I want to do research, where do I start ?

Presentation Overview

• Developing a Research Question

• Key Components of Effective Research Questions

• Applying Frameworks to Construct Research Questions

• From Questions to Objectives

• Population Selection and Eligibility Criteria

• Understanding Clinical Trial Endpoints

• Significance of Sample Size and Power in Research

“Research is to see what everybody else has seen, and to think what nobody else has thought.”
- Albert Szent-Györgyi

A well-formulated question is the first step in thinking differently about what we observe

Developing a Research Question

• Identify a gap: reviewing literature, attending conferences, and engaging with the community.
• Formulate the question: frameworks like PICO (Population, Intervention, Comparator, Outcome) and FINER (Feasible, Intersting, Novel, Ethical , Relvent ).
• Statistical considerations from the start — feasibility of study design, data collection methods, and preliminary data analysis plans.

Crafting Research Questions

• Research question: A clearly defined query that your research aims to answer. It guides the focus of your study and is the starting point of your research.
• A well-crafted research question should be specific , feasible, and relevant, addressing gaps in the current knowledge.

Example

• Bad: Is the new radiotherapy technique beneficial for lung cancer patients?

• Better: Investigating the efficacy of high-precision, image-guided radiotherapy (Intervention) in extending survival (Outcome) among patients with advanced lung cancer (Population) compared to the current standard of care (Comparator) over a two-year period (Time).

The importance of Systematic Review

• Foundation for research: lays the groundwork by compiling and analysing all relevant studies on a topic.
• Identifying gaps: uncovering research gaps, which directly informs the development of research objectives.
• Refining research questions: insights from a systematic review can lead to refining the question for clarity and focus

More considerations for good research questions

• What is the appropriate stage for evaluation?
• Can internal validity be achieved?
• To what extent is external validity (generalisability) achievable?
• What will your circumstances permit?
• What can you afford?
• What is the best balance between “idea” and “feasibility”?

Setting Research Objectives

• Research objectives: Breakdown the research question into smaller, manageable goals.

• Defined using SMART criteria: Specific, Measurable, Achievable, Realistic, Time-defined.

• Aim for clarity and specificity to guide your research direction effectively.

Example Research Objectives

• Primary objective:
  • To determine the difference in two-year overall survival rates between stage III non-small cell lung cancer patients treated with high-precision, image-guided radiotherapy versus those treated with conventional conformal radiotherapy.
• Secondary objective:
  • To assess the impact of high-precision, image-guided radiotherapy on the quality of life of stage III non-small cell lung cancer patients at 12 months post randomisation, compared to the impact of conventional conformal radiotherapy.

Population Selection & Eligibility

• Trial phase impact
  • Early phases: Homogeneous populations for focused insights.
  • Later phases: Heterogeneous populations for broader applicability.
• Considerations
  • Safety is paramount.
  • Flexible entry criteria to ensure timely enrolment.

Clinical Trial Endpoints

• Purpose & types
  • Guide trials: Primary (main outcome) and Secondary (supplementary insights).
  • Common types: Continuous, Categorical, Event-time.
• Characteristics of primary endpoint
  • Clinically relevant, interpretable, sensitive, practical, and validated.
• Considerations
  • Objective vs. Subjective: Importance of blinding.

Common endpoints in Cancer research

• Overall survival: Gold standard primary
• Complete response/ Objective response rate: A surrogate marker
• Progression-free survival: A surrogate marker, common in phase 2 trials
• Health related quality of life

Sample size/power

• Sample size determination
  • Balances precision, feasibility, and resources.
  • Influenced by expected effect size, variability, and significance level (α).
• Power (1-β)
  • Probability of detecting a true effect.
  • Aim for 80-90% power: Minimises risk of Type II error (failing to detect a real effect).
• Impact on clinical trials
  • Adequate sample size and power ensure reliable, generalisable results.

Summary

• Start with an idea
• Use frameworks to get a research question
• Revise this question with literature review
• Start to consider populations and outcomes
• Talk to a statistician as soon as possible