Posted 02 February 2017
By Michael Mezher
In a meeting with pharmaceutical industry executives on Tuesday, President Donald Trump expressed his concern that the US Food and Drug Administration (FDA) is standing in the way of terminally ill patients accessing unapproved drugs as treatments of last resort.
"One thing that's always disturbed me, they come up with a new drug for a patient who is terminal, and the FDA says 'we can't have this drug used on the patient.' But they say, 'But the patient within four weeks will be dead.' They [FDA] say, 'Well, we still can't approve the drug and we don't want to hurt the patient." But the patient is not going to live more than four weeks,'" Trump said at the meeting.
This scenario closely mirrors the one told by "right-to-try" proponents, who have successfully advocated for right-to-try laws that allow patients to get access to unapproved drugs from manufacturers without FDA's involvement in 33 states.
While Trump has not formally endorsed such efforts, Vice President Mike Pence signed Indiana's right-to-try bill into law in 2015 and made statements during the presidential campaign in support of such legislation on a national level.
Dr. Vinay Prasad, assistant professor of medicine at the Knight Cancer Institute at the Oregon Health & Science University, challenged Trump and right-to-try supporters' portrayal of access to unapproved drugs, pointing to FDA's expanded access program.
"Trump is incorrect that dying patients cannot try these drugs. The major barrier to right-to-try is the pharma industry, who declines some requests," Prasad told Focus, as such laws do not require manufacturers to give the drugs to patients.
Through what FDA calls expanded access, the agency allows patients with serious or immediately life-threatening diseases to have their doctors request access to unapproved investigational drugs outside of a clinical trial. Since 2010, FDA has approved between 98% and 99% of the more than 1,000 requests for expanded access each year.
However, FDA has been criticized in the past for the expanded access process being overly cumbersome for patients and physicians.
In response, the agency introduced changes to streamline and simplify the program that went into effect last June.
For its part, the pharmaceutical industry trade group PhRMA has emphasized the role of FDA and clinical trials in light of right-to-try efforts.
"Because investigational medicines have not been approved by the FDA and determined to be safe and effective, FDA must approve expanded access to an investigational drug before a biopharmaceutical company can provide it to a patient," said PhRMA spokesperson Caitlin Carroll. "Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients."
Impact of Right-to-Try
And so far, the impact of state right-to-try laws has been difficult to ascertain.
Despite the widespread adoption of such laws in more than 30 states, examples of patients gaining access to unapproved drugs through such laws are scarce, and outcomes data for those patients are even rarer.
In an email to Focus, Starlee Coleman, vice president of communications at the Goldwater Institute, a major advocate for right-to-try efforts, identified Dr. Ebrahim Delpassand, CEO of Excel Diagnostics in Texas, who says he has treated at least 78 patients under the state's law after FDA denied his request to extend a clinical trial for Lutetium-177-DOTA-Octreotate in patients with neuroendocrine tumors. However, information about those patients' outcomes is not readily available. Focus has reached out to Excel Diagnostics for more information and will update this story accordingly.
Coleman also said she's heard of two other patients, one in Florida and one in Oregon, who have been given access to drugs under their respective state laws, but that the Goldwater Institute had not been in contact with them or their physicians.
On the federal level, efforts to pass right-to-try legislation have been less successful.
In September, Sen. Harry Reid (D-NV) blocked a bill introduced by Sen. Ron Johnson (R-WI) that would have created a federal program. Notably, the bill would have prevented FDA from considering outcomes related to drugs given out under the program in any future assessments.