Real-World Data: Addressing Bias in Healthcare

Real-world data (RWD) is an emerging concept with a broad range of applications for studying patient health data including treatment safety and efficacy, short and long-term health outcomes, and disease burdens. RWD can include data from electronic health records, wearable technology, disease registries, patient-reported outcomes, social media, and various electronic health services and is a growing contributor to clinical and biopharma research on treatment efficacy. Traditional randomized control trials (RCTs) remain the gold standard for assessing treatment or diagnostic efficacy, but they often involve a homogenous population due to their strictly controlled inclusion criteria and limited sample sizes. Thus, RCTs can be limited in their ability to represent patient groups of minority backgrounds—including racial and ethnic minorities, patients with comorbidities, and patients with rare conditions.

Coupled with analysis using AI and machine learning technologies, vast amounts of real-world data from millions of patients have been analyzed to generate real-world evidence (RWE) more rapidly and with more cost-effectiveness than traditional clinical trials. With greater sample sizes, RWD may be better able to summarize the range of patient outcomes by including patient subgroups and demographics that are more representative of the true populations seeking healthcare. Additionally, this data may then be used to further efforts in precision medicine. Companies such as Flatiron Health, which generate RWE for oncology treatments, use data on patient genetic mutational variants and other biomarkers in an attempt to optimize treatment. This ability to capture diverse covariates is particularly useful in fields such as oncology and mental health, where patient outcomes are often associated with a multitude of genetic or demographic factors.

However, there are a number of limitations in the current capacities of RWD. A major limitation is the varying quality of data and lack of data standardization: patient notes involving free-text, incomplete fields, and different reporting formats across clinics all pose challenges. Additionally, rural healthcare systems or clinics with limited resources may be less able to anonymize data to share with RWD companies, potentially leading to their exclusion from RWD studies, and more broadly, the exclusion of minority populations whom their data represents.

Though further research and innovation is needed to improve data quality and analysis methodology, RWD continues to be a growing area of research that is taking steps toward addressing bias and disparities in healthcare.

Edited by: Anne Sacks

Graphic Designed by: Soojin Lee

Citations

1. https://bmcmedresmethodol.biomedcentral.com/articles/10.1186/s12874-022-01768-6

2. https://www.mckinsey.com/industries/life-sciences/our-insights/real-world-data-quality-what-are-the-opportunities-and-challenges

3. https://www.the-scientist.com/sponsored-article/real-world-data-opening-new-avenues-for-health-research-71090

4. https://flatiron.com/real-world-evidence

5. https://www.iqvia.com/solutions/real-world-evidence/real-world-data-and-insights?utm_source=google&utm_medium=cpc&utm_campaign=2023_gadsRWDUS_RWS_RS&utm_content=150775213880&utm_term=real%20world%20data&gad=1&gclid=Cj0KCQjwtJKqBhCaARIsAN_yS_mdudgoTAm9rWhG_uJabVVJcxoXQPnhssagteFuwgyG5LIib1B_rr4aArMZEALw_wcB

Xenotransplantation: The Animal Inside of You

Imagine a giant football stadium, swarming with fans. Every single seat is filled up, and there are even people standing on the field and in the isles, just to be in that stadium. Now add 20,000 more people. That’s how many people were waiting for organ transplants in 2022.

Over 100,000 US patients each year are on a life-or-death organ transplant waiting list, but only around 40,000 transplants occur annually. For a majority of patients on that list, the wait for an organ is excruciating and can have a deadly toll, but the gap between the number of organ donors and the number of recipients is remaining relatively steady. When people are 16 and getting their driver's license, we can’t force them to check the ‘organ donor’ box. So, if we can’t increase the amount of donors, how can we fix this?

While we can’t increase the amount of human donors, we might be able to increase the amount of animal donors. Scientists have been experimenting with xenotransplantation, the transplantation of animal organs or tissue into a human. Within the past two years, there have been significant advances in this field including the first successful genetically modified pig heart transplant in 2022, at the University of Maryland School of Medicine, and the first successful pig kidney transplant in 2023, at NYU Langone. These are the first successful transplants in humans and with each new surgery, we continue to discover more and more about the human physiological response to animal organs.

While these transplants are promising, many physicians and patients are concerned with the ethical dilemma of xenotransplantation. Firstly, many patients may be resistant to receiving an animal organ as it conflicts with their personal religious beliefs. In Judaism and Islam, for example, pigs – or swine – are viewed as inferior and unclean beings. To have the organ of an animal that your religion forbids you from associating with can be mentally challenging. Even in patients who are not religious, the idea of having another animal’s organ inside of you is mentally tolling, especially with the stigma surrounding it. It’s hard to explain to someone who is not familiar with xenotransplantation treatment that you have a pig heart; to some people, it just seems gross. It may be hard for the patient to justify having that organ for themselves if people around them are not accepting of it.

Furthermore, the actual harvesting of pig organs is questionable morally and efficiency-wise. From birth, pigs are genetically modified in order to alleviate the human immune response from rejecting the foreign tissue. The caveat is that the pigs must be raised in a completely sterile lab and constantly undergo medical procedures until they are ready to be used for harvesting. The pigs – who are neurologically advanced animals – are not mentally stimulated, which raises concerns. Also, the entire process is costly and inefficient as a singular pig has to be raised in very specific conditions, so optimizing this procedure will take time.

Even if we develop an efficient way to harvest organs, it will still cost an exorbitant amount, leading to questions of who is prioritized for animal organs versus human organs. The financial costs of xenotransplantation often make it inaccessible for lower income families to obtain treatment immediately. This forces families to accept steep financial penalties if they choose to have the transplant rather than remaining on machinery such as a ventilator or dialysis machine as substitutes for failing organs.

However, if all of these ethical considerations are taken into consideration prior to xenotransplantation becoming a mainstream treatment option, then it will greatly benefit the medical community. So many lives will be saved because of this upcoming technology. It is only a matter of time before it becomes medically feasible.

Edited by: Sanjana Anand

Graphic Designed by: Shanzeh Sheikh

Citations

• Ebner, K., Ostheimer, J., & Sautermeister, J. (2020). The role of religious beliefs for the acceptance of Xenotransplantation. exploring dimensions of xenotransplantation in the field of hospital chaplaincy. Xenotransplantation, 27(4). https://doi.org/10.1111/xen.12579

  • https://onlinelibrary.wiley.com/doi/10.1111/xen.12579

• Grosse H. W. (2003). Xenotransplantation aus christlich-ethischer Sicht [Xenotransplantation from a Christian-ethical perspective]. ALTEX, 20(4), 259–269.

  • https://pubmed.ncbi.nlm.nih.gov/14671705/

• Rollin B. E. (2020). Ethical and Societal Issues Occasioned by Xenotransplantation. Animals : an open access journal from MDPI, 10(9), 1695. https://doi.org/10.3390/ani10091695

  • https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7552641/

• Two-month study of pig kidney xenotransplantation gives new hope to the future of the organ supply. NYU Langone News. (n.d.). https://nyulangone.org/news/two-month-study-pig-kidney-xenotransplantation-gives-new-hope-future-organ-supply

  • https://nyulangone.org/news/two-month-study-pig-kidney-xenotransplantation-gives-new-hope-future-organ-supply

• Health Resources and Services Administration. (n.d.). Organ Donation Statistics. Learn About Donation. https://www.organdonor.gov/learn/organ-donation-statistics

  • https://www.organdonor.gov/learn/organ-donation-statistics

• Science Learning Hub. (n.d.). Xenotransplantation – timeline. Xenotransplantation Timeline. https://www.sciencelearn.org.nz/resources/2125-xenotransplantation-timeline

  • https://www.sciencelearn.org.nz/resources/2125-xenotransplantation-timeline

• Rabin, R. C. (2021, October 19). In a first, surgeons attached a pig kidney to a human, and it worked. New York Times. Retrieved from https://www.nytimes.com/2021/10/19/health/kidney-transplant-pig-human.html.

  • https://www.nytimes.com/2021/10/19/health/kidney-transplant-pig-human.html

Personalized Medicine: The Customized Human Blueprint

When somebody mentions going to the doctor, the common response is, “Are you feeling alright?” Typically, visiting a doctor is associated with feeling unwell. However as more people face health challenges every day, a modernized approach to healthcare known as personalized medicine is gaining prominence. The conventional healthcare model primarily concentrates on treating the disease or symptoms manifested in the patient. It often overlooks the complex web of factors that contribute to the development and progression of the disease. Recent technological advancements have made it possible to detect diseases in their early stages, offering an opportunity for early intervention and prevention.

Personalized medicine takes into account an individual's unique genetic makeup, environmental factors, and lifestyle choices to create a tailored medical plan. This approach aims to make healthcare more personal, predictive, preventative, and participatory. Each person inherits a unique combination of genes from their parents, resulting in an individual with varying susceptibility to different diseases. Moreover, an individual's life experiences further add to the complexity of disease susceptibility.

The personalized medicine approach has the potential to transform the entire healthcare system, with a heightened focus on primary care. In primary care settings, comprehensive information about a patient's health, genetics, and lifestyle is gathered to assess their risk for specific diseases. Collaboratively with the patient, the physician creates a personalized plan for disease prevention and management.

Consider the example of a patient struggling with obesity. During their checkup, various tests are conducted, including genetic markers, BMI measurements, and other baseline assessments. Lifestyle questionnaires delve into diet and physical activity. With this wealth of information, the medical team can work alongside the patient to develop a tailored strategy for managing obesity.

While personalized medicine appears to be a promising addition to our healthcare system, several barriers hinder its implementation. The shortage of primary care physicians has led to high workloads, making it challenging for the physicians to dedicate the necessary time to develop personalized strategies for each patient. Patients may also avoid visiting doctors due to financial constraints or personal preferences. Additionally, the introduction of numerous tests can complicate insurance costs, potentially making healthcare more expensive and subject to disputes over necessity.

To bring about this drastic shift in healthcare, it is critical to reevaluate and restructure parts of the current healthcare system. The current model is unsustainable for the rising complexity of chronic diseases that require strategies for prevention even before symptoms manifest. With more than two-thirds of US healthcare expenditures directed toward chronic diseases, personalized medicine offers a solution to alleviate the burden of noncommunicable diseases. It falls upon the collective demand for change to transform this concept into a system capable of saving countless lives.

Edited by: Sam Shi

Graphic Designed by: David Axon

Sources

1. Snyderman, R. (2012), Personalized health care: From theory to practice. Biotechnology Journal, 7: 973-979. https://doi.org/10.1002/biot.201100297

2. https://www.washingtonpost.com/health/2023/10/17/primary-care-saves-lives/

3. https://time.com/6257775/patient-burnout-health-care/

4. https://health.ucdavis.edu/precision-medicine/what-is-precision-medicine.html