Medical ethicist discusses Right-to-Try legislation

Photo: Marko Javorac via Flickr

Taken at face value, Right-to-Try (RTT) legislation sounds like a no-brainer, promising to improve access to treatments yet unapproved for marketing by the FDA for people with terminal illnesses. But it’s not that simple, according to Alison Bateman-House, Ph.D., M.P.H., M.A., an ethicist and assistant professor at the New York University School of Medicine.

In fact, many of its basic provisions make it unlikely to deliver on its promise. At a New York City Metro AHCJ Chapter meeting in November, she discussed reasons for her argument that RTT will only serve to limit, rather than expand, access to potentially life-saving treatments.

Federal legislation, ostensibly aiming to overcome legal challenges to 38 state RTT laws, was introduced in both houses of Congress earlier this year. The Senate version was passed in August via unanimous consent, in which the chamber agrees to not debate a bill. Championed by Rep. Andy Biggs (R-Ariz.), the House bill’s title reads, “To authorize the use of unapproved medical products by patients diagnosed with a terminal illness in accordance with State law, and for other purposes.”

Proponents of this legislation, led by Phoenix-based libertarian think tank the Goldwater Institute, blame the FDA for “killing patients” and use heart-wrenching patient stories to support their cause. Not surprisingly, families desperate to find help for their critically or terminally-ill loved ones have provided ammunition. This is especially true, says Bateman-House, of people suffering from ALS and supporters of easing regulations on widespread use of stem cells.

RTT opponents argue that the legislation does an end-run on regulatory protections and puts Americans at risk. By releasing health care providers and drug companies from their respective mandates to report adverse events (AE) to the FDA, serious complications, drug interactions, and even deaths attributed to investigational medical products could remain closely held secrets. Unlikely opponents to RTT include pharmaceutical, biotech, and medical device companies, who worry that lacking such information could lead to ineffective or dangerous products entering the market, only to be withdrawn after tragic outcomes for families — and, consequently, for their own marketing.

This provision is what differentiates RTT from “expanded access” or “compassionate use” rules, which do require AE reporting and do a good job of connecting needy patients with investigational drugs. So, is RTT even necessary? Does the American public really need protection from the FDA as the Goldwater Institute claims?

Bateman-House and her colleagues say absolutely not. In talking to stakeholders from patients to manufacturers, she said, the FDA is rarely named as the problem preventing patients from accessing investigational drugs. In fact, more than 99 percent of requests for compassionate use are approved by the FDA, within 24 hours in emergencies and within days for non-emergency requests. Whether the requested drugs help or not, is different story. And, the only requests that to the FDA for approval are ones that the drug companies have agreed to fill: hence, the chief obstacle to obtaining access to investigational drugs is at the company level.

RTT has been widely covered, but there are many angles yet untapped for health care journalists to pursue, including:

  • How might RTT affect physician’s freedom to prescribe marketed drugs off-label for patients with conditions that may benefit? Bateman-House suggests a colleague, Christopher Robertson, who is an expert in this area and may offer insights.
  • Why do the publicly funded patient groups (for example, the American Cancer Society, Friends of Cancer Research, NORD, the Isaac Foundation, Max Cure) opposing RTT feel so strongly about their opposition to such popular legislation that they are willing to take a public stand against it?
  • What is the upside of RTT? Bateman-House and other opponents of RTT credit the movement with raising awareness of expanded access and compassionate use programs for needy patients. For example, gov has added a function that allows users to search for drugs with compassionate use programs and the FDA streamlined its paperwork for single-patient requests.
  • How might a new project by Johnson & Johnson and New York University concerning compassionate use of investigative medicines help patients? Bateman-House admits bias, adding that she is personally involved in this effort. Its goal to use a non-industry, interdisciplinary panel to decide which patients’ requests for investigational drugs to grant and which to decline.

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