Is the Federal "Right to Try" Legislation Righteous, or Rotten?

By: Brandon M. Macsata, CEO, ADAP Advocacy Association

Thirty-eight states have laws on it. The U.S. Senate passed a bipartisan bill on it (S. 204). The current occupant at 1600 Pennsylvania Avenue has endorsed it. Many patient rights groups support it. So it begs the question, what is holding-up federal "Right to Try" legislation in the U.S. Congress? Most likely it all depends on your perspective.

According to Wikipedia, "Right-to-try laws are U.S. state laws that were created to let terminally ill patients try experimental therapies (drugs, biologics, devices) that have completed Phase 1 testing but have not been approved by the Food and Drug Administration (FDA)."[1] Simply put, right-to-try laws allow patients and their doctors to bypass the FDA and seek the an investigational therapy directly from the drug manufacturer.

The libertarian-leaning Goldwater Institute touts that the right-to-try laws are already in place in 38 states and counting: Alabama, Arizona, Arkansas, California, Colorado, Connecticut, Florida, Georgia, Idaho, Illinois, Indiana, Iowa, Kentucky, Louisiana, Maine, Maryland, Michigan, Minnesota, Mississippi, Missouri, Montana, Nevada, New Hampshire, North Carolina, North Dakota, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, West Virginia, Washington and Wyoming. More states are expected to follow suit, too.[2] This effort is supported by numerous national patient advocacy organizations.

Certainly for people living with life-threatening (or potential life-threatening) conditions, right-to-try laws make perfectly good sense to them. In fact, there was a time when living with HIV/AIDS was considered one such condition (and still can be so, if the person is not properly treated or if the person is experiencing treatment failure/resistance). “Dallas Buyers Club” styled arrangements back in the 1980s and early 90s come to mind. Today, it is common to find enthusiasts for right-to-try laws among people living with Alzheimer’s disease, cancer, chronic obstructive pulmonary disease (COPD), or Lou Gehrig’s disease (ALS), among others. People living with HIV/AIDS also appear to support them, at least in theory.

There has been a growing effort to pass a federal right-to-try law, mirrored after what states have already passed. Nathan Nascimento, senior policy adviser at Freedom Partners Chamber of Commerce, previously argued in Forbes Magazine: "The few available remedies for this problem are limited. The agency sometimes issues 'compassionate use' exemptions allowing patients to try certain medicines and treatments still under federal review, but getting those exemptions is easier said than done. Roughly 99% of those seeking compassionate use exemptions never get through their application."[3]

Though there has been increased attention on right-to-try laws, they received a big boost earlier this year. On January 30th during the State of the Union (SOTU), such laws received the unequivocal support from the man delivering the speech:

“We also believe that patients with terminal conditions should have access to experimental treatments that could potentially save their lives. People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home. It is time for the Congress to give these wonderful Americans the ‘right to try.’”

Photo Source: SUSAN WALSH/AP

The remarks were hailed by David Barnes, who serves as the policy director for the millennials advocacy group Generation Opportunity. Barnes penned an Op-Ed in which he argued, "Change to federal policy is needed to ensure that the FDA, or any other federal agency, does not interfere with state-passed laws. And there are terminally ill patients who don’t live in Right to Try states and desperately seek a chance to save their lives."[4]

Not everyone applauded the presidential endorsement.

Alison Bateman-House, assistant professor in the Division of Medical Ethics at NYU School of Medicine, claimed what was extolled in the SOTU would only punish the seriously ill. Writing an opinion piece in Forbes Magazine, Bateman-House summarized, "Rarely has a president spoken so vehemently in favor of a bill that would do so little for the sick. But even allowing for the extemporaneous nature of these remarks, it is clear that the president misunderstands how access to investigational drugs works in the U.S. and how right-to-try legislation, if enacted, would change it."[5]

Ms. Bateman-House was not alone. The Society for Science-Based Medicine (SBM),[6] and the National Organization for Rare Disorders (NORD)[7] vehemently oppose such laws. Other groups have also expressed concerns.

Interestingly enough, an important cancer survivor also has doubts about the pending federal right-to-try law. Scott Gottlieb, who serves as the current FDA Commissioner, testified before Congress, “Adequate policies and processes must be in place to appropriately balance individual patients’ needs for access to investigational therapies while recognizing the importance of maintaining a rigorous clinical trial paradigm for testing investigational products to demonstrate safety and efficacy.”[8]

As stated earlier, it is all about perspective. The ADAP Advocacy Association has not taken a position on this policy issue.

Related articles of potential interest:

• A compromise ‘right-to-try’ bill proceeds with help from FDA, but could be a hard sell, published on 02/05/18 in STAT
• Expanding access to unapproved treatments through right-to-try, published on 02/21/18 in STAT
• Trump's controversial new health care idea, published on 02/02/18 in Politico

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[1] Wikipedia, the free encyclopedia (2018, January 3). Right-to-try law. Retrieved from https://en.wikipedia.org/wiki/Right-to-try_law.
[2] Goldwater Institute (2017). Right to Try. Retrieved from http://righttotry.org.
[3] Nascimento, Nathan (2016, August 31). Right To Try: A Healthcare Reform That Can Save Lives. Forbes Magazine. Retrieved from https://www.forbes.com/sites/realspin/2016/08/31/right-to-try-a-healthcare-reform-that-can-save-lives/#1ee2adf44c67.
[4] Barnes, David (2018, February 15). Congressman Flores should press ‘Right to Try’ bill. Waco Tribune-Herald. Retrieved from http://www.wacotrib.com/opinion/columns/guest_columns/david-barnes-guest-columnist-congressman-flores-should-press-right-to/article_42dc32a4-4b2f-54d8-b0e5-b780fd31109d.html.
[5] Bateman-House, Alison, Lisa Kearns and Arthur Caplan (2018, February 1). Trump's 'Right To Try' Push Would Only Punish The Seriously Ill. Forbes Magazine. Retrieved from https://www.forbes.com/sites/alisonbatemanhouse/2018/02/01/why-right-to-try-is-fake-news/#2bb50a541265.
[6] Ballamy, Jann (2014, March 6). The illusions of “right to try” laws. Science-Based Medicine. Retrieved from https://sciencebasedmedicine.org/the-illusions-of-right-to-try-laws/.
[7] Mullen, Laura (2018, February 13). One-pager from NORD on Opposition to Right to Try Act.  National Organization for Rare Disorders. Retrieved from https://rarediseases.org/one-pager-nord-opposition-right-try-act/.
[8] Gottlieb, Scott (2017, October 3). Examining Patient Access to Investigational Drugs. Before the Subcommittee on Health, Committee on Energy and Commerce, US House of Representatives. Retrieved from https://www.fda.gov/NewsEvents/Testimony/ucm578634.htm.