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Newt Gingrich emailed the following report on the outdated and bureaucratic FDA.
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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:
"Is it rite to save 1 child
an[d] not the rest?” wondered one commenter on a news forum. “It’s really not
fair to the thousands of others that were turned down just because they didn’t
make a big public outcry,” said another.
"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:
“You couldn’t get a more troubling
and impossible-to-resolve moral dilemma than this one,” said Arthur Caplan,
director of the division of medical ethics at New York University’s Langone
Medical Center.
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.
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The FDA is not the only federal agency that should be
eliminated or radically changed.
