Purpose

The purpose of this memo is to dissect the effects variation of unproven therapy have on end-of-life care for chronically ill patients on Medicare.

Although niche, it is an important and interesting topic to explore for a couple of reasons - unproven resources are applicable exclusively to terminal patients such as those in this study. The nature of experimental treatment also magnifies the political and policy impact on health, by stretching the bounds of access, exposing variations of factors more clearly.

What are Unproven therapies?

Unproven therapies are treatments or technology lacking approval of safety and or proof of effectiveness. Such opportunities are restricted to terminal patients as the threat of their imminent death supersedes any potential or unknown threats of the experimental treatment. The terminal patient is therefore attempting extra possibilities, regardless of the success rate, at no extra risk of harm greater than that of their expected death.

Such therapies can be medical, behavioral, or surgical and are most commonly found in the form of clinical trials of biomedical devices or chemical drugs.[1]

Terminal patients of chronic illness and clinical trials

Unproven therapies are applicable for those with chronic illness as their conditions are highly prevalent in the wider population, and therefore treatment is in constant demand. Treatment-developers and physicians therefore often utilize clinical trials to achieve greater probability of breakthroughs, or faster approval of in demand treatments. It is also, however, an expanded potential of end-of-life care for the participants in trials, and hence it is important to study the variations in Medicare and in the inequitable benefits different patients may reap from unproven therapies.

Study of data

All data from the Dartmouth Atlas and trial statistics are that of 2019. The measure of study in the data is the characteristics of the deaths. Clinical trials have consistent evidence of survival prolongation and mortality reduction, especially in chronic illness, making data of the deaths an effective representation or explanation of compounding effects.[2] Overall survival rates for participants in clinical trials also have a six month to one year increase average,

meaning the efficiency of unproven interventions can makes a significant enough difference in death data by year to use as evidence for certain correlations.[3]

Comparing the states of California and Wyoming can reveal the biggest impact of variation as they are the two states with the most and the least existing clinical trials.[4] They are also the states with the greatest and least spending on Medicare associated with Chronic diseases (Figure 3.1) and on Hospitalization and Inpatient Physician services associated with Chronic diseases (Figure 3.2). The strong positive liner correlation between spending and advantaged care is applicable to the variations in unproven therapies, as more financial input displays greater opportunity for a wider range of unproven therapies.

Figure 3.1

Source: The Dartmouth Atlas of Health Care 2008

Figure 3.2

Source: The Dartmouth Atlas of Health Care 2008

This is also directly and positively correlated with the associated care required with clinical trials, such as physician visit frequency and inpatient duration. Further notable is the significantly greater range between medical specialist opposed to primary care visit. As specialists are those who have connections to trials, the contrast accounts for inequitable access to trial opportunities. (Figure 2).

Figure 2

Source 1: PhRMA Clinical Trials State Map

Source 2: The Dartmouth Atlas of Health Care 2008

Explanations for variation in medical practice

The most notable explanations of variation in unproven interventions include, but are not limited to, differences in access and practice, both of which are also subjected to a plethora of determinants.

The “access” hypothesis applies to unproven therapies through many ways. Inequitable access to clinical trials and the highly specific resources required to conduct them limit the chronically ill patients accessible to the trials. Those in urbanized and profitable states like Los Angeles, near certain hospitals, have a notable difference in opportunities impossible to the often underfunded and overworked public facilities in rural areas like Wyoming. Furthermore, the of physicians in states like Los Angeles historically work in well-funded and resource saturated centers, increasing their exposure and connections to existing clinical trials, as well as the power and credibility to enroll their patients in beneficial trials. The same case applies to the chronically ill patients themselves. The health literacy of patients in richer states make them more able to make educated requests, anecdotal research and even informed consent to the complex and unique unproven treatments for their terminal state.

The “professional uncertainty” hypothesis applies to the ethical and moral judgement of physicians, as well as the motives certain doctors have for providing trial options or lack thereof. The simplest difference is between those who do and don’t believe in clinical trials as a benefit for their terminal patients. As physicians are often the only pathway to clinical trials, strictly based on physician autonomy, chronically ill patients may or may not even be presented to opportunity to receive experimental care. More complex variations are result subjective to each practitioner’s practice style and beliefs. There is a scale of acceptance towards the ethical debates around unproven treatments, such as the recipients of placebo or drug and the unstudied toxicities and complications of trials. There are also the differences in motives, whether a doctor has beliefs in a “treatment” versus a “cure”, that is, whether the patient’s enrollment in trials should benefit the prolongment of their individual life, or that their expected death creates reasonable sources to contribute clinical study and success of future treatments. Both biases are skewed to the physicians social and work environment, as well as medical education and mentorship culture. The imbalance of power and academia status between doctor and patient makes this hypothesis especially pertinent to unproven therapies as end-of-life care, as clinical trials become paternalistic: a privilege only grantable by physicians instead of a form of care accessible to all terminal patients.

As is the case with all aspects of healthcare, there are countless other potential explanations for the variations amongst patients than the ones mentioned previously. Furthermore, all these factors are deeply intertwined and feed into one another. The four domains of the social determinants of health make a quick generalization of other potential explanations for variations: socio-economic status, physical environment, social environment and discrimination. It is also important to address a common confusion of clinical trials and Medicare. It could be assumed that, because of equal coverage of trials for all patients, so long as the patient requests to enroll in a trial there should be little variation in receiving the unproven treatment. (Figure 1) However, there are often free-of-cost and sponsored opportunities, and these are only presented or even plausible for Medicare individuals in advantaged environments.

Figure 1

Source: Medicare your medicare coverage: Clinical Research Studies

Original Medicare Part A and/or Part B

Medicare Advantage Plan 

Ultimately, it is the long- and short-term effects of these subgroups, in tandem with the numerous variation hypotheses manifest through the difference in clinical trial benefits, as in turn a level of care quality for patients with terminal chronic illness.

Suggested interventions to reduce variation

It is first important to consider the factors that differ between unproven therapies from other healthcare variables. While there are numerous contributors, the two biggest and most specific to the demographic of patients in this study are the lack of easy public access to clinical studies, and the control the physician has on their patient’s opportunity to enroll.

To address both these issues, the most realistic and useful intervention approach would be to firstly increase information access and health literacy on a public level, then to increase patient autonomy of unproven therapy opportunities, instead of physicians making the judgement based on their opinion on the matter.

As all clinical trials are regulated by the FDA, complete and transparent communication between Medicare, the FDA and all hospitals is imperative to successful and equitable benefits of clinical trials for patients. Routine updates of all existing clinical trials should be evaluated by Medicare, and a published list of Medicare covered, and sponsor covered trials should be of public availability. Finally, and most importantly, there needs to be a standardization or policy that obligates informing terminal patients of clinical studies as an option, as well as providing all the available trials applicable to their condition. The resources needed for this implementation would not be financially demanding, but hinges on commitment from all contributing parties, and unbiased compliance by hospitals and their physicians.

Conclusion

Lack of equitable access to clinical trials for end-of-life chronically ill Medicare patients

is an unfortunate result of its premature awareness, and therefore lose regulations. Through comparison of data between the state with most and with least clinical studies, it becomes apparent that there is a wide contrast between financial advantage, opportunity and connection to medical resources in the two areas. These explanations, amongst endless other social determinants, ultimately result in the variation of unproven therapies as a form of care for chronic illness amongst decedents end-of-life care.

References

[1] What are clinical trials and studies? National Institute on Aging. Accessed February 19, 2024. https://www.nia.nih.gov/health/clinical-trials-and-studies/what-are-clinical-trials-and-studies.

[2] Cancer clinical trials - mayo clinic research. Accessed February 19, 2024. https://www.mayo.edu/research/clinical-trials/diseases-conditions/cancer/.

[3] Merkhofer CM, Eaton KD, Martins RG, Ramsey SD, Goulart BHL. Impact of clinical trial participation on survival of patients with metastatic non-small cell lung cancer. Clinical lung cancer. November 2021. Accessed February 19, 2024. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8531178/#:~:text=Overall%20Survival%20by%20Trial%20Participation&text=Median%20overall%20survival%20for%20trial,years)%20among%20non%2Dparticipants.

[4] State map - clinical trials. PhRMA Org. Accessed February 21, 2024. https://phrma.org/en/resource-center/Resources/State-Map/Clinical-Trials.

[5] Tracking the Care of Patients with Severe Chronic Illness, data.dartmouthatlas.org/downloads/atlases/2008_Chronic_Care_Atlas.pdf.