Right to Try Legislation Further Muddles Expanded Access Issues for Experimental Treatments

Beth E. Roxland, JD, MBioethics

Beth E. Roxland, JD, MBioethics

Legislation at the federal level to advance “right to try” (RTT) options for patients seeking expanded access to experimental medical treatments has created more problems than it solves, Beth E. Roxland, JD, MBioethics, told attendees of her session, “On the Soapbox: Right to Try,” at the DIA 2018 Global Meeting in Boston yesterday (June 25).

The new legislation “effectively alters jurisdiction and oversight rules,” Roxland says. “It adds—rather than subtracts—confusion and burdens.”

Broadly speaking, RTT is a state and/or federal initiative to empower patients to take control of their healthcare by accessing experimental drugs or devices that are still in clinical testing, Roxland says.

Expanded access is the use of an investigational treatment outside a clinical trial on a patient with a serious disease or condition who does not have comparable or satisfactory alternative therapies to treat the disease or condition. The primary “intent is clearly treatment,” Roxland says, rather than research. It’s also a misfire in that it doesn’t focus on the main players, she says; the law doesn’t mandate access be granted by sponsors, who are the primary decision-makers.

Two common misconceptions are clouding the waters, Roxland says. “Sponsors [and not the U.S. Food and Drug Administration] are the primary decision-makers” on granting use of their experimental treatments in preapproval conditions, and right to try legislation in and of itself “does not grant patients an actual right to try.”

Patients can’t apply for such access; the request must come from the sponsor, a physician investigator, or a qualified treating physician, either for a single patient or a small group of up to 100 patients.

Instead, the federal legislation “cuts a small hole in an otherwise cohesive regime that aims at efficiently getting effective treatments to the majority of patients in need,” Roxland says.

Further, there is no requirement that companies make or provide the product being requested, nor do insurers have to pay for the product.

There are a number of forces at work—some scientific-based, others reflective of a more visceral personal experience—driving an increasing focus on compassionate use and expanded access, Roxland says. At the top of the list, “situations are tragic, personal, and very understandable,” she says. In addition, there’s been a shift from “paternalistic” decisions made on behalf of patients to patient autonomy.

At the same time, there is more and more personal willingness and acceptability of taking on high or unknown risks.

Other factors propelling demands for expanded access and compassionate use:

  • Strong patient advocacy group mobilization and communications
  • Greater social and traditional media savviness to tell personal stories and reach large audiences
  • Societal movements to limit government oversight and bureaucracy and an emphasis on individual rights

Roxland also cited the media’s failure to cover every angle of the issue. For example, there’s been little media or public discussion of:

  • The effectiveness of, and processes for, current expanded access and compassionate use
  • The challenges of, and competing ethical grounds for, granting or withholding individual preapproval access
  • Any examples of negative health impact on patients who are granted early access, but are not helped or are affirmatively harmed

Author: Michael Causey