Right to Try Must Include Responsibility to Report Results


February 22, 2019

This transcript has been edited for clarity.

Hello and welcome. I'm Dr George Lundberg and this is At Large at Medscape.

Many of us have been saying for a long time that people should take charge of their health. After all, it is their health. Once the Internet came alive a couple of decades ago, it became much easier to actually do so.

What about safety from all sorts of harmful information, practices, and products? That is a problem. It ranges from fake products and contaminated drugs to erroneous information, all presented in very convincing ways. The normal defense is caveat emptor (let the buyer beware), caveat lector (let the reader beware), or caveat aeger (let the patient beware).

In our country, we have state and federal licensing agencies to protect us from dangerous hospitals and dangerous doctors. We have the US Food and Drug Administration (FDA) to protect us against unsafe or ineffective drugs and devices.

But what if I have a really bad cancer that has spread and is unresponsive to all approved therapies that have been tried? May I declare myself a human "guinea pig" and take whatever I can find and afford that might give me a chance? The libertarian in me says, "Absolutely," but the physician in me says, "Not so fast."

In this burgeoning era of personalized, precision medicine, with so many new targeted, immune, and investigational cancer drugs, why not—after fully informed consent—give it a try?

This debate raged between patient autonomy and physician-legal regulation at a state and federal level for years until the US Congress enacted Senate Bill 204 - Right to Try, which was signed into law in May 2018. It is applicable to those drugs that have passed safety phase 1 and are deemed safe enough to be used in efficacy clinical trials in phase 2. Many states (more than 40) had already enacted similar laws.

We learned in January 2019 about the first patient reported to have used the federal Right to Try legislation. This was a patient with aggressive brain cancer being treated at UC Irvine with experimental drug ERC-1671 (called Gliovac in Europe).

The new law has not been welcomed by the FDA, in part because the FDA already has a process in place called "expanded access." This program is applicable to similar patients and is very active, with what is described as 99% approval for patients who go through the expanded-access protocol.

The FDA Oncology Center of Excellence is said to be developing an easier approach to help patients and physicians achieve approval for a single-patient IND (investigational new drug) to use via their existing FDA protocol.

As a molecular pathologist who praised the clinical trial with an N of 1 design as far back as 2010, I am very pleased by the progressive actions of states and this iteration of the FDA under Scott Gottlieb's imaginative, patient-centered leadership. My biggest remaining concern is that the knowledge gained by each such case may be lost.

The Right to Try must be accompanied by the responsibility to report the results to the company, the FDA, the medical profession, and the general public via open-access journals that publish case studies (such as Cureus) via social media (Facebook or Twitter), or in a blog format (such as Curious Dr. George, hosted by Cancer Commons).

Expanded access and Right to Try represent progress. They could even be scientifically important as we use more rapid access to new drugs and new data to transition us into rapid learning communities.

That's my opinion. I'm Dr George Lundberg, at large for Medscape.

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