Straightening out the set record on ‘Right to Try’

The Hill recently ran an article by Naomi Lopez Bauman, of the Goldwater Institute, called “Setting the Record Straight on Right to Try.” In her commentary, Lopez Bauman mounted a defense of right to try legislation, which purports to let terminally ill patients access drugs before they’ve received FDA approval. Although neither her arguments nor her targets are new, the level of invective directed at those targets is. As a frequent critic of right to try, and thus presumably one of her targets, I was moved to draft a rebuttal of some of the most egregious accusations.

1. “As political support and momentum for Right to Try legislation increases, some opponents are resorting to scare tactics in a desperate attempt to keep Washington bureaucrats firmly in charge of who gets access to potentially life-saving treatments.”

Critics agree that the ethical shortcomings of right to try laws are indeed scary, but we don’t consider pointing out well-founded concerns about patient safety to be a “tactic.” Neither is the aim of showing how the laws can harm patients, especially those facing the direst prognoses.

2. “Opponents are falsely claiming that terminal patients accessing treatments under Right to Try will be stranded without access to hospice care should they need it. This is simply not true.”

From the Goldwater Institute’s model right to try bill, Sec. 1, part 2, sec. vi, with identical or similar language included in 17 of the state bills: Informed consent document must contain “A statement that the patient’s eligibility for hospice care may be withdrawn if the patient begins curative treatment with the investigational drug, biological product, or device and that care may be reinstated if this treatment ends and the patient meets hospice eligibility requirements.”

3. “Eligibility for hospice care is largely set by the Center for Medicaid Services and neither the state nor proposed federal Right to Try laws would change this in the slightest. Eligibility for hospice care will still be determined by the payer for the care, or the non-profit organization providing the services.”

And expanded access is run by the FDA. But Goldwater, in its obsession with the FDA, ignores the key issue concerning hospice access. Why not go after CMS with equal gusto and allow hospice care for right to try patients?

4. “In fact, the only mention of hospice in any of the 50 state Right to Try laws … is to ensure patients understand that if they are receiving hospice services but begin to seek curative treatment under Right to Try, their insurance company may determine they cannot also receive hospice care at the same time. And that makes sense. If a person is seeking curative treatment, then they are not ready for hospice care. Patients can go in and out of hospice care without penalty and this law does nothing to change that.”

First, there’s some fancy linguistic footwork going on here with “curative treatment”: The drugs being sought under right to try are experimental; they cannot be said to “cure” anything.

Second, canceling or denying hospice care to a patient trying a drug under right to try does not make sense (unless you’re an insurance company). There’s big difference between deciding to halt or forgo active treatment of an approved drug (which is the usual scenario for entering hospice) and trying an unapproved drug, after exhausting other treatment options (as fancifully might occur under Right to Try). It is morally unconscionable to threaten terminally ill patients with loss of their hospice care under laws designed to restore their freedom to try to save their lives. If this legislation were really patient-centric, it would strive to do everything to help patients while they take the drugs that right to try advocates are working so hard for them to obtain.

Finally, Bauman suggests a nonsensical yet nonetheless very troubling scenario in her defense: A terminally ill patient, in hospice care, decides to try an experimental agent; per all right to try bills, that could be something that has passed just Phase 1 testing. So the dying patient tries an agent with very little safety data and zero efficacy — and has to leave hospice care to do so. (Let’s hope the patient doesn’t live in Connecticut, North Dakota, Oklahoma, or West Virginia, which don’t let hospitalized inpatients request drugs under right to try!)

5. “This latest ‘alternative fact’ about Right to Try really deflects from the central issue: Should terminally patients have to seek permission from Washington bureaucrats in order to try to save their own lives?”

(“Alternative fact”? Really? You’re hitting critics of major legislative reform with a term invented to defend the president’s lies about inauguration crowds?) Terminally ill patients don’t need permission from Washington bureaucrats; they need permission from the company that’s making the drug being sought. Sorry, but that’s just the way it is, and no amount of saying otherwise changes the fact that companies, not the FDA, control access to their drugs.

6. “Right to Try is now law in 33 states and under consideration in the other 17, dozens of patients are being treated under the law, and, based on evidence from overseas, making early access to treatments for terminal patients the rule, rather than the exception, is both possible and desirable.”

And yet neither Goldwater nor anyone else can substantiate the claim about those dozens of patients receiving a drug under right to try that they couldn’t have accessed through the FDA’s Expanded Access Program. After nearly three years since the first law was passed with no one getting access to anything they could not have otherwise received, it remains inhumane to dangle these hollow laws in front of very sick patients.

7. “Yet the FDA’s compassionate use program, which is supposed to give patients access to investigational treatments, granted fewer than 1,300 requests in 2015. And even though the FDA did approve almost all of the requests they received, that means, at best, fewer than one-tenth of one percent of terminal patients suffering from one of these diseases is getting early access to a potentially life-saving treatment.”

One more time: The FDA can only approve requests it receives from companies. It cannot force companies to forward requests to it. So why isn’t Goldwater haranguing industry for agreeing to approve just a tenth of a percent of the requests it receives? To repeat the pathway once again: Physician requests drug from manufacturer, manufacturer says yes or no and then contacts FDA.

8. “Right to Try legislation offers a new path to addressing this problem.”

The legislation does nothing of the sort. From the Goldwater model bill, and echoed in every right to try law and bill: “This act does not require that a manufacturer make available an investigational drug, biological product, or device to an eligible patient.”

Goldwater is a political organization whose goal is reducing government regulation. Unfortunately, the regulations it has in its sights with right to try are ones that were created with the welfare of patients, and the safety and efficacy of the drugs they take, as driving the regulatory mission. You want to help patients? Work to get manufacturers to up that “one-tenth of one percent” figure instead of wasting time repeating the same myths and falsehoods over and over and over again.

Lisa Kearns, MS, is a senior research associate at the NYU School of Medicine’s Division of Medical Ethics and a member of its Working Group on Compassionate Use and Pre-Approval Access.

The views expressed by this author are their own and are not the views of The Hill.