HOPA Annual Conference 2019

The Right to Try Investigational Anticancer Therapies

There exist several barriers to access investigational cancer therapies outside of clinical trials, and many of these barriers have gone unaddressed, according to a review presented by R. Donald Harvey, PharmD, BCOP, FCCP, FHOPA, Associate Professor and Director at the Emory Winship Cancer Institute.

The FDA oversees all aspects of expanded access to investigational therapies, and by definition, expanded access describes “a potential pathway for a patient an immediately life threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.” However, the presentation notes that public perception is that the FDA hinders and delays access to many of these expanded therapies. The passage of the Compassionate Use Act in 2009 made available agents that were previously accessible only through clinical trials. The benefits of this act have lessened reporting requirements as these investigational agents requests no longer have to meet stringent use criteria.

Types of expanded access requests include individual patient investigational new drug (IND) application, intermediate-size population IND, individual patient protocol, emergency use individual patient protocol, intermediate-size population protocol, treatment protocol, and emergency use individual patient IND. With respect to the latter, to obtain an individual patient IND (non-emergency) must first request a letter of authorization (LOA) from the product developer and submit a special FDA form in conjunction with that LOA via certified mail.

Potential barriers to single patient IND submissions may involve industry contacts who are unwilling to provide expanded therapies due to risk (and the industry is under no obligation to provide these agents), and a protracted physician request process which includes not only making a formal request, but completing paperwork, submitting to an institutional review board (IRB), and making documentations before the therapy can be procured and dispensed.

To provide legislative support to terminal cancer patients in need of investigational therapies, the Right to Try legislative movement, which began in 2013 with backing from the Goldwater Institute, has attempted to facilitate easier access to these potentially beneficial experimental agents. This movement received executive support from President Trump in 2018. However, proponents note barriers, such as insurance coverage being jeopardized if investigational drug complications arise, and the possible decrease of safety and efficacy from reduced drug vetting.  The presentation further noted that in 2017, then FDA commissioner Scott Gottlieb was quoted as saying that the “most common obstacle to access the investigational product is the willingness or ability of companies to provide it.”

Harvey R. The Right to Try Investigational Anticancer Therapies Presented at the Hematology/Oncology Pharmacy Association Annual Conference; April 3-6, 2019; Fort Worth, TX.

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