Written by Dr. Mace Rothenberg, Chief Medical Officer, Pfizer Inc.
The concepts of rigor, transparency, and urgency have always been critically important in clinical research. What makes each component essential? Are there limits to which strict adherence to one component can be relaxed in the interest of another? And how has Covid-19 influenced the balance between each of these factors? Now more than ever, when information can be broadly disseminated almost immediately, we must ensure that the scientific community does not become an inadvertent source of misinformation.
The risks of releasing information that is not properly vetted looms large when the healthcare community is desperate for answers. Adhering to — and properly balancing — the tenets of scientific rigor, transparency, and urgency in clinical research and reporting findings are the guideposts we must follow to ensure this doesn’t happen.
Scientific rigor requires strict adherence to the highest standards and best practices of the scientific method, ensuring robust and unbiased experimental design, conduct, analysis, and interpretation. Scientific rigor is essential to building confidence in the veracity of findings.
Transparency relates to the dissemination of findings in a way that is explicit, clear, and open about the research design, methods, and results. This includes a commitment to publishing results so that others may review, refute, or reproduce the results and by doing so, further extend our body of knowledge and understanding in that field. A robust system of reporting research results creates a comprehensive foundation of scientific knowledge and instills confidence in both the medical community and the general public.
There is an expectation that research results will be shared publicly within a reasonable period of time. Given the fact that Covid-19 emerged as a new disease that was having a rapid and devastating impact on a global scale, there was clearly a need for research results to be shared with the widest audience by the fastest means possible. Lives were at stake.
Since the pandemic took hold, we have seen what the consequences can be when one of these parameters is stretched beyond a breaking point. Covid-19 has challenged medical journals that have tried to maintain the high quality of peer review while trying to rapidly disseminate study results that have the potential to inform and change patient treatment decisions. The unprecedented urgency for information that could positively impact patient care during this crisis has given rise to a substantial expansion of non-peer reviewed pathways for sharing this information, including the use of pre-print servers and “publication by press release.”
Some practitioners or key opinion leaders may place inappropriate weight on the results of a small study, or even the collected experience from a small cohort of patients to tout the efficacy of certain therapies. These results have not always stood the test of time and further examination. While the intentions of researchers who disseminate their findings in this manner may be good, the consequences of doing so without proper review and context can impact our ability to successfully foil Covid-19 and can even harm patients.
The medical research community has a major role to play in helping colleagues and the general public understand the value of having both pathways, while pointing out the differences in confidence and reliability of research results released via each (and in many cases, both). This would help strengthen the level of trust that the general public has in medical research and restore their confidence in data-driven medical practice. The benefit of true advances in the treatment of patients with Covid-19 will only be achieved through a trustable body of medical evidence, delivered by a trustworthy medical process and accepted by a trusting general public.
Transparency and urgency need to go hand in hand with scientific rigor. If we fail to deliver on all three of these fronts, the very same people we are working so hard to protect may not trust our science, potentially impeding our ability to rapidly deliver effective treatments and vaccines to those individuals in greatest need.