‘Right to try’ impact on patients uncertain

President Donald J. Trump
Donald J. Trump
Scott Gottlieb
Scott Gottlieb

On May 30, President Donald J. Trump signed “right to try” legislation, which allows terminally ill patients to seek investigational drugs that have passed the first phase of FDA approval but have not received final approval, into law.

The bill was previously approved by the House of Representatives on May 22 and the Senate in August 2017.

“At the FDA, we stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients,” Scott Gottlieb, MD, commissioner of the FDA, said in a press release.

The law aims to help patients with life-threatening illnesses or conditions to access promising investigational drugs, Gottlieb said.

“Although significant from a political and legislative standpoint, whether the right to try bill will have a meaningful impact on the lives of terminally ill patients remains to be seen,” Elizabeth Trentacost, from Arnold & Porter, told Healio Internal Medicine. “Right to try provides patients with an alternative to FDA’s expanded access program, under which patients must seek FDA’s approval to be administered an investigational drug or biologic.”

Previous research published in Therapeutic Innovation & Regulatory Science in 2016 indicated that the FDA approved 99% of compassionate use exemptions over a 10-year period between January 2005 and December 2014. Gottlieb said in a statement before addressing the Subcommittee on Health, Committee on Energy and Commerce, US House of Representatives that “emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days.”

On May 22, the House of Representatives voted 250-169 to pass the “right to try” legislation which was previously approved by the Senate in August 2017.
Photo credit: Shutterstock

 

Trentacost reasoned that the manufacturers who choose not to make their experimental drugs available impede patients’ access to investigational therapies rather than the FDA, which approves a vast majority of expanded access requests.

“Under both expanded access and right to try, manufacturers are under no obligation to provide access to an investigational drug or biologic,” she said. “Manufacturers may have good reason to deny permission, including limited supply of investigational treatments.”

Trentacost noted that patients who do access treatments under right to try may still be financially liable for some costs since manufacturers can recover some direct costs for providing the treatment under the bill.

“Although removal of FDA’s involvement in accessing investigational treatments is a victory to some, it may come at a cost of FDA’s insight into confidential therapy development information which can be used, for example, to modify treatment regimens to make a course of an investigational drug safer,” she said. “FDA’s commissioner has noted though, that a pathway remains for administratively incorporating additional patient protections in right to try that is consistent with the new law.”

More than 100 advocacy groups, including the American Lung Association and American Society of Clinical Oncology, sent a letter to Congress strongly opposing the law. They favor a revised version of right to try legislation from Greg Walden (R -Ore.) that included more patient protections and included other clarifications. That measure passed the House in March, but the Senate declined to bring it to a vote. This week’s vote in the House was on the original Senate bill.

“The Senate version of the legislation is less safe than the pathway proposed in the House version and is dangerous compared to the current expanded access process,” they wrote. “The Senate’s bill would allow unproven therapies to be given to patients without FDA notification for up to a full year and would not establish any standards for informed consent.”

The organizations argued that eliminating FDA approval and consultation does not address barriers to access.

“While we did not support the recent House passed version of this legislation, the House legislation includes improved patient safeguards compared to the Senate version,” they wrote. “The Senate version would negatively impact patient safety substantially... We appreciate past efforts in the House to consider stakeholder perspectives and desire to continue the dialogue, but returning to the Senate version is simply not the way forward.” – by Alaina Tedesco

Editor’s note: This story has been updated on May 30 to include the Trumps approval of the legislation.

Disclosure: Healio Internal Medicine was unable to confirm relevant financial disclosures at the time of publication.

President Donald J. Trump
Donald J. Trump
Scott Gottlieb
Scott Gottlieb

On May 30, President Donald J. Trump signed “right to try” legislation, which allows terminally ill patients to seek investigational drugs that have passed the first phase of FDA approval but have not received final approval, into law.

The bill was previously approved by the House of Representatives on May 22 and the Senate in August 2017.

“At the FDA, we stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients,” Scott Gottlieb, MD, commissioner of the FDA, said in a press release.

The law aims to help patients with life-threatening illnesses or conditions to access promising investigational drugs, Gottlieb said.

“Although significant from a political and legislative standpoint, whether the right to try bill will have a meaningful impact on the lives of terminally ill patients remains to be seen,” Elizabeth Trentacost, from Arnold & Porter, told Healio Internal Medicine. “Right to try provides patients with an alternative to FDA’s expanded access program, under which patients must seek FDA’s approval to be administered an investigational drug or biologic.”

Previous research published in Therapeutic Innovation & Regulatory Science in 2016 indicated that the FDA approved 99% of compassionate use exemptions over a 10-year period between January 2005 and December 2014. Gottlieb said in a statement before addressing the Subcommittee on Health, Committee on Energy and Commerce, US House of Representatives that “emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days.”

On May 22, the House of Representatives voted 250-169 to pass the “right to try” legislation which was previously approved by the Senate in August 2017.
Photo credit: Shutterstock

 

Trentacost reasoned that the manufacturers who choose not to make their experimental drugs available impede patients’ access to investigational therapies rather than the FDA, which approves a vast majority of expanded access requests.

“Under both expanded access and right to try, manufacturers are under no obligation to provide access to an investigational drug or biologic,” she said. “Manufacturers may have good reason to deny permission, including limited supply of investigational treatments.”

Trentacost noted that patients who do access treatments under right to try may still be financially liable for some costs since manufacturers can recover some direct costs for providing the treatment under the bill.

“Although removal of FDA’s involvement in accessing investigational treatments is a victory to some, it may come at a cost of FDA’s insight into confidential therapy development information which can be used, for example, to modify treatment regimens to make a course of an investigational drug safer,” she said. “FDA’s commissioner has noted though, that a pathway remains for administratively incorporating additional patient protections in right to try that is consistent with the new law.”

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More than 100 advocacy groups, including the American Lung Association and American Society of Clinical Oncology, sent a letter to Congress strongly opposing the law. They favor a revised version of right to try legislation from Greg Walden (R -Ore.) that included more patient protections and included other clarifications. That measure passed the House in March, but the Senate declined to bring it to a vote. This week’s vote in the House was on the original Senate bill.

“The Senate version of the legislation is less safe than the pathway proposed in the House version and is dangerous compared to the current expanded access process,” they wrote. “The Senate’s bill would allow unproven therapies to be given to patients without FDA notification for up to a full year and would not establish any standards for informed consent.”

The organizations argued that eliminating FDA approval and consultation does not address barriers to access.

“While we did not support the recent House passed version of this legislation, the House legislation includes improved patient safeguards compared to the Senate version,” they wrote. “The Senate version would negatively impact patient safety substantially... We appreciate past efforts in the House to consider stakeholder perspectives and desire to continue the dialogue, but returning to the Senate version is simply not the way forward.” – by Alaina Tedesco

Editor’s note: This story has been updated on May 30 to include the Trumps approval of the legislation.

Disclosure: Healio Internal Medicine was unable to confirm relevant financial disclosures at the time of publication.

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