Health Economics & Outcomes Research (HE&OR) guides stakeholders to make informed decisions regarding patient access to drugs and services. This document highlights specific use cases for healthcare information technology that add value to HE&OR.

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Rising Importance of Health Economics &
Outcomes Research (HE&OR)
October 2019 | Author: Sameer Gokhale
CitiusTech Thought
Leadership

2. 2
Agenda
▪ Introduction
▪ Challenges
▪ Significance of HE&OR
▪ Use Cases

3. 3
Introduction
▪ Recently, healthcare industry is undergoing a paradigm shift
▪ There is an increasing patient needs
▪ Patient satisfaction has become more important to hospitals’ bottom line
▪ Healthcare budget constraints are increasing across developed and developing countries
▪ Payer dilemma is “not everything is affordable for all patients”
▪ HE&OR plays an important role in meeting patient demands, keeping in mind budget
constraints

4. 4
Challenges (1/2)
▪ Pricing concerns and need for evidence of product value have added hurdles to market access
beyond registration
▪ HE&OR provides an efficient way of allocating scarce healthcare resources to ensure optimal
treatment to patients
Therapeutic Value
Safety Efficacy
Budget
Impact
Cost
Effectiveness
Effectiveness
(Real-world)
Market
Access
Economic Value
Registration

5. 5
Challenges (2/2)
▪ Product value is increasingly being questioned by patients, physicians, payers and policy
makers
▪ Pharmaceutical product value is increasingly being questioned by healthcare stakeholders
(patients, physicians, payers and policy makers)
▪ HE&OR conducts studies and develops models to quantify differential value for a pharmaceutical
products
Patients
▪ Will this newer
treatment improve
my survival?
▪ Will this newer
treatment improve
my quality of life?
▪ Why is this treatment
so expensive?
▪ Give me real-life
examples
Physicians
▪ Show us the
performance of the
newer drug as an
improvement in
primary and
secondary outcomes
▪ Is this a cost-effective
option?
▪ Can you show us the
real-world evidence
of the newer
therapy?
Payers
▪ Is this drug therapy
needed?
▪ How safe and
efficacious is this
treatment?
▪ Is this a cost-effective
option?
▪ Can you show us the
comparative or
relative
effectiveness?
Policy Makers
▪ Can this new
treatment provide
performance-based
pricing?
▪ Can you produce any
economic analysis?
▪ Does this treatment
qualify for
comparative
effectiveness?
▪ Can you show us real-
world evidence?

6. 6
Significance of HE&OR
▪ HE&OR justifies the differential value of a new product as compared to an existing therapeutic
option(s)
▪ HE&OR conducts qualitative studies and develops economic models to quantify the differential
value for a product
▪ This can help mitigate some doubts from healthcare stakeholders
Value of
Comparator
Improvement
in clinical
outcomes
Healthcare costs
avoidedProductivity
gains
Patient
outcomes
benefits
(e.g., Quality of Life)
Value of
new product
DifferentialValue
Value

7. 7
Usage of HE&OR is Limited to Few Payers and Providers
▪ As Randomized Clinical Trial (RCT) is the gold standard, clinical trials are used extensively in
comparison with a limited usage of HE&OR
▪ In an ideal scenario, HE&OR plan should be executed parallel to Clinical Development Plan as
shown below:

8. 8
Healthcare Technology can Accelerate HE&OR Value
From HE&OR perspective, pertinent hypotheses can be tested, or research questions can be
answered using various instruments / databases.
Instruments
Solutions
Treatment Patterns
& Trends
Clinical Decision
Support System
Medication
Adherence
Comparative
Effectiveness
Risk Predictor
Models
Disease Burden /
Cost of Illness
CEA Registry, Tufts Medical Center Cost-Effectiveness Analysis registry; CPRD, Clinical Practice Research Datalink; HCUP, Healthcare Cost
and Utilization Project; HRQoL, health related quality of life; NAMCS, National Ambulatory Medical Care Survey; NHANES, National
Health and Nutrition Examination Survey; NHCS, National Hospital Care Survey; PRO, patient reported outcome; QoL, quality of life

9. 9
Use Case 1 – Risk Predictors
Risk prognostic algorithm to predict an individual’s risk of relapse after first-line treatment for
ovarian cancer.
Actionable Business Outcomes
▪ Improved Progression Free Survival (PFS)
▪ Improved Surveillance / Follow-up
▪ Scientific Publications (Abstract, Poster, Manuscript)
▪ Clinical Decision Support Solution (CDSS) / Tool for efficient Niraparib Uptake
Niraparib is indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian
tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy

10. 10
Use Case 2 – Risk Predictors
Identify predictors for better outcomes in platinum-resistant ovarian cancer patients using claims
and registry databases.
Actionable Business Outcomes
▪ Improved HRQoL
▪ Improved Health Outcomes
▪ Scientific Publications (Abstract, Poster, and Manuscript)
▪ Clinical Decision Support Solution (CDSS) / Tool for Safe Olaparib Uptake
* Matching to be done on various variables to avoid selection bias; OC, ovarian cancer; Pts, patients; QoL,
quality of life; TRT, treatment; References: 1. Liu et al. 2018; 2. Zhou et al. 2017

11. 11
Use Case 3 – Clinical Decision Support System (CDSS)
CDSS solution will predict best treatment regimen leveraging scientific hypotheses in specific types
of epilepsy patients.
Actionable Business Outcome
▪ Current XYZ™ solution predicts ABC at diagnosis while proposed solution in CDSS (XYZ
plus) will predict best treatment regimen leveraging scientific hypotheses.

12. 12
Use Case 4 – Comparative Effectiveness
Comparator outcomes profile can be an important construct for defining value proposition for
high-end products.
▪ Construct of comparator outcomes profile helps pharmaceutical company assess Clinical and
Economic value
▪ Applications in HE&OR set-up:
• Indirect comparisons
• Study end-point discussion
• Post-launch observational studies
• Local HE&OR strategy for country organizations
Actionable Business Outcome
▪ Comparator outcomes profile helps client to define value proposition for a product based on
concepts of health economics and outcomes research.
Select Comparators
Select comparators
suitable for
conducting research
Key Secondary
Outcomes
Select outcomes &
measuring tools
from published and
unpublished sources
Reach & Acceptability
of PRO Tools
Generate graphs and
tables to assess
acceptability and
reach of tools
Results and
Conclusions
Present collated
summary statistics
to make informed
decision

13. 13
References
▪ Coulter A, Ham C, editors. The global challenge of health care rationing. Buckingham: Open University Press; 2000
▪ Hall, Melvin F 2008. “Looking to Improve Financial Results? Start by Listening to Patients” Healthcare Financial Management,
October 2008
▪ Anke-Peggy Holtorf 2012. Current and Future Use of HEOR Data in Healthcare Decision-Making in the United States and in
Emerging Markets. Am Health Drug Benefits. 2012 Nov-Dec; 5(7): 428–438
▪ Lakdawalla DN et al. 2018. Defining Elements of Value in Health Care—A Health Economics Approach: An ISPOR Special Task
Force Report [3]. VALUE IN HEALTH 21 (2018) 131 – 139. https://www.valueinhealthjournal.com/article/S1098-3015(17)33892-
5/pdf
▪ World Economic Forum Insight Report 2017. Value in Healthcare; Laying the Foundation for Health System Transformation.
http://www3.weforum.org/docs/WEF_Insight_Report_Value_Healthcare_Laying_Foundation.pdf
▪ Akobeng AK 2005. Understanding randomised controlled trials. Arch Dis Child 2005;90:840–844. doi: 10.1136/adc.2004.058222
▪ Manns BJ 2009. The role of health economics within clinical research. Methods Mol Biol. 2009;473:235-50. doi: 10.1007/978-1-
59745-385-1_14
▪ Rizzuto I et al. Int J Gynecol Cancer 2015;25: 416Y422
▪ ZEJULA USFDA Label 2017; Highlights of Prescribing Information
▪ SmPC – Zejula from EMA; https://www.ema.europa.eu/en/documents/product-information/zejula-epar-product-
information_en.pdf
▪ Liu Y et al. (2018); Risk of selected gastrointestinal toxicities associated with poly (ADP-ribose) polymerase (PARP) inhibitors in the
treatment of ovarian cancer: a meta-analysis of published trials. Drug Design, Development and Therapy 2018:12 3013–3019
▪ Zhou JX et al. (2017); Risk of severe hematologic toxicities in cancer patients treated with PARP inhibitors: a meta-analysis of
randomized controlled trials. Drug Design, Development and Therapy 2017:11 3009–3017
▪ Gokhale SB. 2015. Comparator outcomes profile: need for an informed assessment of secondary outcomes for conducting post-
launch observational studies in the HE&OR setting. JHPOR, 2015, 2. DOI:10.7365 / JHPOR.2015.2.2

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14
Thank You
Author:
Sameer Gokhale
Sr. Healthcare Consultant
thoughtleaders@citiustech.com