First Patient Treated Under Right to Try Law

Regulatory NewsRegulatory News | 09 January 2019 |  By 

A patient diagnosed with an aggressive form of brain cancer became the first person in the US to access an experimental treatment under the Right to Try law of 2017. The law has been touted as a new way to help people with terminal illnesses and few other therapeutic options, although until now, no patients had ever used the law.

ERC-USA and the University of California, Irvine initiated treatment with the company’s investigational compound ERC-1671, also known as Gliovac in Europe, and which is in Phase 2 clinical trials in the US. The patient’s treatment with ERC-1671 began at the university in late November 2018.

The patient resorted to Right to Try after failing to qualify for enrollment in the ongoing trial. But rather than work around the agency that may eventually approve the experimental treatment, the company said in August that it informed the US Food and Drug Administration (FDA) that it intended to make the experimental treatment available to this one patient. FDA acknowledged acceptance of the company’s notification on 13 July, ERC said.

As far as why the company used Right to Try rather than FDA's expanded access, ERC told Focus that time was of the essence and Right to Try "had a lower paperwork requirement and likely faster implementation."

The law has proven to be a thorn in the side of FDA, which has said it will work to implement it in a manner consistent with congressional intent and with FDA's public health mission, but the agency still has its own process for helping terminally ill patients receive experimental treatments, known as expanded access. FDA grants about 99% of the expanded access requests it receives.

Holly Fernandez Lynch, a professor of medical ethics at the University of Pennsylvania, explained to Focus that the company's use of the Right to Try law in this case "seems totally unnecessary and unhelpful" as FDA likely would have accepted this application through its expanded access pathway.

"If you use Right to Try when expanded access is available, it muddies the waters for patients and gives the impression FDA is a barrier when it isn't," she added. It's short-sighted of the company to try to undercut FDA's role in the process.

Other companies, including Therapeutics Solutions International, have promotionally announced their intentions to use Right to Try, though according to Goldwater Institute, no other patients have yet to use the new law.

According to BioCentury, FDA’s Oncology Center of Excellence is also working on a new initiative, known as Project Facilitate, under which the agency will provide a telephone number that patients and physicians seeking expanded access to an experimental treatment can call. FDA staff will answer calls and fill out the form required to apply for a single-patient IND request. The paperwork will be forwarded to the manufacturer. A pilot version of this initiative is expected to launch in the first half of 2019.


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