Physician Responsibility to Discuss Palliative Unproven Therapies with Out-of-Option Patients

We agree with Lynch et al that patients with chronic diseases and Band-Aid treatments are unlikely to benefit from a version of Operation Warp Speed or by deprioritizing standards of scientific evidence to bring new products to market with questionable safety and efficacy. However, we challenge their underlying claim that “marginally effective or ineffective products do little to help” very sick patients’ unmet needs (Lynch et al. 2021). We argue that under specific circumstances, some unproven and scientifically unsubstantiated interventions do have a place in conventional medicine for out-of-option patients who desire control. For such patients, suggesting benign, unproven interventions under certain conditions may provide hope and a sense of control over their illness. This is likely to strengthen the patient-physician relationship and physicians can continue to support patients and offer guidance throughout their treatment. In this commentary, we highlight the necessity for physicians to offer alternative options in these circumstances and outline criteria for considering how such treatments can be recommended.

Many patients with chronic and severely debilitating conditions seek experimental interventions that leads to a discussion about clinical trials, expanded access, or possibly Right-to-Try with their physician (Smith et al. 2021). Among those who reach this point in their therapeutic journey, many have already experienced emotional burdens, dismissive providers, and have received limited health improvement from conventional medicine. Upon consideration of these research options, additional burdens weigh on patients including lengthy trial wait times, uncertainty about meeting inclusion criteria, and doubts about the trial’s success. Patients often invest months (or years) of hope that academic research centers may call them to enroll in a trial. When patients are confronted by the limited progress of research, their needs for closure about their future and control over their condition remain unmet, leading to feelings of despair.

In these vulnerable moments, physicians can play a critical role in shaping patient priorities and attitudes about the prospects of treatment, quality of life, and emotional well-being. In the absence of a “therapeutic” outlet, many patients, feeling abandoned by the medical establishment, may undertake an unproven and potentially risky alternative. This may result in a loss of trust and deteriorate the patient-physician relationship. Patients under this loss frame may fully dismiss conventional medicine and move towards unscrupulous providers who offer any glimmer of hope. All of this would occur outside the purview of medical professionals who can support patients when considering unproven interventions.

From our research, we have heard all too often from patients facing just such scenarios. Patients against all odds feel deserted and actively turn to online sources to seek health information. Nearly 1 in 4 websites have misinformation (Al Khaja et al. 2018) and none more rampant than what is seen among chronic disease patients seeking unproven stem cell interventions from for-profit clinics. In one case, a woman in her 50s with an end-stage neurological illness felt dismissed by her physicians when suggesting whether physical therapy or other alternatives would help. While she was interested in clinical trials, she was ineligible for studies at major medical institutions. She felt hopelessness and her depression spiralled. Disaffected, she and her family pursued a stem cell treatment from another country and described the alternative clinic, its facility, staff, and procedure as “outstanding.” Quickly her spirits were lifted. As she shared her story, the patient mentioned she was trying something new every day, including physical therapy, yoga, and brain mapping. Other times, the options explored by patients are not so safe or benign, including her stem cell treatment, which cost her over $25,000, not including travel. It is unclear whether the stem cell intervention did anything to improve her condition as she recently passed away. While her case remains elusive, a recent report showed substantial physical harms that result from unproven stem cell interventions, including blindness, tumors, lesions, infections/sepsis, stroke, heart attacks, significant pain, and death (Pew Charitable Trusts 2021). Similarly, treatments like IV injections of hydrogen peroxide to fight infection, a therapy espoused by some clinicians, among others, are a clear threat to patient safety and promotes blatant disregard of information and risks to patients (Plevin 2017).

Such examples offer points of reflection. Out-of-option patients seek emotional well-being and receive benefit from continued engagement with conventional care providers where riskier interventions can be deliberated and discussed openly. For the sake of safety and harm reduction, physicians should widen conversations about care to preemptively include physician-vetted, non-research interventions and therapies and accommodate choices for unproven interventions that have a decent safety profile. Various complementary and alternative medicine modalities including Reiki, cupping, herbal therapy, acupuncture, and natural remedies among others may not be risk free but offer a known and generally tolerable safety profile.

Physicians are well within their ethical bounds to offer such interventions to out-of-option patients even if they lack efficacy evidence. Afterall, their primary duty centers on patient welfare first and foremost (American Board of Internal Medicine Foundation 2005). If nothing else, such options give patients meaningful control in how they relate to their condition, which may improve patient reported outcomes. Such emotional strength, added resolve to persist and endure, and knowledge that they are being accompanied by their physician can help prevent risky behavior and positively affect their symptoms even if its genesis mirrors a placebo effect. If an open-label placebo treatment for Irritable Bowel Syndrome can improve patient well-being compared to no treatment, then it is a therapeutic win (Kaptchuk et al. 2010). Similar effects have been shown in cancer (Hoenemeyer et al. 2018). Even when patients knew they were receiving a placebo, they still perceived symptom improvement. Such data should nudge us to reconsider what a clinician is and is not ethically obligated to endorse or at least accommodate within their fiduciary bounds.

In recommending palliative unproven therapies to out-of-option patients, we believe a set of ethical criteria should be followed. First, physicians should only suggest therapies that have little or no known physical, emotional, or financial harm to individuals and society. Not all therapies are alike, and when patients confront their providers with clearly risky interventions, ones that cost too much, or make outlandish claims of benefit, physicians have a duty to inform patients of the harms and suggest safer alternatives that can fulfill the same emotional and spiritual needs (Hyun 2013). Second, we suggest that treatment alternatives must at least have some psycho-somatically relevant potential benefit to patients. These conditions might extend to cultural, social, and personally ingrained meaning or relevance for patients; if a patient believes a therapy can be effective for a variety of personally or socially held convictions or assumptions, such a treatment could be a welcomed option to at least tolerate within the bounds of safety. Deeper knowledge and awareness of patients’ community and cultural artefacts may be an avenue when suggesting treatment options. Therapeutic use of honey, for example, holds religious significance for members of the Muslim faith and is also culturally perceived to have curative effects (Khan 2020). Patients may already be willing to experience the curative effects on faith alone, and this may be sufficient to combat despair, improve symptoms, and build trust between patients and their religiously and culturally sensitive provider. Such positive outcomes, broadly construed, can serve not only to empower patients but also foster greater trust, humility, and empathy in a physician-patient relationship. Third, physicians must honestly inform patients about the therapeutic benefit of unproven options based on scientific evidence known to date. They must not engage in deception or omission but explain to patients that the treatment is unlikely to result in substantial relief of disease symptoms, but they may receive emotional and other health benefits. An obligation to be honest aligns with good clinical practice (American Board of Internal Medicine Foundation 2005), but caution must be heeded when discussing unproven interventions to ensure a connection with patients, show compassion and support, and communicate empathically.

While we are not arguing that physicians should discourage evidence-based options, we emphasize that they should not be presented as the only avenue for achieving patient welfare in the case conventional medicine can no longer offer suitable alternatives. Physicians have a responsibility to retain the trust of patients, remain present, and enhance emotional health and well-being, and not close the door for patients curious to try benign alternatives. Through this, patients can perceive their doctors and the wider medical establishment as being invested in providing a diversity of patient-centered options, unwilling to abandon patients in their greatest time of need, and valuing patients’ lived experience (physiologically, psychologically, spiritually, and culturally) as necessary tools of the healing process, especially when research hits its limit.

By offering options for patient who will see little progress in research or therapy development – despite best efforts at increasing access and optimization – the medical establishment, though not endorsing policy or regulatory shortcuts, can in the meantime utilize safe (palliative) interventions under the guidance of physicians while inviting increased patient autonomy, trust and empowerment, and the promotion of ongoing engagement in a supportive patient-physician relationship.