How do we know what causes disease? How do we know if a treatment works? Behind every medical breakthrough is a carefully designed investigation. Learning to think like a scientist — to ask good questions, design fair tests, and interpret evidence — is one of the most powerful skills you can develop.
Imagine you notice that students who eat lunch in the cafeteria get stomach aches more often than those who bring lunch from home.
Write down your answers before reading on:
Asking and answering questions
Global Disease
Investigating disease follows the same scientific method used in all science:
In disease research, forming a good question is crucial. Questions must be:
Different tools for different questions
Different research questions require different approaches:
Case-control study:
Compares people who have a disease (cases) with similar people who do not (controls) to identify risk factors.
Cohort study:
Follows a group of people over time to see who develops disease.
Randomised controlled trial (RCT):
The gold standard for testing treatments. Participants are randomly assigned to treatment or placebo (control) groups.
Making sense of numbers
Collecting data is only half the task — interpreting it correctly is equally important:
Measures of disease frequency:
Understanding risk:
Correlation vs causation:
Just because two variables are correlated does not mean one causes the other. Ice cream sales and drowning deaths are correlated (both increase in summer), but ice cream does not cause drowning — hot weather causes both.
Establishing causation requires:
Doing good science responsibly
Medical research involves unique ethical challenges because it affects human lives:
Informed consent: Participants must voluntarily agree after understanding risks, benefits, and their right to withdraw. For children or unconscious patients, parents or guardians provide consent.
Beneficence: Research must benefit participants or society. The potential benefits must outweigh the risks.
Non-maleficence: Researchers must not harm participants. "Do no harm" is a fundamental principle.
Justice: Research benefits and burdens must be distributed fairly. Historically, vulnerable groups (prisoners, poor people, Indigenous communities) were exploited in research. Modern ethics requires equitable inclusion and protection.
Animal research: Many disease treatments are tested in animals before human trials. This raises ethical questions about animal welfare. Australian research follows the 3Rs principle: Replace animals where possible, Reduce numbers used, and Refine procedures to minimise suffering.
Research integrity: Fabricating data, plagiarising, or failing to report negative results undermines trust in science and can harm patients.
"A single study can prove that something causes disease." No — establishing causation requires multiple studies using different methods, consistency across populations, and biological plausibility. A single study can suggest associations but rarely proves causation.
"If two things are correlated, one must cause the other." No — correlation does not imply causation. Many correlated variables are both caused by a third factor, or the correlation may be coincidental.
The National Health and Medical Research Council (NHMRC): Australia's peak body for funding health and medical research and developing ethical guidelines. The NHMRC's National Statement on Ethical Conduct in Human Research sets standards for all Australian research involving humans.
Indigenous research ethics: The NHMRC has specific guidelines for research involving Aboriginal and Torres Strait Islander peoples, developed in partnership with Indigenous communities. Key principles include: community engagement, benefit to communities, cultural sensitivity, and Indigenous control over data. Researchers must demonstrate that their work will benefit the communities involved, not just advance scientific knowledge.
Clinical Trials Australia: Australia is a leading destination for clinical trials due to high-quality healthcare, rigorous ethical oversight, and diverse populations. The Therapeutic Goods Administration (TGA) regulates medicines and medical devices, ensuring safety and efficacy before approval. During COVID-19, Australian researchers conducted trials for vaccines and treatments that contributed to global pandemic response.
1. What is a hypothesis?
2. In a case-control study, researchers compare:
3. Why are randomised controlled trials considered the gold standard for testing treatments?
4. "Correlation does not imply causation" means:
5. Which ethical principle requires that research participants voluntarily agree after understanding risks?
1. Describe the difference between a case-control study and a randomised controlled trial. Include one advantage and one limitation of each. 4 MARKS
2. Explain why "correlation does not imply causation" using a disease-related example. 4 MARKS
3. Evaluate the ethics of testing a new vaccine on children before it has been tested on adults. Consider both the potential benefits and the ethical concerns. 4 MARKS
Go back to your Think First answer. Has your understanding changed?
B — A hypothesis is a testable prediction about the relationship between variables that can be investigated through observation or experiment.
B — In a case-control study, researchers compare people who have a disease (cases) with similar people who do not (controls) to identify risk factors.
B — Randomised controlled trials eliminate bias by randomly assigning participants to treatment or control groups, ensuring that differences in outcomes are due to the treatment rather than other factors.
B — This means that just because two variables change together does not mean one causes the other. A third factor may cause both, or the relationship may be coincidental.
C — Informed consent is the process by which participants voluntarily agree to participate in research after understanding the risks, benefits, and their right to withdraw.
Model answer: A case-control study compares people who have a disease (cases) with similar people who do not (controls) to identify risk factors. Advantage: efficient for studying rare diseases and can examine multiple risk factors simultaneously. Limitation: cannot prove causation because it looks back in time; researchers cannot control exposures, and recall bias may affect results. A randomised controlled trial (RCT) randomly assigns participants to treatment or control groups and compares outcomes. Advantage: randomisation eliminates selection bias, and the control group provides a direct comparison, allowing researchers to establish causation. Limitation: expensive, time-consuming, and ethically constrained — it may be unethical to withhold effective treatments or expose participants to potential harm.
Model answer: "Correlation does not imply causation" means that just because two variables are associated does not mean one causes the other. A disease-related example: studies have found that people who consume more red wine have lower rates of heart disease. However, this does not prove red wine prevents heart disease. Red wine drinkers may have higher incomes, better diets, or more exercise — factors that independently reduce heart disease risk. The correlation may reflect these confounding variables rather than a causal effect of wine. Another example: ice cream sales and drowning deaths are correlated because both increase in summer, but ice cream does not cause drowning — hot weather causes both. In disease research, establishing causation requires controlled experiments, biological plausibility, and consistency across multiple studies.
Model answer: Testing a new vaccine on children before adult testing raises significant ethical concerns. Potential benefits: Children are vulnerable to infectious diseases and may need vaccine protection urgently. Some diseases (like rotavirus) primarily affect children, so adult testing would not provide relevant data. Early testing in children could lead to faster protection for this vulnerable group. Ethical concerns: Children cannot give fully informed consent — parents must decide on their behalf, creating a vulnerability. Children's immune systems and bodies are still developing, so side effects may differ from adults. The principle of non-maleficence requires protecting vulnerable populations from unnecessary risk. Standard practice: Most vaccine development follows a staged approach — safety testing in adults first, then gradually younger age groups. This balances the need to protect children with the ethical obligation to minimise risk. Exceptions may occur during emergencies (like COVID-19) when the disease poses greater risk to children than the vaccine. Ultimately, the decision requires careful ethical review, parental informed consent, and strong safety monitoring.
Design experiments and analyse data! Solve disease mysteries by applying scientific methodology in this investigation adventure.
Tick when you have finished all activities and checked your answers.