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US FDA commissioner tells House hearing that the Senate's legislation in its current form could undermine the intent of addressing terminally ill patients.



Capitol House 



The Senate's right-to-try legislation should be narrowed to specify patients who are "terminally ill" as a criterion for experimental drug eligibility from its current language of "a life-threatening disease or condition," US FDA Commissioner Scott Gottlieb urges lawmakers.



Testifying before the House Energy and Commerce Subcommittee on Health Oct. 3, Gottlieb cautioned that Sen. Ron Johnson's (R-Wisc.) right-to-try legislation, known as the Trickett Wendler Right-to-Try Act (S.204), would encompass too broad a patient population. The bill in its current form could apply to patients with chronic illnesses such as diabetes or other diseases that don’t immediately set a patient on a terminal course, but that are still life-threatening, the commissioner said.



"If you look across the state laws in states that have passed right-to-try laws, the language typically speaks about a patient being terminally ill to qualify for consideration under the right-to-try provisions," Gottlieb said. "Congress, in consideration of some of this legislation … has broadened that to include diseases that are either terminal or life threatening.



"The component of a life-threatening disease is a broader definition. … There are a lot of illnesses that are certainly life-threatening, but not immediately terminal."


In his prepared remarks, Gottlieb recommended defining a terminal illness as "a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months."



Drugmakers could be incentivized to produce more product in the pre-approval setting through a change in clinical trial design, Gottlieb said, but he stressed that the issue is not addressed in the bill.



Johnson's right-to-try law has the goal of increasing access to investigational drugs in accordance with state right-to-try laws. The Senate passed the bill by unanimous consent the same day it voted to pass the FDA Reauthorization Act (FDARA). (Also see "Not Quite FDARA Add-Ons: Right-To-Try, Patient Experience, Opioid Measures Also Clear US Senate" - Pink Sheet, 3 Aug, 2017.)



The Senate, however, may not be welcome to any changes the House makes to the bill. Rep. Greg Walden (R-Ore.), who chairs the full Energy and Commerce Committee, explained that at least one Senate sponsor of the bill told him that the upper chamber is not looking for any changes "out of fear it may fail if it goes back with changes."



Gottlieb explained that the broad "life-threatening" diseases definition could force FDA to interpret the bill "expansively," and that it could "sweep in a whole range of conditions for which it didn't intend."



"The more we broaden this provision, and the more we potentially sweep in conditions for which we might be exposing people to unwanted side effects from experimental therapies, the more we risk undermining the whole venture that we are trying to engage in here, which is to narrowly tailor something to people who really don’t have good options from available therapy," Gottlieb said.



In addition to the Senate legislation, Rep. Andy Biggs, R-Ariz, is sponsoring the Right to Try Act of 2017 (H.R.878). Bigg's bill uses the phrase "terminal illness" instead of "a life-threatening disease or condition." The bill defines terminal illness in accordance with state laws.



The House bill has 43 co-sponsors, which includes four Democrats. Other Democrats, however, such as subcommittee Ranking Member Gene Green (D-Texas), have raised concerns about right-to-try legislative efforts taking FDA out of the equation.



The fate of legislative efforts remains unclear. Subcommittee Chairman Rep. Michael Burgess (R-Texas) closed out the hearing noting that the conversations "set the stage for perhaps our second hearing in this regard," adding that "clearly, this is not the end of the story."



Availability, Safety Also Issues


The commissioner maintained FDA's usual stances on the issue of expanded access, noting that the agency approves 99% of requests, and that it can approve emergency requests over the phone within 24 hours.



For the requests that were denied, Gottlieb explained that roughly half have been due to the experimental drug not being available.



"The biggest reason is that when companies do clinical trials, they don't have continuous manufacturing," Gottlieb said. "They don't have large facilities online pumping out endless supplies of drugs. They will do what we call 'discontinuous batches.' They will do runs just to create batches of drug supply and active pharmaceutical ingredient sufficient for the clinical trial."



Gottlieb said that drugmakers could be incentivized to produce more product in the pre-approval setting through a change in clinical trial design, but stressed that the issue is not addressed in the bill.



In other cases, Gottlieb said, the denial comes as a result of a clinical hold, which the public doesn't know about since the hold is confidential information.


Not A Perfect System, But A Very Good One


Gottlieb noted at the hearing that FDA's expanded access system over the years has not been perfect, it has been remained effective.



In an Oct. 3 FDA Voice blog post, the commissioner touted several measures that the agency has taken to remove hurdles that delay or discourage expanded access applications. He announced that physicians now only need approval from one Institutional Review Board (IRB) member – either the chair or another "appropriate" member – at their facility to treat a patient under expanded access. Previously, physicians were required to obtain approval from the full board.



IRB requirements were clarified in both the "Form FDA 3926" guidance, as well as the "Waiver of IRB Requirements for Drug and Biological Product Studies" guidance.



"This is an important step to protect the rights, safety and well-being of human subjects in clinical research – but assembling the full board may cause delays because they may not routinely meet," Gottlieb writes.



"I believe the simplified IRB process will facilitate access while still protecting patients."



He also announced in the blog post that FDA has its June 2016 Q&A guidance with clarifications about how sponsors should adverse event data for expanded access investigational new drug applications (INDs). The updated guidance specifies that sponsors are only required to report adverse events "if there is evidence to suggest a causal relationship between the drug and the adverse event."



Sponsors have often been hesitant to provide drugs under the expanded access program with the uncertainty about how FDA will view the product's adverse event data in the review process.



Gottlieb worked to assuage these fears at the hearing, however, pointing to a Government Accountability Office (GAO) report that found that have only been two instances where adverse events from expanded access use contributed to a decision to put development of an investigational drug on a partial clinical hold. (Also see "Expanded Access: FDA To Clarify How Adverse Events Impact Drug Approval Process" - Pink Sheet, 20 Jul, 2017.)



The commissioner added in his blog post that more "simplifications and clarifications" regarding the expanded access program are on the way.



Industry Rep Not A Fan


An industry representative at the hearing, Cognition Therapeutics Inc. President and CEO Kenneth Moch, was the harshest critic of the right-to-try legislation at the hearing.



Along with Gottlieb, Moch explained that the legislation cannot force drugmakers to provide their experimental products to patients. Moch added that FDA has never been a hindrance to granting expanded access requests, and that the legislation also does not take into the account the complexities of drug development.



"No ethical company that I know of would ever release an experimental medicine outside of the FDA’s regulatory process. A basic mantra is that 'all drugs have side effects.' And cutting scientific corners creates unbounded risks."



Moch further criticized anecdotal arguments in favor of right-to-try legislation, and stressed that taking FDA out of the equation does nothing to address the reasons as to why drugmakers don't provide access to experimental treatments.



"You have to look at the totality [of data]," Moch said, also referring to the bill as "feel-good legislation."

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