Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018

Floor Speech

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Mr. PALLONE.

Mr. Speaker, I rise today to voice my strong opposition to H.R. 5247, the Right to Try Act of 2018.

This legislation, introduced only last week, is an egregious attempt, in my opinion, by the Goldwater Institute to undermine the gold standard drug approval process at the Food and Drug Administration.

The supporters of this bill claim to be helping desperate patients who are looking for hope.

If this is such a patient-centered bill, then why does every major patient organization overwhelmingly oppose it?

More than 100 patient organizations, including the National Organization for Rare Disorders, the Friends of Cancer Research, and the American Cancer Society have all written in opposition to this legislation.

In a letter to congressional leadership, these 103 patient organizations noted ``that the alternative pathway in the latest version of this legislation is still less safe for our patients than the current expanded access process under the FDA.''

It is not only the patient organizations that are voicing concerns. Four former FDA Commissioners--Drs. Hamburg and Califf, who served under the Obama administration; and Drs. McClellan and Andrew von Eschenbach, who served under the Bush administration--also oppose this legislation. That is two former Republican Commissioners and two former Democratic Commissioners who are opposed to both the House bill and the Senate bill on this same issue.

These four Commissioners explained their opposition by saying: ``There is no evidence that either bill would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients.''

Mr. Speaker, I think most importantly, I would stress that this legislation is simply not needed. There is already a successful program in place today at the FDA in which seriously ill patients and their doctors can request access to an experimental treatment from a manufacturer. This application process, which takes as little as 45 minutes for a physician to complete, has been overwhelmingly successful.

Last summer, a review by the Government Accountability Office found that the FDA approves 99 percent of the requests submitted to the agency. In fact, of the nearly 1,700 requests the FDA received last year, only 9 were not approved.

Physicians and patients also receive approval quickly. Emergency requests are often granted immediately over the phone and, on average, receive a response within 4 days.

While the FDA approves 99 percent of the treatments it reviews through this expanded access process, as it is called, it also adjusts applications for 11 percent of the patients to improve patient safety protections.

In order to protect patients, this review, in my opinion, should continue. We must protect patients from bad actors or from dangerous treatments that might make their lives worse. Just imagine the health consequences to patients if these 11 percent of applicants had not been adjusted.

This is the very reason that the FDA must be involved in the process. If you eliminate FDA review, as this bill does, you are putting patients at risk.

I want to talk a little bit about the fact that many States now have right-to-try statutes. I fear that some Members--and I heard this last week when the bill was on the suspension list--might support this legislation under the false belief that the State right-to-try laws in their States have provided help to patients. But nothing could be further from the truth.

One example supporters of this legislation like to bring up is Dr. Delpassand from Texas, who claims to have treated patients under the State right to try.

Mr. Speaker, I include in the Record a letter from Mr. Andrew McFadyen of The Isaac Foundation, who dispels this myth. The Isaac Foundation, March 20, 2018. Rep. Greg Walden, Chair, Rep. Frank Pallone, Ranking Member, Energy & Commerce Committee.

Dear Mr. Pallone and Mr. Walden: I am writing to you regarding your upcoming debate on HR 5247, the Right to Try initiative fronted by the Goldwater Institute. I am the Executive Director of The Isaac Foundation, an organization that is dedicated to providing advocacy and support to patients dealing with a wide range of disorders and needing access to rare disease treatments. Our work pushes international boundaries, with the bulk of our efforts taking place in Canada and the United States. I am also a member of the NYU Working Group on Compassionate Use and Pre-Approval Access where we are making a concerted effort to improve and address the issues around access to experimental medications, and I'm involved with a non-profit called GE2P2.

I'm proud to say at The Isaac Foundation that we've never been unsuccessful gaining access to life-saving medications and treatments for patients in Canada, and our work directly with pharmaceutical companies is helping countless patients see similar results in the United States. We have had success by being collaborative partners with industry, regulatory authorities, and patients in need.

I watched the discussion last week with growing consternation that many of our elected officials have not taken the opportunity to fact-check claims being made by RTT proponents. Most notably, continued mention of Right to Try being used by Dr. Delpassand out of Texas is both egregiously wrong and, indeed, is the perfect example of why RTT should not be passed by lawmakers.

In October 2016, I testified during Senator Ron Johnson's hearing on Right to Try, at which Johnson introduced and played a video created by the Goldwater Institute of Dr. Delpassand. During that 3-minute video, Dr. Delpassand explained that he was using the state RTT law to treat his patients because the FDA would not allow him to do it through an Expanded Access Program. Senator Johnson asked me what I thought about this video--which included few facts, no context, and was edited by the people fronting the RTT push themselves. I explained that there must be a reason why Dr. Delpassand was in the 1% of cases not allowed by the FDA and vowed I would investigate.

In March of 2017, I received a set of documents from the FDA under a FOIA request. They show that Dr. Delpassand's clinic failed inspections during the clinical trial of Lutathera (lutetium Lu 177 dotatate). Specifically, he failed inspection due to 3 key and very important reasons:

1. Enrolling subjects into the study during a partial clinical hold, issued by the Agency.

2. Underreporting of Adverse Events.

3. 1572-protocol noncompliance.

The failed inspections were discovered after complaint from the CDER Good Clinical Practice Compliance Oversight Branch, Division of Good Clinical Practice Compliance Evaluation, Office of Scientific Investigations (OSI). A ``Clinical Hold'' was placed on the lab and Dr. Delpassand. During a clinical hold, subjects may not be given an investigational drug. Dr. Delpassand and his clinic disregarded this clinical hold and enrolled 6 patients.

Additionally, and just as concerning in terms of patient safety, Dr. Delpassand's clinic failed to promptly report significant new adverse events or risks to the FDA. This failure to report was noted numerous times during the inspection. The inspection also found numerous other areas of concern. I have attached the full report for your consideration.

After these inspections, the FDA would not allow Dr. Delpassand to open an EAP at his clinic for patients in need, and rightly so. They FDA did, however, allow 42 different locations the ability to provide this drug for patients requiring access, including two sites in Texas. A quick search on ClinicialTrials.gov shows this information, further proving that the FDA has been able to provide patients the required access they need, ensuring the environment that they are receiving the drug they need is safe.

My understanding of the situation is that the company running the clinical trial distanced themselves from Dr. Delpassand after these failed inspections. Without company support, and without the FDA's permission to open an EAP, Dr. Delpassand had to use the state legislation to provide drug to his patients. Questions remain, however, such as how Dr. Delpassand paid for the product he was giving his patients, did patients themselves have to pay for that drug supply (which isn't allowed under the Texas RTT law) and who, if anyone, was overseeing the program to ensure safety of the patients, especially after multiple infractions were seen during the failed FDA inspection.

Most important, it should be noted that the FDA process here worked exactly how it is supposed to. A lab was inspected for safety to ensure patients are looked after in the appropriate fashion. That inspection placed a hold on further treating of patients due to numerous infractions. The FDA worked with the company to ensure access for patients across the USA in 42 different sites, helping to monitor adverse events while also allowing the product to advance to approval. That product was approved by the FDA in January 2018.

Also importantly, RTT was used because it was the only way for Dr. Delpassand to treat patients in his clinic after it failed inspection. RTT is a loophole designed to allow people who cannot otherwise follow safety rules set forth by the FDA that are meant to protect vulnerable patients. It's not being used--anywhere--to provide patients with hope or access to life-saving drugs.

One final note, and one that I've not see mentioned anywhere. HR 5247 includes the name of a young child--a brave child battling Duchenne Muscular Dystrophy--named Jordan McLinn. Jordan has been photographed numerous times with Vice President Pence, and is often used as an example of why Right to Try is needed. The problem with these optics is that Jordan has never received any treatment under Right to Try, even though Right to Try has been available in his state of Indiana for 3 years. He already has access to the life-saving treatment he needs--through an FDA approved clinical trial. He's doing well on that trial drug, as I understand it, and receives all the benefits of FDA oversite to ensure his safety on that trial. In essence, the child used to promote RTT is the perfect example of why the FDA process works and is needed.

The true reality is that the landscape for access to medications for dying patients does not change tomorrow if a Federal Right to Try law is passed today. Very clearly, those patients in dire need of help today will wake up tomorrow needing access to the same life-saving treatments, and feel the same despair because they will not be getting the access they need through Right to Try.

The barrier to that access here isn't the FDA, and no Right to Try law enacted by lawmakers in this country is going to remove the true barrier--pharmaceutical companies. The gatekeepers to these medications are the pharmaceutical companies themselves, and we need to be working collaboratively as a team--Industry, Government, physicians, and Patients--to craft solutions that will work for everyone, keeping in mind that we are all on the same side, that we all want the same thing--broad and expeditious access to life- saving medications for patients in need.

I understand how difficult this is for patients--I see it every day, and I feel it every night as I check in on my son (who is battling his own devastating and very rare disease) to make sure he is still breathing, to make sure he is still with us. But I also understand that the change we all need will not come with Right to Try. It will come through collaboration with all stakeholders and by providing companies the safety and assurances they need to make their medications available to our dying patients.

Lawmakers should be spending their time helping make that collaboration happen because that is how we are going to save our dying patients. They should not spin their wheels passing legislation like Right to Try that looks good, and feels good, but will do nothing for those in need. If they do, they are doing a disservice to a large and very vulnerable group of patients now and in the future, my own son, my own hero Isaac, included.

Thank you for your time on this matter. Sincerely, Andrew McFadyen, Executive Director, The Isaac Foundation.

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Mr. PALLONE. Andrew McFadyen said:

Dr. Delpassand claims to have used right to try because FDA would not allow him to do expanded access. And this was for a very good reason. FDA placed a clinical hold on a study, due to the fact that his clinic was not reporting serious, adverse events, as required; and he continued to enroll patients, despite the clinic hold.

The work of Dr. Delpassand's study was associated with 40 deaths and 2 hospitalizations. FDA's clinical hold on Dr. Delpassand's work is a sign to me that FDA's expanded access pathway was working to prevent bad actors from continuing to expose vulnerable patients to experimental treatments.

Mr. Speaker, H.R. 5247 is dangerous for our patients. It is an unprecedented attempt to roll back the FDA's oversight of investigational treatments. I urge my colleagues to stand with more than 100 organizations that have come forward to oppose this misguided and, I believe, harmful legislation.

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Mr. PALLONE. Schakowsky), who is the ranking member for the Digital Commerce and Consumer Protection Subcommittee.

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Mr. PALLONE. Matsui).

Mr. Speaker, I want to explain some other reasons why I am very opposed to this bill. I am concerned that H.R. 5247 essentially does nothing to address what may be the true barrier to expanded access, and that is the determination by the manufacturer as to whether or not they will provide access to their product that is under development. And I want to stress, there is nothing in this legislation before us today that would compel a manufacturer to grant access upon request.

Further, I believe that trusted manufacturers like J&J, or Johnson & Johnson, which is headquartered in my district, have already said that any compassionate use request must be subject to FDA review. Now, I have heard my colleagues refer to this as a Hail Mary pass for the terminally ill. I think, in reality, it is offering false hope of a cure to patients and their families when there is no guarantee that any patient will receive access to treatment from a manufacturer.

In fact, H.R. 5247 sets an extremely low threshold for the types of experimental treatments that may be available through this alternative pathway by allowing patients access to investigational treatments that have only completed a phase 1 clinical trial. Patients will be exposed to treatments with no or relatively little data that they are actually effective. These extremely small trials only examine the safety and toxicity of a drug and do not determine the effectiveness or potential side effects. Access at this phase 1 stage in the development could expose patients to untested products and further harm and result in delaying access to a treatment that may be more appropriate and more beneficial for their underlying disease or condition.

Only 1 in 10 products move on from phase 1 clinical trials to FDA approval. Mr. Speaker, the bill does not make any adverse-event reporting to the FDA immediate. It also 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.

Again, these are some of the many reasons that more than 100 organizations oppose this dangerous bill.

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Mr. PALLONE. Mr. Speaker, may I inquire how much time I have remaining?

Mr. GENE GREEN of Texas. Mr. Speaker, I thank my ranking member for yielding to me. I rise in opposition to the right-to-try legislation that would bypass the Food and Drug Administration's longstanding review and oversight of drug treatments and endanger patients with life-threatening diseases.

Many States have passed this right-to-try piece of legislation, including my home State of Texas, but the States don't have the FDA. The Federal Government has the right to be able to make sure we can protect both constituents and consumers. My heart goes out to the loved ones who are terminally ill and desperate for a breakthrough treatment. I cannot support legislation that offers false hope to the terminally ill and their families.

The FDA has a pathway whereby those in need of investigational medications may seek to obtain them. This program is known as the expanded access pathway, or compassionate use, and has been in the law since 1987. Over the last decade, the FDA has a clinical hold on only two commercial drug development programs due to adverse events associated with compassionate use.

There are many patient advocacy groups that are opposing this legislation. Groups such as the Alliance for Aging Research, the American Cancer Society Cancer Action Network, American Lung Association, the American Society of Clinical Oncology, the Cystic Fibrosis Foundation, Defeat MSA, the Disability Rights Legal Center, and dozens more that are committed to seeking effective treatment cures to many diseases which are terminal, are against this bill. These patients' rights groups seek to ensure that the medication that is offered to individuals is safe, has been tested, and has gone through the proper approval process before it is given to a patient.

The most vulnerable and terminally ill individuals deserve to have access to safe therapies that have undergone the necessary approval process before being given to those who can least afford to receive unproven treatment that may do them more harm than good. In addition to the physical harm which unproven treatments may cause, there is also the risk of financial exploitation of terminally ill patients given that such treatments are not covered by insurance. Manufacturers are not required to cover the cost of investigational treatment.

The majority's decision to go around our committee's consideration and effort to pass the bill on suspension last week exemplifies what this legislation is trying to do, circumvent existing rules and processes that have been created to protect Americans from hasty decisions.

I ask my colleagues on both sides of the aisle to stand up for Americans facing serious and life-threatening diseases by opposing this unnecessary and potentially dangerous legislation.

Mr. Speaker, there has been a lot of misinformation spread by supporters of this legislation that FDA is a barrier to patients receiving access to these investigational treatments, and I want to be very clear that that is simply not the case.

FDA's expanded access program approves nearly all requests for investigational drugs or biologics it receives. For the past 5 years, FDA's approval rate for expanded access requests has been over 99 percent. In fiscal year 2017, as I previously mentioned, only nine individual requests were denied.

FDA also conducts its review quickly. FDA physicians are available 24 hours a day to approve any emergency expanded access requests the agency receives, typically granting emergency requests immediately, over the phone, and nonemergency requests in a median time of 4 days and, generally, no longer than 30 days.

FDA has also taken actions to streamline the expanded access request process for physicians to make it less burdensome. I think that was mentioned by Mr. Walden, the chairman.

Pharmaceutical companies can choose to deny a patient access to an experimental treatment because, for example, there is not enough of the drug available or they are concerned about dangerous side effects. The fact is, when a patient is denied access to an experimental treatment, it is because the company has said no, not the FDA.

So let's be clear as to what this legislation is. It is an attempt to undermine the authority of the expert public health agency charged with reviewing drugs to ensure their safety and efficacy.

I would urge my colleagues to oppose this grab at FDA's authority. That is really what this legislation is all about.

Mr. Speaker, I said before that I have found that some Members were looking to vote for this bill because they said: Well, we have the right to try in our State by State statute, so what is the difference if we do it on the Federal level?

I just want to stress again that the State right-to-try laws do not give patients a right to try effectively and have done little to expand access to investigational treatments.

There are 37 States and the District of Columbia that have enacted right-to-try laws, and 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 company restrictions. Neither the FDA nor States require insurers or pharmaceutical companies to cover the cost or reduce the costs of these often expensive treatments. Instead, these laws put patients at higher risk by prohibiting or weakening FDA's oversight of investigational treatments.

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Mr. PALLONE. Mr. Speaker, may I inquire how much time remains and whether the gentleman has additional speakers on his side.

Mr. Speaker, I just want to stress that, as I said before, we have the four previous FDA Commissioners, two Democrats and two Republicans appointed by President Bush, who have raised serious concerns about this legislation because it excludes FDA review and they think could pose serious risks to vulnerable patients.

I just wanted to read, once again, a statement that they made jointly to The Washington Post, where they said: ``There is no evidence that either bill would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients.''

Mr. Speaker, I just want to stress to my colleagues on both sides of the aisle that my concern is that no one is actually going to be able to get an experimental drug by this bill. In other words, if you are a manufacturer that actually has done something and come up with an experimental drug that you believe will make a difference to someone who is terminally ill, you are likely going to want to go through the FDA expanded access process because then there is a seal of approval that the FDA has actually looked at this and said that it is relatively safe to use.

So my real fear is that the only thing this is going to do is open up to the possibility of some charlatan, fly-by-night snake oil drug company or manufacturer who is going to make all kinds of claims that have not been reviewed by the FDA for any kind of safety, and that then people may say: Okay. Well, I will take that because I am terminally ill and I might as well try something.

But that isn't really what we should be doing here. We should be providing a process, as the FDA does right now, where, if someone is terminally ill and they want to try something, they at least have some certification of approval by the FDA that this is something that may help them, that may make a difference, and that, in the case of about 11 percent of the cases where the application is made to the FDA, some changes are made to make sure that even though there is a certain level of risk, that that level of risk is reduced by the FDA putting on additional safety precautions.

So my real concern here is I don't want people to vote for this legislation thinking that somehow it is going to make a difference. I really don't believe that is true. Otherwise, I wouldn't urge the opposition that I am.

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Mr. PALLONE. Mr. Speaker, I have a motion to recommit at the desk.

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Mr. PALLONE. I am opposed to the bill in its current form.

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Mr. PALLONE. Mr. Speaker, this is an amendment to the bill, or the final amendment to the bill, which will not kill the bill or send it back to committee. If adopted, the bill will immediately proceed to final passage, as amended. And this amendment would offer a more targeted approach to improving the FDA's current expanded access program.

In October, the Energy and Commerce Committee held a hearing on the widely opposed Senate right-to-try legislation. At that hearing, we heard concerns from FDA Commissioner Gottlieb and also from manufacturers, academic experts, and patient groups that S. 204 was legislation that would expose broad numbers of patients to harm, and sought to hamstrung the FDA's ability to oversee or engage in any meaningful way on the use of investigational treatments.

Since that time, my colleagues on the other side of the aisle have drafted new legislation that maintains, in my opinion, the same harmful approach prohibiting FDA review of experimental treatments. The 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.

So my motion to recommit, Mr. Speaker, abandons this harmful attempt to undermine the FDA's expanded access pathway and, instead, seeks to make two improvements that have been identified as meaningful by both manufacturers and patient groups.

This proposal will also not be any surprise to Chairman Walden or Chairman Burgess because it was the bipartisan proposal our staffs were negotiating prior to the introduction of the current Republican bill.

So I want to stress that, unlike the current bill, H.R. 5247, this proposal is not based on the false premise that FDA approval is a barrier to accessing investigational treatments. Rather, it addresses the two key problems identified by expert witnesses at our hearing: how the FDA will utilize clinical outcomes of investigational treatments and liability protection.

To that end, under this motion to recommit, the FDA is directed to issue guidance to manufacturers specifically on how and when the FDA will consider clinical outcomes, and when a sponsor may request the consideration of such outcomes when it comes time to submit an application for approval for the investigational treatment.

This will provide manufacturers with the clarity they are seeking regarding how allowing patients access to drugs that are still under development may impact their ability to gain full FDA approval. It will also ensure that there is a public process for such guidance, ensuring that stakeholders will have the opportunity to offer their views on this issue.

Mr. Speaker, the motion to recommit also provides liability protection to manufacturers, physicians, clinical investigators, and hospitals, if they are in compliance with the current law and regulations for expanded access. If you are a manufacturer, a physician, or a hospital that is in compliance with current rules and requirements related to expanded access, you will receive protection for allowing access to the investigational treatment.

Finally, it also provides transparency around the number of expanded access requests the FDA receives and grants, how many requests a manufacturer receives and grants, and if there are any serious adverse events. This transparency, I believe, will provide clear data as to how many patients are making expanded access requests and how often these requests are granted or denied by the FDA and manufacturers.

Mr. Speaker, I believe that these legislative fixes will go a long way to bolstering the existing successful expanded access pathway, while maintaining the critical review and oversight of the agency charged with protecting our public health, that being the FDA.

I just want to say that, last fall, FDA Commissioner Gottlieb testified on right-to-try efforts and told our committee: ``There is a perception that certain products that aren't being offered under FDA expanded access will be offered under right-to-try, and I don't see that.''

That is our current Commissioner Gottlieb, who I respect a great deal.

Rather than creating an unnecessary alternative pathway that threatens our drug approval process and our clinical trial program, I would urge my colleagues to join with Democrats and 103 patient organizations in supporting the current expanded access program.

These targeted improvements under the motion to recommit to the existing program are, I think, a way to achieve a better goal. So I urge my colleagues to support my motion to recommit and oppose this, what I consider, dangerous Republican proposal in the bill before us.

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Mr. PALLONE. Mr. Speaker, on that I demand the yeas and nays.

The yeas and nays were ordered.

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