What Patients Want To Know About Their Medications



An Introduction to Sociology – and what it can do for pharmacy practice research

Bissel P, Traulsen JM, Haugbolle LS. Int J Pharm Pract. 2001;9:289-95.

Why Sociology Important for Pharmacy Research?

1) Training of health professionals have increasing emphasis on social &interpersonal aspects of health care practice

2) Growing role of Pharmacy in promoting health, enhancing compliance, providing medicines management services

3) Health&illness are social (not just biological) phenomena and so are amenable to sociological analysis

(being ill affects our lives and relationships with others in society

(there are social aspects to medication usage and beliefs about those medications

4) All healthcare practice is conducted within social relationships and social structures

(ex. a hospital is a mini-society

What is Sociology?

Characteristics: - ongoing influence by popular culture and evolving society

- tends to focus on “problems”, especially idea of social order

- rarely provides certainty or objectivity in responses to questions

Three Major Concerns:

1) The nature of social structure

2) The relationship between self and society

3) The historical development of society

Sociological Theories:

To address these concerns sociologists develop theories. These theories often emphasize either “agency” or “structure”, the degree to which characterizes the theory.

if focus on agency = seek to understand how individuals influence the wider society through their thoughts, actions and behaviours

if focus on structure = seek to understand how the thoughts, actions and behaviours of individuals are influenced by wider society

Eg. how is compliance affected by individual beliefs and behaviours? (agency) vs. how is compliance affected by culture, family and financial resources? (structure)

What are the Goals of Sociology?

- to provide a framework within which to interpret and understand research findings

- to determine the “nature of power”

ex. the consequences of how a health professional’s use of their power and authority

affects patient health outcomes (compliance with difficult regimen, etc)

- to question current healthcare practices and policies

Engaging sociological theory can provide deeper insights into health, medicine and human behaviour…this can add to the strength and diversity of pharmacy practice research.

Science and the Scientific Approach

In Foundations of behavioural research 2nd ed. New York: Holt, Reinhart, Winston, 1973:2-15.

Sept 4th Readings (i.e. the one Thorsen HINTED would be on the exam!!!)

Intro: -point of article is to explain scientific language and the scientific approach to problem solving

-this is because in order to understand ANY complex human activity, we must grasp the language and the approach of the individuals who use it

-the scientists approach to problems IS different than layman’s, but it’s not strange or esoteric (when understood it will seem natural)

Science and Common Sense: -“in creative thought common sense is a bad master”

-How are common sense and science alike and different?

|Similar |Different in 5 ways |

|-science is a systematic and controlled |Layman may use the idea of “concepts” loosely, i.e. “ a person gets sick b/c they need to|

|extension of common sense |be punished for committing a crime”. Scientists systematically builds theoretical |

|-both are a series of concepts |structures and tests them for internal consistiency. Realizes that the concepts are |

|satisfactory |“man-made” and may not represent reality |

|for the practical uses of mankind |Scientist systematically and empirically tests his theories and hypothesis. They |

|PROBLEM: sometimes these “concepts” |carefully guard against preconceptions, predilections and selective support of their |

|Can be misleading i.e. last century it |hypothesis |

|was |Control in science-scientist tries to systematically rule out variables that he has |

|“common sense” to use punishment for |hypothesized to be the causes. Layman don’t do that. |

|pedagogy. Now evidence to show that |Cause and effect: Layman may see two phenomena occurring coincidently and label as cause |

|rewards work better than punishment |and effect. Scientist consciously and systematically pursues relations |

| |Explanations of observed phenomena: scientist carefully rules out “metaphysical” |

| |relations: i.e people are poor b/c God wills it, studying hard subjects improves child’s |

| |moral character. None of these things can be tested—metaphysical. Doesn’t mean that |

| |scientist doesn’t believe them, he just can’t be concerned with them while in the |

| |scientist role. |

Four Methods of Knowing (or fixing belief):

1) Method of TENACITY: -men hold firmly to what they believe is true because they have always known it to be true.

-repetitions of the truth increase its validity

-men tend to cling to their beliefs when there are conflicting facts

2) Method of AUTHORITY: -established belief (i.e. if the Bible says so, it is. If a noted physicist says there’s a God, there is).

-superior than tenacity. Life couldn’t go on w/o this method (i.e. we all take large body of fact and information on the basis of authority)

-human progress can still be achieved w/this method (unlike tenacity)

3) PRIORI Method (method of intuition): -priori propositions agree with “Reason”…but not necessarily with experience

-idea seems to be that men, w/free communication can reach the truth by reasoning b/c natural inclinations are towards the truth

-problems: what happens when 2 men reason things out differently? who is right?

4) Method of SCIENCE: - one characteristic that no other method has: self correction

-bulit-in checks along the way used to control and verify scientist’s activities and conclusions

-scientists INSIST on testing every hypothesis no matter how “promising” it looks

-scientists insist that testing procedure be open to public inspection

-OBJECTIVITY: no bias, preconceptions…etc

-most DEPENDABLE form of knowledge

Science and it’s Function:

-science is a badly misunderstood word—3 popular beliefs:

1) whitecoat, stethescope stereotype—scientist is a PECULIAR person who works with facts in the lab

2) scientist is a brilliant individual—thinks, spins complex theories, generally spends time in ivory tower aloof from the world and it’s problems

3) equates science with engineering and technology (i.e. building bridges, improving misiles etc)

In the scientific world there are 2 broad views of science:

STATIC VIEW: -an activity that contributes systemized information to the world, adds to the existing body of knowledge

DYNAMIC VIEW: an activity that scientists DO. It’s a base for further scientific theory. Heuristic view: the word heuristic means to serving to discover or reveal. May be called problem solving with an emphasis on imaginative not routine problem-solving. Established facts are important b/c they add to further theory, discovery etc

FUNCTION OF SCIENCE: --2 views

1) science as an activity aimed at improving things (view held by layman)

2) similiar to heuristic view, but with an added element of establishment of general laws

The Aims of Science, Scientific Explanantion, and Theory:

-basic aim of science is theory, to explain natural phenomena

-the point of THEORIES is to find general explanations that can be applied to specific behaviors

-note, the basic aim of science is NOT betterment of mankind (hard to believe for students)

Theory def’n: A theory is a set of interrelated constructs (concepts), definitions, and propositions that present a systematic view of phenomena by specifiying relations among variables, with the purpose of explaining and predicting the phenomena.

-think of the bolded parts as three important parts of the def’n.

-take example of failure in school as a theory: variables could be intelligence, anxiety, motivation; the phenomena is failure in school; the scientist “understand” school failure by relating the variables to the phenomena, and then is able to “predict” it.

-explaination and prediction are RELATED (if u can explain phenomena, u can predict it)

-by its very nature, theory PREDICTS.

Scientific Research – A definition

Scientific Research is systematic, controlled, empirical, and critical investigation of hypothetical propositions about the presumed relations among natural phenomena.

Two points:

1. Systematic and controlled means, in effect, that scientific investigation is ordered that investigators can have critical confidence in research outcomes. This means that the research observations are tightly disciplined.

2. Scientific research is empirical – Subject belief must be tested against objective reality

The Scientific Approach

- it a special systematized form of all reflective thinking and inquiry.

- what follows is based on Dewey’s analysis

Problem – obstacle idea

The scientist will usually experience an obstacle to understanding. His first step is to get the idea out in the open and express the problem in some reasonable manageable form. Problem will not spring full-blown at this stage. Then he states the problem, explicitly or implicitly and here he intellectualized what was first an emotional quality of the whole situation.

Hypothesis

It is a conjectural statement, a tentative proposition, about the relation between two or more phenomena or variables. (“If such and such occurs, then so and so results”)

Reasoning –Deduction

The scientist now deduces the consequences of the hypothesis he has formulated. Experience, knowledge, and perspicuity are important.

He may arrive at a problem quite different from the one he started with, or the deductions led him to believe that the problem can’t be solved with present technical tools. (eg. Before modern statistics were developed, it was difficult to test more than one hypotheses at the same time, but now there’s reason to believe that certain problems are insoluble unless they are tackled in a multivariate manner, ie teaching methods).

Eg. investigator looks at aggressive behaviour, and notes that aggressive behaviour seems to occur when people have experienced difficulties of some kind. He formulated hypothesis: Frustration leads to aggression and reasons that, if frustration leads to aggression than then we should find a great deal of aggression in children who are in schools that are restrictive. This shows that reasoning may change the problem. The initial problem was only a special case of a broader problem. Important because of its almost heuristic quality. Reasoning can help lead to wider, more basic, and more significant problems, as well as provide operational (testable) implications of the original hypothesis.

Observation –Test – Experiment

Test hypothesis by testing the relation expressed by the hypothesis, (test the relation between variables) for the purpose of putting the problem relation to empirical test. A vague and poor stated question can lead to ignorance and misguided information.

We test the deduced implications of the hypothesis. Eg. Hypothesis may be “writing remarks on student papers will improve future papers”, which was deduced from a broader hypothesis, “Reinforcement of responses leads to an increment in the response rate and strength”. We are testing the relation between writing remarks on papers and the improvement of future papers.

Temporal sequence of reflective thinking or inquiry is not fixed. (ie. the first step is not neatly completed before the second step begins).

We may test before adequately deducing the implications of the hypothesis. The hypothesis itself may seem to need elaboration or refinement as a result of deducing implications from it.

Feedback to the problem, hypothesis, and theory of the results of research is very important. People have altered their theories and research because of experimental findings. Part of the essential core of scientific research is the constant effort to replicate and check findings, to correct theory on the basis of empirical evidence, and to find better explanations of natural phenomena. Science has a cyclic aspect. Theory and research is challenged by other researcher.

Summary of scientific approach.

1. doubt, a barrier, and indeterminate situation

2. formulation of problem.

3. literature search / scans own experiences

4. hypothesis is constructed – then empirical implications are deduced. (original hypothesis may be changed)

5. relation expressed by the hypothesis is tested by observation and experimentation.

6. hypothesis is accepted or rejected on the basis of research evidence

7. information is then fed back to original problem, which is kept or changed based on the evidence

Phases of the process can be expanded or skipped. What’s important is the controlled rationality of scientific research as a process of reflective inquiry, and the interdependent nature of the parts of the process, and the importance the problem and its statements.

Contributions of the Social Sciences

Mount JK. In Foundations of behavioural research 3rd ed., William and Wilkins, 1989. 1-16.

Preliminaries

• Growing concern among public/health care professionals that health needs are not being met by biomedical research alone (which is considered the dominant approach to health care).

• We also have to consider the “human side” of things, or the psychosocial issues, for complete patient care.

• George Engel proposed a biomedical model of health care that adopts the biopsycosocial idea, and considers the social sciences (SS) as a complement to the physical and biological sciences.

• Inclusion of the social sciences offers a new approach to health care

• This is important because pharmacy practice is continually changing (ie. we need to take on new professional roles and patient care orientations, and the social sciences can help us understand these new roles better and focus on the human side of things).

• Social sciences (eg. anthropology, sociology, psychology, epidemiology) focus on understanding patterns and meanings of human behaviour.

• Definition of Science – (1) it is theory based;

(2) it uses precise principles for investigation which

specify appropriate techniques for collecting and

analyzing data;

(3) knowledge base is always expanding and cumulative,

and can support or refute what is already known;

4) identifies cause-effect relationships so we can make

predictions.

• Definition of Theory – a series of related, testable propositions that describe how a set of facts are related to one another and under what conditions these relationships hold. Eg. the germ theroy

• The social sciences are considered a science because they are theory based, use systematic research techniques, and are cumulative and predictive (but this is the weakest aspect for SS because they are still at the early stages of development, unlike chemistry/biology).

• Solving DRPs have mainly focused on physical and biological sciences, but over the last ~ 20 years, pharmacists have started using the SS.

• SS deal with phenomenon that are part of our “life-world”. They are not described by common sense.

• SS are accurately described as interpretive sciences, highlighting peoples understanding of the social world and how it affects them.

• Social scientists must be more explicit in their recognition of their interpretations, and spend time on validating their techniques and theories.

• Basic SS researchers seek enhanced understanding and have the primary goal of knowledge discovery for its own sake. Eg. does patient recall of info affect their medication compliance?

• Applied social or behavioural research want to apply this knowledge about human behaviour to address real concerns or issues. Eg. What memory enhancing techniques best improve patient compliance?

• The weaknesses of the biomedical model:

o Once the best of the physical and biological science knowledge is applied to the treatment of diseases a number of problems are not completely resolved

o The model has a tendency to consider patients as biologically active organisms and its frequent failure to consider patients as socially active participants in health care endeavors

• Weaknesses are addressed in the biopsychosocial model which allows us to view pharmacy and drugs within the human context in which they exist

o Model provides a ‘systems approach’ to understanding the world

Contributions of the social sciences

• There are 3 specific areas in which the social sciences contribute to our understanding of pharmacy and pharmacy practice. It provides with:

o Approaches to asking questions in pharmacy

o Conceptual and explanatory frameworks

o Research tools and techniques

Contribution 1: Approaches to Analyzing pharmacy

• Most important contribution of social sciences to pharmacy is their assistance in identifying questions and subjects that need to be addressed.

• It helps to identify the central problematics of social and behavioural pharmacy

• Question of central interest can be grouped into

o Drugs and drug use

o Pharmacy practice and services

o Pharmacy as a profession

• Social sciences also help to understand the basic work of pharmacy

• 2 major interrelated aspects of this work are

o at the microsocial level: describe pharmacy as the discrete activities and interactions carried out as part of pharmacists’ day to day functioning (Eg. Discussing how belonging to a profession affects individual members)

o at a broader level (ie macrosocial) : pharmacy services describe formally or informally organized sets or systems of pharmacists’ work activities (Eg. Use of an international comparative approach to assessing the status of pharmacy)

• another topic informed by the social sciences focuses on the state and status of the pharmacy profession.

Contribution no. 2: Conceptual and Explanatory Frameworks:

The social sciences assist provide explanatory frameworks for interpreting our question and it’s answers. Such an explanatory framework is a theory or a theoretical approach.

Several broad theoretical orientations( underpin most of the theories social scientists examine.

1. Psychological

• how personality and psychological processes (learning, feeling, thinking) affect behaviour

2. Social Psychological

• like psychological but emphasizes how individual behaviour is affected by social group membership

3. Functional

• influences that result from existence, organization and operation of large social groups

• assumes that consensus and agreement dominate these social groups

4. Political

• influences that result from existence, organization and operation of large social groups

• assumes that conflict and competition dominate

5. cultural

• focus on social groups within their environmental context

• greater emphasis on social/man-made environmental influences on human behaviours

6. ecological

• focus on social groups within their environmental context

• emphasizes physical/natural environmental influences

( theoretical orientation is a basic set of assumptions regarding the factors that influence human behaviour

Contribution no. 3: Research Techniques and Tools

A: Research Design

|Research Method |Strengths |Weaknesses |Example of Application |

|Experiment |*Good internal control |Situations often artificially |Educational interventions |

| |*Random assignment used |structured | |

|Survey Research |*Random sampling can be used |Generally only cross-sectional |*consumer surveys |

| |*Good representation of population |data collected |*drug epidemiology |

| |possible | | |

|Field Research |Can study events in natural |Internal control very difficult |Observation of patient- |

| |surroundings |to achieve |professional interaction |

|Content Analysis |Researcher ahs direct effect on |Problems with data completeness,|Analysis of professional |

| |subjects being studied |accuracy and verification |curriculum |

|Existing Data Research |Same as above |Same as above |Pharmacy records review |

|Historical Research |Allows comparisons not possible at |Restrictions of types, amount |Development of professional |

| |one point in time |and quality of data available |codes of ethics |

|Comparative Research |Allows comparisons not possible in |Comparability of data difficult |State of pharmacy throughout the|

| |any single community society |to assess (as is accuracy often |world |

| | |times) | |

|Evaluation Research |Determination of effects of |Value biases and values conflict|Evaluation of patient counseling|

| |interventions possible |problems often arise |techniques |

B: Data collection

• collection of social science data takes a variety of forms

i. direct observation

ii. review of written records

iii. personal interviews

iv. paper and pencil surveys

C: Data Analysis

• social scientists require specialized data analysis techniques because;

i. of the research methods and data collection procedures

ii. broad social science concepts have to be incorporated into an overall measure or scale that is reliable and valid

Conclusion:

The social sciences contributions

a. provide a unique perspective for enhances understanding of contemporary pharmacy

b. outline new questions for the pharmacy research and education agenda

c. supply a set of approaches, tools and techniques for addressing these questions.

The provision of enhanced pharmacy services in Ontario

Dolovich L, Ho CS, Wichman K, Bowles-Jordan J. CPJ/RPC 2002:May;29-36.

Objective:

Proile the types of enhanced services provided based on definitions in OPA fee guide

Determine the level and frequency of provision

Identify sources and rate of compensation

Method:

Study Design

Cross-sectional, survey by mail, returned by fax or mail

Follow-up reminder to non-responders, second reminder by phone

Survey

27 items, 3 sections with questions related to OPA fee guide, 4th section demographics

800 sent out, with target response rate of 30%

Data Analysis

Descriptive statistics, (mean, median, range etc.)

Results:

24% response, chain and independent, usually had more than one pharmacist/technician working

Additional dispensing services

rate of provision and patient volume:

high: compounding, DRP’s, delivery

low: refill reminder, trial Rx

Time requirements: simple 6-10 mins, complex 11-15

Compensation: most free of charge, except for compliance aid package, compounding, colostomy supply consultative services

Pharmaceutical Care Services

Rate of provision: (high to low)

Med review, advanced patient education, care plan prep, patient assessment, HCP team consultation

Patient volume: 1-3/week

Time: 16-30 mins

Compensation: mostly fee of charge (93-100%), compensation usually from pt not TPP

Specialized Pharmacy Services

Rate of provision: (high to low)

Smoking cessation, complementary care consultation vs. home care, drug abuse consultation

Patient Volume: 1-3/week except complementary care (7-9)

Time: most under 15 mins, some up to 30

Compensation: mostly free of charge (90-100%), compensation usually from pt not TPP

Discussion:

- as the complexity of enhanced pharmacy services increased, the # of pharmacies providing them & the # of patients receiving them decreased.

- Few pharmacies receive compensation for providing enhanced services.

- To provide enhanced services you need: therapeutic knowledge, comprehensive advanced service delivery techniques, time management, and supportive staff.

- Services provided the most: medication reviews & complementary med consult

- Limitations of the study: low response rate (24%), not all questions were answered, bias – those who responded are more likely to be those who provide phm care, the questions contained terms that could be interpreted differently by different pharmacists, study relies on self-reported data.

What Patients Want To Know About Their Medications

Nair K, Dolovich L, Cassels A, McCormack J, Levine M, Gray J, Mann K, Burns S. Canadian Family Physician. 2002;48:104-9.

I think this article was just an example of a research article and the information in it is irrelevant, but we summarized it anyways. (Don’t concentrate too hard on this one!)

Abstract

Objective: - describe what patients want to know about their meds and how they access info.

- describe how MDs and pharmacists respond to patients’ info needs.

- to use patients’, MDs and pharmacists’ feedback to develop evidence-based treatment info sheets

Conclusion: patients and clinicians appear to differ in what and how much info patients should receive about meds.

Introduction

- patients want and seek more info about drug and non drug treatment options, but much of information contains conflicting, inaccurate, poorly written or non-evidence-based information.

- Need for balanced, accessible patient information

- Objectives as per abstract.

Methods

Design: - qualitative study used grounded-theory approach; focus groups held with patients, physicians, pharmacists in 3 regions of Canada (Ont, NS, BC).

Study sample: - used combination of purposeful and convenience sample.

- purposeful sampling consciously seeks out participants who can contribute to subject area.

- Reflected cultural and demographics of targeted cities ( stratification of potential participants ( convenience sampling used to recruit patients

- 18yo or older with more than 1 med eligible in patient focus groups

- pharmacists and MDs that practiced in clinical capacity for at least 1 yr invited to participate

- focus groups conducted until theoretical saturation (repetition of themes) reached.

Focus group format: - patients focus groups consisted of 17 open-ended question asking patients general med info needs and opinions about draft info sheets.

- MDs and pharmacists asked for opinions about themes emerging from patient focus groups.

- Interview guide pilot-tested to ensure clarity, timing, and wording of questions.

- Audiotaped, 2 research team member present in each group; one facilitated group, other wrote notes.

- After each interview, researchers held debriefing, tapes transcribed ( data analysis.

Data analysis: - common themes defined from verbatim statements; transcripts coded independently by at least 2 research members.

- themes continually developed and explored ( further analysis conducted as new themes emerged ( summaries of each theme produced ( reviewed to elicit confirming and nonconfirming data for themes generated ( 3 researchers ensured consensus for report.

Results

- patients wanted general info about their condition before they made decisions about treatment.

- patients also identified 5 specific areas for info: side effects and risks (including drug interactions and contraindications); range of treatment options (including nonpharms and alternative remedies); how long to take medications; cost of meds (if covered by drug plans or more cost-effective alternatives available, ie. Generics); and whether the meds were right for them (ie. Reflecting individual health situation).

- overwhelmingly, patients thought pharmacists were most accessible as source of info; other sources were tv, newspapers, librairies, internet, family and friends.

- MDs and pharmacists generally felt that extra info on drugs should be given only if it did not contribute to info overload, confusion or noncompliance.

- MDs and pharmacists unaware patients wanted treatment info for their own unique health situations, why a particular med or treatment was suggested for them.

- developed model of how and why patients seek information:

Context

Consequences

↓

↓

Strategies

Causal condition Phenomenon (

( (

(

↑

Intervening factors ↑

Discussion

Strengths : - focus groups conducted with diverse patient sample which facilitated analysis process to reach saturation.

- 2 qualitative verification procedures were carried out: negative case analysis and investigator triangulation

- negative case analysis examined data to find contradictory evidence to acknowledge breadth of possible responses ( this ensures bias doesn’t not unduly affect analysis

- investigator triangulation also used to diminish bias and affirm consistency of findings by having more than one person conduct analysis.

Limitations: - study not large enough to allow saturation for identification of subtle differences between subgroups (eg. urban vs rural).

- use of volunteer participants likely resulted in overrepresentation of those more interested in patient info needs.

- Most participants in groups were older women.

Conclusion

- both patients and clinicians acknowledge patients need info to make informed decisions about treatments.

- Patients want more info about side effects than clinicians think they should provide.

- Patients and clinicians support use of evidence-based treatment info sheets as means of reinforcing oral advice.

- Results of study were used to help develop patient info sheets compatible with patients’ info needs.

Development of a successful research grant application

Woodward DK and Clifton GD. American Journal of Hospital Pharmacy. 1994;51:813-22.

• Careful planning is essential

• Competition is steep

Sources of Funding:

• Association and foundations

• Associations are membership organizations consisting of individuals with common goals and interests eg. Canadian diabetes society.

• Foundations are non-profit organizations governed by a board of directors.

• Should be sure that your research is consistent with the views of such organizations.

• Government.

• Industry.

• Intramural. This is when institution for which you work (university) funds your research.

Types of Funding:

• Grant.

• Contracts. Usually given out by industry. Monitored more closely.

Locating Funding Source:

• Associations publish RFA’s in journals or newsletters.

• Directories. example The foundation directory.

• Grant offices. Some institutions have departments devoted to research funding.

• Newsletter.

• Computer networks.

Drafting a grant proposal:

1. Develop the idea

2. Determine specific aims and long term goals.

3. Estimate the duration of the project.

4. Estimate the cost of the project.

5. Identify potential funding sources.

6. Obtain application forms and information packets from potential funding sources.

7. Identify colleagues who can review the proposal and request their assiatance.

8. Set deadlines for completion of the first, second and final drafts (more drafts if time allows) and for mailing the proposal. Make sure to find out if the application deadline is the postmark date or the date of receipt by the funding agency.

• Title should be informative and as specific as possible.

• Specific aims and long-term objectives.

• Background and significance.

• Experience and qualifications of the investigator.

• Letters form collaborators and consultants.

• Experimental design and methods

• Budget

• Abstract

• Appendixes (questionnaires, photos, graphs, figures)

Revise the proposal.

Include self addressed stamped envelope for them to reply to you.

Proposal will be reviewed by institutions review group.

Response to Proposal:

• Funding-Granting agencies require strict accounting records. Once research is done final expenditure report is expected.

• Renewal/continuance-Should you go overtime you should inform the granting institution and find out what must be done to get more money if needed.

• Rejection-Reviewers comments and critique should be carefully analyzed. Resubmission is possible if the problem areas can be dealt with.

What is the Question?

Friedman LM, Furberg CD, DeMets L. Fundamentals of Clinical Trials, 3rd ed. St Louis: Mosby-Year Book, 1995.

Fundamental point: Each clinical trial must have a primary question. The primary question, as well as any secondary or subsidiary questions, should be carefully selected, defined, and stated in advance.

Function of the questions:

(primary question is the one that investigators most want to answer and CAN answer

(there can also be secondary questions, related to the primary question. They come in 2 flavours:

1. response (dependent) variable differs from that in the primary question (ie. primary question asks whether mortality from any cause is altered by the intervention; secondary question asks about incidence of cause-specific death, or nonfatal MI, or some other dependent variable that ISN’T mortality)

2. secondary question relates to subgroup hypothesis. For example – investigator may want to divide subjects by stage of disease at entry into the trial, and see if their outcomes differ. Subgroup hypotheses must be specified before data collection begins, based on reasonable expectations, and limited in number (too many = false positives). Usually the number of participants in a subgroup is too small to prove or disprove anything.

(both types of secondary questions raise methodological issues, eg. if you do a significance test for each secondary question you have, they’ll start being significant by chance alone (5% chance). Answers gleaned from multiple testing should be used to suggest new hypotheses, not to propose definite answers.

(Studies can be used to assess adverse effects, but what adverse effects might occur and how severe they might be is unpredictable – you can’t always specify in advance the question you’ll answer. It’s also pretty unethical to continue a study to the point where a drug is conclusively shown to do more harm than good. Statistical significance and false positives aren’t as important as clinical judgment and participant safety, so some coincidental adverse events may be labelled as adverse effects.

(Studies can be used to answer questions that may not bear directly on the intervention you’re testing but are still of interest. These are ancillary (ancillary = of secondary importance) questions. (eg. the Beta Blocker Heart Attack Trial found that low education among survivors of MI was a marker for poor survival risk; ancillary study found that inability to cope with stress was also associated with mortality, even though stress wasn’t the point of the study.)

(Doing a natural history study of the control group is a good use of collected data; it lets you see how baseline factors affected specific outcomes for better understanding of disease under study. Can only infer predictive association, not causation, from such data. Don’t collect unnecessary baseline information thinking that it might be useful – that’s too expensive; collect only what is known to be related to prognosis.

(In a large, simple clinical trial, you have a very large sample size and you do a very modest, simple intervention. These trials are more common for serious conditions, where even modest interventions can make a useful difference. An easily administered intervention is required, as are very brief data collection forms, an easily ascertained outcome (eg. mortality), and a brief follow-up time.

Intervention:

(investigators have a type of intervention in mind when they come up with a question; they often know the exact drug, procedure, or lifestyle modification they’ll study.

(intervention must have maximum benefit with minimum toxicity.

(availability of intervention being tested (eg. if it’s an experimental drug) must be determined – if not yet licensed, get special approval.

(investigators must take into account things like time intervention is started, how long it will last, and how to blind patients to the intervention, among other considerations.

Response variables:

(response variables are outcomes measured during the course of the trial; they answer your question. Examples: total mortality, death from specific cause, disease incidence, specific adverse effect, symptomatic relief, lab measurement, cost of administering intervention…

(Specific response variables may only partially reflect the original question (eg. using extent of mobility as a measurement for “how severe is arthritis?”)

(Pick only one response variable to answer your question, or chance of false positive increases (checking for statistical significance more than once, with a 5% chance of false positive each time…).

(Events can be combined to make up one response variable – but you can only count one event per participant. eg. in a study of heart disease, combined events might be “death from CHD” + “nonfatal MI”. This makes sense, since these two conditions together represent a measure of coronary heart disease. If both events could occur in one individual, you must establish a hierarchy to decide which one counts more – eg. if pat dies, discount nonfatal MI (obviously).

(Another kind of combination response variable: multiple events of the same sort. Instead of “did it happen?” look at “how often did it happen?” eg. frequency of TIA within a specific follow-up period.

(No matter what you’re measuring, some rules apply:

1. Define and write the question in advance, being as specific as possible. “In population W, is drug A at daily dose X more efficacious in reducing Z over a period of time T than drug B at daily dose Y?” Stating the question this way lets you plan your study design and calculate your sample size. (Calculating sample size requires specification of response variables and establishing what happens with a “successful” intervention – 10% improvement? 25%?)

2. The primary response variable must be capable of being assessed in all participants. Response variables must be measured the same way for all participants.

3. Participation ends when the primary response variable occurs, generally. (Investigator may still wish to watch patient – eg. deaths occurring after the nonfatal primary response variable but before the study ends can still be of interest.)

4. Response variables should be capable of unbiased assessment. Double-blinding helps, as do independent reviewers. If a study can’t be blinded, using only “hard,” purely objective response variables (eg. blood pressure) could ensure honest results.

5. Response variables must be able to be ascertained as completely as possible. If you lose a patient to follow-up and the response variable depends on an interview, you’ve lost your information and could unbalance your final results. Death is a good response variable because it is easily assessed, but it is not always applicable.

Surrogate response variables

(Sometimes we use surrogate response variables to substitute for what we actually care about, eg. osteoporosis in bones as surrogate for bone fractures, change in tumour size as surrogate for mortality. Changes in these variables are likely to occur before the clinical event does, shortening the time required for the trial; also, these variables are more often continuous, allowing for smaller sample size and cheaper studies. Surrogate variables aren’t always appropriate – eg. reducing ventricular arrhythmia does not reduce risk of sudden cardiac death; and although use of inotropic drugs in heart failure improves exercise tolerance and symptomatic manifestations, it also increases risk of death.

(Surrogate response variables are useful in the early stages of development of a new intervention (eg. phase I or II) because they speed things up. Before using a SRV in phase III or further on, though, consider:

1. does the variable really reflect the clinical outcome?

2. can the surrogate be assessed accurately and reliably?

3. will the assessment be so unacceptable to the patient that study can’t be done?

4. will assessment require expensive equipment and highly trained staff ($)?

5. small sample size of SRV trials means you may not get all important data on safety. (you may not see all adverse effects)

6. will the conclusions of the trial be accepted by the scientific and medical communities?

\

Impact of total mortality

(Even if the primary response variable isn’t mortality, mortality must still be noted:

1. mortality reduces the overall sample size

2. if mortality is related to the intervention, excluding those who died from study results can seriously bias the study

(To get around this, pick mortality as the primary response variable, or combine it with a nonfatal response variable for a combined response variable.

(If neither of those are feasible, monitor both your primary response variable and mortality and evaluate considering those who died during the study.

Evaluating the Outcomes of Pharmaceutical Care

Lipowski EE. Journal of the American Pharmaceutical Association. 1996;12:726-35.

Measuring pharm care and relationship to patient benefit requires documentation of activity and mechanism of evaluation.

Currently phm’s in a circular argument – phm’s will provide PC with adequate funding, funding only after evidence of PC benefit proved.

Payors skeptical of direct link b/w PC and improved pt. outcomes, no definitive studies.

PC should be proved on key outcomes which patients value most.

Requires participation of many pharmacists – improves exposure and uniformity of care

Evaluation:

Step 1: define the desired outcomes (physiologic parameters, patient’s symptom’s or saticfaction)

Usually focus on 5 D’s – death, disease, disability, discomfort, dissatisfaction.

May be able to measure multiple outcomes in one study.

Step 2: describe the sequence of events starting with phm intervention and ending at desired outcome.

Process may take many steps. (flow chart).

It may be hard to link cause& effect between steps, must validate assumptions used, identify other factors that may lead to outcome

Necessary to find the most efficient way for patient & phm to reach the outcome.

Step 3: determine what evidence needs to be gathered. May need to resort to indirect measurement of

outcome (validate relationship with indirect marker first e.g. BP and risk of MI) Used when:

• not feasible to measure outcome directly

• outcome far removed from process (5 yr survival)

• outcome is hard to define

• too many factors contribute to outcome (can’t control everything)

Selecting Indicators

The best indicators show direct connection b/w phm activity and phm performance to patient outcome.

Want to measure events that are monitored frequently and/or have significant health effect (eg BP)

Also, measure should vary in intensity, frequency and duration (can see if things are changing)

Try to eliminate outside factors, quantitative data better than qualitative.

Evaluate the ability of patients to self report measures - using patients saves effort (eg diabetics)

Patients can fill out standardized forms to assess qualitative data (QOL, Pain inventory)

Positive results in combined qual & quant study reinforce benefit of PC

Evaluation in Prof. Practice

Could get results from one or more pharmacy, individual or groups of patients (need to be defined)Requires documentation, possible claim made for reimbursement for services.

Make other HCP’s aware of the study- may refer patients or help monitor outcomes.

Steps in documenting pharmaceutical care:

Ask following questions in series:

1. What are the objectives of the pharmaceutical care intervention?

- e.g. improve inhaler technique, monitor theophylline serum levels

2. How can progress toward the desired outcomes be measured?

- e.g. observe the inhaler technique performed by the patient

- e.g. observe theophylline level in the lab report

3. What should we do if we are not observing progress toward the goals?

- e.g. modify the process to achieve the goal

- also document the steps you took

4. What evidence can support a claim that a program of pharmaceutical care has accomplished its stated goals?

- record therapeutic goals, actions taken, and results achieved for each patient

- compare this result with other patients with similar conditions and goals

- do the recording from the beginning of the program; don’t leave it to the end!

Using aggregate data to evaluate performance:

aggregating and comparing the experience of multiple patients in the same practice

routine review of success/failure of patient will help the health team appreciate variations among patients

also helps to set realistic goals and expectations for the pharmacist and the patient

• recording the same type of information as other practitioners helps the pharmacist to compare his or her performance to other health practitioners and the literature.

comparisons of data are useful in finding a better way of providing services.

Need for software:

• we need software to do this, but there is lack of good software for pharmaceutical care

• this lack of software is a barrier

• today’s pharmacy software is not equipped for doing pharmaceutical care

• software and integrated electronic medical records system will come from a growing recognition of their importance.

• to provide PC, we need software program that allows us to gather information systematically and make the info available to decision makers

Evaluation as research activity

• We will need two things in order to evaluate the impact of pharmaceutical care: formal research and pharmacist evaluations.

1. we will need more formal research on the impact of pharmaceutical care, because there is not much evidence now.

2. We will also need to know if the results of the research can be extended to real life situation. So, we will need the pharmacists’ evaluation of how well certain pharmaceutical protocol is making an impact on a particular patient.

• Formal research demonstrates what is possible under specified conditions; pharmacist’s (practitioner’s) evaluation demonstrates the achievement of expected results and how to improve upon the results

Evaluation and pharmacy practice:

it is important to document your interventions and outcome measures

results of evaluations will be different from person to person.

understand that even a small intervention will make a significant impact

individual evaluations will help us understand how health interventions work

Philosophy of design and research

Sharpe TR. In Handbook of Institutional Pharmacy Practice. Williams & Wilkins, 1979:337-46.

Evidence and Causal Analysis

• Aim of research is to establish knowledge based upon reliable and valid information and facts.

• There are methods for establishing this knowledge:

- Method of tenacity – people hold firmly to the truth because they know it is the truth. Repetition of facts makes them truth.

o Assumes all people know truth

- Method of authority – an idea is the truth if it has public sanction and tradition behind it.

o Assumes that some people know truth

- Method of a priori (or intuition) – truth is established if the idea agrees with reason or the way things should be.

o Assumes people can find truth without empirical evidence (observation)

- Method of science – truth is based on observation and experimentation

o Assumes truth can only be found with empirical evidence

o This is the focus of the discussion in research design

The Stimulus-Response Model

• All experimental designs have some variation of the stimulus-response model

S R

• This model states that a stimulus treatment elicits a direct and measurable response when applied to a treatment subject.

- eg- a stimulus treatment may be an intervention program to provide a patient with information concerning the importance of finishing his/her antibiotics therapy. The response is an increase in patient compliance

• a variation of the S-R model is when an intervening mechanism takes place within an organism prior to the response

O

S R

• according to this model a stimulus treatment elicits a measurable response which is first mediated by some intervening mechanism

Establishing Causality

• There are 3 criteria for establishing causality in the S-R model

1. Association

• Includes 2 characteristics that strengthen the conclusion that one variable is the cause of another: magnitude and consistency

• The greater the magnitude of the relationship between 2 variables, the more confidence one has that the association is truly causal

• If a relationship persists under a variety of conditions, this causes consistent confidence in the causal nature of the relationship.

o eg – the relationship between smoking and cancer has been found in prospective and retrospective studies, animal and human studies, in different ethnic and racial groups and in males and females.

• A causal association may also exist when two variables are just barely related because there may be several factors producing an event.

• All causal relationships apply only under certain circumstances, therefore if results are not consistent, it may be that the causal relationships apply only under certain circumstances

• Also, consistence is a necessary but not sufficient condition to establish causality.

2. Time Priority

• An event in the future cannot determine an event in the past or present

• Without knowledge concerning time priority, we are limited to making conclusions only with respect to association among variables.

3. Spurious (false) Relationship

• Assuming that the effects of all relevant variables have been eliminated, then we can be more confident in interpreting the relation as being causal and not due to other factors

o eg – if it can be shown that genetic factors predispose to a smoker to getting cancer, then it would be spurious to interpret the relation between smoking and cancer causal.

• Therefore, only when all three conditions of association, time priority and non-spurious relationship exist can we draw conclusions concerning causal relationships

Characteristics of experimental designs

See figure 1.1 – Implementation of an experimental design

• There are 3 main conditions of the experimental method : (1) Sampling equivalent experimental and control groups; (2) isolation and control of the stimulus; (3) definition and measurement of response

1. Sampling equivalent experimental and control groups

i. Defining the Study population

o The definition of the population to be studied will largely depend on how results of the study are to be applied

o Most common problem is defining the population too broadly

o The population should not be defined as greater that the group from which the researcher can randomly sample

ii. Sample selection

o Many populations are impossible or impractical to study directly because of lack of accessibility

o Data is usually gathered on only a part or sample of the population

o Conclusions are then specified concerning the population

o The size of the sample depends on the anticipated degree of effect: the greater the degree, the smaller the sample required

o Random sampling is a method of drawing a sample population such that every member has an equal chance or being selected. (therefore the sample represents the population from which it was drawn)

o Stratified random sampling first divides the population into strata (ie age groups, men and women) and random samples are drawn from each of these strata in proportion to their presence in the population. This is useful if there is some basis for expecting that the strata are related to the phenomenon being studied.

o Non random sampling include:

▪ Convenience sampling – selection is based on the convenience to the investigation

▪ Quota sampling – the population is divided into groups according to selected characteristics which are considered important

▪ Interval sampling – individuals are selected in a periodic sequence.

▪ Judgement sampling – selection is based on the investigator’s judgement that the sample is representative of the population

▪ Systematic sampling – choosing every nth person from a population. Common if a list of people in population is available.

o Without some form of random sampling, no conclusions can be inferred concerning relationships in the population.

iii. Random assignment to experimental and control

o The essence of a true experimental design is that the available pool of subjects is randomly sorted into the various groups

o An equal or nearly equal number of people should be placed in each group

o Random assignment ensures that the subjects will not differ on the average with respect to any characteristic more than could be expected by chance alone

o The randomization process ensures that there are no systematic influences that tend to make the average value of a variable higher in one group than in another.

2. Isolation and control of the stimulus

• Careful formulation and definition of the stimulus (independent variable) are important to increasing reliability of the study measurements

• It is important that each member in the experimental group receive the same treatment.

• This is important for the immediate study and for those who with to replicate the investigation

• Stimulus variables should be clearly defined including specific content, message, medium and situational environment. eg- unacceptable to say “experimental group received intensive counseling”. The intensive counseling must be more clearly defined.

• Isolation of the stimulus is also important since introducing extraneous stimuli can confound the effects resulting from the stimulus treatment.

• Also important to isolate treatment from control.

3. Definition and Measurement of Response

• Defining response (dependent variables) is also important

• The dependent variable should also be defined in measurable terms

Reliability and Validity

Reliability

• Reliability is consistency

• Refers to the degree to which the measure produces consistent results upon repeated application

• Classified into four types:

1. Congruency: the extent to which several indicators measure the same thing

2. Precision: the extent to which an indicator is consistent for a single observer

3. Objectivity: the extent to which the same instrument is consistent for 2 or more variables

4. Constancy: the extent to which the object being measured does not fluctuate

• Five sources of unsystematic variables (unreliability) in experiments include:

1. Subject reliability: subjects mood may affect physical and mental health

2. Observer reliability: subject factors also affect the way an observer makes their measurements

3. Situational reliability: conditions under which the measurement is made may produce changes in results

4. Instrument reliability: the factors of the instrument itself may affect is reliability, ie – poorly worded questions in an interview

5. Processing reliability: coding or mechanical errors occurring at random in an unsystematic manner

• All results contain error, therefore the researcher must reduce this error such that it does not interfere with the valid use of the measurement instrument

• Reliability is best controlled by careful attention to factors which permit large chance error to enter the experiment.

Validity

• Validity is the degree to which a measure or procedure succeeds in doing what it claims to do ( Are we measuring what we say we are measuring?

• It reflects systematic errors that represent bias, slanting the results in a particular direction rather than at random

• Because of this non-randomness, errors may falsely be interpreted as causes of these trends

• Internal validity

□ Did the experimental treatments make a difference in this specific instance?

□ This is the basic minimum, so that without it, any experiment is uninterpretable

• External validity

□ To what populations, setting, treatment variables and measurement variables can this effect be generalized?

• Campbell and Stanley identified 8 classes of extraneous variables which might produce effects confounded with the effect of the experimental stimulus if not controlled:

1. History: events that occur between pre-test and post-test that could provide an alternate explanation of the effect

2. Maturation: processes producing changes as a function of the passage of time (eg. growth, fatigue)

3. Testing: the effect that taking a test will have upon the scores of a second test

4. Instrumentation: changes in calibrations, observers or scores used may produce changes in measurements

5. Regression artifacts: pseudo-shifts occurring when persons or treatment units have been selected upon the basis of their extreme scores

6. Selection: biases resulting from differential recruitment of comparison groups

7. Experimental mortality: differential loss of respondents from comparison groups

8. Selection-maturation interaction: selection biases that result in differential rates of maturation

• They also outlined 4 factors that jeopardize external validity:

1. Interaction effect of testing: the effect of a pre-test influencing respondents sensitivity or responsiveness to the experimental variable, making it unrepresentative of an unpre-tested universe

2. Interaction of selection and experimental treatment: unrepresentative responsiveness of treated population

3. Reactive effects of experimental arrangements: conditions making the experimental setting atypical of regular application of the treatment (“artificiality”, Hawthorne effects)

4. Multiple-treatment interference: multiple treatments are jointly applied, effects atypical of separate applications

Classification of Research Designs

• Campbell and Stanley classified experimental designs and evaluated them according to their typology of threats to internal and external validity

• Legend to understanding the graphical representations below:

|X |Exposure of group to experimental treatment |

|O |Process of observation or measurement |

|Xs & Os in a row |Applied to same specific persons (in temporal order) |

|Xs & Os vertically |Simultaneous |

|R |Random assignment to experiment & control groups |

|Rows separated by a line |Comparison groups not equated by random assignment |

Pre-experimental Designs

1. The One-shot Case Study: X O

• Example: 8 cases of thrombosis (incl. 1 death) in patients treated with azarbrin (anti-psoriatic) during the 1st year of marketing when 500-100 patients were being treated with it ( this lead to an FDA request to withdraw from the market

• In this design observations are made after treatment has been administered

• Simplest form of research design, the one most frequently used in pharmacy and medicine

• Weakest form of design, because no baseline measurements or control groups ( no way to evaluate if response was due to the stimulus

• Results are testimonials

• Primary sources of invalidity: history, maturation, selection, mortality

2. The One-group, Pre-test, Post-test Design: O1 X O2

• Example: Study to determine the analgesic activity of an investigational new drug AX-735. It involved administering a pin prick to patients right forearm to measure their pain threshold (PT), before giving drug and 4 hours after. A lower PT was found post-medication which the researchers attributed to the drug.

• Introduces baseline measurements before stimulus administered, followed by a measurement after

• Researcher can objectively measure change, improvement over method #1

• It doesn’t allow researcher to attribute this change to the stimulus administered

• 5 main threats to internal validity are:

□ History ( other events may occur simultaneously influencing effect

□ Maturation ( people may improve with/without exposure to stimulus

□ Testing ( the first measurement may constitute a stimulus itself

□ Instrumentation ( after measurement may be due to fatigue or instrument reliability

□ Statistical regression ( unreliability may produce statistical regression with shifting values toward the mean

3. The Static Group Comparison X O1

O2

• Example: Another study to determine analgesic activity of AX-735. 25 patients on one floor of a hospital were given a 200 mg dose and PT measurements were done 4 hours later. 25 patients on another floor were given placebo and PT measurements were taken 4 hours later. A lower PT was found in the treatment group vs. placebo which researchers attributed to AX-735.

• Merely gives the appearance of providing a control group since it was not randomly assigned

• 2 threats to internal validity:

□ Selection ( differential recruitment of observation groups can lead to differences in response regardless of stimulus

□ Mortality ( differential drop-out may create differences between observation groups

True Experiment Designs

• Allow researcher to objectively measure change and to attribute it to experimental stimuli

4. The Pre-test, Post-test Control Group Design R O1 X O2

R O3 O4

• Example: Pharmacists who attend weekly CE meetings were randomly assigned to treatment and control groups. Week 1: Pharmacists were given a Diabetes Mellitus drug therapy test. Week 5: Experimental group shown a DM drug therapy film. Week12: Both groups re-administered DM test. Mean improvement score was greater for treatment group. Investigators attributed this to the film stimulus.

• Most frequently used of true experiment designs

• Groups are equivalent due to randomization

• Baseline is measured and compared to the “after” measurement

• All threats to internal validity are controlled since extraneous variables would produce differences in both groups since they are equivalent and mortality can be evaluated by examining differential drop-out rates

5. The Post-test Only, Control Group Design R X O1

R O2

• Example: Double-blind study to determine effectiveness of methods to prevent cephalothin-induces phlebitis. Patients were randomly assigned to 2 treatment groups: 1. Heparin 1000 mcg + hydrocortisone 10 mg, 2. Heparin 500 mcg + hydrocortisone 1 mg. Patients were given cephalothin and phlebitis was assessed q12h. Significant differences were found in both groups, found attributable to the treatment.

• Similar to design # 4 except without the pre-test which is not essential since randomization can suffice without it, and this is the most adequate way to ensure equivalency of both groups

• Controls all threats of validity without actually measuring them like in design #4

6. The Solomon Four-group Design R O1 X O2 This is part of Design #4

R O3 O4

R X O5 This is part of Design #5

R O6

• Controls and measures experimental and possible interaction effects of measuring process

• Allows researcher to determine whether pre-test was a stimulus itself, this would be indicated by significant differences between O2 and O5 and/or between O4 and O6

Quasi-Experimental Designs

• Often realities prevent researcher from controlling sample selection, randomization and scheduling stimuli, making a true design not possible

• Don’t control threats of internal validity but can determine whether a rival hypothesis can be ruled out through circumstantial evidence

7. The Time-Series Experiment O1 O2 O3 O4 X O5 O6 O7 O8

8. The Equivalent Time-Samples Design X1 O X0 O X1 O X0 O

9. Non-Equivalent Control Group Design O1 X O2

O3 O4

Practical Guidelines of Effective Study Design

1. Carefully define the study population

2. Sample randomly from the population, if possible

3. Randomly assign subjects to experimental and control groups

4. Carefully define all variables and conditions

5. Carefully isolate experimental and control conditions

6. Carefully develop measurement instrument and pre-test it

7. Carefully train observers in proper use of measurement instrument

8. Observe inconspicuously, if possible

9. Conduct investigation double-blindedly

10. Choose an appropriate statistic for making comparisons

11. Use a true design vs. a quasi design, if possible

12. Be careful that conclusions are firmly supported by data

Determining the value of pharmacy services — the search for rigorous research designs

Malone DC. Journal of Managed Care Pharmacy. 2002;8:153-5.

Basic message: Author briefly summarizes studies evaluating clinical pharmacy services with respect to study design and results. He presents suggestions to improve the quality of research associated with pharmacy services.

Example of a good study (conducted by the author of course)

IMROVE study to evaluate ambulatory clinical pharmacy services plus medical care (n = 523) vs. medical care only (n = 531)

Subjects were randomized into treatment and control groups at 9 VA medical centres.

No difference in terms of SF-36 scores or overall resource utilization, but costs of pharmacy services did not increase overall costs.

Subset of hyperlipidemic patients showed significant decrease in TC and LDL vs. control

Conducting Rigorous Research fo Pharmacy Practice

It is important not to overstate or understate the value of pharmacy services based upon studies with poor research designs.

Common study problems are:

Lack of control group

• Control group serves to rule out alternative explanations for observations.

• Control group crucial in IMPROVE study because entire VA health care system simultaneously initiated program to improve hyperlipidemia treatment.

Selection bias

• Especially problematic in retrospective studies because difficult to find identical treatment and control groups; they tend to differ in disease severity.

• Disease severity measures generally not available in administrative databases.

• Alternative is to identify a surrogate e.g., clinic visits, hospitalizations, medication use..

• Often still difficult to find identical control groups so may need to normalize differences by regression of outcomes such as post-period costs and hospitalizations.

• Be ware of self-selection bias e.g., patients who agree to be study measuring patient satisfaction will likely report higher satisfaction anyway; sometimes difficult to avoid with interventions that are obvious or cannot be masked.

Selecting cases based on extreme scores

• Patients selected on basis of extreme values of observed parameter tend to “regress to the mean” of the population.

• This is because out-of-range subjects statistically will become more normal even when no intervention is made.

• So studies that select especially poorly performing subjects at outset will observe improvement regardless of intervention.

• Therefore especially important to have control group.

• Control group can be formed in a separate but similar setting as the one providing care.

• Randomization is preferred method of forming control group to eliminate selection bias.

• Pharmacist performing intervention should not participate in group assignment to avoid bias.

Poor measurement of principal outcomes

• Use previously validated measurement tools; published tool is not necessarily validated.

• Tool should correlate with an objective pathophysiological parameter (e.g., asthma QoL with lung function).

• Should be sufficiently sensitive, but not prone to false positives (test-retest reliability).

Inadequate follow-up periods

• Many researchers conduct a study with 3- to 6-month pre- and post-intervention measurement periods.

• Changes in chronic disease control are likely to take months to manifest e.g., hypertension, diabetes or with rare events e.g., asthmatic attack

• Recommend collect data for 12 months retrospectively before implementing service (baseline) and 12 months prospectively when possible.

Recruiting & Retaining Patients in Research

Farley E. In Conducting research in the practice setting, Eds: Bass MJ et al, 1993. Sage Publications, 58-67.

|Factors/Determinants of patients’ |Discussion |

|willingness to participate in research | |

|-type of study proposed |-in any study that involve patients directly, the patients should acquire a clear understanding of the purpose, |

|-risks, time, inconvenience involved |procedures, risks, potential outcomes for the subject, and potential significance for others |

|-presence or absence of invasive |-most practice-based research is associated with no risk (requires minimal to moderate input from pt) |

|procedures |-Characteristics of studies most accepted by pts: low risk study that offers greatest potential good to society |

|-potential value to themselves |-most demanding & disruptive routine: clinical drug trials (esp. in Phase III) |

|& others | |

|-quality of provider/pt relationship |-**provider-patient relationship can be a significant factor in patient’s acceptance |

|(>85-90% of patients are willing to |(e.g. Dr. David Hahn reported that majority of the patients who declined to participate in an asthma study |

|become involved in research in |were new patients to the practice; those patients with whom he had an established relationship not only |

|approached by physicians) |participated willingly but at times also ask when he will be starting another study) |

| |-patients who have limited or no ability to select their personal providers (e.g. in isolated communities with |

| |one physician, in group practices, or restrictions of a health insurance plan) may be less likely to participate |

| |-acceptance is also higher if the patients feel that there is a sense of personal |

| |connection (if the practice is organized so that patients and their doctor(s) know |

| |each other) |

|-nature of practice |-practice type, function, and structure of a practice can determine what types of research can be done and |

| |patients’ acceptance |

| |-traditionally, large, impersonal hospital clinics have had more difficulty with compliance than smaller, more |

| |personal private practices. However, a review conducted by the author shows little difference between these |

| |two types of environments. This finding reflects that patients develop great loyalty to any organization or |

| |institution from which they receive ongoing care |

| |-the nature and size of a practice also influence acceptance |

| |-mixed specialty practices may be able to undertake studies different from those of single-specialty practices |

| |-primary care practices can undertake studies on a subset of the population different from those of |

| |subspecialty practices |

| |-e.g. patients in a health maintenance organization (HMO) may be more amenable to studies on |

| |prevention than patients in fee-for-service practices |

|-practice management |-factors that affect patients’ willingness to participate in research are: |

| |how patients are scheduled with their physicians or other providers |

| |how available the practitioners are to the patients |

| |the hours the practice is open |

| |how support staff relate to the providers, the patients, and the community |

| |how the business office handles billings and collections (particularly for those who |

| |have difficulty paying or who receive Medicare benefits |

| |the general ambience of the facility |

| |-how a practice organizes the data it collects in the ongoing care of patients helps determine the type of |

| |research that can be done |

| |-an important starting point is the minimal basic data set (MBDS) collected on all patients |

| |-even if the MBDS only include the usual registration data (name, date of birth, address, telephone number, |

| |head of household, and insurance company payer), its an excellent basis on which to define research |

| |questions and to manage the practice (by dividing the population served into cohorts) |

| |-MBDS is expanded to include disease or problem, education, occupation, race, religion, ethnicity & |

| |members of household |

|-patient education |-practices that emphasize on patient education for health maintenance and disease prevention usually |

| |attract patients who are more motivated to remain in good health |

| |-some practices use the service of a nurse practitioner or health educator who are |

| |usually very comfortable in communicating with patients and often can be |

| |used to help recruit and retain patients into studies |

| |-patient participation usually is excellent when studies involve some connection with health maintenance |

| |or disease prevention |

|-relationship of practice to the |-practices that use a community-oriented primary care (COPC) approach are involved fully in the community, |

|community |have an advisory committee of community members, and work closely with the community to improve the |

| |overall health status of the population |

| |-success of such practices depend on the strength of the advisory committee (composed of representatives |

| |from the community, including medical personnel) |

| |-the committee gave the physicians, nurses, health visitors, and medical anthropologists an opportunity to |

| |work with the community on common health concerns, to explain what we could and could not do in the |

| |way of prevention, diagnosis and treatment, and to explain what we were doing in the way of research |

| |-examples of studies that use a COPC approach are studies on the prevalence and ambulatory treatment |

| |of TB, streptococcal disease |

| |-COPC practices usually have little problem enrolling and retaining patients in studies |

| |-patients do not want to be “guinea pigs”; when a community believes the practice group has a sense of |

| |responsibility toward its |

| |members, individuals are usually very willing to participate in research |

|-previous involvement of practice in |-patients who have had good research experience with a particular practice or |

|research projects |provider usually will be more willing to participate in other projects |

|-role of learners in the practice |-learners, such as residents or medical, nursing, and physician assistant students, can have a significant |

| |impact b/c if these learners are integrated into practice setting in a manner that wins patient acceptance |

| |and interest, they can increase the willingness of patients to participate in a specific projects |

| |-from the author’s experience, practices that include learners have no problem with patient involvement in |

| |research if the patients are made aware of the activities and feel included in the process and if the learners |

| |are monitored closely and have clearly delineated roles that are consistent with their learning stage |

|-characteristics of population served |-it is important to understand the population served because socioeconomic and sociocultural status may |

| |affect how an individual or group accepts involvement |

| |-clearly defined distinguishing attributes of some groups allow the researchers to develop hypotheses of |

| |questions that practice- or population-based research may answer |

| |-with the diverse people who make up the North American continent, it can be expected that practice-based |

| |research will come to focus increasingly on health and illness patterns associated with cultural and religious |

| |diversity |

| |-understanding sociocultural diversity within a racial or cultural group can enable us to provide care, determine |

| |individuals’ willingness to participate in studies, to ask the right questions, and to undertake the right studies |

| |concerning the effect of our care on individual health and function |

| |-another population aspect that must be considered is access to medical care which is affected by |

| |socioeconomic (e.g. people who are under- or uninsured and do not have easy access to public- |

| |supported care) or cultural factors |

|-whether they get paid or not |-remuneration is very important that require considerable patient time & commitment in order to maintain |

| |continuing |

| |involvement in studies |

| |-patients should also be paid for any study-related costs |

| |-NOT usually the primary factor in determining whether patients will participate or not |

|-social/moral implications of the problem |-ethical issues are always present & must be dealt with in any research |

|(e.g. HIV/AIDS) |-all practice-based research MUST be done in a way that ensures continued confidentiality & minimizes |

|-public awareness of the problem |the chance |

|-confidence in whether confidentiality |for any social, psychological or physical harm to the patient as a result |

|be maintained | |

|-retention of patients in prospective |-one reason why some pts enroll in studies is b/c they believe they will benefit from the extra attention |

|studies |& tests, |

| |therefore, it’s important to maintain that hope in the pts in order to get continual involvement from them |

| |-patients seen for a scheduled study visits will continue to present other problems that must be either |

| |attended to at |

| |that time or followed up at a later time. |

Increasing response rates to postal questionnaires: systematic review

Edwards P, Roberts I, Clarke M, DiGuiseppi C, Pratap S, Wentz R, Kwan I. BMJ. 2002;324:1-9.

The article describes the systematic review of 292 RCTs of any method to influence response to postal questionnaires

Objective of the study: To identify methods to increase response to postal questionnaires.

Main outcome measure: The proportion of completed or partially completed questionnaires returned.

Postal questionnaires:

- used to collect data in health research

- the only financially viable option when collecting info from large, geographically dispersed populations

Non-response

- reduces the effective sample size

- can introduce bias, i.e. affects the validity of epidemiological studies

There is a need to identify effective strategies to increase response to postal questionnaires to improve the quality of health research

Methods:

The RCTs

- were found through both electronic database and manual searching (reference lists of relevant trials and reviews)

- were not restricted to medical surveys; 1/3 medical, epidemiological, or health related; ¼ psychological, educational or sociological; 2/5 marketing, business or statistical

- included any postal questionnaire topic in any population

- included studies in languages other than English

- included strategies requiring telephone contact

- excluding (due to cost) the strategies requiring home visits.

75 different strategies for increasing response to postal questionnaires were identified

- the average # of participants per trial was 1091 (range 39-10047)

- all tests for selection bias were significant (P ................
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