May 4, 2021

Real-World Evidence

Physicians in practice and their patients create real-world evidence. Through guidelines-based care and clinical judgment, physicians and advanced care providers prescribe diagnostic studies and therapies that constitute it. Every type of healthcare setting from tertiary care, and National Cancer Institute-designated comprehensive cancer centers, to community clinics is included. When evaluating healthcare resource utilization and costs, real-world evidence provides insight into divergent healthcare delivery systems and the demographics of America. Real-world evidence also reflects the health of the nation. Diagnosis of disease and comorbidities reflect the nation’s diversity in age, geographic location, and access to care, which is often dictated by socioeconomic status and, by proxy, the type of healthcare insurance. These factors all influence the outcomes of disease.

Reflecting the issues within public health, real-world evidence provides the most important type of translational research—translating outcomes from highly controlled clinical trials with a limited number of patients that meet strict eligibility criteria, to a broader patient population in a wide range of healthcare settings. Therapeutic improvements within broader, often more compromised, patient populations are the most meaningful as they impact the nation’s health.

Lessons Learned

We have learned a lot about healthcare from the COronVIrusDisease-2019 (COVID-19) pandemic. The pandemic has ingrained information about personal health risks and population-based healthcare policy. Medical researchers and the patients who participated in the clinical trials accomplished unprecedented breakthroughs in therapeutics and vaccines. Healthcare systems triaged resources to meet the needs of patients. Operating rooms were repurposed into intensive care units.

Exhausted healthcare professionals felt an obligation to share their observations and COVID-19 outcomes as soon as possible with colleagues and the public. Also sharing in this obligation, the publishers of medical journals created free access to pandemic-related peer-reviewed publications. Vulnerable populations were defined. Unexpected comorbidities, like anosmia and hypercoagulopathy were observed. Healthcare professionals, despite a lack of experience with this novel disease and cytokine storm, risked becoming COVID-19’s next victim or bringing it home to their families. Every essential worker who provided or supported the delivery of healthcare during this pandemic deserves the nation’s profound gratitude.

So, what is next for medicine as we look beyond the acute phase of the pandemic?  The COVID-19 pandemic has changed medical research, impacted healthcare outcomes, and influenced how healthcare systems function. Real-world outcomes since 2020, whether related to the pandemic or other diseases, will have a long-term influence on healthcare practices and policies.

While instituted with an abundance of caution, it has been demonstrated that many of the sclerotic practices in clinical trials have profound human costs—namely, the delay in the availability of therapeutic advances that could save lives. Also raised are the ethics of randomizing patients to a potentially inferior standard of therapy rather than using real-world data to create an external control arm in a clinical trial. Especially important is that the early stopping rule and other critical statistics for an experimental arm in a clinical trial are primarily dependent on the outcomes and toxicities of therapy being tested.  Instead of highly selected patients in the control arm of a prospective, randomized clinical trial, the outcomes of the control arm cohort in a clinical trial should be more representative of real-world data. COVID-19 has taught medical research and the nation’s regulatory agencies the urgency of providing practice-changing diagnostics and therapeutics on behalf of our patients, while maintaining strict safety standards.

Outcomes and Consequences

As a population health endpoint, real-world evidence will be critical to fully understand the broad range of the pandemic’s outcomes. The second toll of the pandemic will be the currently unknown long-term consequences of COVID-19. End-organ COVID-19 morbidity and mortality, including cardiovascular, coagulopathy, and neurologic pathology will need to be assessed by age when the patient was infected. Healthcare resource utilization and patterns of morbidity may change in older populations that survived COVID-19 and who already have baseline end-organ comorbidities. Like Chagas’ disease, years may be required in younger patients before the long-term end-organ effects of COVID-19 manifest. Mental health effects will impact generations.

The third human toll of the COVID-19 pandemic, as discovered through real-world data, will be the increased morbidity and mortality from the delay of routine care. By necessity, COVID-19 forced most healthcare systems to be nimbler, rather than function as a burdensome bureaucracy. The efficiencies gained will need to be continued as healthcare facilities ramp up to deliver diagnostics and procedures delayed from the start of the pandemic. 

Most importantly, it was the coordination of healthcare professionals of every specialty and at every level in medicine that cared for the nation. Now having heightened significance, the ethical oath taken by healthcare professionals of all ages was fulfilled honorably. Every data point within real-world evidence was and continues to be a function of a healthcare professional, risking his or her life to care for the life of a patient during the pandemic.

This great sacrifice will be analyzed as the consequences of COVID-19 continue. In fact, it will only be through real-world evidence that medicine will more fully understand it as an infection, and the lessons learned in response to a public health emergency, especially in vulnerable populations, including the socioeconomically disadvantaged. The lessons learned must also include an analysis of resources necessary to restore and expand the healthcare infrastructure and workforce and resolve gaps in access to care. Skirting possible Severe Acute Respiratory Syndrome (SARS), Ebola virus and Zika virus pandemics over the past two decades, the COVID-19 pandemic may not be a centennial event.

Lasting Impact

The COVID-19 pandemic taught the nation much about readiness, responsibility, and resilience. We also learned about the dedication of the healthcare system, and the sacrifice, willingly taken by healthcare professionals in fulfillment of their oath to service, to care for every patient. Critical to the recovery from the COVID-19 pandemic were the innovations of medical research, including the efficiencies introduced within clinical trial design. At STATinMED Research, we identified risk factors for COVID-19 survival using machine learning, and look forward to publishing our findings soon. Analysis of and the efficiencies allowed by real-world evidence will have a lasting impact on the nation’s healthcare system.

References

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