Why Stem Cell Advocates Texans for Cures say “Right to Try” Legislation Should be Fought

Texans for Cures 

This week in Washington DC a delegation from the stem cell advocacy group Texans for Cures is meeting with members of Congress from both parties. The focus of the meetings are three bills promoting “Right to Try” legislation. Supporters of the bills say they will empower patients battling terminal illness. Texans for Cures say, quite the contrary, that these laws will endanger patients. In this guest blog, Texans for Cures explain why they feel these laws are bad.

In 2014, the Goldwater Institute published a policy report titled, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment.”[i] The report calls for states to pass “Right to Try” legislation as a means to reclaim patients’ medical autonomy and right to determine their own medical treatment.

This policy recommendation is built on the theory that the Food and Drug Administration (FDA) should not be able to restrict terminal patients’ access to potentially life-saving treatments so long as the treatment has been tested for basic safety. While this idea may seem immediately appealing, the policy undermines medical research in several ways that are harmful to the development of new treatments.

Texans for Cures opposes this legislation because it harms the sound development of treatments for future patients on the mere chance that it may provide relief to current patients that have received a terminal diagnosis. In short, Right to Try policies ignore the attendant risks and overemphasize the potential benefits.

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“Right to Try” Model Legislation and State Enacted Variants

The Goldwater Institute’s policy report included model legislation for interested legislators, which it summed up as follows:

Simply stated, Right to Try allows a patient to access investigational medications that have passed basic safety tests without interference by the government when the following conditions are met:

  1. The patient has been diagnosed with a terminal disease;
  2. The patient has considered all available treatment options;
  3. The patient’s doctor has recommended that the investigational drug, device, or biological product represents the patient’s best chance at survival;
  4. The patient or the patient’s guardian has provided informed consent; and
  5. The sponsoring company chooses to make the investigational drug available to patients outside the clinical trial.

Since the Goldwater Institute published this policy report in 2014, 33 states have enacted Right to Try laws.[ii] These laws contain minor variations from the model legislation, but each operates similarly to limit the FDA’s oversight roll.

Right to Try is Loosely Grounded in the Constitution and May Require Federal Action

Due to the fact that these laws may infringe on the FDA’s authority over drug development and distribution, the policy report attempts to ground Right to Try in one’s constitutional right to liberty. This constitutional underpinning is loose and is not firmly supported by Supreme Court precedent.[iii] With the constitutional basis of Right to Try resting on a weak foundation, it is important for Right to Try proponents to pass a complimentary Right to Try statute on the federal level in Congress.

There are three bills actively working through the Congressional process that would prohibit the FDA or any other federal agency from interfering with a patient’s Right to Try: H.R. 878 by Representative Biggs,[iv] H.R. 2368 by Representative Fitzpatrick,[v] and S. 204 by Senator Johnson.[vi] Each of these bills shares three common provisions, while H.R. 2368 has two additional provisions:

Common Provisions:

  1. Prohibition on federal action
  2. No liability
  3. No use of outcomes

Provisions Unique to H.R. 2368:

  1. Manufacturers are not required to make treatments available
  2. Permits manufacturers to receive compensation or recover costs

All three of the federal bills would remove the FDA’s ability to intervene in state Right to Try programs. They also create a liability shield for any producer, manufacturer, distributor, prescriber, dispenser, possessor, or user participating in the program. And finally, each prohibits the use of outcomes from patients participating in Right to Try as a criteria for FDA review of the treatment. This means that harmed patients would have limited or no legal recourse, and the FDA may need another Act of Congress to grant them the authority to intervene in any programs that prove to be dangerous. However, it may be difficult to know if these programs are harming patients or not, because the bills do not provide any mechanism for tracking outcomes and using that information for oversight.

Each bill is drafted in a way that would remove FDA oversight authority and would allow states to proceed with Right to Try policies and grants states broad discretion to tailor these programs without federal oversight. However, H.R. 2368 contains two additional provisions that would compliment and potentially override state statute. First, the bill gives manufacturers the authority to deny patients access to investigational treatments, which is consistent with the Goldwater Institute’s model legislation. Second, the bill allows manufacturers to receive compensation or recover costs involved in making the drug available to patients. This second provision is particularly problematic in that it would allow manufacturers to charge patients for unproven treatments unless they were explicitly prohibited from doing so by state law.

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How Right to Try Laws Structurally Harm the Research Process

Right to Try laws create a number of problematic incentives and penalties that would likely harm the long term development of new therapies. First and foremost, under Right to Try, patients will be able to bypass the clinical trial process, request investigational treatments, and pay the cost of the drug, rather than enter into a clinical trial. Given that clinical trials may involve the use of placebos, Texans for Cures is concerned that patients may choose to exercise Right to Try rather than participate in a clinical trial, because under Right to Try the patient avoids the possibility of receiving a placebo.

Additionally, there is no mechanism in the proposed bills for tracking outcomes of patients participating in Right to Try, and there is no mechanism for government intervention if Right to Try proves to be unreasonably risky.

Right to Try seeks to shield all participants from liability, meaning that patients who are harmed will have limited or no legal recourse, even if manufacturers or physicians are negligent. Furthermore, Right to Try laws typically allow manufacturers to recover the cost of manufacturing the treatment for participating patients, but cost is not defined. Does cost include the cost of research and development or is it exclusively the cost of creating that specific treatment? The ambiguity surrounding this term is a cause for concern, because companies may be tempted to use this ambiguity to cover a broader sets of costs than the authors intended.

Conclusion

Texans for Cures opposes this legislative effort because the program could potentially harm patients and, if it does, the law does not provide adequate safeguards or remedies. Additionally, the law does not require any monitoring of outcomes and is therefore unscientific in its approach to treatments that are currently undergoing clinical research.

The FDA is already working to ease the burdens associated with Expanded Access programs, which achieve the end that Right to Try desires: providing access to research drugs for terminal patients. The difference is that Expanded Access has additional safeguards and a mechanism for FDA intervention if treatment is found to be dangerous or harmful to the clinical trial process.

Finding scientifically sound treatments for patients in need is the primary concern of Texans for Cures. Texans for Cures sympathizes with, and its members have similarly experienced, the pain of losing loved ones. The hope and emotion involved in Right to Try laws is not to be taken lightly, but it is precisely because strong emotions can cloud our judgment that we, as a society, must approach the clinical trial process with a clear mental state. Texans for Cures believes that Right to Try will harm the long term development of new treatments and therefore asks for your help in fighting this legislative effort.

Footnotes:

[1] Christina Corieri, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment,” Goldwater Institute (2014), https://goldwater-media.s3.amazonaws.com/cms_page_media/2015/1/28/Right%20To%20Try.pdf

2 KHN Morning Briefing, “‘Right to Try’ Advocates Help Pass Laws In 33 States As Movement Gains National Foothold,” Kaiser Health News (2017), http://khn.org/morning-breakout/right-to-try-advocates-help-pass-laws-in-33-states-as-movement-gains-national-foothold/

3 The Goldwater Institute’s sole source for constitutional grounding for this law comes from a concurrence by Justice Douglas in Doe v. Bolton, 410 U.S. 179, 218 (1973), where he noted that individuals have a “right to care for one’s health and person.” The Goldwater Institute appears to recognize the precarious footing of their model legislation, stating in their policy report, “Although the right of terminal patients to access investigational medications has not yet been recognized by the Supreme Court, it is consistent with and can be supported by existing precedent.”

[1] H.R. 878 by Representative Biggs, https://www.congress.gov/bill/115th-congress/house-bill/878/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=2

[1] H.R. 2368 by Representative Fitzpatrick, https://www.congress.gov/bill/115th-congress/house-bill/2368/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=1

[1] S. 204 by Senator Johnson, https://www.congress.gov/bill/115th-congress/senate-bill/204/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=3

‘Right To Try’ laws called ‘Right To Beg’ by Stem Cell Advocates

In recent years, ‘Right to Try’ laws have spread rapidly across the US, getting approved in 32 states, with at least three more states trying to pass their own versions.

The organization behind the laws says they serve a simple purpose:

‘Right To Try’ allows terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves. ‘Right To Try’ expands access to potentially life-saving treatments years before patients would normally be able to access them.”

That certainly sounds like a worthy goal; one most people could get behind. And that’s what is happening. Most ‘Right To Try’ laws are passed with almost unanimous bi-partisan support at the state level.

Beth Roxland

Beth Roxland

But that’s not the view of Beth Roxland, an attorney and health policy advisor with an extensive history in both regenerative medicine and bioethics. At the recent World Stem Cell Summit Roxland said ‘Right To Try’ laws are deceptive:

“These are not patient friendly but are actually patient unfriendly and could do harm to patients. The problem is that they are pretending to do something that isn’t being done. It gives patients a sense that they can get access to a treatment, but they don’t have the rights they think they do. This is a right to ask, not a right to get.”

Roxland says the bills in all 32 states are almost all identical, and use the same cookie-cutter language from the Goldwater Institute – the libertarian organization that is promoting these laws. And she says these laws have one major flaw:

“There is no actual right provided in the bill. The only right is the right to try and save your life, “by requesting” from a manufacturer a chance to try the therapy. The manufacturer doesn’t have to do anything; they aren’t obliged to comply. The bills don’t help; they give people false hopes.”

Roxland says there isn’t one substantiated case where a pharmaceutical company has provided access to a therapy solely because of a ‘Right To Try’ law.

However, Starlee Coleman, the Vice President for Communications at the Goldwater Institute, says that’s not true. She says Dr. Ebrahim Delpassand, a cancer specialist in Texas, has testified before Congress that he has treated dozens of patients under his state’s ‘Right To Try’ law. You can see a video of Dr. Delpassand here.

Coleman says ;

“We think the promise of ‘Right To Try’ is self-evident. If one doctor alone can treat 80 patients in one fell swoop, but the FDA can only manage to get 1200 people through its expanded access program each year, we think the potential to help patients is significant.”

Other speakers at the panel presentation at the World Stem Cell Summit said these laws can at the very least play an important role in at least raising the issue of the need for people battling terminal illnesses to have access to experimental therapies. Roxland agreed it was important to have that conversation but she pointed out that what often gets lost in the conversation is that these laws can have hidden costs.

  • 13 states may withdraw hospice eligibility to people who gain access to an early or experimental intervention
  • 4 states may withdraw home care
  • 6 states say patients taking part in these therapies may lose their insurance
  • Several states allow insurers to deny coverage for conditions that may arise from patients getting access to these therapies
  • 30 states say the companies can charge the patients for access to these therapies

Roxland says the motives behind the ‘Right To Try’ laws may be worthy but the effect is misleading, and diverts attention from efforts to create the kind of reforms that would have real benefits for patients.

Here is a blog we wrote on the same topic last year.