"Data analysis of 500,000 patients…clinical utility of choline alfoserate reconfirmed"

'Choline alfoserate expert forum' reviewed a large-scale real-world data (RWD) analysis

"Addresses the structural limitations of previous Italian RCT study…strengthened the evidence for choline alfoserate's clinical efficacy"

"This study aligns with the recent government stance on utilizing RWD…has established clinical evidence for real-world clinical practice"

'Choline alfoserate expert forum' recently hosted by DailyPharm

A large-scale real-world data (RWD) analysis of 500,000 Korean patients with mild cognitive impairment (MCI) has reconfirmed the clinical utility of choline alfoserate in reducing the risk of progression to dementia.

Experts evaluate that this study significantly strengthens the evidence base for prescribing choline alfoserate. It is considered academically meaningful because it revalidated the efficacy evidence from existing randomized controlled trials (RCTs) using massive datasets from actual clinical settings.

Analysis of 500,000 Korean MCI patients...“Reduction in risks for both Alzheimer’s disease‧vascular dementia”

At the 'Choline alfoserate expert forum' recently hosted by DailyPharm, Professors Han-kyeol Kim (Wonju Severance Christian Hospital), Choi Hojin (Hanyang University Guri Hospital), Lee Chan Nyung (Korea University Anam Hospital), and Kim Geon Ha (Ewha Womans University Mokdong Hospital), all neurologists, focused on the results of the study titled 'Effect of Choline Alfoserate: NHIS Cohort Study,' published last year.

Professor Han-kyeol Kim (Wonju Severance Christian Hospital)

Professor Han-kyeol Kim, who delivered the keynote presentation, introduced the research findings based on the National Health Insurance Service (NHIS) big data, which followed 508,107 patients newly diagnosed with MCI between 2013 and 2016.

The study results showed that the choline alfoserate user group had a significantly lower risk of progression to Alzheimer’s dementia by 10.1% (HR 0.899) and vascular dementia by 16.8% (HR 0.832) compared to the non-user group. Furthermore, the risk of ischemic stroke decreased by 16.7% (HR 0.833), and the risk of hemorrhagic stroke decreased by 15.3% (HR 0.847).

Professor Han-kyeol Kim said, ​"This was not a simple statistics of prescriptions. We linked health examination data to precisely adjust for variables such as smoking, alcohol consumption, income, and chronic diseases (hypertension, diabetes, dyslipidemia), as well as the duration of drug exposure to enhance objectivity," and added, "The study confirms that the use of choline alfoserate can be effective in reducing the risk of dementia progression and stroke in MCI patients, serving as a useful option for early intervention."

Combining the scientific achievement of RCTs with the confirmed RWD… “Completing complementary evidence for dementia suppression”

A discussion continued, chaired by Professor Choi Hojin, with Professors Lee Chan Nyung and Kim Geon Ha in attendance. They noted that this RWD study addresses the structural limitations of previous RCTs.

The previous 'ASCOMALVA' study was designed as a multicenter, randomized, double-blind, controlled trial (RCT). The study targeted 210 patients with Alzheimer’s disease dementia accompanied by ischemic stroke, comparing a donepezil monotherapy group with a choline alfoserate + donepezil combination therapy group. The results confirmed significant improvements in the combination group across ▲the Mini-Mental State Examination (MMSE) ▲ Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-cog) ▲Instrumental Activities of Daily Living (IADL). Long-term follow-up over four years also showed that the rate of cognitive decline was inhibited in the combination therapy group.

Experts agreed that this RWD study supplements the limitations of the RCT. Professor Choi Hojin evaluated, "The evidence for the efficacy of choline alfoserate has become even more robust by adding the analysis results of a large-scale Korean patient population to the ASCOMALVA findings," and added, "Results tracking the effects of drugs prescribed in the real world over a long period are highly meaningful as evidence from actual clinical practice, distinct from RCTs conducted in controlled environments."

Professors Choi Hojin (Hanyang University Guri Hospital) ·Lee Chan Nyung (Korea University Anam Hospital) ·Kim Geon Ha (Ewha Womans University Mokdong Hospital)

Professor Lee Chan Nyung stated, "While RCTs are suitable for proving drug efficacy in standardized environments, it is not easy to confirm actual conversion rates in slowly progressing diseases like dementia due to limitations in sample size and follow-up duration," and "This RWD study serves as powerful complementary evidence to existing RCTs by reaffirming the outcome of 'inhibiting dementia progression' through data accumulated over several years in actual clinical settings."

​“MFDS policy to enhance RWD reliability…increasing potential for utilization in clinical practice”

Experts also emphasized that this study aligns with the recent government stance on the importance of RWD. In this regard, the Ministry of Food and Drug Safety (MFDS) announced the 'Guidelines for the Utilization of RWD/RWE in the Approval and Licensing of Medicines, etc.' in June 2024. Through this, the ministry presented specific criteria for actively recognizing RWD as clinical evidence for indication expansion or efficacy validation. In contrast, it had previously been used only as a supplementary tool for post-marketing surveillance (PMS) of side effects.

The view among experts is that RWD research, which precisely analyzes NHIS big data from over 500,000 citizens, can yield new clinical evidence amid these changing regulatory environments. Professor Lee Chan Nyung emphasized, "As the vast RWD figures of 500,000 show, the dementia suppression effect confirmed in a large-scale patient group will serve as crucial evidence that gives clinical specialists the confidence to prescribe."

Professor Kim Geon Ha said, "The 16.8% lower risk of progression to vascular dementia shown in this study holds great significance for the Korean elderly population with high vascular risk factors such as hypertension," and Kim concluded, "It raises the level of evidence for preemptive treatment of MCI patients in actual clinical settings."

Professor Choi Hojin highlighted the public health significance of early intervention at the MCI stage. Choi added, "Delaying the onset of dementia by even just a few years can drastically reduce national medical costs and the caregiving burden on patients' families. Preemptive response through proven options will be a practical solution to alleviate the dementia burden in an aging society."