The $650,000 Gamble: Ethical Challenges of High-Cost Experimental Therapies

Julia Young is no stranger to the harsh realities of battling cancer. After enduring 11 years of numerous surgeries, radiation therapy, and rounds of chemotherapy, doctors once again delivered the disheartening news: her ovarian cancer was still growing.

For advanced cancer patients like Julia, who have exhausted conventional treatment options available in mainstream medicine, the chance of a cure, as uncertain as it may be, can serve as a glimmer of hope. Julia has opted to receive a promising, but unproven therapy - a personalised neoantigen vaccine that comes with an exorbitant price tag of ​​$83,000 (~650,000 HKD), a substantial financial commitment for a treatment with unknown efficacy (1).

The personalised neoantigen cancer vaccine represents a monumental advancement in precision immunotherapy, where it works by priming the patient’s immune system to recognize, and destroy cancer cells with specific mutations (2). This tailored approach to cancer treatment has demonstrated potential in certain clinical trials, yet it remains experimental and unproven. Despite this, experimental therapies are sometimes made available to terminally ill patients in the United States through the Food and Drug Administration (FDA)'s "Compassionate Use" program, which allows certain therapies to be used clinically before they have achieved full FDA approval (3). When patients resort to similar high-priced, experimental therapies of unproven efficacy, a myriad of ethical conundrums arises (4).

First of all, the use of experimental therapies, particularly when they come with substantial costs, can be potentially exploitative (4). Critics contend that companies might exploit the desperation of patients, using them as human guinea pigs to accelerate the pace of their research. Given the numerous obstacles to initiating clinical trials, such as lengthy timelines, lack of eligible participants, and limited funding, the concern of researchers offering experimental therapies to bypass the stringent research protocols and guidelines can be alarming (5). The Jaime Leandro Foundation, the non-profit that manufactures Julia’s cancer vaccines, says that the $83,000 fee merely covers the direct expenses of creating the vaccines and generates no profit (6). However, as well-intentioned as the foundation may be, it could be argued that patients are still exploited in the process of paying enormous amounts for a treatment that may not offer any concrete benefit (1). This also links to a separate issue surrounding the ethical pricing of drugs - how can we strike a balance between allowing pharmaceutical companies to recoup the substantial costs tied to drug development and making to make enough profits to incentivize innovation, while still ensuring medications remain affordable for patients (7)?

Another prominent ethical consideration is the question of safety. Experimental therapies like the neoantigen vaccine are, by definition, in the exploratory stage and their effects, both positive and negative, are not entirely predictable. The lack of larger, comprehensive clinical trials for these therapies means that our understanding of their safety profile is limited, as small-scale, early trials may mask significant risks that may further deteriorate the patient’s health (8). 

Yet, some might argue that as long as the patient is well aware of the risks, and still chooses to pay willingly, then there is nothing ethically wrong with the patient paying for a treatment that might not yield any benefits. However, this perspective overlooks the issue of unrealistic therapeutic optimism, which can compromise patient autonomy and informed consent. Therapeutic optimism refers to patients’ tendency to overestimate the benefits of a treatment but underestimate its risks, often due to a heightened state of hope or desperation. This can distort their judgement and obscure their understanding of the experimental nature of certain therapies, impeding their ability to make informed healthcare decisions (9). On top of that, when patients are financially invested in their treatment, they might feel additional pressure to believe in its efficacy, further amplifying their therapeutic optimism. Consequently, this could cause patients to persist with a treatment that is ineffective, or even harmful due to their financial commitment and psychological need to prove their expenditure worthwhile (10). 

Moreover, the issue of distributive justice is particularly pronounced in the case of expensive experimental therapies. As insurance or governmental agencies are unlikely to subsidise unproven treatments, the high cost of these therapies creates an access barrier for many patients who cannot pay out of pocket. As such, these potentially life-saving treatments may only be accessible to the wealthy and privileged, further exacerbating existing health disparities. In addition to monetary costs, the allocation of substantial resources for a single patient’s treatment raises ethical concerns. For instance, creating a single personalised neoantigen vaccine requires 4 months of concentrated scientific effort, which involves significant time and expertise from highly trained professionals (1). From a utilitarian perspective, might we be neglecting a larger patient population whose treatment costs are comparatively lower and whose therapies are proven to be effective? 

The ethical dilemmas surrounding the use of experimental therapies are complex. In the case of Julia and countless other patients, the hope of a potential cure is powerful and understandably so. It is therefore incredibly important that we protect patients from exploitation, ensure that they fully understand the potential risks and benefits, and that access to experimental therapies does not become yet another enabler of health disparities. 

References

1. Saltzman J. Foundation stirs controversy by charging cancer patients $83,000 for unproven but promising experimental drug Boston Globe2023 [updated March 4, 2023. Available from: https://www.bostonglobe.com/2023/03/04/business/foundation-stirs-controversy-by-charging-cancer-patients-83000-unproven-promising-experimental-drug/?s_campaign=bostonglobe%3Asocialflow%3Atwitter.

2. Biswas N, Chakrabarti S, Padul V, Jones LD, Ashili S. Designing neoantigen cancer vaccines, trials, and outcomes. Front Immunol. 2023;14:1105420.

3. Expanded Access U.S. Food & Drug Administration2022 [updated 12/21/2022. Available from: https://www.fda.gov/news-events/public-health-focus/expanded-access.

4. Borysowski J, Ehni HJ, Górski A. Ethics review in compassionate use. BMC Med. 2017;15(1):136.

5. Patient access to experimental treatments Nuffield Bioethics2018 [updated 20/11/2018. Available from: https://www.nuffieldbioethics.org/publications/experimental-treatments/introduction/ethical-issues-arising-from-the-use-of-experimental-treatments.

6. FAQs: The Jaime Leandro Foundation; 2022 [Available from: https://jaimeleandrofoundation.org/faqs.

7. Parker-Lue S, Santoro M, Koski G. The ethics and economics of pharmaceutical pricing. Annu Rev Pharmacol Toxicol. 2015;55:191-206.

8. Michelle Rubin KRWM. The Impact of Right to Try Laws on Medical Access in the United States: Baker Institute for Public Policy; 2016 [1/10/2023]. Available from: https://www.bakerinstitute.org/research/right-try-unproven-drugs.

9. Gold RS, Aucote HM. 'I'm less at risk than most guys': gay men's unrealistic optimism about becoming infected with HIV. Int J STD AIDS. 2003;14(1):18-23.

10. Jansen LA. Two concepts of therapeutic optimism. J Med Ethics. 2011;37(9):563-6.