Right to Try and the FDA’s Expanded Access Program: No oversight? Or considered compassion.
Updated: Dec 29, 2019
"We need to fix this. But we do not need to undermine a working program that benefits patients by creating a deeply flawed alternative program.” ~ Alison Bateman-House, PhD, MPH; assistant professor of medical ethics at NYU Langone Health, and co-chair of the Working Group on Compassionate Use and Pre-Approval Access
If it were me or my loved one with a terminal illness, it would be difficult to objectively assess the “Right to Try” bill now in the U.S. House of Representatives. Fortunately, I am not experiencing such horrific pressure, and so was able to spend the afternoon somewhat unemotionally researching the topic. I say “somewhat” because the stories are heart-rending. Who wouldn’t want to offer some of these folks a chance at life?
The bill would allow patients with life-threatening conditions access to drugs that have not been approved by the FDA—in effect, circumventing FDA oversight. The U.S. Senate passed it in August 2017, and it is now in the House for consideration. In fact, 38 states already have similar laws, which were adopted due to support (eg, lobbying and donations) by organizations such as the Goldwater Institute*, which calls itself a “free-market public policy research and litigation organization,” and FreedomWorks, a nonprofit conservative advocacy group associated with the Tea Party movement. The rhetoric in favor of Right to Try is often overheated, as in this statement by FreedomWorks: “Right now there are millions of Americans lying in hospital beds, fighting for their lives. And Congressman Greg Walden [R–Oregon] isn't doing anything to help them!”1 Nice.
Opponents of the bill include 40 patient-advocacy groups (eg, American Lung Association,
Oncology Nursing Society),2 which sent a letter to Congress outlining their stance, and 330 physicians, medical ethicists, patients, lawyers, and others, who signed another letter.3 The former was drafted by Dr Alison Bateman-House and colleagues; she also made a statement to Congress on the matter in October 2017. In fact, the FDA already offers Expanded Access for just these purposes, and over 99% of requests are approved within days of receipt. In response to concerns about a lengthy application process, FDA simplified it, and it is now estimated to require about 45 minutes by the requesting physician. For emergency cases, treatment can be requested and authorized by telephone or other rapid means, and may start immediately upon FDA authorization—ie, within hours.
The Working Group on Compassionate Use and Pre-Approval Access, which Dr Bateman-House co-chairs, spent 3 years studying the FDA’s program and concluded that it is doing an excellent job of helping patients obtain access to experimental drugs. The group also discovered two key issues needing improvement: (1) widespread lack of knowledge about the program; and (2) widespread inaccurate beliefs about the program. The inaccuracies are so rife that Bateman-House, et al resort to labeling them “myths.” These myths include:
• The FDA can force companies to provide their investigational drugs.
• The FDA is slow handling requests for compassionate use.
• Pharmaceutical companies may be exposed to legal risks.
• If a patient has a serious problem or dies while taking the investigational drug, then eventual FDA approval if the drug is threatened.
Importantly, patients, families, and advocates often don’t know how to request an experimental drug from a company—for example, the request must be made by a physician. The Working Group recommends that the FDA be more proactive in educating industry, doctors, patient advocacy organizations, and patients.
The Goldwater Institute, in a fine example of misstatement and obfuscation, states that “the FDA drug approval process can take up to 15 years. That’s far too long for dying patients to wait.”4 It is the creation of the drug that can take up to 15 years, beginning with laboratory tests and proceeding to animal tests and then studies in humans. This is an entirely necessary process. Just as surgeons don’t agree to perform 15-hour operations in 2 hours to allow the patient to return home sooner, pharmaceutical companies do not, and cannot, make half-baked medicines available to the public.
As the Institute points out, “No one knows how many requests for compassionate use drug companies receive or reject. They are not required to keep or report that information.”5 This is worth exploring. Should companies be required to record requests? Michael B. Becker, writing for STAT, raises other important questions: Do patients undergoing Right-to-Try therapies lose their coverage for hospice? Are insurers absolved of any responsibility for covering additional medical expenses once a patient starts a drug under Right to Try? What if the experimental drug causes hospitalization—who pays for that?6
The answers to some of these questions are alarming. Bateman-House described the harm caused by 38 different state Right to Try laws: “In 19 states, patients using an investigational drug obtained via Right to Try can lose their hospice coverage, and 6 states say these patients may be denied coverage for home healthcare assistance.”7 In five states, Right-to-Try laws don’t apply to those ≤18 years old.
Experts assert that “best, safest way to speed access to drugs in development to the largest number of patients is through the clinical trial process.”7 In addition to strongly increasing FDA education about the Expanded Access program another route for action is expansion of access to clinical trials.
* Before he died, Senator Barry Goldwater protested the direction of the institute to which he’d lent his name. He didn't like that it was turning into a special-interest, big-business lobbying group.
1. Weixel N. Conservative group prods Walden over “right to try” bill. July 6, 2017. Accessed February 20, 2018 at http://thehill.com/policy/healthcare/medical-devices-and-prescription-drug-policy/340898-conservative-group-prods
2. Letter from patient advocacy groups, February 2018. Accessed February 20, 2018 at www.acscan.org/sites/default/files/National%20Documents/February%20Right%20to%20Try%20Coalition%20Letter%20-%20Final.pdf
3. Letter from 330 stakeholders, February 2018. Accessed February 20, 2018 via http://thehill.com/policy/healthcare/372440-hundreds-sign-on-to-letter-opposing-right-to-try-drug-bill
4. Goldwater Institute. About Right to Try. 2017. Accessed February 20, 2018 at http://righttotry.org/about-right-to-try/
5. Goldwater Institute. Dead on arrival: Federal “compassionate use” leaves little hope for dying patients. 2016. Accessed February 20, 2018 at http://righttotry.org/dead-on-arrival/
6. Becker MD. I’m the ideal person to support right to try. But I can’t — it’s a disaster in the making. STAT. February 1, 2018. Accessed at https://www.statnews.com/2018/02/01/right-to-try-cancer-fda/
7. Statement of Alison Bateman-House before the Health Subcommittee of the Committee on Energy and Commerce, U.S. House of Representatives. Examining patient access to investigational drugs. October 3, 2017. Available at http://docs.house.gov/meetings/IF/IF14/20171003/106461/HHRG-115-IF14-Wstate-Bateman-HouseA-20171003.pdf