https://www.ajmc.com/newsroom/right-to-try-legislation-headed-for-a-vote-as-doubts-about-its-necessity-remain
"Right-to-Try" Legislation Defeated in House Amid Doubts About Its Necessity

Allison Inserro

A revamped “right-to-try” bill was defeated in the House of Representatives 259-140 Tuesday night, which advocates had said would give desperate, dying patients a last chance at experimental treatments. The legislation, released over the weekend after backers made some changes since the the original bill was introduced last fall, failed to convince enough Democrats.

The House Energy and Commerce Committee chairman, Greg Walden, R-Oregon, and chair of the health subcommittee, Michael C. Burgess, MD, R-Texas, had endorsed the updated version of the legislation, saying that it incorporates concerns raised by FDA Commissioner Scott Gottlieb, MD, and others.

The bill was pushed by Vice President Mike Pence, who signed a similar bill when he was governor of Indiana, and touted by President Trump in his State of the Union address. Although the defeat of the bill was a surprise, Politico reported Wednesday that GOP leaders intended to keep trying.

After the bill's defeat, Frank Pallone, D-New Jersey, released a statement that read, "By defeating this bill tonight, we protected patients and supported FDA’s continued role in approving experimental treatments that may help save a patient’s life.  This bill should have never been on the House Floor in the first place since it was only introduced today and has never been reviewed or discussed by our Committee.  With tonight’s result, I’d hope Chairman Walden would let the Committee continue its work to develop legislation that protects patients from bad actors while also protecting FDA’s critical role in the review process.” 

The bill failed to get the necessary two-thirds support; the House had voted for the measure under suspension of the rules, which Pallone criticized.

Walden and Burgess said in a statement they were disappointed in fellow Democrats. “For months we sought to strike the right balance by allowing patients greater access to these unapproved treatments and therapies while also ensuring proper patient protections. This bill does just that, with more robust informed consent and real-time reporting, as well as requiring FDA notification of participation." 

Earlier Tuesday, The American Journal of Managed Care® (AJMC®) spoke with Marjorie A. Speers, PhD, executive director of the WCG Foundation, a public charity that works to ensure experimental medicines to very ill patients under the FDA's current expanded acccess and compassionate use programs. Speers has previously participated on a panel on this subject at AJMC®'s Patient-Centered Oncology Care® meeting in Philadelphia, Pennsylvania, in 2017. 

“My overall reaction to the bill is that it doesn’t add much to the process that we already have in place,” said Speers, referring to the FDA’s expanded access program.

In more than 99% of cases where a pharmaceutical, biotech, or device company seeks an expedited approval for a patient, the agency usually responds within days, or even 24 hours, if the case is deemed an emergency, she said. An institutional review board (IRB) reviews patient safety and informed consent. IRB review and informed consent requirements stem from FDA regulations, Speers noted

From her perspective, everyone involved works very hard on behalf of patients to ensure their safety and welfare. “This version of the bill, from what I see it does, is it removes the FDA from the process. But it keeps a lot of the other types of safety protections in place.”

If that’s the case, she questioned, “why do we need this bill? It reflects a lot of the good process that we already have in place.”

The latest version of the bill does narrow eligibility to patients who are at a stage of disease or condition where “there is reasonable liklihood that death will occur within a matter of months,” or that will result in “significant irreversible morbidity that is likely to lead to premature death.”

Pharmaceutical companies—which are not required to make their treatments available under this law—are required to tell the secretary of HHS about adverse events, not the FDA commissioner.  

One of her concerns has to do with the fact that for an investigational drug to get cleared through this new pathway, the only hurdle is that it must complete a phase 1 trial. A phase 1 trial only determines the highest dose level at which a drug appears to be safe but it tells you nothing about efficacy, Speers said.

She questioned how anyone can make a decision to treat desperately ill patients based on such information.  

The bill requires adverse event reporting, yet at the same time the clinical outcomes associated with the use of that reporting cannot be used by the HHS secretary to delay or adversely effect the approval of the drug, unless the HHS secretary decides it is necessary for safety reasons or the company wants it reported.

The manufacturer that provides the investigational drug shall post on a website information such as the number of requests received, the number of patients treated, and more.

Speers said the House “is trying to make investigational medicine more readily available, but at the same time they are trying to have some control or restrictions on it so that patients are not harmed,” based on the concerns they heard last October. “I think they really heard that message,”

At the same time, Speers noted, “This bill removes FDA but it seems to me…by trying to keep some of the restrictions, limitations on these uses, it’s an implicit acknowledgement that the current FDA’s expanded access program works.”

In states that do have right-to-try laws, they are not being used much for several reasons, she said. “Patients, physicians, and product manufacturers, they don’t want to go outside of the clinical trials process. They don’t want to go outside the regulations that seem to be working."

“This bill really doesn’t fix a problem, because the problem it’s intended to fix doesn’t exist,” Speers said, noting that the FDA streamlined the application for expedited access in 2016 and overall has been more responsive.

Another value of the FDA reviewing this process, she said, is that when an application is filed, an IND number is given and a letter sent to the physician. Sometimes, she said, that FDA letter will include suggestions, perhaps having to do with monitoring, doses, or administration. It is information helpful to the physician, the IRB, and also the patient, thus improving patient care. None of that is included in the House bill being considered today, she said.

“This formal review process can be invaluable to patients,” she said.

Patient groups remained unconvinced about the bill as well—75 of them sent a letter Monday to House Speaker Paul Ryan, R-Wisconsin, and Minority Leader Nancy Pelosi, D-California, saying the changes do not go far enough.
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