This is interesting: The Right-to-Try Revolt
Terminally ill patients are fed up with slow FDA drug approvals.
It should not go unnoticed that last week the Food and Drug Administration announced it would “simplify and accelerate” the application process for “unapproved investigational drugs” that have passed Phase I safety trials. We are back on the most contentious ground of FDA drug approval, involving the most desperate, terminally ill patients.
The current form used by such patients for access to unapproved drugs is so complex that it takes about 100 hours to complete it and requires seven attachments. The new form will take 45 minutes, asks attending physicians to fill in eight pieces of patient information and requires one attachment.
For sick patients seeking access to experimental drugs this would appear to rank with Moses parting the Red Sea. It is surely less than that, but patient groups already know it takes an army to move the FDA bureaucracy. So-called “compassionate-use” protocols exist mainly because in 1988 some 1,000 people from the AIDS advocacy group called ACT Up marched on and surrounded the FDA’s main building in Rockville, Md. The FDA bent.
No such mass movement exists today for seriously ill people, who of late have turned to state legislatures to enact so-called “right to try” laws. Last year, at the urging of grassroots groups like the Goldwater Institute, five states enacted such laws with wide bipartisan majorities. Another 26 states, including California and Florida, may follow.
More than anything, the FDA’s bureaucrats have proven they are politically astute. The right-to-try laws do not eliminate the FDA’s regulatory authority. The state laws essentially say that patients and their doctors may seek to use drugs that have passed the FDA’s first phase of safety tests. Right-to-try laws allow patients and doctors to negotiate directly with drug companies for access to experimental drugs.
The agency said last week it’s “committed to streamlining its processes,” but the more likely explanation is that the bureaucracy felt pressured by a groundswell of frustrated patients. As with the AIDS era’s creation of compassionate use, the spreading popularity of these new state-based access laws has the makings of a movement that could eventually find its way to Congress.
That is where the FDA’s control of the American drug-development process could come under real pressure, if Republicans in 2017 control both Congress and the Presidency. Most likely, the FDA recognized it had to do something to slow down the right-to-try political juggernaut. Whatever its improvements, the new form still keeps the process under FDA control.
The issue of access to experimental drugs for terminally ill patients is fraught with moral, ethical, scientific and economic concerns. Drug makers, especially smaller biotechnology companies, can feel caught between patients on one side and an FDA approval process that can cost at least $1 billion before a single sale of the new drug. None of those issues will disappear.
We’re not going to look a gift horse in the mouth. Whether the decision to expedite access means the agency knows its processes are broken or is merely an act of bureaucratic realpolitik, the long battle to help the nation’s sickest patients has moved forward again—and at last.
http://investorstemcell.com/forum/ocata-main-.../49571.htm
Terminally ill patients are fed up with slow FDA drug approvals.
It should not go unnoticed that last week the Food and Drug Administration announced it would “simplify and accelerate” the application process for “unapproved investigational drugs” that have passed Phase I safety trials. We are back on the most contentious ground of FDA drug approval, involving the most desperate, terminally ill patients.
The current form used by such patients for access to unapproved drugs is so complex that it takes about 100 hours to complete it and requires seven attachments. The new form will take 45 minutes, asks attending physicians to fill in eight pieces of patient information and requires one attachment.
For sick patients seeking access to experimental drugs this would appear to rank with Moses parting the Red Sea. It is surely less than that, but patient groups already know it takes an army to move the FDA bureaucracy. So-called “compassionate-use” protocols exist mainly because in 1988 some 1,000 people from the AIDS advocacy group called ACT Up marched on and surrounded the FDA’s main building in Rockville, Md. The FDA bent.
No such mass movement exists today for seriously ill people, who of late have turned to state legislatures to enact so-called “right to try” laws. Last year, at the urging of grassroots groups like the Goldwater Institute, five states enacted such laws with wide bipartisan majorities. Another 26 states, including California and Florida, may follow.
More than anything, the FDA’s bureaucrats have proven they are politically astute. The right-to-try laws do not eliminate the FDA’s regulatory authority. The state laws essentially say that patients and their doctors may seek to use drugs that have passed the FDA’s first phase of safety tests. Right-to-try laws allow patients and doctors to negotiate directly with drug companies for access to experimental drugs.
The agency said last week it’s “committed to streamlining its processes,” but the more likely explanation is that the bureaucracy felt pressured by a groundswell of frustrated patients. As with the AIDS era’s creation of compassionate use, the spreading popularity of these new state-based access laws has the makings of a movement that could eventually find its way to Congress.
That is where the FDA’s control of the American drug-development process could come under real pressure, if Republicans in 2017 control both Congress and the Presidency. Most likely, the FDA recognized it had to do something to slow down the right-to-try political juggernaut. Whatever its improvements, the new form still keeps the process under FDA control.
The issue of access to experimental drugs for terminally ill patients is fraught with moral, ethical, scientific and economic concerns. Drug makers, especially smaller biotechnology companies, can feel caught between patients on one side and an FDA approval process that can cost at least $1 billion before a single sale of the new drug. None of those issues will disappear.
We’re not going to look a gift horse in the mouth. Whether the decision to expedite access means the agency knows its processes are broken or is merely an act of bureaucratic realpolitik, the long battle to help the nation’s sickest patients has moved forward again—and at last.
http://investorstemcell.com/forum/ocata-main-.../49571.htm
