to public pressure by unanimously approving the RTT bill. President Trump endorsed the move in his first State of the Union address.
Since it is backed by so much public support, you might assume that the new RTT law will have a major impact on the practice of medicine. I initially did, largely because the legislation included liability protection for companies that make their therapies available to terminal patients.
I’ve always heard that the reason biotechs balked at expanded access, the prior program for accessing unapproved therapies, was a fear of lawsuits. RTT addressed that issue by reducing drug companies’ legal liabilities, but it can’t stop malevolent lawsuits.
We know this is a serious problem because “short and distort” attackers have brought lawsuits that they knew would fail in court. These lawsuits, often accompanied by notices on financial websites seeking other participants for class actions, target biotech firms with the aim of sowing fear and driving down stock prices.
Drug companies routinely win these court battles, but they are expensive, distracting, and can be exploited by unscrupulous market manipulators to seriously hurt targeted companies.
Fear of the FDA’s Hawk Eyes
Having spoken with scientists and executives in the drug development business since RTT became law, I no longer believe that lawsuits are their only concern. What I’ve learned is that startup biotechs weren’t telling me the whole story. They’re equally if not more afraid that they will bias the FDA by utilizing a program that the agency largely opposed.
Remember, RTT only applies to drugs that are in the clinical-trial process, so any company that participates still depends on FDA approval. RTT complicates the picture.
When formal clinical trials are designed for terminal diseases, scientists and clinicians go to tremendous lengths to find patients who are not already so sick that they will die before the trial is complete. This is extremely difficult, and mistakes have derailed promising drugs in the past.
A biotech firm offering a drug for RTT could screen the patients requesting its drug, but that screening process would be too expensive for most startups. Without it, though, the odds go up that patients will die or have dramatic side effects, which generates bad press.
Theoretically, the FDA wouldn’t hold such events against a company, but it’s a risk that few drug developers want to take. I therefore predict that consumers are going to be disappointed by the actual results of RTT, which will further aggravate public distrust of the pharmaceutical industry and the FDA.
Tensions are already rising due to demographic pressures. The US population is aging, and the number of people with expensive, life-shortening diseases is growing. This not only increases the cost of healthcare for the aged population—already the biggest component of the federal budget and debt—it is creating a powerful political lobby of older people who want faster access to new drugs.
Simultaneously, medical science is accelerating, and stories about miracle cures just a few years down the road have become increasingly common. Those who were surprised that RTT passed so quickly and easily shouldn’t have been. Much more serious changes, probably along the lines of Japan’s progressive approval process, are coming soon.
Japan has aged ahead of America, so the oldest population in the world wants and needs new therapies that can extend healthspans. Japanese politicians know this and have reformed their system to give conditional approval to therapies that directly address aging and the diseases of age, allowing sales following phase-1 safety trials.
The Japanese are not reckless, they’re ahead of the curve. The same change will come to the US, probably before you expect it.