Introduction to Health Economics

and Outcomes Research (HEOR)

for Writing Professionals
B e t h L e s h e r, P h a r m D , B C P S
Catherine O’Connor Mir vis, BA

www.pharmerit.com
4350 East West Highway, Suite 1110 | Bethesda, MD 20814
Agenda
Introductions
What is HEOR?
How can I break into HEOR writing/editing?
Who uses HEOR evidence?
How is HEOR evidence used?

2
Presenters
Beth Lesher, PharmD, BCPS

Associate Director,
PharmD Clinical Medical
Undergrad
Pharmacist/ Writing Freelance
Strategic Market Access
Critical Care
BS Pharm Academia Fellowship
Residency

Catherine Mirvis, BA

Sr. Communications Analyst,

NCI Internship English Major
Smithsonian Editorial Strategic Market Access
Volunteer Assistant

3
 PART 1:
WHAT IS HEOR?
 Health Economics & Outcomes Research
Health Economics
Analyzes the economic aspects of health and healthcare, with a focus on the costs (inputs) and
consequences (outcomes) of healthcare interventions.

Outcomes Research
Evaluates the effect of healthcare interventions on patient-related clinical, humanistic, and
economic outcomes.

Health service Health economic

Clinical research
research evaluation
Clinical Clinical outcomes
Policy research
epidemiology assessment

5
Berger et al. Health Care Cost, Quality, and Outcomes. ISPOR Book of Terms 2003
 What Outcomes Are Generated?
Economic Clinical Humanistic
ECHO MODEL

Impact of intervention on costs; Impact of an intervention on

Measurable changes in health
direct and indirect costs derived patient-reported endpoints; also
status due to an intervention
from clinical outcomes derived from clinical outcomes

Includes: Resource use, work

productivity, loss of work, burden Includes: effectiveness, morbidity, Includes: Health-related quality
of illness, cost-effectiveness, mortality, function of life (HRQOL), preference,
transportation caregiver burden

Evaluated using economic or

pharmacoeconomic analyses Evaluated using general or
Evaluated through clinical trials,
(e.g., cost-benefit, cost- disease-based patient/caregiver
post-marketing reports
effectiveness, cost-minimization, questionnaires or surveys
cost-utility, budget impact
models)
Examples: Cure, clinical goal Examples: SF-36, EQ-5D,
Examples: ICER, QALYs, PPPM, (HbA1c, BP), secondary patient satisfaction, patient
PPPY absenteeism, LOS, office prevention, remission, adverse preference, validated and
visits event rates, compliance unvalidated tools/surveys

Berger et al. Health Care Cost, Quality, and Outcomes. ISPOR Book of Terms 2003; Kozma et al. Clin Ther 1993;15:1121-32; Outcomes Research; available at
http://www.amcp.org/WorkArea/DownloadAsset.aspx?id=19128. Accessed July 17, 2018 6
 Where Does HEOR Evidence Come From?

Indirect Prospective
Chart Reviews Treatment Observational
Comparison Studies
Real-world Clinical
Surveys
Evidence Studies

Open-label Patient-
Prospective Health
Studies reported
RCTs Status
Outcomes

Observation Meta-
Productivity
al Studies analyses

Clinical Post- Claims

Outcomes marketing Registries Database
Assessment Studies Analyses

Systematic
Symptoms
Reviews

7
Who Performs Outcomes Research?
Academic
institutions

Health plans/
Physicians
Medical groups

Pharmaceutical
Pharmacists HTA bodies
companies

Government
Nurses
agencies

Other healthcare
professionals

Economists

8
 HEOR Evidence and Product Life Cycle
Preclinical • Exploratory research • Unmet needs/gap analysis • Very early modelling
• Market assessment • KOL research

• Market assessment • Early modelling, early pricing models • Piggyback studies

Phase 1 and 2 • Unmet needs/gap analysis • Burden of illness studies • PRO development, testing, validation

• Payor assessment • Registries • Pricing and reimbursement

Phase 3 and 3b • Model development/validation • Comparative effectiveness research • AMCP dossier
• Piggyback studies • Value message development • Global value dossier

Product Approval and Launch

• Phase 4 studies • Comparative effectiveness • Retrospective studies • AMCP dossier

Post-launch • Model refinement research • Database analyses • Global value dossier
• Piggyback studies • Prospective observational • Chart reviews • HTA
• Registries studies • Safety surveillance

Loss of • Safety surveillance • Comparative effectiveness research • HTA

Exclusivity • Real-world studies • Global value dossier

9
Why Do We Need HEOR Evidence?

 Identify unmet
 Supplement
 Address
 Promote
patient-
needs RCT with RWE evidence gaps centered
research

 Help develop/
evaluate cost
 Adapt data to
different
 Respond to
changes in
 Comply with
HTA
containment market
populations submission
strategies environments

10
 Summary: Who, What, Where, When, How

• • Outcomes • Clinical studies

What

Where
Who

Regulators
• Patients – Economic • Real-world evidence
• HCDM – Clinical • Patient-reported
• HCP – Humanistic outcomes
• Politicians/Advocacy • Modelling

• •
When

How
Pre-clinical Product approval
• Phase 1/2 • Education/awareness
• Phase 3/Pre-launch • Reimbursement
• Post-launch • Formulary placement
• Loss of exclusivity • Guidelines

11
 PART 2:
WRITING AND EDITING IN HEOR
Roles for Writing Professionals in HEOR
Writing Editing Project Management

• Dossiers • Dossiers • Dossiers

• Publications • Publications • Publications
• Value messaging • Slide decks • Reports
• Objection • Reports
handlers
• Study reports
• Modelling reports

13
 Tips for How to Enter the HEOR Space

Leverage Your Know Your Know Your Expand Your

Skills Audience Resources Knowledge
• Manuscript • Journal selection • ISPOR website • ISPOR short
writing • HCDMs / Payers • AMCP format courses
• Editing • Global • HealthEconomics. • AMCP meetings
• Scientific • National com • HEOR meetings
background
• Slide decks
• Reports

14
Tips for Nontechnical Backgrounds
Learn or brush up on statistics
Promote your Microsoft Word knowledge
Know your audience (HCDMs, HTAs, patients)
Don’t take your liberal arts skills for granted!
• Audience analysis
• Big picture
• Writing mechanics
• Organizing ideas
Insight into non-expert audiences
AMWA workshops
Focus on your “highest and best use”

15
Where Can I Get More Information?

ISPOR ISOQOL AMCP CHEERS

ispor.org isoqol.org amcp.org Guidelines

NICHSR AHRQ PCORI

nlm.nih.gov/nichsr/hta101 ahrq.gov pcori.org

HealthEconomics.com

16
 PART 3:
HEOR EXAMPLES
Pharmacy and Therapeutics (P&T)
Committee
What is a Formulary?
What is a P&T committee?
Who is on the P&T committee?
• Pharmacists
• Doctors
• Nurses
• Lawyer
• Quality assurance
• Lay member

18
Pharmacy and Therapeutics (P&T)
Committee
What are their functions
• Manage education programs on drug
utilization
• Establish policies to ensure safe and
effective drug use
• Develop policies promoting cost-
effective drug use
• Provide guidance on drug distribution
and control policies

This Photo by Unknown Author is licensed under CC BY-NC-ND

19
Factors Considered by P&T Committees

Clinical efficacy and effectiveness

Safety: RCT, real-world data
Therapeutic need: first in class, 5 similar agents
Clinical guidelines
Standards of practice
Treatment options
Economics: costs, PMPM costs, QALY, ICER

20
Case Studies
Dose Cost, $ Efficacy Side Effects
Drug A 1 tablet daily 1.25 per tablet 77%-80% Nausea, vomiting
Drug B 1 tablet daily 1.50 per tablet 78%-80% Nausea, headache

Dose Cost, $ Efficacy Side Effects

Drug A 2 tablets daily 0.60 per tablet 77%-80% Nausea, vomiting
Drug B 1 tablet daily 1.50 per tablet 78%-80% Nausea, headache

Dose Cost, $ Efficacy Side Effects Adherence (DBA)

Drug A 1 tablet daily 1.25 per tablet 77%-80% Nausea, vomiting 65%
Drug B 1 tablet daily 1.50 per tablet 78%-80% Nausea, headache 80%

21
 Case Studies
Dose Cost, $ Efficacy Side Effects
Drug A 1 tablet daily 1.25 per tablet 77%-80% Nausea, vomiting, irreversible hepatotoxicity (5%)
Drug B 1 tablet daily 1.50 per tablet 78%-80% Nausea, headache

Dose Cost, $ Efficacy Side Effects Nursing Administration

Drug A 2 injections daily 30.00 per dose 77%-80% Nausea, vomiting 5 minutes
Drug B 2 injections daily 15.00 per dose 78%-80% Nausea, headache IV push over 30 min,
(drug cost) observe for 30 min

Dose Cost, $ Efficacy Side Effects Hospital Length of Stay

Drug A 2 tablets daily 250.00 per tablet 77%-80% Nausea, vomiting 5 days
Drug B 2 injections daily 25.00 per dose 78%-80% Nausea, headache 7 days / 5 days + 2 days
(drug cost) home IV therapy

22
Case Studies
Drug A Drug B
Cost, $ 500,000 per dose 1.50 per dose
Efficacy Effective Effective
PMPM cost, $ 0.0001 0.02

Drug A Drug B
Dose 1 injections daily 2 injections daily
Cost, $ 30.00 per dose 10.00 per dose (drug cost)
Efficacy 80% 60%
Side effects Nausea, vomiting, severe rash Nausea, headache
Nursing administration IV push over 10 minutes IV push over 10 minutes
Laboratory testing Q day (renal) Q 3 days (renal panel, CBC)
ICU time 1 day 1.5 days
Ventilator time 0.5 day 1 day

23
Questions?

24