Dare to Dream REALworld: AML
Your experience with pediatric AML can help move care forward
Nevaeh
leukemia survivor
REAL stands for Real-world Evidence Accelerating New Treatments. The initiative was created to address a very important, very urgent problem:
New treatments for pediatric acute myeloid leukemia (AML) aren’t being developed quickly enough. By choosing to share electronic health records from your real-world experience, families and survivors can help researchers learn faster and work toward safer, more effective options.
More information coming soon.
What is Dare to Dream REALworld: AML?
A new way to approach pediatric AML clinical trials
Clinical trials are necessary to make sure new treatments are safe and effective. But trials for rare diseases, like pediatric AML, present challenges. With how few patients get diagnosed, it takes a long time to enroll in a randomized trial, which can make them expensive, lengthy, or difficult to complete.
Blood Cancer United is changing this through Dare to Dream REALworld: AML, and we need your help. By helping to build out our real-world evidence dataset, you help doctors and researchers learn from real-world experiences—so new treatments can be developed and evaluated faster for children and adolescents with AML.
How real-world evidence speeds up clinical trials
Clinical trials are broken up into three phases, designed to determine if investigational drugs are safe, test their efficacy, and benchmark them against the standard of care.
Phase 1 Trial
Key question
Is the investigational drug safe?
- Number of patients: 10 – 50
- May be the first trial of the drug in humans (or in specific age categories)
- May compare two or more different doses (strengths) of the drug, which helps determine the optimal dosage and tolerability
Goals
- Determine possible side effects of the drug
- Identify the pharmacokinetic profile (how the drug affects or moves through the body)
Phase 2 Trial
Key question
Is the investigational drug effective?
- Number of patients: 25 - 75
- Assesses the frequency and severity of side effects in a larger sample size
- Measures the patients’ response to the drug (how well it works)
Goals
- Establish that investigational drug produces the desired outcome and baseline efficacy
- Begins to establish the potential that the investigational drug may produce better outcomes than existing treatments (standard of care)
Phase 3 Trial
Key question
How does the investigational drug compare to the standard of care?
- Number of patients: 50 – 100+
- Continued assessment of frequency and severity of side effects in an even larger sample size
- Measures patient response to the drug (how well it works) compared with existing treatments (standard of care)
- Usually a randomized clinical trial (RCT)
- Some patients receive new drug and some receive standard of care
- Patients assigned to treatment arm randomly
Goals
- Establish clear evidence that the investigational drug produces better outcomes for patients than the standard of care
When many families choose to share their health records, that real-world information can be used as a comparison in future studies (by acting as a control group).
As a result, more patients would be able to get the new treatment, and it speeds up the testing and approval process. This approach follows FDA standards and does not compromise patient safety.
How real-world evidence can improve...
Phase 1 Trials
Data can be queried to establish benchmarks and inform the statistical plan for the trial
Determines an acceptable frequency of side effects for the new drug
Phase 2 Trials
Data can be queried to evaluate effectiveness of standard of care
Determines appropriate benchmarks for efficacy of the new drug
Phase 3 Trials
Data can be used to find “matched” controls (e.g., patients with the same disease and similar demographics who received the standard of care)
Serves as a "virtual" control group in typical randomized clinical trials, so all enrolled patients receive the investigational drug and the study can be completed much more quickly
Help create more life-saving treatments
Why families and survivors choose to participate
- To help speed up the development of desperately needed treatments for pediatric AML
- To contribute to research through a groundbreaking trial that can serve as a template for future clinical trials
- To stay organized, keep all your records in one place, and share information with care teams, all in one convenient app
- To access reliable education and resources from Blood Cancer United
- To know your experience is helping improve care for the next family
Put simply, it’s a way to speed up clinical trials. By using real-world data from people who opt in, researchers can gather information and draw conclusions that can normally only be answered by large, randomized clinical trials. It can make clinical trials faster and more efficient, leading to the development of new therapies more quickly.
Whether you are a patient or are the parent of one, signing up means that you help build the control group for pediatric AML clinical trials, just by going through a normal treatment plan. This helps researchers get information they need for how the standard of care works.
Email [email protected] to signify your interest in getting involved. When the platform is ready to accept patients, which we expect will happen in Summer 2026, you will be notified of the next steps.
Everything will be done through the olivia app, which you can either download or use on a web browser. Anyone who signs up for the app will receive free, personalized information from Blood Cancer United that is specific to their own needs, plus it aggregates health information across all the hospitals and health systems that a patient visits and has an AI interface that lets you ask questions that receive personalized answers.
Yes! The safety of your child is our number one priority. That applies to the information that you’re sharing to make this project possible.
You, your care team, and researchers who need this information can access it for free. Commercial companies that pay a fee can also access it. Before any other people access your data, your (or your child’s) name and any other identifying information will be removed.
Yes. You can opt out at any time by reaching out to [email protected] and expressing that you would like to unenroll.
The program is free to join, as is access to the olivia app and all of its features.
It is! Using real-world evidence is an FDA-approved approach—for more information, head here.
While you do not need their permission, we would highly encourage anyone who is interested in participating to first speak with their care team about enrolling.
For now, our focus is on using this for pediatric AML clinical trials. However, we are confident that this can serve as a blueprint that others can follow to make clinical trials for other rare diseases faster, more efficient, and more cost-effective.
Yes! Thank you for wanting to help inform the project. Please reach out to [email protected] for next steps.
Thank you for wanting to support this initiative, and we are sorry for your loss. Your child’s information can be used in this, and you can contact [email protected] to learn more about how you can help.
It can, so long as you were diagnosed when you were 21 or younger. To express your interest, please reach out to [email protected].
Blood Cancer United’s top priority is to find better, safer treatments for people living with blood cancer. We’ve worked with patients, researchers, healthcare providers, and drug developers—all of whom are necessary for this project to succeed. And our work with both patient data and clinical trials means we understand the complexities that come with this project, and are committed to making sure anyone who participates feels safe.
Feel free to email [email protected], and we will do our best to answer your question in a timely manner.
Help transform the treatment of pediatric AML
Better treatments. Better outcomes. And a better process for clinical trials that does not compromise on safety.
All of this becomes possible when your family becomes part of Dare to Dream REALworld: AML. Enroll today and help create a tomorrow with better ways to treat pediatric AML.
More information coming soon.