Pharmacoeconomics & Health Outcomes

Pharmacoeconomics & Health Outcomes

Health & Economic Data Sources

Leon E. Cosler, R.Ph., Ph.D. Associate Professor of Pharmacoeconomics Albany College of Pharmacy

Road Map: Data Sources 1. Identify various data sources useful for this field 2. Advantages & disadvantages of different types 3. List several government databases

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Selected examples

4. Current laws / regulations in human research

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Patient informed consent process

“Where’s he getting this?” • Parts of Bootman Text Ch. 12

Recommended Steps for P-E study

• • • • •

Well defined hypothesis Perspective is specified Alternative therapies / options considered Clinical effectiveness is considered All relevant costs / outcomes identified • and accurately measured

• •

Study design / methodology appropriate Results interpreted and communicated

Data Sources

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Selecting source for data depends on: • • • • •

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The type of data needed The research question(s) The study perspective Access to the data Resources ($$$) you have to spend

Common sources include: • • • • •

Nationally collected data Payers (insurers, medicaid, medicare) Providers (inpatient*, MCOs, PBMs) Rx clinical trials (more on this later…) Patient surveys

The type of data needed:

- Economic data ($$$$) • Direct Medical Costs (easiest) - hospitalizations - visits: MD, clinic, ED, DDS, - diagnostic procedures - lab tests - medications - home health care - medical equipment / supplies

The type of data needed:

- Economic data ($$$$) • Some $$$ only available from Pt.: - out-of-pocket $$$ (co-pays) > OOP or TrOOP (true out of pocket) costs - non-medical direct $$$

• Requires surveying the patient - Pt. diaries or journals - difficult, but can be very important - driven by study perspective

The type of data needed:

- Patient Outcomes data • clinical outcomes get from: - Ex: medical records

• humanistic outcomes get from: - Ex: patient surveys or questionnaires

Data Sources

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The research question(s):

- Specifically defined study objective(s): • clearly drives the data you will need

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The study perspective determines:

- “costs” or “charges” - which categories are needed? • and suggests where to get them...

Access to health databases:

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Provider / Payer cost data • often considered proprietary info. • also private medical info. restrictions • “willingness to share” differs

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Surveying patients • often requires “informed consent” • IRB review of protocol

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Release of medical information • HIPAA & PHI (protected health information)

http://www.hhs.gov/ocr/hipaa/

Resources ($$$) for data:

- Some organizations charge for data - Researchers (i.e.: you) may need: • computer programming assistance • technical support to understand the data • extra computer equipment / software • statistical consultants

External Data Sources

- already published studies • comparable therapy to your objective? • quality study? • “meta-analysis”

- Medical claims and databases • ex: Medicare / Medicaid / Insurers • very limited clinical measures • rarely any patient outcomes • no humanistic outcomes

Internal data •

inside your (hospital / medical center)

- requires sufficient # of cases - generalizable only to your situation •

Medical Records have rich clinical info. • difficult to extract & recompile • difficult to get large samples • often a costly source of data • New trends toward electronic medical records (EMRs)

Frequently Used Data Sources

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Hospital based data Medical Record Information

Billing Data

Admission / Discharge

Pt. Demographics

No. of products received Length of stay

Diagnosis Codes Procedure Codes MD information Severity of Illness

No. of tests Department costs Total hospital costs Total hospital charges

Admission type Place of admission Discharge status

- Remember these are different: • hospital “cost” • hospital “charges”

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Requires permission to access records

Pharmacoeconomics & Health Outcomes

Sources of Health Care Data

Leon E. Cosler, R.Ph., Ph.D. Associate Professor of Pharmacoeconomics Albany College of Pharmacy

http://www.nlm.nih.gov/hsrinfo/datasites.html

Government Databases

Census Data (www.census.gov)

Census Data

Government Databases: CMS

Government Databases: CDC - NCHS

Government Databases: CDC - NCHS

Government Databases: AHRQ

Pharmacoeconomics & Health Outcomes

Human Subjects in Research Research Designs

Leon E. Cosler, R.Ph., Ph.D. Associate Professor of Pharmacoeconomics Albany College of Pharmacy

Nazi Medical Experiments

1940s

Key Historical Events...

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Nazi Medical Experiments

1940s

- Nuremberg Medical Trials - Nuremberg Code

1946 - 47 1947

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Declaration of Helsinki

1964

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Belmont Report

1978 *

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National Research Act

1974

- 45 CFR 46 (PHS) - 21 CFR 50, 56, 315, 812 (FDA)

1974+ 1981+

Key Historical Events... The Nuremberg Code: 1947 1. Voluntary consent essential 2. Legal capacity to give consent 3. Without force, deceit or coercion 4. Have knowledge & comprehension of the experiment’s elements to make an informed decision

Tuskegee Syphilis Study

( 1932 - 72 )

Tuskegee Syphilis Study (1932 - 72)

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US PHS funded study of untreated syphilis

- re-reviewed in 1969 “justified & beneficial”

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Tuskegee Syphilis Review Panel (1972)

- found PHS study “ethically unjustified”

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Called for new federal office (the office of human protections) “… with the authority to regulate all federally supported research involving human subjects…”

Key Historical Events...

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National Research Act of 1974:

- National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research

- Published “Ethical Principles and Guidelines for the Protection of Human Subjects (aka The Belmont Report, 1978)

The Belmont Report (1978) :

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Defines “research”

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3 Basic ethical principles • •

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Respect for persons Beneficence – cannot lie to them…full disclosure…do not do experiments that will hurt them Justice – pick patients fairly…don’t pick poor men in Tuskegee b/c they have syphilis..dont pick prisoners

“Informed Consent”

CFR Title 45 Part 46 (1991): “Protection of Human Subjects”

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all research involving humans supported by any Federal agency

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established the role of Institutional Review Boards (IRBs)

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Defines • “research” • Human subjects • “informed consent”

CFR Title 45 Part 46 (1991): Research •

“…(d) Research means a systematic investigation, including research development, testing and evaluation, designed to develop or contribute to generalizable knowledge. Activities which meet this definition constitute research for purposes of this policy, whether or not they are conducted or supported under a program which is considered research for other purposes. For example, some demonstration and service programs may include research activities…”

Human Subject •

“… (f) Human subject means a living individual about whom an investigator (whether professional or student) conducting research obtains: (1) Data through intervention or interaction with the individual, or (2) Identifiable private information….”

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

FDA - Regulated Research

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INDs / IDEs in addition to IRB review Additional Laws:

- 21 CFR 312, 314, 600, 812, 814

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Investigators required to sign FDA 1572 Obligations of the study sponsor

- industry-sponsored studies

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Safety Reports: SAEs reported promptly

http://www.fda.gov/oc/ohrt/irbs/default.htm

Institutional Review Boards (IRBs)

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Must have at least 5 members

- diverse with at least one “non-scientist” - one outside the organization - Three “levels” of protocol review: a. “exempted” from review b. “expedited” review c. “full” board review if it is high risk study

IRB reviews the following:

2.

Assess risks to patients

3.

Equitable selection of patients

4.

Informed Consent

5.

Adequate monitoring of patient safety

6.

Adequate protection of privacy (PHI)

Informed Consent components

1. -

Declaration statement Risks / benefits Alternative procedures Confidentiality procedures Medical treatment if needed •

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Usually responsibility of sponsor

“Who do you call… “ Voluntary statement •

Says you are volunartily being asked to patricipate…may leave study at any time you want

Jesse Gelsinger

(1981 - 1999)

Current Developments (2000 +)

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Federal Office Renamed: OHRP

- All P.I.s must be trained - NIH considers adding funds for IRBs - FDA updating information sheets and adverse event monitoring

- changes to 45 CFR 46 considered

Ellen Roche (1977 - 2001)

Important Web sites http://www.hhs.gov/ohrp/ http://www.primr.org/

http://www.fda.gov/oc/ohrt/irbs/default.htm http://www.nih.gov/sigs/bioethics/IRB.html

That’s all for today… !