Trump hints that he favors national ‘right to try’ legislation

President Donald J. Trump this week hinted that he may favor a national law giving terminally ill patients the right to try drugs that are still in development.

Thirty-three U.S. states already have so-called “right to try” laws on the books, and the other 17 are considering them, according to the Goldwater Institute, a conservative think tank that wrote the model legislation that is the basis for the state laws.

President Donald J. Trump
Donald J. Trump

Although no such law exists at the federal level, the FDA says it has granted thousands of requests from terminally ill patients asking to try investigational drugs and devices under its expanded access program, and it was unclear how a national “right to try” law would be different.

Steven Joffe, MD, MPH, vice chair of medical ethics at the University of Pennsylvania, told Infectious Disease News that “right to try” laws at the state and federal level are “superfluous,” given the FDA’s track record of approving nearly 100% of the requests made by terminally ill patients to try experimental treatments.

“If you’re looking for a law that makes the FDA say yes rather than no, well, the FDA never says no, so what function does a ‘right to try’ law have?” Joffe said during an interview.

The White House said Trump touched on a number of issues in a closed-door meeting Tuesday with representatives of the pharmaceutical industry, including drug pricing and deregulation. In public remarks, Trump addressed how the FDA handles requests from terminally ill patients.

“One thing that’s always disturbed me [is] they come up with a new drug for a patient who’s terminal and the FDA says, we can’t have this drug used on a patient,” Trump said. “But the patient within 4 weeks will be dead. They say, well, we still can’t approve the drug. Now we don’t know if the drug works or it doesn’t work, but we can’t approve the drug because we don’t want to hurt the patient, but the patient is not going to live more than 4 weeks. So, we’re going to be changing a lot of the rules.”

Starlee Coleman, spokeswoman for the Goldwater Institute, said the organization believes Trump was stating his support for national “right to try” legislation. Coleman said Vice President Mike Pence, an early supporter of “right to try” legislation, pledged during the presidential campaign to make the issue a priority. Pence, who also attended Tuesday’s meeting with pharmaceutical representatives, signed a “right to try” bill in 2015 when he was governor of Indiana.

“We believe he is keeping his campaign promise and the administration is going to make this happen for terminal patients across the country,” Coleman told Infectious Disease News.

Republicans have introduced similar legislation in the U.S. House and Senate aimed at protecting the state laws from federal interference.

Under the FDA’s expanded access program — also called “compassionate use” — physicians, pharmaceutical companies and the FDA must cooperate to get experimental treatments into the hands of patients. But the FDA cannot make companies approve requests from physicians for access to experimental treatments for their patients. In fact, according to the FDA, companies appear to reject those claims more often than the agency turns down expanded use requests.

If a request is granted by a company, the physician must then apply to the FDA for permission. Since the FDA revamped the expanded access program in 2009, it says it has denied only 39 of the 7,291 requests made by physicians. In comparison, just one company reportedly rejected hundreds of applications for experimental treatments filed by physicians over a 2-year period, according to Peter Lurie, MD, MPH, associate commissioner for public health strategy and analysis at the FDA.

During his testimony last September in front of the U.S. Senate Committee on Homeland Security and Governmental Affairs, Lurie underscored the FDA’s preference that investigational products be used by patients as part of a clinical trial.

“Nonetheless,” he said, “FDA recognizes that there are circumstances when patients with serious or life-threatening conditions and no comparable or satisfactory alternative therapy are not eligible for a clinical trial, either because of where they live, their age, or some other disqualifying factor. These patients may consider seeking access to investigational drugs, and FDA’s expanded access program is intended to serve them.”

An FDA spokeswoman contacted by Infectious Disease News would not say if the agency had heard Trump’s comments or if it believed he was advocating for a national “right to try” law. Trump has not yet appointed a new FDA commissioner to replace Robert M. Califf, MD, who stepped down when the new president was inaugurated, per tradition.

The FDA said it streamlined its expanded use application process in 2015 to make it quicker and easier to complete, including simplifying the form and reducing the number of attachments from seven to one.

The Goldwater Institute says state-level “right to try” laws are designed to make it easier for patients who want access to drugs that have passed initial safety tests in phase 1 trials.

But writing in The New England Journal of Medicine in 2015, Aaron S. Kesselheim, MD, JD, MPH, associate professor of medicine in the Center for Bioethics at Harvard Medical School, and colleagues said these laws will have “limited effect” because they do not compel pharmaceutical and insurance companies to supply and pay for experimental treatments.

“In addition, [the laws] are unlikely to withstand a constitutionality challenge that is based on conflict with the FDA's enabling legislation and existing expanded-access regulations,” Kesselheim and colleagues wrote. “Under the Supreme Court's long-standing preemption doctrine, state laws that conflict with federal statutes or regulations are ‘without effect.’ Limiting the reach of these state laws to patients with terminal illnesses cannot avoid the conflict.”

Under the FDA program, pharmaceutical companies are required to seek written authorization to charge patients for an experimental treatment and can recoup only the direct costs of making the treatment available.

Joffe said laws giving patients access to experimental drugs are ethical, but legislators must be careful not to undermine the clinical trial process.

“At the end of the day what we really want, what patients really want and what the public really wants is evidence that our drugs are safe and effective,” he said. “If we undermine the clinical trials that get us that evidence, then we are shooting ourselves in the foot.” – by Gerard Gallagher

References:

Darrow JJ, et al. N Engl J Med. 2015;doi:10.1056/NEJMhle1409465.

FDA. Expanded access (compassionate use). 2017. http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm. Accessed February 2, 2017.

Goldwater Institute. Right to try model legislation. 2016. https://goldwater-media.s3.amazonaws.com/cms_page_media/2016/1/5/GoldwaterInstituteRighttoTryModel.pdf. Accessed February 2, 2017.

Lurie P, et al. N Engl J Med. 2015;doi:10.1056/NEJMc1501823.

Disclosures: Coleman is a spokeswoman for the Goldwater Institute. Joffe reports no relevant financial disclosures.

President Donald J. Trump this week hinted that he may favor a national law giving terminally ill patients the right to try drugs that are still in development.

Thirty-three U.S. states already have so-called “right to try” laws on the books, and the other 17 are considering them, according to the Goldwater Institute, a conservative think tank that wrote the model legislation that is the basis for the state laws.

President Donald J. Trump
Donald J. Trump

Although no such law exists at the federal level, the FDA says it has granted thousands of requests from terminally ill patients asking to try investigational drugs and devices under its expanded access program, and it was unclear how a national “right to try” law would be different.

Steven Joffe, MD, MPH, vice chair of medical ethics at the University of Pennsylvania, told Infectious Disease News that “right to try” laws at the state and federal level are “superfluous,” given the FDA’s track record of approving nearly 100% of the requests made by terminally ill patients to try experimental treatments.

“If you’re looking for a law that makes the FDA say yes rather than no, well, the FDA never says no, so what function does a ‘right to try’ law have?” Joffe said during an interview.

The White House said Trump touched on a number of issues in a closed-door meeting Tuesday with representatives of the pharmaceutical industry, including drug pricing and deregulation. In public remarks, Trump addressed how the FDA handles requests from terminally ill patients.

“One thing that’s always disturbed me [is] they come up with a new drug for a patient who’s terminal and the FDA says, we can’t have this drug used on a patient,” Trump said. “But the patient within 4 weeks will be dead. They say, well, we still can’t approve the drug. Now we don’t know if the drug works or it doesn’t work, but we can’t approve the drug because we don’t want to hurt the patient, but the patient is not going to live more than 4 weeks. So, we’re going to be changing a lot of the rules.”

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Starlee Coleman, spokeswoman for the Goldwater Institute, said the organization believes Trump was stating his support for national “right to try” legislation. Coleman said Vice President Mike Pence, an early supporter of “right to try” legislation, pledged during the presidential campaign to make the issue a priority. Pence, who also attended Tuesday’s meeting with pharmaceutical representatives, signed a “right to try” bill in 2015 when he was governor of Indiana.

“We believe he is keeping his campaign promise and the administration is going to make this happen for terminal patients across the country,” Coleman told Infectious Disease News.

Republicans have introduced similar legislation in the U.S. House and Senate aimed at protecting the state laws from federal interference.

Under the FDA’s expanded access program — also called “compassionate use” — physicians, pharmaceutical companies and the FDA must cooperate to get experimental treatments into the hands of patients. But the FDA cannot make companies approve requests from physicians for access to experimental treatments for their patients. In fact, according to the FDA, companies appear to reject those claims more often than the agency turns down expanded use requests.

If a request is granted by a company, the physician must then apply to the FDA for permission. Since the FDA revamped the expanded access program in 2009, it says it has denied only 39 of the 7,291 requests made by physicians. In comparison, just one company reportedly rejected hundreds of applications for experimental treatments filed by physicians over a 2-year period, according to Peter Lurie, MD, MPH, associate commissioner for public health strategy and analysis at the FDA.

During his testimony last September in front of the U.S. Senate Committee on Homeland Security and Governmental Affairs, Lurie underscored the FDA’s preference that investigational products be used by patients as part of a clinical trial.

“Nonetheless,” he said, “FDA recognizes that there are circumstances when patients with serious or life-threatening conditions and no comparable or satisfactory alternative therapy are not eligible for a clinical trial, either because of where they live, their age, or some other disqualifying factor. These patients may consider seeking access to investigational drugs, and FDA’s expanded access program is intended to serve them.”

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An FDA spokeswoman contacted by Infectious Disease News would not say if the agency had heard Trump’s comments or if it believed he was advocating for a national “right to try” law. Trump has not yet appointed a new FDA commissioner to replace Robert M. Califf, MD, who stepped down when the new president was inaugurated, per tradition.

The FDA said it streamlined its expanded use application process in 2015 to make it quicker and easier to complete, including simplifying the form and reducing the number of attachments from seven to one.

The Goldwater Institute says state-level “right to try” laws are designed to make it easier for patients who want access to drugs that have passed initial safety tests in phase 1 trials.

But writing in The New England Journal of Medicine in 2015, Aaron S. Kesselheim, MD, JD, MPH, associate professor of medicine in the Center for Bioethics at Harvard Medical School, and colleagues said these laws will have “limited effect” because they do not compel pharmaceutical and insurance companies to supply and pay for experimental treatments.

“In addition, [the laws] are unlikely to withstand a constitutionality challenge that is based on conflict with the FDA's enabling legislation and existing expanded-access regulations,” Kesselheim and colleagues wrote. “Under the Supreme Court's long-standing preemption doctrine, state laws that conflict with federal statutes or regulations are ‘without effect.’ Limiting the reach of these state laws to patients with terminal illnesses cannot avoid the conflict.”

Under the FDA program, pharmaceutical companies are required to seek written authorization to charge patients for an experimental treatment and can recoup only the direct costs of making the treatment available.

Joffe said laws giving patients access to experimental drugs are ethical, but legislators must be careful not to undermine the clinical trial process.

“At the end of the day what we really want, what patients really want and what the public really wants is evidence that our drugs are safe and effective,” he said. “If we undermine the clinical trials that get us that evidence, then we are shooting ourselves in the foot.” – by Gerard Gallagher

References:

Darrow JJ, et al. N Engl J Med. 2015;doi:10.1056/NEJMhle1409465.

FDA. Expanded access (compassionate use). 2017. http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm. Accessed February 2, 2017.

Goldwater Institute. Right to try model legislation. 2016. https://goldwater-media.s3.amazonaws.com/cms_page_media/2016/1/5/GoldwaterInstituteRighttoTryModel.pdf. Accessed February 2, 2017.

Lurie P, et al. N Engl J Med. 2015;doi:10.1056/NEJMc1501823.

Disclosures: Coleman is a spokeswoman for the Goldwater Institute. Joffe reports no relevant financial disclosures.

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