“I read this on PubMed…”: Navigating Research Articles
Navigating Research Articles for Better Health Decisions
Many of our families spend hours digging through the internet to try to find any promise of improvement to their child’s quality of life with neonatal and pediatric-acquired Hypoxic Ischemic Encephalopathy. There is so much research going on, and very little resources for the layperson to understand the full context, nuance and applicability to what they are reading to their own situation.
Research articles, particularly those involving preclinical and clinical data, can be complex and sometimes misleading if not properly understood. Let’s tackle some of the key concepts to help you better navigate health-related research and make informed decisions.
Preclinical Data vs. Human Clinical Trials: What’s the Difference?
Research typically begins with preclinical studies, which often involve cell cultures or animal models. These studies are crucial for understanding the basic mechanisms of a disease and testing the initial safety and efficacy of a new therapeutic approach. However, it’s important to remember:
- Preclinical Data is Not the Same as Human Data: Results obtained from animal studies do not always translate directly to humans due to physiological and genetic differences.
- Understanding Chemistry, Mechanisms of Action: It’s common to see preclinical data in animals, or for a different diagnosis in adult humans, post the question of something being effective for treatment. These articles typically analyze specific molecules in animal models found in a current medication, but it does not mean that current medication will work for the diagnosis.
- Chemical compounds that are useful for additional diagnoses have to be re-engineered, tested for safety, and then tested for effectiveness in the population studied.
- When talking about neonatal or pediatric populations, adult dosing, or even a compound that’s indicated for one use, may not be effective for the other population, could be dangerous, and shouldn’t be used “off the shelf” based on a report of the potential benefit.
- Clinical Trials are Essential: Clinical trials involve human participants and are necessary to confirm whether a treatment is safe and effective in people. These trials are conducted in multiple phases to rigorously test the new therapy.
Preclinical Animal Data: Promise and Pitfalls
Preclinical studies are an important first step in the development of new therapies. They help scientists understand how a treatment works and identify potential side effects. However, there are some key points to consider:
- Success in Animals ≠ Success in Humans: A significant number of treatments that show promise in animal models fail when tested in humans. For example, while animal studies may suggest a treatment is effective, it might not work in humans due to differences in biology.
- Variability in Success: According to some estimates, only about 10-15% of drugs that enter clinical trials after successful preclinical testing in animals end up being safe, effective and approved for human use. This highlights the complexity of translating findings from animals to humans.
- For HIE, there are different models: HIE has several animal models to test different molecules and mechanisms — small: mice and rats, to large: sheep, pigs and primates, to human organoid models with human cells. Because so many therapeutics have failed in HIE, regulatory bodies like the FDA and EMA are calling on therapeutic developers to show promise in both the small and larger animal models before heading to human trials.
- If you read an article talking about mouse trials, it is NOT ready for prime time in humans and may not translate! This is why there are many regulations behind clinical trials.
Headlines and Individual Diagnosis: One Size Doesn’t Fit All – Read Past the Headline
Media and research headlines about new medical breakthroughs can be exciting but also misleading. It’s important to approach these headlines with a critical eye:
- Context Matters: A study’s findings may not be applicable to everyone. For example, a headline about a new cancer treatment may only be relevant to specific types of cancer, stages of the disease, or particular patient populations.
- Populations Matter: Pulling a study from one area of the world may be completely irrelevant to the population you are in. For example, therapeutic hypothermia has been shown to be harmful in the HELIX study — from India, Sri Lanka and Bangladesh — but it is effective in other low and middle income countries like those in South America, and in many high-income countries.
- Safety does not mean it’s Effective: A therapy or medication may be deemed “safe”, but that doesn’t mean it is effective! Many families will share basic safety articles about a treatment or medication that have no evidence, or have been disproven to be effective.
- Personalization of Care: Each individual’s situation is unique. Factors such as age, overall health, genetic background, and other treatments being taken can influence how a new therapy might work for them.
The Dangers of Self-Medication
Taking medications not indicated for your specific diagnosis can be dangerous for several reasons:
- Safety Concerns: Medications are tested and approved for specific conditions. Using them for an unapproved purpose can lead to unexpected side effects or interactions with other medications you may be taking.
- Lack of Efficacy: A drug that works for one condition may not be effective for another. Using it without proper medical guidance can delay effective treatment and worsen your health.
- Professional Guidance is Crucial: Always consult a healthcare provider before starting or changing any medication, including supplements and over-the-counter medications. They can provide personalized advice based on your unique health needs.
Safety Over Everything!
Our families and children have been through enough. The last thing we want is for our families to experience further trauma, financial instability, and preventable harm. We ALWAYS recommend you bring what you read to your medical team to explain how it may or may not be beneficial to your child!
Helpful Resources to Build Health Literacy Skills:
Hope for HIE is dedicated to helping families build health literacy skills to empower them in advocating for the best quality of life for children impacted by HIE. All of our educational materials are vetted by Hope for HIE’s Medical Advisory Board members to ensure families have evidence-based, data-driven information to make important decisions for their children and families.
