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Terminal patients who have exhausted all approved drug options would be able to seek out investigational treatments – even if they do not qualify for clinical trials – under a bill passed in the U.S. House, despite opposition from more than 100 patient and physician groups.
The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina 
For an unapproved drug to be made available to patients, it must have an active application that is not subject to any kind of clinical hold. Sponsors and manufacturers must notify the Food and Drug Administration when an unapproved drug is made available to the patient.
The bill also includes safeguards to prevent manufacturers from purposefully misbranding or mislabeling drugs.
H.R. 5247 provides liability protections to manufacturers, sponsors, physicians, clinical investigators, and hospitals that participate in providing experimental drugs to terminal patients through this new alternative pathway, although it does not shield them from liability stemming from reckless misconduct, gross negligence, or any other intentional violations. It requires sponsors and manufacturers to report all adverse events to the FDA.
It also provides certainty to manufacturers as to how the FDA will use patient outcomes from the use of treatments outside of clinical trials when it is evaluating the applications on these new drugs.
Rep. Michael Burgess (R-Tex.), the House Energy & Commerce Health Subcommittee chairman and a physician, spoke in support of the bill during a debate on the House floor.
“Mr. Speaker, as a physician, I understand that access to investigational drugs and therapies is a deeply personal priority for those seeking treatment for their loved ones with serious, life-threatening conditions,” he said. “To my friends on the other side of the aisle, I have a simple question: Why do you not want to allow these patients to exercise their right to fight for their future?”
Rep. Frank Pallone (D-N.J.), the top-ranking Democrat on the House Energy & Commerce Committee, responded by asking, “if this is such a patient-centered bill, then why does every major patient organization overwhelmingly oppose it?”
In a March 19 letter to congressional leaders, a coalition of more than 100 physician and patient advocacy groups called the alternative pathway laid out in legislation “less safe” for patients than the FDA’s current expanded access process.
“This alternative pathway would allow for a 7-day lag between access to investigational therapies (as well as potential ensuing adverse effects) and FDA notification. FDA also is prohibited from halting access to these experimental therapies short of placing a clinical hold on all clinical research on the therapy in question, which is a blunt and disproportionate measure. The legislation would also remove FDA’s consultation on dosing, route of administration, dosing schedule, and other important safety measures available under FDA’s current expanded access program,” the groups wrote.
The groups that signed the letter included the American Society of Clinical Oncology, the Cystic Fibrosis Foundation, Friends of Cancer Research, the Leukemia & Lymphoma Society, the National Comprehensive Cancer Network, the National Organization for Rare Disorders, the Platelet Disorder Support Association, and Vietnam Veterans of America.
“The current compassionate use program at the Food and Drug Administration does make a good faith effort to help patients who do not qualify for clinical trials,” Rep. Burgess said. “But ‘right to try’ would actually offer patients an alternative pathway to access eligible investigational drugs, so long as they are certified by a physician who is in good standing and abides by the rules laid out in the bill.”
But Rep. Pallone noted that a review by the Government Accountability Office found that the FDA approves 99% of the requests submitted to the agency. Of the nearly 1,700 requests the FDA received in 2017, just 9 were not approved. However, the agency also adjusted applications for 11% of the patients in order to improve patient safety protections and that type of review should be allowed to continue, he said.
Patient groups expressed disappointment following the House vote. “The House has voted for a proposal that would create a less-safe, redundant pathway for accessing investigational therapies outside of clinical trials,” the National Organization for Rare Disorders said in a statement. “We hope the Senate will recognize that patients deserve legislation that will genuinely increase access. For example, senators should focus on legislation that reduces the financial disincentives companies encounter in offering their therapy through expanded access.”
The Senate passed a version of “right to try” in 2017 through the unanimous consent process (S. 204). No schedule has been set yet to either combine the two bills in committee or for the Senate to take up the House bill. President Trump voiced support for “right to try” legislation during his 2018 State of the Union address.
Terminal patients who have exhausted all approved drug options would be able to seek out investigational treatments – even if they do not qualify for clinical trials – under a bill passed in the U.S. House, despite opposition from more than 100 patient and physician groups.
The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina
For an unapproved drug to be made available to patients, it must have an active application that is not subject to any kind of clinical hold. Sponsors and manufacturers must notify the Food and Drug Administration when an unapproved drug is made available to the patient.
The bill also includes safeguards to prevent manufacturers from purposefully misbranding or mislabeling drugs.
H.R. 5247 provides liability protections to manufacturers, sponsors, physicians, clinical investigators, and hospitals that participate in providing experimental drugs to terminal patients through this new alternative pathway, although it does not shield them from liability stemming from reckless misconduct, gross negligence, or any other intentional violations. It requires sponsors and manufacturers to report all adverse events to the FDA.
It also provides certainty to manufacturers as to how the FDA will use patient outcomes from the use of treatments outside of clinical trials when it is evaluating the applications on these new drugs.
Rep. Michael Burgess (R-Tex.), the House Energy & Commerce Health Subcommittee chairman and a physician, spoke in support of the bill during a debate on the House floor.
“Mr. Speaker, as a physician, I understand that access to investigational drugs and therapies is a deeply personal priority for those seeking treatment for their loved ones with serious, life-threatening conditions,” he said. “To my friends on the other side of the aisle, I have a simple question: Why do you not want to allow these patients to exercise their right to fight for their future?”
Rep. Frank Pallone (D-N.J.), the top-ranking Democrat on the House Energy & Commerce Committee, responded by asking, “if this is such a patient-centered bill, then why does every major patient organization overwhelmingly oppose it?”
In a March 19 letter to congressional leaders, a coalition of more than 100 physician and patient advocacy groups called the alternative pathway laid out in legislation “less safe” for patients than the FDA’s current expanded access process.
“This alternative pathway would allow for a 7-day lag between access to investigational therapies (as well as potential ensuing adverse effects) and FDA notification. FDA also is prohibited from halting access to these experimental therapies short of placing a clinical hold on all clinical research on the therapy in question, which is a blunt and disproportionate measure. The legislation would also remove FDA’s consultation on dosing, route of administration, dosing schedule, and other important safety measures available under FDA’s current expanded access program,” the groups wrote.
The groups that signed the letter included the American Society of Clinical Oncology, the Cystic Fibrosis Foundation, Friends of Cancer Research, the Leukemia & Lymphoma Society, the National Comprehensive Cancer Network, the National Organization for Rare Disorders, the Platelet Disorder Support Association, and Vietnam Veterans of America.
“The current compassionate use program at the Food and Drug Administration does make a good faith effort to help patients who do not qualify for clinical trials,” Rep. Burgess said. “But ‘right to try’ would actually offer patients an alternative pathway to access eligible investigational drugs, so long as they are certified by a physician who is in good standing and abides by the rules laid out in the bill.”
But Rep. Pallone noted that a review by the Government Accountability Office found that the FDA approves 99% of the requests submitted to the agency. Of the nearly 1,700 requests the FDA received in 2017, just 9 were not approved. However, the agency also adjusted applications for 11% of the patients in order to improve patient safety protections and that type of review should be allowed to continue, he said.
Patient groups expressed disappointment following the House vote. “The House has voted for a proposal that would create a less-safe, redundant pathway for accessing investigational therapies outside of clinical trials,” the National Organization for Rare Disorders said in a statement. “We hope the Senate will recognize that patients deserve legislation that will genuinely increase access. For example, senators should focus on legislation that reduces the financial disincentives companies encounter in offering their therapy through expanded access.”
The Senate passed a version of “right to try” in 2017 through the unanimous consent process (S. 204). No schedule has been set yet to either combine the two bills in committee or for the Senate to take up the House bill. President Trump voiced support for “right to try” legislation during his 2018 State of the Union address.
Terminal patients who have exhausted all approved drug options would be able to seek out investigational treatments – even if they do not qualify for clinical trials – under a bill passed in the U.S. House, despite opposition from more than 100 patient and physician groups.
The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina
For an unapproved drug to be made available to patients, it must have an active application that is not subject to any kind of clinical hold. Sponsors and manufacturers must notify the Food and Drug Administration when an unapproved drug is made available to the patient.
The bill also includes safeguards to prevent manufacturers from purposefully misbranding or mislabeling drugs.
H.R. 5247 provides liability protections to manufacturers, sponsors, physicians, clinical investigators, and hospitals that participate in providing experimental drugs to terminal patients through this new alternative pathway, although it does not shield them from liability stemming from reckless misconduct, gross negligence, or any other intentional violations. It requires sponsors and manufacturers to report all adverse events to the FDA.
It also provides certainty to manufacturers as to how the FDA will use patient outcomes from the use of treatments outside of clinical trials when it is evaluating the applications on these new drugs.
Rep. Michael Burgess (R-Tex.), the House Energy & Commerce Health Subcommittee chairman and a physician, spoke in support of the bill during a debate on the House floor.
“Mr. Speaker, as a physician, I understand that access to investigational drugs and therapies is a deeply personal priority for those seeking treatment for their loved ones with serious, life-threatening conditions,” he said. “To my friends on the other side of the aisle, I have a simple question: Why do you not want to allow these patients to exercise their right to fight for their future?”
Rep. Frank Pallone (D-N.J.), the top-ranking Democrat on the House Energy & Commerce Committee, responded by asking, “if this is such a patient-centered bill, then why does every major patient organization overwhelmingly oppose it?”
In a March 19 letter to congressional leaders, a coalition of more than 100 physician and patient advocacy groups called the alternative pathway laid out in legislation “less safe” for patients than the FDA’s current expanded access process.
“This alternative pathway would allow for a 7-day lag between access to investigational therapies (as well as potential ensuing adverse effects) and FDA notification. FDA also is prohibited from halting access to these experimental therapies short of placing a clinical hold on all clinical research on the therapy in question, which is a blunt and disproportionate measure. The legislation would also remove FDA’s consultation on dosing, route of administration, dosing schedule, and other important safety measures available under FDA’s current expanded access program,” the groups wrote.
The groups that signed the letter included the American Society of Clinical Oncology, the Cystic Fibrosis Foundation, Friends of Cancer Research, the Leukemia & Lymphoma Society, the National Comprehensive Cancer Network, the National Organization for Rare Disorders, the Platelet Disorder Support Association, and Vietnam Veterans of America.
“The current compassionate use program at the Food and Drug Administration does make a good faith effort to help patients who do not qualify for clinical trials,” Rep. Burgess said. “But ‘right to try’ would actually offer patients an alternative pathway to access eligible investigational drugs, so long as they are certified by a physician who is in good standing and abides by the rules laid out in the bill.”
But Rep. Pallone noted that a review by the Government Accountability Office found that the FDA approves 99% of the requests submitted to the agency. Of the nearly 1,700 requests the FDA received in 2017, just 9 were not approved. However, the agency also adjusted applications for 11% of the patients in order to improve patient safety protections and that type of review should be allowed to continue, he said.
Patient groups expressed disappointment following the House vote. “The House has voted for a proposal that would create a less-safe, redundant pathway for accessing investigational therapies outside of clinical trials,” the National Organization for Rare Disorders said in a statement. “We hope the Senate will recognize that patients deserve legislation that will genuinely increase access. For example, senators should focus on legislation that reduces the financial disincentives companies encounter in offering their therapy through expanded access.”
The Senate passed a version of “right to try” in 2017 through the unanimous consent process (S. 204). No schedule has been set yet to either combine the two bills in committee or for the Senate to take up the House bill. President Trump voiced support for “right to try” legislation during his 2018 State of the Union address.