Terminally ill patients would be put at risk by changes to a House bill giving them access to experimental drugs, bioethicists and patients groups told Bloomberg Law.

The bill, set to be voted on in the House March 13, narrowed the Senate version’s definition of who could seek unapproved drugs. It also expanded the Senate version’s liability protections for drug companies and hospitals. Both bills give access to drugs that are in clinical trials and haven’t yet been approved by the Food and Drug Administration.

The Senate passed a version of the “right to try” legislation more than six months ago ( S. 204), but FDA Commissioner Scott Gottlieb and Republicans leading the House’s main health committees had concerns it was too broadly written. The latest version appears to have appeased Gottlieb and House leaders, but not its critics in the research community.

Gottlieb and House leaders were concerned the bill would permit people who have chronic illnesses like diabetes that aren’t immediately life-threatening to turn to experimental drugs, potentially putting their lives at risk. Under the House bill, only patients who have a disease or condition where they will likely die within a matter of months, or a disease or condition that would likely lead to severely premature death, would have access to experimental treatments.

The bill has major support in the White House, with President Donald Trump calling for its passage in his State of the Union address and Vice President Mike Pence personally lobbying Congress for its passage. So far, 38 states have enacted their own right-to-try laws, and the governor of Wisconsin is slated to sign one into law soon.

But bioethicists and patients groups told Bloomberg Law they are still concerned the bill cuts the FDA out of any role in making decisions about access to experimental drugs, which could put patients at risk.

Cutting the FDA out of the process “is bad for patients because they won’t benefit from the FDA’s knowledge,” Steve Joffe, a pediatric oncologist and bioethicist at the University of Pennsylvania, told Bloomberg Law in a March 12 email. “In addition, it’s a terrible precedent because it establishes the principle that drugs don’t need to establish safety and efficacy before being administered to patients.”

The bioethicists and patient groups also said the bill would shield drug companies from liability if patients are harmed. But they did praise a provision that would require public reporting of any adverse events experienced by patients.

Senate to Vote Again
If the bill passes in the House, the Senate will have to vote again on the measure or, less likely, form a conference committee to work out the differences, according to a statement from House Majority Leader Rep. Kevin McCarthy (R-Calif.).

The bill’s sponsors, Rep. Andy Biggs (R-Ariz.) and Brian Fitzpatrick (R-Pa.) in the House and Sen. Ron Johnson (R-Wis.), backed the changes and called for the bill to be passed quickly.

Democrats, however, have been reluctant to support right-to-try measures. Rep. Frank Pallone Jr. (D-N.J.), the lead Democrat on the House Energy and Commerce Committee, has publicly opposed the bill out of concern it’ll cause more harm than good to the terminally ill.

A spokesman for Pallone’s office told Bloomberg Law March 12 the Democrat’s opinion of the bill hasn’t changed.

Safety Concerns
The FDA offers people with terminal or serious illnesses ways to access drugs in the early stages of approval through clinical trials and a program called expanded access.

The agency approves most expanded access requests—in 2016, the agency approved 99.4 percent of the requests it received (1,545 out of 1,554). But even if the FDA approves a request, a drug company can still deny a request due to supply or safety concerns.

Some companies also have their own expanded access programs. New Jersey-based pharmaceutical giant Merck & Co. recently disclosed in the U.S. an expanded access program for its cancer immunotherapy drug Keytruda (pembrolizumab) for patients with metastatic melanoma who have tried standard treatments. Its website lays out the criteria used for participating in expanded access as well as directions for requesting access to experimental drugs.

While the House bill contains narrow circumstances under which a patient can obtain access to experimental drugs, “once the principle is established, critics of FDA regulation will seek to expand it over time,” Joffe said. “The FDA’s oversight role is essential to protect the public from unsafe & ineffective medicines, and right to try puts it at risk.”

Drug companies “are likely to prefer to work with rather than around the FDA,” Joffe said. “So I don’t see the bill markedly increasing companies’ willingness to say yes” to patients requesting access to experimental drugs.

Joffe also said there’s no assurance that physicians who prescribe drugs under this pathway will have the expertise to do so safely. “Thus the administration of drugs outside expert hands risks causing frequent and severe harm to patients,” he said.

Joffe and a group of bioethicists last month got more than 300 signatories to oppose a right-to-try legislation and are reigniting their efforts in a new letter. The new letter, dated March 13, provided to Bloomberg Law, and addressed to House leadership, acknowledges the House bill is an improvement from what they opposed last month. But they said the bill would exclude the FDA from oversight of how unapproved products are used and put patients at risk.

The Pharmaceutical Research and Manufacturers of America (PhRMA) hasn’t taken a formal position on the bill, but Andrew Powaleny, director of public affairs for PhRMA, told Bloomberg Law in an email his group “believes any legislation must benefit patients and not disrupt the future of clinical trials and the research and approval of new medicines.”

Opposition to Bill
Several patient and consumer groups said they still oppose the House bill even though it offers some improvements to the Senate version.

For example, the consumer group Public Citizen “strongly” opposes the House bill, Mike Carome, director of Public Citizen’s Health Research Group, told Bloomberg Law March 12.

“Although the legislation has several improvements over the Senate-passed version, the bill still has fundamental flaws that would put vulnerable patients at risk and undermine their rights,” Carome said. “In particular, the bill would offer false hope to patients by creating a dangerous pathway for access to experimental drugs that bypasses the FDA’s existing expanded access program.”

The FDA’s expanded access program “helps ensure proposed uses of experimental drugs don’t pose unacceptable risks to patients,” Carome said.

Also, Ellen V. Sigal, chairwoman of Friends of Cancer Research, said the House bill still falls short. “We cannot support a bill that removes this agency from the equation, and we cannot support a bill that creates false hope and may harm those who are most vulnerable,” she said in a March 12 statement to Bloomberg Law.

Paul Melmeyer, director of federal policy for the National Organization for Rare Disorders (NORD), said in a statement NORD still opposes the bill and plans on reiterating that opposition in a letter to the House.

“This version, though considerably improved, will still create an unnecessary and less safe alternative pathway for accessing investigational therapies outside of a clinical trial,” Melmeyer said. “This would not benefit our patients, and would only generate confusion and false hope.”

Meanwhile, Starlee Coleman, a spokeswoman for the Goldwater Institute, praised the new bill in a statement, saying it “should answer any questions from doctors and pharmaceutical and biotech companies about exactly who qualifies and how the FDA can use patient data collected and reported from Right to Try patients.” The Goldwater Institute, a conservative think thank, has been pushing for state right-to-try laws.

Reporting Adverse Events

The House bill would require the public reporting of adverse events experienced by patients who take experimental drugs under the right-to-try pathway.

“It’s critical that such events are not kept under wraps,” Joffe said. But he said, the requirement to report adverse events could make companies less likely to agree to provide access to experimental drugs.

“Figuring out whether an adverse event in a sick patient is caused by an experimental drug is exceptionally difficult,” Joffe said. “The FDA has the expertise to do this; reporting adverse events without the agency’s adjudication may paradoxically make the drugs look more toxic than they are.”

Liability Protections
The House bill also “would broadly immunize from liability manufacturers of drugs, sponsors, physicians, clinical investigators, and hospitals for a wide range of conduct related to providing experimental medications to patients under this pathway,” Carome said.

While the Senate bill had a similar provision, the House bill extends the liability immunization provisions to the FDA’s existing expanded access program, Carome said.

“In our view, that would eliminate a strong incentive for manufacturers and health-care providers to act responsibly and in the best interest of patients when providing these experimental medicines,” Carome said.

But Joffe said the liability protections in the bill are most likely meaningless, because there hasn’t been any litigation against companies for the use of experimental drugs and the reasons companies say no to allowing access aren’t related to litigation risks.

https://www.bna.com/opposition-experimental-drug-n57982089743/