Scientific Approach to Clinical Research and Practice

the engine of medicine. it drives the discovery of new drugs, new services, and new clinical strategies.

research

new clinic models, telepharmacy, collaborative care.

practice-based innovations

whether you're making new knowledge or just applying it, you must understand research ______________.

principles

research in the biological and medical sciences that aims to improve health.

biomedical research

curiosity-driven, understanding the "why" of biology.

basic research

solving practical problems with basic knowledge.

applied research

testing in humans.

clinical research

how drugs and pharmacy systems affect care.

pharmaceutical practice and policy research

systematic study of fundamental phenomena — the "pure science" tier.

basic research

which type of research: figuring out how sodium channels open/close in neurons (before we even think about seizures or AEDs). it's done in labs, often involving biology, physiology, biochemistry.

basic research

builds the foundation for applied research — applied scientists can't design new drugs without ________________ researchers first explaining the target.

basic

research with a specific, practical goal in mind.

applied research

if basic research discovers how a receptor works, _____________ research asks: "Can we make a drug to target it?"

applied

conducted in animals or other models before humans.

focus areas: pharmacology (drug effects), medicinal chemistry (drug design), and pharmaceutics (drug formulation/delivery).

applied research

the systematic application of knowledge to create new products (drugs, devices, methods).

drug development

creative, structured work to expand knowledge and turn it into useful applications.

research and development

research that directly involves humans or human tissue/behavior.

focus: understanding, preventing, diagnosing, treating, and curing diseases in people.

clinical research

studies mechanisms of disease, drug effects, new interventions.

patient-oriented research

looks at disease patterns, risk factors, health behaviors.

epidemiological and behavioral research

studies effectiveness/efficiency of care delivery (real-world practice).

health services research

the pharmacy lens on health services research. focuses on pharmaceuticals, pharmacy services, and pharmacy systems. multidisciplinary: pulls from clinical, behavioral, economic, organizational, and technological perspectives. examines cost, access, and quality of pharmaceutical care.

pharmaceutical practice and policy research

how drugs are used and their effects in populations.

pharmacoepidemiology

cost-effectiveness of therapies.

pharmacoeconomics

patient results from pharmacy interventions.

pharmaceutical outcomes research

innovation in pharmacy practice (e.g., collaborative MTM clinics, telepharmacy).

pharmacy practice-based research

this type of research creates the evidence base that justifies pharmacists' expanding role in healthcare. without it, we'd be stuck defending our value with anecdotes.

pharmaceutical practice and policy research

all about moving discoveries along the pipeline — from lab → humans → clinical settings → practice → populations.

translational research

lab discoveries → first human testing ("bench to bedside").

T1: translation to humans

patients → clinical guidelines (turning trial results into standard recommendations).

T2: translation to clinical settings

guidelines → everyday clinical practice (doctors, pharmacists, nurses actually using them).

T3: translation to practice

practice → populations (studying effectiveness at the community and public health level).

T4: translation to populations

head-to-head testing of different options to see which one actually works better (and for whom).

comparative effectiveness research

compares benefits vs harms of alternative methods for prevention, diagnosis, treatment, or care delivery. goal: optimize patient outcomes by making decisions based on real comparisons, not just "this drug works in theory."

comparative effectiveness research

evaluation of alternatives (Drug A vs Drug B, pill vs injection, telehealth vs in-person).

comparative effectiveness

as data grows, it's easier to directly compare therapies. there's a rising demand for treatment comparisons so clinicians can choose smarter. the real audience = decision-makers (providers, payers, policymakers) at both the individual (patient-level) and system (population-level).

example: should Medicaid cover Drug A or Drug B for diabetes? CER provides the evidence.

comparative effectiveness analysis

instead of focusing only on disease or drugs, it centers patients' voices and preferences. patients and caregivers are included in research → helping them make informed decisions.

patient centered outcomes research

funds this work in the U.S.

example: a patient with arthritis might care less about lab results and more about whether a new med lets them walk their dog without pain. that outcome is equally important.

the Patient-Centered Outcomes Research Institute

knowledge must be based on observation and experience (not vibes or authority).

empiricism

no bias — data collection, design, and analysis must be neutral.

objectivity

provides the framework to ask meaningful questions (why we think X might affect Y).

theory

protects human subjects, ensures moral responsibility, guided by IRBs and federal regulations.

ethics

select all that apply:

what are the four steps in the process of scientific inquiry?

1. pose a research question + hypothesis.

2. develop and implement a research plan.

3. collect and analyze data.

4. report findings.

good ________________ come from clinical practice, policy gaps, current issues, or theory.

research questions

a strong question has to pass the ________________:

feasible (can it realistically be studied?)

interesting/novel (does it add something new?)

ethical (safe, approved, protects subjects)

relevant (to pharmacy, medicine, or society).

FINER test

do you have funding, expertise, access to patients/data?

feasibility

does it contribute beyond what's already known?

novelty

governed by IRBs, must follow federal rules.

ethics

must matter to pharmacy practice and stakeholders (patients, providers, payers, policymakers).

relevance

how science moves from lab to lives:

translational research makes discoveries useful.

_____________________ shows which options are best.

PCOR ensures patients' voices shape care.

the scientific method and FINER criteria ensure research is solid, ethical, and impactful.

comparative effectiveness research

this framework is like a recipe for building a sharp, testable clinical research question.

PICOTS

kids, adults, elderly, or specific disease groups.

population

med, device, service, policy.

intervention

placebo, usual care, or another treatment.

comparator

economic (cost), clinical (mortality, morbidity), or humanistic (quality of life).

outcomes

short-, intermediate-, long-term.

timeline

outpatient, inpatient, community.

setting

instead of saying "does drug X work?" you'd ask:

"In elderly patients with hypertension (________________), does drug X (intervention) compared to drug Y (comparator) improve quality of life (outcome) over 12 months (timeline) in an outpatient setting (setting)?"

population

research often focuses on economical, clinical, and humanistic outcomes

ECHO

outcomes: costs of care (direct and indirect).

economic

outcomes: morbidity/mortality, safety, effectiveness.

clinical

outcomes: patient-reported quality of life, functional status.

humanistic

__________ for research: some outcomes are short-term (blood pressure drop after 4 weeks) vs long-term (reduced stroke risk over 10 years).

timeline

classification of research: just describing what is (prevalence of diabetes in Houston).

descriptive

classification of research: testing relationships between variables (does obesity increase diabetes risk?).

analytical

classification of research: specifying an expected relationship (new drug ↓ HbA1c more than standard care).

hypothesis-driven

usually rooted in existing theories and evidence.

example: pathophysiology of diabetes + pharmacology of GLP-1 agonists → semaglutide improves glycemic control and reduces weight.

the expected relationship is often based on biomedical or sociobehavioral theory.

hypotheses

blueprint for answering your question.

research design

strongest (randomized controlled trials, or RCTs). test causality directly.

experimental research design

weaker (cohort, cross-sectional). they don't control variables but show associations. useful for real-world data.

observational research design

gold standard research design, but costly and not always feasible.

randomized clinical trial

cohort or cross-sectional. give insight into real-world practice, but prone to bias/confounding.

observational study design

example of a study design:

retrospective study on immunotherapy duration in lung cancer.

population: NSCLC patients on immunotherapy.

exposure: stopping at 2 years vs continuing indefinitely.

outcome: survival.

design: _____________________

key finding: stopping at 2 years gave similar outcomes to indefinite continuation → saves cost, reduces unnecessary treatment.

cohort (observational)

PICOTS helps craft a strong research question.

ECHO outcomes make sure the question is clinically, economically, and humanistically relevant.

hypotheses guide the direction, grounded in biology and prior research.

study design determines the strength of evidence ( _____________ > observational).

ultimately, all of this is about creating useful, credible, patient-centered evidence.

randomized clinical trial

how you collect and measure data.

research methodology

collect new data (surveys, lab tests, interviews, clinical assessments).

primary methods

use existing data (medical records, insurance claims, Medicare data).

secondary methods

the toolbox for answering your research question with numbers.

statistics

summarize data (means, medians, modes).

descriptive statistics

test hypotheses, generalize findings (t-tests, ANOVA, regression, Cox models).

inferential statistics

reports (journal articles, posters, abstracts) communicate findings. all credible research is ___________________ (other experts critique it before publication).

peer-reviewed

typical structure = IMRD format:

introduction: background, rationale, objective.

methods: how study was done.

results: data + analysis.

__________________ : interpretation, implications, limitations

discussion

visual tool to show survival probability over time.

Kaplan-Meier survival curves

multivariable regression model for survival data.

Cox proportional-hazard model

methodology matters — design + data collection drive credibility.

___________________ turn raw data into meaningful results.

reports communicate findings in a structured way (IMRD).

example study in the slides shows how real-world secondary data can identify safety risks (and potentially shape prescribing guidelines).

statistics

using the best scientific evidence to guide healthcare decisions for both individuals and populations.

evidence based medicine

goal of ________________ research: provide evidence that improves health and functioning.

clinical

delivering care that improves outcomes and aligns with professional knowledge.

quality in healthcare

select all that apply:

what are the six qualities of good healthcare?

1. safe

2. effective

3. patient-centered

4. timely

5. efficient

6. equitable

avoids harm.

safe

treatments should actually benefit patients.

effective

respects individual preferences and values.

patient-centered

doesn't delay needed care.

timely

avoids waste of resources.

efficient

no discrimination—care should not vary by race, gender, income, etc.

equitable

__________________ drives EBM. without good research, you're just guessing.

research evidence is the backbone—everything else flows from it.

goal: use relevant evidence to deliver highest quality patient care.

science

select all that apply:

what are the four steps in EBM?

1. ask an answerable question

2. find evidence

3. appraise evidence critically

4. apply to practice

this structure forces you to ask specific, testable questions. example: "in older adults with dementia and OAB, are selective antimuscarinics safer than non-selective ones?"

PICO method

the PICO method:

P = __________________

I = intervention

C = comparator

O = outcome

patient/population

are the study methods sound? did they avoid bias?

internal validity

can the findings apply to my patients, or was the study population too different?

external validity