Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018
Floor Speech
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I rise today in strong opposition to H.R. 5247, or the Right to Try Act of 2018. Supporters of this legislation, Mr. Speaker, have claimed that it will provide seriously ill patients, who have exhausted all of their available treatment options, access to experimental therapies free from the barriers of FDA oversight.
While it is understandable that someone suffering from a disease who has no more options would want to try anything that could help them fight their disease, this legislation delivers the false hope to patients and their families that they will receive a cure to their underlying disease or condition.
In fact, this legislation provides patients and their families nothing more than the right to ask a manufacturer for access to early stage, unproven treatments. Like other so-called right-to-try proposals, H.R. 5247 is based on the false premise that patients are not receiving access to the investigational treatments as a result of the Food and Drug Administration, and this simply not the case.
Through the FDA's existing expanded access program, seriously ill patients are able to request access to investigational products. The FDA approves 99 percent of all requests for investigational drugs or biologics that it receives through this program.
Last year, FDA received more than 1,500 requests, and only 9 were not approved. Despite this high-approval rate, supporters of right-to-try laws have argued that the process is too slow and burdensome, but I have not seen evidence that this is the case, Mr. Speaker. In fact, FDA often grants emergency requests for expanded access immediately over the phone, and nonemergency requests are processed in an average of 4 days.
FDA has even made improvements to streamline the process. For example, FDA has revised the application for physicians to ensure that it now takes less than an hour to complete. FDA has also released additional guidance to industry, outlining the expanded access program's requirements and addressing common questions related to the different programs and submission process, and how outcomes will be considered as part of the review process.
Last fall, FDA Commissioner Gottlieb testified on right-to-try efforts and told our committee that: ``There is a perception that certain products that aren't being offered under FDA expanded access . . . will be offered under right-to-try. I don't see that,'' the commissioner said. As I have said, the review process is working well, but this legislation would completely take FDA out of the review process. This is dangerous and could put patients at serious risk.
FDA is part of the process for a reason. It protects patients from potentially bad actors or from experimental treatments that might do more harm than good. While FDA approves 99 percent of the treatments it reviews, it also revises applications for 11 percent of patients to improve patient safety protections.
In order to protect patients, this review should continue. We must protect patients from bad actors or from dangerous treatments that would make their lives worse. I am extremely concerned that the legislation we are considering today is advancing a solution to address barriers to investigational treatments that do not exist and could expose seriously ill patients to greater harm instead of the greater access that they are looking for.
The true barrier to any expanded access is the determination by the manufacturer as to whether or not they will provide access to their products that are under development. But nothing in the legislation before us today would compel a manufacturer to grant access upon request.
Further, H.R. 5247 would allow patients access to investigational treatments that have only completed a phase I clinical trial. That is an extremely small trial. It does not determine the effectiveness, or the potential side effects of a drug. Access at this stage in the development could expose patients to untested products, further harm, and result in delaying access to a treatment that may be more appropriate and more beneficial for their underlying disease or condition.
H.R. 5247 also erodes important patient safeguards. It limits FDA's ability to use clinical outcomes associated with the use of an investigational product when reviewing a product for approval if it could adversely impact its review. It also prevents any entity from being held liable for use of the treatment.
And while I appreciate, Mr. Speaker, the intent of this bill, I can't support it. The last thing I want to do is give patients false hope and to potentially put them at risk by completely removing FDA from the review and approval process.
Finally, Mr. Speaker, it is outrageous, in my opinion, that a bill of this magnitude is being considered under a suspension of the rule. As my Republican colleagues well know, bills considered under suspension are traditionally bipartisan bills that have worked their way through the appropriate committees with overwhelming bipartisan support.
This bill was never considered by the Energy and Commerce Committee. In fact, it was only introduced today. A bill with such critical patient safety implications should not be considered in this fashion. So I urge my colleagues to oppose this misguided legislation and stand with the more than 100 organizations that have come forward expressing their concern for patients and the unnecessary risk this legislation could expose our Nation's most vulnerable to.
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Mr. GENE GREEN of Texas. Mr. Speaker, I thank my ranking member for allowing me to speak tonight.
Mr. Speaker, I rise in opposition to the Right to Try Act, legislation that would bypass the Food and Drug Administration's longstanding review and oversight of drug treatments and endanger patients with life-threatening diseases.
My heart goes out to the families of loved ones who are terminally ill and desperate for a breakthrough treatment. I, too, have lost loved ones and wished there was an experimental therapy available to save them.
FDA has decades of experience dealing with experimental therapies that have not received final approval. In 1987, the FDA created expanded access, better known as compassionate use, and gives terminally ill patients access to therapies still under clinical trials. FDA approves nearly all requests for investigational drugs. For the last 5 years, the FDA approval rate for this expanded access is over 99 percent. In fact, FDA physicians are available 24 hours a day to approve emergency requests.
My daughter is an infectious disease physician at the University of Nebraska Medical Center. They used the FDA's compassionate pathway to provide experimental therapy for an American doctor, a U.S. citizen, who had contracted Ebola while in Africa in 2014. FDA approved that request for that experimental treatment over the telephone in less than 24 hours. There is a solution other than this bill.
The new path created in this legislation is not necessary, and, in fact, may endanger the health and safety of terminally ill patients by bypassing FDA's oversight and expertise.
Mr. Speaker, I also want to speak on the importance of following regular order. The House Energy and Commerce Committee has been working with stakeholders and Federal agencies for years on creating incentives and pathways for the new generation of breakthrough therapies.
Two years ago, these efforts culminated with the passage of the 21st Century Cures Act, which I am proud to be a champion of. The 21st Century Cures Act went through regular order, including hearings; Member discussions; and compromises between regulators, stakeholders, and regulators.
It is not easy or quick, but regular order works because it gives the committees of jurisdiction the opportunity to debate and refine the legislation. This legislation we are currently considering did not go through regular order. In fact, it was just introduced earlier today, purposely avoiding consideration before our Energy and Commerce Committee due to its shortcomings.
I hope we can agree on the importance of following regular order and observe our Chamber's rules and traditions. The American people deserve nothing less. I ask my colleagues on both sides of the aisle to stand up for Americans facing these serious and life-threatening diseases by opposing this unnecessary and potentially dangerous legislation.
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Mr. PALLONE. Schakowsky).
Mr. Speaker, as I have said, I have great concerns that H.R. 5247 would expose our most vulnerable and desperate patients to unnecessary risk.
Supporters of this legislation have argued that those patients who are suffering from a terminal illness deserve the right to take their health and treatment into their own hands, as they are faced without any other treatment options. Some have even asked: What risk could be worse than the risk of death?
As Arthur Caplan, a bioethicist from NYU, has pointed out: ``There are things worse than death; being made to die faster, being made to die more miserably.''
These are all very real scenarios that patients could be exposed to under the misleading and ill-conceived right-to-try pathway.
As I stated before, while the FDA approved 99 percent of the requests it received, of those, they revised 11 percent in order to protect patients. If this bill becomes law, the FDA no longer will have the opportunity to make those revisions and to protect vulnerable patients.
We must protect patients from bad actors or from dangerous treatments that might make their lives worse. That is why more than 100 organizations have written in opposition to this legislation, including 83 patient organizations like the National Organization for Rare Disorders, the Friends of Cancer Research, the American Cancer Society, Cancer Action Network.
In a letter to the Speaker and the Democratic leader, the patient organizations noted that ``the alternative pathway in the latest version of the legislation is still less safe for our patients than the current expanded access process'' that the FDA uses.
Dr. Ellen Sigal, the chair and founder of Friends of Cancer Research, said: ``In its current form, the proposed legislation does nothing for patients other than provide false hope by allowing them to request a drug with no evidence of efficacy they may never receive and, should they receive it, may do more harm than good.''
So I think we should all be concerned about protecting patients. Rather than rushing this bill through today, I would urge my colleagues to oppose this legislation and to come back to the table to find a solution that will streamline Expanded Access Programs while protecting patients from unnecessary harm.
Mr. Speaker, there are a lot of reasons to oppose this bill today, and I have given a number of them, but the primary reason being the need to continue protecting patients by ensuring that the FDA remains a part of the process.
While we are speaking about process, I have to also oppose this legislation based on the inappropriate way the Republican majority is bringing this bill to the floor. Bills considered under suspension have traditionally gone through the committee process with overwhelming bipartisan support, and neither of those things is the case with this bill.
It was introduced today.
Does the majority really believe they are giving Members the appropriate time to view this bill when it was introduced at 2 p.m.?
Patient access and patient safety should be shared goals among Democrats and Republicans, goals that could be achieved if this legislation was not being rushed to the floor under an arbitrary deadline.
Legislation such as this, that carries such great risk of patient harm, should be considered carefully, with attention paid to the unintended consequences that could follow.
Mr. Speaker, I would urge my colleagues to oppose this unnecessary and risky legislation, and to return to the regular order of the committee to consider legislation that would protect both patients from harm and the FDA from the weakening of the agency's role in our drug approval process.
We should not be voting on a bill of this consequence that was introduced this afternoon.
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Mr. PALLONE.
Mr. Speaker, I just want to talk about two other aspects of this bill that I haven't so far. One is the fact that States have actually implemented right-to-try laws that have done little to expand access to investigational treatment. Although 17 States and the District of Columbia have enacted right-to-try laws, there is no evidence that anyone has obtained an investigational treatment via these laws that couldn't have been obtained through FDA's expanded access program.
Right-to-try laws do not compel companies to provide patients access to investigational treatments. Therefore, under these State laws, patients still do not have a right to try, only the right to request the treatment from the company. State right-to-try laws do not address the fundamental barriers of cost and accompanying restrictions.
Neither the FDA nor States require insurers or pharmaceutical companies to cover the cost or reduce the cost of these expensive treatments. Instead, these laws put patients at a higher risk by prohibiting or weakening FDA oversight of investigational treatments.
With regard to clinical trials, the legislation could also expose patients to unnecessary risk by allowing access to investigational drugs that have only completed a phase I clinical trial. Phase I trials are extremely small trials, in the range of 20 to 80 patients, and are used primarily to determine toxicity. They do not determine effectiveness or potential side effects. Patients could suffer from harmful side effects or delay enrolling in a clinical trial program for a treatment that actually has evidence of efficacy for their disease or condition.
Finally, the bill would weaken the FDA's ability to oversee the adverse events or other clinical outcomes from the use of investigational drugs and provide broad liability protections for manufacturers, leaving patients with no recourse in the case of an adverse effect.
I just wanted to mention those.
Mr. Speaker, I just want to conclude, if I could, in opposition to this bill by quoting some of the former FDA Commissioners.
This is from Dr. Margaret Hamburg, who said:
I am deeply concerned by the draft legislation being considered to remove the FDA from the proposals around right to try. Excluding the FDA will not benefit those patients and would be a mistake. There is no need to create a new potentially dangerous paradigm by passing this legislation which does not address the real issues at hand and could have unintended negative consequences, leading to a possible impediment of the development and approval of safe and effective therapies.
And then, finally, is the former FDA Principal Deputy Commissioner, Joshua Sharfstein, who said:
FDA review allows doctors and patients to tell the difference between a medication that works and one that does not. Evidence also orients the pharmaceutical market towards developing products that produce meaningful benefits for patients instead of just hope. Undermining FDA review by giving a right to patients to try anything at any time will leave more patients in desperate situations with fewer options and less understanding of what could really make a difference.
Again, Mr. Speaker, I would urge opposition to this legislation.
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