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Abstract Details

Real World Data as a control group, an ALS case study
P10 - Poster Session 10

In clinical research, the gold standard is to use a randomized controlled trial (RCT) to assess the effect of a treatment.

Thanks to the effort of many centers and institutes, the availability of retrospective data about Amyotrophic lateral sclerosis (ALS) is growing.

Since it’s a rare disease, enrolling patients for a study is always challenging, and providing the placebo to patients raises ethical questions.

Propensity score matching (PSM) aims to equate treatment groups concerning measured baseline covariates to achieve a comparison with reduced selection bias. It is a valuable statistical methodology mimics the RCT and may create an "apples to apples" comparison while reducing bias due to confounding.
Assess the effect of treatment with Real World Data (RWD) using Propensity Score Matching

Ceftriaxone injection treats certain infections caused by bacteria.

The ALS/MND Natural History consortium, comprising nine ALS clinics, collects real-world data on patients with ALS/MND, including medical history, medication, longitudinal outcomes, survival, and demographics. To extract causal inferences from this observational data set, PSM was used to create a dataset balanced on common factors known or believed to associate with ALS survival and progression and inclusion and exclusion criteria.

P-values were calculated using the Sharp Null Hypothesis and simulation rather than asymptotically.

First results show no differences in survival and ALSFRS-R functional between patients who received CEFTRIAXONE and matched patients from RWD. These results are the same as from the RCT.

Thanks to the matching methodologies, RWD can be used as the control group avoiding the placebo.
Matteo Locatelli, BS (MGH)
Mr. Locatelli has nothing to disclose.
Alexander Sherman (Massachusetts General Hospital) The institution of Mr. Sherman has received research support from The ALS Association. The institution of Mr. Sherman has received research support from NIH. The institution of Mr. Sherman has received research support from FDA. Mr. Sherman has a non-compensated relationship as a Member, Board of Directors with ALD Connect that is relevant to AAN interests or activities.
Alex Berger, BS Mr. Berger has nothing to disclose.