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Planning Critical Research
To understand whether a treatment works and is safe, doctors and reviewers like Health Canada use data from research studies.
Researchers need to decide many things before starting a study. They must decide exactly what kinds of patients they will look at, how many they will include, exactly how they will treat patients, and how they will measure outcomes.
Study Endpoints
One important thing that researchers must decide are the study Endpoints. These are the key outcomes that researchers will measure to decide whether a treatment worked as intended. The most important result is called the Primary Endpoint.
Different studies can use different endpoints. Some examples are:
• Patient answers to a series of standardized questions
• Average number of new lesions in an MRI brain scan
• Percentage of patients in remission
In some cases, multiple measures can be combined into one endpoint.
NEDA = “No Evidence of Disease Activity”
In multiple sclerosis research, multiple measures of how patients are impacted have been combined into one endpoint called:
NEDA, No Evidence of Disease Activity.
What is NEDA?
In 2006, researchers were testing whether a drug was effective at treating relapsing-remitting multiple sclerosis (RRMS). They designed their clinical trial with two primary endpoints: Rate of relapse after 1 year and probability of worsening disability after 2 years. They also looked at the number and size of lesions using MRI.
After collecting and analyzing the data, researchers were encouraged and wanted to know how well the drug worked if they combined the endpoints into one, more holistic measure of the effect it had on the disease.
The type of combined endpoint they came up with continues to be used and has come to be called NEDA (commonly pronounced: “NEE-dah”).
For a RRMS, NEDA commonly
means that a patient has:
1
No relapses
2
no worsening
of disability
3
no new or growing
lesions on their
brain scans
More and more clinical and real-world studies are using NEDA as one of their endpoints, although the specific definition of NEDA used in each study can vary as determined by the researcher team. Because NEDA combines different measures of how well a drug works, it is said to be a more stringent efficacy endpoint.
Ask your doctor about the data behind your treatment.
You might be wondering if your treatment has been shown to achieve NEDA.
Keep in mind that interpreting and comparing clinical data can be difficult. Your doctor can help. He or she is probably very familiar with the data supporting your prescribed treatment, and they can help you understand more about the clinical and real-world trials.
Ask your doctor whether your treatment has been shown to achieve NEDA in either clinical trials or in recent analyses of standard clinical practice. Discuss whether no evidence of disease activity is an obtainable goal for your MS.
Check your knowledge!
What did you learn?
True or false?
All medications for MS are measured with the same outcomes (efficacy endpoints) in clinical research studies.
Not true! Researchers design clinical and real-world studies in different ways. Studies use a variety of individual endpoints that are relevant to patients and doctors, like relapses or disability progression.
Some trials use NEDA (no evidence of disease activity), considered to be a more stringent efficacy endpoint because it combines measures like relapse, disability, and brain lesions into one.
Discuss with your doctor the clinical trial data behind your treatment.
That’s right! Researchers design clinical and real-world studies in different ways. Studies use a variety of individual endpoints that are relevant to patients and doctors, like relapses or disability progression.
Some trials use NEDA (no evidence of disease activity), considered to be a more stringent efficacy endpoint because it combines measures like relapse, disability, and brain lesions into one.
Discuss with your doctor the clinical trial data behind your treatment.
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References:
1. Havrdova E, Galetta S, Hutchinson M, et al. Effect of natalizumab on clinical and radiological disease activity in multiple sclerosis: A retrospective analysis of the Natalizumab Safety and Efficacy in Relapsing- Remitting Multiple Sclerosis (AFFIRM) study. Lancet Neurol. 2009;8:254-60.
2. Polman CH, O’Connor PW, Havrdova E, et al. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med. 2006;354(9):899-910.
3. Perumal J, Balabanov R, Su R, et al. Natalizumab in early relapsing-remitting multiple sclerosis: A 4-year, open-label study. Adv Ther. 2021;38(7):3724-3742.
4. Prosperini L, Saccà F, Cordioli C, et al. Real-world effectiveness of natalizumab and fingolimod compared with self-injectable drugs in non-responders and in treatment-naïve patients with multiple sclerosis. J Neurol. 2017;264:284-294.
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