"Right to Try" Legislation: A Developing Legal Issue Related to Medications

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Pharmacy Times, July 2016 Digestive Health, Volume 82, Issue 7

Individuals working in a health care field are often expected to have up-to-date information about the latest trends in laws and legislative changes related to their specialty.

Individuals working in a health care field are often expected to have up-to-date information about the latest trends in laws and legislative changes related to their specialty. Questions can come from colleagues who serve patients, from patients themselves, or from patients’ relatives or other caregivers. This article presents background, context, and the pros and cons about the current legislative proposals known as Right to Try.

Right to Try laws are legislation enacted at the state level with the goal of helping terminally ill patients have access to investigational drugs that may be lifesaving.1 Currently, the FDA has policies and procedures in place to make sure the drugs being approved are safe and effective. This process starts with the drug being developed and animal-tested, and an investigational new drug application being submitted. Then, phase 1 of clinical testing in humans takes place, using 20 to 80 healthy volunteers. If the drug appears safe, phase 2 studies assess the efficacy of the drug by including hundreds of patients with the disease/condition. If the drug is effective, phase 3 trials study the drug in thousands of patients.2 The FDA then reviews the data accumulated from all studies and either approves the drug for sale in interstate commerce or rejects it. This process can take years.

If a patient is terminally ill, and a medication that could be lifesaving is currently in an FDA clinical trial, the patient may be able to join the clinical trial. If patients do not qualify to join a clinical trial, they may not have the opportunity to try a drug and may not live long enough to see the drug get through the FDA review and approval process and subsequently reach the market.

The Right to Try movement is trying to help terminally ill patients obtain drugs currently in clinical trials. The movement is supported by the Goldwater Institute, a conservative Arizona-based organization.3 The Institute has drafted proposed state legislation to facilitate terminally ill patients’ access to investigational drugs that have secured phase 1 FDA approval.3 Many events led to the Goldwater Institute’s movement to draft legislation for states to consider. Starting in the late 1980s, the HIV and AIDS crisis led to a demand for the FDA to quickly approve drugs for the epidemic. The HIV medication AZT was approved in 1987 by the FDA without a phase 3 clinical trial.4 The FDA then created the Federal Expanded Access Program, now referred to as the Compassionate Use Program, which was designed to facilitate access to investigational therapies.5

Abigail Burroughs was very influential in the Right to Try movement. In 2001, as a 21-year-old who had cancer, she had tried all conventional treatment options.6 Her physician suggested cetuximab, an epidermal growth factor receptor—targeted treatment that was still in clinical trials. Abigail did not qualify for the clinical trials and was unable to get access to the drugs through any other channels. She passed away in 2001.

In response, the Abigail Alliance was created in November 2001, with the purpose of “promoting creative ideas at the FDA and on Capitol Hill, getting information out to the public, and directing cancer patients to reliable sources for further information.” 6 On May 2, 2006, Abigail Alliance for Better Access to Developmental Drugs v Von Eschenbach was heard in the US Court of Appeals for the District of Columbia Circuit by a 3-judge panel. The panel decided by a 2-to-1 margin in favor of the Abigail Alliance, stating that terminally ill patients who have exhausted all other drug treatments have the Constitutional right to try experimental treatments. A little over a year later, the FDA requested a re-hearing of the matter by the full appellate court. The decision following the re-hearing was 8 to 2 in favor of the FDA, thereby reversing the prior panel’s decision. In 2008, the US Supreme Court declined to hear Abigail Alliance for Better Access to Developmental Drugs v Von Eschenbach, meaning that the decision of the lower court was final.7 This final court decision led the FDA to expand the Compassionate Use Program.8 This program, expanded in 2009, allows patients to ask the FDA for access to a drug in clinical trials.5

Stories similar to Abigail’s have helped the Right to Try legislation gain popularity. Currently, 24 states have enacted similar legislation and 17 states are currently seeking approval; California is the only state where a bill passed by the legislature was vetoed by the governor. The topic is still discussed frequently. In March 2016, Iowa advanced its Right to Try bill to the state senate floor.9 The legislature in Maine enacted a Right to Try bill and the governor has signed it.10 Georgia has also enacted a Right to Try law.11

The Table lists the supporting and opposing viewpoints regarding Right to Try. In the eyes of those supporting the legislation, the FDA’s Compassionate Use Program requires completion of a lengthy document, is a slow process, and, even with approval, still presents barriers to access. From the perspective of these individuals, supporting state Right to Try legislation will allow for better health outcomes and will save lives. Right to Try laws give terminally ill patients the opportunity to use investigational drugs because it is their personal right.12 They also give patients another option when they have exhausted conventional options and do not qualify for a clinical trial.13 However, even if approval is secured through the clinical trial program, individuals who are terminally ill say investigational medications are not received in a timely manner.14 Right to Try only allows access to medications that have passed the basic safety testing of a phase 1 study.15 The laws are worded to include legal protection for the referring prescriber from licensing board action.16 An additional argument for enactment of these laws is that allowing more patients to access drugs provides more information about the drugs before they are approved.

On the other hand, those who oppose the legislation argue that the bills could worsen health outcomes. If phase 1 trial drugs are viewed as unproven treatment modalities, expanding their use beyond clinical trials is a concern for individuals, prescribers, and the FDA. More harm than good could come from the use of these drugs. Efficacy has not been established at this stage of the process because phase 1 trials include just a small group of healthy individuals and are designed to assess the safety of use within the group.17 The potential for medication interactions is unknown and untested. Elizabeth Weeks Leonard, a professor of health law at the University of Georgia, outlines the opposition, saying, “There are several assumptions that would be required for the Right to Try laws to be effective: drug companies would willingly provide their nonapproved products to dying patients, patients would willingly pay for the drugs, and doctors would willingly prescribe the drugs.”8

Another argument against Right to Try pertains to the question of where to draw the line between patients who are terminally ill and those who are chronically ill. Once terminally ill patients have access to unproven drugs, will chronically ill patients also request them because they have a right to their own decision making? Individuals opposing the legislation believe that allowing unproven drugs to be used will negatively affect the drug companies and the FDA-approval process. Why would someone join a drug company’s trial and risk getting the placebo? In addition, the FDA’s system could be damaged by Right to Try laws.8 If everyone had access to the real drug, the FDA would have no information for determining whether a drug should be approved for marketing. Those opposing the legislation also say that the FDA’s Compassionate Use application takes only about 45 minutes to complete. During the 2012 fiscal year, 974 of the 977 Compassionate Use requests were granted.18 This program allows the FDA to control the drug product approval process while allowing terminally ill patients to have access to investigational drugs.19

Irrespective of the positions on Right to Try, there are unanswered questions. Both sides of the issue pose good supporting arguments, but the problem boils down to how terminally ill patients will get access to medications. Whether it is through new FDA approval processes or individual state laws, the focus should be on trying to change laws to help terminally ill patients receive available treatment in a timely manner. Although legislative action has been taken on a state-by-state basis, the House of Representatives was presented with the Right to Try Act of 2015, sponsored by Rep. Matt Salmon of Arizona. In July 2015, the legislation was referred to the Subcommittee on Crime, Terrorism, Homeland Security, and Investigations.20 Those developments deserve monitoring. Overall, health care for the terminally ill could greatly change in the next few years.

Erica Krantz is a fourth professional year student, and Joseph L. Fink III, BSPharm, JD, is a professor of pharmacy law and policy in the Department of Pharmacy Practice and Science at the University of Kentucky College of Pharmacy.

References

  • About Right to Try. Right to Try website. righttotry.org/about-right-to-try. Accessed December 20, 2015.
  • US Food and Drug Administration drug approval process. FDA website. www.fda.gov/downloads/Drugs/ResourcesForYou/Consumers/UCM284393.pdf. Accessed May 17, 2016.
  • Monir M. States move to give terminally ill 'right-to-try' drugs. USA Today website. usatoday.com/story/news/nation/2015 /02/19/right-to-try-state-legislation-terminally-ill-patients/23667229/. Published February 19, 2016. Accessed December 22, 2015.
  • HIV/AIDS historical time line 1981-1990. FDA website. www.fda.gov/ForPatients/Illness/HIVAIDS/History/ucm151074.htm. Updated August 8, 2014. Accessed May 17, 2016.
  • Expanded access (Compassionate Use). FDA website. www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/. Updated February 19, 2016. Accessed May 17, 2016.
  • Our story. Abigail Alliance website. abigail-alliance.org/story.php. Accessed May 17, 2016.
  • Abigail Alliance for Better Access to Developmental Drugs v Von Eschenbach, 495 F3d 695 (DC Cir 2008); cert denied, 552 US 1159 (2008).
  • Leonard E. Right to experimental treatment: FDA new drug approval, Constitutional rights, and the public's health. J Law Med Ethics. 2009;37(2):269-279. doi: 10.1111/j.1748-720X.2009.00371.x.
  • Petroski W. ‘Right to Try’ drug bill advances to Iowa Senate floor. The Des Moines Register website. www.desmoinesregister.com/story/news/politics/2016/02/15/right-try-drug-bill-advances-iowa-senate-floor/80415376/. Published February 15, 2016. Accessed May 17, 2016.
  • Maine Senate votes to support ‘Right to Try’ bill [press release]. Augusta, ME: WCSH6 Portland; March 24, 2016. wcsh6.com/news/politics/maine-senate-votes-to-support-right-to-try-bill/98405486. Accessed May 17, 2016.
  • 2015-2016 Regular Session — HB 34 "Georgia Right to Try Act"; enact. Georgia General Assembly website. legis.ga.gov/Legislation/en-US/display/20152016/HB/34. Accessed May 17, 2016.
  • SB139 (15RS). Kentucky Legislature website. lrc.ky.gov/record/15RS/SB139.htm. Accessed December 18, 2015.
  • Olsen D. Dying should have 'Right to Try' new treatments. Time magazine website. http://time.com/4091290/right-to-try/. Published November 10, 2015. Accessed December 29, 2015.
  • Dresser R. “Right to Try” laws: the gap between experts and advocates. Hastings Cent Rep. 2015;45(3):9-10. doi: 10.1002/hast.442.
  • Right to Try model legislation. khi.org/assets/uploads/news/13359/goldwater_institute_right_to_try_model_legislation.pdf. Accessed January 1, 2016.
  • SB139. Kentucky Legislature website. lrc.ky.gov/record/15RS/SB139/bill.pdf. Accessed December 17, 2015.
  • Rubin R. Experts critical of America's right-to-try drug laws. Lancet. 2015;386(10001):1325-1326. doi: 10.1016/S0140-6736(15)00393-1.
  • Tsakopoulos A, Han J, Nodler N, Russo V. The Right to Try: an overview of efforts to obtain expedited access to unapproved treatment for the terminally ill. Food Drug Law J. 2015;70(4):617-641.
  • Bateman-House A, Kimberly L, Redman B, Dubler N, Caplan A. Right-to-Try laws: hope, hype, and unintended consequences. Ann Int Med. 2015;163(10):796-797. doi: 10.7326/M15-0148.
  • HR3012 - 114th Congress (2015-2016): Right to Try Act of 2015 (July 15, 2015).