What is the difference between clinical trial data and real-world evidence? Let’s look at a new drug being investigated for treatment of MS. Before a drug can be approved by a health authority such as Health Canada, the drug must go through the clinical trial process. If a drug has shown promise, it will likely reach a phase 3 trial involving at least 1,000 individuals. Clinical trials are highly controlled, where study participants take the medication being investigated (or mock drug) as prescribed under the watchful eye of a research team. Before the trial even begins, desired endpoints (such as annual relapse rate, lesion load, disability progression, and brain volume mass) are established and will be used to measure the efficacy and safety of the drug in humans. Data from phase 3 trials that meet the desired endpoints in a statistically significant way, along with safety data reported during the trial, will be assessed by Health Canada in order to make a regulatory decision, or approval.
Once a drug has been approved, post-market studies continue to take place, often by way of a patient registry. Registries provide researchers and clinicians with data from uncontrolled settings, or ‘real-world’ data such as the long-term efficacy of a drug, adherence to drugs, trends in healthcare and employment, comorbidities, drug exposure during pregnancy and additional safety data that had not emerged during clinical trials. Though real-world data can come from a variety of sources such as healthcare system databases, post-market studies or cohort studies, there is a growing number of patient registries. At #ECTRIMS2018, data was presented from over ten patient registries worldwide, here are a few of the registries presented along with their findings:
- The optimal time to start treatment in relapsing-remitting multiple sclerosis patients: results from the Big Multiple Sclerosis Data Network– Danish, Italian, French and Swedish national registries, along with global MSBase Neuro-Immunology registry merged data for a special project with the Big MS Data Network (BMSDN) to identify the optimal time to initiate a disease-modifying therapies (DMT) after MS onset to prevent long-term disability accumulation. The data set included a cohort of 11,871 patients with relapsing-remitting MS and identified that the optimal time to initiate a DMT to prevent long-term disability accumulation was within six months of disease onset. This finding provides additional support for the importance of initiating early treatment following disease onset.
- Familial multiple sclerosis: comparing demographic and clinical characteristics with sporadic form — Using data from MSBase Registry, Dr. Sara Eichau (Spain) and a team of researchers investigated if, and how, familial MS and sporadic MS (no family history of MS) differed. This study included 17,739 patients from 32 MS centres worldwide, over a period of eleven years. Patients were identified as having either familial MS or sporadic MS, and their demographic and clinical characteristics were compared. No differences were found between the groups related to gender or course of MS, however, those with familial MS were found to have a lower age at MS onset and earlier diagnosis and a higher number of relapses, including a higher annual relapse rate within the first five years from disease onset. The familial group also demonstrated a shorter conversion time from relapsing-remitting MS to secondary progressive MS as well as shorter time to reach EDSS 4.0 when compared with the sporadic MS group. The findings from this study will help further understand the genetic role in MS risk.
- Pregnancy and infant outcomes with interferon beta: data from the European Interferon Beta Pregnancy Registry and Population-Based Registries in Finland and Sweden — Dr. Kersten Hellwig (Germany) presented findings from the European Interferon Beta Pregnancy Study Group with data from over 800 pregnancies exposed to interferon treatment. The study group found no evidence that exposure to interferon formulations prior to conception, or during pregnancy negatively impacted pregnancy or infant outcomes. Pregnancy registries provide vital data on drug exposure prior to conception and during pregnancy that could not otherwise be known due to the ethical exclusion of pregnant women in clinical trials.
Registries help inform MS research and assist clinicians in treating MS. If you would like to contribute to MS research through a registry, consider the North American Research Committee on Multiple Sclerosis (NARCOMS), iConquerMS, and MSBase Neuro-Immunology Registry.
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