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IDF-BRiDGES Translational Research Grants Course. BRIDGES, an IDF project supported by an educational grant from Eli Lilly and Company Course and Materials developed by K.M. Venkat Narayan and Mary Beth Weber Rollins School of Public Health, Emory University . Acknowledgements.

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Idf bridges translational research grants course

IDF-BRiDGES Translational Research Grants Course

BRIDGES, an IDF project supported by an educational grant from Eli Lilly and Company

Course and Materials developed by K.M. Venkat Narayan and Mary Beth Weber

Rollins School of Public Health, Emory University


Acknowledgements
Acknowledgements

  • Thoughts, comments, slides, and help from the following are acknowledged:

    • Frank Vinicor

    • Edward Gregg

    • Carol Mangione

    • Monique Hennink

    • Russell Glassgow

    • Michael Engelgau

    • David Williamson

    • Ping Zhang

    • Mark Hutcheson



DISTRIBUTION

AVAILABILITY

EFFICIENCY

EFFECTIVENESS

EFFICACY

FUNDAMENTAL

RESEARCH

DETSKY, 1990


Translational research transforms currently available knowledge into useful measures for everyday clinical and public health practice. Translational research aims to assess implementation of standards of care, understand the barriers to their implementation, and intervene throughout all levels of health care delivery and public health to improve quality of care and health outcomes, including quality of life.

Narayan et al. Ann Intern Med, 2004


Knowledge

Need


Knowledge

Need


Etiological vs translational research

Changing practice

Effectiveness

Patient, provider and system factors

Generalizability

Focus on common

Benefit as absolute

Quality as relative and dynamic

Optimal health for many

Etiological vs. translational research

  • Understanding cause

  • Efficacy

  • Patient factors (largely biological)

  • Internal validity

  • Focus on rare

  • Benefit as relative

  • Quality as absolute and static

  • Perfect health for few

Narayan et al. Diabetes Care


Types of translational research
Types of translational research

  • Phase one translation, usually dubbed “bench to-bedside,” applies basic scientific discoveries to human health care under controlled conditions, i.e., clinical research.

  • Phase two translation promotes the adoption of the fruits of promising clinical research by the community-based health care system under uncontrolled and (often) uncontrollable conditions.

http://www.niddk.nih.gov/fund/other/Diabetes-Translation/conf-publication.pdf


What a reasonable observer assumes
What a reasonable observer assumes:

  • That promising new biomedical and behavioral science would be quickly and universally offered to any patient who might benefit from it

  • That the power of the new science to improve the human condition should be sufficient to propel widespread adoption of the new science by our health care system and the patients it serves


Unfortunately this is rarely the case
Unfortunately, this is rarely the case

  • In the real world, phase two translation stumbles unguided towards a very uneven, extraordinarily incomplete, and socially disappointing state of affairs


However that can change
However, that can change

  • Implementation of well-designed translational research projects

  • This seminar will provide you with information on:

    • Necessary project components

    • Tips for grant writing

    • Avoiding common pitfalls



Presentation overview
Presentation overview

  • Study aims and objectives

  • Background

  • Research plan

  • Sustainability

  • Budget



Aims and objectives
Aims and objectives

Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

  • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

    • Primary Outcome: incidence of T2DM

    • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


  • Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet

Aim: Clearly states the point of the study, including important details about the research. The aim can also be stated as a question or as a hypothesis.


  • Aim: To evaluate theeffectiveness, cost-effectiveness, and sustainabilityof a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


  • Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using arandomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


  • Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using arandomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


  • Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


  • Aim: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet

Objective: Describes how the investigators will

answer the research question


Aims and objectives1
Aims and objectives

  • Aim 1:

    Can peer education and support groups be used to influence lifestyle choices and prevent obesity in men living in Karachi?


Aim example
Aim example

  • Aim 1:

    Can peer education and support groups be used to influence lifestyle choices and prevent obesity in men living in Karachi?

  • Problems:

    • Study design?

    • Lifestyle choice outcome is vague


Aims and objectives general information
Aims and objectives: General information

  • Begin by summarizing the need for this research

  • Clearly state your main research objective in the form of aims or a hypothesis

  • Briefly describe how you are going to do the project in objectives or a paragraph

  • Length: 1-2 pages


Aims and objectives general information1
Aims and objectives: General information

  • After reading your aims section, the reader should be able to:

    • State the need for your project

    • Agree that the project is needed

    • Summarize the main purpose and broad methods of your project

    • Be excited and interested to read the rest of your proposal




Background
Background

  • Summarize pertinent literature

    • The problem your project is addressing

    • Other studies addressing this problem

    • Highlight what is missing in the literature

    • The research that you are translating

    • Support for the theories and methods that you are using

    • End with a clear summary

  • Preliminary research

  • Length: 1-2 pages


Background1
Background

  • Your background sections should be:

    • Thorough but succinct

    • Well-written

    • Easy to read and follow

      • Subheadings

    • Pertinent




Research plan components

Study design

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Research plan components1

Study design

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


How to select the best study design
How to select the best study design

  • Study design

    • Should be closely linked to the purpose of the evaluation

    • Should build on a theoretical framework of behavioral change

    • Should consider randomized designs first

    • Non-randomized designs or designs where the unit of randomization is other than the participant may be the most appropriate for translational research questions


Study design selection
Study design selection

  • Challenge in translational research is that the interventions are usually complex (multifaceted with simultaneous changes in different parts of the organization)

  • Researcher has variable control over how the intervention is implemented

Eccles M, et al. Qual Saf Health Care 2003;12;47-52


Critical steps in the research plan before the definitive study
Critical steps in the research plan before the definitive study

  • Development of a theoretical basis for the intervention

  • Define the components of the intervention

  • Exploratory studies of observational data + qualitative research to further refine the intervention and planned evaluation

  • Definitive evaluation

Eccles M, et al. Qual Saf Health Care 2003;12;47-52


Types of studies
Types of studies study

Quantitative non-experimental designs

Quantitative experimental designs

Randomization

Qualitative

Multi-level, multi-factorial interventions

Community-based participatory research


Quantitative non experimental designs
Quantitative non-experimental designs study

  • Cross-sectional designs

  • Uncontrolled before and after (longitudinal observational cohort studies)

  • Controlled before and after

  • Time series analyses

  • In translational research there can be political, practical, and ethical barriers to randomized designs – but randomized designs should first be considered


Quantitative non experimental designs1
Quantitative non-experimental designs study

  • May have little control over the implementation of the intervention…

    • Strength: very “real world”

    • Weakness: hard to know what really happened or which “outcomes” are likely to have changed

    • Researcher should document implementation across heterogeneous settings

  • Lack of randomized controls is always a threat to internal validity but this trade-off must be placed in context of research question and study goals


Uncontrolled before and after
Uncontrolled before and after study

  • Simple to conduct

  • Secular trends make it difficult to attribute changes to the intervention

  • Probably over-estimate the benefit from quality improvement interventions


Controlled before and after
Controlled before and after study

  • A control population with similar characteristics is identified

  • Baseline and post-intervention data are collected on both the control and intervention populations

  • Protects against secular trends

  • Even in well-matched groups, baseline characteristic can differ


Controlled before and after1
Controlled before and after study

  • Looking at the significance of within group change is not appropriate

  • Analyses of these data need to account for clustering by site especially if the intervention is delivered at the organizational level


Time series designs
Time series designs study

  • Does the intervention improve care more than the observed secular trend?

  • Requires the collection of data multiple times both before and after so that you understand the magnitude of the secular trend

  • Analysis must account for the auto-correlation of data collected at multiple time points

  • Strength is that you do not need a control group

    • Weaknesses are that you need to collect data multiple times and the design does not protect against other events occurring at the same time as the intervention


Types of intervention studies
Types of intervention studies study

  • Randomized or quasi-randomized trials

  • Interrupted time series (ITS)

    • Defined intervention

    • 3 points in time before and after

  • Non-randomized studies with controls at a second site

    • Data collected before and after the intervention

    • Key to interpretation hinges on comparability of the sites

  • Most are evaluations within systems rather than between systems


Quantitative experimental designs
Quantitative experimental designs study

  • Individual patient-level randomized controlled trials

  • Cluster randomized controlled trials

  • Strongest designs to establish a causal relationship

  • Often times difficult to implement…but considering them is important


Randomized controlled trials
Randomized controlled trials study

  • Considered to be the gold standard

    • Randomly allocated to either intervention or control group

    • Best way to insure that both known and unknown factors that may influence effectiveness of the intervention are balanced in the two comparison groups

  • Time consuming, expensive, complex, may require a large number of clusters, tight inclusion criteria limit generalizabilty

  • May not tell you whether an intervention will improve routine practice


Randomization vs random sample
Randomization vs. random sample study

  • Randomization = process of randomly allocating people to different study groups

    • Control for confounding

  • Random sample = process of selecting a random group from a larger study population

    • Generalizability


Level of randomization
Level of randomization study

  • Patient

  • Health care professional

  • Practice/hospital

  • Provider group

  • Health plan

  • Community

LOW LEVEL CONTAMINATION

HIGH LEVEL POWER, LOGISTICS


Level of randomization1
Level of randomization study

  • At higher levels of randomization measure-ment of pre-intervention characteristics is critically important

  • Consider stratification on baseline characteristics that are likely to influence the effectiveness of the intervention

  • Cluster randomization is likely to violate the assumption of independence of observations within a cluster

    • Two patients in same practice are likely to be more similar than three from different practices

    • Need to be able to estimate the intracluster correlation coefficient (ICC)


Cluster randomization
Cluster randomization study

  • Many quality improvement (QI) interventions are at provider or system level and if you randomize at individual patient level it is likely there will be contamination

  • Randomization at higher level will reduce contamination but you pay a high price with regard to power and ability to detect clinically meaningful differences in outcomes; additionally, risk for bias is much higher

  • Randomize at a higher level but collect data at the patient level


Analysis of cluster randomization
Analysis of cluster randomization study

  • Analysis at cluster level uses the cluster as unit of randomization and unit of analysis

    • Each cluster is treated as one data point (inefficient!)

  • Patient level analyses that are adjusted for the cluster

  • Patient level analyses that allow for correlation between clusters is explicitly modeled; hierarchical nature of data is accounted for in the analysis

  • Unit of randomization must be accounted for in all analyses


Qualitative studies
Qualitative studies study

  • Results from qualitative studies can provide critical information in the translation of interventions to real world settings that have been shown to be effective in clinical trials

  • Meanings, definitions, and characteristics of phenomenon

  • Increase acceptability, cultural appropriateness, and feasibility

  • Relying on current literature may not be sufficient


Types of qualitative research
Types of qualitative research study

  • Direct observation

    • Observe but do not interact


Types of qualitative research1
Types of qualitative research study

  • Direct observation

  • Written documents


Types of qualitative research2
Types of qualitative research study

  • Direct observation

  • Written documents

  • In-depth interviews

    • Individual interviews

    • Focus group discussions


Questions to ask before including qualitative research in your study
Questions to ask before including qualitative research in your study

  • Is qualitative research the best way to answer your research question?

  • Is the methodology appropriate to the research question?

    • Justify your research method


Mixed methods studies
Mixed Methods Studies your study

  • “Quantitative research excels at summarizing large amounts of data and reaching generalizations based on statistical perceptions. Qualitative research excels at “telling the story” from the participant’s viewpoint, providing the rich descriptive detail that sets quantitative results into their human context.”

Trochim, W.M.K., Research Methods Knowledge Base: www.socialresearchmethods.net


Background premises for multi level multi factorial interventions
Background premises for multi-level, multi-factorial interventions

  • We live in contexts: patients, families, providers, clinics, health systems, societies

  • Behavior change and quality improvement are difficult

  • Multiple interventions are more effective than single

  • Diabetes and obesity are chronic conditions: chronic care / chronic disease models


Community based participatory research
Community-based participatory research interventions

  • Community focus: often vulnerable or hard to reach populations

  • Collaboration: community and academic partners

  • Equal relationships

  • Benefit of community

    • Ultimately interventions

    • Reduce disparities


Research plan components2

Study design interventions

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Study outcomes
Study outcomes interventions

  • One primary outcome

  • One or more secondary outcomes

  • Short-term, intermediate, and long-term outcomes


  • Aim interventions: To evaluate the effectiveness, cost-effectiveness, and sustainability of a community-based diabetes prevention program in Chennai, India using a randomized controlled trial, with 700 people with pre-diabetes randomized to either standard lifestyle advice or a culturally specific, low-cost, intensive lifestyle intervention

    • Objective 1: To evaluate effectiveness of the lifestyle intervention by assessing between group changes in the following:

      • Primary Outcome: incidence of T2DM

      • Secondary Outcomes: body weight, percent body fat, body mass index, waist-to-hip ratio, fasting glucose, blood pressure, plasma lipids, activity, and diet


Health care outcomes continuum

Immediate Outcomes interventions

Intermediate Outcomes

Learning

Knowledge

Skill Acquisition

Post-Intermediate Outcomes

Behavior

Change

Long Term Outcomes

Improved Clinical

Indicators

Improved Health Status

Health care outcomes continuum


Study outcomes1
Study outcomes interventions

  • Can be quantitative or qualitative in nature

  • Should be sensible based on study design and research question

  • Should be practical


What is practical
What is practical? interventions

  • Reliable and valid

  • Succinct and/or engaging

  • Relevant to setting and question

  • Broadly applicable: literacy, culture, language

  • Reasonable burden on the study participants



Re aim dimensions and definitions
Re-aim dimensions and interventionsdefinitions

Individual Level

www.re-aim.org


Re aim dimensions and definitions1
Re-aim dimensions and interventionsdefinitions

Setting Level

Both

www.re-aim.org


Why multiple measures
Why multiple measures? interventions

  • Clinicians and policy makers focus on different issues than much efficacy

  • Research

    • Biological vs. economic outcomes

    • Short vs. long term

    • Patient centered vs. provider/system

    • Patient level vs. setting level


Examples of outcomes

Quantitative Measures interventions

Qualitative Measures

Types of Outcomes

Biological

Psychosocial

Behavioral

Economic

Examples of outcomes


Quantitative measures

Incidence - rate of new cases of a disease or condition that occur over a period of time

i.e. 20% of the the controls and 10% of those in the intervention group developed diabetes by the end of the study period

Prevalence - proportion of existing cases of a disease or condition at a particular point in time

i.e. 7.8% of the U.S. population has diabetes

Quantitative measures


Quantitative measures1
Quantitative measures occur over a period of time

  • Absolute risk (risk difference)- the probability of an event occurring among people not exposed to treatment minus the probability of an event occurring among those exposed to treatment

    • In the DPP:

      • Incidence of diabetes in the placebo group = 11%

      • Incidence of diabetes in the intervention group = 4.8%

      • Absolute risk = 11% - 4.8% = 6.2%


Quantitative measures2
Quantitative measures occur over a period of time

  • Relative risk - risk of developing a disease or an event relative to an exposure

    Probability of the outcome in the exposed

    Probability of the outcome in the unexposed

    • Probability of developing cancer is 20% among smokers

    • Probability of developing cancer is 1% among non-smokers

    • Relative risk = 20, in other words smokers, are 20 times as likely as nonsmokers to get cancer

=


Quantitative measures3
Quantitative measures occur over a period of time

  • Odds ratio - odds of an event occurring in one group (p) relative to the odds of the same event occurring in another group (q)

    p/(1-p)p*(1-q)

    q/(1-q) q*(1-p)

    • If 90 out of 100 men smoke and 20 out of 100 women smoke, the odds ratio of smoking among men compare to women is 36

=

=


Qualitative measures
Qualitative measures occur over a period of time

  • Discussion themes

  • Opinions

  • Knowledge

  • Program acceptability


Biological outcomes
Biological outcomes occur over a period of time

  • Examples

    • Diabetes incidence

    • Blood glucose

    • Hemoglobin A1c

    • Weight

    • Blood pressure


Psychosocial outcomes
Psychosocial outcomes occur over a period of time

  • Examples

    • Quality of life

    • Community integration

    • Depression

    • Anxiety


Quality of life qol
Quality of life (QOL) occur over a period of time

Growth of chronic disease is shifting emphasis from objective (mortality and morbidity) to subjective (SRH, HRQL) health measures

The perspective of the person/patient is gaining importance

Growing importance of economics

Changes in organization and delivery of health care


How is qol measured
How is QOL measured? occur over a period of time

QOL is a multi-dimensional construct comprising the individual’s subjective perception of physical, emotional and social well-being, including both a cognitive component (e.g., satisfaction) and an emotional component (e.g., happiness)

  • Campbell A, et al. The Quality of American Life, New York, Russell Sage, 1976


Measuring qol
Measuring QOL occur over a period of time

Illness-specific

Focus on specific problems

Hypoglycemia, insulin injections, self-monitoring of blood glucose, dietary restrictions

DQOL, Diabetes-39, PAID, DTSR

Global

Use across health and illness groups


Health related quality of life hrqol
Health related quality of life (HRQOL) occur over a period of time

HRQOL refers to the impact of health aspects of an individual’s life on their quality of life or overall wellbeing, or refers to the value of a health state to an individual


Global hrqol ways of measuring
Global HRQOL: Ways of measuring occur over a period of time

Health Profile: non-preference-based approach (e.g., SF-36, SIP)

Can not produce a single score

Can not be used for economic evaluation

Utility measure: preference-based approach (e.g., QWB, HUI, EUROQOL)

Can produce a single score

Designed for economic evaluation


Global measures of qol non preference based
Global measures of QOL occur over a period of time(non-preference based)

Medical Outcomes Study Short-Form General Health Survey (SF-36, SF-20, SF-12)

Physical, social and role functioning capture behavioral dysfunction

Mental health, perceptions of overall health, and pain reflect subjective components

Available in many languages


Quality adjusted life years qaly
Quality-adjusted life years (QALY) occur over a period of time

A summary outcome measure that incorporates the quality or utility of a health state with the duration of survival


Qaly matrix
QALY matrix occur over a period of time

Two possible effects of a disease or an intervention:

Extending life

Improving the health related quality of life

QALY combines the two effects in a multiplicative way


How to calculate qaly
How to calculate QALY occur over a period of time

  • Assign a number that corresponds to the quality of heath state during each period during the survival

    • For example: optimal health = 1 death = 0 blindness = 0.8

    • “Quality” of heath state is often referred to as utility

  • Add the utilities across time periods


Qalys gained from an intervention
QALYs gained from an intervention occur over a period of time

optimal health

1

without program

with program

Quality adjusted life years gained

Health related quality of life

0

Duration (years)

Death

without the program

Death

with the program


Behavioral outcomes
Behavioral outcomes occur over a period of time

  • Examples

    • Adherence

    • Stage of change

    • Behavior change


Economic outcomes
Economic Outcomes occur over a period of time

  • Examples

    • Cost

    • Cost-effectiveness

    • Cost-utility


Cost and cost effectiveness
Cost and cost-effectiveness occur over a period of time

Health care resources are limited

Some interventions have better value than others

All health care choices have an “opportunity cost”

Policy makers, public health agencies, insurers, health care providers, patients, etc. all have to make health care choices


Questions addressed by economic analyses
Questions addressed by economic analyses occur over a period of time

How much does a disease cost?

How much does treating or preventing the disease cost?

How do the benefits compare with the costs? (i.e. What is the value of interventions?)


Alternative occur over a period of time

more effective

but more costly

B

Alternative

cheaper but less

effective

Alternative

more effective

and less costly

COST

Alternative

less effective

and more costly

E

A

OUTCOME

D

C


General study designs in health economics
General study designs in health economics occur over a period of time

Cost analyses

Cost-effectiveness analyses

Cost-utility analyses

Teutsch and Harris, Prevention Effectiveness, 1996


Cost analysis
Cost analysis occur over a period of time

  • How much does an intervention, program, or disease cost?


Cost effectiveness analysis
Cost effectiveness analysis occur over a period of time

An analytical tool in which costs and effects of a program are compared to the costs and effects of another program or doing nothing

Expressed in terms of difference in cost divided by difference in outcomes

Gold et al. Cost-effectiveness in Health and Medicine, 1996


Cost effectiveness examples
Cost effectiveness examples occur over a period of time

  • Among persons > 25 years of age, screening in the doctor’s office costs $236,449 for each year of life gained, compared to not screening

CDC Diabetes Cost-effectiveness Group, JAMA, 1998


Cost utility analysis
Cost utility analysis occur over a period of time

A type of cost-effectiveness in which the outcome is adjusted to account for the impact of the intervention on quality of life

Defined as difference in cost divided by difference in quality-adjusted life years (QALY) achieved


Cost utility examples
Cost utility examples occur over a period of time

Among persons > 25 years of age, the cost utility of opportunistic screening for diabetes is an additional $56,649 for each additional quality-adjusted life year gained, compared to not screening

CDC Diabetes Cost-effectiveness Group, JAMA, 1998


Basic ways of conducting cost effectiveness studies
Basic ways of conducting cost-effectiveness studies occur over a period of time

Within trial: Examination of costs and effects using real data collected during a clinical trial

Model based: Simulation of lifetime costs and effects using mathematical models and data from the literature


Study perspectives
Study perspectives occur over a period of time

When choices about allocation of health resources are considered, who is affected?

On whose behalf are decisions made?

Who gains health benefit, and who pays for it?

The answers to these questions define the perspective of an economic evaluation

Gold MR et al. Cost-Effectiveness in Health and Medicine, Oxford University Press, NY, 1996


Three major perspectives
Three major perspectives occur over a period of time

Health system: An HMO might consider only costs related to the clinical services they provide

Payer: An employer might consider additional costs that affect employee productivity

Societal: Consideration also given to costs incurred directly by the patients


Concepts related to costs
Concepts related to costs occur over a period of time

Opportunity cost

Marginal cost

Time discounting

Types of costs


The economic definition
The economic definition occur over a period of time

In economic theory, ‘costs’ refer to the value of foregone benefits which may or may not be measurable in dollars

Richardson J. Economic evaluation of health promotion: Friend or foe?Aust NZ J Public Health 1998;22:247-53.


Opportunity cost
“Opportunity” cost occur over a period of time

Because you can’t spend a dollar in two places at once, opportunity cost is the value of the opportunity foregone by spending resources in one place and not in another


Marginal vs total costs
“Marginal” vs. total costs occur over a period of time

Economic evaluation of health interventions is generally concerned with marginal costs and not with the total costs of an intervention.

Richardson J. Aust NZ J Public Health 1998;22:247-53.


What does marginal mean
What does “marginal” mean? occur over a period of time

A “marginal” is simply the difference between two quantities

The marginal cost is calculated as the total cost of A minus the total cost of B:

marginal cost = $A - $B


Time discounting
Time discounting occur over a period of time

The practice of reducing the value of costs when they occur in the future so they can be compared with costs that occur in the present

Richardson J. Aust NZ J Public Health 1998;22:247-53.


Time discounting why do it
Time discounting: Why do it? occur over a period of time

If funds are not spent on health programs then they may be invested and earn a rate of interest

At some future date the funds can buy more health

However, when the future date is reached the same argument could be used

Hence, one could justify never spending on health

Thus, we need to express costs relative to the same time period, i.e. present value

Richardson J. Aust NZ J Public Health 1998;22:247-53



Types of costs
Types of costs occur over a period of time

  • Direct medical:

    • Costs of the intervention itself

      • e.g., MD time, lab tests

    • Costs of routine care

      • e.g., treating microvascular complications

    • Costs of treating side-effects

      • e.g., hypoglycemia

  • Direct non-medical:

    • Costs incurred by patient

      • e.g., treadmill, sneakers

  • Indirect:

    • Cost of lost productivity due to illness

    • Cost of lost productivity due to death


Measurements of value
Measurements of “value” occur over a period of time

Cost-effectiveness ratio

Quality adjusted life year

Cost-utility ratio


Cost effectiveness ratio
Cost-effectiveness ratio occur over a period of time

CE ratio = Difference in cost divided by difference in effectiveness

Difference in cost = cost with a program − cost without the program


What is in the cost
What is in the cost ? occur over a period of time

Total Cost =

Direct medical costs +

Direct non-medical costs +

Indirect costs


What is in effectiveness
What is in effectiveness? occur over a period of time

Effectiveness is a health outcome measure

Examples:

Number of diabetes cases prevented

Number of days sight saved

Number of years of life saved


Research plan components3

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Measuring study outcomes
Measuring study outcomes occur over a period of time

  • You should plan for the following when designing your study:

    • How the outcomes will be measured

    • Use validated tests/study instruments

    • Timing of study tests

    • Be conservative about the amount of change


What to include in your grant
What to include in your grant occur over a period of time

Table of all study tests and test schedule

Describe how you will measure all primary and secondary outcomes, as well as other outcomes

Include descriptions of screening, baseline, follow-up testing

Test for randomization, adherence, recruitment


Study testing
Study testing occur over a period of time

  • Clearly state how you will collect data

    • What methods will be used to record data?

    • Who will collect the data?

    • When and where will data collection occur?

    • Be systematic in your data collection and reporting

    • Explain how your choices relate to your research question


Research plan components4

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Study sample
Study sample occur over a period of time

  • Your sample should reflect the larger target community

  • Exclusion and inclusion criteria

  • Recruitment

    • Who is your study population?

    • How and where will you reach them?

    • In qualitative research want to recruit an information-rich sample


Sample size
Sample size occur over a period of time

  • Power - probability of rejecting a false null hypothesis (1-)

  • Depends on study design and method of randomization, desired power, type I error rate, and the outcome being studied

    • Software programs: SIZ, PASS, Egret

    • Online sample size calculators


Sample size1
Sample size occur over a period of time

  • Examples of online sample size calculators (see handout):

    • http://www.openepi.com/Menu/OpenEpiMenu.htm

    • http://stat.ubc.ca/~rollin/stats/ssize/


Research plan components5

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Study timeline
Study timeline occur over a period of time


Research plan components6

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Intervention
Intervention occur over a period of time

Describe intervention and control arm

Do not re-invent the wheel - use tested interventions when possible

Randomization procedures

Theoretical framework


Theoretical frameworks for behavioral research
Theoretical frameworks for behavioral research occur over a period of time

  • Stages of change

  • Health beliefs model

  • Locus of control

  • Diffusion of innovation


1 stages of change
1. Stages of Change occur over a period of time

http://www.adultmeducation.com/FacilitatingBehaviorChange.html


2 health beliefs model
2. Health beliefs model occur over a period of time

http://en.wikipedia.org/wiki/Health_Belief_Model


3 locus of control
3. Locus of control occur over a period of time

  • An aspect of personality that explains who an individual believes is in control of events in that individual’s life

  • Events (life, health, etc.) can be viewed as being controlled internally or externally:

    • Internal - the individual is in change of himself and his life

    • External - something or someone else - the environment, other people, a higher power - control the individual’s decisions and life


4 diffusion of innovation
4. Diffusion of innovation occur over a period of time

http://cq-pan.cqu.edu.au/david-jones/Reading/Adoption/onweb/


Research plan components7

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Data analysis and data management software
Data analysis and data management software occur over a period of time

  • Data entry and management:

    • Excel, Access

  • Textual data analysis:

    • Nudist, Atlas/ti, MAXqda

  • Quantitative data analysis

    • Epi Info, SAS, SUDAAN, Stata, WesVar, SPSS


Data analysis plan
Data analysis plan occur over a period of time

  • A data analysis plan should include descriptions of:

    • How and in what units outcomes will be measured

    • How randomization, response rate, retention, and adherence will be measured

    • How data quality will be assured and assessed

    • How data will be protected


Data analysis plan1
Data analysis plan occur over a period of time

  • A data analysis plan should include descriptions of (cont.):

    • How data will be analyzed

      • Quantitative data: treatment of continuous and categorical variable, planned statistical tests

      • Qualitative data: and/or textual data analysis description

    • List any data analysis or management programs that will be used


Data analysis plan2
Data analysis plan occur over a period of time

  • Be specific about and reference procedures for data analysis

  • For qualitative research

    • Be clear about how themes are derived

    • Be systematic in your analysis to avoid bias

  • Consult an expert


Bias occur over a period of time

  • Types of bias

    • Selection bias

      • Berkson’s Bias

    • Misclassification (information bias)

      • Recall bias

    • Confounding

      • Alcohol and risk of oral cancer


How to deal with bias
How to deal with bias occur over a period of time

  • Confounding should be addressed at the analysis phase

  • Selection bias and misclassification cannot be corrected for statistically and must be avoided


Research plan components8

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Key personnel
Key personnel occur over a period of time

Include a qualified and well-rounded study team

Role in project

Relevant research


Research plan components9

Study design occur over a period of time

Outcomes

Study testing

Study Sample

Timeline

Intervention

Data analysis plan

Key personnel

Ethical Issues

Research plan components


Ethical issues
Ethical Issues occur over a period of time

Subject population

Sources of research materials

Data and safety monitoring plan

Potential risks

Anticipated benefits


Human subjects
Human subjects occur over a period of time

Procedures for protection of human subjects

Procedures for ensuring confidentiality

Consent procedures

Study impact


Resources
Resources occur over a period of time

http://www.hhs.gov/ohrp/

Informed consent checklist: http://www.hhs.gov/ohrp/humansubjects/assurance/consentckls.htm

Institutional Review Boards/Research Oversight Committees


Questions and discussion2
Questions and discussion occur over a period of time


4. Sustainability occur over a period of time


Sustainability
Sustainability occur over a period of time

Translation research is:

“comprehensive, applied research that strives to translate the available knowledge and make it useful in everyday clinical and public health practice”

Narayan KM, et al. Ann Int Med 2004; 140: 958-963.


Sustainability1
Sustainability occur over a period of time

Should include:

Sustainability should be integrated in the study

Detailed description of how the program will be sustained

What is sustainable?

Who will continue the program after the research is complete - community members, the research institution, etc.

Is the project scalable?

Future impact


Questions and discussion3
Questions and discussion occur over a period of time


5. Budget occur over a period of time


Budget
Budget occur over a period of time

See handout for budget and budget justification example

Start early

Personnel, travel, equipment, testing


Budget justification
Budget justification occur over a period of time

Justify the budget items

Explain how costs were derived

If the study team is international, how is salary being determined at each study site

Explain why these items are needed

List percent effort


Questions and discussion4
Questions and discussion occur over a period of time


Overarching Issues occur over a period of time


Grant writing overarching issues
Grant writing: overarching issues occur over a period of time

  • Anticipate problems and put plans in place to avoid them

  • Minimize variation

  • List limitations and how they will be minimized

  • Lay out study costs and milestones clearly


Common errors in idf applications pitfalls to avoid
Common errors in IDF applications: pitfalls to avoid occur over a period of time

  • Not in the BRiDGES framework

  • Not enough detail

  • Lack of cohesion

  • Readability


2 not enough detail
2. Not enough detail occur over a period of time

  • “no clear view of reach…who will participate and why”

  • “unclear which scales will be used”

  • “no mention how data will be obtained”

  • “lack of clear definitions” for outcome measures

  • No description of measurement tools

  • “proposal lacks significant details”

  • No power calculations

  • No support for the feasibility of the study tests and/or intervention

  • “theoretical framework…is not defined”


3 disagreement between different parts of the grant
3. Disagreement between different parts of the grant occur over a period of time

  • “lack of cohesion between” objectives, study measures, and outcomes

  • “timeline and figure” are “somewhat inconsistent” with the study design

  • Costs do not reflect all components of the study


4 readability
4. Readability occur over a period of time

  • “hypotheses are not well articulated”

  • “cursory nature of the written proposal”

  • Inconsistency in terminology


Avoiding common pitfalls
Avoiding common pitfalls occur over a period of time

  • Read the grant announcement carefully

  • Proof-read several times

  • Recruit other readers


Questions and discussion5
Questions and discussion occur over a period of time


Group activities and interactive discussion

Group Activities and Interactive Discussion occur over a period of time


Evaluation criteria final thoughts on grant preparation

Evaluation Criteria: occur over a period of timeFinal Thoughts on Grant Preparation


Checklists for research proposals
Checklists for Research Proposals occur over a period of time

  • Translational Research Checklist

  • STROBE

  • QUOROM


Grant proposal development final thoughts
Grant proposal development: occur over a period of timeFinal thoughts

Start early and develop a timeline

Think of the specific question(s) clearly

Do a good literature search on the topic

Write a one-page summary with objectives and get feedback from colleagues

Meet and discuss study plan

Write and rewrite 1-2 page purpose and objectives


Grant proposal development final thoughts1
Grant proposal development: occur over a period of timeFinal thoughts

Clearly draft the plan of investigation

Get it reviewed and criticized

Redraft plan of investigation

Write the background

Use tables for measurements

Use diagrams for concept and study design


Grants proposal development final thoughts
Grants’ proposal development: occur over a period of timeFinal thoughts

Write, read, and revise several times

Get different people to read over and comment

Check spelling, grammar

Don’t repeat

Write short (but thoroughly)!

Use summaries and highlight key points

Make it interesting to the reviewer

On the last two days, be compulsive


Questions
Questions? occur over a period of time


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