Anyone who works in pharma knows the FDA. We’ve seen it forbid doctors from advising their peers how to dose a drug because the information doesn’t jibe with an ancient package insert. We’ve seen it delay new drug applications to settle scores for unrelated infractions. We’ve heard it promise to be more open yet fail to undertake meaningful reform.
But finally, change is in the wind. The Right to Try, a book written by public policy expert Darcy Olsen, might break down the FDA’s bureaucratic walls. Her book documents, often in heartbreaking detail, the FDA’s refusal to allow terminally ill patients access to promising investigational therapies — even when proven to be effective.
As she explains, the FDA has become so obsessed with ensuring the absolute safety of drugs that it has forgotten its charge to help ensure that effective drugs reach the public. Its reliance on double-blind, placebo-controlled trials with exhaustive inclusion and exclusion criteria is incompatible with today’s precision medicine.
In its defense the FDA points to large numbers of approved drugs, options for enrolling in clinical trials, and compassionate use programs. And while many new drugs are available, the glacial approval process prolongs the wait for thousands of terminally ill patients. Extra time is the one thing they do not have.
See also: Speeding up FDA Approvals Calls for Pause
What about clinical trials? For most people they’re a mirage. Only 3% of cancer patients who try to enroll are accepted. Can you imagine hearing about a new drug that might save your life and then learning that you don’t fit the criteria? Compassionate use programs are a logistical nightmare. Simply completing the application takes an average of 100 hours. Fewer than 9,000 such applications are granted yearly.
What sets Olsen’s book apart is that she goes beyond dramatizing the problem. She suggests a solution. It’s called “Right to Try.” Now enacted in 24 state legislatures, the idea is simple.
Patients with fatal diseases who have exhausted all other options would be allowed access to investigational drugs provided the drug has passed a phase I safety study, it’s still undergoing FDA-sanctioned clinical trials, the patient’s doctor agrees the drug may help, and the manufacturer is willing to provide it.
Assuming these criteria are met, the FDA would no longer be able to deny patients access to potentially lifesaving drugs.
I think that’s reasonable. It’s also worth noting that if this approach isn’t adopted, more radical measures may lie ahead. Sen. Ted Cruz (R-Texas), a presidential candidate, recently introduced legislation that would force the FDA to accept any drug that had been previously approved elsewhere in the world. That law would also establish a congressional committee with the power to reverse any given FDA decision. Based on what I know of Washington, that makes me nervous. I’m not a huge fan of the FDA, but it did save us from thalidomide, which would have been approved under Cruz’s plan.
Does The Right to Try strike a fair balance between sensible reform and eviscerating the FDA? Please read it in order to make an informed decision for yourself.
Sander Flaum is principal of Flaum Navigators.
From the April 01, 2016 Issue of MM+M - Medical Marketing and Media
