This article, published online in JAMA Oncology,1 presented a viewpoint regarding the potential impact of the recently-passed U.S. Federal “Right to Try” Act2 on the timely and effective delivery of palliative care in the setting of pediatric cancer.

The Right to Try Act, which became Federal law in the United States in June 2018, “amends the Federal Food, Drug, and Cosmetic Act to exempt, from specified requirements and restrictions under that Act and other laws, the provision of certain unapproved, investigational drugs to a terminally ill patient who has exhausted approved treatment options and is unable to participate in a clinical trial involving the drugs.”2 It differs from the US Food & Drug Administration (FDA) Expanded Access program3 in that the Right to Try Act is not under the regulatory jurisdiction of the FDA; instead, investigational drugs need only have completed phase 1 testing and be under active development.4

In this opinion piece, the authors expressed concerns regarding “the potential unintended negative consequences” of the Right to Try Act with respect to the delivery of palliative care to children with cancer.1

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In particular, the authors pointed out that hospice or other insurance benefits might be compromised for children under active investigational treatment. However, they mentioned that this risk is likely to be greater for adults, as many states allow for concurrent hospice care and investigational therapy in the pediatric setting.

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The main concern expressed in this article was that Right to Try may exacerbate existing deficiencies regarding the successful integration of palliative care throughout the cancer care continuum, including end-of-life care, for pediatric patients with cancer.

“Although the necessary supporting evidence has not been generated, to our knowledge, it seems reasonable to be concerned that a general increase in cure-seeking behavior would likely exacerbate rather than improve the timeliness of palliative care referrals in pediatric oncology,”1 the authors noted.

Conversely, the authors also mentioned that the Right to Try Act “might offer a high-profile opportunity to promote improved integrative clinical practice.”1

The authors concluded that research is needed to answer this question, as well as whether access to investigational agents is more efficient and equitable under the Right to Try Act compared with the FDA Expanded Access Program; whether children with cancer are living longer lives with improved quality of life as a result of access of investigational drugs made available through the Right to Try Act, and whether children are actively involved in the decision-making process regarding whether to receive an investigational agent under the law.


1. Lanzel AF, Lavery JV. Unintended consequences of the Right to Try Act for palliative care in pediatric oncology [published online March 7, 2019]. JAMA Oncol. doi: 10.1001/jamaoncol.2018.7201

2. Right to Try Act. Pub L No. 115-176. Accessed March 15, 2019.

3. US Food & Drug Administration. Expanded Access. Updated February 27, 2019. Accessed March 13, 2019.

4. Lynch HF, Zettler PJ, Sarpatwari A. Promoting patient interests in implementing the Federal Right to Try Act. JAMA. 2018;320(9):869-870. doi: 10.1001/jama.2018.9880