March 26, 2014
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The Food and Drug Administration is destructive of the health of the American people. It is an obsolete bureaucracy with obsolete rules that do more harm than good.
The time has come to think through a 21st century replacement for a 108-year-old bureaucracy.
Two people, Joshua Hardy and Ron Woodroof, personify the problems with the FDA.
Joshua is a seven-year-old boy who became a national story this week seeking compassionate relief for a drug that might save his life but was not available because of the current bureaucracy, which takes 10 to 15 years to approve a drug.
Ron has been dead since September 1992 but he is currently famous because of a remarkable movie, The Dallas Buyers Club, and an extraordinary Academy Award winning performance by Matthew McConaughey as Ron Woodroof.
Both stories illustrate fatal flaws in the current FDA system.
In Woodroof’s case he was told in 1985 that he only had 30 days to live. By breaking out of the system he found a series of drugs (many of them vitamins) that helped him live for 7 years — 84 times longer than his doctors thought possible.
Instead of seeing Woodroof’s remarkable achievement as something worth studying and learning from, the FDA and the medical establishment found his activities a direct threat to their authority. The movie is painful to watch both because people are dying and because our government is making their lives harder as they struggle to survive.
In effect Woodroof is told he can’t try new solutions because they aren’t approved — even though the approved options will mean certain death.
He keeps pointing out that people are dying but the bureaucratic urgency is reserved for enforcing the establishment’s authority.
This same passion for propping up the old order became apparent when seven-year-old Joshua sought help.
Joshua became famous when concerned citizens launched an online petition and got over 20,000 signatures. The drug he needed was available but the small company that developed it was heavily invested in the lengthy, expensive FDA approval process. A bad result for a single high-profile case like Joshua’s would be a significant risk to its investment.
The FDA bureaucracy has become so cumbersome and so demanding that hardly any inexpensive drug or drug aimed at a small market (people with a rare disease) can overcome the cost of getting through the process.
FDA approval can easily cost $1 billion dollars.
Small companies literally bet their existence on getting their first drug approved.
This was the situation when Joshua needed the new drug. Technically the FDA might approve compassionate use but the company was afraid to risk its investment in the FDA trials.
A woman named Andrea Sloan encountered the same lethal bureaucracy when she tried to get a new, experimental cancer drug last year. I talked with her oncologist at M.D. Anderson, the largest cancer center in the world. He was totally frustrated by the system. She never got the drug and she died at 45.
Similarly Nick Audin got more than 500,000 signatures through an on line petition. He had three children and yet he never got the drug he needed. He died at 41.
The current system is destructive, arbitrary and at times heartless. Yet the reaction of the establishment is to defend it. The opposition to seven-year-old Joshua getting compassionate relief was captured perfectly in the Washington Post headline “Ethicists Worry Josh Hardy Case May Set Bad Precedent“.
The Post described a “backlash” against helping the young boy:
“The Herald-Sun newspaper in Durham, N.C., where the company that makes the drug is based, said it was glad for the boy’s sake that he was able to get the medicine. “But the process leaves us pained,” the editorial board wrote. “This is no way to make health-care decisions.”
“The story of how Joshua Hardy — a first-grader from Fredericksburg, Va., who is fighting off an infection after getting a bone-marrow transplant — got access to an unapproved treatment when others with similar requests were turned down highlights the ethical conundrums facing doctors, companies and regulators in the era of Facebook and Twitter.”
The Post quoted one expert who managed to get the problem exactly backward. Consider his line of reasoning:
From the perspective of the public and future patients, it’s best for the company to focus on getting the drug approved as soon as possible so that the largest number of people can be helped, Caplan said. But from a patient’s point of view, getting immediate access to the drug is what’s important.
“It’s a trade-off between the public good versus self-interest,” Caplan said. “They conflict. There is no way of getting around it.”
Yet Dr. Caplan has the problem exactly backward.
The focus should not be on the ethics of getting drugs through the slow and outdated system.
The focus should be on systems changes that would accelerate access to the newest, best, and most effective solutions.
Rationing transplants, for example, should be replaced with a dramatic acceleration of regenerative medicine so you can replant your own organs grown from your own cells in a manner that will eliminate rationing and waiting.
People faced with terminal illnesses should have informed-consent access to any drug which has passed a stage-one safety test. If the drug won’t kill you and you have been told you are dying, you should have the right to experiment if you choose to live out your challenge (the opposite of the Dallas Buyers Club experience).
The use of placebos (where half of a test group is knowingly given something which will not work) should be ended. First, it is immoral to give people in desperate circumstances a false medicine. Second, with forty or fifty years of clinical trial data and new continuous, real time medical monitoring possible through mobile devices, it is possible to imagine a dramatically different model of testing new therapies. Many more people can be involved, the costs can be dramatically less, and the volume and speed of information flow can be enormous compared to the pre-smartphone, pre-wireless, pre-computer world.
A largely paper-based and slow moving FDA bureaucracy is simply incapable of this kind of modern, personalized model of developing new solutions in healthcare. That is why it needs to be replaced rather than reformed.
Instead of having compassionate access as a rare event requiring massive publicity, we should redesign the system to lower the cost of approval so companies can afford to share breakthroughs even during the testing phase.
Every American deserves compassion and we should design a system to achieve that goal.
Finally there has to be a very inexpensive approval process for breakthroughs with small but important market potential. A drug should not need to be worth billions to merit FDA approval.
Listen to my Breakout podcast next week for much more on this topic.
Remember Joshua and Ron, and demand a new approach to approving drugs and therapies.