HemOnc Today's PharmAnalysis

HemOnc Today's PharmAnalysis

Disclosures: Bota reports a speakers bureau role with Novocure. Unguru reports no relevant financial disclosures.
March 10, 2020
3 min read

Do examples of achieving remission with a drug obtained via Right to Try justify the law’s existence?

Disclosures: Bota reports a speakers bureau role with Novocure. Unguru reports no relevant financial disclosures.
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Click here to read the Cover Story, “Right to Try: A ‘well-intentioned’ but ‘misguided’ law.”



For some pharmaceutical companies, particularly smaller companies, providing an investigational drug to patients at no cost is not financially feasible. Although some patients receive medications for free under Right to Try, the law provides an opportunity for smaller companies to make their drugs available to patients who need them without suffering a significant financial blow. Patients also have the opportunity to explore every possible option in the pursuit of effective treatment.

Daniela A. Bota, MD, PhD
Daniela A. Bota

One of the biggest objections posed by opponents of Right to Try is that there is not adequate oversight or informed consent. These drugs are required to have passed phase 1 clinical trials, and discussions between the patient and their oncologist provide the information needed to make an educated choice. Additionally, some states, including my state of California, have laws that require IRB review. Because the state laws supersede the federal law, drugs that are provided through Right to Try undergo IRB review.

Additionally, California’s Right to Try law requires that the IRB biannually report information to the FDA about the number of Right-to-Try requests made, the status of each request, the length of treatment, the costs paid by patients, the efficacy of the investigational drug in treatment and any adverse events. These additional safeguards provide a level of oversight comparable to that offered through FDA’s Expanded Access program.

The Expanded Access program also is very important and useful in providing investigational drugs to patients in a safe and timely manner. However, in cases where a small pharmaceutical company wants to provide an investigational drug to a patient in a way that is not cost prohibitive, Right to Try affords that opportunity. Possible remissions, such as those seen with Gliovac, provide an indication that in some cases, Right to Try may be an option worth exploring.

Daniela A. Bota, MD, PhD, is associate professor of neurology, senior associate dean for clinical research and chief scientific officer of clinical trials at University of California, Irvine School of Medicine and medical director of the UCI Health Comprehensive Brain Tumor Program. She can be reached at 200 Manchester Ave., Ste. 206, Irvine, CA. 92868; email: dbota@hs.uci.edu. Disclosure: Bota reports a speakers bureau role with Novocure.




It is always good news when a patient achieves remission. However, an n-of-1, as in the case of an isolated patient achieving remission or a single patient experiencing a meaningful response, does not make for evidence.

Yoram Unguru, MD, MS, MA
Yoram Unguru

An argument can be made that the current clinical trial system is less than ideal. That said, it exists for a very important reason, to make sure that medications being used and prescribed by doctors are safe and effective. Skirting that process is a very slippery slope. The FDA has listened to patient complaints and over the past few years has upped its game, such that patients in need who do not have the opportunity to wait for a trial to be completed can access medications through Expanded Access relatively quickly. Further, the process for physicians also has been streamlined with the FDA available to assist and navigate Expanded Access submissions.

Both from an ethical and a practical medical perspective, one major concern is the potential that Right to Try may foster false hope. Some patients who are near the end of their lives and who have run out of standard options understandably begin to look for whatever treatment they can find. Media reports of the “latest and greatest” treatment — sometimes embraced by advocacy groups and medical institutions — may lead patients to view these interventions as a panacea. There have been many instances of promising medications investigated in phase 1 and phase 2 trials that were not confirmed in subsequent phase 3 trials as either safe or effective. In some cases, the investigational drug actually increased the frequency of the underlying problem it was meant to address. Phase 3 trials exist to assure that patients, as well as the medical and scientific establishment, are not misled by preliminary and yet-to-be-confirmed data, no matter how seemingly encouraging. Further, phase 3 trials prevent patients from taking medications with no potential benefit, avoid unnecessary side effects and limit wasteful spending.

As a clinician who cares for kids with life-threatening illnesses, I completely appreciate patients’ and parents’ desperation. Understandably, a dying parent or dying child (and their physician) may want to turn over every stone. However, just because you can, doesn’t necessarily mean you should turn over that stone. In some cases, unproven and seemingly promising treatments may not be effective at all and may ultimately cause more harm than good.

Yoram Unguru, MD, MS, MA, is pediatric hematologist/oncologist at The Herman and Walter Samuelson Children’s Hospital at Sinai and core faculty at Johns Hopkins Berman Institute of Bioethics. He also is assistant professor at Johns Hopkins University School of Medicine. He can be reached at 2401 W. Belvedere Ave., Baltimore, MD 21215; email: yunguru@lifebridgehealth.org. Disclosure: Unguru reports no relevant financial disclosures.