Utilizing Real-World Data to Examine Outcomes in Patients With Amyotrophic Lateral Sclerosis

It is well established that randomized clinical trials (RCTs) are the gold standard for effectiveness and safety in clinical research.¹

In amyotrophic lateral sclerosis (ALS), disease heterogeneity and historically poor survival rates, among other factors, make it a clinically challenging disease to research and evaluate in clinical trials.² Research studies analyzing real-world data (RWD) may be able to provide additional information to the application of evidence from RCTs and inform insights beyond those addressed by the RCTs.³

RWD is defined as data derived from sources other than traditional RCT that relate to a patient’s health status and/ or the delivery of health care. RWD is collected from a variety of sources such as electronic heath records, medical and billing (administrative) claims, and product and disease registries.⁴ Real world evidence (RWE) is the clinical evidence regarding usage and potential benefits or risks of a medical product derived from analysis of RWD and is generated according to a research plan.³

RWD can be particularly helpful in exploring gaps in evidence around patient’s experience with therapeutic approaches in everyday medical practice and how treatment impacts their day-to-day lives. RWD can be used to compare multiple alternative interventions to inform treatment choice; examine outcomes in a more diverse study population and follow a larger cohort over a longer period. Additionally, RWD can be used to inform therapeutic development, research on health care systems, quality improvement, safety surveillance, and well-controlled effectiveness studies.⁵ Stakeholders including clinicians, formulary decision makers, regulators and clinical guideline developers who are tasked with determining the application of evidence to specific populations, may decide to turn to RWE to inform their decision making that previously had been guided only by results from RCTs in more narrowly defined populations.³ Taken together, RWD and data obtained from the RCT may provide additional information after approval.²

Despite the growing spectrum of its uses, RWD can be difficult to interpret due to its lack of randomization, unknown or unmeasured confounding variables and use complex statistical analysis techniques³ therefore understanding the utility and limitations of RWD is critical to proper application of insights.⁴ Furthermore, RWD studies ordinarily cannot determine definitive causal conclusions about the effects of treatment.³

As a leader in developing treatments for patients with ALS, MTPA is exploring the ability of RWE to provide insights into current treatment options including disease progression, time to event measures, treatment duration, and outcomes that are not typically captured in RCTs such as patient reported outcomes and caregiver burden.  It is hoped that examining RWD alongside RCT data will be helpful in uncovering key evidence around how patients with ALS respond to therapy and how treatment impacts their day-to day lives.

While our real-world research efforts in ALS are continuing to evolve, we look forward to sharing more details about our methodologies and findings with the ALS scientific community and we look forward to working together with the community to align on the opportunities and challenges that RWD presents and find ways to effectively incorporate these insights to help guide research into new therapies and optimize treatment selection for patients with ALS.