US Pharm. 2018;43(7):31-32.
On May 30, 2018, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 was signed into law.1 The federal Right to Try Act was intended to authorize the use of unapproved medical products by patients diagnosed with a terminal illness in accordance with state law and for other purposes.2
Laws addressing a terminally ill patient’s right to try have been passed in a number of states. As of the writing of this column, right-to-try statutes have been enacted in Alabama, Arizona, Arkansas, California, Colorado, Connecticut, Florida, Georgia, Idaho, Illinois, Indiana, Iowa, Kentucky, Louisiana, Maine, Maryland, Michigan, Minnesota, Mississippi, Missouri, Montana, Nebraska, Nevada, New Hampshire, North Carolina, North Dakota, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, Washington, West Virginia, Wisconsin, and Wyoming. Other right-to-try bills have been introduced in a number of other states.3
The federal Right to Try Act is intended to provide an alternative pathway to the expanded-access policies of the FDA and allows patients to work directly with pharmaceutical sponsors without direct FDA oversight.4 The Right to Try Act’s author, Senator Ron Johnson (R-WI), stated in a letter to the FDA dated May 31, 2018, that the Act “is not meant to grant FDA more power or enable the FDA to write new guidance, rules, or regulations that would limit the ability of an individual facing a life-threatening disease from accessing treatments.”5
Identifying Eligible Patients
The federal Right to Try Act requires that both the patient and the investigational drug satisfy certain criteria before the patient may be given access to the medication. Under the Right to Try Act, an eligible patient is: a patient who has been diagnosed with a life-threatening disease or condition; a patient who has exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug, as certified by a physician; and a patient who has provided to the treating physician a written informed consent regarding the eligible investigational drug.6-8
Defining Investigational Drugs
The Act defines an eligible investigational drug as an investigational drug9:
• For which a phase I clinical trial has been completed to determine whether the drug is safe10;
• That has not been approved or licensed for any use under Section 505 of the Federal Food, Drug, and Cosmetic Act (FDCA) or Section 351 of the Public Health Service Act (PHSA);
• For which a New Drug Application or a Biologics License Application has been filed under the FDCA or the PHSA, respectively; or that is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of approval or licensure or is the subject of an active Investigational New Drug (IND) application; and
• The active development or production of which is ongoing and has not been discontinued by the manufacturer or placed on clinical hold.
Eligible investigational drugs are exempt from certain requirements under the FDCA and the PHSA. However, the sponsor must comply with other requirements under those laws, such as labeling requirements applicable to INDs.11 In addition, the sponsor may not represent in a promotional context that an IND is safe or effective for the purposes for which it is under investigation or otherwise promote the drug.12 Finally, the sponsor may recover only the direct costs of making its investigational drug available. Direct costs are costs incurred by a sponsor that can be specifically and exclusively attributed to providing the drug for the investigational use for which FDA has authorized cost recovery.13
Reporting Investigational-Drug Use
The manufacturer or sponsor of an eligible investigational drug must submit to the FDA Secretary an annual summary of the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events. The Secretary, in turn, is required to post an annual report of the use on the FDA’s website. As a result, pharmacists, patients, and their caregivers should be aware that certain information on the uses of eligible investigational drugs will become publicly available.14
The Act’s Limited Liability
Under the Act, there is no liability against the sponsor or manufacturer regarding an eligible investigational drug provided to an eligible patient in compliance with the Act. There is also no such liability against a prescriber, dispenser (e.g., pharmacist), or other individual entity (other than a sponsor or manufacturer) unless the relevant conduct constitutes reckless or willful misconduct, gross negligence, or an intentional tort under applicable state law. In addition, there is no liability against any sponsor, manufacturer, prescriber, dispenser, or other individual entity who decides not to provide access to an eligible investigational drug under the Right to Try Act.15 As a result, pharmaceutical companies, physicians, pharmacists, and others have the discretion to determine whether to participate in any program permitted under the Right to Try Act.16
No New Mandate
Finally, pharmacists should be aware that the Right to Try Act does not establish a new entitlement, modify an existing one, or otherwise establish a positive right to any person or establish a new mandate. As a result, pharmacists and patients should be aware that there is no requirement for commercial or government payors to provide a funded benefit for any eligible investigational drug or related services.17
How the pharmaceutical industry will respond to the Act remains to be seen. At least one pharmaceutical manufacturer is reported to have expressed ambivalence about the law because of the potential to reduce enrollment in clinical trials.18
1. Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017. S.204. 115th Cong (2017-2018).
3. Right to try in your state. http://righttotry.org/in-your-state/. Accessed June 11, 2018.
4. S.204, §3. See also: Expanded access (sometimes called “compassionate use”). FDA. www.fda.gov/NewsEvents/PublicHealthFocus/Expanded AccessCompassionateUse/default.htm. Accessed June 11, 2018.
5. U.S. Senate Committee on Homeland Security & Governmental Affairs. Johnson to FDA: agency should comply with Right to Try law. May 31, 2018. www.hsgac.senate.gov/media/majority-media/johnson-to-fda-agency-should-comply-with-right-to-try-law. Accessed June 11, 2018.
6. 21 CFR §312.81. S.204, §2(a).
7. Id. The physician 1) must be in good standing with the physician’s licensing organization or board, and 2) must not be compensated directly by the manufacturer for certifying that the patient has exhausted approved treatment options and is unable to participate in a requisite clinical trial.
8. Id. The Right to Try Act does not specify the content requirements of a patient’s informed consent, but practitioners may wish to be aware that the FDA has previously issued guidance on informed consent requirements at 21 CFR §50.20, et seq.
9. For purposes of the Right to Try Act, an “investigational drug” is defined in Section 561 of the Federal Food, Drug, and Cosmetic Act. See S.204, §2(a), for complete definition of “eligible investigational drug.”
10. Phase 1 trial means a phase 1 clinical investigation of a drug as described in 21 CFR §312.21 (or any successor regulations).
11. 21 CFR §312.6.
12. 21 CFR §312.7.
13. 21 CFR §312.8(d)(1).
14. S.204, §2(a).
15. S.204, §2(b).
16. Reddy S. The ‘Right to Try’ law says yes, the drug company says no. Wall Street Journal. Updated June 6, 2018. www.wsj.com/articles/family-battling-a-rare-disease-sees-roadblocks-despite-right-to-try-law-1528293923. Accessed June 11, 2018.
17. S.204, §3.
18. Dearment A. Gottlieb at BIO: FDA developing processes around right-to-try law. MedCityNews. June 7, 2018. https://medcitynews.com/2018/06/gottlieb-at-bio-fda-developing-processes-around-right-to-try-law/?rf=1. Accessed June 11, 2018.
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